Long Term, Follow-on Study of Lomitapide in Patients With Homozygous Familial Hypercholesterolemia

• Sponsor: Aegerion Pharmaceuticals, Inc. (Industry)
• Overall Status: Completed
• CT.gov ID: NCT00943306
• Collaborator: (none)
• Enrollment: 19
• Locations: 10
• Arm: 1
• Actual Duration (Months): 61.1
• Patients Per Site: 1.9
• Patients Per Site Per Month: 0

Study Details

Study Description

Brief Summary

This was a long term follow on study to assess the continued long term safety and efficacy of lomitapide in patients with homozygous familial hypercholesterolemia.

Condition or Disease	Intervention/Treatment	Phase
Familial Hypercholesterolemia	Drug: lomitapide	Phase 3

Detailed Description

This was a phase III open label clinical trial to evaluate the long-term efficacy and safety of lomitapide at the maximum tolerated dose (for each patient) established during the clinical trial 733-005/UP1002. Subjects completing the 78-week treatment period in study 733-005/UP1002 who have not met any of the stopping criteria will be eligible to participate in 733-012. The treatment period continued until a decision was made by the local competent authority regarding marketing authorization. Lomitapide will be given orally once daily. Patient specific doses will be carried forward from 733-005/UP1002, but will not exceed the maximum tolerated dose the patient received during 733-005 /UP1002. The maximum dose for any patient was 80 mg/day. There was no reference therapy in this trial. The effects of the study drug were compared to baseline data (from 733-005/UP1002). Concomitant lipid-lowering therapy including plasmapheresis or LDL apheresis is permitted.

Note that hereafter, Week 0 in Study 733-005/UP1002 will be referred to as Baseline and Week 48 in Study AEGR-733-012 will be referred to as Week 126 (of overall treatment).

Study Design

• Study Type: Interventional
• Actual Enrollment: 19 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Phase III, Long Term, Open Label, Follow on Study of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor 'Lomitapide' (LOMITAPIDE) in Patients With Homozygous Familial Hypercholesterolemia
• Actual Study Start Date: Oct 29, 2009
• Actual Primary Completion Date: Sep 17, 2012
• Actual Study Completion Date: Dec 1, 2014

Arms and Interventions

Arm	Intervention/Treatment
Experimental: lomitapide Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.	Drug: lomitapide; 5-60 mg po every day; Other Names: AEGR-733 BMS-201038

Outcome Measures

Primary Outcome Measures

1. Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) [Baseline and Week 126]

   Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012).

Secondary Outcome Measures

1. Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) [Baseline and Week 174]

   Percent change in Low Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).

2. Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) [Baseline and Week 222]

   Percent change in Low Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).

3. Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) [Baseline and Week 246]

   Percent change in Low Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).

4. Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) [Baseline and Week 270]

   Percent change in Low Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).

5. Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) [Baseline and Week 294]

   Percent change in Low Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).

6. Percent Change in Total Cholesterol [Baseline and Week 126]

   Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012).

7. Percent Change in Total Cholesterol [Baseline and Week 174]

   Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).

8. Percent Change in Total Cholesterol [Baseline and Week 222]

   Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).

9. Percent Change in Total Cholesterol [Baseline and Week 246]

   Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).

10. Percent Change in Total Cholesterol [Baseline and Week 270]

    Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).

11. Percent Change in Total Cholesterol [Baseline and Week 294]

    Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).

12. Percent Change in Apolipoprotein B (Apo B) [Baseline and Week 126]

    Percent change in Apolipoprotein B (Apo B) from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012).

13. Percent Change in Apolipoprotein B (Apo B) [Baseline and Week 174]

    Percent change in Apolipoprotein B (Apo B) from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).

14. Percent Change in Apolipoprotein B (Apo B) [Baseline and Week 222]

    Percent change in Apolipoprotein B (Apo B) from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).

15. Percent Change in Apolipoprotein B (Apo B) [Baseline and Week 246]

    Percent change in Apolipoprotein B (Apo B) from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).

16. Percent Change in Apolipoprotein B (Apo B) [Baseline and Week 270]

    Percent change in Apolipoprotein B (Apo B) from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).

17. Percent Change in Apolipoprotein B (Apo B) [Baseline and Week 294]

    Percent change in Apolipoprotein B (Apo B) from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).

18. Percent Change in Triglycerides [Baseline and Week 126]

    Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012).

19. Percent Change in Triglycerides [Baseline and Week 174]

    Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).

20. Percent Change in Triglycerides [Baseline and Week 222]

    Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).

21. Percent Change in Triglycerides [Baseline and Week 246]

    Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).

22. Percent Change in Triglycerides [Baseline and Week 270]

    Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).

23. Percent Change in Triglycerides [Baseline and Week 294]

    Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).

24. Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) [Baseline and Week 126]

    Percent change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012).

25. Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) [Baseline and Week 174]

    Percent change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).

26. Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) [Baseline and Week 222]

    Percent change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).

27. Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) [Baseline and Week 246]

    Percent change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).

28. Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) [Baseline and Week 270]

    Percent change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).

29. Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) [Baseline and Week 294]

    Percent change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).

30. Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) [Baseline and Week 126]

    Percent change in Very Low Density Lipoprotein Cholesterol (VLDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012).

31. Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) [Baseline and Week 174]

    Percent change in Very Low Density Lipoprotein Cholesterol (VLDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).

32. Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) [Baseline and Week 222]

    Percent change in Very Low Density Lipoprotein Cholesterol (VLDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).

33. Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) [Baseline and Week 246]

    Percent change in Very Low Density Lipoprotein Cholesterol (VLDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).

34. Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) [Baseline and Week 270]

    Percent change in Very Low Density Lipoprotein Cholesterol (VLDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).

35. Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) [Baseline and Week 294]

    Percent change in Very Low Density Lipoprotein Cholesterol (VLDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).

36. Percent Change in Lp(a) [Baseline and Week 126]

    Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012).

37. Percent Change in Lp(a) [Baseline and Week 174]

    Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).

38. Percent Change in Lp(a) [Baseline and Week 222]

    Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).

39. Percent Change in Lp(a) [Baseline and Week 246]

    Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).

40. Percent Change in Lp(a) [Baseline and Week 270]

    Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).

41. Percent Change in Lp(a) [Baseline and Week 294]

    Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).

42. Percent Change in High Density Lipoprotein Cholesterol (HDL-C) [Baseline and Week 126]

    Percent change in High Density Lipoprotein Cholesterol (HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012).

43. Percent Change in High Density Lipoprotein Cholesterol (HDL-C) [Baseline and Week 174]

    Percent change in High Density Lipoprotein Cholesterol (HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).

44. Percent Change in High Density Lipoprotein Cholesterol (HDL-C) [Baseline and Week 222]

    Percent change in High Density Lipoprotein Cholesterol (HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).

45. Percent Change in High Density Lipoprotein Cholesterol (HDL-C) [Baseline and Week 246]

    Percent change in High Density Lipoprotein Cholesterol (HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).

46. Percent Change in High Density Lipoprotein Cholesterol (HDL-C) [Baseline and Week 270]

    Percent change in High Density Lipoprotein Cholesterol (HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).

47. Percent Change in High Density Lipoprotein Cholesterol (HDL-C) [Baseline and Week 294]

    Percent change in High Density Lipoprotein Cholesterol (HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).

48. Percent Change in Apolipoprotein AI (Apo AI) [Baseline and Week 126]

    Percent change in Apolipoprotein AI (Apo AI) from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012).

49. Percent Change in Apolipoprotein AI (Apo AI) [Baseline and Week 174]

    Percent change in Apolipoprotein AI (Apo AI) from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).

50. Percent Change in Apolipoprotein AI (Apo AI) [Baseline and Week 222]

    Percent change in Apolipoprotein AI (Apo AI) from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).

51. Percent Change in Apolipoprotein AI (Apo AI) [Baseline and Week 246]

    Percent change in Apolipoprotein AI (Apo AI) from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).

52. Percent Change in Apolipoprotein AI (Apo AI) [Baseline and Week 270]

    Percent change in Apolipoprotein AI (Apo AI) from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).

53. Percent Change in Apolipoprotein AI (Apo AI) [Baseline and Week 294]

    Percent change in Apolipoprotein AI (Apo AI) from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

1. Completed UP1002 or 733-005.

2. Willing and able to provide consent and comply with the requirements of the study protocol.

Exclusion Criteria:

1. Met any of the stopping rules for study discontinuation at the final visit of study UP1002 or 733-005.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Cedars-Sinai Medical Center	Los Angeles	California	United States	90048
2	University of Pennsylvania	Philadelphia	Pennsylvania	United States	19104
3	Robarts Research Institute	London	Ontario	Canada	N6A 5K8
4	Lipid Clinic and University of Montreal Community Genomic Medicine Center	Chicoutimi	Quebec	Canada	G7H 5H6
5	Medicina Interna Universitaria	Ferrara	Sicily	Italy
6	Dipartimento di Medicina Clinica e Delle Patologie Emergenti	Palermo	Sicily	Italy
7	Centro Universitario Dislipidemie	Milano		Italy
8	DAI Ematologia, Oncologia, Anatomia Patologica e Medicina	Roma		Italy
9	Cardiology Research	Bloemfontein		South Africa	9300
10	University of Capetown	Cape town		South Africa	7925

Sponsors and Collaborators

• Aegerion Pharmaceuticals, Inc.

Investigators

• Principal Investigator: Marina Cuchel, MD, PhD, University of Pennsylvania
• Study Chair: Mark Sumeray, MD, Aegerion Pharmaceuticals, Inc.

Study Documents (Full-Text)

None provided.

More Information

Publications

• Cuchel M, Bloedon LT, Szapary PO, Kolansky DM, Wolfe ML, Sarkis A, Millar JS, Ikewaki K, Siegelman ES, Gregg RE, Rader DJ. Inhibition of microsomal triglyceride transfer protein in familial hypercholesterolemia. N Engl J Med. 2007 Jan 11;356(2):148-56.
• Cuchel M, Meagher E, Marais AD, et.al. Abstract 1077: A phase III study of microsomal triglyceride transfer protein inhibitor lomitapide (AEGR-733) in patients with homozygous familial hypercholesterolemia: interim results at 6 months. Circulation, Nov 2009; 120: S441

• Responsible Party: Aegerion Pharmaceuticals, Inc.
• ClinicalTrials.gov Identifier: NCT00943306
• Other Study ID Numbers: AEGR-733-012
• First Posted: Jul 22, 2009
• Last Update Posted: Jun 13, 2018
• Last Verified: May 1, 2018

• Keywords provided by Aegerion Pharmaceuticals, Inc.: Homozygous; Familial; Hypercholesterolemia; MTP; Apheresis; lomitapide; Homozygous Familial Hypercholesterolemia (HoFH)
• Additional relevant MeSH terms: Hyperlipoproteinemia Type II; Hypercholesterolemia

Study Results

Participant Flow

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Period Title: Overall Study
STARTED	19
COMPLETED	16
NOT COMPLETED	3

Baseline Characteristics

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Overall Participants	19
Age (Count of Participants)
<=18 years	0 0%
Between 18 and 65 years	19 100%
>=65 years	0 0%
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	30.4 (11.74)
Sex: Female, Male (Count of Participants)
Female	9 47.4%
Male	10 52.6%
Race (NIH/OMB) (Count of Participants)
American Indian or Alaska Native	0 0%
Asian	1 5.3%
Native Hawaiian or Other Pacific Islander	0 0%
Black or African American	0 0%
White	17 89.5%
More than one race	0 0%
Unknown or Not Reported	1 5.3%
Region of Enrollment (participants) [Number]
Canada	2 10.5%
United States	3 15.8%
South Africa	9 47.4%
Italy	5 26.3%

Outcome Measures

1. Primary Outcome

Title	Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)
Description	Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012).
Time Frame	Baseline and Week 126

Outcome Measure Data

Analysis Population Description

Week 126 Completers Population

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	17
Mean (Standard Deviation) [Percent Change]	-45.5 (31.35)

2. Secondary Outcome

Title	Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)
Description	Percent change in Low Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).
Time Frame	Baseline and Week 174

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	16
Mean (Standard Deviation) [Percent Change]	-51.0 (16.03)

3. Secondary Outcome

Title	Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)
Description	Percent change in Low Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).
Time Frame	Baseline and Week 222

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	15
Mean (Standard Deviation) [Percent Change]	-58.5 (24.25)

4. Secondary Outcome

Title	Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)
Description	Percent change in Low Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).
Time Frame	Baseline and Week 246

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	14
Mean (Standard Deviation) [Percent Change]	-60.1 (18.51)

5. Secondary Outcome

Title	Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)
Description	Percent change in Low Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).
Time Frame	Baseline and Week 270

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	9
Mean (Standard Deviation) [Percent Change]	-74.0 (19.10)

6. Secondary Outcome

Title	Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)
Description	Percent change in Low Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).
Time Frame	Baseline and Week 294

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	3
Mean (Standard Deviation) [Percent Change]	-51.1 (10.11)

7. Secondary Outcome

Title	Percent Change in Total Cholesterol
Description	Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012).
Time Frame	Baseline and Week 126

Outcome Measure Data

Analysis Population Description

Week 126 Completers Population

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	17
Mean (Standard Deviation) [Percent Change]	-43.2 (25.35)

8. Secondary Outcome

Title	Percent Change in Total Cholesterol
Description	Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).
Time Frame	Baseline and Week 174

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	16
Mean (Standard Deviation) [Percent Change]	-46.9 (15.21)

9. Secondary Outcome

Title	Percent Change in Total Cholesterol
Description	Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).
Time Frame	Baseline and Week 222

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	15
Mean (Standard Deviation) [Percent Change]	-51.0 (21.34)

10. Secondary Outcome

Title	Percent Change in Total Cholesterol
Description	Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).
Time Frame	Baseline and Week 246

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	14
Mean (Standard Deviation) [Percent Change]	-54.1 (16.88)

11. Secondary Outcome

Title	Percent Change in Total Cholesterol
Description	Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).
Time Frame	Baseline and Week 270

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	9
Mean (Standard Deviation) [Percent Change]	-65.2 (15.97)

12. Secondary Outcome

Title	Percent Change in Total Cholesterol
Description	Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).
Time Frame	Baseline and Week 294

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	3
Mean (Standard Deviation) [Percent Change]	-43.9 (5.01)

13. Secondary Outcome

Title	Percent Change in Apolipoprotein B (Apo B)
Description	Percent change in Apolipoprotein B (Apo B) from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012).
Time Frame	Baseline and Week 126

Outcome Measure Data

Analysis Population Description

Week 126 Completers Population

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	17
Mean (Standard Deviation) [Percent Change]	-53.6 (23.74)

14. Secondary Outcome

Title	Percent Change in Apolipoprotein B (Apo B)
Description	Percent change in Apolipoprotein B (Apo B) from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).
Time Frame	Baseline and Week 174

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	16
Mean (Standard Deviation) [Percent Change]	-59.4 (12.60)

15. Secondary Outcome

Title	Percent Change in Apolipoprotein B (Apo B)
Description	Percent change in Apolipoprotein B (Apo B) from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).
Time Frame	Baseline and Week 222

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	15
Mean (Standard Deviation) [Percent Change]	-65.1 (20.72)

16. Secondary Outcome

Title	Percent Change in Apolipoprotein B (Apo B)
Description	Percent change in Apolipoprotein B (Apo B) from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).
Time Frame	Baseline and Week 246

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	14
Mean (Standard Deviation) [Percent Change]	-65.9 (15.76)

17. Secondary Outcome

Title	Percent Change in Apolipoprotein B (Apo B)
Description	Percent change in Apolipoprotein B (Apo B) from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).
Time Frame	Baseline and Week 270

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	9
Mean (Standard Deviation) [Percent Change]	-76.7 (16.09)

18. Secondary Outcome

Title	Percent Change in Apolipoprotein B (Apo B)
Description	Percent change in Apolipoprotein B (Apo B) from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).
Time Frame	Baseline and Week 294

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	3
Mean (Standard Deviation) [Percent Change]	-60.9 (12.17)

19. Secondary Outcome

Title	Percent Change in Triglycerides
Description	Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012).
Time Frame	Baseline and Week 126

Outcome Measure Data

Analysis Population Description

Week 126 Completers Population

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	17
Mean (Standard Deviation) [Percent Change]	-37.5 (42.52)

20. Secondary Outcome

Title	Percent Change in Triglycerides
Description	Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).
Time Frame	Baseline and Week 174

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	16
Mean (Standard Deviation) [Percent Change]	-31.7 (37.09)

21. Secondary Outcome

Title	Percent Change in Triglycerides
Description	Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).
Time Frame	Baseline and Week 222

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	15
Mean (Standard Deviation) [Percent Change]	-27.6 (48.88)

22. Secondary Outcome

Title	Percent Change in Triglycerides
Description	Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).
Time Frame	Baseline and Week 246

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	14
Mean (Standard Deviation) [Percent Change]	-41.9 (33.16)

23. Secondary Outcome

Title	Percent Change in Triglycerides
Description	Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).
Time Frame	Baseline and Week 270

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	9
Mean (Standard Deviation) [Percent Change]	-48.5 (34.24)

24. Secondary Outcome

Title	Percent Change in Triglycerides
Description	Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).
Time Frame	Baseline and Week 294

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	3
Mean (Standard Deviation) [Percent Change]	29.2 (58.74)

25. Secondary Outcome

Title	Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)
Description	Percent change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012).
Time Frame	Baseline and Week 126

Outcome Measure Data

Analysis Population Description

Week 126 Completers Population

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	17
Mean (Standard Deviation) [Percent Change]	-47.1 (27.83)

26. Secondary Outcome

Title	Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)
Description	Percent change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).
Time Frame	Baseline and Week 174

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	16
Mean (Standard Deviation) [Percent Change]	-53.5 (16.42)

27. Secondary Outcome

Title	Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)
Description	Percent change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).
Time Frame	Baseline and Week 222

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	15
Mean (Standard Deviation) [Percent Change]	-57.0 (24.07)

28. Secondary Outcome

Title	Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)
Description	Percent change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).
Time Frame	Baseline and Week 246

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	14
Mean (Standard Deviation) [Percent Change]	-58.8 (17.73)

29. Secondary Outcome

Title	Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)
Description	Percent change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).
Time Frame	Baseline and Week 270

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	9
Mean (Standard Deviation) [Percent Change]	-71.5 (17.65)

30. Secondary Outcome

Title	Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)
Description	Percent change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).
Time Frame	Baseline and Week 294

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	3
Mean (Standard Deviation) [Percent Change]	-46.6 (5.19)

31. Secondary Outcome

Title	Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)
Description	Percent change in Very Low Density Lipoprotein Cholesterol (VLDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012).
Time Frame	Baseline and Week 126

Outcome Measure Data

Analysis Population Description

Week 126 Completers Population

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	17
Mean (Standard Deviation) [Percent Change]	-36.8 (43.90)

32. Secondary Outcome

Title	Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)
Description	Percent change in Very Low Density Lipoprotein Cholesterol (VLDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).
Time Frame	Baseline and Week 174

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	16
Mean (Standard Deviation) [Percent Change]	-31.5 (36.32)

33. Secondary Outcome

Title	Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)
Description	Percent change in Very Low Density Lipoprotein Cholesterol (VLDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).
Time Frame	Baseline and Week 222

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	15
Mean (Standard Deviation) [Percent Change]	-26.3 (49.94)

34. Secondary Outcome

Title	Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)
Description	Percent change in Very Low Density Lipoprotein Cholesterol (VLDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).
Time Frame	Baseline and Week 246

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	14
Mean (Standard Deviation) [Percent Change]	-41.4 (34.20)

35. Secondary Outcome

Title	Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)
Description	Percent change in Very Low Density Lipoprotein Cholesterol (VLDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).
Time Frame	Baseline and Week 270

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	9
Mean (Standard Deviation) [Percent Change]	-48.7 (33.41)

36. Secondary Outcome

Title	Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)
Description	Percent change in Very Low Density Lipoprotein Cholesterol (VLDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).
Time Frame	Baseline and Week 294

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	3
Mean (Standard Deviation) [Percent Change]	30.6 (59.94)

37. Secondary Outcome

Title	Percent Change in Lp(a)
Description	Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012).
Time Frame	Baseline and Week 126

Outcome Measure Data

Analysis Population Description

Week 126 Completers Population

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	16
Mean (Standard Deviation) [Percent Change]	5.5 (43.62)

38. Secondary Outcome

Title	Percent Change in Lp(a)
Description	Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).
Time Frame	Baseline and Week 174

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	15
Mean (Standard Deviation) [Percent Change]	10.2 (60.64)

39. Secondary Outcome

Title	Percent Change in Lp(a)
Description	Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).
Time Frame	Baseline and Week 222

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	14
Mean (Standard Deviation) [Percent Change]	-12.8 (49.00)

40. Secondary Outcome

Title	Percent Change in Lp(a)
Description	Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).
Time Frame	Baseline and Week 246

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	13
Mean (Standard Deviation) [Percent Change]	3.4 (54.22)

41. Secondary Outcome

Title	Percent Change in Lp(a)
Description	Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).
Time Frame	Baseline and Week 270

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	9
Mean (Standard Deviation) [Percent Change]	-6.6 (48.99)

42. Secondary Outcome

Title	Percent Change in Lp(a)
Description	Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).
Time Frame	Baseline and Week 294

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	3
Mean (Standard Deviation) [Percent Change]	-10.4 (35.64)

43. Secondary Outcome

Title	Percent Change in High Density Lipoprotein Cholesterol (HDL-C)
Description	Percent change in High Density Lipoprotein Cholesterol (HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012).
Time Frame	Baseline and Week 126

Outcome Measure Data

Analysis Population Description

Week 126 Completers Population

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	17
Mean (Standard Deviation) [Percent Change]	-8.3 (19.28)

44. Secondary Outcome

Title	Percent Change in High Density Lipoprotein Cholesterol (HDL-C)
Description	Percent change in High Density Lipoprotein Cholesterol (HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).
Time Frame	Baseline and Week 174

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	16
Mean (Standard Deviation) [Percent Change]	3.8 (26.51)

45. Secondary Outcome

Title	Percent Change in High Density Lipoprotein Cholesterol (HDL-C)
Description	Percent change in High Density Lipoprotein Cholesterol (HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).
Time Frame	Baseline and Week 222

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	15
Mean (Standard Deviation) [Percent Change]	-2.7 (21.22)

46. Secondary Outcome

Title	Percent Change in High Density Lipoprotein Cholesterol (HDL-C)
Description	Percent change in High Density Lipoprotein Cholesterol (HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).
Time Frame	Baseline and Week 246

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	14
Mean (Standard Deviation) [Percent Change]	-12.5 (19.17)

47. Secondary Outcome

Title	Percent Change in High Density Lipoprotein Cholesterol (HDL-C)
Description	Percent change in High Density Lipoprotein Cholesterol (HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).
Time Frame	Baseline and Week 270

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	9
Mean (Standard Deviation) [Percent Change]	-10.3 (27.68)

48. Secondary Outcome

Title	Percent Change in High Density Lipoprotein Cholesterol (HDL-C)
Description	Percent change in High Density Lipoprotein Cholesterol (HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).
Time Frame	Baseline and Week 294

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	3
Mean (Standard Deviation) [Percent Change]	-23.5 (2.27)

49. Secondary Outcome

Title	Percent Change in Apolipoprotein AI (Apo AI)
Description	Percent change in Apolipoprotein AI (Apo AI) from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012).
Time Frame	Baseline and Week 126

Outcome Measure Data

Analysis Population Description

Week 126 Completers Population

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	17
Mean (Standard Deviation) [Percent Change]	-14.0 (17.71)

50. Secondary Outcome

Title	Percent Change in Apolipoprotein AI (Apo AI)
Description	Percent change in Apolipoprotein AI (Apo AI) from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).
Time Frame	Baseline and Week 174

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	16
Mean (Standard Deviation) [Percent Change]	-8.2 (20.12)

51. Secondary Outcome

Title	Percent Change in Apolipoprotein AI (Apo AI)
Description	Percent change in Apolipoprotein AI (Apo AI) from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).
Time Frame	Baseline and Week 222

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	15
Mean (Standard Deviation) [Percent Change]	-2.7 (33.30)

52. Secondary Outcome

Title	Percent Change in Apolipoprotein AI (Apo AI)
Description	Percent change in Apolipoprotein AI (Apo AI) from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).
Time Frame	Baseline and Week 246

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	14
Mean (Standard Deviation) [Percent Change]	-16.8 (26.62)

53. Secondary Outcome

Title	Percent Change in Apolipoprotein AI (Apo AI)
Description	Percent change in Apolipoprotein AI (Apo AI) from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).
Time Frame	Baseline and Week 270

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	9
Mean (Standard Deviation) [Percent Change]	-17.8 (20.12)

54. Secondary Outcome

Title	Percent Change in Apolipoprotein AI (Apo AI)
Description	Percent change in Apolipoprotein AI (Apo AI) from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).
Time Frame	Baseline and Week 294

Outcome Measure Data

Analysis Population Description

Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Measure Participants	3
Mean (Standard Deviation) [Percent Change]	-30.5 (22.2)

Adverse Events

Time Frame	Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Arm/Group Title	Lomitapide
Arm/Group Description	Maximum tolerated dose of lomitapide (up to 80mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
All Cause Mortality
	Lomitapide
	Affected / at Risk (%)	# Events
Total	1/19 (5.3%)
Serious Adverse Events
	Lomitapide
	Affected / at Risk (%)	# Events
Total	7/19 (36.8%)
Cardiac disorders
Angina Pectoris	1/19 (5.3%)
Aortic Value Incompetence	1/19 (5.3%)
Coronary Artery Disease	1/19 (5.3%)
Gastrointestinal disorders
Diarrhoea	1/19 (5.3%)
Reflux Oesophagitis	1/19 (5.3%)
General disorders
Sudden Cardiac Death	1/19 (5.3%)
Hepatobiliary disorders
Hepatotoxicity	1/19 (5.3%)
Infections and infestations
Lower Respiratory Tract Infection	1/19 (5.3%)
Injury, poisoning and procedural complications
Subdural Haematoma	1/19 (5.3%)
Investigations
International Normalized Ratio Increased	1/19 (5.3%)
Metabolism and nutrition disorders
Dehydration	1/19 (5.3%)
Musculoskeletal and connective tissue disorders
Rhabdomyolsis	1/19 (5.3%)
Nervous system disorders
Facial Palsy	1/19 (5.3%)
Surgical and medical procedures
Anticoagulant Therapy	1/19 (5.3%)
Transfusion	1/19 (5.3%)
Vascular disorders
Arteriovenous Fistula	1/19 (5.3%)
Hypovolaemic Shock	1/19 (5.3%)
Other (Not Including Serious) Adverse Events
	Lomitapide
	Affected / at Risk (%)	# Events
Total	17/19 (89.5%)
Blood and lymphatic system disorders
Anaemia	2/19 (10.5%)
Iron Deficiency Anaemia	1/19 (5.3%)
Cardiac disorders
Angina Pectoris	4/19 (21.1%)
Aortic Valve Incompetence	1/19 (5.3%)
Chest Pain	1/19 (5.3%)
Coronary Artery Disease	1/19 (5.3%)
Sinus Bradycardia	1/19 (5.3%)
Ear and labyrinth disorders
Tinnitus	1/19 (5.3%)
Vertigo	1/19 (5.3%)
Endocrine disorders
Hypothyroidism	1/19 (5.3%)
Eye disorders
Blepharitis	1/19 (5.3%)
Gastrointestinal disorders
Abdominal Distension	3/19 (15.8%)
Chapped Lips	1/19 (5.3%)
Dental Caries	1/19 (5.3%)
Diarrhoea	8/19 (42.1%)
Dyspepsia	2/19 (10.5%)
Epigastric Discomfort	2/19 (10.5%)
Flatulence	1/19 (5.3%)
Gastrooesophageal Reflux Disease	1/19 (5.3%)
Gingival Bleeding	1/19 (5.3%)
Haemorrhoidal Haemorrhage	1/19 (5.3%)
Hiatus Hernia	1/19 (5.3%)
Intestinal Mass	1/19 (5.3%)
Nausea	6/19 (31.6%)
Reflux Oesophagitis	1/19 (5.3%)
Stomach Discomfort	1/19 (5.3%)
Toothache	1/19 (5.3%)
Vomiting	5/19 (26.3%)
General disorders
Asthenia	1/19 (5.3%)
Chest Pain	1/19 (5.3%)
Fatigue	1/19 (5.3%)
Influenza	1/19 (5.3%)
Oedema Peripheral	2/19 (10.5%)
Pain	1/19 (5.3%)
Pyrexia	1/19 (5.3%)
Vestibulitis	1/19 (5.3%)
Sudden Cardiac Death	1/19 (5.3%)
Hepatobiliary disorders
Hepatotoxicity	2/19 (10.5%)
Immune system disorders
Drug Hypersensitivity	1/19 (5.3%)
Infections and infestations
Bronchitis	3/19 (15.8%)
Gastroenteritis	3/19 (15.8%)
Gastrointestinal Infection	1/19 (5.3%)
Influenza	5/19 (26.3%)
Lower Respiratory Tract Infection	1/19 (5.3%)
Nasopharyngitis	4/19 (21.1%)
Sinusitus	3/19 (15.8%)
Tooth Abscess	2/19 (10.5%)
Upper Respiratory Tract Infection	1/19 (5.3%)
Urinary Tract Infection	2/19 (10.5%)
Viral Infection	1/19 (5.3%)
Injury, poisoning and procedural complications
Fall	1/19 (5.3%)
Joint Injury	1/19 (5.3%)
Post Procedural Diarrhoea	1/19 (5.3%)
Procedural Headache	1/19 (5.3%)
Skeletal Injury	1/19 (5.3%)
Subdural Haematoma	1/19 (5.3%)
Investigations
Alanine Aminotransferase Increased	3/19 (15.8%)
Asparate Aminotransferase Increased	2/19 (10.5%)
Blood creatine phosphokinase Increased	1/19 (5.3%)
Blood Potassium Increased	1/19 (5.3%)
Blood Pressure Increased	1/19 (5.3%)
Carotene Decreased	1/19 (5.3%)
Carotid Bruit	1/19 (5.3%)
International Normalized Ratio Decreased	1/19 (5.3%)
International Normalized Ratio Increased	1/19 (5.3%)
Liver Function Test Abnormal	1/19 (5.3%)
Prothrombin Time Prolonged	1/19 (5.3%)
Transaminases Increased	1/19 (5.3%)
Vitamin K Decreased	1/19 (5.3%)
Weight Decreased	2/19 (10.5%)
White Blood Cell Count Decreased	1/19 (5.3%)
Metabolism and nutrition disorders
Decreased Appetite	1/19 (5.3%)
Dehydration	1/19 (5.3%)
Iron Deficiency	1/19 (5.3%)
Oral Intake Reduced	1/19 (5.3%)
Vitamin E Deficiency	1/19 (5.3%)
Musculoskeletal and connective tissue disorders
Arthralgia	2/19 (10.5%)
Back Pain	3/19 (15.8%)
Musculoskeletal Pain	1/19 (5.3%)
Neck Pain	1/19 (5.3%)
Rhabdomyolysis	1/19 (5.3%)
Tendonitis	2/19 (10.5%)
Nervous system disorders
Dizziness	1/19 (5.3%)
Facial Palsy	1/19 (5.3%)
Headache	6/19 (31.6%)
Hypoaesthesia	2/19 (10.5%)
Migraine	1/19 (5.3%)
Paraesthesia	2/19 (10.5%)
Sensory Disturbance	1/19 (5.3%)
Syncope	1/19 (5.3%)
Psychiatric disorders
Anxiety	2/19 (10.5%)
Depression	2/19 (10.5%)
Stress	1/19 (5.3%)
Renal and urinary disorders
Nephrolithiasis	1/19 (5.3%)
Reproductive system and breast disorders
Dysmenorrhoea	1/19 (5.3%)
Respiratory, thoracic and mediastinal disorders
Apnoea	1/19 (5.3%)
Cough	2/19 (10.5%)
Dyspnoea	1/19 (5.3%)
Dyspnoea Exertional	1/19 (5.3%)
Epistaxis	1/19 (5.3%)
Painful Respiration	1/19 (5.3%)
Pharyngolaryngeal Pain	1/19 (5.3%)
Sinus Congestion	1/19 (5.3%)
Skin and subcutaneous tissue disorders
Alopecia	1/19 (5.3%)
Dry Skin	1/19 (5.3%)
Hair growth abnormal	1/19 (5.3%)
Scar	1/19 (5.3%)
Surgical and medical procedures
Anticoagulant Therapy	1/19 (5.3%)
Transfusion	1/19 (5.3%)
Vascular disorders
Arterial Stenosis	1/19 (5.3%)
Arteriovenous Fistula	1/19 (5.3%)
Haematoma	1/19 (5.3%)
Hypertension	1/19 (5.3%)
Hypotension	1/19 (5.3%)
Hypovolaemic Shock	1/19 (5.3%)

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

The CTAs generally envision a multisite publication, with the PI's right to publish individually if the pooled publication does not occur within 12 months of study completion. Sponsor has a 45 to 60 day review/approval period to request deletion of confidential information or to request limited deferral to protect its proprietary technology. In one case, the publication provision is more general, specifying that the Sponsor and clinical site will agree on the manner/terms of publication.