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LOWER: Lomitapide Observational Worldwide Evaluation Registry

Sponsor
Amryt Pharma (Industry)
Overall Status
Recruiting
CT.gov ID
NCT02135705
Collaborator
(none)
300
Enrollment
47
Locations
173.5
Anticipated Duration (Months)
6.4
Patients Per Site
0
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This global product exposure registry is a multicentre, long-term, prospective, observational cohort study (exposure registry), designed to evaluate the long term safety and effectiveness of lomitapide.

Condition or Disease Intervention/Treatment Phase

Detailed Description

To evaluate the occurrence of adverse events of special interest, long term effectiveness of lomitapide, and to evaluate whether prescribers of lomitapide are following screening and monitoring recommendations as specified in product labeling.

Study Design

Study Type:
Observational [Patient Registry]
Anticipated Enrollment :
300 participants
Observational Model:
Cohort
Time Perspective:
Prospective
Official Title:
LOWER: Lomitapide Observational Worldwide Evaluation Registry
Actual Study Start Date :
Mar 18, 2014
Anticipated Primary Completion Date :
Mar 1, 2028
Anticipated Study Completion Date :
Sep 1, 2028

Arms and Interventions

Arm Intervention/Treatment
Lomitapide

Lomitapide as prescribed by Physician.

Drug: Lomitapide
As prescribed by Physician.
Other Names:
  • Juxtapid
  • Lojuxta

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Hepatic Abnormalities [patients will be followed for 10 years]

      to evaluate the occurrence of hepatic abnormalities

    Secondary Outcome Measures

    1. Gastrointestinal (GI) Events [patients will be followed for 10 years]

      to evaluate the occurrence of GI events

    2. Tumors [patients will be followed for 10 years]

      to evaluate any occurrence of tumors (small bowel, hepatic, colorectal or pancreatic)

    3. Events associated with coagulopathy [patients will be followed for 10 years]

      to evaluate the occurrence of events associated with coagulopathy (abnormal bleeding, cerebral haemorrhage or GI bleeding)

    4. Major Adverse Cardiovascular Events (MACE) events [patients will be followed for 10 years]

      to evaluate the occurrence of MACE events

    5. Death, including cause of death [patients will be followed for 10 years]

      to evaluate the occurrence and cause of death

    6. Pregnancy [patients will be followed for 10 years]

      to evaluate the occurrence and outcomes of pregnancy in females of reproductive potential treated with lomitapide. Patients who become pregnant will be offered enrolment into a separate Pregnancy Exposure Registry (PER).

    7. Serum lipid levels [patients will be followed for 10 years]

      to evaluate the long-term effectiveness of lomitapide in maintaining control of serum lipid levels in a clinical practice setting.

    8. Prescriber behavior [patients will be followed for 10 years]

      to evaluate whether prescribers of lomitapide enrolled at registry sites are following the screening and monitoring recommendations as specified in the PI and the prescriber educational materials aimed at risk minimization.

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:
    • Adult patients (age ≥18 years) who meet one of the following two criteria:

    Initiating treatment with lomitapide at the time of registry enrolment, or initiated treatment with lomitapide within 36 months prior to enrolment into the registry and after lomitapide commercial availability in the country.

    • Patients who have the ability to understand the requirements of the study and provide written informed consent to comply with the study data collection procedures.
    Exclusion Criteria:

    • Patients who are receiving lomitapide in clinical trials or through compassionate use where patients are followed under a separate protocol

    • Patients receiving an investigational agent, defined as any drug or biologic agent other than lomitapide that has not received MA in the country of participation.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Brookwood Baptist Medical Center Birmingham Alabama United States 35209
    2 Springhill Physician Practices Mobile Alabama United States 36608
    3 Northern Arizona Healthcare Cottonwood Arizona United States 86326
    4 One Medical Gilbert Arizona United States 85296
    5 Scottsdale Family Health Phoenix Arizona United States 85018
    6 Pima Heart Tucson Arizona United States 85718
    7 Arkansas Heart Center Fort Smith Arkansas United States 72901
    8 Cedars-Sinai Medical Center Los Angeles California United States 90048
    9 University of Colorado Health Aurora Colorado United States 80045
    10 Alfieri Cardiology Newark Delaware United States 19713
    11 Preventative Cardiology, Inc. Boca Raton Florida United States 33434
    12 Florida Lipid Institute Winter Park Florida United States 32792-2223
    13 Piedmont Heart Institute Atlanta Georgia United States 30309
    14 Atlanta Heart Specialists Cumming Georgia United States 30041
    15 Comprehensive Cardiovascular Care Gurnee Illinois United States 60031
    16 Community Health Network Indianapolis Indiana United States 46250
    17 St. Vincent Medical Group, Inc. Indianapolis Indiana United States 46290
    18 Henry County Medical Center New Castle Kentucky United States 40050
    19 Cardiovascular Institute of the South Houma Louisiana United States 70360
    20 Get Well Immediate Care Towson Maryland United States 21204
    21 University of Michigan Health System Ann Arbor Michigan United States 48109
    22 Complete Family Care Cholesterol Treatment Center Sterling Heights Michigan United States 48314
    23 Minneapolis Heart Institute Foundation Minneapolis Minnesota United States 55407
    24 HealthEast Care System Saint Paul Minnesota United States 55102
    25 Stern Cardiovascular Foundation Southaven Mississippi United States 38671
    26 Methodist Physicians Clinic Omaha Nebraska United States 68114
    27 Hackensack Meridian Health Edison New Jersey United States 08817
    28 Avinash C. Gupta, MD, PC Lakewood New Jersey United States 08701
    29 201 Route 17 North Rutherford New Jersey United States 07070
    30 Valley Medical Group Wyckoff New Jersey United States 07481
    31 Murray Hill Medical Group New York New York United States 10016
    32 NY Heart Center Syracuse New York United States 13210
    33 Cardiology Specialists Of Carolina Charlotte North Carolina United States 28211
    34 The Heart Care Group, PC Allentown Pennsylvania United States 18103
    35 St. Luke's University Health Network - Pennsylvania Bethlehem Pennsylvania United States 18015
    36 Lehigh Valley Hospital And Health Network East Stroudsburg Pennsylvania United States 18301
    37 Cardiology Consultants of Philadelphia Philadelphia Pennsylvania United States 19107
    38 Thomas Jefferson University Health System Philadelphia Pennsylvania United States 19148
    39 Palmetto Health Columbia South Carolina United States 29203
    40 317 Saint Francis Dr. Greenville South Carolina United States 29601
    41 Cardiovascular Specialists of Texas Austin Texas United States 78758
    42 Baylor Scott & White Health Dallas Texas United States 75226
    43 The University of Vermont Health Network Burlington Vermont United States 05401-1473
    44 West Virginia University Medicine Morgantown West Virginia United States 26506
    45 Ascension Milwaukee Wisconsin United States 53211
    46 Medical College of Wisconsin Milwaukee Wisconsin United States 53226
    47 Aspirus Research Institute Wausau Wisconsin United States 54401

    Sponsors and Collaborators

    • Amryt Pharma

    Investigators

    • Study Director: Sallyann O'Brien, Amryt Pharmaceuticals

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Amryt Pharma
    ClinicalTrials.gov Identifier:
    NCT02135705
    Other Study ID Numbers:
    • AEGR-733-025
    First Posted:
    May 12, 2014
    Last Update Posted:
    Dec 4, 2020
    Last Verified:
    Dec 1, 2020
    Additional relevant MeSH terms:
    Hyperlipoproteinemia Type II
    Hypercholesterolemia

    Study Results

    No Results Posted as of Dec 4, 2020