Long Term Follow-up Study on Safety and Maintenance of Efficacy of ATX-101

• Sponsor: Kythera Biopharmaceuticals (Industry)
• Overall Status: Completed
• CT.gov ID: NCT02159729
• Collaborator: (none)
• Enrollment: 205
• Locations: 20
• Arms: 4
• Duration (Months): 81.9
• Patients Per Site: 10.3
• Patients Per Site Per Month: 0.1

Study Details

Study Description

Brief Summary

This was a long-term follow-up study of participants who completed Kythera-sponsored trials of ATX-101 (06-03, 07-07, 09-15)

Condition or Disease	Intervention/Treatment	Phase
Submental Fat; Healthy	Drug: Placebo; Drug: ATX-101 (1 mg/cm^2); Drug: ATX-101 (2 mg/cm^2); Drug: ATX-101 (4 mg/cm^2)	Phase 2

Detailed Description

No study medication was administered in this study.

Study Design

• Study Type: Interventional
• Actual Enrollment: 205 participants
• Allocation: Non-Randomized
• Intervention Model: Parallel Assignment
• Masking: Double (Participant, Investigator)
• Primary Purpose: Other
• Official Title: Long-term Follow-up Study of Subjects Who Completed Kythera-sponsored Trials of ATX-101 (Sodium Deoxycholate Injection) for the Reduction of Localized Subcutaneous Fat in the Submental Area
• Study Start Date: Feb 1, 2009
• Actual Primary Completion Date: Dec 1, 2015
• Actual Study Completion Date: Dec 1, 2015

Arms and Interventions

Arm	Intervention/Treatment
Placebo Comparator: Placebo Participants treated with placebo in previous ATX-101 studies	Drug: Placebo; Clinical evaluation, patient reported outcome questionnaires, photographs, caliper measurements and recording adverse events
Experimental: ATX-101 (1 mg/cm^2) Participants treated with ATX-101 (1 mg/cm^2) in previous phase 2 studies	Drug: ATX-101 (1 mg/cm^2); Clinical evaluation, patient reported outcome questionnaires, photographs, caliper measurements and recording adverse events
Experimental: ATX-101 (2 mg/cm^2) Participants treated with ATX-101 (2 mg/cm^2) in previous phase 2 studies	Drug: ATX-101 (2 mg/cm^2); Clinical evaluation, patient reported outcome questionnaires, photographs, caliper measurements and recording adverse events
Experimental: ATX-101 (4 mg/cm^2) Participants treated with ATX-101 (4 mg/cm^2) in previous phase 2 studies	Drug: ATX-101 (4 mg/cm^2); Clinical evaluation, patient reported outcome questionnaires, photographs, caliper measurements and recording adverse events

Outcome Measures

Primary Outcome Measures

1. Percentage of Participants Maintaining CR-SMFRS 1-Grade Response During 5 Years of Follow up, i.e. % of Participants Who Were CR-SMFRS 1-Grade Responders at Both Long-term LTFU Baseline and at Subsequent LTFU Visits [Up to 60 months from long-term follow-up (LTFU) baseline (the last visit in the previous study)]

   The investigator evaluated the participant's chin and neck area using the Clinician-Reported Submental Fat Rating Scale (a 5-point scale) where: 0=absent submental convexity (best) to 4= extreme submental convexity (worst). Non-responders at LTFU baseline in each treatment group (including placebo) were not included in the analysis.

2. Percentage of Participants Maintaining PR-SMFRS 1-Grade Response During 5 Years of Follow up, i.e. % of Participants Who Were PR-SMFRS 1-Grade Responders at Both Long-term LTFU Baseline and at Subsequent LTFU Visits [Up to 60 months from long-term follow-up (LTFU) baseline (the last visit in the previous study)]

   The participant evaluated their chin and neck area using the Patient-Reported Submental Fat Rating Scale (a 5-point scale) where: 0=no chin fat at all (best) to 4= a very large amount of chin fat (worst). Non-responders at LTFU baseline in each treatment group (including placebo) were not included in the analysis.

3. Percentage of Participants Maintaining Composite SMFRS 1-Grade Response During 5 Years of Follow up, i.e. % of Participants Who Were CR-SMFRS and PR-SMFRS 1-Grade Responders at Both Long-term LTFU Baseline and at Subsequent LTFU Visits [Up to 60 months from long-term follow-up (LTFU) baseline (the last visit in the previous study)]

   The investigator evaluated the participant's chin and neck area using the Clinician-Reported Submental Fat Rating Scale (a 5-point scale) where: 0=absent submental convexity (best) to 4= extreme submental convexity (worst). The participant evaluated their chin and neck area using the Patient-Reported Submental Fat Rating Scale (a 5-point scale) where: 0=no chin fat at all (best) to 4= a very large amount of chin fat (worst). Non-responders at LTFU baseline in each treatment group (including placebo) were not included in the analysis.

4. Percentage of Participants Maintaining Composite SMFRS 2-Grade Response During 5 Years of Follow up, i.e. % of Participants Who Were CR-SMFRS and PR-SMFRS 2-Grade Responders at Both Long-term LTFU Baseline and at Subsequent LTFU Visits [Up to 60 months from long-term follow-up (LTFU) baseline (the last visit in the previous study)]

   The investigator evaluated the participant's chin and neck area using the Clinician-Reported Submental Fat Rating Scale (a 5-point scale) where: 0=absent submental convexity (best) to 4= extreme submental convexity (worst). The participant evaluated their chin and neck area using the Patient-Reported Submental Fat Rating Scale (a 5-point scale) where: 0=no chin fat at all (best) to 4= a very large amount of chin fat (worst). Non-responders at LTFU baseline in each treatment group (including placebo) were not included in the analysis.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years to 65 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: Yes

Inclusion Criteria:

• Any subject who successfully completed a Kythera-sponsored clinical trial of ATX-101 (06-03, 07-07, 09-15)

• Signed informed consent

• Willingness to comply with schedule and procedures of the study

Exclusion Criteria:

• Subjects who have had or are undergoing treatment that may affect the evaluation of the submental area will be excluded

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Total Skin & Beauty Dermatology Center	Birmingham	Alabama	United States	35205
2	Clinical Testing Center Beverly Hills	Beverly Hills	California	United States	90210
3	Mokusiga, Inc	Beverly Hills	California	United States	90212
4	Plastic & Reconstructive Surgery	San Francisco	California	United States	94115
5	Dermatology Institute of DuPage Medical Group in Naperville	Naperville	Illinois	United States	60563
6	Skin Care Physicians	Chestnut Hill	Massachusetts	United States	02467
7	Minnesota Clinical Study Center	Fridley	Minnesota	United States	55432
8	Body Aesthetic Research Center	Saint Louis	Missouri	United States	63141
9	Skin Specialists, PC	Omaha	Nebraska	United States	68144
10	Research Across America	Plano	Texas	United States	75093
11	Mei-Heng Tan	Sydney	New South Wales	Australia	2000
12	Skin Centre, AHC House	Benowa	Queensland	Australia	4217
13	Southeast Dermatology Belmont Specialist Centre	Carina Heights	Queensland	Australia	4152
14	T/AS Dermatology Institute of Victoria	South Yarra	Victoria	Australia	3141
15	Niagara Falls Dermatology & Skin Care	Niagara Falls	Ontario	Canada	L2E 7H1
16	Institute of Cosmetic & Laser Surgery	Oakville	Ontario	Canada	L6J 7W5
17	Toronto Cosmetic Skin Surgery Centre	Toronto	Ontario	Canada	M4V 1R1
18	Cosmetic Dermatology on Bloor	Toronto	Ontario	Canada	M5S 3B4
19	The Dermatology Centre	Salford	Manchester	United Kingdom	M6 8HD
20	Cranley Clinic, Harcout House	London		United Kingdom	W1G OPN

Sponsors and Collaborators

• Kythera Biopharmaceuticals

Investigators

• Study Director: Frederick Beddingfield, III, MD, PhD, Sponsor GmbH

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Kythera Biopharmaceuticals
• ClinicalTrials.gov Identifier: NCT02159729
• Other Study ID Numbers: ATX-101-08-12
• First Posted: Jun 10, 2014
• Last Update Posted: Feb 17, 2020
• Last Verified: Jan 1, 2020

Study Results

Participant Flow

Recruitment Details	The objective of this non-treatment, placebo-controlled, 5-year follow-up study was to evaluate the long-term efficacy and safety of subcutaneous (SC) injections of deoxycholic acid (ATX-101) in the submental area. No treatment was administered in this study.
Pre-assignment Detail	Participants who previously received deoxycholic acid or placebo injections in studies ATX-101-06-03, ATX-101-07-07, & ATX-101-09-15 were enrolled in this non-treatment follow-up study to further evaluate safety and efficacy.

Arm/Group Title	ATX-101 (Deoxycholic Acid) Injection	Placebo
Arm/Group Description	This is a non-treatment follow-up study. Participants were previously treated with deoxycholic acid injection, 10 mg/mL in 1 of 3 predecessor studies ATX-101-06-03, ATX-101-07-07, and ATX-101-09-15	This is a non-treatment follow-up study. Participants were previously treated with placebo in 1 of 3 predecessor studies ATX-101-06-03, ATX-101-07-07, and ATX-101-09-15
Period Title: Overall Study
STARTED	140	65
COMPLETED	98	44
NOT COMPLETED	42	21

Baseline Characteristics

Arm/Group Title	ATX-101 (Deoxycholic Acid) Injection	Placebo	Total
Arm/Group Description	This is a non-treatment follow-up study. Participants were previously treated with deoxycholic acid injection, 10 mg/mL in 1 of 3 predecessor studies ATX-101-06-03, ATX-101-07-07, and ATX-101-09-15	This is a non-treatment follow-up study. Participants were previously treated with placebo in 1 of 3 predecessor studies ATX-101-06-03, ATX-101-07-07, and ATX-101-09-15	Total of all reporting groups
Overall Participants	140	65	205
Age (Years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [Years]	46.8 (8.86)	48.2 (9.94)	47.2 (9.22)
Sex: Female, Male (Count of Participants)
Female	111 79.3%	43 66.2%	154 75.1%
Male	29 20.7%	22 33.8%	51 24.9%
Race (NIH/OMB) (Count of Participants)
American Indian or Alaska Native	0 0%	0 0%	0 0%
Asian	0 0%	2 3.1%	2 1%
Native Hawaiian or Other Pacific Islander	0 0%	0 0%	0 0%
Black or African American	2 1.4%	2 3.1%	4 2%
White	131 93.6%	56 86.2%	187 91.2%
More than one race	7 5%	5 7.7%	12 5.9%
Unknown or Not Reported	0 0%	0 0%	0 0%
Baseline weight (kg) (kilogram (kg)) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [kilogram (kg)]	80.64 (16.683)	85.93 (15.983)	82.32 (16.610)

Outcome Measures

1. Primary Outcome

Title	Percentage of Participants Maintaining CR-SMFRS 1-Grade Response During 5 Years of Follow up, i.e. % of Participants Who Were CR-SMFRS 1-Grade Responders at Both Long-term LTFU Baseline and at Subsequent LTFU Visits
Description	The investigator evaluated the participant's chin and neck area using the Clinician-Reported Submental Fat Rating Scale (a 5-point scale) where: 0=absent submental convexity (best) to 4= extreme submental convexity (worst). Non-responders at LTFU baseline in each treatment group (including placebo) were not included in the analysis.
Time Frame	Up to 60 months from long-term follow-up (LTFU) baseline (the last visit in the previous study)

Outcome Measure Data

Analysis Population Description

Analysis population included all who received study treatment from a previous study, and who were CR-SMFRS 1-Grade Responders at LTFU Baseline

Arm/Group Title	ATX-101 (Deoxycholic Acid) Injection	Placebo
Arm/Group Description	This is a non-treatment follow-up study. Participants were previously treated with deoxycholic acid injection, 10 mg/mL in 1 of 3 predecessor studies ATX-101-06-03, ATX-101-07-07, and ATX-101-09-15	This is a non-treatment follow-up study. Participants were previously treated with placebo in 1 of 3 predecessor studies ATX-101-06-03, ATX-101-07-07, and ATX-101-09-15
Measure Participants	104	20
Baseline	100.0 71.4%	100.0 153.8%
Visit 1 (Month 3)	93.1 66.5%	83.3 128.2%
Visit 2 (Month 6)	94.0 67.1%	68.8 105.8%
Visit 3 (Month 9)	94.9 67.8%	81.3 125.1%
Visit 4 (Month 12)	94.0 67.1%	82.4 126.8%
Visit 5 (Month 18)	95.1 67.9%	83.3 128.2%
Visit 6 (Month 24)	89.6 64%	75.0 115.4%
Visit 7 (Month 36)	80.8 57.7%	76.5 117.7%
Visit 8 (Month 48)	85.2 60.9%	64.3 98.9%
Visit 9 (Month 60)	71.2 50.9%	71.4 109.8%

2. Primary Outcome

Title	Percentage of Participants Maintaining PR-SMFRS 1-Grade Response During 5 Years of Follow up, i.e. % of Participants Who Were PR-SMFRS 1-Grade Responders at Both Long-term LTFU Baseline and at Subsequent LTFU Visits
Description	The participant evaluated their chin and neck area using the Patient-Reported Submental Fat Rating Scale (a 5-point scale) where: 0=no chin fat at all (best) to 4= a very large amount of chin fat (worst). Non-responders at LTFU baseline in each treatment group (including placebo) were not included in the analysis.
Time Frame	Up to 60 months from long-term follow-up (LTFU) baseline (the last visit in the previous study)

Outcome Measure Data

Analysis Population Description

Analysis population included all who received study treatment from a previous study, and who were PR-SMFRS 1-Grade Responders at LTFU Baseline

Arm/Group Title	ATX-101 (Deoxycholic Acid) Injection	Placebo
Arm/Group Description	This is a non-treatment follow-up study. Participants were previously treated with deoxycholic acid injection, 10 mg/mL in 1 of 3 predecessor studies ATX-101-06-03, ATX-101-07-07, and ATX-101-09-15	This is a non-treatment follow-up study. Participants were previously treated with placebo in 1 of 3 predecessor studies ATX-101-06-03, ATX-101-07-07, and ATX-101-09-15
Measure Participants	41	21
Baseline	100.0 71.4%	100.0 153.8%
Visit 1 (Month 3)	94.7 67.6%	73.3 112.8%
Visit 2 (Month 6)	94.1 67.2%	80.0 123.1%
Visit 3 (Month 9)	87.5 62.5%	94.4 145.2%
Visit 4 (Month 12)	85.0 60.7%	94.4 145.2%
Visit 5 (Month 18)	80.6 57.6%	83.3 128.2%
Visit 6 (Month 24)	85.7 61.2%	82.4 126.8%
Visit 7 (Month 36)	81.8 58.4%	94.1 144.8%
Visit 8 (Month 48)	78.1 55.8%	80.0 123.1%
Visit 9 (Month 60)	84.8 60.6%	93.3 143.5%

3. Primary Outcome

Title	Percentage of Participants Maintaining Composite SMFRS 1-Grade Response During 5 Years of Follow up, i.e. % of Participants Who Were CR-SMFRS and PR-SMFRS 1-Grade Responders at Both Long-term LTFU Baseline and at Subsequent LTFU Visits
Description	The investigator evaluated the participant's chin and neck area using the Clinician-Reported Submental Fat Rating Scale (a 5-point scale) where: 0=absent submental convexity (best) to 4= extreme submental convexity (worst). The participant evaluated their chin and neck area using the Patient-Reported Submental Fat Rating Scale (a 5-point scale) where: 0=no chin fat at all (best) to 4= a very large amount of chin fat (worst). Non-responders at LTFU baseline in each treatment group (including placebo) were not included in the analysis.
Time Frame	Up to 60 months from long-term follow-up (LTFU) baseline (the last visit in the previous study)

Outcome Measure Data

Analysis Population Description

Analysis population included all who received study treatment from a previous study, and who were composite SMFRS 1-Grade Responders at LTFU Baseline

Arm/Group Title	ATX-101 (Deoxycholic Acid) Injection	Placebo
Arm/Group Description	This is a non-treatment follow-up study. Participants were previously treated with deoxycholic acid injection, 10 mg/mL in 1 of 3 predecessor studies ATX-101-06-03, ATX-101-07-07, and ATX-101-09-15	This is a non-treatment follow-up study. Participants were previously treated with placebo in 1 of 3 predecessor studies ATX-101-06-03, ATX-101-07-07, and ATX-101-09-15
Measure Participants	30	7
Baseline	100.0 71.4%	100.0 153.8%
Visit 1 (Month 3)	84.6 60.4%	75.0 115.4%
Visit 2 (Month 6)	82.6 59%	83.3 128.2%
Visit 3 (Month 9)	82.8 59.1%	100.0 153.8%
Visit 4 (Month 12)	79.3 56.6%	71.4 109.8%
Visit 5 (Month 18)	73.1 52.2%	71.4 109.8%
Visit 6 (Month 24)	73.1 52.2%	83.3 128.2%
Visit 7 (Month 36)	65.2 46.6%	85.7 131.8%
Visit 8 (Month 48)	60.9 43.5%	83.3 128.2%
Visit 9 (Month 60)	73.9 52.8%	83.3 128.2%

4. Primary Outcome

Title	Percentage of Participants Maintaining Composite SMFRS 2-Grade Response During 5 Years of Follow up, i.e. % of Participants Who Were CR-SMFRS and PR-SMFRS 2-Grade Responders at Both Long-term LTFU Baseline and at Subsequent LTFU Visits
Description	The investigator evaluated the participant's chin and neck area using the Clinician-Reported Submental Fat Rating Scale (a 5-point scale) where: 0=absent submental convexity (best) to 4= extreme submental convexity (worst). The participant evaluated their chin and neck area using the Patient-Reported Submental Fat Rating Scale (a 5-point scale) where: 0=no chin fat at all (best) to 4= a very large amount of chin fat (worst). Non-responders at LTFU baseline in each treatment group (including placebo) were not included in the analysis.
Time Frame	Up to 60 months from long-term follow-up (LTFU) baseline (the last visit in the previous study)

Outcome Measure Data

Analysis Population Description

Analysis population included all who received study treatment from a previous study, and who were composite SMFRS 2-Grade Responders at LTFU Baseline

Arm/Group Title	ATX-101 (Deoxycholic Acid) Injection	Placebo
Arm/Group Description	This is a non-treatment follow-up study. Participants were previously treated with deoxycholic acid injection, 10 mg/mL in 1 of 3 predecessor studies ATX-101-06-03, ATX-101-07-07, and ATX-101-09-15	This is a non-treatment follow-up study. Participants were previously treated with placebo in 1 of 3 predecessor studies ATX-101-06-03, ATX-101-07-07, and ATX-101-09-15
Measure Participants	3	1
Baseline	100.0 71.4%	100.0 153.8%
Visit 1 (Month 3)	0.0 0%	0.0 0%
Visit 2 (Month 6)	66.7 47.6%	100.0 153.8%
Visit 3 (Month 9)	66.7 47.6%	100.0 153.8%
Visit 4 (Month 12)	100.0 71.4%	100.0 153.8%
Visit 5 (Month 18)	0.0 0%	100.0 153.8%
Visit 6 (Month 24)	100.0 71.4%	100.0 153.8%
Visit 7 (Month 36)	100.0 71.4%	0.0 0%
Visit 8 (Month 48)	100.0 71.4%	0.0 0%
Visit 9 (Month 60)	100.0 71.4%	100.0 153.8%

Adverse Events

Time Frame	Up to 60 months from the last visit in the previous study
Adverse Event Reporting Description	All enrolled participants with a clearly identifiable actual treatment received from a previous ATX-101 study and at least 1 visit in the long-term follow-up (LTFU) study were included in the analysis population. All adverse events (AEs) and serious adverse events (SAEs) that were ongoing at the conclusion of the predecessor Studies ATX-101-06-03, ATX-101-07-07, and ATX-101-09-15, were followed until they resolved or were considered medically stable.
Arm/Group Title	ATX-101 (Deoxycholic Acid) Injection		Placebo
Arm/Group Description	This is a non-treatment follow-up study. Participants were previously treated with deoxycholic acid injection, 10 mg/mL in 1 of 3 predecessor studies ATX-101-06-03, ATX-101-07-07, and ATX-101-09-15		This is a non-treatment follow-up study. Participants were previously treated with placebo in 1 of 3 predecessor studies ATX-101-06-03, ATX-101-07-07, and ATX-101-09-15
All Cause Mortality
	ATX-101 (Deoxycholic Acid) Injection		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/140 (0%)		1/65 (1.5%)
Serious Adverse Events
	ATX-101 (Deoxycholic Acid) Injection		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	3/140 (2.1%)		3/65 (4.6%)
Cardiac disorders
Third degree heart block	1/140 (0.7%)	1	0/65 (0%)	0
Endocrine disorders
Asymptomatic cystic thyroid mass	1/140 (0.7%)	1	0/65 (0%)	0
Thyroid cancer	0/140 (0%)	0	1/65 (1.5%)	1
Metabolism and nutrition disorders
Hypocalcemia	0/140 (0%)	0	1/65 (1.5%)	1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Breast cancer	1/140 (0.7%)	1	0/65 (0%)	0
Patient died of pancreatic cancer	0/140 (0%)	0	1/65 (1.5%)	1
Prostate cancer	0/140 (0%)	0	1/65 (1.5%)	1
Other (Not Including Serious) Adverse Events
	ATX-101 (Deoxycholic Acid) Injection		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/140 (0%)		0/65 (0%)

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

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

A disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 90 days from the time submitted to the sponsor for review. The sponsor cannot require changes to the communication and cannot extend the embargo.