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

• Sponsor: Kythera Biopharmaceuticals (Industry)
• Overall Status: Completed
• CT.gov ID: NCT02163902
• Collaborator: (none)
• Enrollment: 224
• Locations: 15
• Arms: 2
• Duration (Months): 32
• Patients Per Site: 14.9
• Patients Per Site Per Month: 0.5

Study Details

Study Description

Brief Summary

The investigation of the long-term safety and maintenance of efficacy of ATX-101 in reduction of submental fat.

Condition or Disease	Intervention/Treatment	Phase
Submental Fat; Healthy	Drug: ATX-101; Drug: Placebo	Phase 3

Detailed Description

No study medication is administered in this study.

Study Design

• Study Type: Interventional
• Actual Enrollment: 224 participants
• Allocation: Non-Randomized
• Intervention Model: Parallel Assignment
• Masking: Double (Participant, Investigator)
• Primary Purpose: Other
• Official Title: A Multicenter, Double-blind, Nontreatment, Long-term Follow up Study of Subjects Who Completed ATX-101 (Deoxycholic Acid Injection) Clinical Trials ATX-101-11-22 or ATX-101-11-23 for the Reduction of Localized Subcutaneous Fat in the Submental Area
• Study Start Date: Dec 1, 2013
• Actual Primary Completion Date: Jan 1, 2016
• Actual Study Completion Date: Aug 1, 2016

Arms and Interventions

Arm	Intervention/Treatment
Experimental: ATX-101 Participants treated with ATX-101 in previous studies ATX-101-11-22 and ATX-101-11-23.	Drug: ATX-101; Clinical evaluation, patient reported outcome questionnaires, photographs, caliper measurements, recording of adverse events
Placebo Comparator: Placebo Participants treated with placebo in previous studies ATX-101-11-22 and ATX-101-11-23.	Drug: Placebo; Clinical evaluation, patient reported outcome questionnaires, photographs, caliper measurements, recording of adverse events

Outcome Measures

Primary Outcome Measures

1. Percentage of Participants Maintaining CR-SMFRS 1-Grade Response During 3 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 [From 12 weeks after last treatment (in the predecessor study) to up to 36 months after last treatment]

   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.

Secondary Outcome Measures

1. Percentage of Participants Maintaining CR-SMFRS 2-Grade Response During 3 Years of Follow up, i.e. % of Participants Who Were CR-SMFRS 2-Grade Responders at Both Long-term LTFU Baseline and at Subsequent LTFU Visits [From 12 weeks after last treatment (in the predecessor study) to up to 36 months after last treatment.]

   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 3 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 [From 12 weeks after last treatment (in the predecessor study) to up to 36 months after last treatment]

   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 PR-SMFRS 2-Grade Response During 3 Years of Follow up, i.e. % of Participants Who Were PR-SMFRS 2-Grade Responders at Both Long-term LTFU Baseline and at Subsequent LTFU Visits [From 12 weeks after last treatment (in the predecessor study) to up to 36 months after last treatment]

   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 1-Grade Response During 3 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 [From 12 weeks after last treatment (in the predecessor study) to up to 36 months after last treatment]

   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.

5. Percentage of Participants Maintaining Composite SMFRS 2-Grade Response During 3 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 [From 12 weeks after last treatment (in the predecessor study) to up to 36 months after last treatment]

   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:

1. Any participant who completed follow-up visits at 12 and 24 weeks after the last dose in one of the two predecessor studies

2. Willingness to comply with the schedule and procedures of this study.

3. Signed informed consent form (ICF).

Exclusion Criteria:

1. Participants who have had any treatment or condition (e.g., pregnancy or metabolic disease, which may lead to unstable weight) that may affect assessment of safety or efficacy since enrollment in the predecessor study.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Dermatology Cosmetic Laser Medical Associates of La Jolla	San Diego	California	United States	92121
2	AboutSkin Dermatology and DermSurgery, PC	Englewood	Colorado	United States	80113
3	Dermatology Research Institute	Coral Gables	Florida	United States	33146
4	Stephan Baker MD PA	Coral Gables	Florida	United States	33146
5	Baumann Cosmetic and Research Center	Miami	Florida	United States	33137
6	Kenneth R. Beer, MD, PA	West Palm Beach	Florida	United States	33401
7	Altman Dermatology Associates	Arlington Hts	Illinois	United States	60005
8	DeNova Research	Chicago	Illinois	United States	60611
9	DuPage Medical Group, Dermatology Institute	Naperville	Illinois	United States	60563
10	Callender Center for Clinical Research	Glendale	Maryland	United States	20769
11	Aesthetics, Skin Care, Dermatologic Surgery	Rockville	Maryland	United States	20850
12	Tennessee Clinical Research Center	Nashville	Tennessee	United States	37215
13	Research Across America	Plano	Texas	United States	75093
14	Shannon Humphrey, MD	Vancouver		Canada	V5Z 4E1
15	Carruthers Dermatology Centre Inc.	Vancouver		Canada	V5Z4E1

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: NCT02163902
• Other Study ID Numbers: ATX-101-13-35
• First Posted: Jun 16, 2014
• Last Update Posted: Feb 17, 2020
• Last Verified: Jan 1, 2020

Study Results

Participant Flow

Recruitment Details	The objective of this non-treatment, double-blind, placebo-controlled 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-101-11-22 and ATX-101-11-23 were enrolled in this non-treatment, double-blind, placebo-controlled 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 2 predecessor studies ATX-101-11-22 and ATX-101-11-23.	This is a non-treatment follow-up study. Participants were previously treated with placebo in 1 of 2 predecessor studies ATX-101- 11-22 and ATX-101-11-23.
Period Title: Overall Study
STARTED	113	111
COMPLETED	102	98
NOT COMPLETED	11	13

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 2 predecessor studies ATX-101-11-22 and ATX-101-11-23.	This is a non-treatment follow-up study. Participants were previously treated with placebo in 1 of 2 predecessor studies ATX-101- 11-22 and ATX-101-11-23.	Total of all reporting groups
Overall Participants	113	111	224
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	51.0 (8.22)	50.1 (9.00)	50.6 (8.61)
Age, Customized (Count of Participants)
≤ 30 years	3 2.7%	4 3.6%	7 3.1%
> 30 to ≤ 50 years	52 46%	49 44.1%	101 45.1%
> 50 years	58 51.3%	58 52.3%	116 51.8%
< 50 years	47 41.6%	47 42.3%	94 42%
Sex: Female, Male (Count of Participants)
Female	96 85%	95 85.6%	191 85.3%
Male	17 15%	16 14.4%	33 14.7%
Ethnicity (NIH/OMB) (Count of Participants)
Hispanic or Latino	33 29.2%	25 22.5%	58 25.9%
Not Hispanic or Latino	80 70.8%	86 77.5%	166 74.1%
Unknown or Not Reported	0 0%	0 0%	0 0%
Race (NIH/OMB) (Count of Participants)
American Indian or Alaska Native	0 0%	1 0.9%	1 0.4%
Asian	4 3.5%	1 0.9%	5 2.2%
Native Hawaiian or Other Pacific Islander	1 0.9%	0 0%	1 0.4%
Black or African American	12 10.6%	7 6.3%	19 8.5%
White	96 85%	101 91%	197 87.9%
More than one race	0 0%	1 0.9%	1 0.4%
Unknown or Not Reported	0 0%	0 0%	0 0%
Body Mass Index (BMI) (kg/m^2) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [kg/m^2]	29.283 (4.4934)	28.836 (4.0099)	29.062 (4.2570)
Fitzpatrick Score (Count of Participants)
I	4 3.5%	4 3.6%	8 3.6%
II	30 26.5%	39 35.1%	69 30.8%
III	33 29.2%	36 32.4%	69 30.8%
IV	35 31%	20 18%	55 24.6%
V	9 8%	9 8.1%	18 8%
VI	2 1.8%	3 2.7%	5 2.2%

Outcome Measures

1. Primary Outcome

Title	Percentage of Participants Maintaining CR-SMFRS 1-Grade Response During 3 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	From 12 weeks after last treatment (in the predecessor study) to up to 36 months after last treatment

Outcome Measure Data

Analysis Population Description

Participants who had at least a 1-grade reduction from baseline on the CRSMFRS (ie, CR-1 responder) at 12-weeks after last treatment in the predecessor studies.

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 2 predecessor studies ATX-101-11-22 and ATX-101-11-23.	This is a non-treatment follow-up study. Participants were previously treated with placebo in 1 of 2 predecessor studies ATX-101- 11-22 and ATX-101-11-23.
Measure Participants	95	46
Visit 1 (Year 1) Percentage	86.4 76.5%	56.8 51.2%
Visit 2 (Year 2) Percentage	90.6 80.2%	73.8 66.5%
Visit 3 (Year 3) Percentage	82.4 72.9%	65.0 58.6%

2. Secondary Outcome

Title	Percentage of Participants Maintaining CR-SMFRS 2-Grade Response During 3 Years of Follow up, i.e. % of Participants Who Were CR-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). Non-responders at LTFU baseline in each treatment group (including placebo) were not included in the analysis
Time Frame	From 12 weeks after last treatment (in the predecessor study) to up to 36 months after last treatment.

Outcome Measure Data

Analysis Population Description

Analysis Population: Participants who had at least a 2-grade reduction from baseline on the CR-SMFRS (ie, CR-2 responder) at 12-weeks after last treatment in the predecessor studies.

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 2 predecessor studies ATX-101-11-22 and ATX-101-11-23.	This is a non-treatment follow-up study. Participants were previously treated with placebo in 1 of 2 predecessor studies ATX-101- 11-22 and ATX-101-11-23.
Measure Participants	44	12
Visit 1 (Year 1) Percentage	75.0 66.4%	18.2 16.4%
Visit 2 (Year 2) Percentage	72.5 64.2%	33.3 30%
Visit 3 (Year 3) Percentage	65.8 58.2%	33.3 30%

3. Secondary Outcome

Title	Percentage of Participants Maintaining PR-SMFRS 1-Grade Response During 3 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	From 12 weeks after last treatment (in the predecessor study) to up to 36 months after last treatment

Outcome Measure Data

Analysis Population Description

Analysis Population: Participants who had at least a 1-grade reduction from baseline in PR-SMFRS (ie, PR-1 responder) at 12-weeks after last treatment in the predecessor studies.

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 2 predecessor studies ATX-101-11-22 and ATX-101-11-23.	This is a non-treatment follow-up study. Participants were previously treated with placebo in 1 of 2 predecessor studies ATX-101- 11-22 and ATX-101-11-23.
Measure Participants	98	58
Visit 1 (Year 1) Percentage	95.5 84.5%	76.4 68.8%
Visit 2 (Year 2) Percentage	87.8 77.7%	83.0 74.8%
Visit 3 (Year 3) Percentage	77.0 68.1%	75.0 67.6%

4. Secondary Outcome

Title	Percentage of Participants Maintaining PR-SMFRS 2-Grade Response During 3 Years of Follow up, i.e. % of Participants Who Were PR-SMFRS 2-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	From 12 weeks after last treatment (in the predecessor study) to up to 36 months after last treatment

Outcome Measure Data

Analysis Population Description

Analysis Population: Participants who had at least a 2-grade reduction from baseline in PR-SMFRS (ie, PR-2 responder) at 12-weeks after last treatment in the predecessor studies.

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 2 predecessor studies ATX-101-11-22 and ATX-101-11-23.	This is a non-treatment follow-up study. Participants were previously treated with placebo in 1 of 2 predecessor studies ATX-101- 11-22 and ATX-101-11-23.
Measure Participants	43	11
Visit 1 (Year 1) Percentage	59.0 52.2%	63.6 57.3%
Visit 2 (Year 2) Percentage	57.5 50.9%	60.0 54.1%
Visit 3 (Year 3) Percentage	59.5 52.7%	70.0 63.1%

5. Secondary Outcome

Title	Percentage of Participants Maintaining Composite SMFRS 1-Grade Response During 3 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	From 12 weeks after last treatment (in the predecessor study) to up to 36 months after last treatment

Outcome Measure Data

Analysis Population Description

Analysis Population: Participants who were both CR-1 responders and PR-1 responders (SMFRS-1) at 12-weeks after last treatment in the predecessor studies.

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 2 predecessor studies ATX-101-11-22 and ATX-101-11-23.	This is a non-treatment follow-up study. Participants were previously treated with placebo in 1 of 2 predecessor studies ATX-101- 11-22 and ATX-101-11-23.
Measure Participants	83	31
Visit 1 (Year 1) Percentage	86.8 76.8%	44.8 40.4%
Visit 2 (Year 2) Percentage	85.1 75.3%	64.3 57.9%
Visit 3 (Year 3) Percentage	72.6 64.2%	46.2 41.6%

6. Secondary Outcome

Title	Percentage of Participants Maintaining Composite SMFRS 2-Grade Response During 3 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	From 12 weeks after last treatment (in the predecessor study) to up to 36 months after last treatment

Outcome Measure Data

Analysis Population Description

Analysis Population: Participants who were both CR-2 responders and PR-2 responders (SMFRS-2) at 12-weeks after last treatment in the predecessor studies.

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 2 predecessor studies ATX-101-11-22 and ATX-101-11-23.	This is a non-treatment follow-up study. Participants were previously treated with placebo in 1 of 2 predecessor studies ATX-101- 11-22 and ATX-101-11-23.
Measure Participants	18	3
Visit 1 (Year 1) Percentage	41.2 36.5%	0.0 0%
Visit 2 (Year 2) Percentage	43.8 38.8%	0.0 0%
Visit 3 (Year 3) Percentage	50.0 44.2%	50.0 45%

Adverse Events

Time Frame	From the first exposure to study drug in predecessor study to up to 36 months after last treatment in predecessor 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-101-11-22 and ATX-101-11-23 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 2 predecessor studies ATX-101-11-22 and ATX-101-11-23.		This is a non-treatment follow-up study. Participants were previously treated with placebo in 1 of 2 predecessor studies ATX-101- 11-22 and ATX-101-11-23.
All Cause Mortality
	ATX-101 (Deoxycholic Acid) Injection		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/113 (0%)		0/111 (0%)
Serious Adverse Events
	ATX-101 (Deoxycholic Acid) Injection		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	5/113 (4.4%)		1/111 (0.9%)
Gastrointestinal disorders
Diverticulitis	2/113 (1.8%)	2	0/111 (0%)	0
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Small cell lung cancer stage unspecified	1/113 (0.9%)	1	0/111 (0%)	0
Breast cancer in situ	0/96 (0%)	0	1/95 (1.1%)	1
Reproductive system and breast disorders
Ovarian cancer recurrent	1/96 (1%)	1	0/95 (0%)	0
Surgical and medical procedures
Breast reconstruction	1/96 (1%)	1	0/95 (0%)	0
Other (Not Including Serious) Adverse Events
	ATX-101 (Deoxycholic Acid) Injection		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/113 (0%)		0/111 (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.