Phase 2 Study of Deoxycholic Acid Injection (ATX-101) for the Reduction of Submental Fat

• ClinicalTrials.gov Identifier: NCT00618618
• Recruitment Status: Completed
• First Posted: February 20, 2008
• Results First Posted: June 15, 2015
• Last Update Posted: July 14, 2015
• Sponsor: Kythera Biopharmaceuticals
• Information provided by (Responsible Party): Kythera Biopharmaceuticals

Study Description

Brief Summary:

Phase 2 trial to evaluate the safety and potential efficacy of one concentration of deoxycholic acid injection, given in three dosing paradigms, compared to placebo for the reduction of submental fat (fat beneath the chin).

Condition or disease	Intervention/treatment	Phase
Moderate or Severe Submental Fullness	Drug: Deoxycholic Acid Injection; Drug: Placebo	Phase 2

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 73 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Double (Participant, Investigator)
• Primary Purpose: Treatment
• Official Title: Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Study of the Safety and Efficacy of ATX-101 (Sodium Deoxycholate for Injection) Given by Three Dosing Paradigms for the Reduction of Localized Subcutaneous Fat in the Submental Area
• Study Start Date: April 2008
• Actual Primary Completion Date: December 2008
• Actual Study Completion Date: December 2008

Arms and Interventions

Arm	Intervention/treatment
Experimental: Deoxycholic Acid Injection 0.2 mL/0.7 cm; Participants received deoxycholic acid administered in 0.2 mL injections, 0.7 cm apart, up to 9.6 mL per treatment session at intervals of approximately 1 month for up to a maximum of 4 treatments.	Drug: Deoxycholic Acid Injection; Formulated as an injectable solution containing deoxycholic acid at a concentration of 10 mg/mL.; Other Name: ATX-101
Placebo Comparator: Placebo 0.2 mL/0.7 cm; Participants received placebo administered in 0.2 mL injections, 0.7 cm apart, up to 9.6 mL per treatment session at intervals of approximately 1 month for up to a maximum of 4 treatments.	Drug: Placebo
Experimental: Deoxycholic Acid Injection 0.2 mL/1.0 cm; Participants received deoxycholic acid administered in 0.2 mL injections, 1.0 cm apart, up to 4.8 mL per treatment session at intervals of approximately 1 month for up to a maximum of 4 treatments.	Drug: Deoxycholic Acid Injection; Formulated as an injectable solution containing deoxycholic acid at a concentration of 10 mg/mL.; Other Name: ATX-101
Placebo Comparator: Placebo 0.2 mL/1.0 cm; Participants received placebo administered in 0.2 mL injections, 1.0 cm apart, up to 4.8 mL per treatment session at intervals of approximately 1 month for up to a maximum of 4 treatments.	Drug: Placebo
Experimental: Deoxycholic Acid Injection 0.4 mL/1.0 cm; Participants received deoxycholic acid administered in 0.4 mL injections, 1.0 cm apart, up to 9.6 mL per treatment session at intervals of approximately 1 month for up to a maximum of 4 treatments.	Drug: Deoxycholic Acid Injection; Formulated as an injectable solution containing deoxycholic acid at a concentration of 10 mg/mL.; Other Name: ATX-101
Placebo Comparator: Placebo 0.4 mL/1.0 cm; Participants received placebo administered in 0.4 mL injections, 1.0 cm apart, up to 9.6 mL per treatment session at intervals of approximately 1 month for up to a maximum of 4 treatments.	Drug: Placebo

Outcome Measures

Primary Outcome Measures :

1. Number of Participants With Adverse Events [ Time Frame: From the first dose of study drug until 12 weeks after the last dose (up to 24 weeks after first treatment). ]

   The investigator determined the relationship of each adverse event to the administration of study drug.

   Severity of adverse events was determined using the following scale:

   • Mild: The participant is aware of a sign or symptom, but it is easily tolerated
   • Moderate: Discomfort or interference with usual activity
   • Severe: Incapacitating, with inability to engage in usual activity.

   A serious AE (SAE) was defined as an event that may constitute a significant medical hazard or side-effect, regardless of the investigator or sponsor's opinion regarding relatedness to study material. Serious events included, but were not limited to, any event that:

   • was fatal
   • was life-threatening
   • required inpatient hospitalization or prolongation of existing hospitalization
   • resulted in persistent or significant disability/incapacity
   • was a congenital anomaly/birth defect
   • other significant medical hazard
2. Number of Participants With Clinically Significant Changes From Baseline in Laboratory Values, Weight, Vital Signs, and Physical Examinations [ Time Frame: From the first dose of study drug until 12 weeks after the last dose (up to 24 weeks after first treatment). ]

Secondary Outcome Measures :

1. Change From Baseline in Submental Fat (SMF) Rating Scale Score [ Time Frame: Baseline and 4 weeks after last treatment (up to 16 weeks after first dose) ]

   The SMF rating scale score is based on the investigator's clinical evaluation of the participant, where submental fullness is scored on a 5-point ordinal scale (0-4) with 0 = absent, 1 = mild, 2 = moderate, 3 = severe, and 4 = extreme.

   A negative change from Baseline indicates improvement.

2. Change From Baseline in Subject Satisfaction With Appearance Rating Scale [ Time Frame: Baseline and 4 weeks after last treatment (up to 16 weeks after first dose) ]

   The Subject Satisfaction with Appearance Rating Scale assesses participants' satisfaction with their appearance in association with the face and chin on a 7-point scale from 0 to 6 where 0 = Extremely dissatisfied, 1 = Dissatisfied, 2 = Slightly dissatisfied, 3 = Neither satisfied nor dissatisfied, 4 = Slightly satisfied, 5 = Satisfied and 6 = Extremely satisfied.

   A positive change from Baseline indicates improvement.

3. Percentage of Participants With a Response in the Subject Global Improvement Rating [ Time Frame: 4 weeks after last treatment (up to 16 weeks after first dose) ]

   Participants were asked to rate their total improvement or worsening in the appearance and physical feeling of their chin and neck area since before they received study treatment, whether or not they believed it was due to study treatment or to any other cause.

   0 = Very much worse, 1 = Much worse, 2 = Minimally worse, 3 = No change, 4 = Minimally improved, 5 = Much improved, 6 = Very much improved.

   Response is defined as any improvement, ie, a global improvement rating of 4, 5, or 6.

4. Percentage of Participants With an SMF Response [ Time Frame: Baseline and 4 weeks after last treatment (up to 16 weeks after first dose) ]
   Response is defined as a participant with at least a 1-grade improvement in SMF Rating Scale score at Week 16 from Baseline. The SMF rating scale score is based on the investigator's clinical evaluation of the participant, where submental fullness is scored on a 5-point ordinal scale (0-4) with 0 = absent, 1 = mild, 2 = moderate, 3 = severe, and 4 = extreme.
5. Change From Baseline in Skin Laxity Rating [ Time Frame: Baseline and Week 4, Week 8, Week 12, Week 16 (4 weeks after last treatment) and Week 24 (12 weeks after last treatment) ]

   Skin laxity assessment was based on clinical evaluation and palpation of the submental area on the following scale:

   1 = no laxity; 2 = minimal laxity; 3 = moderate laxity; 4 = very lax. A negative change from Baseline indicates improvement.

6. Change From Baseline to Each Visit in Submental Fat (SMF) Rating Scale Score [ Time Frame: Baseline and Week 4, Week 8, Week 12, Week 16 (4 weeks after last treatment) and Week 24 (12 weeks after last treatment) ]

   The SMF rating scale score is based on the investigator's clinical evaluation of the participant, where submental fullness is scored on a 5-point ordinal scale (0-4) with 0 = absent, 1 = mild, 2 = moderate, 3 = severe, and 4 = extreme.

   A negative change from Baseline indicates improvement.

7. Change From Baseline in the Cervicomental Angle [ Time Frame: Baseline and 4 weeks after last treatment (up to 16 weeks after first dose) ]
   The cervicomental angle was measured using a profile view photograph obtained at each visit. A goniometer was used to determine the angle. Cervicomental angle measurements less than 80 degrees are excluded, due to error in measurement.
8. Visual Analogue Scale Pain Intensity Rating [ Time Frame: Approximately 60 minutes after completion of each treatment session at Week 0, Week 4, Week 8 and Week 12 ]
   Participants rated pain associated with the submental area on a 100 mm horizontal axis ranging from 0 (no pain) to 100 (most severe pain possible)

Eligibility Criteria

• Ages Eligible for Study: 25 Years to 65 Years (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

Inclusion Criteria:

• Submental fat that was considered undesirable by the subject and graded by the investigator as 2 or 3 using the submental fat (SMF) rating scale
• Good general health
• Signed informed consent

Exclusion Criteria:

• History of any treatment in the neck or chin area
• Loose skin or prominent platysmal bands in the neck or chin area
• Recent treatment with anticoagulants
• Presence of clinically significant health problems

Contacts and Locations

Locations

Australia, Victoria

Investigational Site

Toorak, Victoria, Australia, 3142

Australia

Investigational Site

Sydney, Australia, 2000

Canada, Ontario

Investigational Site

Niagara Falls, Ontario, Canada, L2E 7H1

Investigational Site

Oakville, Ontario, Canada, L6J 7W5

Investigational Site

Toronto, Ontario, Canada, M4V 1R1

Investigational Site

Toronto, Ontario, Canada, M5S 3B4

United Kingdom

Investigational Site

Manchester, United Kingdom, M68HD

Sponsors and Collaborators

Kythera Biopharmaceuticals

Investigators

• Study Director: Frederick Beddingfield, III, M.D., Ph.D.; Kythera Biopharmaceuticals, Inc.

More Information

• Responsible Party: Kythera Biopharmaceuticals
• ClinicalTrials.gov Identifier: NCT00618618
• Other Study ID Numbers: ATX-101-07-07; 2007-006303-21 ( EudraCT Number )
• First Posted: February 20, 2008
• Results First Posted: June 15, 2015
• Last Update Posted: July 14, 2015
• Last Verified: June 2015

Additional relevant MeSH terms:

Deoxycholic Acid; Cholagogues and Choleretics; Gastrointestinal Agents