Trial Outcomes & Findings for Open-Label Study of Deoxycholic Acid for the Reduction of Localized Subcutaneous Fat in the Submental Area (NCT NCT01426373)

The study was conducted at 18 investigational centers in the United States.

Open-Label Study of Deoxycholic Acid for the Reduction of Localized Subcutaneous Fat in the Submental Area

Serious AEs include any event that met one or more of the following criteria: was fatal or life-threatening, required inpatient hospitalization or prolonged a hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect or a significant medical hazard. The severity of each AE was defined as either: Mild: The participant was aware of the sign or symptom, but it was easily tolerated. Moderate: The sign or symptom caused discomfort and interfered with usual activity. Severe: The sign or symptom was incapacitating, and the participant was unable to engage in usual activity. The investigator determined the relationship of each AE to the study drug using the question: "Is there a reasonable possibility that the event may have been caused by treatment with the study drug?"

The CR-SMFRS score is based on the investigator's clinical evaluation of the participant, where submental fullness is scored on a 5-point ordinal scale (0 = absent, 1 = mild, 2 = moderate, 3 = severe, and 4 = extreme). A negative change from baseline indicates improvement.

The PR-SMFRS is based on the participant's response to the question "How much fat do you have under your chin right now?" and answered on a 5-point ordinal scale (0 = no chin fat at all, 1 = a slight amount of chin fat, 2 = a moderate amount of chin fat, 3 = a large amount of chin fat, and 4 = a very large amount of chin fat). A negative change from baseline indicates improvement.

A composite 1-grade response is defined as at least a 1-grade improvement from baseline on both the CR-SMFRS and PR-SMFRS. The CR-SMFRS score is based on the investigator's clinical evaluation of the participant, where submental fullness is scored on a 5-point ordinal scale (0 = absent, 1 = mild, 2 = moderate, 3 = severe, and 4 = extreme). The PR-SMFRS is based on the participant's response to the question "How much fat do you have under your chin right now?" and answered on a 5-point ordinal scale (0 = no chin fat at all, 1 = a slight amount of chin fat, 2 = a moderate amount of chin fat, 3 = a large amount of chin fat, and 4 = a very large amount of chin fat).

A composite 2-grade response is defined as at least a 2-grade improvement from baseline on both the CR-SMFRS and PR-SMFRS. The CR-SMFRS score is based on the investigator's clinical evaluation of the participant, where submental fullness is scored on a 5-point ordinal scale (0 = absent, 1 = mild, 2 = moderate, 3 = severe, and 4 = extreme). The PR-SMFRS is based on the participant's response to the question "How much fat do you have under your chin right now?" and answered on a 5-point ordinal scale (0 = no chin fat at all, 1 = a slight amount of chin fat, 2 = a moderate amount of chin fat, 3 = a large amount of chin fat, and 4 = a very large amount of chin fat).

The PR-SMFIS assesses the impact of submental fat on self-perception of 6 emotional and visual characteristics (unhappy, bothered, self-conscious, embarrassed, look older, and look overweight) related to the appearance of submental fullness as evaluated by the participant. Each item is rated on an 11-point numeric scale from 0 to 10. Scores for the 6 items were averaged to generate a PR-SMFIS total scale score ranging from 0 to 10 where 0 is a positive outcome and 10 is a negative outcome. A negative change from baseline indicates improvement.

The SSRS assesses participants' satisfaction with their appearance in association with the face and chin on a 7-point scale from 0 to 6 (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.

Self-rating of Attractiveness assesses aspects of appearance from the participant's perspective with a series of 6 questions: How attractive do you think your overall appearance (chin/neck, eyes, nose, mouth, entire face) is/are?" Each question was answered on a scale from 1 to 9 (1 = not at all attractive, 5 = neither attractive nor unattractive, and 9 = extremely attractive). A positive change from baseline indicates improvement.

Participants answered 3 questions on a 7-point scale that ranged from "a great deal worse" to "a great deal better" (questions 1 and 2) or from "extremely dissatisfied" to "extremely satisfied" (question 3). Question 1: Since the start of the study, how would you rate the fat under your chin right now? Question 2: Since the start of the study, how would you rate the definition between your chin and neck right now? Question 3: How satisfied are you with the treatment you received in this study?

Submental fat thickness was measured using calipers.

SMSLG assessment was based on clinical evaluation and palpation of the submental area. The SMSLG scale incorporates 3 features: skin wrinkling, adherence to underlying neck structures (bone and muscle) and redundancy (horizontal and vertical folds). Grade 1 (none): no or minimal superficial wrinkling, skin well apposed to deeper neck structures, no skin redundancy (no skin draping (vertical folds) or skin sagging (horizontal folds)); Grade 2 (mild): mild superficial wrinkling, skin well apposed to deeper neck structures, minimal skin redundancy (slight skin draping and sagging); Grade 3 (moderate): may have mild to moderate superficial wrinkling, skin has mild to moderate separation from deeper neck structures, moderate skin redundancy (moderate skin draping and skin sagging); Grade 4 (severe): mild to marked superficial wrinkling, loose skin separated from deeper neck structures, marked skin redundancy (marked skin draping and sagging).

Each participant was given 2 example line drawings representing each of the 5 submental fat grades (0 = absent, 1 = mild, 2 = moderate, 3 = severe, and 4 = extreme) and asked to select the drawing that best represents their current profile. Improvement is any decrease in grade, and worsening is any increase in grade.