Double Blind Study to Evaluate the Efficacy of Collagenase Histolyticum in the Treatment of Lipoma

A Double Blind Study to Evaluate the Safety and Efficacy of Collagenase Clostridium Histolyticum (AA4500) in the Treatment of Lipoma

The purpose of this study is to determine whether collagenase is effective in reducing the surface area of a subcutaneous benign lipoma.

Study Overview

• Status: Completed
• Conditions: Lipoma
• Intervention / Treatment: Drug: placebo; Drug: AA4500

Detailed Description

This study will evaluate the safety and efficacy of a Food and Drug Administration (FDA) approved drug (XIAFLEX) in the treatment of lipoma (fatty tumors). The fat in the lipoma is like normal fat except that it is enclosed in a balloon-like structure which is made of collagen (fibrous tissue). Treatment of the lipoma with an injection of XIAFLEX (a protein that breaks down collagen fibers) may dissolve the collagen/fibrous strands thereby decreasing the size of the lipoma or removing it. Each subject must have two lipomas; one lipoma to be treated with placebo and one lipoma to be treated with study drug, both to be observed similarly throughout the trial.

• Study Type: Interventional
• Enrollment (Actual): 19
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • New York
    • Hewlett, New York, United States, 11557
      • Aesthetic Center
  • Tennessee
    • Nashville, Tennessee, United States, 37232
      • Vanderbilt University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 75 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• 18-75 years of age, of either sex or any race
• Clinical history of at least 2 lipomas for at least one year
• Lipomas diagnosed as benign
• Two lipomas on distinctly separate parts of the body, or on the torso if 30 cm apart, with easily definable edges
• Each lipoma is a single mass with easily definable edges
• Lipomas are 5 to 24 cm squared
• Women of childbearing potential must use an acceptable method of birth control

Exclusion Criteria:

• Lipomas on the head, neck, hand or foot, or female breast
• Women who are nursing or pregnant
• Multi-lobular lipomas
• Subjects who have received an investigational drug within 30 days before receiving the first dose of study drug in this study
• Subjects with a known allergy to collagenase or any of the inactive ingredients in XIAFLEX
• Subjects with uncontrolled diabetes, hypertension, or thyroid disease, or any medical condition that would make the subject unsuitable for enrollment
• Subjects having prior treatment or trauma of the lipoma that could interfere with study assessments
• Subjects with a history of connective tissue diseases, rheumatological diseases
• Subjects taking anticoagulants or planning to receive anticoagulants (except for lo dose aspirin and over-the-counter nonsteroidal anti-inflammatory drugs) within 7 days of injection of study drug

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: AA4500; single injection of 0.58 mg study drug	Drug: AA4500; Subjects must present with 2 lipomas; one to receive AA4500 and one to receive placebo simultaneously; Other Names: XIAFLEX, Collagenase Clostridium Histolyticum
Placebo Comparator: Placebo; single injection of placebo	Drug: placebo; Placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percent Change From Baseline in Surface Area of the Lipoma at Six Months	The primary efficacy outcome is lipoma visible surface area defined as the longest dimension ("length") times the longest dimension perpendicular to length ("width"). Visible surface area will be analyzed as the percent change from baseline at the 6-month visit.	Baseline and 6 months post injection

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Responder Analysis	The number of participants with at least 50% decrease in visible lipoma surface area of lipoma relative to baseline	Baseline and 6 months
Percent Change From Baseline in Greatest Dimension (Length) of Lipoma at 6 Months	Change from baseline in lipoma length Calculated as the percent change from baseline for length of the lipoma treated with AA4500 and the lipoma treated with placebo.	Baseline and 6 months
Subject Satisfaction	Subjects very satisfied or somewhat satisfied with study treatment based upon Subject Questionnaire	6 months

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in Visible Surface Area	Visible surface area is defined as the longest dimension ("length") times the longest dimension perpendicular to length ("width"). Visible surface area will be analyzed as the percent change from baseline.	1 Month post injection
Change in Visible Surface Area	Visible surface area is defined as the longest dimension ("length") times the longest dimension perpendicular to length ("width"). Visible surface area will be analyzed as the percent change from baseline.	3 month post injection

Collaborators and Investigators

• Sponsor: Gerut, Zachary, M.D.
• Collaborators: Advance Biofactures Corporation
• Investigators: Principal Investigator: Zachary E Gerut, MD, Private Practice

Study record dates

Study Major Dates

• Study Start: August 1, 2014
• Primary Completion (Actual): April 1, 2016
• Study Completion (Actual): June 1, 2016

Study Registration Dates

• First Submitted: August 18, 2014
• First Submitted That Met QC Criteria: September 23, 2014
• First Posted (Estimate): September 25, 2014

Study Record Updates

• Last Update Posted (Actual): February 23, 2017
• Last Update Submitted That Met QC Criteria: January 6, 2017
• Last Verified: August 1, 2016

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Adipose Tissue; Lipoma
• Other Study ID Numbers: 10-Lipoma II

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Undecided