A Dose Escalation Study Using Collagenase Clostridium Histolyticum in the Treatment of Lipoma

The purpose of this research is to evaluate the safest and most effective dose of a Food and Drug Administration (FDA) approved drug (XIAFLEX) in the treatment of lipoma (fatty tumors of varying sizes that occur commonly in the adult population). 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.

Study Overview

• Status: Completed
• Conditions: Lipoma
• Intervention / Treatment: Drug: Collagenase Clostridium Histolyticum

Detailed Description

Lipomas are common mesenchymal, benign, fatty tumors of varying sizes that occur in the general adult population. They usually present as painful or annoying lumps that are palpable and often visible in the subcutaneous tissue. Many subcutaneous lipomas are asymptomatic and are removed for non-medical reasons. However, they may cause the subject pain or discomfort or interfere with normal activity.

Spontaneous remission of lipomas has not been reported. A lipoma, once it presents itself, remains there for the lifetime of a person who carries it and may stay small or become larger. Alternative treatments of the lipoma are surgery or liposuction. If a large excision is performed there is the problem of having a very large scar and the accompanying issues of healing a large scar, or possible formation of a hematoma followed by consolidation of the hematoma and the remnants of a mass of scar tissue. This is often painful and more problematic than the original lipoma. liposuction also has the potential for hematoma formation followed by scar mass residual.

To avoid the complications listed above, a non-invasive method of treatment, such as enzyme mediated disassociation of adipose tissue, would be desirable. The primary aim of this study is to perform a step-wise approach in dosing to evaluate the safety and efficacy of clostridium histolyticum as a non-operative treatment for lipoma.

• Study Type: Interventional
• Enrollment (Actual): 14
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • New York
    • Hewlett, New York, United States, 11557
      • Zachary E. Gerut, M.D.

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:

• Clinical history lipoma at least one year and no treatment within last 90 days
• Lipoma diagnosed as benign
• Lipoma is a single mass with easily definable edges
• Lipoma has an area of 2.0 to 17 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
• Subjects who have participated in any investigational drug trial within 45 days of enrollment in this study
• Subjects with known allergy to collagenase or any of the inactive ingredients in XIAFLEX
• Subjects with diabetes or any medical condition that would make the subject unsuitable for enrollment.
• Subjects having a history of trauma in the area of the lipoma
• Subjects with a history of connective tissue diseases or rheumatological diseases.
• Subjects with uncontrolled hypertension
• Subjects currently receiving or planning to receive anticoagulant medication except for less than 150 mg aspirin daily and over-the-counter nonsteroidal anti-inflammatory drugs) within 7 days before injection of study drug
• Subjects having the following laboratory abnormalities:
• ALT/AST greater than 3 times the upper limit of normal
• Creatinine greater than 2
• Hemoglobin/hematocrit and WBC outside the normal range

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Dose #1; single injection 0.058 mg Collagenase Clostridium Histolyticum	Drug: Collagenase Clostridium Histolyticum; This is a dose escalation study in which subjects will sequentially receive single injections of 0.058 mg, 0.15 mg, 0.29 mg, 0.44 mg.; Other Names: XIAFLEX
Experimental: Dose #2; single injection 0.15 mg Collagenase Clostridium Histolyticum	Drug: Collagenase Clostridium Histolyticum; This is a dose escalation study in which subjects will sequentially receive single injections of 0.058 mg, 0.15 mg, 0.29 mg, 0.44 mg.; Other Names: XIAFLEX
Experimental: Dose #3; single injection 0.29 mg Collagenase Clostridium Histolyticum	Drug: Collagenase Clostridium Histolyticum; This is a dose escalation study in which subjects will sequentially receive single injections of 0.058 mg, 0.15 mg, 0.29 mg, 0.44 mg.; Other Names: XIAFLEX
Experimental: Dose #4; single injection 0.44 mg Collagenase Clostridium Histolyticum	Drug: Collagenase Clostridium Histolyticum; This is a dose escalation study in which subjects will sequentially receive single injections of 0.058 mg, 0.15 mg, 0.29 mg, 0.44 mg.; Other Names: XIAFLEX

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Relative Change in Volume of the Lipoma	A secondary outcome is the relative change in volume of the lipoma as determined by MRI. This outcome will be analyzed as the change from baseline to 6 months post injection.	Baseline and 6 months post injection of study drug

Collaborators and Investigators

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

Study record dates

Study Major Dates

• Study Start: May 1, 2012
• Primary Completion (Actual): September 1, 2013
• Study Completion (Actual): October 1, 2013

Study Registration Dates

• First Submitted: June 5, 2012
• First Submitted That Met QC Criteria: June 5, 2012
• First Posted (Estimate): June 7, 2012

Study Record Updates

• Last Update Posted (Estimate): October 6, 2016
• Last Update Submitted That Met QC Criteria: August 25, 2016
• Last Verified: August 1, 2016

More Information

Terms related to this study

• Keywords: Lipoma; Fatty Tumor
• Additional Relevant MeSH Terms: Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Adipose Tissue; Lipoma
• Other Study ID Numbers: Lipoma DE/01

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No