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CompletedNCT02249052Updated Feb 23, 2017Results posted

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

A Phase 2 interventional study of AA4500 and placebo in Lipoma, sponsored by Gerut, Zachary, M.D.. Completed at 2 sites in United States. Open to participants aged 18 Years to 75 Years, including healthy volunteers. Per ClinicalTrials.gov, last updated 2017-02-23.

Sponsored by Gerut, Zachary, M.D. · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
19
Allocation
Randomized
Ages
18 Years to 75 Years
Sex
All
01

Study summary

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

Read the 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.

02

Conditions studied

  • Lipoma

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03

Who can participate

Ages eligible
18 Years to 75 Years
Sexes eligible
All
Accepts healthy volunteers
Yes

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

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
04

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
19 participants (actual)

Study arms

  • Active comparator
    AA4500

    single injection of 0.58 mg study drug

    Drug: AA4500

  • Placebo comparator
    Placebo

    single injection of placebo

    Drug: placebo

Interventions

  • DrugAA4500

    Subjects must present with 2 lipomas; one to receive AA4500 and one to receive placebo simultaneously

    Also known as: XIAFLEX, Collagenase Clostridium Histolyticum

  • Drugplacebo

    Placebo

05

What researchers measure

Primary outcomes

  1. 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.

    Time frame: Baseline and 6 months post injection

Secondary outcomes

  1. Responder Analysis

    The number of participants with at least 50% decrease in visible lipoma surface area of lipoma relative to baseline

    Time frame: Baseline and 6 months

  2. 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.

    Time frame: Baseline and 6 months

  3. Subject Satisfaction

    Subjects very satisfied or somewhat satisfied with study treatment based upon Subject Questionnaire

    Time frame: 6 months

Other outcomes

  1. 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.

    Time frame: 1 Month post injection

  2. 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.

    Time frame: 3 month post injection

06

Results

Posted Dec 13, 2016

Participant flow

Subjects were screened and enrolled at two study sites in the United States.

Participant flow — Overall Study
MilestoneAA4500 and Placebo
Started19
Completed19
Not completed0

Outcome measures

PrimaryPercent 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.

Time frame:
Baseline and 6 months post injection
Reported as:
Mean · percentage change from baseline
Percent Change From Baseline in Surface Area of the Lipoma at Six Months
percentage change from baselineAA4500Placebo
Percent Change From Baseline in Surface Area of the Lipoma at Six Months-81.277 ± 22.92.053 ± 12.1
Statistical analysis
  • AA4500 vs Placebo · t-test, 2 sided · p = <0.0001 · Mean difference (final values): -83.330 · 95% CI -95.394 to -71.265
SecondaryResponder Analysis

The number of participants with at least 50% decrease in visible lipoma surface area of lipoma relative to baseline

Time frame:
Baseline and 6 months
Reported as:
Number · participants
Responder Analysis
participantsAA4500Placebo
Responder Analysis170
Statistical analysis
  • AA4500 vs Placebo · Sign test · p = <.0001 · Binomial proportion: .8947 · 95% CI .6686 to .9870
SecondaryPercent 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.

Time frame:
Baseline and 6 months
Reported as:
Mean · Percent change from baseline
Percent Change From Baseline in Greatest Dimension (Length) of Lipoma at 6 Months
Percent change from baselineAA4500Placebo
Percent Change From Baseline in Greatest Dimension (Length) of Lipoma at 6 Months-64.1818 ± 28.970.156 ± 7.78
SecondarySubject Satisfaction

Subjects very satisfied or somewhat satisfied with study treatment based upon Subject Questionnaire

Time frame:
6 months
Reported as:
Number · participants satisfied
Subject Satisfaction
participants satisfiedAA4500Placebo
Subject Satisfaction187
Other pre-specifiedChange 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.

Time frame:
1 Month post injection
Reported as:
Mean · percentage change from baseline
Change in Visible Surface Area
percentage change from baselineAA4500Placebo
Change in Visible Surface Area-26.74 ± 35.570.174 ± 8.38
Other pre-specifiedChange 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.

Time frame:
3 month post injection
Reported as:
Mean · percentage change from baseline
Change in Visible Surface Area
percentage change from baselineAA4500Placebo
Change in Visible Surface Area-62.51 ± 32.140.402 ± 12.92

Adverse events

Collected over 6 months post administration study medication. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
AA4500—0/19 (0%)19/19 (100%)
Placebo—0/19 (0%)3/19 (15.8%)
Most frequent other events
Most frequent other events
EventAA4500Placebo
Injection site BruisingGeneral disorders17/193/19
Injection site swellingGeneral disorders15/190/19
Injection site painGeneral disorders10/190/19
Injection site pruritisGeneral disorders4/190/19
Injection site haemorrhageGeneral disorders2/190/19
Skin disorderSkin and subcutaneous tissue disorders2/190/19
Injection site discolourationGeneral disorders1/190/19
Muscle spasmsMusculoskeletal and connective tissue disorders1/190/19
Musculoskeletal stiffnessMusculoskeletal and connective tissue disorders1/190/19

Baseline characteristics

Age, Continuous
Age, Continuous(years)AA4500 and Placebo
Mean49.9 ± 9.13
Gender
Gender(Participants)AA4500 and Placebo
Female4
Male15
Race (NIH/OMB)
Race (NIH/OMB)(Participants)AA4500 and Placebo
American Indian or Alaska Native0
Asian0
Native Hawaiian or Other Pacific Islander0
Black or African American1
White18
More than one race0
Unknown or Not Reported0
07

Study locations

2 sites
  • Aesthetic Center
    Hewlett, New York 11557, United States
  • Vanderbilt University
    Nashville, Tennessee 37232, United States
08

References and documents

Individual participant data

Plan to share: Undecided

No publications or documents are linked to this record.

09

Registry details

Key details

Study ID
NCT02249052
Lead sponsor
Gerut, Zachary, M.D.
Collaborators
Advance Biofactures Corporation
Responsible party
Sponsor
First posted
Sep 25, 2014
Start date
Aug 2014
Primary completion
Apr 2016
Completion
Jun 2016
Results posted
Dec 13, 2016
Last update
Feb 23, 2017

Study contacts

Zachary E Gerut, MD
principal investigator · Private Practice

Oversight

Data monitoring committee
No
View the source record on ClinicalTrials.gov ↗

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