Lipoma
Conditions
Brief summary
The purpose of this study is to determine whether collagenase is effective in reducing the surface area of a subcutaneous benign lipoma.
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.
Interventions
DRUGAA4500
Subjects must present with 2 lipomas; one to receive AA4500 and one to receive placebo simultaneously
DRUGplacebo
Placebo
Sponsors
Advance Biofactures Corporation
Gerut, Zachary, M.D.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
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
* 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percent Change From Baseline in Surface Area of the Lipoma at Six Months | Baseline and 6 months post injection | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Subject Satisfaction | 6 months | Subjects very satisfied or somewhat satisfied with study treatment based upon Subject Questionnaire |
| Responder Analysis | Baseline and 6 months | The number of participants with at least 50% decrease in visible lipoma surface area of lipoma relative to baseline |
| Percent Change From Baseline in Greatest Dimension (Length) of Lipoma at 6 Months | Baseline and 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. |
Other
| Measure | Time frame | Description |
|---|---|---|
| Change in Visible Surface Area | 1 Month post injection | 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. |
Countries
United States
Participant flow
Recruitment details
Subjects were screened and enrolled at two study sites in the United States.
Pre-assignment details
Subjects must present with 2 lipomas meeting inclusion criteria
Participants by arm
| Arm | Count |
|---|---|
| AA4500 and Placebo single injection of 0.58 mg study drug and placebo
Subjects must present with 2 lipomas; one to receive AA4500 and one to receive placebo simultaneously | 19 |
| Total | 19 |
Baseline characteristics
| Characteristic | AA4500 and Placebo |
|---|---|
| Age, Continuous | 49.9 years STANDARD_DEVIATION 9.13 |
| Gender Female | 4 Participants |
| Gender Male | 15 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 1 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 18 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 19 / 19 | 3 / 19 |
| serious Total, serious adverse events | 0 / 19 | 0 / 19 |
Outcome results
Primary
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
Population: All 19 subjects enrolled with 2 lipomas. All received AA4500 in one lipoma and placebo in the other lipoma.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| AA4500 | Percent Change From Baseline in Surface Area of the Lipoma at Six Months | -81.277 percentage change from baseline | Standard Deviation 22.9 |
| Placebo | Percent Change From Baseline in Surface Area of the Lipoma at Six Months | 2.053 percentage change from baseline | Standard Deviation 12.1 |
p-value: <0.000195% CI: [-95.394, -71.265]t-test, 2 sided
Secondary
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
Population: All study participants
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| AA4500 | Percent Change From Baseline in Greatest Dimension (Length) of Lipoma at 6 Months | -64.1818 Percent change from baseline | Standard Deviation 28.97 |
| Placebo | Percent Change From Baseline in Greatest Dimension (Length) of Lipoma at 6 Months | 0.156 Percent change from baseline | Standard Deviation 7.78 |
Secondary
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
Population: All 19 participants
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| AA4500 | Responder Analysis | 17 participants |
| Placebo | Responder Analysis | 0 participants |
p-value: <0.000195% CI: [0.6686, 0.987]Sign test
Secondary
Subject Satisfaction
Subjects very satisfied or somewhat satisfied with study treatment based upon Subject Questionnaire
Time frame: 6 months
Population: 1 participant did not complete the questionnaire at the final visit; therefore the analysis population was based upon 18 participants
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| AA4500 | Subject Satisfaction | 18 participants satisfied |
| Placebo | Subject Satisfaction | 7 participants satisfied |
Other Pre-specified
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
Population: All 19 subjects enrolled with 2 lipomas. All received AA4500 in one lipoma and placebo in the other lipoma.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| AA4500 | Change in Visible Surface Area | -26.74 percentage change from baseline | Standard Deviation 35.57 |
| Placebo | Change in Visible Surface Area | 0.174 percentage change from baseline | Standard Deviation 8.38 |
Other Pre-specified
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
Population: All 19 subjects enrolled with 2 lipomas. All received AA4500 in one lipoma and placebo in the other lipoma.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| AA4500 | Change in Visible Surface Area | -62.51 percentage change from baseline | Standard Deviation 32.14 |
| Placebo | Change in Visible Surface Area | 0.402 percentage change from baseline | Standard Deviation 12.92 |