Trial Outcomes & Findings for Double Blind Study to Evaluate the Efficacy of Collagenase Histolyticum in the Treatment of Lipoma (NCT NCT02249052)
NCT ID: NCT02249052
Last Updated: 2017-02-23
Results Overview
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.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
19 participants
Primary outcome timeframe
Baseline and 6 months post injection
Results posted on
2017-02-23
Participant Flow
Subjects were screened and enrolled at two study sites in the United States.
Subjects must present with 2 lipomas meeting inclusion criteria
Participant milestones
| Measure |
AA4500 and Placebo
Each subject received a single injection of 1 mL containing 0.58 mg AA4500 in one lipoma and a single injection of 1 mL placebo in another lipoma
Lipoma #1 was randomized to AA4500 or placebo with Lipoma #2 receiving the alternate intervention
|
|---|---|
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Overall Study
STARTED
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19
|
|
Overall Study
COMPLETED
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19
|
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Overall Study
NOT COMPLETED
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0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Double Blind Study to Evaluate the Efficacy of Collagenase Histolyticum in the Treatment of Lipoma
Baseline characteristics by cohort
| Measure |
AA4500 and Placebo
n=19 Participants
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
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|---|---|
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Age, Continuous
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49.9 years
STANDARD_DEVIATION 9.13 • n=99 Participants
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|
Gender
Female
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4 Participants
n=99 Participants
|
|
Gender
Male
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15 Participants
n=99 Participants
|
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Race (NIH/OMB)
American Indian or Alaska Native
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0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Asian
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0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
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0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Black or African American
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1 Participants
n=99 Participants
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Race (NIH/OMB)
White
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18 Participants
n=99 Participants
|
|
Race (NIH/OMB)
More than one race
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0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
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PRIMARY outcome
Timeframe: Baseline and 6 months post injectionPopulation: All 19 subjects enrolled with 2 lipomas. All received AA4500 in one lipoma and placebo in the other lipoma.
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.
Outcome measures
| Measure |
AA4500
n=19 Participants
Each subject received a single injection of 1 mL containing 0.58 mg AA4500
|
Placebo
n=19 Participants
Each subject received a single injection of 1 mL placebo
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|---|---|---|
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Percent Change From Baseline in Surface Area of the Lipoma at Six Months
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-81.277 percentage change from baseline
Standard Deviation 22.9
|
2.053 percentage change from baseline
Standard Deviation 12.1
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SECONDARY outcome
Timeframe: Baseline and 6 monthsPopulation: All 19 participants
The number of participants with at least 50% decrease in visible lipoma surface area of lipoma relative to baseline
Outcome measures
| Measure |
AA4500
n=19 Participants
Each subject received a single injection of 1 mL containing 0.58 mg AA4500
|
Placebo
n=19 Participants
Each subject received a single injection of 1 mL placebo
|
|---|---|---|
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Responder Analysis
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17 participants
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0 participants
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SECONDARY outcome
Timeframe: Baseline and 6 monthsPopulation: All study participants
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.
Outcome measures
| Measure |
AA4500
n=19 Participants
Each subject received a single injection of 1 mL containing 0.58 mg AA4500
|
Placebo
n=19 Participants
Each subject received a single injection of 1 mL placebo
|
|---|---|---|
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Percent Change From Baseline in Greatest Dimension (Length) of Lipoma at 6 Months
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-64.1818 Percent change from baseline
Standard Deviation 28.97
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0.156 Percent change from baseline
Standard Deviation 7.78
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SECONDARY outcome
Timeframe: 6 monthsPopulation: 1 participant did not complete the questionnaire at the final visit; therefore the analysis population was based upon 18 participants
Subjects very satisfied or somewhat satisfied with study treatment based upon Subject Questionnaire
Outcome measures
| Measure |
AA4500
n=18 Participants
Each subject received a single injection of 1 mL containing 0.58 mg AA4500
|
Placebo
n=18 Participants
Each subject received a single injection of 1 mL placebo
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|---|---|---|
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Subject Satisfaction
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18 participants satisfied
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7 participants satisfied
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OTHER_PRE_SPECIFIED outcome
Timeframe: 1 Month post injectionPopulation: All 19 subjects enrolled with 2 lipomas. All received AA4500 in one lipoma and placebo in the other lipoma.
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.
Outcome measures
| Measure |
AA4500
n=19 Participants
Each subject received a single injection of 1 mL containing 0.58 mg AA4500
|
Placebo
n=19 Participants
Each subject received a single injection of 1 mL placebo
|
|---|---|---|
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Change in Visible Surface Area
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-26.74 percentage change from baseline
Standard Deviation 35.57
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0.174 percentage change from baseline
Standard Deviation 8.38
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OTHER_PRE_SPECIFIED outcome
Timeframe: 3 month post injectionPopulation: All 19 subjects enrolled with 2 lipomas. All received AA4500 in one lipoma and placebo in the other lipoma.
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.
Outcome measures
| Measure |
AA4500
n=19 Participants
Each subject received a single injection of 1 mL containing 0.58 mg AA4500
|
Placebo
n=19 Participants
Each subject received a single injection of 1 mL placebo
|
|---|---|---|
|
Change in Visible Surface Area
|
-62.51 percentage change from baseline
Standard Deviation 32.14
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0.402 percentage change from baseline
Standard Deviation 12.92
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Adverse Events
AA4500
Serious events: 0 serious events
Other events: 19 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
AA4500
n=19 participants at risk
single injection of 0.58 mg study drug
AA4500: Subjects must present with 2 lipomas; one to receive AA4500 and one to receive placebo simultaneously
|
Placebo
n=19 participants at risk
single injection of placebo
placebo: Placebo
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|---|---|---|
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General disorders
Injection site Bruising
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89.5%
17/19 • Number of events 17 • 6 months post administration study medication
Treatment emergent adverse events were collected after the date of administration of study medication through the last visit at 6 months post administration.
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15.8%
3/19 • Number of events 3 • 6 months post administration study medication
Treatment emergent adverse events were collected after the date of administration of study medication through the last visit at 6 months post administration.
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General disorders
Injection site swelling
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78.9%
15/19 • Number of events 15 • 6 months post administration study medication
Treatment emergent adverse events were collected after the date of administration of study medication through the last visit at 6 months post administration.
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0.00%
0/19 • 6 months post administration study medication
Treatment emergent adverse events were collected after the date of administration of study medication through the last visit at 6 months post administration.
|
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General disorders
Injection site pain
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52.6%
10/19 • Number of events 10 • 6 months post administration study medication
Treatment emergent adverse events were collected after the date of administration of study medication through the last visit at 6 months post administration.
|
0.00%
0/19 • 6 months post administration study medication
Treatment emergent adverse events were collected after the date of administration of study medication through the last visit at 6 months post administration.
|
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General disorders
Injection site pruritis
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21.1%
4/19 • Number of events 4 • 6 months post administration study medication
Treatment emergent adverse events were collected after the date of administration of study medication through the last visit at 6 months post administration.
|
0.00%
0/19 • 6 months post administration study medication
Treatment emergent adverse events were collected after the date of administration of study medication through the last visit at 6 months post administration.
|
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General disorders
Injection site haemorrhage
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10.5%
2/19 • Number of events 2 • 6 months post administration study medication
Treatment emergent adverse events were collected after the date of administration of study medication through the last visit at 6 months post administration.
|
0.00%
0/19 • 6 months post administration study medication
Treatment emergent adverse events were collected after the date of administration of study medication through the last visit at 6 months post administration.
|
|
General disorders
Injection site discolouration
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5.3%
1/19 • Number of events 1 • 6 months post administration study medication
Treatment emergent adverse events were collected after the date of administration of study medication through the last visit at 6 months post administration.
|
0.00%
0/19 • 6 months post administration study medication
Treatment emergent adverse events were collected after the date of administration of study medication through the last visit at 6 months post administration.
|
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Musculoskeletal and connective tissue disorders
Muscle spasms
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5.3%
1/19 • Number of events 1 • 6 months post administration study medication
Treatment emergent adverse events were collected after the date of administration of study medication through the last visit at 6 months post administration.
|
0.00%
0/19 • 6 months post administration study medication
Treatment emergent adverse events were collected after the date of administration of study medication through the last visit at 6 months post administration.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal stiffness
|
5.3%
1/19 • Number of events 1 • 6 months post administration study medication
Treatment emergent adverse events were collected after the date of administration of study medication through the last visit at 6 months post administration.
|
0.00%
0/19 • 6 months post administration study medication
Treatment emergent adverse events were collected after the date of administration of study medication through the last visit at 6 months post administration.
|
|
Skin and subcutaneous tissue disorders
Skin disorder
|
10.5%
2/19 • Number of events 2 • 6 months post administration study medication
Treatment emergent adverse events were collected after the date of administration of study medication through the last visit at 6 months post administration.
|
0.00%
0/19 • 6 months post administration study medication
Treatment emergent adverse events were collected after the date of administration of study medication through the last visit at 6 months post administration.
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Additional Information
Dr. Zachary E. Gerut, Sponsor/Investigator
Dr. Zachary E. Gerut
Phone: 516 295-2100
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 30 days from the time submitted to the sponsor for review. The sponsor cannot require changes to the communication and cannot extend the embargo.
- Publication restrictions are in place
Restriction type: OTHER