Lipoma
Conditions
Keywords
Lipoma, Fatty Tumor
Brief summary
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.
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.
Interventions
DRUGCollagenase 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.
Sponsors
Advance Biofactures Corporation
Zachary Gerut
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in Visible Surface Area of the Lipoma | Baseline and Six months post injection of study drug | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Relative Change in Volume of the Lipoma | Baseline and 6 months post injection of study drug | 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. |
Countries
United States
Participant flow
Recruitment details
Subjects were screened and enrolled at 1 site in the United States
Participants by arm
| Arm | Count |
|---|---|
| Dose #1 single injection 0.058 mg Collagenase Clostridium Histolyticum
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. | 2 |
| Dose #2 single injection 0.15 mg Collagenase Clostridium Histolyticum
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. | 4 |
| Dose #3 single injection 0.29 mg Collagenase Clostridium Histolyticum
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. | 4 |
| Dose #4 single injection 0.44 mg Collagenase Clostridium Histolyticum
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. | 4 |
| Total | 14 |
Baseline characteristics
| Characteristic | Dose #1 | Dose #2 | Dose #3 | Dose #4 | Total |
|---|---|---|---|---|---|
| Age, Continuous | 48.0 Years STANDARD_DEVIATION 5.66 | 51.5 Years STANDARD_DEVIATION 12.5 | 41.3 Years STANDARD_DEVIATION 11.09 | 57.0 Years STANDARD_DEVIATION 4.97 | 49.6 Years STANDARD_DEVIATION 10.6 |
| Region of Enrollment United States | 2 participants | 4 participants | 4 participants | 4 participants | 14 participants |
| Sex: Female, Male Female | 2 Participants | 3 Participants | 3 Participants | 1 Participants | 9 Participants |
| Sex: Female, Male Male | 0 Participants | 1 Participants | 1 Participants | 3 Participants | 5 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk |
|---|---|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — | — / — | — / — |
| other Total, other adverse events | 1 / 2 | 3 / 4 | 3 / 4 | 4 / 4 |
| serious Total, serious adverse events | 0 / 2 | 0 / 4 | 0 / 4 | 0 / 4 |
Outcome results
Primary
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.
Time frame: Baseline and Six months post injection of study drug
Population: Analysis population consists of all subjects who were enrolled and received the administration of study drug.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Dose #1 | Change in Visible Surface Area of the Lipoma | -31.69 Percent change from baseline | Standard Deviation 80.83 |
| Dose #2 | Change in Visible Surface Area of the Lipoma | -47.57 Percent change from baseline | Standard Deviation 23.48 |
| Dose #3 | Change in Visible Surface Area of the Lipoma | -52.42 Percent change from baseline | Standard Deviation 50.61 |
| Dose #4 | Change in Visible Surface Area of the Lipoma | -66.60 Percent change from baseline | Standard Deviation 17.82 |
Comparison: This is a proof of concept dose escalation study. No power justification has been implemented. Descriptive data provided for each arm.
Secondary
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.
Time frame: Baseline and 6 months post injection of study drug
Population: Analysis is based on the population that consists of all subjects who are enrolled and received administration of study drug
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Dose #1 | Relative Change in Volume of the Lipoma | -54.16 Percent reduction from baseline | Standard Deviation 34.3 |
| Dose #2 | Relative Change in Volume of the Lipoma | -62.33 Percent reduction from baseline | Standard Deviation 30.39 |
| Dose #3 | Relative Change in Volume of the Lipoma | -11.35 Percent reduction from baseline | Standard Deviation 32.12 |
| Dose #4 | Relative Change in Volume of the Lipoma | -34.75 Percent reduction from baseline | Standard Deviation 24.21 |
Comparison: This is a proof of concept dose escalation study. No power of justification was implemented. Descriptive statistics was provided for each arm only.