Phase 2 Study for the Treatment of Superficial Lipomas

Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study of the Safety and Efficacy of ATX-101 (Sodium Deoxycholate for Injection) Intralipomal Injections for the Treatment of Superficial Lipomas

The purpose of this research is to compare the safety and effectiveness of 3 different concentrations of deoxycholic acid for injection against a placebo in the treatment of superficial lipomas.

Study Overview

• Status: Completed
• Conditions: Lipoma
• Intervention / Treatment: Drug: Deoxycholic Acid Injection; Drug: Placebo

Detailed Description

A lipoma is a fatty lump typically located on the trunk, shoulder, arms, or legs. For the purposes of this study, only lipomas on the trunk, arms, legs, or neck were treated. (Lipomas on the face, wrists, hands, lower portion of the spine, genitals, ankles, or feet were not treated.)

• Study Type: Interventional
• Enrollment (Actual): 62
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Alabama
    • Birmingham, Alabama, United States, 35205
      • Gary D. Monheit, M.D.
  • California
    • San Diego, California, United States, 92123
      • Stacy R. Smith
  • Michigan
    • Warren, Michigan, United States, 48088
      • Steven Grekin, D.O.
  • Nebraska
    • Omaha, Nebraska, United States, 68144
      • Joel Schlessinger, M.D.
  • New Jersey
    • Westwood, New Jersey, United States, 07675
      • David J. Goldberg, M.D.
  • New York
    • New York, New York, United States, 10021
      • Neil S. Sadick, M.D.
  • Tennessee
    • Nashville, Tennessee, United States, 37215
      • Michael H. Gold, M.D.

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years to 63 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• One or more lipomas, based on clinical and histological diagnosis, which are accessible for treatment and assessment, are quantifiable along at least 2 perpendicular diameters, and have the following characteristics:

  • History of slow growth followed by dormancy, and stable for at least 6 months
  • Greatest length multiplied by greatest perpendicular width between 1 and 16 cm², inclusive
  • Discrete, oval to rounded in shape, not hard or attached to underlying tissue
  • Without changes in overlying skin (ie, inflammation, pain or tenderness, hyperpigmentation)
  • Located on the trunk, arms, legs, or neck
• Signed informed consent.

Exclusion Criteria:

• Absence of significant medical conditions that could affect safety
• History of surgical or deoxycholate treatment for lipomas
• Treatment with an investigational agent within 30 days before ATX-101 treatment

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Deoxycholic Acid 1%; Participants received 1.0% deoxycholic acid administered at a volume dependent on the size of the lipoma, up to a maximum of 4.8 mL per treatment session, at 28-day intervals for up to a maximum of 4 treatments.	Drug: Deoxycholic Acid Injection; Administered via intralipomal injection.; Other Names: ATX-101; sodium deoxycholate
Experimental: Deoxycholic Acid 2%; Participants received 2.0% deoxycholic acid administered at a volume dependent on the size of the lipoma, up to a maximum of 4.8 mL per treatment session, at 28-day intervals for up to a maximum of 4 treatments.	Drug: Deoxycholic Acid Injection; Administered via intralipomal injection.; Other Names: ATX-101; sodium deoxycholate
Experimental: Deoxycholic Acid 4%; Participants received 4.0% deoxycholic acid administered at a volume dependent on the size of the lipoma, up to a maximum of 4.8 mL per treatment session, at 28-day intervals for up to a maximum of 4 treatments.	Drug: Deoxycholic Acid Injection; Administered via intralipomal injection.; Other Names: ATX-101; sodium deoxycholate
Placebo Comparator: Placebo; Participants received matching vehicle placebo administered at a volume dependent on the size of the lipoma, up to a maximum of 4.8 mL per treatment session, at 28-day intervals for up to a maximum of 4 treatments.	Drug: Placebo; Matching vehicle placebo administered via intralipomal injection.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants With Adverse Events (AEs)	Severity of AEs was determined using the following scale: Mild: The participant was aware of a sign or symptom, but it was easily tolerated; Moderate: Discomfort or interference with usual activity; Severe: Incapacitating, with inability to engage in usual activity. The investigator determined the relationship of each AE to the administration of study material by answering the question: "Was there a reasonable possibility that the event may have been caused by treatment with study material?" A serious AE was an event that constituted a significant medical hazard or side effect, regardless of the investigator's or sponsor's opinion regarding relatedness to study material. Serious AEs included any event that was fatal or life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect or other significant medical hazard.	Up to 24 weeks
Number of Participants With Newly Occurring or Worsening Biochemistry/Hematology/Urinalysis Abnormalities	An abnormality is defined as a value outside the limits of the expanded normal range/notable range.	24 weeks
Number of Participants With Clinically Significant Changes in Vital Signs or Weight		Up to 24 weeks
Number of Participants With Positive Histopathology Results at Screening	A needle core tissue sample biopsy was performed at screening for all treated lipomas.	Screening (prior to randomization)
Number of Participants With Positive Histopathology Results at Week 20	After the completion of all tests and procedures scheduled for week 20, participants with treated lipomas that remained palpable could have their treated lipomas excised.	Week 20

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Participants With Complete Clearance or ≥ 75% Clearance	At randomization 1 to 3 lipomas were selected for treatment. Lipomas were measured in 3 dimensions (longest length, perpendicular width, and height if possible) using digital calipers. Complete clearance indicates target lipoma(s) not present or detectable, and ≥ 75% clearance is defined as a ≥ 75% reduction from baseline in the area of target lipoma(s). For participants with > 1 target lipoma, the total area of all target lipomas was used in the calculation of response.	Baseline and week 20 (8 weeks after last dose)
Percent Change From Baseline in the Sum of the Areas of All Treated Lipomas	Percent change from baseline was calculated as the baseline total lipoma area - postbaseline total lipoma area / baseline total lipoma area * 100. A positive change indicates a reduction in size.	Baseline and week 12 (last treatment session), week 16 (4 weeks after last treatment), and week 20 (8 weeks after last treatment)

Collaborators and Investigators

• Sponsor: Kythera Biopharmaceuticals
• Investigators: Study Director: Patricia S. Walker, M.D., Ph.D., Kythera Biopharmaceuticals, Inc.

Study record dates

Study Major Dates

• Study Start: November 1, 2007
• Primary Completion (Actual): August 1, 2008
• Study Completion (Actual): January 1, 2010

Study Registration Dates

• First Submitted: January 23, 2008
• First Submitted That Met QC Criteria: February 5, 2008
• First Posted (Estimate): February 6, 2008

Study Record Updates

• Last Update Posted (Estimate): January 14, 2016
• Last Update Submitted That Met QC Criteria: December 8, 2015
• Last Verified: December 1, 2015

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Adipose Tissue; Lipoma; Gastrointestinal Agents; Cholagogues and Choleretics; Deoxycholic Acid
• Other Study ID Numbers: ATX-101-07-05