RZL-012 for Dercum's Disease Lipomas

A Double Blind, Randomized, Multi-Center, Placebo-Controlled Phase 2B Clinical Trial for the Evaluation of Efficacy and Safety of RZL-012 in Subjects Having Dercum's Disease Lipomas

Thirty-eight (38) subjects will be included in the study. Subjects will be randomized in a ration of 1:1 to be treated with RZL-012 or placebo. Subjects will be injected with a different doses of RZL-012 according to their lipomas sizes. The total of 38 subjects will be enrolled in 3 clinical sites.

Study Overview

• Status: Completed
• Conditions: Dercum's Disease Lipomas
• Intervention / Treatment: Drug: RZL-012; Drug: Vehicle

Detailed Description

This is a multi-center, randomized, double blind, placebo controlled clinical trial in DD subjects having lipomas. Subjects will be randomized, in a 1:1 ratio, into two groups injected with either RZL-012 or vehicle.

Once the study ends and codes are opened, 84 days after dosing, placebo-treated subjects will be offered the option of receiving treatment with RZL-012 and followed up for an additional 84 days.

At least 4 lipomas/nodules, preferably 6, and no more than 8, will be injected per subject, two of which will be medium (4-5.9 cm), large (6-7.9 cm) or extra-large (8-10 cm) in diameter. Dosing will be according to lipoma size, where the total injected dose will not exceed 240 mg per patient (48 injections at 5mg/injection).

• Study Type: Interventional
• Enrollment (Actual): 38
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • California
    • Los Angeles, California, United States, 90025
      • Limitless Therapeutics

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years to 68 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• At least 4 painful lipomas of appropriate size to be injected on a background of Dercum's Disease.
• Generally considered healthy according to medical history, physical examination, electrocardiogram (ECG) and laboratory evaluation with an emphasis on metabolic parameters (fasting glucose concentration < 200 mg/dL).
• Subjects must be able to adhere to the visit schedule and protocol requirements and be capable of completing the study.
• Males or females in the age of fertility are willing to refrain from sexual activity or agree to use a double-barrier contraceptive device (e.g., condom and spermicide) for 4 weeks after treatment with RZL 012.
• Subjects must sign an informed consent indicating they are aware of the investigational nature of the study.

Exclusion Criteria:

• Unable to tolerate subcutaneous injections.
• Pregnant women.
• Subjects with uncontrolled cardiac, hepatic, renal or neurologic/psychiatric disorders, that in the opinion of the investigator places the subject at significant risk.
• Positive blood screen for Hepatitis B surface antigen (HbSAg), Hepatitis C virus (HCV), or Human immunodeficiency virus (HIV).
• Subjects with a clinical history of active primary or secondary immunodeficiency, autoimmune disease or subjects taking immunosuppressive drugs such as corticosteroids
• Subjects with dysfunctional gallbladder activity, e.g. underwent cholecystectomy or cholecystitis.
• As a result of medical review and physical examination, the PI (or medically qualified nominee) considers the subject unfit for the study.
• Known sensitivity to components of the injection formulation.
• Prior wound, tattoo or infection in the treated area.
• Prior invasive treatment such as surgery or injectable drug at the RZL-012 injected area.
• Subjects treated chronically at least 3 months prior to study entry with systemic steroids or immunosuppressive drugs.
• Subjects treated chronically at least one week prior to study entry with Non-Steroidal Anti-Inflammatory Drugs (NSAIDs).
• Current participation or participation within 3 months prior to the start of this study in a drug or other investigational research study.

Study Plan

How is the study designed?

Design Details

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

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: RZL-012; A single-treatment injection, multiple subcutaneous injections of RZL-012 administered into 4-8 lipomas in each subject, preferably 6. Dosing will be done according to lipomas size, as will be determined by Ultrasound. Each injection contains 0.1mL RZL-012 (5mg). Lipomas in the size of: 2-3.9 cm will be dozed with 0.4mL RZL-012 (20mg). 4-5.9 cm will be dozed with 0.8mL RZL-012 (80mg). 6-7.9 cm will be dozed with 1 mL RZL-012 (100mg). 8-10 cm will be dozed with 1.2 mL RZL-012 (120mg).	Drug: RZL-012; small synthetic molecule for the treatment of Dercum's Disease lipomas
Placebo Comparator: Vehicle of RZL-012; A single-treatment injection, multiple subcutaneous injections of vehicle administered into 4-8 lipomas in each subject, preferably 6. Dosing will be done according to lipomas size, as will be determined by Ultrasound. Each injection contains 0.1mL vehicle. Lipomas in the size of: 2-3.9 cm will be dozed with 0.4mL vehicle. 4-5.9 cm will be dozed with 0.8mL vehicle. 6-7.9 cm will be dozed with 1 mL vehicle. 8-10 cm will be dozed with 1.2 mL vehicle.	Drug: Vehicle; Vehicle of RZL-012 drug product; Other Names: placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Efficacy - Change in Lipomas Dimensions	Evaluation of the efficacy of RZL-012 following injection into lipomas/nodules of Dercum's Disease (DD) subjects. Efficacy will be determined by ultrasound assessment of the lipoma/nodule dimensions after treatment vs baseline. Lipomas Dimensions will be measured for active vs. placebo at days 28,56,84	0-84 days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Key Secondary Outcome - Efficacy - Improvement in Pain Assessment of Individual Lipomas	Assessment of lipoma/nodule associated pain using the Comparative Pain Scale. 0 is "no pain" and 10 is "worst pain". Pain measured for active vs. placebo at days 28,56,84	0-84 days
Safety Number of Participants With One or More Abnormal Laboratory Values up to Day 28	Safety will be assessed by by change-from-baseline values for clinical laboratory tests. measured for active vs. placebo at days 1 and 28 following injection. Number of subjects with abnormal laboratory values will be compared in both study arms.	0-28 days

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Exploratory	Improvement in Quality of Life - QOL questionnaire. Improvement will be measured for active vs. placebo at Day 0 and Day 84 following treatment.	0-84 Days

Collaborators and Investigators

• Sponsor: Raziel Therapeutics Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): July 30, 2020
• Primary Completion (Actual): February 20, 2021
• Study Completion (Actual): February 20, 2021

Study Registration Dates

• First Submitted: January 9, 2020
• First Submitted That Met QC Criteria: January 13, 2020
• First Posted (Actual): January 14, 2020

Study Record Updates

• Last Update Posted (Actual): February 7, 2024
• Last Update Submitted That Met QC Criteria: January 15, 2024
• Last Verified: January 1, 2020

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Skin Diseases; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Lipid Metabolism Disorders; Neoplasms, Adipose Tissue; Lipomatosis; Adiposis Dolorosa; Lipoma
• Other Study ID Numbers: RZL-012-DD-P2bUS-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No