RZL-012 for Dercum’s Disease Lipomas

Overview

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.

Full Title of Study: “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”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Randomized
    • Intervention Model: Parallel Assignment
    • Primary Purpose: Treatment
    • Masking: Double (Participant, Investigator)
  • Study Primary Completion Date: June 30, 2021

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).

Interventions

  • Drug: RZL-012
    • small synthetic molecule for the treatment of Dercum’s Disease lipomas
  • Drug: Vehicle
    • Vehicle of RZL-012 drug product

Arms, Groups and Cohorts

  • 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).
  • 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.

Clinical Trial Outcome Measures

Primary Measures

  • Efficacy – Change in lipomas dimensions
    • Time Frame: 0-84 days
    • 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

Secondary Measures

  • Key secondary outcome – efficacy – Improvement in pain assessment of individual lipomas
    • Time Frame: 0-84 days
    • 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
  • Safety change from baseline in clinical laboratory tests
    • Time Frame: 0-28 days
    • 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.

Participating in This Clinical Trial

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.

Gender Eligibility: All

Minimum Age: 18 Years

Maximum Age: 70 Years

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Raziel Therapeutics Ltd.
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Karen Herbst, MD, Principal Investigator, Arizona
  • Overall Contact(s)
    • Racheli Gueta, PhD, 972-8-9126941, racheli@raziel-therapy.com

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