Treatment of Women With Lipedema Involving Substantial Fat Above the Knee or of Women and Men With Nodular Dercum's Disease

An Open Label, Phase 2a Clinical Trial for the Evaluation of Safety and Efficacy of RZL-012 for the Treatment of Women With Lipedema Involving Substantial Fat Above the Knee or of Women and Men With Nodular Dercum's Disease

This is an open label, 2 cohort, clinical trial in women with lipedema with substantial fat above the knee or women and men with nodular Dercum's disease. Each cohort will have 6 subjects who will receive RZL-012.

Study Overview

• Status: Completed
• Conditions: Dercum Disease; Lipedema
• Intervention / Treatment: Drug: RZL-012

Detailed Description

This is an open label, 2 cohort clinical trial in women with lipedema with substantial fat above the knee or women and men with nodular Dercum's disease. Each cohort will have 6 subjects who will receive RZL-012.

• The 1st cohort will be comprised of subjects with Dercum's disease
• The 2nd cohort will be comprised of subjects with lipedema with substantial fat above the knee.

Within each cohort, dosing of the subjects will progress consecutively from one individual to the other with a minimum of 7-days between subjects to assess safety. This study design will allow the physicians to monitor safety for at least 7 days prior to dosing the next subject. Cohort 2 will be conducted in a dose escalation manner and the decision to proceed to the next dose level will be made after reviewing all safety data collected by Day 14 within 2 ± 1d of the last dosed subject. The trial will proceed within a cohort provided that no more than one subject experiences intolerable side effects in a cohort, and based on the decision made by the Principal Investigator (PI) and the Medical Monitor.

• Study Type: Interventional
• Enrollment (Actual): 12
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Arizona
    • Tucson, Arizona, United States, 85724
      • University of Arizona Clinical and Translational Science Research Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 20 years to 65 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• 1. Post-menopausal (at least 2 years) women no more than 65 years old, with lipedema involving substantial fat above the knee or nodular Dercum's disease in such women and in men 20 - 65 years with nodular Dercum's disease.

  2. For Dercum's disease subjects - at least 2 nodules to be injected of at least 2cm diameter each, as determined by ultrasound 3. For lipedema subjects - Significant subcutaneous fat above the knee as determined by circumference of 50cm 4. Generally considered healthy according to medical history, physical examination, ECG and laboratory evaluation with a special emphasis on metabolic parameters (fasting glucose concentration < 200 mg, normal blood pressure).

  5. Subjects must be able to adhere to the visit schedule and protocol requirements and be available to complete the study.

  6. Subjects must sign an informed consent indicating that they are aware of the investigational nature of the study.

Exclusion Criteria:

• 1. Unable to tolerate subcutaneous injection. 2. Subjects with uncontrolled cardiac, hepatic, renal or neurologic/psychiatric disorders, that in the opinion of the investigator, put the subject at significant risk, are not eligible.

  3. Positive blood screen for Hepatitis B surface antigen (HbSAg), Hepatitis C virus (HCV), or Human immunodeficiency virus (HIV), or positive urine screen for alcohol or drugs of abuse (unless prescribed by a physician).

  4. Subjects with a clinical history of primary or secondary immunodeficiency, autoimmune disease or subjects taking immunosuppressive drugs such as corticosteroids are ineligible.

  5. As a result of medical review, physical examination, the PI (or medically qualified nominee) considers the subject unfit for the study.

  6. Known sensitivity to components of the injection formulation. 7. Prior wound, tattoo or infection in the treated area. 8. Prior invasive treatment such as surgery or injectable drug at the RZL-012 injected area.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Cohort 1 - Dercum's disease; Nodule size - diameter (cm) 2-2.9 3-3.9 4-8 Total Dose of RZL-012 (mg) 10 15 20 Dose per NOAEL* 1/25th 1/18.75th 1/12.5th Number of Injections 2 3 4	Drug: RZL-012; The dosing regimen will be a single dose treatment injection of RZL-012 in multiple sites.
Active Comparator: Cohort 2 - Lipedema; Total Dose RZL-012 (mg) 60 80 Dose per NOAEL* 1/4.688 1/3.125 Number of Injections 12 16	Drug: RZL-012; The dosing regimen will be a single dose treatment injection of RZL-012 in multiple sites.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The incidence of any Grade 3 or greater event intolerable side effect experienced in a cohort	Trial will proceed as long as no more than one subject experiences an intolerable side effect in a cohort	To 14 days

Collaborators and Investigators

• Sponsor: Raziel Therapeutics Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): June 28, 2018
• Primary Completion (Actual): May 2, 2019
• Study Completion (Actual): May 2, 2019

Study Registration Dates

• First Submitted: August 29, 2019
• First Submitted That Met QC Criteria: August 30, 2019
• First Posted (Actual): September 3, 2019

Study Record Updates

• Last Update Posted (Actual): September 6, 2019
• Last Update Submitted That Met QC Criteria: September 4, 2019
• Last Verified: September 1, 2019

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Skin Diseases; Connective Tissue Diseases; Lipid Metabolism Disorders; Lipomatosis; Lipedema; Adiposis Dolorosa
• Other Study ID Numbers: RZL-012-FD-P2aUS-001.7

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
• product manufactured in and exported from the U.S.: No