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

Primary Purpose

Dercum Disease, Lipedema

Status

Completed

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

RZL-012

About this trial

This is an interventional treatment trial for Dercum Disease

Eligibility Criteria

20 Years - 65 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

University of Arizona Clinical and Translational Science Research Center

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Arm Label

Cohort 1 - Dercum's disease

Cohort 2 - Lipedema

Arm Description

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

Total Dose RZL-012 (mg) 60 80 Dose per NOAEL* 1/4.688 1/3.125 Number of Injections 12 16

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

Full Information

NCT ID

NCT04076891

First Posted

August 29, 2019

Last Updated

September 4, 2019

Sponsor

Raziel Therapeutics Ltd.

1. Study Identification

Unique Protocol Identification Number

NCT04076891

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

September 2019

Overall Recruitment Status

Completed

Study Start Date

June 28, 2018 (Actual)

Primary Completion Date

May 2, 2019 (Actual)

Study Completion Date

May 2, 2019 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Raziel Therapeutics Ltd.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

Brief Summary

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.

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Dercum Disease, Lipedema

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

12 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Cohort 1 - Dercum's disease

Arm Type

Active Comparator

Arm Description

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

Arm Title

Cohort 2 - Lipedema

Arm Type

Active Comparator

Arm Description

Total Dose RZL-012 (mg) 60 80 Dose per NOAEL* 1/4.688 1/3.125 Number of Injections 12 16

Intervention Type

Drug

Intervention Name(s)

RZL-012

Intervention Description

The dosing regimen will be a single dose treatment injection of RZL-012 in multiple sites.

Primary Outcome Measure Information:

Title

The incidence of any Grade 3 or greater event intolerable side effect experienced in a cohort

Description

Trial will proceed as long as no more than one subject experiences an intolerable side effect in a cohort

Time Frame

To 14 days

10. Eligibility

Sex

All

Minimum Age & Unit of Time

20 Years

Maximum Age & Unit of Time

65 Years

Accepts Healthy Volunteers

Eligibility Criteria

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.

Facility Information:

Facility Name

University of Arizona Clinical and Translational Science Research Center

City

Tucson

State/Province

Arizona

ZIP/Postal Code

85724

Country

United States

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

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

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