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Evaluation of Safety and Efficacy of RZL-012 for the Treatment of Lipedema or of Nodular Dercum's Disease

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ClinicalTrials.gov Identifier: NCT03492840
Recruitment Status : Completed
First Posted : April 10, 2018
Last Update Posted : May 14, 2019
Sponsor:
Collaborator:
University of Arizona
Information provided by (Responsible Party):
Raziel Therapeutics Ltd.

Brief Summary:
Lipedema and Dercum's disease are known as Rare Adipose Disorders; they are painful, progressive disorders that result in abnormal accumulations of fat in the form of lipomas, excess lymphatic fluid, and many other symptoms. This trial aims to study RZL-012, a novel compound, in treating lipedema and Dercum's disease by triggering lipolysis at selected sites and reducing fat bringing pain relief and improvement in quality of life.

Condition or disease Intervention/treatment Phase
Lipedema Dercum Disease Drug: RZL-012 Phase 2

Detailed Description:

A vast amount of published information proves that for some people, obesity is not the result of excessive food consumption, poor food choices, and failure to exercise . Problems with mitochondria, leaky lymphatics and other mutations can drive and maintain fat growth that cannot be lost through traditional diet and exercise. Lipedema and Dercum's disease are known as Rare Adipose Disorders. These are painful and progressive disorders that result in abnormal accumulations of fat in the form of lipomas, excess lymphatic fluid, and many other symptoms.

RZL-012 is a novel molecule that enables de-novo generation of thermogenic tissue at favorable anatomical sites. As a result, the extra accumulated fat will be turned into heat and induce lipolysis. This is an open label , Phase 2a clinical trial for the evaluation of safety, efficacy and lipolysis-induction of RZL-012 in women with lipedema involving substantial fat above the knee or women and men with nodular Dercum's disease.


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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 12 participants
Intervention Model: Single Group Assignment
Intervention Model Description: An open label clinical trial - subjects will receive the investigational drug only
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : May 1, 2018
Actual Primary Completion Date : May 6, 2019
Actual Study Completion Date : May 6, 2019

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: RZL-012

A single-time injection, multiple subcutaneous injections of RZL-012 administered into the subcutaneous fat.

Dercum's disease - dosing according to nodule size:

Nodule of 2-2.9cm - 2 injections (0.1 mL each); total of 10 mg RZL-012.

Nodules of 3-3.9cm - 3 injections (0.1 mL each); total of 15 mg RZL-012.

Nodules of 4-8cm - 4 injections (0.1 mL each); total of 20 mg RZL-012.

Lipedema -

2 subjects will receive 20mg RZL-012 in 4 injections in each leg adding up to 8 injections of 40mg RZL-012.

2 subjects will receive 30mg RZL-012 in 6 injections in each leg adding up to 12 injections of 60mg RZL-012.

2 subjects will receive 40mg RZL-012 in 8 injections in each leg adding up to 16 injections of 80mg RZL-012.

Drug: RZL-012
Subject receive a single-time injection. Multiple injections of RZL-012 are administered at 2-16 sites (0.1mL per site) into the subcutaneous fat.




Primary Outcome Measures :
  1. The incidence of intolerable side effects (a related adverse event) as defined in the protocol. [ Time Frame: 0-56 days ]
    Adverse events [AEs] will be assessed by significant clinical changes in safety parameter (e.g. vital signs, ECG, clinical laboratory evaluations)


Secondary Outcome Measures :
  1. For lipedema subjects - The reduction of fat above the knee in the injection site region [ Time Frame: 0-56 days ]
    Evaluated by measurement of leg circumference above the knee compared to baseline.

  2. For Dercum's disease subjects - The reduction in local subcutaneous fat in the injection site region. [ Time Frame: 0-56 days ]
    Evaluated by the reduction in nodules size and/or n number of nodules as assessed by ultrasound compared to baseline

  3. Extended duration of the fat reduction effect [ Time Frame: 0-56 days ]
    Assessed by leg circumference measurement that will be followed for 56 days

  4. Elucidation of the tissue changes [ Time Frame: 0-56 days ]
    Assessed by nodular quality at the injection site by ultrasound and compared to baseline

  5. Improvement in local pain [ Time Frame: 0-56 days ]
    Assessed by the Comparative Pain Scale and compared to baseline. Comparative Pain Scale ranges are: 1-3 Minor pain, 4-6 Moderate pain, 7-10 Severe pain. Higher values represent worst outcome.

  6. Improvement in pain [ Time Frame: 0-56 days ]
    Assessed by reduction in the use of analgesics and compared to baseline.

  7. Improvement in physical function in lipedema subjects [ Time Frame: 0-56 days ]
    Assessed by The Lower Extremity Functional Scale (LEFS). LEFS is used to assess if the patient has difficulties in their physical function activities. For each physical function activity the range is 1-4 when higher values are considered worse outcome. Subscales are combined to compute a total score. The percentage of physical function is calculated by dividing the total score by 80.

  8. For lipedema subjects - The reduction in local subcutaneous fat in the injection site region [ Time Frame: 0-56 days ]
    Assessed by ultrasound images that will be followed for 56 days



Information from the National Library of Medicine

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Ages Eligible for Study:   20 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Gender Based Eligibility:   Yes
Gender Eligibility Description:   For lipedema - women only. For lipedema and Dercum- Post-menopausal women
Accepts Healthy Volunteers:   No
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. Subjects with stable weight in the last 3 months by medical history.
  3. For Dercum's disease subjects - at least 2 nodules to be injected of at least 2cm diameter each, as determined by ultrasound
  4. For lipedema subjects - Significant subcutaneous fat above the knee as determined by circumference of 50cm
  5. Generally considered healthy according to medical history, physical examination, ECG and laboratory evaluation with a special emphasis on metabolic parameters (fasting glucose concentration < 100 mg, normal blood pressure).
  6. Subjects must be able to adhere to the visit schedule and protocol requirements and be available to complete the study.
  7. 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03492840


Locations
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United States, Arizona
University of Arizona Medical Center
Tucson, Arizona, United States, 85724
Sponsors and Collaborators
Raziel Therapeutics Ltd.
University of Arizona
Investigators
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Principal Investigator: Karen Herbst, MD University of Arizona

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Responsible Party: Raziel Therapeutics Ltd.
ClinicalTrials.gov Identifier: NCT03492840     History of Changes
Other Study ID Numbers: RZL-012-FD-P2aUS-001.2
First Posted: April 10, 2018    Key Record Dates
Last Update Posted: May 14, 2019
Last Verified: April 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Lipedema
Adiposis Dolorosa
Connective Tissue Diseases
Lipomatosis
Skin Diseases
Lipid Metabolism Disorders
Metabolic Diseases