Back to resultsEvaluation of Safety and Efficacy of RZL-012 for the Treatment of Lipedema or of Nodular Dercum's Disease
Primary Purpose
Lipedema, Dercum Disease
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
RZL-012
Sponsored by
About this trial
This is an interventional treatment trial for Lipedema
Eligibility Criteria
Inclusion Criteria:
- 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.
- Subjects with stable weight in the last 3 months by medical history.
- For Dercum's disease subjects - at least 2 nodules to be injected of at least 2cm diameter each, as determined by ultrasound
- For lipedema subjects - Significant subcutaneous fat above the knee as determined by circumference of 50cm
- 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).
- Subjects must be able to adhere to the visit schedule and protocol requirements and be available to complete the study.
- Subjects must sign an informed consent indicating that they are aware of the investigational nature of the study.
Exclusion Criteria:
- Unable to tolerate subcutaneous injection.
- 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.
- 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).
- Subjects with a clinical history of primary or secondary immunodeficiency, autoimmune disease or subjects taking immunosuppressive drugs such as corticosteroids are ineligible.
- As a result of medical review, 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.
Sites / Locations
- University of Arizona Medical Center
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
RZL-012
Arm Description
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.
Outcomes
Primary Outcome Measures
The incidence of intolerable side effects (a related adverse event) as defined in the protocol.
Adverse events [AEs] will be assessed by significant clinical changes in safety parameter (e.g. vital signs, ECG, clinical laboratory evaluations)
Secondary Outcome Measures
For lipedema subjects - The reduction of fat above the knee in the injection site region
Evaluated by measurement of leg circumference above the knee compared to baseline.
For Dercum's disease subjects - The reduction in local subcutaneous fat in the injection site region.
Evaluated by the reduction in nodules size and/or n number of nodules as assessed by ultrasound compared to baseline
Extended duration of the fat reduction effect
Assessed by leg circumference measurement that will be followed for 56 days
Elucidation of the tissue changes
Assessed by nodular quality at the injection site by ultrasound and compared to baseline
Improvement in local pain
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.
Improvement in pain
Assessed by reduction in the use of analgesics and compared to baseline.
Improvement in physical function in lipedema subjects
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.
For lipedema subjects - The reduction in local subcutaneous fat in the injection site region
Assessed by ultrasound images that will be followed for 56 days
Full Information
NCT ID
NCT03492840
First Posted
February 19, 2018
Last Updated
May 13, 2019
Sponsor
Raziel Therapeutics Ltd.
Collaborators
University of Arizona
1. Study Identification
Unique Protocol Identification Number
NCT03492840
Brief Title
Evaluation of Safety and Efficacy of RZL-012 for the Treatment of Lipedema or of 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
April 2019
Overall Recruitment Status
Completed
Study Start Date
May 1, 2018 (Actual)
Primary Completion Date
May 6, 2019 (Actual)
Study Completion Date
May 6, 2019 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Raziel Therapeutics Ltd.
Collaborators
University of Arizona
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
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.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Lipedema, Dercum Disease
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Model Description
An open label clinical trial - subjects will receive the investigational drug only
Masking
None (Open Label)
Allocation
N/A
Enrollment
12 (Actual)
8. Arms, Groups, and Interventions
Arm Title
RZL-012
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
RZL-012
Intervention Description
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 Measure Information:
Title
The incidence of intolerable side effects (a related adverse event) as defined in the protocol.
Description
Adverse events [AEs] will be assessed by significant clinical changes in safety parameter (e.g. vital signs, ECG, clinical laboratory evaluations)
Time Frame
0-56 days
Secondary Outcome Measure Information:
Title
For lipedema subjects - The reduction of fat above the knee in the injection site region
Description
Evaluated by measurement of leg circumference above the knee compared to baseline.
Time Frame
0-56 days
Title
For Dercum's disease subjects - The reduction in local subcutaneous fat in the injection site region.
Description
Evaluated by the reduction in nodules size and/or n number of nodules as assessed by ultrasound compared to baseline
Time Frame
0-56 days
Title
Extended duration of the fat reduction effect
Description
Assessed by leg circumference measurement that will be followed for 56 days
Time Frame
0-56 days
Title
Elucidation of the tissue changes
Description
Assessed by nodular quality at the injection site by ultrasound and compared to baseline
Time Frame
0-56 days
Title
Improvement in local pain
Description
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.
Time Frame
0-56 days
Title
Improvement in pain
Description
Assessed by reduction in the use of analgesics and compared to baseline.
Time Frame
0-56 days
Title
Improvement in physical function in lipedema subjects
Description
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.
Time Frame
0-56 days
Title
For lipedema subjects - The reduction in local subcutaneous fat in the injection site region
Description
Assessed by ultrasound images that will be followed for 56 days
Time Frame
0-56 days
10. Eligibility
Sex
All
Gender Based
Yes
Gender Eligibility Description
For lipedema - women only. For lipedema and Dercum- Post-menopausal women
Minimum Age & Unit of Time
20 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
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.
Subjects with stable weight in the last 3 months by medical history.
For Dercum's disease subjects - at least 2 nodules to be injected of at least 2cm diameter each, as determined by ultrasound
For lipedema subjects - Significant subcutaneous fat above the knee as determined by circumference of 50cm
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).
Subjects must be able to adhere to the visit schedule and protocol requirements and be available to complete the study.
Subjects must sign an informed consent indicating that they are aware of the investigational nature of the study.
Exclusion Criteria:
Unable to tolerate subcutaneous injection.
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.
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).
Subjects with a clinical history of primary or secondary immunodeficiency, autoimmune disease or subjects taking immunosuppressive drugs such as corticosteroids are ineligible.
As a result of medical review, 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Karen Herbst, MD
Organizational Affiliation
University of Arizona
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of Arizona Medical Center
City
Tucson
State/Province
Arizona
ZIP/Postal Code
85724
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Evaluation of Safety and Efficacy of RZL-012 for the Treatment of Lipedema or of Nodular Dercum's Disease
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