- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05351164
Evaluation of Safety and Efficiency of Metreleptin Treatment for Patients With Multiple Symmetric Lipomatosis (MSL)
April 17, 2024 updated by: Elif Oral, University of Michigan
Patients (homozygous MFN2 [gene that provides instructions to produce the Mitofusin 2 protein] R707W) will be treated with Metreleptin, and effects on body composition, metabolic parameters and safety will be assessed over a 6 month intervention period.
Additional safety will be assessed for 1 more year (up to 1.5 years total) in which adverse event data will be collected.
Study Overview
Status
Active, not recruiting
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Actual)
4
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
-
Michigan
-
Ann Arbor, Michigan, United States, 48109
- University of Michigan
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
No older than 60 years (Child, Adult)
Accepts Healthy Volunteers
No
Description
Inclusion Criteria:
- Have the clinical diagnosis of MSL and being followed at University of Michigan (cohort to be studied in this proof-of-concept study is already available at Michigan).
- Willing and able to tolerate the study procedures.
- Willing and able to tolerate blood sampling.
- Having no condition that may impede successful data collection or interfere with testing parameters.
- <60 years of age.
If female of childbearing potential:
- Not breastfeeding.
- Negative pregnancy test (human chorionic gonadotropin, beta subunit) at baseline.
- Can read, understand and sign approved informed consent form, communicate with study physician, and study team, and understand and comply with protocol requirements.
Exclusion Criteria:
- Presence of advanced liver disease (abnormal synthetic function, prothrombin time [PT], or albumin) in medical records
- Evidence of other etiologies of viral hepatitis in medical records
- Presence of active hematologic, bone marrow or other abnormalities that may increase risk of bleeding in medical records.
- Presence of HIV infection in medical records.
- Presence of End-stage renal disease (ESRD), active cancer, or >class 2 congestive heart failure based on medical history and physical examination.
- Active chronic infection (e.g., known chronic osteomyelitis or Tuberculosis [TB]). May have transient infections but must be free of active infection for two weeks prior to study visits.
- Unable to ambulate or tolerate trips to the University of Michigan Clinical Research Unit.
- Clinically relevant Coronary artery disease (CAD): history of stent or Coronary artery bypass graft surgery (CABG) with cardiologist confirmed angina.
- Presence of autoimmune disease.
- Hypersensitivity to metreleptin.
- General obesity not associated with congenital leptin deficiency.
- Any other condition that, in our opinion, may impede successful data collection.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Patients with MSL
|
Study drug for injection is supplied in a carton containing 30 vials for reconstitution.
Each vial contains 11.3 mg of the study drug as a sterile, white, solid, lyophilized cake or powder to deliver 5 mg/mL of the study drug when reconstituted with 2.2 mL of water for injection (WFI).
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change in truncal adiposity
Time Frame: Baseline, Week 24
|
Measured using a Dual-energy X-ray absorptiometry (DEXA) scan
|
Baseline, Week 24
|
Change in total adiposity
Time Frame: Baseline, Week 24
|
Measured using a Dual-energy X-ray absorptiometry (DEXA) scan
|
Baseline, Week 24
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Collaborators
Investigators
- Principal Investigator: Elif Oral, University of Michigan
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
August 1, 2023
Primary Completion (Actual)
April 8, 2024
Study Completion (Estimated)
April 1, 2025
Study Registration Dates
First Submitted
April 22, 2022
First Submitted That Met QC Criteria
April 22, 2022
First Posted (Actual)
April 28, 2022
Study Record Updates
Last Update Posted (Actual)
April 19, 2024
Last Update Submitted That Met QC Criteria
April 17, 2024
Last Verified
April 1, 2024
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- HUM00206598
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.
Yes
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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