Clinical Trial

Evaluation of Safety and Efficiency of Metreleptin Treatment for Patients With Multiple Symmetric Lipomatosis (MSL)

Active, Not Recruiting Phase 2
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Summary
Participants (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.
Protocol Amendment History 7 amendments
This ClinicalTrials.gov record has been amended 7 times since 2022-04-22; most recent amendment 2025-06-10.
Status change: Enrolling by Invitation → Active, Not Recruiting 2024-04-17
Status change: Not Yet Recruiting → Enrolling by Invitation 2023-02-10
Trial Details
NCT Number NCT05351164
Lead Sponsor University of Michigan
Collaborators: Amryt Pharma
Conditions Lipomatosis, Multiple Symmetrical
Enrollment 4 participants
Start Date 2023-08-01
Primary Completion 2024-04-08 (estimated)
Study Completion 2028-10 (estimated)
Updated on ClinicalTrials.gov 2025-06-12