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Evaluation of Safety and Efficiency of Metreleptin Treatment for Patients With Multiple Symmetric Lipomatosis (MSL)
Sponsor: University of Michigan
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.
Key Details
Gender
All
Age Range
Any - 60 Years
Study Type
INTERVENTIONAL
Enrollment
4
Start Date
2023-08-01
Completion Date
2028-10
Last Updated
2025-06-12
Healthy Volunteers
No
Conditions
Interventions
Metreleptin
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).
Locations (1)
University of Michigan
Ann Arbor, Michigan, United States