NCT05351164

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

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 4 (actual)
• Sponsor: University of Michigan · Academic / Other
• Sex: All
• Age: 60 Years
• Healthy volunteers: Not accepted

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.

Detailed description

In 2024, the IRB granted an amendment that allowed for continued data collection, including adverse events, up to 5 years per participant.

Conditions

• Lipomatosis, Multiple Symmetrical

Interventions

Type	Name	Description
DRUG	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).

Timeline

• Start date: 2023-08-01
• Primary completion: 2024-04-08
• Completion: 2028-10-01
• First posted: 2022-04-28
• Last updated: 2025-06-12
• Results posted: 2025-06-12

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study