NCT02654977

CLINICAL PROTOCOL to Investigate the Long-term Safety and Efficacy of Metreleptin in Various Forms of Partial Lipodystrophy

CLINICAL PROTOCOL to Investigate the Long-term Safety and Efficacy of Recombinant Human Leptin (METRELEPTIN) in Various Forms of Partial Lipodystrophy

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 11 (actual)
• Sponsor: University of Michigan · Academic / Other
• Sex: All
• Age: 5 Years
• Healthy volunteers: Not accepted

Summary

The study investigators' aim is to determine the long term safety and efficacy of Metreleptin (Myalept,) in promoting amelioration of metabolic abnormalities in patients with all forms of partial lipodystrophy. Patients will be offered this protocol under the following condition: Subjects have completed University of Michigan research protocol MB002-014 and have shown improved clinical benefit as judged by clinical criteria set forth in this protocol.

Detailed description

Leptin is now an approved therapeutic in the form of Myalept in patients with generalized forms of lipodystrophy. However, it is still under investigation for patients with partial forms of the disease based on FDA decision on February 24, 2014. The study investigators have been carrying out a protocol in patients with partial lipodystrophy, specifically familial partial lipodystrophy. There have been a number of patients who have been treated under this protocol who are not covered by the currently approved label, but who have experienced significant clinical benefit. This study allowed continued treatment of patients with partial forms of lipodystrophy who volunteered and completed treatment under the investigators' completed protocol (MB002-014) and who derived significant clinical benefit as judged by an amelioration of their hemoglobin A1c, triglyceride levels, and/or reduction in their baseline diabetes or lipid therapies that affect quality of life. In this long-term study, there were three individuals whose data were excluded from final data analyses as explained in the results, however all participants were included in the presentation of baseline data as well as tabulated safety data.

Conditions

• Familial Partial Lipodystrophy
• Nonalcoholic Steatohepatitis
• NAFLD

Interventions

Type	Name	Description
DRUG	Metreleptin	MyaLept (Recombinant-methionyl Human Leptin (r-metHuLeptin) METRELEPTIN) subcutaneous injections

Timeline

• Start date: 2015-09-29
• Primary completion: 2019-11-14
• Completion: 2019-11-14
• First posted: 2016-01-13
• Last updated: 2020-11-24
• Results posted: 2020-11-24

Locations

1 site across 1 country: United States