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

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

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

Brief 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 Summary:

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 would allow continued treatment of patients with partial forms of lipodystrophy who volunteered and completed treatment under the investigators' ongoing protocol (MB002-014) and who have derived significant clinical benefit as judged by an amelioration of their HbA1c, triglyceride levels, and/or reduction in their baseline diabetes or lipid therapies that affect quality of life.


Sponsor: University of Michigan

Current Primary Outcome: Improvement in Non alcoholic fatty liver disease [ Time Frame: 5 years ]

Improvement in total NAS score by liver biopsy


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Improvement in triglyceride levels [ Time Frame: 5 years ]
    reduction in triglyceride levels
  • Improvement in hemoglobin A1c values [ Time Frame: 5 years ]
    reduction in hemoglobin A1c


Original Secondary Outcome: Same as current

Information By: University of Michigan

Dates:
Date Received: January 20, 2015
Date Started: April 2015
Date Completion: February 2025
Last Updated: October 17, 2016
Last Verified: October 2016