Hope for rare fat disorder: expanded access to metreleptin offered

NCT ID NCT02404896

AVAILABLE Disease control Sponsor: University of Michigan Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This expanded access program provides metreleptin (MyaLept) to patients with familial partial lipodystrophy who also have diabetes or very high triglycerides. Metreleptin is already approved for generalized lipodystrophy, but this program aims to help those with the partial form. Participants receive open-label treatment, and the goal is to manage blood sugar and fat levels.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: metreleptin (MyaLept)
What this could lead to: If successful, this could provide ongoing disease control for patients with partial lipodystrophy who have diabetes or high triglycerides.
What could go wrong: This is an expanded access program, not a formal trial, so results are limited. Metreleptin is already approved for generalized lipodystrophy, but its benefits in partial lipodystrophy are less certain and may not work for everyone.

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Conditions

The condition(s) this trial relates to.

familial partial lipodystrophy partial lipodystrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

FAMILIAL PARTIAL LIPODYSTROPHY

Contacts and locations

Locations

University of Michigan
AVAILABLE

Ann Arbor, Michigan, 48105, United States

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