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Evaluating daily metreleptin treatment for Partial Lipodystrophy

A 12-Month Randomized, Multicenter, Double-Blind, Placebo-Controlled Phase 3 Study to Evaluate the Safety and Efficacy of Daily Subcutaneous Metreleptin Treatment in Subjects With Partial Lipodystrophy

Phase 3 Interventional Amryt Pharma · NCT05164341

This study is testing if daily injections of a medication called metreleptin can help people with Familial Partial Lipodystrophy improve their blood sugar and fat levels.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment65 (estimated)
Ages12 Years and up
SexAll
SponsorAmryt Pharma Industry-sponsored
Drugs / interventionsradiation
Locations31 sites (Birmingham, Alabama and 30 other locations)
Trial IDNCT05164341 on ClinicalTrials.gov

What this trial studies

This Phase III clinical trial is designed to assess the safety and efficacy of daily subcutaneous administration of metreleptin in patients diagnosed with Familial Partial Lipodystrophy. The study employs a double-blind, placebo-controlled methodology to ensure unbiased results. Participants will be monitored for metabolic control, specifically looking at HbA1c levels and fasting triglycerides. The goal is to determine whether metreleptin can improve metabolic outcomes in this patient population.

Who should consider this trial

Good fit: Ideal candidates for this study are individuals diagnosed with Familial Partial Lipodystrophy who have poor metabolic control.

Not a fit: Patients with acquired or radiation-induced partial lipodystrophy or those who have previously received metreleptin will not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve metabolic control and quality of life for patients with Partial Lipodystrophy.

How similar studies have performed: While this approach is novel in the context of Partial Lipodystrophy, similar studies with metreleptin have shown promise in other lipodystrophy conditions.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* Diagnosis of Familial Partial Lipodystrophy (FPLD)
* Subjects with poor metabolic control defined as:

HbA1c ≥7% (at Visit 1 and Visit 3) and/or Fasting TGs ≥500 mg/dL (5.65 mmol/L, at Visit 1 and Visit 3)

* Patients should be receiving optimized stable therapy

Exclusion Criteria:

* Previous treatment with metreleptin
* Leptin levels \>20.0 ng/mL
* Acquired or radiation induced partial lipodystrophy (APL)

Other protocol defined inclusion/exclusion criteria apply

Where this trial is running

Birmingham, Alabama and 30 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Partial Lipodystrophymetreleptin
Last reviewed 2026-06-09 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.