Study to Evaluate the Safety and Efficacy of Daily Subcutaneous Metreleptin Treatment in Subjects With PL

Phase 3

Recruiting

• Conditions: Partial Lipodystrophy
• Interventions: Drug: metreleptin; Drug: Placebo

• Registration Number: NCT05164341
• Lead Sponsor: Amryt Pharma

Brief Summary

This is a Phase III, double-blind, placebo-controlled, safety and efficacy study of daily SC metreleptin in subjects with Partial Lipodystrophy.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2021-11-11
• Study First Submitted QC: 2021-12-06
• Study Start: 2021-12-17
• Study First Posted: 2021-12-20
• Status Verified: 2025-04
• Last Update Submitted: 2025-04-01
• Last Update Posted: 2025-04-03
• Primary Completion: 2025-06-30
• Study Completion: 2026-01-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 65

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
metreleptin	metreleptin	Metreleptin \[Recombinant-methionyl human Leptin; r-metHuLeptin\] for daily injection is a sterile, white, solid lyophilised cake
placebo	Placebo	Placebo for daily injection is a sterile, white, solid lyophilised cake

Primary Outcome Measures

Name	Time	Method
Percent change from Baseline to month 6 in fasting TGs in subjects with partial lipodystrophy (PL)	6 months	To evaluate the efficacy (TGs) of daily SC metreleptin treatment
Change from baseline to month 6 in HbA1c in subjects with partial lipodystrophy (PL)	6 months	To evaluate the efficacy (HbAa1c) of daily SC metreleptin treatment

Secondary Outcome Measures

Name	Time	Method
Change from baseline to months 9 and 12 in HbA1c in subjects with partial lipodystrophy (PL)	12 months	To assess the effect of metreleptin on HbA1c
Percent change from baseline to months 9 and 12 in fasting TGs in subjects with partial lipodystrophy (PL)	12 months	To assess the effect of metreleptin on TGs
Safety analysis of AEs, AESIs, SAEs by treatment arm	12 months	To evaluate the safety of daily SC metreleptin treatment in subjects with PL
Change from Baseline to each assessment time point in quality of life (QoL) in all subjects	12 months	To assess the effect of metreleptin on quality of life (QoL) in all subjects

Trial Locations

Locations (31)

UC Davis; 🇺🇸 Sacramento, California, United States

Emory University; 🇺🇸 Atlanta, Georgia, United States

The Children's Hospital of Philadelphia; 🇺🇸 Philadelphia, Pennsylvania, United States

Care Access Clinic; 🇨🇦 Sydney, Nova Scotia, Canada

University of Alabama; 🇺🇸 Birmingham, Alabama, United States

Flourish Boca Raton; 🇺🇸 Boca Raton, Florida, United States

Massachusetts General Hospital; 🇺🇸 Boston, Massachusetts, United States

University of Michigan; 🇺🇸 Ann Arbor, Michigan, United States

Mayo Clinic; 🇺🇸 Rochester, Minnesota, United States

University of Missouri; 🇺🇸 Columbia, Missouri, United States

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