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

Amryt Pharma

Status and phase: Enrolling; Phase 3

Conditions: Partial Lipodystrophy

Treatments: Drug: metreleptin; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT05164341

APG-20

Details and patient eligibility

About

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

Enrollment: 65 estimated patients

Sex: All

Ages: 12+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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65 participants in 2 patient groups, including a placebo group

metreleptin

Active Comparator group

Description:

Metreleptin \[Recombinant-methionyl human Leptin; r-metHuLeptin\] for daily injection is a sterile, white, solid lyophilised cake

Treatment:

Drug: metreleptin

placebo

Placebo Comparator group

Description:

Placebo for daily injection is a sterile, white, solid lyophilised cake

Treatment:

Drug: Placebo