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

Purpose

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

Condition

  • Partial Lipodystrophy

Eligibility

Eligible Ages
Over 12 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

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

Phase
Phase 3
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
Active Comparator
metreleptin 		Metreleptin [Recombinant-methionyl human Leptin; r-metHuLeptin] for daily injection is a sterile, white, solid lyophilised cake
  • Drug: metreleptin
    Metreleptin is a recombinant human leptin analog that is indicated as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency
Placebo Comparator
placebo 		Placebo for daily injection is a sterile, white, solid lyophilised cake
  • Drug: Placebo
    Placebo

Recruiting Locations

Massachusetts General Hospital
Boston, Massachusetts 02114
Contact:
Lindsay Fourman

More Details

Status
Recruiting
Sponsor
Amryt Pharma

Study Contact

Janet Boylan
+3905212791
clinicaltrials_info@chiesi.com