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

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

Official Title
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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

Conditions
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Partial Lipodystrophy

Intervention / Treatment
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* Drug: metreleptin
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosis of Familial Partial Lipodystrophy (FPLD) Exclusion Criteria: Previous treatment with metreleptin

Ages Eligible for Study
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Minimum Age: 12 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: metreleptin<br>Metreleptin [Recombinant-methionyl human Leptin; r-metHuLeptin] for daily injection is a sterile, white, solid lyophilised cake | Drug: metreleptin<br>* 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<br>|
| Placebo Comparator: placebo<br>Placebo for daily injection is a sterile, white, solid lyophilised cake | Drug: Placebo<br>* Placebo<br>|

What is the study measuring?
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Primary Outcome Measures

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

Secondary Outcome Measures

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

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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metreleptin