Metreleptin for Partial Lipodystrophy
(METRE-PL Trial)
Recruiting in Palo Alto (17 mi)
+51 other locations
Age: Any Age
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 3
Recruiting
Sponsor: Amryt Pharma
Disqualifiers: Previous metreleptin, High leptin, APL, others
Pivotal Trial (Near Approval)
Prior Safety Data
Trial Summary
What is the purpose of this trial?This trial tests the safety and effectiveness of daily injections of metreleptin for people with Partial Lipodystrophy. The treatment aims to replace a missing hormone to help their bodies manage fat and sugar levels better. Metreleptin is approved by the United States Food and Drug Administration for the treatment of generalized forms of lipodystrophy and has shown improvement in many patients.
Will I have to stop taking my current medications?
The trial does not specify if you need to stop taking your current medications, but it mentions that patients should be on optimized stable therapy. This suggests you may need to continue your current treatment.
What data supports the effectiveness of the drug metreleptin for partial lipodystrophy?
Research shows that metreleptin helps improve eating behavior in lipodystrophy patients by increasing feelings of fullness and reducing hunger, and these benefits last for a long time.
12345How is the drug metreleptin unique for treating partial lipodystrophy?
Metreleptin is unique because it is specifically designed to replace leptin, a hormone that helps regulate fat and sugar levels in the body, which is often deficient in people with lipodystrophy. Unlike other treatments, it directly addresses the underlying hormone imbalance rather than just managing symptoms like high blood sugar or cholesterol.
13567Eligibility Criteria
This trial is for individuals with a condition called Partial Lipodystrophy, specifically those diagnosed with Familial Partial Lipodystrophy (FPLD). It's not open to people who have already been treated with metreleptin.Inclusion Criteria
I have been diagnosed with Familial Partial Lipodystrophy.
Exclusion Criteria
I have been treated with metreleptin before.
Trial Timeline
Screening
Participants are screened for eligibility to participate in the trial
2-4 weeks
Treatment
Participants receive daily subcutaneous metreleptin or placebo for 12 months
12 months
Follow-up
Participants are monitored for safety and effectiveness after treatment
4 weeks
Participant Groups
The study is testing the safety and effectiveness of a daily injection of a drug called metreleptin compared to a placebo. This is done in a controlled way where neither the participants nor the researchers know who gets the real treatment or placebo.
2Treatment groups
Active Control
Placebo Group
Group I: metreleptinActive Control1 Intervention
Metreleptin \[Recombinant-methionyl human Leptin; r-metHuLeptin\] for daily injection is a sterile, white, solid lyophilised cake
Group II: placeboPlacebo Group1 Intervention
Placebo for daily injection is a sterile, white, solid lyophilised cake
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
University of PennsylvaniaPhiladelphia, PA
UT Southwestern Medical CenterDallas, TX
Medical College of WisconsinMilwaukee, WI
Nova Scotia HealthHalifax, Canada
More Trial Locations
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Who Is Running the Clinical Trial?
Amryt PharmaLead Sponsor
References
Long-term effectiveness and safety of metreleptin in the treatment of patients with partial lipodystrophy. [2022]To evaluate the effects of metreleptin in patients with partial lipodystrophy (PL).
Therapeutic indications and metabolic effects of metreleptin in patients with lipodystrophy syndromes: Real-life experience from a national reference network. [2022]To describe baseline characteristics and follow-up data in patients with lipodystrophy syndromes treated with metreleptin in a national reference network, in a real-life setting.
Beneficial effects of leptin substitution on impaired eating behavior in lipodystrophy are sustained beyond 150 weeks of treatment. [2020]Metreleptin treatment in lipodystrophy patients improves eating behavior with increased satiety and reduced hunger. However, no data are available whether effects are maintained beyond 52 weeks of treatment.
Effect of Leptin Therapy on Survival in Generalized and Partial Lipodystrophy: A Matched Cohort Analysis. [2022]Data quantifying the impact of metreleptin therapy on survival in non-human immunodeficiency virus (HIV)-related generalized lipodystrophy (GL) and partial lipodystrophy (PL) are unavailable.
Long-term effectiveness and safety of metreleptin in the treatment of patients with generalized lipodystrophy. [2023]The purpose of this study is to summarize the effectiveness and safety of metreleptin in patients with congenital or acquired generalized lipodystrophy.
Effects of metreleptin in patients with lipodystrophy with and without baseline concomitant medication use. [2021]To evaluate the effects of metreleptin in distinct subgroups of patients with generalized lipodystrophy (GL) and partial lipodystrophy (PL), using multivariate linear regression modeling to account for the role of patients' baseline usage of concomitant glucose and lipid-lowering medications and other covariates on their outcomes.
Efficacy and Safety of Metreleptin in Patients with Partial Lipodystrophy: Lessons from an Expanded Access Program. [2020]Patients with lipodystrophy have severe metabolic abnormalities (insulin resistance, diabetes, and hypertriglyceridemia) that may increase morbidity and mortality. Metreleptin is approved by the United States Food and Drug Administration for treatment of generalized forms of lipodystrophy. We aimed to determine the efficacy and safety of metreleptin among patients with partial lipodystrophy using an expanded-access model.