Clinical trial NCT05164341: Study to Evaluate the Safety and Efficacy of Daily Subcutaneous Metreleptin Treatment in Subjects With PL
Official title: 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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Partial Lipodystrophy
Keywords: metreleptin
Lead sponsor: Amryt Pharma
Sponsor class: INDUSTRY
First posted: 2021-12-20
Enrollment: 65
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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


Eligibility criteria: 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

Primary outcomes:
measure: Change from baseline to month 6 in HbA1c in subjects with partial lipodystrophy (PL)
timeFrame: 6 months
measure: Percent change from Baseline to month 6 in fasting TGs in subjects with partial lipodystrophy (PL)
timeFrame: 6 months
Secondary outcomes:
measure: Safety analysis of AEs, AESIs, SAEs by treatment arm
timeFrame: 12 months
measure: Change from baseline to months 9 and 12 in HbA1c in subjects with partial lipodystrophy (PL)
timeFrame: 12 months
measure: Percent change from baseline to months 9 and 12 in fasting TGs in subjects with partial lipodystrophy (PL)
timeFrame: 12 months
measure: Change from Baseline to each assessment time point in quality of life (QoL) in all subjects
timeFrame: 12 months
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama
state: Alabama
city: Sacramento
country: United States
facility: UC Davis
state: California
city: Boca Raton
country: United States
facility: Flourish Boca Raton
state: Florida
city: Atlanta
country: United States
facility: Emory University
state: Georgia
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan
state: Michigan
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
city: Columbia
country: United States
facility: University of Missouri
state: Missouri
city: Columbus
country: United States
facility: Amryt Research Site, Endocrinology Research Associates Inc
state: Ohio
city: Columbus
country: United States
facility: The Ohio State University Wexner Medical Center
state: Ohio
city: Philadelphia
country: United States
facility: The Children's Hospital of Philadelphia
state: Pennsylvania
city: Dallas
country: United States
facility: UT Southwestern Medical Center
state: Texas
city: Milwaukee
country: United States
facility: Medical College of Wisconsin
state: Wisconsin
city: Leuven
country: Belgium
facility: UZ Leuven
city: Fortaleza
country: Brazil
facility: Universitário Walter Cantídio
city: Porto Alegre
country: Brazil
facility: Insight Centro de Pesquisas
city: Ribeirão Preto
country: Brazil
facility: Hospital das Clinicas FMRP-USP
city: Rio De Janeiro
country: Brazil
facility: Instituto Estadual de Diabetes e Endocrinologia (IEDE-RJ)
city: Halifax
country: Canada
facility: Nova Scotia Health
state: Nova Scotia
city: Sydney
country: Canada
facility: Care Access Clinic
state: Nova Scotia
city: Hamilton
country: Canada
facility: Hamilton General Hospital- McMaster University
state: Ontario
city: London
country: Canada
facility: London Health Science Centre
state: Ontario
city: Chicoutimi
country: Canada
facility: Ecogene-21
state: Quebec
city: Québec
country: Canada
facility: Faculty of Medicine, Universite Laval
state: Quebec
city: Santiago
country: Chile
facility: Universidad Catolica
city: Jerusalem
country: Israel
facility: Hadassah University Hospital - Ein Kerem
city: Leiden
country: Netherlands
facility: Leids Universitair Medisch Centrum
city: Nijmegen
country: Netherlands
facility: Radboud University Medical Centre Interne Geneeskund
city: Katowice
country: Poland
facility: Uniwersyteckie Centrum Kliniczne
city: Kraków
country: Poland
facility: SPZOZ Szpital Uniwersytecki w Krakowie
city: Santiago De Compostela
country: Spain
facility: University Clinical Hospital of Santiago de Compostela/Hospital Universitario
Study officials:
References: