Partial LipodystrophyPhase 3METRE-PLNCT05164341
Study to Evaluate the Safety and Efficacy of Daily Subcutaneous Metreleptin Treatment in Subjects With PL
A Phase 3 study testing metreleptin in people with Partial Lipodystrophy, run by Chiesi Farmaceutici S.p.A. at 31 sites in 9 countries.
Trial summary
This is a Phase III, double-blind, placebo-controlled, safety and efficacy study of daily SC metreleptin in subjects with Partial Lipodystrophy. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Partial Lipodystrophy
- What is being tested?
- metreleptin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 69 participants
- Where
- 31 sitesin 9 countries
- Study dates
- 17 Dec 2021to 31 Oct 2026
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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 locations and contacts
31 sites in 9 countries.
31 sites in 9 countries
United States13 sites
University of Michigan
Ann Arbor, Michigan, 48109
Emory University
Atlanta, Georgia, 30329
University of Alabama
Birmingham, Alabama, 35233
Flourish Boca Raton
Boca Raton, Florida, 33431
Massachusetts General Hospital
Boston, Massachusetts, 02114
University of Missouri
Columbia, Missouri, 65212
Amryt Research Site, Endocrinology Research Associates Inc
Columbus, Ohio, 43201
The Ohio State University Wexner Medical Center
Columbus, Ohio, 43210
UT Southwestern Medical Center
Dallas, Texas, 75390
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Mayo Clinic
Rochester, Minnesota, 55902
UC Davis
Sacramento, California, 95817
Canada6 sites
Ecogene-21
Chicoutimi, Quebec, G7H 7K9
Nova Scotia Health
Halifax, Nova Scotia, B3H 2Y9
Hamilton General Hospital- McMaster University
Hamilton, Ontario, L8N 3Z5
London Health Science Centre
London, Ontario, N6A 5B7
Faculty of Medicine, Universite Laval
Québec, Quebec, G1V4W2
Care Access Clinic
Sydney, Nova Scotia, B1M 0A1
Brazil4 sites
Universitário Walter Cantídio
Fortaleza
Insight Centro de Pesquisas
Porto Alegre
Hospital das Clinicas FMRP-USP
Ribeirão Preto
Instituto Estadual de Diabetes e Endocrinologia (IEDE-RJ)
Rio de Janeiro
Netherlands2 sites
Leids Universitair Medisch Centrum
Leiden
Radboud University Medical Centre Interne Geneeskund
Nijmegen
Poland2 sites
Uniwersyteckie Centrum Kliniczne
Katowice
SPZOZ Szpital Uniwersytecki w Krakowie
Krakow
Spain1 site
University Clinical Hospital of Santiago de Compostela/Hospital Universitario
Santiago de Compostela
About this study
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
This is a Phase III, double-blind, placebo-controlled, safety and efficacy study of daily SC metreleptin in subjects with Partial Lipodystrophy.
- Conditions
- Partial Lipodystrophy
- Being tested
- metreleptin, Placebo
- Keywords
- metreleptin
- Medical subject headings
- Lipodystrophy, Partial, Acquired
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
metreleptinDrug
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
PlaceboDrug
Placebo
| Study group | Role | What they receive |
|---|---|---|
| metreleptin | Active comparator | Metreleptin [Recombinant-methionyl human Leptin; r-metHuLeptin] for daily injection is a sterile, white, solid lyophilised cake Receives: Drug: metreleptin |
| placebo | Placebo comparator | Placebo for daily injection is a sterile, white, solid lyophilised cake Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline to month 6 in HbA1c in subjects with partial lipodystrophy (PL)
To evaluate the efficacy (HbAa1c) of daily SC metreleptin treatment
Measured: 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
Measured: 6 months
Secondary outcomes (4)
Safety analysis of AEs, AESIs, SAEs by treatment arm
To evaluate the safety of daily SC metreleptin treatment in subjects with PL
Measured: 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
Measured: 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
Measured: 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
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Dec 2021
- Study start17 Dec 2021
- Primary completion21 Jul 2026 (estimated)
- Study completion31 Oct 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Partial LipodystrophyStudies of the same condition, open and under way.
- More trials run by Chiesi Farmaceutici S.p.A.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 69 participants (actual)
Sponsor and investigators
- Lead sponsor
- Chiesi Farmaceutici S.p.A.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05164341
- Sponsor's protocol ID
- APG-20
- Acronym
- METRE-PL
- Status last verified
- Jun 2026
- First submitted
- 11 Nov 2021
- Last updated
- 15 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No