Generalized LipodystrophyPhase 3NCT06502990
Open-label Study to Evaluate Metreleptin in Children Under 6 Years of Age With Generalised Lipodystrophy
A Phase 3 study testing Metreleptin in people with Generalized Lipodystrophy, run by Amryt Pharma at 12 sites in 4 countries.
Trial summary
This is an open-label, Phase 3b study to evaluate effectiveness, safety and pharmacokinetic parameters of metreleptin in patients under 6 years of age with generalised lipodystrophy and associated diabetes mellitus and/or hypertriglyceridaemia Read more about this study
- Who is it for?
- Ages Up to 5 years
- Conditions the study is for
- Generalized Lipodystrophy
- What is being tested?
- Metreleptin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 12 sitesin 4 countries
- Study dates
- 23 May 2025to 11 Feb 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 Up to 5 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Confirmed diagnosis of Generalised Lipodystrophy
- Metreleptin treatment naive
Who cannot join
- Weight <9 kg at Screening (Visit 1)
Trial locations and contacts
12 sites in 4 countries.
12 sites in 4 countries
Italy7 sites
IRCCS Ospedale Pediatrico Bambino Gesù
Chieti, Chieti, 66100
Azienda Ospedaliera Universitaria "Federico II"
Naples, Naples, 80131
Azienda Ospedaliero Universitaria Maggiore della Carità di Novara
Novara, Novara, 28100
Azienda Ospedaliero-Universitaria di Parma
Parma, Parma, 43126
Azienda Ospedaliero Universitaria Pisana
Pisa, Pisa, 56124
IRCCS Ospedale Pediatrico Bambino Gesù
Rome, Rome, 00165
Ospedale Filippo Del Ponte Varese - ASST Sette Laghi
Varese, Varese, 21100
France2 sites
Hôpital Necker - Enfants Malades
Paris, Paris, 75015
Assistance Publique-Hopitaux de Paris (AP-HP) - Hopital Universitaire Robert-Debre
Paris
Germany2 sites
Universitätsklinikum Hamburg-Eppendorf
Hamburg, Free and Hanseatic City of Hamburg, 20251
Universitaetsklinikum Ulm - Klinik fuer Kinder- und Jugendmedizin
Ulm, Ulm, 89075
About this study
Official title: Open-label, Phase 3b Study to Evaluate Effectiveness, Safety and Pharmacokinetic Parameters of Metreleptin in Patients Under 6 Years of Age With Generalised Lipodystrophy and Associated Diabetes Mellitus and/or Hypertriglyceridaemia
This is an open-label, Phase 3b study to evaluate effectiveness, safety and pharmacokinetic parameters of metreleptin in patients under 6 years of age with generalised lipodystrophy and associated diabetes mellitus and/or hypertriglyceridaemia
- Conditions
- Generalized Lipodystrophy
- Being tested
- Metreleptin
- Keywords
- Metreleptin
- Medical subject headings
- Lipodystrophy, Congenital Generalized
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
| Study group | Role | What they receive |
|---|---|---|
| Metreleptin | Experimental | Metreleptin [Recombinant-methionyl human Leptin; rmetHuLeptin] for daily injection is a sterile, white, solid lyophilised cake Receives: Drug: Metreleptin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent change from baseline in fasting serum TG levels at Month 12 for subjects with fasting TG levels ≥2.3 mmol/L (200 mg/dL) at baseline
To evaluate the effectiveness of metreleptin in patients under 6 years of age with GL and associated diabetes mellitus and/or hypertriglyceridaemia
Measured: 12 months
Absolute change from baseline in glycated haemoglobin (HbA1c) at Month 12 for subjects with HbA1c ≥6.5% at baseline
To evaluate the effectiveness of metreleptin in patients under 6 years of age with GL and associated diabetes mellitus and/or hypertriglyceridaemia
Measured: 12 months
Secondary outcomes (6)
Proportion of subjects of those with Baseline HbA1c ≥6.5% achieving target actual decreases of at least 0.5%, 1%, 1.5%, 2% absolute decrease in HbA1c or HbA1c <6.5% or HbA1c <5.7% at Month 12
To evaluate the efficacy of metreleptin treatment in patients with GL
Measured: 12 months
Proportion of subjects of those with Baseline HbA1c ≥5.7% achieving target actual decreases of at least 0.5%, 1%, 1.5%, 2% absolute decrease in HbA1c or HbA1c <5.7% at Month 12
To evaluate the efficacy of metreleptin treatment in patients with GL
Measured: 12 months
Proportion of subjects of those with fasting serum TG ≥ 1.7 mmol/L (150 mg/dL) achieving target actual decreases from baseline of at least 15%, 20%, 25%, 30%, 35%, 40% at Month 12
To evaluate the efficacy of metreleptin treatment in patients with GL
Measured: 12 months
Proportion of subjects of those with fasting serum TG ≥ 2.3 mmol/L (200 mg/dL) achieving target actual decreases from baseline of at least 15%, 20%, 25%, 30%, 35%, 40% at Month 12
To evaluate the efficacy of metreleptin treatment in patients with GL
Measured: 12 months
Change from baseline in liver volume and liver span as assessed by ultrasound at each post-baseline visit through Month 12
To evaluate the efficacy of metreleptin treatment in patients with GL
Measured: 12 months
Incidence of, treatment emergent adverse events (TEAEs), serious adverse events (SAEs), treatment related adverse events (AEs), AEs of special interest (AESIs) and AEs leading to study drug discontinuation
To assess safety and tolerability of metreleptin
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 posted16 Jul 2024
- Study start23 May 2025
- Primary completion11 Feb 2026
- Study completion11 Feb 2026
Why it stopped: This clinical trial was closed early due to no longer being a requirement to the European Medicines Agency.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Generalized LipodystrophyStudies of the same condition, open and under way.
- 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
- Not applicable
- Intervention model
- Single group
- Model description
- Single Group Assignment
- Masking
- None (open label)
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- Amryt Pharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06502990
- Sponsor's protocol ID
- APL-20
- Other IDs
- 2022-501781-22-00
- Status last verified
- Jul 2026
- First submitted
- 9 Jul 2024
- Last updated
- 22 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No