Evaluating Metreleptin for Young Children with Lipodystrophy
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
PHASE3 · Amryt Pharma · NCT06502990
This study is testing if a medication called metreleptin can help young children with a rare condition called lipodystrophy and its related health issues.
Quick facts
| Phase | PHASE3 |
|---|---|
| Study type | Interventional |
| Enrollment | 15 (estimated) |
| Ages | N/A to 5 Years |
| Sex | All |
| Sponsor | Amryt Pharma (industry) |
| Locations | 12 sites (Leuven and 11 other locations) |
| Trial ID | NCT06502990 on ClinicalTrials.gov |
What this trial studies
This open-label, Phase 3b study aims to assess the effectiveness, safety, and pharmacokinetics of metreleptin in children under 6 years of age diagnosed with generalized lipodystrophy, along with associated conditions such as diabetes mellitus and hypertriglyceridemia. The study will involve administering metreleptin to treatment-naive patients to gather data on its impact. The trial is designed to provide insights into how this treatment can help manage the symptoms and complications of this rare condition in a vulnerable population.
Who should consider this trial
Good fit: Ideal candidates for this study are children under 6 years of age with a confirmed diagnosis of generalized lipodystrophy who have not previously received metreleptin treatment.
Not a fit: Patients weighing less than 9 kg at screening will not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly improve metabolic control and quality of life for young patients with generalized lipodystrophy.
How similar studies have performed: While this approach is relatively novel, previous studies on metreleptin in older populations have shown promising results, suggesting potential for success in this younger demographic.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Confirmed diagnosis of Generalised Lipodystrophy * Metreleptin treatment naive Exclusion Criteria: * Weight \<9 kg at Screening (Visit 1)
Where this trial is running
Leuven and 11 other locations
- UZ Leuven — Leuven, Belgium (RECRUITING)
- Hôpital Necker - Enfants Malades — Paris, France (RECRUITING)
- Assistance Publique-Hopitaux de Paris (AP-HP) - Hopital Universitaire Robert-Debre — Paris, France (RECRUITING)
- Universitätsklinikum Hamburg-Eppendorf — Hamburg, Germany (NOT_YET_RECRUITING)
- Universitaetsklinikum Ulm - Klinik fuer Kinder- und Jugendmedizin — Ulm, Germany (RECRUITING)
- IRCCS Ospedale Pediatrico Bambino Gesù — Chieti, Italy (RECRUITING)
- Azienda Ospedaliera Universitaria "Federico II" — Naples, Italy (RECRUITING)
- Azienda Ospedaliero Universitaria Maggiore della Carità di Novara — Novara, Italy (RECRUITING)
- Azienda Ospedaliero-Universitaria di Parma — Parma, Italy (RECRUITING)
- Azienda Ospedaliero Universitaria Pisana — Pisa, Italy (RECRUITING)
- IRCCS Ospedale Pediatrico Bambino Gesù — Rome, Italy (RECRUITING)
- Ospedale Filippo Del Ponte Varese - ASST Sette Laghi — Varese, Italy (RECRUITING)
Study contacts
- Study coordinator: Janet Boylan
- Email: clinicaltrials_info@chiesi.com
- Phone: +3905212791
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Generalized Lipodystrophy, Metreleptin