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
Status: Recruiting
Location: See all (12) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
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
Eligibility
Participation Requirements
Sex: All
Maximum Age: 5
Healthy Volunteers: f
View:
• Confirmed diagnosis of Generalised Lipodystrophy
• Metreleptin treatment naive
Locations
Other Locations
Belgium
UZ Leuven
RECRUITING
Leuven
France
Assistance Publique-Hopitaux de Paris (AP-HP) - Hopital Universitaire Robert-Debre
RECRUITING
Paris
Hôpital Necker - Enfants Malades
NOT_YET_RECRUITING
Paris
Germany
Universitätsklinikum Hamburg-Eppendorf
NOT_YET_RECRUITING
Hamburg
Universitaetsklinikum Ulm - Klinik fuer Kinder- und Jugendmedizin
RECRUITING
Ulm
Italy
IRCCS Ospedale Pediatrico Bambino Gesù
RECRUITING
Chieti
Azienda Ospedaliera Universitaria Federico II
RECRUITING
Naples
Azienda Ospedaliero Universitaria Maggiore della Carità di Novara
RECRUITING
Novara
Azienda Ospedaliero-Universitaria di Parma
RECRUITING
Parma
Azienda Ospedaliero Universitaria Pisana
RECRUITING
Pisa
IRCCS Ospedale Pediatrico Bambino Gesù
RECRUITING
Rome
Ospedale Filippo Del Ponte Varese - ASST Sette Laghi
RECRUITING
Varese
Contact Information
Primary
Janet Boylan
clinicaltrials_info@chiesi.com
+3905212791
Time Frame
Start Date: 2025-05-23
Estimated Completion Date: 2026-09-30
Participants
Target number of participants: 15
Treatments
Experimental: Metreleptin
Metreleptin \[Recombinant-methionyl human Leptin; rmetHuLeptin\] for daily injection is a sterile, white, solid lyophilised cake
Related Therapeutic Areas
Sponsors
Leads: Amryt Pharma