Clinical trial NCT06502990: Open-label Study to Evaluate Metreleptin in Children Under 6 Years of Age With Generalised Lipodystrophy
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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Generalized Lipodystrophy
Keywords: Metreleptin
Lead sponsor: Amryt Pharma
Sponsor class: INDUSTRY
First posted: 2024-07-16
Enrollment: 15
Sex: ALL
Maximum age (years): 5
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Single Group Assignment
Primary purpose: TREATMENT

Brief 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 criteria: Inclusion Criteria:

* Confirmed diagnosis of Generalised Lipodystrophy
* Metreleptin treatment naive

Exclusion Criteria:

* Weight \<9 kg at Screening (Visit 1)

Primary outcomes:
measure: 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
timeFrame: 12 months
measure: Absolute change from baseline in glycated haemoglobin (HbA1c) at Month 12 for subjects with HbA1c ≥6.5% at baseline
timeFrame: 12 months
Secondary outcomes:
measure: 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
timeFrame: 12 months
measure: 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
timeFrame: 12 months
measure: 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
timeFrame: 12 months
measure: 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
timeFrame: 12 months
measure: Change from baseline in liver volume and liver span as assessed by ultrasound at each post-baseline visit through Month 12
timeFrame: 12 months
measure: 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
timeFrame: 12 months
Other outcomes:
Locations:
city: Leuven
country: Belgium
facility: UZ Leuven
city: Paris
country: France
facility: Hôpital Necker - Enfants Malades
city: Hamburg
country: Germany
facility: Universitätsklinikum Hamburg-Eppendorf
city: Ulm
country: Germany
facility: Universitaetsklinikum Ulm - Klinik fuer Kinder- und Jugendmedizin
city: Chieti
country: Italy
facility: IRCCS Ospedale Pediatrico Bambino Gesù
city: Naples
country: Italy
facility: Azienda Ospedaliera Universitaria "Federico II"
city: Novara
country: Italy
facility: Azienda Ospedaliero Universitaria Maggiore della Carità di Novara
city: Parma
country: Italy
facility: Azienda Ospedaliero-Universitaria di Parma
city: Pisa
country: Italy
facility: Azienda Ospedaliero Universitaria Pisana
city: Rome
country: Italy
facility: IRCCS Ospedale Pediatrico Bambino Gesù
city: Varese
country: Italy
facility: Ospedale Filippo Del Ponte Varese - ASST Sette Laghi
Study officials:
References: