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
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
15
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
UZ Leuven
Leuven, Belgium
RECRUITINGHôpital Necker - Enfants Malades
Paris, France
RECRUITINGPercent 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
Time frame: 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
Time frame: 12 months
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
Time frame: 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
Time frame: 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
Time frame: 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
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Assistance Publique-Hopitaux de Paris (AP-HP) - Hopital Universitaire Robert-Debre
Paris, France
RECRUITINGUniversitätsklinikum Hamburg-Eppendorf
Hamburg, Germany
NOT_YET_RECRUITINGUniversitaetsklinikum Ulm - Klinik fuer Kinder- und Jugendmedizin
Ulm, Germany
RECRUITINGIRCCS Ospedale Pediatrico Bambino Gesù
Chieti, Italy
RECRUITINGAzienda Ospedaliera Universitaria "Federico II"
Naples, Italy
RECRUITINGAzienda Ospedaliero Universitaria Maggiore della Carità di Novara
Novara, Italy
RECRUITINGAzienda Ospedaliero-Universitaria di Parma
Parma, Italy
RECRUITINGAzienda Ospedaliero Universitaria Pisana
Pisa, Italy
RECRUITING...and 2 more locations
To evaluate the efficacy of metreleptin treatment in patients with GL
Time frame: 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
Time frame: 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
Time frame: 12 months