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| ID | Type | Description | Link |
|---|---|---|---|
| 2022-501781-22-00 | EU Trial (CTIS) Number |
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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
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Metreleptin | Experimental | Metreleptin [Recombinant-methionyl human Leptin; rmetHuLeptin] for daily injection is a sterile, white, solid lyophilised cake |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Metreleptin | Drug | 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 |
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| Measure | Description | Time Frame |
|---|---|---|
| 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 | 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 | 12 months |
| Measure | Description | Time Frame |
|---|---|---|
| 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 | 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 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Janet Boylan | Contact | +3905212791 | clinicaltrials_info@chiesi.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UZ Leuven | Recruiting | Leuven | 3000 | Belgium |
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| ID | Term |
|---|---|
| D052497 | Lipodystrophy, Congenital Generalized |
| ID | Term |
|---|---|
| D008052 | Lipid Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| ID | Term |
|---|---|
| C415771 | metreleptin |
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Single Group Assignment
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To evaluate the efficacy of metreleptin treatment in patients with GL |
| 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 | 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 | 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 | 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 | 12 months |
| Hôpital Necker - Enfants Malades | Recruiting | Paris | 75015 | France |
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| Assistance Publique-Hopitaux de Paris (AP-HP) - Hopital Universitaire Robert-Debre | Recruiting | Paris | France |
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| Universitätsklinikum Hamburg-Eppendorf | Not yet recruiting | Hamburg | 20251 | Germany |
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| Universitaetsklinikum Ulm - Klinik fuer Kinder- und Jugendmedizin | Recruiting | Ulm | 89075 | Germany |
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| IRCCS Ospedale Pediatrico Bambino Gesù | Recruiting | Chieti | 66100 | Italy |
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| Azienda Ospedaliera Universitaria "Federico II" | Recruiting | Naples | 80131 | Italy |
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| Azienda Ospedaliero Universitaria Maggiore della Carità di Novara | Recruiting | Novara | 28100 | Italy |
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| Azienda Ospedaliero-Universitaria di Parma | Recruiting | Parma | 43126 | Italy |
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| Azienda Ospedaliero Universitaria Pisana | Recruiting | Pisa | 56124 | Italy |
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| IRCCS Ospedale Pediatrico Bambino Gesù | Recruiting | Rome | 00165 | Italy |
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| Ospedale Filippo Del Ponte Varese - ASST Sette Laghi | Recruiting | Varese | 21100 | Italy |
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| D008060 | Lipodystrophy |
| D012875 | Skin Diseases, Metabolic |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D052439 | Lipid Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |