Partial Lipodystrophy
Conditions
Keywords
Metreleptin
Brief summary
This is an Open Label, Phase IV, Post Authorisation Study to Evaluate the Efficacy, Safety and Immunogenicity of Daily Subcutaneous Metreleptin Treatment in people with Partial Lipodystrophy
Interventions
DRUGMetreleptin
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
Sponsors
Amryt Pharma
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Confirmed diagnosis of familial or acquired partial lipodystrophy
Exclusion criteria
* Treatment with any Investigational Medicinal Product (IMP) within 6 months or 5 times the terminal half-life of the corresponding IMP, whichever is longer, before the screening visit. Other protocol defined inclusion/
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of patients with decrease of at least 0.5% in glycated haemoglobin (HbA1c) at Month 12 compared to Baseline or HbA1c <6.5 % at Month 12, in patients with baseline HbA1c ≥6.5%. | 12 months | To evaluate the efficacy (HbA1c) of metreleptin treatment in patients with PL |
| Number of patients with decrease of at least 30% in triglycerides (TG) at Month 12 compared to Baseline, in patients with baseline TG levels ≥500 mg/dL (5.65 mmol/L) | 12 months | To evaluate the efficacy (TG) of metreleptin treatment in patients with PL |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of patients with decrease of at least 0.5% in HbA1c at Month 24 compared to Baseline or HbA1c <6.5 % at Month 24, in patients with baseline HbA1c ≥6.5%. | 24 months | To evaluate the long-term efficacy of metreleptin treatment in patients with PL |
| Number of patients with decrease of at least 30% in TG levels at Month 24 compared to Baseline, in patients with baseline TG levels ≥500 mg/dL (5.65 mmol/L). | 24 months | To evaluate the long-term efficacy of metreleptin treatment in patients with PL |
| Change from baseline in liver volume at Month 12 and Month 24 | 12 months and 24 months | To assess changes in liver volume. |
| Incidence of, Treatment emergent adverse events (TEAEs), Deaths and other serious adverse events (SAEs), Treatment related adverse events (AEs), AEs of special interest (AESIs) and AEs leading to study drug discontinuation | 24 months | To evaluate the safety of metreleptin treatment in patients with PL |
Countries
France, Germany, Italy, United Kingdom
Contacts
Primary ContactJanet Boylan
Outcome results
None listed