Open-label Study to Evaluate Metreleptin in Patients With Partial Lipodystrophy

ConditionPartial Lipodystrophy

Trial statusRecruiting

Trial phasePhase 4

Trial typeInterventional

Biological sexAll

Age12+

SponsorAmryt Pharma

About this trial

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

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Confirmed diagnosis of familial or acquired partial lipodystrophy

Disqualifiers

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.

Trial design

Design model

Single group

Treatments tested in this trial

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

Treatment groups

12 Participants

are divided into 1 treatment group

Group A: MetreleptinExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

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%.

To evaluate the efficacy (HbA1c) of metreleptin treatment in patients with PL

Time frame

12 months

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)

To evaluate the efficacy (TG) of metreleptin treatment in patients with PL

Time frame

12 months

Secondary outcomes

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%.

To evaluate the long-term efficacy of metreleptin treatment in patients with PL

Time frame

24 months

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).

To evaluate the long-term efficacy of metreleptin treatment in patients with PL

Time frame

24 months

Change from baseline in liver volume at Month 12 and Month 24

To assess changes in liver volume.

Time frame

12 months and 24 months

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

To evaluate the safety of metreleptin treatment in patients with PL

Time frame

24 months