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Open-label Study to Evaluate Metreleptin in Children Under 6 Years of Age With Generalised Lipodystrophy

ConditionGeneralized Lipodystrophy
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
AgeUp to 5
SponsorAmryt Pharma

About this trial

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

This trial does not accept healthy volunteers

Qualifiers

Confirmed diagnosis of Generalised Lipodystrophy

Metreleptin treatment naive

Disqualifiers

Weight <9 kg at Screening (Visit 1)

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

15 Participants
are divided into 1 treatment group
Group A: MetreleptinExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

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

Time frame
12 months
2

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

Secondary outcomes

1

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
2

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
3

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
4

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

Time frame
12 months

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Amryt Pharma

Lead sponsor

Source ClinicalTrials.gov
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