Metreleptin for Generalized Lipodystrophy

Recruiting in Palo Alto (17 mi)

+7 other locations

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 4

Waitlist Available

Sponsor: Aegerion Pharmaceuticals, Inc.

Stay on Your Current Meds

No Placebo Group

Prior Safety Data

Approved in 2 Jurisdictions

Trial Summary

What is the purpose of this trial?

MYALEPT™ (metreleptin) has been approved as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy (MYALEPT Prescribing Information). This study is a multicenter, open-label, Phase 4 trial to provide an assessment of the immunogenicity associated with metreleptin and of any major potential risks due to development of antibodies to metreleptin. The study is being conducted to comply with a postmarketing requirement.

Research Team

Janet Boylan

Principal Investigator

Aegerion Pharmaceuticals, Inc.

Eligibility Criteria

This trial is for individuals over 1 year old with congenital or acquired generalized lipodystrophy who are starting MYALEPT treatment. Participants need to consent, use contraception if of childbearing potential, and not have used metreleptin before. They shouldn't be at high risk as per the investigator's opinion, have a recent history of substance abuse, HIV positive status, or severe reactions to metreleptin.

Inclusion Criteria

If <18 years of age, has a parent or guardian able to read, understand, and sign the Informed Consent Form (ICF) and a Child Assent form, communicate with the Investigator, and understand and comply with protocol requirements. Adolescent patients must also read and understand the Child Assent Form. If the child is too young or unable to read, then the Child Assent form must be explained to the child

I will not donate blood during the study or for 3 months after my last dose of metreleptin.

I am using or willing to use approved birth control methods if I can have children.

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Exclusion Criteria

Participation in another clinical study with an investigational product during the last 6 months

My kidney function, measured by creatinine clearance, is below 30 mL/min.

I am HIV positive, have a weakened immune system, or am taking drugs that affect my immune system.

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Treatment Details

Interventions

Metreleptin (Leptin Replacement Therapy)

Trial OverviewThe study tests the immune response to Metreleptin in patients with generalized lipodystrophy. It's an open-label Phase 4 trial assessing risks associated with developing antibodies against Metreleptin following its market approval.

Participant Groups

1Treatment groups

Experimental Treatment

Group I: MetreleptinExperimental Treatment1 Intervention

Subjects will receive prescribed dosage of metreleptin as indicated in the USPI Patients (males and females) ≤ 40 kg: 0.06mg/kg Male patients \> 40 kg: 2.5mg Female patients \> 40 kg: 5mg