Metreleptin for Generalized Lipodystrophy

Recruiting at 7 trial locations

Overseen By Ambika Ashraf, MD

Age: Any Age

Sex: Any

Trial Phase: Phase 4

Sponsor: Aegerion Pharmaceuticals, Inc.

Must be taking: Myalept

Must not be taking: Immunomodulatory drugs

Disqualifiers: HIV, Immunocompromised, Drug abuse, others

Stay on Your Current Meds You can continue your current medications while participating

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 12

Duration: 36 months

10 Participants Needed

What You Need to Know Before You Apply

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.

Who Is on the Research Team?

Janet Boylan

Principal Investigator

Aegerion Pharmaceuticals, Inc.

Are You a Good Fit for This Trial?

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

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.

I am a male either surgically sterile or willing to use barrier contraception during the study.

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.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive daily SC metreleptin treatment to assess immunogenicity and potential antibody development

36 months

Monthly visits for the first 12 months, then every 6 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Metreleptin

Trial Overview The 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.

How Is the Trial Designed?

Group I: Metreleptin Experimental Treatment 1 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

Who Is Running the Clinical Trial?

Aegerion Pharmaceuticals, Inc.

Lead Sponsor

Trials

20

Recruited

1,630,000+