Metreleptin for Generalized Lipodystrophy

No longer 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial examines how metreleptin, a medication, works for people with generalized lipodystrophy. Generalized lipodystrophy prevents the body from properly storing fat, causing various health issues. The trial aims to determine if the body creates antibodies against metreleptin and to ensure its safety for new users. It targets individuals with a confirmed diagnosis of this condition who have not previously tried metreleptin. Participants should not have received metreleptin treatment before and should not be involved in other studies. As a Phase 4 trial, metreleptin has already received FDA approval and proven effective; this research seeks to understand how it benefits more patients.

Do I have to stop taking my current medications for the trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the study team or your doctor to get a clear answer.

What is the safety track record for metreleptin?

Research has shown that metreleptin, approved by the FDA for treating leptin deficiency in generalized lipodystrophy, has undergone safety studies. In one study, 48 patients with this condition were monitored. Common side effects included low blood sugar in 13% of patients and injection site reactions, such as redness and hives, in 4% of patients. While these side effects are important to note, most participants did not experience them.

Given its approval and existing safety data, metreleptin is generally well-tolerated for its approved use. However, this study aims to further explore any risks related to the development of antibodies against metreleptin. ^{1 2 3 4 5}

Why are researchers enthusiastic about this study treatment?

Metreleptin is unique because it directly addresses leptin deficiency, which is a core issue in generalized lipodystrophy. Unlike other treatments that manage symptoms indirectly, metreleptin acts as a recombinant form of human leptin, effectively replacing the missing hormone. This approach not only targets the root cause of the metabolic problems associated with lipodystrophy but also offers a more tailored treatment option that can improve both metabolic control and quality of life for patients. Researchers are excited about metreleptin because it represents a more precise and potentially more effective way to manage this challenging condition compared to existing options.

What is the effectiveness track record for metreleptin in treating generalized lipodystrophy?

Research shows that metreleptin, the treatment under study in this trial, helps address metabolic problems in people with generalized lipodystrophy. Studies have found that it reduces food intake and improves insulin use, which is crucial for controlling blood sugar levels. One study found that metreleptin can increase both lifespan and quality of life. It is already approved to treat issues from leptin deficiency in these patients, indicating strong evidence of its effectiveness for this condition. ^{6 7 8 9 10}

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+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC7993583/

Effects of Metreleptin on Patient Outcomes and Quality of Life ... Quality-adjusted life-year gains associated with 12 months of treatment with metreleptin were estimated at 0.313 for generalized and 0.117 for partial ...

2. myalept.com myalept.com/hcp/efficacy

Efficacy | Myalept® (metreleptin) for injection | HCP Effects of Metreleptin on patient outcomes and quality of life in generalized and partial lipodystrophy. J Endocrin Soc. 2021;5(4):1-16. IMPORTANT SAFETY ...

3. academic.oup.com academic.oup.com/jcem/article/110/4/e1051/7675889

Effects of Metreleptin in Patients With Generalized ... Metreleptin was shown to be effective in causing sustained improvement in metabolic abnormalities in patients with generalized lipodystrophy (4-6). However, ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT02262832

NCT02262832 | Compassionate Use of Metreleptin in ... This study showed that metreleptin ameliorates metabolic and endocrine abnormalities in lipodystrophy, including reducing food intake, improving insulin ...

5. ncbi.nlm.nih.gov ncbi.nlm.nih.gov/books/NBK610033/

Pharmacoeconomic Review - Metreleptin (Myalepta) - NCBI In the CADTH base case, metreleptin plus supportive care was associated with an incremental cost-effectiveness ratio (ICER) of $5,308,188 per QALY gained ...

6. myalept.com myalept.com/hcp/safety

Safety | Myalept® (metreleptin) for injection | HCP Six patients (13%) had 7 adverse reactions of hypoglycemia. · Two patients (4%) with generalized lipodystrophy reported injection-site erythema and urticaria.

7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10214317/

The Metreleptin Effectiveness and Safety Registry (MEASuRE) One approach to address the paucity of information regarding rare diseases, including lipodystrophy, is to use patient registries—organized ...

8. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/label/2014/125390s004lbl.pdf

MYALEPT Label - accessdata.fda.gov The safety of MYALEPT was evaluated in 48 patients with generalized lipodystrophy in a single-arm, open-label study [see Clinical Studies (14.1)]. The median ...

9. aetna.com aetna.com/cpb/medical/data/800_899/0882.html?ref=counterforcehealth.org

Metreleptin (Myalept) - Medical Clinical Policy Bulletins The safety and effectiveness of Myalept were evaluated in an open-label, single-arm study that included 48 patients with congenital or acquired generalized ...

10. clinicalstudies.info.nih.gov clinicalstudies.info.nih.gov/ProtocolDetails.aspx?id=16-DK-0173

Metreleptin Effectiveness and Safety Registry People with GL have a lack of fatty tissue. Myalept is a drug that is approved by the Food and Drug Administration (FDA) to treat GL. It is also called ...