Access to Metreleptin for Patients with Generalized Lipodystrophy
Compassionate Use of Metreleptin in Previously-Treated Patients With Generalized Lipodystrophy
This study is trying to provide the drug metreleptin to people with generalized lipodystrophy who need it but can't get it anywhere else.
Quick facts
| Phase | Phase 3 |
|---|---|
| Study type | Interventional |
| Enrollment | 30 (estimated) |
| Ages | 6 Months to 98 Years |
| Sex | All |
| Sponsor | National Institutes of Health Clinical Center (CC) NIH |
| Locations | 1 site (Bethesda, Maryland) |
| Trial ID | NCT02262832 on ClinicalTrials.gov |
What this trial studies
This study aims to provide access to the drug metreleptin for individuals with generalized lipodystrophy who have previously received the treatment but cannot obtain it through other means. Participants, aged 6 months and older, will visit the NIH Clinical Center approximately every six months during the first year and then annually. During these visits, they will receive metreleptin for daily injections and undergo various medical assessments, including blood tests and physical exams, to evaluate the drug's safety and effectiveness.
Who should consider this trial
Good fit: Ideal candidates include individuals aged 6 months and older diagnosed with generalized lipodystrophy who cannot access metreleptin through other approved channels.
Not a fit: Patients who can obtain metreleptin through approved or compassionate use mechanisms in their home country will not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly improve metabolic health and quality of life for patients with generalized lipodystrophy.
How similar studies have performed: Previous studies have shown that metreleptin effectively improves metabolic and endocrine abnormalities in patients with lipodystrophy, indicating a promising approach.
Eligibility criteria
Show full inclusion / exclusion criteria
* INCLUSION CRITERIA: 1. Age greater than or equal to 6 months. 2. Generalized lipodystrophy (either congenital or acquired). 3. Those who cannot obtain metreleptin through approved or compassionate use mechanisms in their home country. EXCLUSION CRITERIA: 1. Availability of metreleptin to the patient either as an approved drug, or through local compassionate use or expanded access programs. 2. Known HIV infection or HIV-associated lipodystrophy. 3. Any medical condition or medication that will increase risk to the subject. 4. Current alcohol or substance abuse. 5. Subjects who have a known hypersensitivity to E. coli derived proteins (as leptin is derived from such proteins).
Where this trial is running
Bethesda, Maryland
- National Institutes of Health Clinical Center — Bethesda, Maryland, United States (Recruiting)
Study contacts
- Principal investigator: Rebecca J Brown, M.D. — National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
- Study coordinator: Megan S Startzell, R.N.
- Email: megan.startzell@nih.gov
- Phone: (301) 402-6371
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.