Status:
ACTIVE_NOT_RECRUITING
Compassionate Use of Metreleptin in Previously Treated People With Partial Lipodystrophy
Lead Sponsor:
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Conditions:
Diabetes
Lipodystrophy
Eligibility:
All Genders
6-98 years
Phase:
PHASE2
Brief Summary
Background: \- Partial lipodystrophy can cause high blood fat levels and resistance to insulin. This can lead to health problems including diabetes. Researchers have found that the drug metreleptin i...
Detailed Description
Leptin is an adipocyte-derived hormone that can be thought of as a signal from adipose tissue to the rest of the body conveying information about long-term nutritional status. Patients with lipodystro...
Eligibility Criteria
Inclusion
- INCLUSION CRITERIA
- Age greater than or equal to 6 months
- Partial lipodystrophy (either genetic or acquired)
- Previously or currently treated with metreleptin under NIH study 02-DK-0022 and/or NIH study 13-DK-0057.
- Documented metabolic benefit from prior or current metreleptin treatment, defined as one or more of the following:
- TG reduction greater than or equal to 30% OR
- HbA1c reduction greater than or equal to 1% OR
- Decrease in insulin requirements greater than or equal to 40% OR
- Decrease in episodes of pancreatitis OR
- Improvement in steatohepatitis OR
- Withdrawal of metreleptin led to marked worsening of metabolic parameters
- EXCLUSION CRITERIA
- Availability of metreleptin to the patient either as an approved drug, or through local compassionate use or expanded access programs.
- Known HIV infection or HIV-associated lipodystrophy.
- Psychiatric disorder impeding competence or compliance.
- Any medical condition or medication that will increase risk to the subject.
- Current alcohol or substance abuse.
- Subjects who have a known hypersensitivity to E. coli derived proteins.
Exclusion
Key Trial Info
Start Date :
October 14 2014
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
July 31 2027
Estimated Enrollment :
29 Patients enrolled
Trial Details
Trial ID
NCT02262806
Start Date
October 14 2014
End Date
July 31 2027
Last Update
January 8 2026
Active Locations (1)
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1
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892