Compassionate Use of Metreleptin in Previously Treated People With Partial Lipodystrophy

• Register: ClinicalTrials.gov
• Last refreshed on: 4 March 2024
• Main ID: NCT02262806
• Date of registration: 09/10/2014
• Prospective Registration: Yes
• Primary sponsor: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
• Public title: Compassionate Use of Metreleptin in Previously Treated People With Partial Lipodystrophy
• Scientific title: Compassionate Use of Metreleptin in Previously-Treated Patients With Partial Lipodystrophy
• Date of first enrolment: October 14, 2014
• Target sample size: 30
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT02262806
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Rebecca J Brown, M.D.
• Affiliation: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Key inclusion & exclusion criteria

- 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.



Age minimum: 6 Months
Age maximum: 98 Years
Gender: All

Health Condition(s) or Problem(s) studied

Lipodystrophy

Hyperlipidemia

Diabetes

Intervention(s)

Drug: Metreleptin

Primary Outcome(s)

Serum hemoglobin A1C [Time Frame: every 6-12 months]

Serum triglycerides [Time Frame: every 6-12 months]

Secondary ID(s)

150002

15-DK-0002

Source(s) of Monetary Support

Please refer to primary and secondary sponsors