- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02404896
Expanded Access Metreleptin Study
Expanded-Access for the Use of Metreleptin in Patients With Partial Lipodystrophy Associated With Diabetes Mellitus or Hypertriglyceridemia
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
Leptin is a naturally occurring hormone and an important regulator of energy homeostasis and other diverse physiological functions. Circulating levels of leptin closely correlate with the amount of adipose tissue present. Metreleptin, a recombinant analogue of human leptin, is a 147-amino acid polypeptide that differs from the human leptin sequence by 1 additional amino acid, methionine, located at the amino-terminal end. Metreleptin has the same physiological effects as leptin, including regulation of energy homeostasis and metabolic function.
The patient group covered under this expanded access submission has demonstrated evidence of clinical benefit from treatment with metreleptin in clinical study FHA101/MB002-002, and needs expanded access to continue treatment without interruption.
New enrollment, subject to approval by the FDA, can be considered on a case-by-case basis.
Study Type
Expanded Access Type
- Individual Patients
Contacts and Locations
Study Contact
- Name: Elif A Oral, MD
- Phone Number: 734-615-7271
- Email: eliforal@med.umich.edu
Study Contact Backup
- Name: Adam H Neidert, MS
- Phone Number: 734-615-0539
- Email: aneidert@med.umich.edu
Study Locations
-
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Michigan
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Ann Arbor, Michigan, United States, 48105
- Available
- University of Michigan
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Contact:
- Elif A Oral, MD
- Phone Number: 734-615-7271
- Email: eliforal@med.umich.edu
-
Contact:
- Adam H Neidert, MS
- Phone Number: 734-615-0539
- Email: aneidert@med.umich.edu
-
Sub-Investigator:
- Nevin A Ajluni, MD
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-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
Inclusion Criteria:
Signed Written Informed Consent
a) Before any program procedures are performed, the details of the program will be described to the patient and the patient will be given a written informed consent document to read. If the patient agrees to participate in the program, consent will be indicated by signing and dating of the informed consent document in the presence of program personnel.
Target Population
- Ability to comply with visits and procedures required by program
- Previously enrolled in study FHA101/MB002-002
Has physician-confirmed partial lipodystrophy and had evidence of benefit with metreleptin treatment based on the following metabolic criteria demonstrated within the last year of metreleptin treatment (if on treatment over 1 year) from baseline values:
- TG reduction ≥ 30% OR
- HbA1c reduction ≥ 1% OR
- Decrease in insulin requirements ≥ 40% OR
- Decrease in episodes of pancreatitis OR
- Improvement in steatohepatitis OR
- Withdrawal of metreleptin led to marked worsening of metabolic parameters
Age and Reproductive Status
- Male or female, over the age of 6 months
- Women of childbearing potential (WOCBP) must have a negative serum or urine pregnancy test (minimum sensitivity 25 IU/L or equivalent units of HCG) within 24 hours prior to the restart of study drug.
- Women must not be breastfeeding
- WOCBP must agree to follow instructions for method(s) of contraception for the duration of treatment with metreleptin plus 5 half-lives of metreleptin plus 30 days (duration of ovulatory cycle) for a total of 6 months post-treatment completion.
- Men who are sexually active with WOCBP must agree to follow instructions for method(s) of contraception for the duration of treatment with metreleptin plus 5 half-lives of the metreleptin plus 90 days (duration of sperm turnover) for a total of 3 months post-treatment completion.
Exclusion Criteria:
Target Disease Exceptions
a) Has acquired lipodystrophy and clinically significant hematologic abnormalities (such as neutropenia and/or lymphadenopathy)
Medical History and Concurrent Diseases
- Has been diagnosed with generalized lipodystrophy
- Has been diagnosed with HIV infection
- Has a clinically significant medical condition that could potentially affect the risk/benefit ratio for metreleptin treatment and/or the personal well-being of the patient, as judged by the primary treating physician
- Has known infectious liver disease
- Has known allergies to E. coli-derived proteins or hypersensitivity to any component of metreleptin treatment
Other Exclusion Criteria
- Prisoners or patients who are involuntarily incarcerated.
- Patients who are compulsorily detained for treatment of either a psychiatric or physical (eg, infectious disease) illness.
Study Plan
How is the study designed?
Collaborators and Investigators
Sponsor
Investigators
- Principal Investigator: Elif A Oral, MD, University of Michigan
Study record dates
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimated)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- HUM00094843
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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