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Expanded Access Metreleptin Study

Primary Purpose

Familial Partial Lipodystrophy

Status

Available

Phase

Locations

United States

Study Type

Expanded Access

Intervention

Metreleptin

About this trial

This is an expanded access trial for Familial Partial Lipodystrophy

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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
1. Ability to comply with visits and procedures required by program
2. Previously enrolled in study FHA101/MB002-002
3. 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
1. Male or female, over the age of 6 months
2. 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.
3. Women must not be breastfeeding
4. 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.
5. 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
1. Has been diagnosed with generalized lipodystrophy
2. Has been diagnosed with HIV infection
3. 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
4. Has known infectious liver disease
5. Has known allergies to E. coli-derived proteins or hypersensitivity to any component of metreleptin treatment
Other Exclusion Criteria
1. Prisoners or patients who are involuntarily incarcerated.
2. Patients who are compulsorily detained for treatment of either a psychiatric or physical (eg, infectious disease) illness.

Sites / Locations

University of Michigan

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

NCT ID

NCT02404896

First Posted

January 20, 2015

Last Updated

February 10, 2023

Sponsor

University of Michigan

1. Study Identification

Unique Protocol Identification Number

NCT02404896

Brief Title

Expanded Access Metreleptin Study

Official Title

Expanded-Access for the Use of Metreleptin in Patients With Partial Lipodystrophy Associated With Diabetes Mellitus or Hypertriglyceridemia

Study Type

Expanded Access

2. Study Status

Record Verification Date

February 2023

Overall Recruitment Status

Available

Study Start Date

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Primary Completion Date

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Study Completion Date

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3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

University of Michigan

4. Oversight

5. Study Description

Brief Summary

Metreleptin was approved in the United States as adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy in February 2014. The approval was based on results obtained in 2 open-label, investigator-sponsored studies (Studies 991265 and 20010769) conducted at the National Institutes of Health (NIH) to evaluate the safety and efficacy of metreleptin treatment in patients with lipodystrophy and 1 treatment IND (FHA101/MB002-002/MB002-002) conducted by Bristol-Myers Squibb on behalf of AstraZeneca (BMS/AZ) in patients with diabetes mellitus and/or hypertriglyceridemia related to lipodystrophy. These studies enrolled patients with lipodystrophy including both generalized and partial lipodystrophy. Although the marketing authorization restricted the indication to patients with generalized lipodystrophy, meaningful clinical benefit was achieved in a subset of patients with partial lipodystrophy, and these patients from FHA101/MB002-002 form the basis of the request for ongoing treatment under expanded access.

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Familial Partial Lipodystrophy

7. Study Design

8. Arms, Groups, and Interventions

Intervention Type

Drug

Intervention Name(s)

Metreleptin

Other Intervention Name(s)

MyaLept

Intervention Description

Metreleptin open-label treatment

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

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.

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Elif A Oral, MD

Phone

734-615-7271

eliforal@med.umich.edu

First Name & Middle Initial & Last Name or Official Title & Degree

Adam H Neidert, MS

Phone

734-615-0539

aneidert@med.umich.edu

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Elif A Oral, MD

Organizational Affiliation

University of Michigan

Official's Role

Principal Investigator

Facility Information:

Facility Name

University of Michigan

City

Ann Arbor

State/Province

Michigan

ZIP/Postal Code

48105

Country

United States

Individual Site Status

Available

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Elif A Oral, MD

Phone

734-615-7271

eliforal@med.umich.edu

First Name & Middle Initial & Last Name & Degree

Adam H Neidert, MS

Phone

734-615-0539

aneidert@med.umich.edu

First Name & Middle Initial & Last Name & Degree

Nevin A Ajluni, MD

12. IPD Sharing Statement

Learn more about this trial

Expanded Access Metreleptin Study

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