CLINICAL PROTOCOL to Investigate the Long-term Safety and Efficacy of Metreleptin in Various Forms of Partial Lipodystrophy

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02654977
• Date of registration: 20/01/2015
• Prospective Registration: Yes
• Primary sponsor: University of Michigan
• Public title: CLINICAL PROTOCOL to Investigate the Long-term Safety and Efficacy of Metreleptin in Various Forms of Partial Lipodystrophy
• Scientific title: CLINICAL PROTOCOL to Investigate the Long-term Safety and Efficacy of Recombinant Human Leptin (METRELEPTIN) in Various Forms of Partial Lipodystrophy
• Date of first enrolment: September 29, 2015
• Target sample size: 11
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02654977
• 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: Elif A Oral, MD
• Affiliation: University of Michigan

Key inclusion & exclusion criteria

Inclusion Criteria:

- Previously completed study protocol:

o CLINICAL PROTOCOL to investigate the efficacy of recombinant human leptin
(METRELEPTIN) in nonalcoholic steatohepatitis (NASH) or nonalcoholic fatty liver
disease (NAFLD) associated with lipodystrophy, MB002-014 (IRMBED: HUM00058708)

- Demonstrates clinical benefit as defined by meeting at least one of the following
criteria upon completion of the above stated protocols:

- Reduction of HbA1c = 1.0% or,

- Reduction of triglycerides = 30% of baseline or,

- Decrease in insulin requirements = 40% or,

- Reduction in total NASH score by = 2 points,

- Significant worsening of metabolic parameters after discontinuation of
Metreleptin if discontinuation has been undertaken.

- A health condition that appears to have significantly improved by metreleptin for
which two independent health care providers make a request to prevent drug
discontinuation. In addition, the PI has to document absence of contraindications
for drug continuation (such as bone marrow suppression).

- Is male or female = 5 years old at baseline.

- Is male, female not of childbearing potential, or meets all the following criteria if
female of childbearing potential (including perimenopausal women who have had a
menstrual period within one year):

- Not breastfeeding

- Negative pregnancy test result (human chorionic gonadotropin, beta subunit) at
baseline (not applicable to hysterectomized females).

- Must practice and be willing to continue to practice appropriate birth control
(defined as a method which results in a low failure rate when use consistently
and correctly, such as implants, injectables, oral contraceptives, some
intrauterine contraceptive devices, sexual abstinence, tubal ligation, or a
vasectomized partner) during the entire duration of metreleptin treatment.

- Has physician-confirmed lipodystrophy as defined by evidence of partial (limbs) loss
of body fat outside the range of normal variation.

- If = 18 years of age, is able to read, understand and sign the University of Michigan
institutional review board (IRBMED) approved informed consent form (ICF), communicate
with study physician and study team, understand and comply with protocol requirements.

- If < 18 and = 7 years of age, is able to read, understand and sign the appropriate
University of Michigan IRBMED approved assent form and has a parent or legal guardian
that is able to read, understand and sign the ICF.

- If < 7 and = 5 years of age or unable to read, the appropriate assent form must be
explained to the child.

- If previously treated with thiazolidinediones or Vitamin E, stable dose of these
medications for at least 3 months.

Exclusion Criteria:

- Presence of advanced liver disease (as evidenced by abnormal synthetic function,
abnormal partial thromboplastin time or albumin).

- Evidence of other etiologies of viral hepatitis.

- Presence of clinically significant hematologic abnormalities (such as neutropenia
and/or lymphadenopathy).

- Presence of HIV infection.

- Inability to give informed consent.

- Presence of end stage renal disease, any type of active cancer, or >class 2 congestive
heart failure ((New York Heart Association Functional Classification System), based on
medical history and physical examination.

- Has known allergies to E. coli-derived proteins or hypersensitivity to any component
of metreleptin treatment.

- Any other condition in the opinion of the investigators that may impede successful
data collection.



Age minimum: 5 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Nonalcoholic Steatohepatitis

Familial Partial Lipodystrophy

NAFLD

Intervention(s)

Drug: Metreleptin

Primary Outcome(s)

Percent Change in Fasting Triglyceride Levels [Time Frame: 5 years on metreleptin or last observation carried forward]

Secondary Outcome(s)

Percent Change in Hemoglobin A1c Levels [Time Frame: 5 years on metreleptin or last observation carried forward]

Secondary ID(s)

HUM00093399

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 24/11/2020
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02654977