Clinical Protocol to Investigate the Efficacy of Recombinant Human Leptin (Metreleptin) in Nonalcoholic Steatohepatitis (NASH) or Nonalcoholic Fatty Liver Disease (NAFLD) Associated With Lipodystrophy

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT01679197
• Date of registration: 31/08/2012
• Prospective Registration: Yes
• Primary sponsor: University of Michigan
• Public title: Clinical Protocol to Investigate the Efficacy of Recombinant Human Leptin (Metreleptin) in Nonalcoholic Steatohepatitis (NASH) or Nonalcoholic Fatty Liver Disease (NAFLD) Associated With Lipodystrophy
• Scientific title: Clinical Protocol to Investigate the Efficacy of Recombinant Human Leptin (Metreleptin) in Nonalcoholic Steatohepatitis (NASH) or Nonalcoholic Fatty Liver Disease (NAFLD) Associated With Lipodystrophy
• Date of first enrolment: October 8, 2012
• Target sample size: 23
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01679197
• Study type: Interventional
• Study design: 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, MS
• Affiliation: University of Michigan

Key inclusion & exclusion criteria

Inclusion Criteria:

- 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
[ßhCG]) 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 generalized (whole
body) or partial (limbs) loss of body fat outside the range of normal variation.

- Alcohol consumption of less than 40 grams/week.

- A liver ultrasound confirming non-alcoholic fatty liver disease, or previous liver
biopsy confirming NASH status.

- If = 18 years of age, is able to read, understand and sign the U of M 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 U
of M 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 PT 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.

- Very poorly controlled diabetes; HbA1c >10%

- Inability to give informed consent.

- Presence of ESRD, 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.

- Active infection (may be transient).

- 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

Fatty Liver Disease, Nonalcoholic

Lipodystrophy

Nonalcoholic Steatohepatitis

Intervention(s)

Drug: Metreleptin

Primary Outcome(s)

Liver Histopathology [Time Frame: 1 year]

Secondary Outcome(s)

Fasting Glucose [Time Frame: 1 year]

Liver Fat by MRI and MR Spectroscopy [Time Frame: 1 year]

Fasting Lipids [Time Frame: 1 year]

Liver Function Tests [Time Frame: 1 year]

Body Weight [Time Frame: 1 year]

Secondary ID(s)

MCRU 2834

R01DK088114-02

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Results

• Results available: Yes
• Date Posted: 14/06/2017
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01679197