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

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ClinicalTrials.gov Identifier: NCT02654977
Recruitment Status : Active, not recruiting
First Posted : January 13, 2016
Last Update Posted : October 22, 2018
Sponsor:
Information provided by (Responsible Party):
Elif Oral, University of Michigan

Brief Summary:
The study investigators' aim is to determine the long term safety and efficacy of Metreleptin (Myalept,) in promoting amelioration of metabolic abnormalities in patients with all forms of partial lipodystrophy. Patients will be offered this protocol under the following condition: Subjects have completed University of Michigan research protocol MB002-014 and have shown improved clinical benefit as judged by clinical criteria set forth in this protocol.

Condition or disease Intervention/treatment Phase
Familial Partial Lipodystrophy Nonalcoholic Steatohepatitis NAFLD Drug: Metreleptin Phase 2

Detailed Description:

Leptin is now an approved therapeutic in the form of Myalept in patients with generalized forms of lipodystrophy. However, it is still under investigation for patients with partial forms of the disease based on FDA decision on February 24, 2014. The study investigators have been carrying out a protocol in patients with partial lipodystrophy, specifically familial partial lipodystrophy. There have been a number of patients who have been treated under this protocol who are not covered by the currently approved label, but who have experienced significant clinical benefit.

This study would allow continued treatment of patients with partial forms of lipodystrophy who volunteered and completed treatment under the investigators' ongoing protocol (MB002-014) and who have derived significant clinical benefit as judged by an amelioration of their HbA1c, triglyceride levels, and/or reduction in their baseline diabetes or lipid therapies that affect quality of life.


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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 11 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: CLINICAL PROTOCOL to Investigate the Long-term Safety and Efficacy of Recombinant Human Leptin (METRELEPTIN) in Various Forms of Partial Lipodystrophy
Study Start Date : April 2015
Estimated Primary Completion Date : February 2025
Estimated Study Completion Date : February 2025


Arm Intervention/treatment
Experimental: Metreleptin
Metreleptin open-label
Drug: Metreleptin
MyaLept (Recombinant-methionyl Human Leptin (r-metHuLeptin) METRELEPTIN) subcutaneous injections
Other Name: MyaLept




Primary Outcome Measures :
  1. Improvement in Non alcoholic fatty liver disease [ Time Frame: 5 years ]
    Improvement in total NAS score by liver biopsy


Secondary Outcome Measures :
  1. Improvement in triglyceride levels [ Time Frame: 5 years ]
    reduction in triglyceride levels

  2. Improvement in hemoglobin A1c values [ Time Frame: 5 years ]
    reduction in hemoglobin A1c



Information from the National Library of Medicine

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Ages Eligible for Study:   5 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02654977


Locations
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United States, Michigan
University of Michigan
Ann Arbor, Michigan, United States, 48105
Sponsors and Collaborators
University of Michigan
Investigators
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Principal Investigator: Elif A Oral, MD University of Michigan

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Responsible Party: Elif Oral, Associate Professor, Internal Medicine, University of Michigan
ClinicalTrials.gov Identifier: NCT02654977     History of Changes
Other Study ID Numbers: HUM00093399
First Posted: January 13, 2016    Key Record Dates
Last Update Posted: October 22, 2018
Last Verified: October 2018

Additional relevant MeSH terms:
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Fatty Liver
Non-alcoholic Fatty Liver Disease
Lipodystrophy
Lipodystrophy, Familial Partial
Liver Diseases
Digestive System Diseases
Skin Diseases, Metabolic
Skin Diseases
Lipid Metabolism Disorders
Metabolic Diseases