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

NCT02654977 | Completed Phase 2 Results

• 1 other identifier: HUM00093399
• Study Type: interventional
• Target: 11
• Locations: 1 country
• Sites: 1

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.

Conditions

Familial Partial Lipodystrophy; Nonalcoholic Steatohepatitis; NAFLD

Outcome Measures

Primary Outcomes (1)

• Percent Change in Fasting Triglyceride Levels

  Fasting triglyceride levels after 5 years on metreleptin, measured in mg/dL are compared to fasting triglyceride levels at baseline). The median percent increase (positive numbers) or decrease from baseline is shown, along with the full range.

  5 years on metreleptin or last observation carried forward

Secondary Outcomes (1)

• Percent Change in Hemoglobin A1c Levels

  5 years on metreleptin or last observation carried forward

Study Arms (1)

Metreleptin

EXPERIMENTAL

Metreleptin open-label

Drug: Metreleptin

Interventions

Metreleptin DRUG

MyaLept (Recombinant-methionyl Human Leptin (r-metHuLeptin) METRELEPTIN) subcutaneous injections

Also known as: MyaLept

Metreleptin

Eligibility Criteria

• Age: 5 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• University of Michigan: lead

Study Sites (1)

University of Michigan

Ann Arbor, Michigan, 48105, United States

Location

MeSH Terms

Conditions

Lipodystrophy, Familial Partial; Non-alcoholic Fatty Liver Disease

Interventions

metreleptin

Condition Hierarchy (Ancestors)

Laminopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipodystrophy; Skin Diseases, Metabolic; Skin Diseases; Skin and Connective Tissue Diseases; Lipid Metabolism, Inborn Errors; Lipid Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Fatty Liver; Liver Diseases; Digestive System Diseases

Limitations and Caveats

Definitive conclusions cannot be drawn from sample sizes as small as the four individuals who completed the trial period.

Results Point of Contact

• Title: Dr. Elif Oral
• Organization: University of Michigan

eliforal@umich.edu

734-615-7271

Study Officials

• Elif A Oral, MD

  University of Michigan

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor, Internal Medicine

Study Record Dates

• First Submitted: January 20, 2015
• First Posted: January 13, 2016
• Study Start: September 29, 2015
• Primary Completion: November 14, 2019
• Study Completion: November 14, 2019
• Last Updated: November 24, 2020
• Results First Posted: November 24, 2020

Record last verified: 2020-10

Locations

US (1)