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Immunogenicity of Metreleptin in Patients With Generalized Lipodystrophy

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ClinicalTrials.gov Identifier: NCT04026178
Recruitment Status : Recruiting
First Posted : July 19, 2019
Last Update Posted : July 19, 2019
Sponsor:
Information provided by (Responsible Party):
Aegerion Pharmaceuticals, Inc.

Brief Summary:
MYALEPT™ (metreleptin) has been approved as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy (MYALEPT Prescribing Information). This study is a multicenter, open-label, Phase 4 trial to provide an assessment of the immunogenicity associated with metreleptin and of any major potential risks due to development of antibodies to metreleptin. The study is being conducted to comply with a postmarketing requirement.

Condition or disease Intervention/treatment Phase
Generalized Lipodystrophy Drug: Metreleptin Phase 4

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A 36-Month, Multicenter, Open Label Phase 4 Study to Evaluate the Immunogenicity of Daily SC Metreleptin Treatment in Patients With Generalized Lipodystrophy
Actual Study Start Date : November 14, 2018
Estimated Primary Completion Date : January 2022
Estimated Study Completion Date : January 2022


Arm Intervention/treatment
Experimental: Metreleptin
Subjects will receive prescribed dosage of metreleptin as indicated in the USPI Patients (males and females) ≤ 40 kg: 0.06mg/kg Male patients > 40 kg: 2.5mg Female patients > 40 kg: 5mg
Drug: Metreleptin
Subjects will receive prescribed dosage of metreleptin as indicated in the USPI




Primary Outcome Measures :
  1. Percentage of participants who develop anti-metreleptin/leptin antibodies at any timepoint after treatment with metreleptin [ Time Frame: Month 36 ]
    Antibody titers are determined by the ADA Binding Assay

  2. Percentage of participants who develop anti-metreleptin/leptin antibodies with neutralizing activity at any timepoint after treatment with metreleptin [ Time Frame: Month 36 ]

    In vitro neutralizing activity is determined in samples that tested positive for metreleptin/leptin-antibody by the ADA Binding Assay by two methods:

    • Method A: Cell-Based Assay
    • Method B: Receptor-Binding Assay


Secondary Outcome Measures :
  1. Percentage of participants with SAEs, AESIs or adverse events leading to discontinuation [ Time Frame: Baseline and Months 1, 2, 4, 6, 9, 12, 18, 24, 30, 36 ]
    Percentage of participants with SAEs, AESIs or adverse events leading to discontinuation

  2. Percentage of participants with neutralizing or non-neutralizing antibodies who also show one or several of: a. Significant change of HbA1c b. Significant change of triglycerides c. Concomitant SAEs, AESIs or AEs leading to discontinuation [ Time Frame: Baseline and Months 1, 2, 4, 6, 9, 12, 18, 24, 30, 36 ]

    Percentage of participants with neutralizing or non-neutralizing anti-metroleptin/leptin antibodies who also show one or several of:

    1. Significant change of hemoglobin A1c
    2. Significant change of triglycerides
    3. Concomitant SAEs, AESIs or adverse events leading to discontinuation


Other Outcome Measures:
  1. Percentage of participants who show a significant change in hemoglobin A1c [ Time Frame: Baseline and Months 1, 2, 4, 6, 9, 12, 18, 24, 30, 36 ]
    Absolute change of Hemoglobin A1c from baseline level

  2. Percentage of participants who show a significant change in fasting triglycerides [ Time Frame: Baseline and Months 1, 2, 4, 6, 9, 12, 18, 24, 30, 36 ]
    Percentage change of triglycerides from baseline level



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Ages Eligible for Study:   1 Year and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Provision of informed consent prior to any study specific procedures. If <18 years of age, has a parent or guardian able to read, understand, and sign the Informed Consent Form (ICF) and a Child Assent form, communicate with the Investigator, and understand and comply with protocol requirements. Adolescent patients must also read and understand the Child Assent Form. If the child is too young or unable to read, then the Child Assent form must be explained to the child.
  2. Female and/or male patients ≥1 years of age.
  3. Physician-confirmed diagnosis of congenital or acquired generalized lipodystrophy and will begin treatment with MYALEPT for the first time.
  4. Negative pregnancy test (urine or serum) for female patients of childbearing potential.
  5. Female patients of childbearing potential must be 1 year postmenopausal, surgically sterile, or be willing to use an acceptable method of contraception (an acceptable method of contraception is defined as a barrier method in conjunction with a spermicide) for the duration of the study (from the time they sign consent). In addition, oral contraceptives, approved contraceptive implant, long-term injectable contraception, intrauterine device, or tubal ligation are allowed. Oral contraception alone is not acceptable; additional barrier methods in conjunction with spermicide must be used.
  6. Male patients must be surgically sterile or be willing to use an acceptable method of contraception (defined as barrier methods in conjunction with spermicides) for the duration of the study (from the time they sign consent).
  7. Patients who are blood donors should not donate blood during the study and for 3 months following their last dose of metreleptin.

Exclusion Criteria:

  1. Involvement in the planning and/or conduct of the study (applies to both Aegerion staff and/or staff at the study site.)
  2. Previous treatment with metreleptin.
  3. Participation in another clinical study with an investigational product during the last 6 months.
  4. Patients with prior severe hypersensitivity reactions to metreleptin or to any of the product components.
  5. Known to have tested positive for human immunodeficiency virus, are immunocompromised, or are receiving immunomodulatory drugs.
  6. Known history of drug or alcohol abuse within 1 year of screening.
  7. Creatinine clearance <30 mL/min using institutional standards:

    e.g., calculated using Cockcroft-Gault formula for patients ≥18 years of age; calculated using Schwartz equation for patients <18 years of age.

  8. For women only - currently pregnant (confirmed with positive pregnancy test) or breast-feeding.
  9. Any condition where, in the opinion of the Investigator, participation in this study may pose a significant risk to the patient or could render the patient unable to successfully complete the study.

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): NCT04026178


Contacts
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Contact: Michael Pankratz, MBA, BSN 857-242-5141 michael.pankratz@aegerion.com

Locations
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United States, Michigan
University of Michigan Recruiting
Ann Arbor, Michigan, United States, 48109
Principal Investigator: Elif Oral         
United States, Pennsylvania
Childrens Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19104
Principal Investigator: Shana McCormack         
United States, Texas
University Texas Southwestern INT Recruiting
Dallas, Texas, United States, 75390
Principal Investigator: Abhimanyu Garg, M.D.         
Sponsors and Collaborators
Aegerion Pharmaceuticals, Inc.
Investigators
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Study Chair: Oscar Segurado, M.D. Aegerion Pharmaceuticals, Inc.

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Responsible Party: Aegerion Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT04026178     History of Changes
Other Study ID Numbers: AEGR-734-401
First Posted: July 19, 2019    Key Record Dates
Last Update Posted: July 19, 2019
Last Verified: July 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: only IPD that underly results in a publication
Supporting Materials: Study Protocol
Informed Consent Form (ICF)
Time Frame: A copy of data generated at the study site will be provided to individual sites after the overall publication by the sponsor is completed and submitted

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Aegerion Pharmaceuticals, Inc.:
Immunogenicity
Antibodies
Lipodystrophy

Additional relevant MeSH terms:
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Lipodystrophy, Congenital Generalized
Lipodystrophy
Skin Diseases, Metabolic
Skin Diseases
Lipid Metabolism Disorders
Metabolic Diseases
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn