An Open-Label Treatment Protocol to Provide Metreleptin for the Treatment of Diabetes Mellitus and/or Hypertriglyceridemia Associated With Lipodystrophy

Study Overview
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Brief Summary
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This is an open-label study to provide metreleptin for the treatment of diabetes mellitus and/or hypertriglyceridemia associated with lipodystrophy. This study intends to provide guidance to investigators with respect to identification of appropriate subjects for metreleptin treatment, guidance on metreleptin dosing, and collection of safety and efficacy data following metreleptin treatment in this population

Official Title
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An Open-Label Treatment Protocol to Provide Metreleptin for the Treatment of Diabetes Mellitus and/or Hypertriglyceridemia Associated With Lipodystrophy

Conditions
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Lipodystrophy

Intervention / Treatment
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* Drug: metreleptin


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Is male or female ≥5 years old If female of childbearing potential (including perimenopausal women who have had a menstrual period within one year): Not breastfeeding Negative pregnancy test result Must practice and be willing to continue to practice appropriate birth control (defined as a method which results in a low failure rate, i.e., less than 1% per year, when used 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 the study (Double barrier methods including the use of female diaphragm and male condom with spermicide can also be used.) 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 Has been diagnosed with at least one of the following 2 metabolic disorders: Diabetes Mellitus Hypertriglyceridemia as defined by fasting triglyceride concentrations >200 mg/dL If ≥18 years of age, is able to read, understand, and sign the Informed Consent Form (ICF) and an Authorization to Use and Disclose Protected Health Information form, communicate with the investigator, and understand and comply with protocol requirements If <18 years of age, has a parent or legal guardian to read and understand the ICF and Child Assent Form, communicate with the investigator, and understand and comply with protocol requirements. Adolescent subjects 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. Exclusion Criteria: Has been diagnosed with HIV infection Has known infectious liver disease Has known allergies to E. coli-derived proteins or hypersensitivity to any component of study treatment

Ages Eligible for Study
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Minimum Age: 5 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: 1<br> | Drug: metreleptin<br>* metreleptin injection<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To provide metreleptin, an investigational medication, under a treatment protocol to subjects with lipodystrophy that is associated with diabetes mellitus and/or hypertriglyceridemia |  | open ended | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To monitor the safety and tolerability of metreleptin in subjects with lipodystrophy that is associated with diabetes mellitus and/or hypertriglyceridemia |  | open ended | 
| Information on the efficacy of metreleptin as assessed by its effects on fasting triglyceride concentrations, HbA1c, and fasting glucose concentrations in subjects with lipodystrophy that is associated with diabetes mellitus and/or hypertriglyceridemia |  | open ended | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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metreleptin, leptin, Amylin, diabetes mellitus, hypertriglyceridemia, lipodystrophy