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

• Sponsor: Bristol-Myers Squibb (Industry)
• Overall Status: Completed
• CT.gov ID: NCT00677313
• Collaborator: (none)
• Enrollment: 41
• Locations: 5
• Arm: 1
• Duration (Months): 70
• Patients Per Site: 8.2
• Patients Per Site Per Month: 0.1

Study Details

Study Description

Brief Summary

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

Condition or Disease	Intervention/Treatment	Phase
Lipodystrophy	Drug: metreleptin	N/A

Study Design

• Study Type: Interventional
• Actual Enrollment: 41 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: An Open-Label Treatment Protocol to Provide Metreleptin for the Treatment of Diabetes Mellitus and/or Hypertriglyceridemia Associated With Lipodystrophy
• Study Start Date: Mar 1, 2009
• Actual Primary Completion Date: Jan 1, 2015
• Actual Study Completion Date: Jan 1, 2015

Arms and Interventions

Arm	Intervention/Treatment
Experimental: 1	Drug: metreleptin; metreleptin injection

Outcome Measures

Primary Outcome Measures

1. 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

1. To monitor the safety and tolerability of metreleptin in subjects with lipodystrophy that is associated with diabetes mellitus and/or hypertriglyceridemia [open ended]

2. 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]

Eligibility Criteria

Criteria

• Ages Eligible for Study: 5 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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):

1. Not breastfeeding

2. Negative pregnancy test result

3. 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:

1. Diabetes Mellitus

2. 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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Research Site	Santa Barbara	California	United States
2	Research Site	Chicago	Illinois	United States
3	Research Site	Ann Arbor	Michigan	United States
4	Local Institution	Reno	Nevada	United States	89502
5	Research Site	Greenville	North Carolina	United States

Sponsors and Collaborators

• Bristol-Myers Squibb

Investigators

• Study Director: Bristol-Myers Squibb, Bristol-Myers Squibb

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Bristol-Myers Squibb
• ClinicalTrials.gov Identifier: NCT00677313
• Other Study ID Numbers: MB002-002; FHA101
• First Posted: May 14, 2008
• Last Update Posted: Apr 30, 2015
• Last Verified: Feb 1, 2015

• Keywords provided by Bristol-Myers Squibb: metreleptin; leptin; Amylin; diabetes mellitus; hypertriglyceridemia; lipodystrophy
• Additional relevant MeSH terms: Lipodystrophy; Diabetes Mellitus; Hypertriglyceridemia

Study Results

No Results Posted as of Apr 30, 2015