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| Sponsor: | The Cooper Health System |
|---|---|
| Information provided by: | The Cooper Health System |
| ClinicalTrials.gov Identifier: | NCT00683189 |
Purpose
Objectives/Purpose:
To determine the safety and efficacy of a Vitamin K (Vit K) antagonist (warfarin) in treating Metachromatic Leukodystrophy (MLD).
| Condition | Intervention |
|---|---|
|
Metachromatic Leukodystrophy |
Drug: Warfarin |
| Study Type: | Interventional |
| Study Design: | Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy |
| Estimated Enrollment: | 10 |
| Study Start Date: | June 2007 |
| Study Completion Date: | May 2008 |
| Primary Completion Date: | May 2008 (Final data collection date for primary outcome measure) |
Hypothesis:
Vit K has an essential role in biosynthesis of sulfatides and other sphingolopids in the brain. Administering warfarin, a Vit K antagonist, may ameliorate the phenotype in MLD by decreasing t he amount of sphingolipid storage in the neuronal cells.
Study Design Prospective: we will enroll eligible consenting subjects into the study. The study will not include a control group and the families and treating physicians are informed administration of the drug.
Eligibility| Ages Eligible for Study: | 1 Year to 10 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Contacts and Locations| United States, New Jersey | |
| Cooper University Hospital | |
| Camden, New Jersey, United States, 08103 | |
| Study Director: | Paola Leone, Ph.D. | UMDNJ/SOM |
| Principal Investigator: | Mitra Assadi, M.D. | Cooper University Hospital |
More Information
| Responsible Party: | Mitra Assadi, M.D., Cooper University Hospital |
| ClinicalTrials.gov Identifier: | NCT00683189 History of Changes |
| Other Study ID Numbers: | RP#07/063 |
| Study First Received: | May 21, 2008 |
| Last Updated: | March 18, 2011 |
| Health Authority: | United States: Institutional Review Board |
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MLD |
|
Leukodystrophy, Metachromatic Hereditary Central Nervous System Demyelinating Diseases Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases Nervous System Diseases Sulfatidosis Sphingolipidoses Lysosomal Storage Diseases, Nervous System Leukoencephalopathies Demyelinating Diseases |
Metabolism, Inborn Errors Genetic Diseases, Inborn Lipidoses Lipid Metabolism, Inborn Errors Lysosomal Storage Diseases Metabolic Diseases Lipid Metabolism Disorders Warfarin Anticoagulants Hematologic Agents Therapeutic Uses Pharmacologic Actions |