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Sponsored by: |
The Cooper Health System |
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Information provided by: | The Cooper Health System |
ClinicalTrials.gov Identifier: | NCT00683189 |
Objectives/Purpose:
To determine the safety and efficacy of a Vitamin K (Vit K) antagonist (warfarin) in treating Metachromatic Leukodystrophy (MLD).
Condition | Intervention |
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Metachromatic Leukodystrophy |
Drug: Warfarin |
Study Type: | Interventional |
Study Design: | Treatment, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study |
Official Title: | Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy |
Estimated Enrollment: | 10 |
Study Start Date: | June 2007 |
Estimated Study Completion Date: | March 2009 |
Estimated Primary Completion Date: | March 2009 (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.
Ages Eligible for Study: | 1 Year to 10 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Contact: Paola Leone, Ph.D. | 856 566-6334 | leonepa@umdnj.edu |
Contact: Kelly Anderson, MPH | 856-566-6389 | anderskm@umdnj.edu |
United States, New Jersey | |
Cooper University Hospital | Recruiting |
Camden, New Jersey, United States, 08103 | |
Contact: Paola leone, Ph.D. 856-566-6334 leonepa@mac.com | |
Contact: kelly Anderson, MPH 856-566-6389 anderskm@umdnj.edu |
Study Director: | Paola Leone, Ph.D. | UMDNJ/SOM |
Principal Investigator: | Mitra Assadi, M.D. | Cooper University Hospital |
Responsible Party: | Cooper University Hospital ( Mitra Assadi, M.D. ) |
Study ID Numbers: | RP#07/063 |
Study First Received: | May 21, 2008 |
Last Updated: | May 22, 2008 |
ClinicalTrials.gov Identifier: | NCT00683189 History of Changes |
Health Authority: | United States: Institutional Review Board |
MLD |
Lipid Metabolism, Inborn Errors Metachromatic Leukodystrophy Sphingolipidoses Anticoagulants Leukodystrophy, Metachromatic Metabolic Diseases Demyelinating Diseases Lysosomal Storage Diseases Sphingolipidosis Central Nervous System Diseases |
Warfarin Brain Diseases Leukodystrophy Metabolism, Inborn Errors Genetic Diseases, Inborn Brain Diseases, Metabolic, Inborn Lipidoses Metabolic Disorder Lipid Metabolism Disorders Brain Diseases, Metabolic |
Lipid Metabolism, Inborn Errors Sphingolipidoses Anticoagulants Metabolic Diseases Leukodystrophy, Metachromatic Demyelinating Diseases Lysosomal Storage Diseases, Nervous System Lysosomal Storage Diseases Hematologic Agents Nervous System Diseases Sulfatidosis Central Nervous System Diseases |
Warfarin Brain Diseases Hereditary Central Nervous System Demyelinating Diseases Pharmacologic Actions Metabolism, Inborn Errors Genetic Diseases, Inborn Therapeutic Uses Lipidoses Brain Diseases, Metabolic, Inborn Lipid Metabolism Disorders Brain Diseases, Metabolic |