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Trial record 10 of 55 for:
Leukodystrophy
Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy
This study has been completed.
Sponsor:
The Cooper Health System
Information provided by:
The Cooper Health System
ClinicalTrials.gov Identifier:
NCT00683189
First received: May 21, 2008
Last updated: March 18, 2011
Last verified: March 2011
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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: | Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy |
Resource links provided by NLM:
Genetics Home Reference related topics:
RNAse T2-deficient leukoencephalopathy
leukoencephalopathy with vanishing white matter
megalencephalic leukoencephalopathy with subcortical cysts
metachromatic leukodystrophy
Genetic and Rare Diseases Information Center resources:
Leukodystrophy
Metachromatic Leukodystrophy
Sphingolipidosis
U.S. FDA Resources
Further study details as provided by The Cooper Health System:
Primary Outcome Measures:
- Quantitative Neurological Assessment [ Time Frame: 4 weeks ]
- Urine Sulfatides Quantification [ Time Frame: 4 weeks ]
Secondary Outcome Measures:
- Brain MRI [ Time Frame: before and after treatment ]
| 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) |
Intervention Details:
Detailed Description:
-
Drug: Warfarin
Oral administration (QD), variable dosage: patients will undergo blood test for PT/INR at baseline and afterwards, at weekly bases for 4 weeks. The INR will be kept in a safe range of 2-2.5
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.
- Duration of Treatment: 4 weeks
- Pharmacological Intervention: The patients will receive warfarin 1.5 mg at the beginning of the study period. The dosage then will be adjusted to the INR values on weekly basis.
- Clinical evaluation: The patients will undergo clinical assessment prior to starting the treatment and at the end of the treatment period. The clinical assessment will also include administration of Gross Motor Function Measure (GMFM), a clinical toll for evaluation of motor development in children.
- Urine Sulfatide Quantification: Urine samples for quantification of the sulfatide level will be collected at the time of enrollment, after 2 weeks and at the end of treatment period.
- Blood Monitoring: The patients will undergo blood test for PT/INR at baseline and afterwards, at weekly bases for 4 weeks. The INR will be kept in a safe range of 2-2.5. If the INR is greater than 4.0 the dosage of warfarin will be lowered and another blood draw will be performed in 3 days.
Eligibility| Ages Eligible for Study: | 1 Year to 10 Years (Child) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Children with MLD, 1 to 10 years of age who have received and failed bone marrow transplantation or are excluded from the treatment due to delayed diagnosis or any other reasons.
Exclusion Criteria:
- Any Children with MLD who are eligible for and might receive ABMT.
- Any Children with MLD who suffer with a bleeding disorder, moderate to severe anemia or any other hematological disorders.
- Any contraindications systemic for anti-coagulation
Contacts and Locations
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To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below.
For general information, see Learn About Clinical Studies.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00683189
Please refer to this study by its ClinicalTrials.gov identifier: NCT00683189
Locations
| United States, New Jersey | |
| Cooper University Hospital | |
| Camden, New Jersey, United States, 08103 | |
Sponsors and Collaborators
The Cooper Health System
Investigators
| Study Director: | Paola Leone, Ph.D. | UMDNJ/SOM |
| Principal Investigator: | Mitra Assadi, M.D. | The Cooper Health System |
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 |
Keywords provided by The Cooper Health System:
|
MLD |
Additional relevant MeSH terms:
|
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 |
ClinicalTrials.gov processed this record on July 19, 2017