Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy
To determine the safety and efficacy of a Vitamin K (Vit K) antagonist (warfarin) in treating Metachromatic Leukodystrophy (MLD).
|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|
- Quantitative Neurological Assessment [ Time Frame: 4 weeks ] [ Designated as safety issue: No ]
- Urine Sulfatides Quantification [ Time Frame: 4 weeks ] [ Designated as safety issue: No ]
- Brain MRI [ Time Frame: before and after treatment ] [ Designated as safety issue: No ]
|Study Start Date:||June 2007|
|Study Completion Date:||May 2008|
|Primary Completion Date:||May 2008 (Final data collection date for primary outcome measure)|
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.
|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|