Metachromatic Leukodystrophy Clinical Trial
Official title:
Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy
Objectives/Purpose:
To determine the safety and efficacy of a Vitamin K (Vit K) antagonist (warfarin) in
treating Metachromatic Leukodystrophy (MLD).
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.
1. Duration of Treatment: 4 weeks
2. 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.
3. 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.
4. 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.
5. 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.
;
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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