Status:
COMPLETED
Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy
Lead Sponsor:
The Cooper Health System
Conditions:
Metachromatic Leukodystrophy
Eligibility:
All Genders
1-10 years
Phase:
NA
Brief Summary
Objectives/Purpose: To determine the safety and efficacy of a Vitamin K (Vit K) antagonist (warfarin) in treating Metachromatic Leukodystrophy (MLD).
Detailed Description
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 decreasi...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
June 1 2007
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
May 1 2008
Estimated Enrollment :
10 Patients enrolled
Trial Details
Trial ID
NCT00683189
Start Date
June 1 2007
End Date
May 1 2008
Last Update
March 21 2011
Active Locations (1)
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1
Cooper University Hospital
Camden, New Jersey, United States, 08103