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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.
Information provided by:
The Cooper Health System Identifier:
First received: May 21, 2008
Last updated: March 18, 2011
Last verified: March 2011


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:

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:
    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
Detailed Description:


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.

Ages Eligible for Study:   1 Year to 10 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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
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Please refer to this study by its identifier: NCT00683189

United States, New Jersey
Cooper University Hospital
Camden, New Jersey, United States, 08103
Sponsors and Collaborators
The Cooper Health System
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 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:

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
Lysosomal Storage Diseases, Nervous System
Demyelinating Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders
Anticoagulants processed this record on July 19, 2017