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Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00683189
Recruitment Status : Completed
First Posted : May 23, 2008
Last Update Posted : March 21, 2011
Information provided by:
The Cooper Health System

Brief Summary:


To determine the safety and efficacy of a Vitamin K (Vit K) antagonist (warfarin) in treating Metachromatic Leukodystrophy (MLD).

Condition or disease Intervention/treatment Phase
Metachromatic Leukodystrophy Drug: Warfarin Not Applicable

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy
Study Start Date : June 2007
Actual Primary Completion Date : May 2008
Actual Study Completion Date : May 2008

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

Primary Outcome Measures :
  1. Quantitative Neurological Assessment [ Time Frame: 4 weeks ]
  2. Urine Sulfatides Quantification [ Time Frame: 4 weeks ]

Secondary Outcome Measures :
  1. Brain MRI [ Time Frame: before and after treatment ]

Information from the National Library of Medicine

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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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00683189

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United States, New Jersey
Cooper University Hospital
Camden, New Jersey, United States, 08103
Sponsors and Collaborators
The Cooper Health System
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Study Director: Paola Leone, Ph.D. UMDNJ/SOM
Principal Investigator: Mitra Assadi, M.D. The Cooper Health System
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Responsible Party: Mitra Assadi, M.D., Cooper University Hospital Identifier: NCT00683189    
Other Study ID Numbers: RP#07/063
First Posted: May 23, 2008    Key Record Dates
Last Update Posted: March 21, 2011
Last Verified: March 2011
Keywords provided by The Cooper Health System:
Additional relevant MeSH terms:
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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