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Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00683189
First Posted: May 23, 2008
Last Update Posted: March 21, 2011
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.
Information provided by:
The Cooper Health System
  Purpose

Objectives/Purpose:

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: None (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:

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.
  Eligibility

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
Criteria

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
  Contacts and Locations
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 ClinicalTrials.gov identifier (NCT number): NCT00683189


Locations
United States, New Jersey
Cooper University Hospital
Camden, New Jersey, United States, 08103
Sponsors and Collaborators
The Cooper Health System
Investigators
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
ClinicalTrials.gov Identifier: NCT00683189     History of Changes
Other Study ID Numbers: RP#07/063
First Submitted: May 21, 2008
First Posted: May 23, 2008
Last Update Posted: March 21, 2011
Last Verified: March 2011

Keywords provided by The Cooper Health System:
MLD

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
Sulfatidosis
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Leukoencephalopathies
Demyelinating Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders
Warfarin
Anticoagulants