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Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy
This study is currently recruiting participants.
Verified by The Cooper Health System, May 2008
First Received: May 21, 2008   Last Updated: May 22, 2008   History of Changes
Sponsored by: The Cooper Health System
Information provided by: The Cooper Health System
ClinicalTrials.gov Identifier: NCT00683189
  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

Genetics Home Reference related topics: Chanarin-Dorfman syndrome cholesteryl ester storage disease Farber lipogranulomatosis leukoencephalopathy with vanishing white matter long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency megalencephalic leukoencephalopathy with subcortical cysts metachromatic leukodystrophy mitochondrial trifunctional protein deficiency primary carnitine deficiency succinic semialdehyde dehydrogenase deficiency
MedlinePlus related topics: Blood Thinners Leukodystrophies
Drug Information available for: Warfarin Warfarin sodium Warfarin potassium
U.S. FDA Resources
Study Type: Interventional
Study Design: Treatment, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study
Official Title: Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy

Further study details as provided by The Cooper Health System:

Primary Outcome Measures:
  • Quantitative Neurological Assessment [ Time Frame: 4 weeks ] [ Designated as safety issue: No ]
  • Urine Sulfatides Quantification [ Time Frame: 4 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Brain MRI [ Time Frame: before and after treatment ] [ Designated as safety issue: No ]

Estimated Enrollment: 10
Study Start Date: June 2007
Estimated Study Completion Date: March 2009
Estimated Primary Completion Date: March 2009 (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

Ages Eligible for Study:   1 Year to 10 Years
Genders Eligible for Study:   Both
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
Please refer to this study by its ClinicalTrials.gov identifier: NCT00683189

Contacts
Contact: Paola Leone, Ph.D. 856 566-6334 leonepa@umdnj.edu
Contact: Kelly Anderson, MPH 856-566-6389 anderskm@umdnj.edu

Locations
United States, New Jersey
Cooper University Hospital Recruiting
Camden, New Jersey, United States, 08103
Contact: Paola leone, Ph.D.     856-566-6334     leonepa@mac.com    
Contact: kelly Anderson, MPH     856-566-6389     anderskm@umdnj.edu    
Sponsors and Collaborators
The Cooper Health System
Investigators
Study Director: Paola Leone, Ph.D. UMDNJ/SOM
Principal Investigator: Mitra Assadi, M.D. Cooper University Hospital
  More Information

No publications provided

Responsible Party: Cooper University Hospital ( Mitra Assadi, M.D. )
Study ID Numbers: RP#07/063
Study First Received: May 21, 2008
Last Updated: May 22, 2008
ClinicalTrials.gov Identifier: NCT00683189     History of Changes
Health Authority: United States: Institutional Review Board

Keywords provided by The Cooper Health System:
MLD

Study placed in the following topic categories:
Lipid Metabolism, Inborn Errors
Metachromatic Leukodystrophy
Sphingolipidoses
Anticoagulants
Leukodystrophy, Metachromatic
Metabolic Diseases
Demyelinating Diseases
Lysosomal Storage Diseases
Sphingolipidosis
Central Nervous System Diseases
Warfarin
Brain Diseases
Leukodystrophy
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Brain Diseases, Metabolic, Inborn
Lipidoses
Metabolic Disorder
Lipid Metabolism Disorders
Brain Diseases, Metabolic

Additional relevant MeSH terms:
Lipid Metabolism, Inborn Errors
Sphingolipidoses
Anticoagulants
Metabolic Diseases
Leukodystrophy, Metachromatic
Demyelinating Diseases
Lysosomal Storage Diseases, Nervous System
Lysosomal Storage Diseases
Hematologic Agents
Nervous System Diseases
Sulfatidosis
Central Nervous System Diseases
Warfarin
Brain Diseases
Hereditary Central Nervous System Demyelinating Diseases
Pharmacologic Actions
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Therapeutic Uses
Lipidoses
Brain Diseases, Metabolic, Inborn
Lipid Metabolism Disorders
Brain Diseases, Metabolic

ClinicalTrials.gov processed this record on May 07, 2009