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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
May 21, 2008
May 23, 2008
March 21, 2011
June 2007
May 2008   (Final data collection date for primary outcome measure)
  • Quantitative Neurological Assessment [ Time Frame: 4 weeks ]
  • Urine Sulfatides Quantification [ Time Frame: 4 weeks ]
Same as current
Complete list of historical versions of study NCT00683189 on ClinicalTrials.gov Archive Site
Brain MRI [ Time Frame: before and after treatment ]
Same as current
Not Provided
Not Provided
 
Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy
Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy

Objectives/Purpose:

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

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.
Interventional
Not Provided
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Metachromatic Leukodystrophy
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
Not Provided
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
10
May 2008
May 2008   (Final data collection date for primary outcome measure)

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
Sexes Eligible for Study: All
1 Year to 10 Years   (Child)
No
Contact information is only displayed when the study is recruiting subjects
United States
 
 
NCT00683189
RP#07/063
No
Not Provided
Not Provided
Mitra Assadi, M.D., Cooper University Hospital
The Cooper Health System
Not Provided
Study Director: Paola Leone, Ph.D. UMDNJ/SOM
Principal Investigator: Mitra Assadi, M.D. The Cooper Health System
The Cooper Health System
March 2011

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP
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