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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.
 
ClinicalTrials.gov Identifier: NCT00683189
Recruitment Status : Completed
First Posted : May 23, 2008
Last Update Posted : March 21, 2011
Sponsor:
Information provided by:
The Cooper Health System

Tracking Information
First Submitted Date  ICMJE May 21, 2008
First Posted Date  ICMJE May 23, 2008
Last Update Posted Date March 21, 2011
Study Start Date  ICMJE June 2007
Actual Primary Completion Date May 2008   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 22, 2008)
  • Quantitative Neurological Assessment [ Time Frame: 4 weeks ]
  • Urine Sulfatides Quantification [ Time Frame: 4 weeks ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT00683189 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: May 22, 2008)
Brain MRI [ Time Frame: before and after treatment ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy
Official Title  ICMJE Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy
Brief Summary

Objectives/Purpose:

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

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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Not Applicable
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Metachromatic Leukodystrophy
Intervention  ICMJE 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
Study Arms  ICMJE Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Estimated Enrollment  ICMJE
 (submitted: May 22, 2008)
10
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE May 2008
Actual Primary Completion Date May 2008   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 1 Year to 10 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00683189
Other Study ID Numbers  ICMJE RP#07/063
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Mitra Assadi, M.D., Cooper University Hospital
Study Sponsor  ICMJE The Cooper Health System
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Paola Leone, Ph.D. UMDNJ/SOM
Principal Investigator: Mitra Assadi, M.D. The Cooper Health System
PRS Account The Cooper Health System
Verification Date March 2011

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