Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy
Primary Purpose
Metachromatic Leukodystrophy
Status
Completed
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
Warfarin
Sponsored by
About this trial
This is an interventional treatment trial for Metachromatic Leukodystrophy focused on measuring MLD
Eligibility 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
Sites / Locations
- Cooper University Hospital
Outcomes
Primary Outcome Measures
Quantitative Neurological Assessment
Urine Sulfatides Quantification
Secondary Outcome Measures
Brain MRI
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT00683189
Brief Title
Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy
Official Title
Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy
Study Type
Interventional
2. Study Status
Record Verification Date
March 2011
Overall Recruitment Status
Completed
Study Start Date
June 2007 (undefined)
Primary Completion Date
May 2008 (Actual)
Study Completion Date
May 2008 (Actual)
3. Sponsor/Collaborators
Name of the Sponsor
The Cooper Health System
4. Oversight
Data Monitoring Committee
No
5. Study Description
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.
Duration of Treatment: 4 weeks
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.
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.
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.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Metachromatic Leukodystrophy
Keywords
MLD
7. Study Design
Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
10 (Anticipated)
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
Warfarin
Intervention Description
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 Measure Information:
Title
Quantitative Neurological Assessment
Time Frame
4 weeks
Title
Urine Sulfatides Quantification
Time Frame
4 weeks
Secondary Outcome Measure Information:
Title
Brain MRI
Time Frame
before and after treatment
10. Eligibility
Sex
All
Minimum Age & Unit of Time
1 Year
Maximum Age & Unit of Time
10 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Paola Leone, Ph.D.
Organizational Affiliation
UMDNJ/SOM
Official's Role
Study Director
First Name & Middle Initial & Last Name & Degree
Mitra Assadi, M.D.
Organizational Affiliation
The Cooper Health System
Official's Role
Principal Investigator
Facility Information:
Facility Name
Cooper University Hospital
City
Camden
State/Province
New Jersey
ZIP/Postal Code
08103
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy
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