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Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy

Sponsor
The Cooper Health System (Other)
Overall Status
Completed
CT.gov ID
NCT00683189
Collaborator
(none)
10
Enrollment
1
Location
11
Duration (Months)
0.9
Patients Per Site Per Month

Study Details

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).

Condition or Disease Intervention/Treatment Phase
N/A

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 Design

Study Type:
Interventional
Anticipated Enrollment :
10 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy
Study Start Date :
Jun 1, 2007
Actual Primary Completion Date :
May 1, 2008
Actual Study Completion Date :
May 1, 2008

Outcome Measures

Primary Outcome Measures

  1. Quantitative Neurological Assessment [4 weeks]

  2. Urine Sulfatides Quantification [4 weeks]

Secondary Outcome Measures

  1. Brain MRI [before and after treatment]

Eligibility Criteria

Criteria

Ages Eligible for Study:
1 Year to 10 Years
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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

Locations

Site City State Country Postal Code
1 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

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
, ,
ClinicalTrials.gov Identifier:
NCT00683189
Other Study ID Numbers:
  • RP#07/063
First Posted:
May 23, 2008
Last Update Posted:
Mar 21, 2011
Last Verified:
Mar 1, 2011
Keywords provided by , ,
MLD
Additional relevant MeSH terms:
Leukodystrophy, Metachromatic
Warfarin

Study Results

No Results Posted as of Mar 21, 2011