Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy

Study Description

Go to 

Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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
Metachromatic Leukodystrophy	Drug: Warfarin	Not Applicable

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

Go to 

Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Study Type :	Interventional (Clinical Trial)
Estimated Enrollment :	10 participants
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 :	June 2007
Actual Primary Completion Date :	May 2008
Actual Study Completion Date :	May 2008

Arms and Interventions

Go to 

Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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

Outcome Measures

Go to 

Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Primary Outcome Measures :

1. Quantitative Neurological Assessment [ Time Frame: 4 weeks ]
2. Urine Sulfatides Quantification [ Time Frame: 4 weeks ]

Secondary Outcome Measures :

1. Brain MRI [ Time Frame: before and after treatment ]

Eligibility Criteria

Go to 

Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Ages Eligible for Study:	1 Year to 10 Years (Child)
Sexes Eligible for Study:	All
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

Go to 

Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Locations

United States, New Jersey
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

More Information

Go to 

Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Responsible Party:	Mitra Assadi, M.D., Cooper University Hospital
ClinicalTrials.gov Identifier:	NCT00683189 History of Changes
Other Study ID Numbers:	RP#07/063
First Posted:	May 23, 2008
Last Update Posted:	March 21, 2011
Last Verified:	March 2011

Keywords provided by The Cooper Health System:

MLD

Additional relevant MeSH terms:

Leukodystrophy, Metachromatic; Hereditary Central Nervous System Demyelinating Diseases; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Sulfatidosis; Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Leukoencephalopathies	Demyelinating Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Lipid Metabolism Disorders; Warfarin; Anticoagulants