Effect of Warfarin in the Treatment of Metachromatic Leuk... | NCT00683189

Studies
Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy

NCT00683189

Sponsor

The Cooper Health System

Status

Completed

Last Update Posted

Mar 21, 2011Estimated

Enrollment

10Estimated

Phase

Not Applicable

Conditions

Metachromatic Leukodystrophy

Interventions

Warfarin

Countries

United States

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Mar 2011

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jun 2007

Primary Completion Date

May 2008Actual

Completion Date

May 2008Actual

First Submitted Date

May 21, 2008

First Submission Date that Met QC Criteria

May 22, 2008

First Posted Date

May 23, 2008Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Mar 18, 2011

Last Update Posted Date

Mar 21, 2011Estimated

Sponsor/Collaborators Module

Responsible Party, by Official Title

Not provided

Lead Sponsor

The Cooper Health SystemOTHER

Collaborators

Not provided

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Not Applicable

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

10Estimated

Arms/Interventions Module

Arm Groups

Not provided

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Warfarin	Drug	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

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Quantitative Neurological Assessment		4 weeks
Urine Sulfatides Quantification		4 weeks

Secondary Outcomes

Measure	Description	Time Frame
Brain MRI		before and after treatment

Other Outcomes

Not provided

Eligibility Module

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

1 Year

Maximum Age

10 Years

Standard Ages

Child

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Paola Leone, Ph.D.	UMDNJ/SOM	Study Director
Mitra Assadi, M.D.	The Cooper Health System	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Cooper University Hospital		Camden	New Jersey	08103	United States

Condition Browse Module

MeSH Terms

ID	Term
D007966	Leukodystrophy, Metachromatic

Ancestor Terms

ID	Term
D020279	Hereditary Central Nervous System Demyelinating Diseases
D020739	Brain Diseases, Metabolic, Inborn
D001928	Brain Diseases, Metabolic
D001927	Brain Diseases

Browse Leaves

Not provided

Browse Branches

Not provided

Intervention Browse Module

MeSH Terms

ID	Term
D014859	Warfarin

Ancestor Terms

ID	Term
D015110	4-Hydroxycoumarins
D003374	Coumarins
D001578	Benzopyrans
D011714	Pyrans
D006573

Browse Leaves

Not provided

Browse Branches

Not provided