NCT00683189

Brief Summary

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

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
10

participants targeted

Target at below P25 for not_applicable

Timeline
Completed

Started Jun 2007

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

June 1, 2007

Completed
11 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 1, 2008

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

May 1, 2008

Completed
20 days until next milestone

First Submitted

Initial submission to the registry

May 21, 2008

Completed
2 days until next milestone

First Posted

Study publicly available on registry

May 23, 2008

Completed
Last Updated

March 21, 2011

Status Verified

March 1, 2011

Enrollment Period

11 months

First QC Date

May 21, 2008

Last Update Submit

March 18, 2011

Conditions

Metachromatic Leukodystrophy

Keywords

MLD

Outcome Measures

Primary Outcomes (2)

  • Quantitative Neurological Assessment

    4 weeks

  • Urine Sulfatides Quantification

    4 weeks

Secondary Outcomes (1)

  • Brain MRI

    before and after treatment

Interventions

WarfarinDRUG

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

Eligibility Criteria

Age1 Year - 10 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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.

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Cooper University Hospital

Camden, New Jersey, 08103, United States

Location

MeSH Terms

Conditions

Leukodystrophy, Metachromatic

Interventions

Warfarin

Condition Hierarchy (Ancestors)

Hereditary Central Nervous System Demyelinating DiseasesBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesSulfatidosisSphingolipidosesLysosomal Storage Diseases, Nervous SystemLeukoencephalopathiesDemyelinating DiseasesMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesLipidosesLipid Metabolism, Inborn ErrorsLysosomal Storage DiseasesMetabolic DiseasesNutritional and Metabolic DiseasesLipid Metabolism Disorders

Intervention Hierarchy (Ancestors)

4-HydroxycoumarinsCoumarinsBenzopyransPyransHeterocyclic Compounds, 1-RingHeterocyclic CompoundsHeterocyclic Compounds, 2-RingHeterocyclic Compounds, Fused-Ring

Study Officials

  • Paola Leone, Ph.D.

    UMDNJ/SOM

    STUDY DIRECTOR

  • Mitra Assadi, M.D.

    The Cooper Health System

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
not applicable
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER

Study Record Dates

First Submitted

May 21, 2008

First Posted

May 23, 2008

Study Start

June 1, 2007

Primary Completion

May 1, 2008

Study Completion

May 1, 2008

Last Updated

March 21, 2011

Record last verified: 2011-03

Locations

US(1)