Metachromatic LeukodystrophyNCT00683189
Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy
A study testing Warfarin in people with Metachromatic Leukodystrophy, run by Cooper Norcross Health at 1 site in United States.
Trial summary
Objectives/Purpose: To determine the safety and efficacy of a Vitamin K (Vit K) antagonist (warfarin) in treating Metachromatic Leukodystrophy (MLD). Read more about this study
- Who is it for?
- Ages 1 year to 10 years
- Conditions the study is for
- Metachromatic Leukodystrophy
- What is being tested?
- Warfarin
- Type of treatment
- Pill or oral medicine
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2007to May 2008
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 10 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Cooper University Hospital
Camden, New Jersey, 08103
About this study
Objectives/Purpose:
To determine the safety and efficacy of a Vitamin K (Vit K) antagonist (warfarin) in treating Metachromatic Leukodystrophy (MLD).
Read the 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.
- Conditions
- Metachromatic Leukodystrophy
- Being tested
- Warfarin
- Keywords
- MLD
- Medical subject headings
- Leukodystrophy, Metachromatic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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
What is being measured
The primary outcome is the main question the study is designed to answer.
Quantitative Neurological Assessment
Measured: 4 weeks
Urine Sulfatides Quantification
Measured: 4 weeks
Secondary outcomes (1)
Brain MRI
Measured: before and after treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 May 2008 (estimated)
- Study startJun 2007
- Primary completionMay 2008
- Study completionMay 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Metachromatic LeukodystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Cooper Norcross Health
- Sponsor type
- University, hospital or other
- Investigators
- Paola Leone, Ph.D., Study director, UMDNJ/SOMMitra Assadi, M.D., Principal investigator, Cooper Norcross Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00683189
- Sponsor's protocol ID
- RP#07/063
- Status last verified
- Mar 2011
- First submitted
- 21 May 2008
- Last updated
- 21 Mar 2011
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Mar 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy
- ClinicalTrials.gov ID
- NCT00683189
- Acronym / sponsor ID
- RP#07/063
- Conditions
- Metachromatic Leukodystrophy
- Keywords
- MLD
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Cooper Norcross HealthUniversity, hospital or other
- Study start
- Jun 2007
- Primary completion
- May 2008 (actual)
- Study completion
- May 2008 (actual)
- First posted
- 23 May 2008 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 1 year to 10 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (1 listed)
- 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.
Key exclusion criteria (3 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| WarfarinOral 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 | Drug | – |
Primary outcome
Quantitative Neurological Assessment
Time frame: 4 weeks
Urine Sulfatides Quantification
Time frame: 4 weeks
1 secondary outcome
Brain MRI
Time frame: before and after treatment
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Mar 2011.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Paola Leone, Ph.D. study director
UMDNJ/SOM
Mitra Assadi, M.D. principal investigator
Cooper Norcross Health
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Warfarin in people with Metachromatic Leukodystrophy, run by Cooper Norcross Health at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 21 Mar 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Quantitative Neurological Assessment (time frame: 4 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Mar 2011.