Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
Register:
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ClinicalTrials.gov |
Last refreshed on:
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4 May 2015 |
Main ID: |
NCT00657748 |
Date of registration:
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09/04/2008 |
Prospective Registration:
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Yes |
Primary sponsor: |
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Public title:
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Lithium and Acetate for Canavan Disease
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Scientific title:
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Evaluation of the Tolerance and Efficiency of a Combined Oral Therapy With Lithium and GTA in Patients With Canavan Disease |
Date of first enrolment:
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September 2009 |
Target sample size:
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0 |
Recruitment status: |
Withdrawn |
URL:
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http://clinicaltrials.gov/show/NCT00657748 |
Study type:
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Interventional |
Study design:
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Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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Phase:
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Phase 2
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Countries of recruitment
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France
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Contacts
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Name:
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Patrick Aubourg, MD, PhD |
Address:
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Telephone:
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Email:
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Affiliation:
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Assistance Publique - Hôpitaux de Paris |
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Name:
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Caroline Sevin, MD, PhD |
Address:
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Telephone:
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Email:
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Affiliation:
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Assistance Publique - Hôpitaux de Paris |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Clinical and biochemical diagnosis of Canavan disease
Exclusion Criteria:
- Renal disease
- Thyroid disease
- Cardiac disease
- Impossibility to perform brain MRI
Age minimum:
1 Year
Age maximum:
15 Years
Gender:
Both
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Health Condition(s) or Problem(s) studied
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Leukodystrophy, Spongiform
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Canavan Disease
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Deficiency Disease
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Aspartoacylase
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Infantile
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Intervention(s)
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Drug: Lithium Gluconate (drug) Glyceryl Triacetate GTA (drug)
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Primary Outcome(s)
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The primary outcome will be a decrease of the NAA peak (> 20%) or the appearance of an acetate peak at the end of the treatment (10 months), using spectroscopy-MRI.
[Time Frame: 10 months]
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Secondary Outcome(s)
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Secondary outcomes will be assessed at 10 months (end of the treatment): -Improvement of neuromotor performances (GMFM and Mullen scales), spasticity, and neurological severity
[Time Frame: 10 months]
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-Decrease in NAA and increase in acetate contents in fluids (CSF, plasma, urine).
[Time Frame: 10 months]
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-Improvement of epilepsy (number of seizures)
[Time Frame: 10 months]
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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