Canavan Disease Clinical Trial
Official title:
Evaluation of the Tolerance and Efficiency of a Combined Oral Therapy With Lithium and GTA in Patients With Canavan Disease
Verified date | April 2015 |
Source | Assistance Publique - Hôpitaux de Paris |
Contact | n/a |
Is FDA regulated | No |
Health authority | France: Ministry of Health |
Study type | Interventional |
The aim of this study is to determine whether oral supplementation with lithium and acetate may improve the biological and clinical prognosis in patients with Canavan Disease.
Status | Withdrawn |
Enrollment | 0 |
Est. completion date | January 2011 |
Est. primary completion date | September 2010 |
Accepts healthy volunteers | No |
Gender | Both |
Age group | 1 Year to 15 Years |
Eligibility |
Inclusion Criteria: - Clinical and biochemical diagnosis of Canavan disease Exclusion Criteria: - Renal disease - Thyroid disease - Cardiac disease - Impossibility to perform brain MRI |
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Country | Name | City | State |
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n/a |
Lead Sponsor | Collaborator |
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Assistance Publique - Hôpitaux de Paris | European Leukodystrophy Association |
Baslow MH, Kitada K, Suckow RF, Hungund BL, Serikawa T. The effects of lithium chloride and other substances on levels of brain N-acetyl-L-aspartic acid in Canavan disease-like rats. Neurochem Res. 2002 May;27(5):403-6. — View Citation
Janson CG, Assadi M, Francis J, Bilaniuk L, Shera D, Leone P. Lithium citrate for Canavan disease. Pediatr Neurol. 2005 Oct;33(4):235-43. — View Citation
Madhavarao CN, Arun P, Moffett JR, Szucs S, Surendran S, Matalon R, Garbern J, Hristova D, Johnson A, Jiang W, Namboodiri MA. Defective N-acetylaspartate catabolism reduces brain acetate levels and myelin lipid synthesis in Canavan's disease. Proc Natl Ac — View Citation
Mathew R, Arun P, Madhavarao CN, Moffett JR, Namboodiri MA. Progress toward acetate supplementation therapy for Canavan disease: glyceryl triacetate administration increases acetate, but not N-acetylaspartate, levels in brain. J Pharmacol Exp Ther. 2005 O — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | 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. | 10 months | Yes | |
Secondary | Secondary outcomes will be assessed at 10 months (end of the treatment): -Improvement of neuromotor performances (GMFM and Mullen scales), spasticity, and neurological severity | 10 months | Yes | |
Secondary | -Improvement of epilepsy (number of seizures) | 10 months | Yes | |
Secondary | -Decrease in NAA and increase in acetate contents in fluids (CSF, plasma, urine). | 10 months | Yes |
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