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| Descriptive Information Fields | |||||||||
| Brief Title † | Lithium and Acetate for Canavan Disease | ||||||||
| Official Title † | Evaluation of the Tolerance and Efficiency of a Combined Oral Therapy With Lithium and GTA in Patients With Canavan Disease | ||||||||
| Brief Summary | 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. |
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| Detailed Description | Canavan Disease is an autosomal recessive devastating demyelinating disease caused by a deficiency in Aspartoacylase (ASPA) enzyme. There is no available treatment. ASPA deficiency leads to:- the accumulation of high levels of N-acetylaspartate (NAA), involved in myelin degeneration and epilepsy;- the deficient synthesis of acetate in oligodendrocytes, that could impair CNS myelination.Lithium administration induces a decrease in NAA in the brain of the tremor rats (animal model for CD) and in one patient (JANSON, 2005). On the other hand, administration of acetate could improve myelination in Canavan patients.For this reason, we propose to combine both treatments: Lithium Gluconate and Glyceryl Triacetate (GTA). Eighteen patients, aged 1 to 15 years, will receive oral GTA or Lithium during 4 months, then both treatment in association during 6 months. Patients will be sequentially evaluated up to the end of the treatment and 2 months thereafter for:-tolerance of the therapy (careful monitoring of clinical and biological parameters).- efficacy of the therapy on clinical, biological and radiological parameters. Particularly, we will evaluate using MRI-spectroscopy and CSF samples the decrease in NAA and increase in acetate levels in the brain. |
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| Study Phase | Phase II | ||||||||
| Study Type † | Interventional | ||||||||
| Study Design † | Treatment, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study | ||||||||
| Primary Outcome Measure † | 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 ] [ Designated as safety issue: Yes ] | ||||||||
| Secondary Outcome Measure † | 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 ] [ Designated as safety issue: Yes ] -Improvement of epilepsy (number of seizures) [ Time Frame: 10 months ] [ Designated as safety issue: Yes ] -Decrease in NAA and increase in acetate contents in fluids (CSF, plasma, urine). [ Time Frame: 10 months ] [ Designated as safety issue: Yes ] |
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| Condition † | Canavan Disease Infantile Deficiency Disease Aspartoacylase Leukodystrophy, Spongiform |
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| Intervention † | Drug: Lithium Gluconate (drug) Glyceryl Triacetate GTA (drug) | ||||||||
| MEDLINE PMIDs | 16002461, 15784740, 16194720, 12064356 | ||||||||
| Links | ELA (European Leukodystrophy Association)  |
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| Recruitment Information Fields | |||||||||
| Recruitment Status † | Not yet recruiting | ||||||||
| Enrollment † | 18 | ||||||||
| Start Date † | June 2008 | ||||||||
| Completion Date | January 2011 | ||||||||
| Eligibility Criteria † | Inclusion Criteria:
Exclusion Criteria:
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| Gender | Both | ||||||||
| Ages | 1 Year to 15 Years | ||||||||
| Accepts Healthy Volunteers | No | ||||||||
| Contacts †† |
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| Location Countries † | France | ||||||||
| Administrative Information Fields | |||||||||
| NCT ID † | NCT00657748 | ||||||||
| Organization ID | P070803 | ||||||||
| Secondary IDs †† | |||||||||
| Study Sponsor † | Assistance Publique - Hôpitaux de Paris | ||||||||
| Collaborators †† | ELA foundation (European Leukodystrophy Association) | ||||||||
| Investigators † |
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| Information Provided By | Assistance Publique - Hôpitaux de Paris | ||||||||
| Verification Date | April 2008 | ||||||||
| First Received Date † | April 9, 2008 | ||||||||
| Last Updated Date | April 11, 2008 | ||||||||
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