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Lithium and Acetate for Canavan Disease

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ClinicalTrials.gov Identifier: NCT00657748
Recruitment Status : Withdrawn
First Posted : April 14, 2008
Last Update Posted : April 21, 2015
European Leukodystrophy Association
Information provided by:
Assistance Publique - Hôpitaux de Paris

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.

Condition or disease Intervention/treatment Phase
Canavan Disease Infantile Deficiency Disease Aspartoacylase Leukodystrophy, Spongiform Drug: Lithium Gluconate (drug) Glyceryl Triacetate GTA (drug) Phase 2

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 Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Evaluation of the Tolerance and Efficiency of a Combined Oral Therapy With Lithium and GTA in Patients With Canavan Disease
Study Start Date : September 2009
Actual Primary Completion Date : September 2010
Actual Study Completion Date : January 2011

Arm Intervention/treatment
Experimental: 1 Drug: Lithium Gluconate (drug) Glyceryl Triacetate GTA (drug)
Lithium 1.3 mEq/kg/day (three administrations a day)during the study GTA 500 mg/kg/day in four administrations a day during the study
Other Names:
  • Lithium Gluconate (drug)
  • Glyceryl Triacetate, GTA (drug)

Primary Outcome Measures :
  1. 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 ]

Secondary Outcome Measures :
  1. 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 ]
  2. -Improvement of epilepsy (number of seizures) [ Time Frame: 10 months ]
  3. -Decrease in NAA and increase in acetate contents in fluids (CSF, plasma, urine). [ Time Frame: 10 months ]

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Ages Eligible for Study:   1 Year to 15 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Clinical and biochemical diagnosis of Canavan disease

Exclusion Criteria:

  • Renal disease
  • Thyroid disease
  • Cardiac disease
  • Impossibility to perform brain MRI

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00657748

Sponsors and Collaborators
Assistance Publique - Hôpitaux de Paris
European Leukodystrophy Association
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Principal Investigator: Patrick Aubourg, MD, PhD Assistance Publique - Hôpitaux de Paris
Principal Investigator: Caroline Sevin, MD, PhD Assistance Publique - Hôpitaux de Paris
Additional Information:
Publications of Results:
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Responsible Party: Yannick VACHER, Department Clinical Research and Developpement
ClinicalTrials.gov Identifier: NCT00657748    
Other Study ID Numbers: P070803
First Posted: April 14, 2008    Key Record Dates
Last Update Posted: April 21, 2015
Last Verified: April 2015
Keywords provided by Assistance Publique - Hôpitaux de Paris:
Canavan disease,
Glyceryl Triacetate
Additional relevant MeSH terms:
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Canavan Disease
Deficiency Diseases
Nutrition Disorders
Hereditary Central Nervous System Demyelinating Diseases
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Demyelinating Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Metabolic Diseases
Lithium Carbonate
Antidepressive Agents
Psychotropic Drugs
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Antimanic Agents
Tranquilizing Agents
Central Nervous System Depressants
Physiological Effects of Drugs
Antifungal Agents
Anti-Infective Agents