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Oral Glyceryl Triacetate (GTA) in Newborns With Canavan

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00724802
Recruitment Status : Unknown
Verified June 2011 by Shaare Zedek Medical Center.
Recruitment status was:  Recruiting
First Posted : July 30, 2008
Last Update Posted : June 21, 2011
Information provided by:
Shaare Zedek Medical Center

Brief Summary:
Canavan disease is caused by Aspartoacylase deficiency. There is no treatment for the disease, but there is a food additive that includes acetate . We suggest an early treatment with acetate and a neurologic evaluation, including MRI, after 4 months of treatment. In any case the treatment will be stopped at the age of 22 months, when myelinization is ended.

Condition or disease Intervention/treatment
Canavan Disease Dietary Supplement: GTA (Glyceryl triacetate) Drug: GTA glyceryl triacetate

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 1 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Oral Glyceryl Triacetate (GTA) in Newborns With Canavan
Study Start Date : March 2008
Primary Completion Date : August 2008

Intervention Details:
    Dietary Supplement: GTA (Glyceryl triacetate)
    0.5 gr/kg x2/day syrup two times a day with increasing dose each 3 days till max 5gr /kg each day for 6 months
    Other Name: No other names are available for this drug
    Drug: GTA glyceryl triacetate
    0.5 gr/kg twice a day with increase of 0.5 gr/kg every 3 days up to 5 gr/kg

Primary Outcome Measures :
  1. brain MRI at the end of the study [ Time Frame: 6 months ]

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 18 Months   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • clinical diagnosis of Canavan disease

Exclusion Criteria:

  • Age above 18 months

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 identifier (NCT number): NCT00724802

Contact: Gheona Alterescu, MD 972-2-6666435

Shaare Zedek Medical Center Recruiting
Jerusalem, Israel, 91031
Contact: Gheona Alterescu, MD    972-26666435   
Principal Investigator: Gheona Alterescu, MD         
Sponsors and Collaborators
Shaare Zedek Medical Center
Principal Investigator: Gheona Alterescu, M.D. Shaare Zedek Medical Center

Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: Dr. Gheona Alteresco, Shaare Zedek Medical Center Identifier: NCT00724802     History of Changes
Other Study ID Numbers: canavangta
First Posted: July 30, 2008    Key Record Dates
Last Update Posted: June 21, 2011
Last Verified: June 2011

Keywords provided by Shaare Zedek Medical Center:
babies with canavan disease

Additional relevant MeSH terms:
Canavan Disease
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
Antifungal Agents
Anti-Infective Agents