Oral Glyceryl Triacetate (GTA) in Newborns With Canavan

The recruitment status of this study is unknown because the information has not been verified recently.
Verified June 2011 by Shaare Zedek Medical Center.
Recruitment status was  Recruiting
Sponsor:
Information provided by:
Shaare Zedek Medical Center
ClinicalTrials.gov Identifier:
NCT00724802
First received: June 18, 2008
Last updated: June 20, 2011
Last verified: June 2011

June 18, 2008
June 20, 2011
March 2008
August 2008   (final data collection date for primary outcome measure)
brain MRI at the end of the study [ Time Frame: 6 months ] [ Designated as safety issue: No ]
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Complete list of historical versions of study NCT00724802 on ClinicalTrials.gov Archive Site
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Oral Glyceryl Triacetate (GTA) in Newborns With Canavan
Oral Glyceryl Triacetate (GTA) in Newborns With Canavan

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.

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Interventional
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Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Canavan Disease
  • 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
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Segel R, Anikster Y, Zevin S, Steinberg A, Gahl WA, Fisher D, Staretz-Chacham O, Zimran A, Altarescu G. A safety trial of high dose glyceryl triacetate for Canavan disease. Mol Genet Metab. 2011 Jul;103(3):203-6. doi: 10.1016/j.ymgme.2011.03.012. Epub 2011 Mar 15.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
1
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August 2008   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • clinical diagnosis of Canavan disease

Exclusion Criteria:

  • Age above 18 months
Both
up to 18 Months
No
Contact: Gheona Alterescu, MD 972-2-6666435 gheona@szmc.org.il
Israel
 
NCT00724802
canavangta
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Dr. Gheona Alteresco, Shaare Zedek Medical Center
Shaare Zedek Medical Center
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Principal Investigator: Gheona Alterescu, M.D. Shaare Zedek Medical Center
Shaare Zedek Medical Center
June 2011

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP