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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
Information provided by:
Shaare Zedek Medical Center Identifier:
First received: June 18, 2008
Last updated: June 20, 2011
Last verified: June 2011
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 Intervention
Canavan Disease
Dietary Supplement: GTA (Glyceryl triacetate)
Drug: GTA glyceryl triacetate

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Oral Glyceryl Triacetate (GTA) in Newborns With Canavan

Resource links provided by NLM:

Further study details as provided by Shaare Zedek Medical Center:

Primary Outcome Measures:
  • brain MRI at the end of the study [ Time Frame: 6 months ] [ Designated as safety issue: No ]

Estimated Enrollment: 1
Study Start Date: March 2008
Primary Completion Date: August 2008 (Final data collection date for primary outcome measure)
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

Ages Eligible for Study:   up to 18 Months
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • clinical diagnosis of Canavan disease

Exclusion Criteria:

  • Age above 18 months
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: 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
  More Information

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 
Study First Received: June 18, 2008
Last Updated: June 20, 2011
Health Authority: United States: Institutional Review Board

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

Additional relevant MeSH terms:
Canavan Disease
Brain Diseases
Brain Diseases, Metabolic
Brain Diseases, Metabolic, Inborn
Central Nervous System Diseases
Demyelinating Diseases
Genetic Diseases, Inborn
Hereditary Central Nervous System Demyelinating Diseases
Heredodegenerative Disorders, Nervous System
Metabolic Diseases
Metabolism, Inborn Errors
Nervous System Diseases
Neurodegenerative Diseases
Anti-Infective Agents
Antifungal Agents processed this record on May 26, 2016