Oral Glyceryl Triacetate (GTA) in Newborns With Canavan

The recruitment status of this study is unknown. The completion date has passed and the status has not been verified in more than two years.

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

History of Changes

Purpose

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: None (Open Label) Primary Purpose: Treatment
Official Title:	Oral Glyceryl Triacetate (GTA) in Newborns With Canavan

Resource links provided by NLM:

Genetics Home Reference related topics: Canavan disease RNAse T2-deficient leukoencephalopathy leukoencephalopathy with vanishing white matter megalencephalic leukoencephalopathy with subcortical cysts

MedlinePlus related topics: Leukodystrophies

Genetic and Rare Diseases Information Center resources: Canavan Disease

U.S. FDA Resources

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 ]


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

Intervention Details:

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

0.5 gr/kg twice a day with increase of 0.5 gr/kg every 3 days up to 5 gr/kg

Eligibility


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

Criteria

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 ClinicalTrials.gov identifier: NCT00724802

Contacts


Contact: Gheona Alterescu, MD	972-2-6666435	gheona@szmc.org.il

Locations


Israel
Shaare Zedek Medical Center	Recruiting
Jerusalem, Israel, 91031
Contact: Gheona Alterescu, MD 972-26666435 gheona@szmc.org.il
Principal Investigator: Gheona Alterescu, MD

Sponsors and Collaborators

Shaare Zedek Medical Center

Investigators


Principal Investigator:	Gheona Alterescu, M.D.	Shaare Zedek Medical Center

More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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.


Responsible Party:	Dr. Gheona Alteresco, Shaare Zedek Medical Center
ClinicalTrials.gov Identifier:	NCT00724802 History of Changes
Other Study ID Numbers:	canavangta
Study First Received:	June 18, 2008
Last Updated:	June 20, 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 Leukoencephalopathies Demyelinating Diseases	Heredodegenerative Disorders, Nervous System Neurodegenerative Diseases Genetic Diseases, Inborn Metabolism, Inborn Errors Metabolic Diseases Triacetin Antifungal Agents Anti-Infective Agents

ClinicalTrials.gov processed this record on September 20, 2017

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