Oral Glyceryl Triacetate (GTA) in Newborns With Canavan

This study is currently recruiting participants.

Verified June 2011 by Shaare Zedek Medical Center

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

Tracking Information

First Received Date ^ICMJE

June 18, 2008

Last Updated Date

June 20, 2011

Start Date ^ICMJE

March 2008

Primary Completion Date

August 2008 (final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

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

Original Primary Outcome Measures ^ICMJE

Not Provided

Change History

Complete list of historical versions of study NCT00724802 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures ^ICMJE

Not Provided

Original Secondary Outcome Measures ^ICMJE

Not Provided

Current Other Outcome Measures ^ICMJE

Not Provided

Original Other Outcome Measures ^ICMJE

Not Provided

Descriptive Information

Brief Title ^ICMJE

Oral Glyceryl Triacetate (GTA) in Newborns With Canavan

Official Title ^ICMJE

Oral Glyceryl Triacetate (GTA) in Newborns With Canavan

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.

Detailed Description

Not Provided

Study Type ^ICMJE

Interventional

Study Phase

Not Provided

Study Design ^ICMJE

Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment

Condition ^ICMJE

Canavan Disease

Intervention ^ICMJE

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

Study Arm (s)

Not Provided

Publications *

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. Epub 2011 Mar 15.

* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.

Recruitment Information

Recruitment Status ^ICMJE

Recruiting

Estimated Enrollment ^ICMJE

Completion Date

Not Provided

Primary Completion Date

August 2008 (final data collection date for primary outcome measure)

Eligibility Criteria ^ICMJE

Inclusion Criteria:

clinical diagnosis of Canavan disease

Exclusion Criteria:

Age above 18 months

Gender

Both

Ages

up to 18 Months

Accepts Healthy Volunteers

Contacts ^ICMJE

Contact: Gheona Alterescu, MD

972-2-6666435

gheona@szmc.org.il

Location Countries ^ICMJE

Israel

Administrative Information

NCT Number ^ICMJE

NCT00724802

Other Study ID Numbers ^ICMJE

canavangta

Has Data Monitoring Committee

Not Provided

Responsible Party

Dr. Gheona Alteresco, Shaare Zedek Medical Center

Study Sponsor ^ICMJE

Shaare Zedek Medical Center

Collaborators ^ICMJE

Not Provided

Investigators ^ICMJE

Principal Investigator:

Gheona Alterescu, M.D.

Shaare Zedek Medical Center

Information Provided By

Shaare Zedek Medical Center

Verification Date

June 2011

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

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