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

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. 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.

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 (Child)

Accepts Healthy Volunteers

Contacts ^ICMJE

Contact: Gheona Alterescu, MD

972-2-6666435

gheona@szmc.org.il

Listed Location Countries ^ICMJE

Israel

Removed Location Countries

Administrative Information

NCT Number ^ICMJE

NCT00724802

Other Study ID Numbers ^ICMJE

canavangta

Has Data Monitoring Committee

Not Provided

Plan to Share Data

Not Provided

IPD Description

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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