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Oral Glyceryl Triacetate (GTA) in Newborns With Canavan

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00724802
Recruitment Status : Unknown
Verified June 2011 by Shaare Zedek Medical Center.
Recruitment status was:  Recruiting
First Posted : July 30, 2008
Last Update Posted : June 21, 2011
Sponsor:
Information provided by:
Shaare Zedek Medical Center

Tracking Information
First Submitted Date  ICMJE June 18, 2008
First Posted Date  ICMJE July 30, 2008
Last Update Posted Date June 21, 2011
Study Start Date  ICMJE March 2008
Actual Primary Completion Date August 2008   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 18, 2009)
brain MRI at the end of the study [ Time Frame: 6 months ]
Original Primary Outcome Measures  ICMJE Not Provided
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures 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  ICMJE Not Applicable
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (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 Arms  ICMJE 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 Unknown status
Estimated Enrollment  ICMJE
 (submitted: March 18, 2009)
1
Original Enrollment  ICMJE Not Provided
Study Completion Date  ICMJE Not Provided
Actual 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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE up to 18 Months   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
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
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Current Responsible Party Dr. Gheona Alteresco, Shaare Zedek Medical Center
Original Responsible Party Not Provided
Current Study Sponsor  ICMJE Shaare Zedek Medical Center
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Gheona Alterescu, M.D. Shaare Zedek Medical Center
PRS Account 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