Oral Glyceryl Triacetate (GTA) in Newborns With Canavan

• ClinicalTrials.gov Identifier: NCT00724802
• Recruitment Status: Unknown
• First Posted: July 30, 2008
• Last Update Posted: June 21, 2011
• Sponsor: Shaare Zedek Medical Center
• Information provided by: Shaare Zedek Medical Center

Tracking Information

• First Submitted Date: June 18, 2008
• First Posted Date: July 30, 2008
• Last Update Posted Date: June 21, 2011
• Study Start Date: March 2008
• Actual Primary Completion Date: August 2008 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: March 18, 2009): brain MRI at the end of the study [ Time Frame: 6 months ]
• Original Primary Outcome Measures: Not Provided
• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Oral Glyceryl Triacetate (GTA) in Newborns With Canavan
• Official Title: 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: Interventional
• Study Phase: Not Applicable
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Canavan Disease

Intervention

• 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: 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.

Recruitment Information

• Recruitment Status: Unknown status
• Estimated Enrollment (submitted: March 18, 2009): 1
• Original Enrollment: Not Provided
• Study Completion Date: Not Provided
• Actual Primary Completion Date: August 2008 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• clinical diagnosis of Canavan disease

Exclusion Criteria:

• Age above 18 months

Sex/Gender

• Sexes Eligible for Study: All

• Ages: up to 18 Months (Child)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Israel

Administrative Information

• NCT Number: NCT00724802
• Other Study ID Numbers: canavangta
• Has Data Monitoring Committee: Not Provided
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Current Responsible Party: Dr. Gheona Alteresco, Shaare Zedek Medical Center
• Original Responsible Party: Not Provided
• Current Study Sponsor: Shaare Zedek Medical Center
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

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

• PRS Account: Shaare Zedek Medical Center
• Verification Date: June 2011