Oral Glyceryl Triacetate (GTA) in Newborns With Canavan

Canavan Disease Clinical Trial

Official title:

Oral Glyceryl Triacetate (GTA) in Newborns With Canavan

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT00724802
• Other study ID #: canavangta
• Status: Recruiting
• Phase: N/A
• First received: June 18, 2008
• Last updated: June 20, 2011
• Start date: March 2008

Study information

• Verified date: June 2011
• Source: Shaare Zedek Medical Center
• Contact: Gheona Alterescu, MD
• Phone: 972-2-6666435
• Email: gheona[@]szmc.org.il
• Is FDA regulated: No
• Health authority: United States: Institutional Review Board
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 1
• Est. primary completion date: August 2008
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A to 18 Months

Eligibility

Inclusion Criteria:

• clinical diagnosis of Canavan disease

Exclusion Criteria:

• Age above 18 months

Study Design

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

Related Conditions & MeSH terms

• 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

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

Locations

Country	Name	City	State
Israel	Shaare Zedek Medical Center	Jerusalem

Sponsors (1)

Lead Sponsor	Collaborator
Shaare Zedek Medical Center

Country where clinical trial is conducted

Israel, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	brain MRI at the end of the study		6 months	No