Oral Glyceryl Triacetate (GTA) in Newborns With Canavan

Status

UNKNOWN

Phases

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT00724802

Enrollment

Registered

2008-07-30

Start date

2008-03-31

Completion date

Unknown

Last updated

2011-06-21

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Canavan Disease

Keywords

babies with canavan disease

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.

Interventions

DIETARY_SUPPLEMENTGTA (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

DRUGGTA 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 design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

No minimum to 18 Months

Healthy volunteers

Inclusion criteria

* clinical diagnosis of Canavan disease

Exclusion criteria

* Age above 18 months

Design outcomes

Primary

Measure	Time frame
brain MRI at the end of the study	6 months

Countries

Israel

Contacts

Primary ContactGheona Alterescu, MD

gheona@szmc.org.il972-2-6666435

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026