Oral Glyceryl Triacetate (GTA) in Newborns With Canavan

Shaare Zedek Medical Center

Status: Unknown

Conditions: Canavan Disease

Treatments: Drug: GTA glyceryl triacetate; Dietary Supplement: GTA (Glyceryl triacetate)

Study type: Interventional

Funder types: Other

Identifiers

NCT00724802

canavangta

Details and patient eligibility

About

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.

Enrollment: 1 estimated patient

Sex: All

Ages: Under 18 months old

Volunteers: No Healthy Volunteers

Inclusion criteria

• clinical diagnosis of Canavan disease

Exclusion criteria

• Age above 18 months

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)