Oral Glyceryl Triacetate (GTA) in Newborns With Canavan

NCT ID: NCT00724802

Last Updated: 2011-06-21

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

NA

Total Enrollment

1 participants

Study Classification

INTERVENTIONAL

Study Start Date

2008-03-31

Brief Summary

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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

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Conditions

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Canavan Disease

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Interventions

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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

Intervention Type DIETARY_SUPPLEMENT

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

Intervention Type DRUG

Other Intervention Names

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No other names are available for this drug

Eligibility Criteria

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Inclusion Criteria

* clinical diagnosis of Canavan disease

Exclusion Criteria

* Age above 18 months
Maximum Eligible Age

18 Months

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Shaare Zedek Medical Center

OTHER

Sponsor Role lead

Responsible Party

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Shaare Zedek Medical Center

Principal Investigators

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Gheona Alterescu, M.D.

Role: PRINCIPAL_INVESTIGATOR

Shaare Zedek Medical Center

Locations

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Shaare Zedek Medical Center

Jerusalem, , Israel

Site Status RECRUITING

Countries

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Israel

Central Contacts

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Gheona Alterescu, MD

Role: CONTACT

972-2-6666435

Facility Contacts

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Gheona Alterescu, MD

Role: primary

972-26666435

References

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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.

Reference Type DERIVED
PMID: 21474353 (View on PubMed)

Other Identifiers

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canavangta

Identifier Type: -

Identifier Source: org_study_id

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