NCT00724802

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.

Trial Health

55
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
1

participants targeted

Target at below P25 for not_applicable

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

March 1, 2008

Completed
4 months until next milestone

First Submitted

Initial submission to the registry

June 18, 2008

Completed
1 month until next milestone

First Posted

Study publicly available on registry

July 30, 2008

Completed
2 days until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 1, 2008

Completed
Last Updated

June 21, 2011

Status Verified

June 1, 2011

Enrollment Period

5 months

First QC Date

June 18, 2008

Last Update Submit

June 20, 2011

Conditions

Canavan Disease

Keywords

babies with canavan disease

Outcome Measures

Primary Outcomes (1)

  • brain MRI at the end of the study

    6 months

Interventions

GTA glyceryl triacetateDRUG

0.5 gr/kg twice a day with increase of 0.5 gr/kg every 3 days up to 5 gr/kg

Eligibility Criteria

AgeUp to 18 Months
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • clinical diagnosis of Canavan disease

You may not qualify if:

  • Age above 18 months

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Shaare Zedek Medical Center

Jerusalem, 91031, Israel

RECRUITING

Related Publications (1)

  • 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.

    PMID: 21474353DERIVED

MeSH Terms

Conditions

Canavan Disease

Interventions

Triacetin

Condition Hierarchy (Ancestors)

Hereditary Central Nervous System Demyelinating DiseasesBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesLeukoencephalopathiesDemyelinating DiseasesHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolism, Inborn ErrorsMetabolic DiseasesNutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

TriglyceridesGlyceridesLipids

Study Officials

  • Gheona Alterescu, M.D.

    Shaare Zedek Medical Center

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Gheona Alterescu, MD

CONTACT

972-2-6666435gheona@szmc.org.il

Study Design

Study Type
interventional
Phase
not applicable
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER

Study Record Dates

First Submitted

June 18, 2008

First Posted

July 30, 2008

Study Start

March 1, 2008

Primary Completion

August 1, 2008

Last Updated

June 21, 2011

Record last verified: 2011-06

Locations

IL(1)