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Oral Glyceryl Triacetate (GTA) in Newborns With Canavan

Primary Purpose

Canavan Disease

Status
Unknown status
Phase
Not Applicable
Locations
Israel
Study Type
Interventional
Intervention
GTA (Glyceryl triacetate)
GTA glyceryl triacetate
Sponsored by
Shaare Zedek Medical Center
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Canavan Disease focused on measuring babies with canavan disease

Eligibility Criteria

undefined - 18 Months (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • clinical diagnosis of Canavan disease

Exclusion Criteria:

  • Age above 18 months

Sites / Locations

  • Shaare Zedek Medical CenterRecruiting

Outcomes

Primary Outcome Measures

brain MRI at the end of the study

Secondary Outcome Measures

Full Information

First Posted
June 18, 2008
Last Updated
June 20, 2011
Sponsor
Shaare Zedek Medical Center
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1. Study Identification

Unique Protocol Identification Number
NCT00724802
Brief Title
Oral Glyceryl Triacetate (GTA) in Newborns With Canavan
Official Title
Oral Glyceryl Triacetate (GTA) in Newborns With Canavan
Study Type
Interventional

2. Study Status

Record Verification Date
June 2011
Overall Recruitment Status
Unknown status
Study Start Date
March 2008 (undefined)
Primary Completion Date
August 2008 (Actual)
Study Completion Date
undefined (undefined)

3. Sponsor/Collaborators

Name of the Sponsor
Shaare Zedek Medical Center

4. Oversight

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Canavan Disease
Keywords
babies with canavan disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
1 (Anticipated)

8. Arms, Groups, and Interventions

Intervention Type
Dietary Supplement
Intervention Name(s)
GTA (Glyceryl triacetate)
Other Intervention Name(s)
No other names are available for this drug
Intervention Description
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
Drug
Intervention Name(s)
GTA glyceryl triacetate
Intervention Description
0.5 gr/kg twice a day with increase of 0.5 gr/kg every 3 days up to 5 gr/kg
Primary Outcome Measure Information:
Title
brain MRI at the end of the study
Time Frame
6 months

10. Eligibility

Sex
All
Maximum Age & Unit of Time
18 Months
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: clinical diagnosis of Canavan disease Exclusion Criteria: Age above 18 months
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Gheona Alterescu, MD
Phone
972-2-6666435
Email
gheona@szmc.org.il
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Gheona Alterescu, M.D.
Organizational Affiliation
Shaare Zedek Medical Center
Official's Role
Principal Investigator
Facility Information:
Facility Name
Shaare Zedek Medical Center
City
Jerusalem
ZIP/Postal Code
91031
Country
Israel
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Gheona Alterescu, MD
Phone
972-26666435
Email
gheona@szmc.org.il
First Name & Middle Initial & Last Name & Degree
Gheona Alterescu, MD

12. IPD Sharing Statement

Citations:
PubMed Identifier
21474353
Citation
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.
Results Reference
derived

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Oral Glyceryl Triacetate (GTA) in Newborns With Canavan

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