Canavan DiseaseNCT00724802
Oral Glyceryl Triacetate (GTA) in Newborns With Canavan
A study testing GTA (Glyceryl triacetate) and GTA glyceryl triacetate in people with Canavan Disease, run by Shaare Zedek Medical Center at 1 site in Israel.
Trial 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. Read more about this study
- Who is it for?
- Ages Up to 18 months
- Conditions the study is for
- Canavan Disease
- What is being tested?
- GTA (Glyceryl triacetate) and GTA glyceryl triacetate
- Type of treatment
- Pill or oral medicine
- How many people
- 1 participants
- Where
- 1 siteIsrael
- Study dates
- Mar 2008to Aug 2008
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 18 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- clinical diagnosis of Canavan disease
Who cannot join
- Age above 18 months
Trial locations and contacts
1 site in 1 country.
Israel1 site, 1 recruiting
- Recruiting
Shaare Zedek Medical Center
Jerusalem, 91031
Gheona Alterescu, MD, 972-26666435, gheona@szmc.org.il
Gheona Alterescu, MD
About this study
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.
- Conditions
- Canavan Disease
- Being tested
- GTA (Glyceryl triacetate), GTA glyceryl triacetate
- Keywords
- babies with canavan disease
- Medical subject headings
- Canavan Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GTA (Glyceryl triacetate)Dietary supplement
Also called No other names are available for this drug
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
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
What is being measured
The primary outcome is the main question the study is designed to answer.
brain MRI at the end of the study
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jul 2008 (estimated)
- Study startMar 2008
- Primary completionAug 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Canavan DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 1 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shaare Zedek Medical Center
- Sponsor type
- University, hospital or other
- Investigators
- Gheona Alterescu, M.D., Principal investigator, Shaare Zedek Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00724802
- Sponsor's protocol ID
- canavangta
- Status last verified
- Jun 2011
- First submitted
- 18 Jun 2008
- Last updated
- 21 Jun 2011
- Results posted
- No
Publications
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.
PubMed 21474353
Study at a glance
From the registry record, last updated by the sponsor on 21 Jun 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Oral Glyceryl Triacetate (GTA) in Newborns With Canavan
- ClinicalTrials.gov ID
- NCT00724802
- Acronym / sponsor ID
- canavangta
- Conditions
- Canavan Disease
- Keywords
- babies with canavan disease
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 1 participants (estimated)
- Lead sponsor
- Shaare Zedek Medical CenterUniversity, hospital or other
- Study start
- Mar 2008
- Primary completion
- Aug 2008 (actual)
- First posted
- 30 Jul 2008 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- Up to 18 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (1 listed)
- clinical diagnosis of Canavan disease
Key exclusion criteria (1 listed)
- Age above 18 months
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GTA (Glyceryl triacetate)Also: No other names are available for this drug0.5 gr/kg x2/day syrup two times a day with increasing dose each 3 days till max 5gr /kg each day for 6 months | Dietary supplement | – |
| GTA glyceryl triacetate0.5 gr/kg twice a day with increase of 0.5 gr/kg every 3 days up to 5 gr/kg | Drug | – |
Primary outcome
brain MRI at the end of the study
Time frame: 6 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Jun 2011.
- Derived from this study: PubMed 21474353Segel 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Gheona Alterescu, M.D. principal investigator
Shaare Zedek Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing GTA (Glyceryl triacetate) and GTA glyceryl triacetate in people with Canavan Disease, run by Shaare Zedek Medical Center at 1 site in Israel.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 21 Jun 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Israel. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Gheona Alterescu, MD (972-2-6666435, gheona@szmc.org.il), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
brain MRI at the end of the study (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00724802, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Jun 2011.