Infantile Canavan DiseasePhase 1NCT00278707
GTA-Glyceryltriacetate for Canavan Disease
A Phase 1 study testing GTA: Glyceryltriacetate in people with Infantile Canavan Disease and Deficiency Disease, Aspartoacylase, run by Sheba Medical Center at 1 site in Israel.
Trial summary
The purpose of this study is to determine whether oral supplementation of glyceryl triacetate improves the clinical prognosis of Canavan Disease. Read more about this study
- Who is it for?
- Ages 0 years to 15 months
- Conditions the study is for
- Infantile Canavan Disease, Deficiency Disease, Aspartoacylase
- What is being tested?
- GTA: Glyceryltriacetate
- How many people
- 5 participants
- Where
- 1 siteIsrael
- Study dates
- Jan 2006to Jul 2006
- Phase
- Phase 1
- 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 0 years to 15 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Age below 15 months
- Biochemically diagnosed with Canavan Disease
Who cannot join
- None
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Phase 1 Treatment With GTA in Two Infant With Canavan Disease
The purpose of this study is to determine whether oral supplementation of glyceryl triacetate improves the clinical prognosis of Canavan Disease.
Read the detailed description
Canavan Disease is caused by a deficiency in the enzyme named Aspartoacylase (ASPA). This disease is a devastating, progressive disease with no available treatment. As a result of the ASPA deficiency, there are high levels of N-acetylaspartate (NAA) and low levels of L-aspartate and acetate.
We hypothesize that one of the functions of ASPA is to provide sufficient levels of acetate for CNS myelinization. For this reason, we offer to supplement acetate levels by the oral administration of glyceryl triacetate (GTA). Such treatment must be offered to patients before the age of 18 months, prior to the termination of CNS myelinization.
- Two patients, aged less than 15 months, will receive daily doses of oral GTA
- The daily dose will be increased incrementally until the maintenance dose is reached. This will be done under close monitoring of the patients, including periodic blood gas sampling.
- GTA has not been shown to cause any known toxicity, according to the Cosmetic Ingredient Review Expert Panel (Fiume, 2003).
- Being tested
- GTA: Glyceryltriacetate
- Keywords
- Canavan Disease, Aspartoacylase Deficiency, NAA, Acetate, Glyceryltriacetate
- 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: GlyceryltriacetateDrug
What is being measured
The primary outcome is the main question the study is designed to answer.
All primary outcome will be evaluated 4 months following the initiation of treatment:
Neurological Status
Brain Imaging: MRI & MRS
NAA Levels in Urine
Ophthalmologic Examination
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jan 2006 (estimated)
- Study startJan 2006
- Study completionJul 2006
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Infantile Canavan DiseaseStudies of the same condition, open and under way.
- More trials run by Sheba Medical CenterThe sponsor's other registered studies.
- 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 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants
Sponsor and investigators
- Lead sponsor
- Sheba Medical Center
- Sponsor type
- Other government
- Investigators
- Yair Anikster, MD PI, Principal investigator, Director Metabolic Disease Unit
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00278707
- Sponsor's protocol ID
- SHEBA-05-3968-YA-CTIL
- Status last verified
- Aug 2006
- First submitted
- 15 Jan 2006
- Last updated
- 15 Aug 2006
- Results posted
- No
Publications
Mathew R, Arun P, Madhavarao CN, Moffett JR, Namboodiri MA. Progress toward acetate supplementation therapy for Canavan disease: glyceryl triacetate administration increases acetate, but not N-acetylaspartate, levels in brain. J Pharmacol Exp Ther. 2005 Oct;315(1):297-303. doi: 10.1124/jpet.105.087536. Epub 2005 Jul 7.
PubMed 16002461Madhavarao CN, Arun P, Moffett JR, Szucs S, Surendran S, Matalon R, Garbern J, Hristova D, Johnson A, Jiang W, Namboodiri MA. Defective N-acetylaspartate catabolism reduces brain acetate levels and myelin lipid synthesis in Canavan's disease. Proc Natl Acad Sci U S A. 2005 Apr 5;102(14):5221-6. doi: 10.1073/pnas.0409184102. Epub 2005 Mar 22.
PubMed 15784740
Study at a glance
From the registry record, last updated by the sponsor on 15 Aug 2006. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 1 Treatment With GTA in Two Infant With Canavan Disease
- ClinicalTrials.gov ID
- NCT00278707
- Acronym / sponsor ID
- SHEBA-05-3968-YA-CTIL
- Keywords
- Canavan Disease, Aspartoacylase Deficiency, NAA, Acetate, Glyceryltriacetate
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 5 participants
- Lead sponsor
- Sheba Medical CenterOther government
- Study start
- Jan 2006
- Study completion
- Jul 2006
- First posted
- 18 Jan 2006 (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.
The purpose of this study is to determine whether oral supplementation of glyceryl triacetate improves the clinical prognosis of Canavan Disease.
Detailed description
Canavan Disease is caused by a deficiency in the enzyme named Aspartoacylase (ASPA). This disease is a devastating, progressive disease with no available treatment. As a result of the ASPA deficiency, there are high levels of N-acetylaspartate (NAA) and low levels of L-aspartate and acetate.
We hypothesize that one of the functions of ASPA is to provide sufficient levels of acetate for CNS myelinization. For this reason, we offer to supplement acetate levels by the oral administration of glyceryl triacetate (GTA). Such treatment must be offered to patients before the age of 18 months, prior to the termination of CNS myelinization.
- Two patients, aged less than 15 months, will receive daily doses of oral GTA
- The daily dose will be increased incrementally until the maintenance dose is reached. This will be done under close monitoring of the patients, including periodic blood gas sampling.
- GTA has not been shown to cause any known toxicity, according to the Cosmetic Ingredient Review Expert Panel (Fiume, 2003).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 0 years to 15 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (2 listed)
- Age below 15 months
- Biochemically diagnosed with Canavan Disease
Key exclusion criteria (1 listed)
- None
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: Glyceryltriacetate | Drug | – |
Primary outcome
All primary outcome will be evaluated 4 months following the initiation of treatment:
Neurological Status
Brain Imaging: MRI & MRS
NAA Levels in Urine
Ophthalmologic Examination
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Aug 2006.
- Background: PubMed 16002461Mathew R, Arun P, Madhavarao CN, Moffett JR, Namboodiri MA. Progress toward acetate supplementation therapy for Canavan disease: glyceryl triacetate administration increases acetate, but not N-acetylaspartate, levels in brain. J Pharmacol Exp Ther. 2005 Oct;315(1):297-303. doi: 10.1124/jpet.105.087536. Epub 2005 Jul 7.
- Background: PubMed 15784740Madhavarao CN, Arun P, Moffett JR, Szucs S, Surendran S, Matalon R, Garbern J, Hristova D, Johnson A, Jiang W, Namboodiri MA. Defective N-acetylaspartate catabolism reduces brain acetate levels and myelin lipid synthesis in Canavan's disease. Proc Natl Acad Sci U S A. 2005 Apr 5;102(14):5221-6. doi: 10.1073/pnas.0409184102. Epub 2005 Mar 22.
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.
Yair Anikster, MD PI principal investigator
Director Metabolic Disease Unit
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing GTA: Glyceryltriacetate in people with Infantile Canavan Disease and Deficiency Disease, Aspartoacylase, run by Sheba 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 2 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 15 Aug 2006. 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.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
All primary outcome will be evaluated 4 months following the initiation of treatment:. The study lists 5 primary outcomes in all. 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 NCT00278707, which also lists 2 linked publications.
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 15 Aug 2006.