Condition (MeSH)
Canavan Disease
ClinicalTrials.gov lists 13 trials for Canavan Disease or a narrower condition beneath it. Aspa Therapeutics has the most among industry and government lead sponsors, with 2.
Search trials, drugs, companies and investigators, and follow each one to the others. Individual plan, $249/seat/mo. Card required.
Trials per phase
13 of the 13 trials are tagged with Canavan Disease itself; the rest are tagged with a narrower condition beneath it.
When did Canavan Disease trials start? (9)
Shown in this order: most recent first, 1990 onwards. 2026 is partial: trials registered with a 2026 start date so far, not a full year.
Trials over time, by phase
- Phase 1
- Phase 1/2
- Phase 2
- Phase 3
- Not applicable
- Observational
Top of scale: 2
Trials registered with a start date in each year, 1990 onward, stacked by phase (complete years only; the current partial year is in the table's notes). The table below lists the same counts, most recent years first.
| Year | Trials started |
|---|---|
| 2021 | 2 |
| 2019 | 1 |
| 2018 | 1 |
| 2017 | 1 |
| 2016 | 2 |
| 2014 | 1 |
| 2009 | 1 |
| 2008 | 2 |
| 2006 | 1 |
No forecast is made for this condition: it averages 0.0 trial starts a year over the five years to September, under the 5 a year the method needs.
Recruitment duration, a model prediction: none of the 13 trials evaluated at the 2026-10-09 snapshot is predicted and still running. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Canavan Disease trials in? (7)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 1 |
| Phase 1/2 | 2 |
| Phase 2 | 1 |
| Phase 3 | 1 |
| Not applicable | 2 |
| Observational | 5 |
| Expanded access | 1 |
How many Canavan Disease trials are recruiting? (8)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Recruiting | 2 |
| Enrolling by invitation | 1 |
| Active, not recruiting | 1 |
| Completed | 4 |
| Terminated | 1 |
Showing 5 of 8 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Canavan Disease trials? (4)
Shown in this order: most trials first. Lead sponsors the registry classes as industry, NIH, US federal, other government or network. A hospital or university the registry files as government can appear here. Sponsors it classes as academic or other are shown on the full page, because that class cannot separate an institution from a named investigator.
| Sponsor | Trials |
|---|---|
| Aspa Therapeutics | 2 |
| Aldagen | 1 |
| Myrtelle Inc. | 1 |
| Sheba Medical Center | 1 |
Which drugs are being tested for Canavan Disease? (1)
Shown in this order: most trials first. One row per drug, matched to a single molecule under all its names, ranked by number of trials.
| Drug | Trials |
|---|---|
| Prednisone | 1 |
Interventions as named in the registry (8)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Lithium Gluconate (drug) Glyceryl Triacetate GTA (drug) | 1 |
| GTA: Glyceryltriacetate | 1 |
| rAAV9-CB6-AspA | 1 |
| AAV9 BBP-812 | 1 |
| Levetiracetam XR | 1 |
Showing 5 of 8 interventions.
These are the names sponsors entered on ClinicalTrials.gov that we have not matched to a single molecule. Some are drugs under a brand or code name; others are procedures, doses or arm labels.
Which publications cover Canavan Disease? (7)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Oligodendrocyte-targeted adeno-associated virus gene therapy for Canavan disease in children: a phase 1/2 trial. | Nature medicine | 2025 | Yes |
| N-acetylaspartate supports the energetic demands of developmental myelination via oligodendroglial aspartoacylase. | Neurobiology of disease | 2016 | Yes |
| A safety trial of high dose glyceryl triacetate for Canavan disease. | Molecular genetics and metabolism | 2011 | Yes |
| Defective N-acetylaspartate catabolism reduces brain acetate levels and myelin lipid synthesis in Canavan's disease. | Proceedings of the National Academy of Sciences of the United States of America | 2005 | Yes |
| Progress toward acetate supplementation therapy for Canavan disease: glyceryl triacetate administration increases acetate, but not N-acetylaspartate, levels in brain. | The Journal of pharmacology and experimental therapeutics | 2005 | Yes |
Showing 5 of 7 publications.
Prevalence (9)
Shown in this order: jurisdiction, then disease name. Orphanet observation for the mapped ORPHA concept; the mapping relation column says whether that concept is exactly this condition or broader or narrower.
| Orphanet disease | Type | Class | Geography | Jurisdiction | Mapping relation |
|---|---|---|---|---|---|
| Canavan disease | Prevalence at birth | 1-9 / 100 000 | Worldwide | Not stated | Exact |
| Canavan disease | Annual incidence | 1-9 / 100 000 | Specific population | Not stated | Unspecified |
| Canavan disease | Point prevalence | Unknown | Worldwide | Not stated | Unspecified |
| Canavan disease | Annual incidence | 1-9 / 100 000 | Specific population | Not stated | Exact |
| Canavan disease | Point prevalence | Unknown | Worldwide | Not stated | Exact |
Showing 5 of 9 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Canavan disease | 141 | Exact | Orphanet Rare Disease Ontology (ORDO) |
| Canavan disease | 141 | Exact | Orphadata Science knowledge files, Product 1 |
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: serious adverse events in this condition's trials and sub-conditions in mesh.