An observational study in Canavan Disease, sponsored by Aspa Therapeutics. Completed at 3 sites in 2 countries. Per ClinicalTrials.gov, last updated 2026-04-17.
Sponsored by Aspa Therapeutics · Observational
This study uses medical records that allow retrospective data extraction of critical milestone and motor function data. In addition, prospective assessments collect data relevant to the natural history of Canavan disease in children.
The CANinform natural history study is the first multinational effort to rigorously gather both retrospective and prospective data from this patient population. Data collection includes extraction of retrospective data from medical records of living and deceased patients, and collection of prospective, longitudinal data from living patients and their parent(s)/caregiver(s). Motor function assessments are performed remotely in the home via video or in the clinic by qualified study team members. Families will be invited to attend clinic visits and/or will be followed remotely by the clinical site for approximately 3 years.
592 studies on the registry are indexed under Rare Diseases; 125 are open to participants now.
This study's enrollment of 67 is below the median of 133 across 208 observational studies indexed under Rare Diseases.
Browse Rare Diseases studies →Aspa Therapeutics is the lead sponsor of 2 studies on the registry; 1 is open to participants now.
Counted across the registry records on this site, refreshed daily.
Living and deceased patients with Canavan disease
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
1. Patient does not meet the Inclusion Criteria.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
* Motor function assessments (remote or in-clinic) every 2 months * Clinic assessments every 6 months
* Motor function assessments (remote or in-clinic) every 4 months * Clinic assessments every 6 months
* Motor function assessments (remote or in-clinic) every 6 months * Clinic assessments every 6 months
* Motor function assessments (remote or in-clinic) 12 months * Clinic assessments every 12 months
• The patient's medical history records will be reviewed. In addition, a parent interview will be performed.
To characterize the natural history of Canavan disease
To enhance the understanding of the natural history of Canavan disease through retrospective data collection from patient medical records and prospective data collection from living patients, including: phenotypic characteristics and variability, genotype characteristics and variability, and disease progression and natural history.
Time frame: approximately 3 years
Plan to share: Yes — Data will be made available for researchers using a defined process.
Ran from Oct 2019 to primary completion in May 2025, 67 months.
Of 88 observational studies under Rare Diseases, half reached primary completion within 118 months of starting (the median), counting those still running; a quarter within 39 and three quarters within 183. This study's 67 months is below that.
Rare Diseases covers many different conditions, not one, so the studies compared here vary widely. Read these figures as a rough guide.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Apr 2026. You cannot join it, but the record below documents what was studied.
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