Last reviewed · How we verify
NCT04126005
Natural History Study of Patients With Canavan Disease (CANinform Study)
trial in Canavan Disease in 67 participants. Completed in 31 August 2025.
21 May 2025
Quick facts
| Lead sponsor | Aspa Therapeutics |
|---|---|
| Status | Completed |
| Study type | OBSERVATIONAL |
| Enrollment | 67 |
| Start date | 10 October 2019 |
| Primary completion | 21 May 2025 |
| Estimated completion | 31 August 2025 |
| Sites | 3 locations across United States, Germany |
Conditions studied
- Canavan Disease — all drugs for Canavan Disease →
Sponsor
Aspa Therapeutics — full company profile →
Who can join
Eligibility, any sex, with Canavan Disease. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
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.
Publications & conference data
1 peer-reviewed publication reference this trial (live from Europe PMC):
-
Urine <i>N</i>-Acetylaspartate Distinguishes Phenotypes in Canavan Disease.
Nagy A, Eichler F, Bley A, Bredow J, et al · · 2024 · cited 3× · PMID 39628365 · DOI 10.1089/hum.2024.168
Verify or expand the search:
- PubMed search for NCT04126005
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other recruiting trials for Canavan Disease
Currently open trials in the same condition.
- NCT04998396 — A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial) · Phase 1, PHASE2 · recruiting
- NCT03047369 — The Myelin Disorders Biorepository Project · recruiting
Other Aspa Therapeutics trials
Trials by the same sponsor.
- NCT04998396 — A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial) · Phase 1, PHASE2 · recruiting
Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT04126005 (US National Library of Medicine, public domain)
- Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Aspa Therapeutics
- Last refreshed: 17 April 2026
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04126005.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing