Scientists track canavan disease in 67 children to map its progression
NCT ID NCT04126005
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study followed 67 children with Canavan disease, a rare genetic brain disorder, to learn how the condition naturally changes over time. Researchers reviewed medical records and conducted checkups to track symptoms, milestones, and disease progression. The goal was to better understand the disease, which may help design future treatments and clinical trials.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- What this could lead to
- If this study succeeds, it will give doctors and researchers a clearer picture of how Canavan disease progresses, which could help design future treatments and clinical trials.
- What could go wrong
- This is an observational study, not a treatment trial, so it does not test any therapy. The results may not apply to all patients, and the small number of participants (67) limits how much we can learn.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for CANAVAN DISEASE are added.
By submitting, you agree to our Terms of use
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
-
UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609, United States
-
University Medical Center Hamburg-Eppendorf
Hamburg, 20246, Germany
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Newborn screening study aims to catch rare diseases at birth
- Experimental gene therapy hopes to slow rare brain disease in toddlers
- Gene therapy breakthrough offers hope for rare brain disease
- Could a single DNA test solve the mystery of rare brain diseases in kids?
- Massive leukodystrophy biobank aims to unlock disease secrets