Phase 2 Rare Disease Trial, Recruiting

Written by an AI model from ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

• Children who are 30 months old or younger at the time of joining
• Children who are in stable general health, with no serious blood, kidney, liver, immune system, or neurological conditions other than Canavan disease (based on the investigator's assessment, medical history, and lab results)
• Children who have been diagnosed with Canavan disease, confirmed by all three of the following: higher-than-normal levels of a substance called NAA in the urine; a specific gene change (mutation) in both copies of the ASPA gene, identified either during screening or recorded in prior medical history; and active signs of Canavan disease
• Children who are up to date on all vaccinations recommended in their local area

Who may not be able to join:

• Children whose blood test shows the presence of certain antibodies against a substance called AAV9 (detected using a specific lab test called ELISA)
• Children who have previously received any gene therapy or any other treatment — including vaccines — that involved a substance called AAV
• Children who are currently receiving high doses of medications that suppress the immune system
• Children whose Canavan disease has progressed significantly, meaning they have any of the following: constant abnormal body posturing caused by severe brain involvement; repeated episodes of prolonged, uncontrolled seizures (status epilepticus); or seizures that cannot be controlled even while taking three or more seizure medications at the same time

Important: Always verify eligibility with the trial site directly before applying.