Early Phase 1 Gaucher 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:

• Your child must be 24 months old or younger.
• Your child must have been diagnosed with Gaucher disease, confirmed by a specific enzyme test and genetic testing showing changes in both copies of the GBA1 gene.
• Your child must show signs or symptoms of the neurological form of Gaucher disease (known as type 2).
• A parent or legal guardian must give written permission for the child to take part in the study.
• A parent or legal guardian must agree to follow the study requirements, including sharing information about the disease and helping with symptom assessments.

Who may not be able to join:

• Children who have another significant brain or nervous system condition besides type 2 Gaucher disease.
• Children who have already learned to walk independently.
• Children whose Gaucher disease has caused severe damage to organs (such as the liver or spleen) that could put them at serious risk or make it hard to take part in the study.
• Children who currently have an active infection, including HIV, hepatitis B, hepatitis C, or syphilis.
• Children who have been on enzyme replacement therapy, substrate reduction therapy, or ambroxol for Gaucher disease for less than 2 months before joining (treatment must have been stable for at least 2 months).
• Children currently taking certain strong medications, herbal products, or over-the-counter products that affect how the body processes drugs (confirm with trial site).
• Children who have previously received any type of gene therapy or cell therapy.
• Children who have received a live vaccine within the past 4 weeks.
• Children currently taking immune-suppressing medications or steroids (except skin creams for skin conditions).
• Children whose blood test shows a high level of antibodies against a specific virus used in the therapy (AAV9), above a certain threshold.
• Children whose brain MRI scan shows an abnormality that would make a specific injection procedure unsafe.
• Children who cannot safely receive sedation (being put into a relaxed or sleep-like state) for surgery or imaging scans.
• Children with other serious medical conditions that could put them at risk or make it difficult to take part in the study.

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