The original Abeona Sanfilippo Type A gene therapy trial (ABT-001) changed its criteria at the beginning of 2019 to enrol only patients under the age of two or with a minimum Cognitive Development Quotient (DQ) of 60 or above. DQ is a measure of a child’s cognitive ability. This new trial aims to enrol up to 12 patients with a DQ below 60 who are still able to walk.
Key criteria for eligible patients include the following, but a full list of inclusion and exclusion criteria are available here
- Diagnosis of MPS IIIA (Sanfilippo Type A)
- Cognitive Development Quotient (DQ) lower than 60
- Must be ambulatory, with/without assistance
What will be required of patients and their families?
- Up to two screening visits.
- A treatment visit, once eligibility is confirmed. Patients will be admitted to the hospital for gene transfer and will stay for approximately 48 hours for observation.
- Patients and their caregiver(s) will be required to stay near the clinical site for 30 days after gene therapy is administered.
- After gene transfer, a total of nine follow up visits will take place at the clinical site, with the last visit occurring 24 months after gene transfer.
- Home visits for collection of blood in between the follow-up visits to the clinical site.
- After 24 months patients may be invited to participate in a long-term follow up study.
