Phase 1/2 Clinical Trial of ABO-101
An open-label, dose-escalation clinical trial assessing the safety of one-time gene therapy ABO-101 for patients with MPS IIIB.
Visit Clinical Trials.gov for more information.
Who is eligible to participate in the study?
Key criteria for eligible patients include:
- Diagnosis of MPS IIIB
- Age 6 months or older
- Evidence of neurologic dysfunction
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.
- Additional visits may be required for eligible patients receiving long-term follow-up care.
About the Study
MPS IIIB, or Sanfilippo syndrome type B, is caused by a build-up of toxic sugars called glycosaminoglycans (GAGs) because of a mutation in the gene encoding the NAGLU enzyme. The study involves a one-time intravenous administration of ABO-101 using an adeno-associated virus vector 9 (AAV9) to deliver a normal copy of the NAGLU gene. The delivery of the normal NAGLU gene may allow cells to produce the NAGLU enzyme responsible for breaking down GAGs. After treatment, patients are followed for 24 months to assess safety and efficacy.
Nationwide Children's Hospital
Hospital Clínico Universitario de Santiago
Santiago De Compostela
The safety and efficacy of the investigational use of this product has not been determined. There is no guarantee that the investigational use listed will be filed with and/or approved for marketing by a regulatory agency.