Study ABT-003


Study ABT-003: A Phase 1/2 Clinical Trial of ABO-102


An open-label clinical trial assessing the safety of one-time gene therapy ABO-102 for patients with middle and advanced phases of MPS IIIA.


Now Enrolling

Is my child eligible to participate in the study?

Key criteria for eligible patients include the following, but a full list of inclusion and exclusion criteria are available here.

  • Diagnosis of MPS IIIA
  • 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.

About the Study

Sanfilippo syndrome type A, or MPS IIIA, is caused by a build-up of toxic sugars called glycosaminoglycans (GAGs) because of a mutation in the gene encoding the SGSH enzyme. The study involves a one-time intravenous administration of ABO-102 using an adeno-associated virus vector (AAV) to deliver a normal copy of the SGSH gene. The delivery of the normal SGSH gene may allow cells to produce the SGSH enzyme responsible for breaking down GAGs. After treatment, patients are followed for 24 months to assess safety and efficacy.


For more information about enrolling in Study ABT-003, contact us at

Locations & Contacts

Women's and Children's Hospital

North Adelaide, South Australia
Louise Jaensch: 

Hospital Clínico Universitario de Santiago

Santiago De Compostela
Maria Luz Couce, MD:
Maria de Castro, MD:

Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania
Maria L. Escolar, MD:

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.