NCT04088734 - Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

September 18, 2019

Primary Completion Date

March 10, 2022

Study Completion Date

March 10, 2022

Conditions

MPS IIIASanfilippo SyndromeSanfilippo AMucopolysaccharidosis III

Interventions

DRUG

ABO-102

Single dose of ABO-102 (scAAV9.U1a.hSGSH) administered by intravenous injection through a peripheral limb vein at a dose of 3 X 10\^13 vg/kg

Trial Locations (3)

Adelaide Women's and Children's Hospital, North Adelaide

Children's Hospital of Pittsburgh, Pittsburgh

Hospital Clínico Universitario de Santiago, Santiago de Compostela