NCT04360265View on ClinicalTrials.gov

Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA

PHASE3Enrolling by invitationINTERVENTIONAL
Enrollment

41

Participants

Timeline

Start Date

September 28, 2020

Primary Completion Date

August 31, 2027

Study Completion Date

August 31, 2027

Conditions
Mucopolysaccharidosis IIIAMPS IIIASanfilippo SyndromeSanfilippo A
Interventions
OTHER

No Investigational Product

No investigational product will be administered in this follow-up trial.

DRUG

Adjuvant Immunomodulatory (IM) Therapy

The Principal Investigator and/or caregiver, in consultation with the medical monitor, will determine whether to initiate adjuvant IM therapy. Not all participants may receive IM therapy.

Trial Locations (4)

43205

Nationwide Children's Hospital, Columbus

Unknown

Women's and Children's Hospital, North Adelaide

Hospital Clínico Universitario de Santiago, Santiago de Compostela

08035

Vall d'Hebron Barcelona Campus, Barcelona

All Listed Sponsors
lead

Ultragenyx Pharmaceutical Inc

INDUSTRY