A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients

PHASE1/PHASE2CompletedINTERVENTIONAL

Enrollment

Participants

Timeline

Start Date

January 19, 2019

Primary Completion Date

April 30, 2021

Study Completion Date

May 7, 2021

Conditions

Sanfilippo Syndrome Type A (MPS IIIA)

Interventions

DRUG

SOBI003

weekly i.v. infusion

Trial Locations (3)

University of North Carolina hospitals, Chapel Hill

Children´s Hospital and research center, Oakland

Gazi University Hospital, Ankara