NCT05492799 - Safety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLE | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

December 2, 2022

Primary Completion Date

October 31, 2027

Study Completion Date

December 31, 2027

Conditions

MPS III B

Interventions

DRUG

AX 250

biweekly infusion by intracerebroventricular (ICV) infusion

Trial Locations (6)

UPMC Children's Hospital Pittburgh, Pittsburgh

UCSF Benioff Children's Hospital Oakland, Oakland

Fundación Cardio Infantil - Instituto de Cardiología, Bogotá

University Medical Center Hamburg-Eppendorf, Hamburg

Gazi University Faculty of Medicine, Ankara

Great Ormond Street Hospital For Children, NHS Foundation Trust, London