NCT03784287 - A Treatment Extension Study of Mucopolysaccharidosis Type IIIB | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

February 19, 2018

Primary Completion Date

January 31, 2025

Study Completion Date

February 28, 2025

Conditions

Mucopolysaccharidosis Type IIIBMPS III B

Interventions

DRUG

AX 250

Chimeric fusion of recombinant human alpha-N-acetylglucosaminidase and truncated human insulin-like growth factor 2 (rhNAGLU-IGF2)

Trial Locations (8)

UPMC Children's Hospital Pittburgh, Pittsburgh

Children's Hospital and Research Center Oakland, Oakland

Fundación Cardio Infantil - Instituto de Cardiología, Bogotá

University Medical Center Hamburg-Eppendorf, Hamburg

Complexo Hospitalario Universitario de Santiago (CHUS) - Hospital Clínico Universitario, Santiago de Compostela

MacKay Memorial Children's Hospital, Taipei

Gazi University Faculty of Medicine, Ankara

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