A Treatment Study of Mucopolysaccharidosis Type IIIB

PHASE1/PHASE2CompletedINTERVENTIONAL
Enrollment

23

Participants

Timeline

Start Date

April 30, 2016

Primary Completion Date

June 24, 2020

Study Completion Date

July 31, 2020

Conditions
MPS III BMucopolysaccharidosis Type IIIB
Interventions
DRUG

AX 250

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

Trial Locations (7)

10449

Mackay Memorial Hospital, Taipei

94609

Children's Hospital Oakland, Oakland

Unknown

Fundación Cardio Infantil - Instituto de Cardiología, Bogotá

University Medical Center Hamburg Eppendorf, Department of Pediatrics, Hamburg

Hospital Clinico Universitario de Santiago, Santiago de Compostela

Gazi Üniversitesi Tıp Fakültesi, Ankara

Somers Clinical Research Facility, Great Ormond Street Hospital, London

Sponsors
All Listed Sponsors
lead

Allievex Corporation

INDUSTRY