NCT03315182 - Gene Transfer Clinical Trial for Mucopolysaccharidosis (MPS) IIIB | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

October 16, 2017

Primary Completion Date

April 7, 2022

Study Completion Date

April 7, 2022

Conditions

Mucopolysaccharidosis Type 3 B

Interventions

BIOLOGICAL

rAAV9.CMV.hNAGLU

Adeno-associated virus serotype 9 carrying the human NAGLU gene under the control of a CMV enhancer/promoter (rAAV9.CMV.hNAGLU) will be delivered one-time through a venous catheter inserted into a peripheral limb vein.

Trial Locations (4)

15706

Hospital Clinico Universitario de Santiago, Santiago de Compostela

43205

Nationwide Children's Hospital, Columbus

Unknown

Armand-Trousseau Hospital, Paris

University Hospital Hamburg-Eppendorf, Hamburg