Gene Transfer Clinical Trial for Mucopolysaccharidosis (MPS) IIIB — MPSIIIB  

Mucopolysaccharidosis Type 3 B Clinical Trial

Official title: Phase I/II Gene Transfer Clinical Trial of rAAV9.CMV.hNAGLU for Mucopolysaccharidosis (MPS) IIIB

Status Terminated

Clinical Trial Summary

Open-label, dose-escalation clinical trial of rAAV9.CMV.hNAGLU injected intravenously through a peripheral limb vein

Clinical Trial Description

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. A tapering course of prophylactic enteral prednisone or prednisolone will be administered for a period of at least two months. ;

Related Conditions & MeSH terms

• Mucopolysaccharidoses
• Mucopolysaccharidosis III
• Mucopolysaccharidosis Type 3 B

• NCT number: NCT03315182
• Study type: Interventional
• Source: Abeona Therapeutics, Inc
• Status: Terminated
• Phase: Phase 1/Phase 2
• Start date: October 16, 2017
• Completion date: April 7, 2022