NCT03315182

Gene Transfer Clinical Trial for Mucopolysaccharidosis (MPS) IIIB

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

• Status: Terminated
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 11 (actual)
• Sponsor: Abeona Therapeutics, Inc · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

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

Detailed 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.

Conditions

• Mucopolysaccharidosis Type 3 B

Interventions

Type	Name	Description
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.

Timeline

• Start date: 2017-10-16
• Primary completion: 2022-04-07
• Completion: 2022-04-07
• First posted: 2017-10-20
• Last updated: 2022-05-05

Locations

4 sites across 4 countries: United States, France, Germany, Spain

Regulatory

• FDA-regulated drug study