NCT04174105View on ClinicalTrials.gov

Gene Transfer Study in Patients With Late Onset Pompe Disease

PHASE1/PHASE2Active, not recruitingINTERVENTIONAL
Enrollment

11

Participants

Timeline

Start Date

October 27, 2020

Primary Completion Date

February 28, 2030

Study Completion Date

February 28, 2030

Conditions
Pompe Disease (Late-onset)
Interventions
GENETIC

zocaglusagene nuzaparvovec

AT845 is an AAV8 vector delivering a functional copy of the human GAA gene, under the control of a muscle-specific promoter

Trial Locations (4)

84108

University of Utah, Division of Medical Genetics, Salt Lake City

92868

University of California Irvine, Department of Neurology, Orange

94304

Stanford University, Palo Alto

NE1 4LP

Newcastle Upon Tyne Hospitals Foundation Trust Clinical Research Facility, Newcastle upon Tyne

Sponsors
All Listed Sponsors
lead

Astellas Gene Therapies

INDUSTRY

NCT04174105 - Gene Transfer Study in Patients With Late Onset Pompe Disease | Biotech Hunter | Biotech Hunter