NCT03533673View on ClinicalTrials.gov

AAV2/8-LSPhGAA (ACTUS-101) in Late-Onset Pompe Disease

PHASE1/PHASE2Active, not recruitingINTERVENTIONAL

Enrollment

7

Participants

Timeline

Start Date

December 17, 2018

Primary Completion Date

September 30, 2022

Study Completion Date

March 31, 2026

Conditions

Pompe Disease

Interventions

BIOLOGICAL

ACTUS-101

Adeno-associated virus serotype 8 carrying the human GAA gene under the control of the LSP promoter.

Trial Locations (1)

27705

Duke University, Durham

Sponsors

Lead Sponsor

All Listed Sponsors

lead

AskBio Inc

INDUSTRY

NCT03533673 - AAV2/8-LSPhGAA (ACTUS-101) in Late-Onset Pompe Disease | Biotech Hunter | Biotech Hunter