NCT03533673

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

A Phase 1 Study of the Safety of AAV2/8-LSPhGAA (ACTUS-101) in Late-onset Pompe Disease

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 7 (actual)
• Sponsor: AskBio Inc · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

Open-label, ascending dose trial of ACTUS-101 administered intravenously.

Detailed description

This study will be a prospective, open-label trial designed to objectively assess the safety and bioactivity of ACTUS-101 in subjects diagnosed with Pompe disease, which is caused by a defect in acid α-glucosidase (GAA) gene. ACTUS-101 is intended to enable expression of a functional copy of the GAA gene in subject's hepatocytes.

Conditions

• Pompe Disease

Interventions

Type	Name	Description
GENETIC	ACTUS-101	Adeno-associated virus serotype 8 carrying the human GAA gene under the control of the LSP promoter.

Timeline

• Start date: 2018-12-17
• Primary completion: 2022-09-30
• Completion: 2026-03-12
• First posted: 2018-05-23
• Last updated: 2026-04-13

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study