AAV2/8-LSPhGAA (ACTUS-101) in Late-Onset Pompe Disease
ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD
Brief 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.
Study Type: Interventional (Clinical Trial)
Estimated Enrollment: 13 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 1 Study of the Safety of AAV2/8-LSPhGAA (ACTUS-101) in Late-onset Pompe Disease
Actual Study Start Date: December 17, 2018
Estimated Primary Completion Date: April 2025
Estimated Study Completion Date: April 2028
Arms:
- Experimental: Cohort 1
- Experimental: Cohort 2
- Experimental: Cohort 3
- Experimental: Cohort 4
- Experimental: Cohort 5
| Category | Value |
|---|---|
| Study type(s) | Interventional |
| Estimated enrolment | 13 |
| Actual Study start date | 17 December 2018 |
| Estimated study completion date | 01 April 2028 |