AAV2/8-LSPhGAA (ACTUS-101) in late-onset Pompe disease
AAV2/8-LSPhGAA (ACTUS-101) in late-onset Pompe disease
ClinicalTrials.gov ID: NCT03533673
Sponsor: Asklepios Biopharmaceutical, Inc.
Information provided by: Asklepios Biopharmaceutical, Inc. (Responsible Party)
Last Update Posted: 2023-06-28
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.
Official Title:
A Phase 1 Study of the Safety of AAV2/8-LSPhGAA (ACTUS-101) in Late-onset Pompe Disease
Intervention / Treatment:
- Biological: ACTUS-101
| Category | Value |
|---|---|
| Study Start (Actual) |
2018-12-17
|
| Primary Completion (Actual) |
2022-09-30
|
| Study Completion (Estimated) |
2026-03-01
|
| Enrollment (Actual) | 7 |
| Study Type | Interventional |
| Phase | Phase 1 Phase 2 |
| Other Study ID Numbers |
ACT-CS101
|