drugset / Trial / NCT02790723
Safety of Intra-Articular Sc-rAAV2.5IL-1Ra in Subjects With Moderate Knee OA
Non-randomizedParallel-groupOpen-labelTreatment
Summary
This study seeks to determine the safety of three different doses of sc-rAAV2.5IL-1Ra delivered by intra-articular injection into one knee joint of patients with moderate OA of the knee.
Timeline
- Start
- 2019-06-05
- Primary completion
- 2022-03-17
- Completion
- 2022-03-17
Outcome
Met primary endpoint
paper This trial met its primary outcome measure, safety, and demonstrated that sustained expression of transgenic IL-1Ra was possible after a single injection of the vector. PMID 41607103 ↗
release “Results showed that GNSC-001 met the primary safety endpoint with no dose-limiting toxicity up to 1x10 13 vg high dose.” genascence.com ↗
Publications
- Evans CH, Ghivizzani SC, Robbins PD. Osteoarthritis Gene Therapy: A 30-Year Journey From Concept to Clinical Trials. J Orthop Res. 2026 Feb;44(2):e70141. doi: 10.1002/jor.70141.
- Evans CH, Ghivizzani SC, Robbins PD. The 2024 OREF Clinical Research Award: Progress Toward a Gene Therapy for Arthritis. J Am Acad Orthop Surg. 2024 Dec 1;32(23):1052-1060. doi: 10.5435/JAAOS-D-24-00831. Epub 2024 Oct 17.
- 2022 AMSSM Rising With Research Presentations. Clin J Sport Med. 2022 Mar 1;32(2):168-176. doi: 10.1097/JSM.0000000000001014. No abstract available.
- 2021 AMSSM Oral Research Poster Presentations. Clin J Sport Med. 2021 Mar 1;31(2):185-221. doi: 10.1097/JSM.0000000000000914. No abstract available.
- Mehta S, He T, Bajpayee AG. Recent advances in targeted drug delivery for treatment of osteoarthritis. Curr Opin Rheumatol. 2021 Jan;33(1):94-109. doi: 10.1097/BOR.0000000000000761.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | sc-rAAV2.5IL-1Ra | Gene therapy (AAV / viral vector) | 1e+11 vg | Other |
| Subject | sc-rAAV2.5IL-1Ra | Gene therapy (AAV / viral vector) | 1e+12 vg | Other |
| Subject | sc-rAAV2.5IL-1Ra | Gene therapy (AAV / viral vector) | 1e+13 vg | Other |