drugset / Trial / NCT02247843
Stem Cell Gene Therapy for Sickle Cell Disease
Phase 1/2
Completed
4 enrolled
Donald B. Kohn, M.D.
California Institute for Regenerative Medicine (CIRM) · collab
NaSingle-groupOpen-labelTreatment
Summary
This Phase I clinical trial will assess the safety and initial evidence for efficacy of an autologous transplant of lentiviral vector modified peripheral blood for adults with severe sickle cell disease.
Timeline
- Start
- 2014-12
- Primary completion
- 2025-09-24
- Completion
- 2025-09-24
Publications
- Background Romero Z, Urbinati F, Geiger S, Cooper AR, Wherley J, Kaufman ML, Hollis RP, de Assin RR, Senadheera S, Sahagian A, Jin X, Gellis A, Wang X, Gjertson D, Deoliveira S, Kempert P, Shupien S, Abdel-Azim H, Walters MC, Meiselman HJ, Wenby RB, Gruber T, Marder V, Coates TD, Kohn DB. beta-globin gene transfer to human bone marrow for sickle cell disease. J Clin Invest. 2013 Jul 1;123(8):3317-30. doi: 10.1172/JCI67930. Online ahead of print.
- Prueksapraopong C, Fernandes A, Campo Fernandez B, Roy S, Hollis RP, Habtemariam B, Pellin D, Ceoldo G, Lin TY, Dang TT, Cornetta K, Romero Z, Blazar BR, Shah AJ, Moore TB, Sehl M, Schiller GJ, Kohn DB. Clinical Outcomes of Lentiviral Vector Gene Therapy for Sickle Cell Disease. Blood Adv. 2026 Apr 20:bloodadvances.2026019869. doi: 10.1182/bloodadvances.2026019869. Online ahead of print.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | βAS3-FB | Cell therapy | — | — |