drugset / Trial / NCT03003533
A Gene Transfer Study for Hemophilia A
Non-randomizedSequentialOpen-labelTreatment
Summary
This clinical research study is being conducted by Spark Therapeutics, Inc. to determine the safety and efficacy of the factor VIII gene transfer treatment with SPK-8011 in individuals with hemophilia A.
Timeline
- Start
- 2017-01-26
- Primary completion
- 2023-12-05
- Completion
- 2023-12-05
Publications
- George LA, Monahan PE, Eyster ME, Sullivan SK, Ragni MV, Croteau SE, Rasko JEJ, Recht M, Samelson-Jones BJ, MacDougall A, Jaworski K, Noble R, Curran M, Kuranda K, Mingozzi F, Chang T, Reape KZ, Anguela XM, High KA. Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A. N Engl J Med. 2021 Nov 18;385(21):1961-1973. doi: 10.1056/NEJMoa2104205.
- Ran G, Chen X, Xie Y, Zheng Q, Xie J, Yu C, Pittman N, Qi S, Yu FX, Agbandje-McKenna M, Srivastava A, Ling C. Site-Directed Mutagenesis Improves the Transduction Efficiency of Capsid Library-Derived Recombinant AAV Vectors. Mol Ther Methods Clin Dev. 2020 Mar 13;17:545-555. doi: 10.1016/j.omtm.2020.03.007. eCollection 2020 Jun 12.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | DIRLOCTOCOGENE SAMOPARVOVEC | Gene therapy (AAV / viral vector) | 5e+11 vg/kg | Intravenous |
| Subject | DIRLOCTOCOGENE SAMOPARVOVEC | Gene therapy (AAV / viral vector) | 1e+12 vg/kg | Intravenous |
| Subject | DIRLOCTOCOGENE SAMOPARVOVEC | Gene therapy (AAV / viral vector) | 1.5e+12 vg/kg | Intravenous |
| Subject | DIRLOCTOCOGENE SAMOPARVOVEC | Gene therapy (AAV / viral vector) | 2e+12 vg/kg | Intravenous |