1. Cell Therapy for Chronic TBI: Interim Analysis of the Randomized Controlled STEMTRA Trial.
- Author
-
Kawabori, Masahito, Weintraub, Alan H, Imai, Hideaki, Zinkevych, Laroslav, McAllister, Peter, Steinberg, Gary K, Frishberg, Benjamin M, Yasuhara, Takao, Chen, Jefferson W, Cramer, Steven C, Achrol, Achal S, Schwartz, Neil E, Suenaga, Jun, Lu, Daniel C, Semeniv, Ihor, Nakamura, Hajime, Kondziolka, Douglas, Chida, Dai, Kaneko, Takehiko, Karasawa, Yasuaki, Paadre, Susan, Nejadnik, Bijan, Bates, Damien, Stonehouse, Anthony H, Richardson, R Mark, and Okonkwo, David O
- Subjects
Injury (total) Accidents/Adverse Effects ,Injury - Trauma - (Head and Spine) ,Clinical Research ,Brain Disorders ,Injury - Traumatic brain injury ,Clinical Trials and Supportive Activities ,Neurosciences ,Patient Safety ,Clinical Sciences ,Cognitive Sciences ,Neurology & Neurosurgery - Abstract
ObjectiveTo determine if chronic motor deficits secondary to traumatic brain injury (TBI) can be improved by implantation of allogeneic modified bone marrow-derived mesenchymal stromal/stem cells (SB623).MethodsThis 6-month interim analysis of the 1-year double-blind, randomized, surgical sham-controlled, phase 2 STEMTRA trial (NCT02416492) evaluated safety and efficacy of the stereotactic intracranial implantation of SB623 in patients with stable chronic motor deficits secondary to TBI. Patients in this multi-center trial (N = 63) underwent randomization in a 1:1:1:1 ratio to 2.5 × 106, 5.0 × 106, 10 × 106 SB623 cells or control. Safety was assessed in patients who underwent surgery (N = 61), and efficacy in the modified intent-to-treat population of randomized patients who underwent surgery (N = 61; SB623 = 46, control = 15).ResultsThe primary efficacy endpoint of significant improvement from baseline of Fugl-Meyer Motor Scale score at 6 months for SB623-treated patients was achieved. SB623-treated patients improved by (LS mean [SE]) +8.3 (1.4) vs +2.3 (2.5) for control at 6 months, the LS mean difference was 6.0 (95% CI: 0.3-11.8); p = 0.040. Secondary efficacy endpoints improved from baseline, but were not statistically significant vs control at 6 months. There were no dose-limiting toxicities or deaths, and 100% of SB623-treated patients experienced treatment-emergent adverse events vs 93.3% of control patients (p = 0.25).ConclusionsSB623 cell implantation appeared to be safe and well tolerated, and patients implanted with SB623 experienced significant improvement from baseline motor status at 6 months compared to controls.Classification of evidenceThis study provides Class I evidence that implantation of SB623 was well tolerated and associated with improvement in motor status.
- Published
- 2021