Abstract Details

Title Pre-Specified Interim Analysis of the STEMTRA Trial: Clinical Outcomes in Chronic Traumatic Brain Injury Patients

Topic Neuro-rehabilitation

Presentation(s) S42 - Neuro-rehabilitation (3:30 PM-3:42 PM)

Poster/Presentation
Number 001

Objective

To determine if chronic motor deficits secondary to traumatic brain injury (TBI) can be improved by intracranial implantation of SB623 cells.

Background

TBI is a major cause of chronic motor deficits for which no effective pharmacological treatments are available.

Design/Methods

This 6-month pre-specified interim analysis of the 1-year double-blind, randomized, surgical sham-controlled, Phase 2 STEMTRA trial (NCT02416492) evaluated safety and efficacy of stereotactic intracranial implantation of allogeneic modified bone marrow-derived mesenchymal stem cells (SB623) in patients with stable chronic motor deficits secondary to TBI. Sixty-three patients underwent randomization in a 1:1:1:1 ratio to either 2.5x10⁶, 5.0x10⁶, 10x10⁶ SB623 cells or surgical sham procedure. Safety was assessed in patients who underwent surgery (N=61). Efficacy analyses were conducted on the modified intent-to-treat population of randomized patients completing surgery (N=61; SB623=46, sham controls=15).

Results

Primary efficacy outcome of significant change from baseline in the Fugl-Meyer Motor Scale score (FMMS) of SB623-treated patients compared to controls at 6 months was achieved (LS mean [SE]: +8.3 [1.4] SB623 versus +2.3 [2.5] control, P=0.04). Significantly more SB623-treated than control patients achieved the FMMS clinically meaningful threshold change of ≥10 points at Month 6 (39.1% versus 6.7%, P=0.04), a threshold that was achieved by 53.3% of patients in the 5.0x10⁶ SB623 group. All SB623-treated patients experienced treatment-emergent adverse events compared with 93.3% of patients in the control group (P=0.25). At the time of this analysis there were no dose-limiting toxicities or deaths. Six treatment emergent serious adverse events occurred in four (8.7%) SB623-treated patients, compared with three TESAEs in three (20%) control patients.

Conclusions

SB623 cell implantation appeared to be safe and well tolerated, with no significant difference in adverse events between SB623 treatment and surgical sham controls. SB623 treatment was associated with improvement in motor status at 6 months.

Authors/Disclosures
Peter J. McAllister, MD, FAAN (New England Inst for Neurology and Headache) PRESENTER	Dr. McAllister has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for lilly. Dr. McAllister has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for abbvie. Dr. McAllister has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for lundbeck.
	No disclosure on file
	No disclosure on file
Benjamin M. Frishberg, MD, FAAN (The Neurology Center of Southern California)	Dr. Frishberg has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for BMS. Dr. Frishberg has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for novartis. Dr. Frishberg has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for genentech. Dr. Frishberg has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Lilly. Dr. Frishberg has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Roche. Dr. Frishberg has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Allergan. Dr. Frishberg has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Lundbeck. Dr. Frishberg has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Novartis. Dr. Frishberg has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for BMS. Dr. Frishberg has received personal compensation in the range of $50,000-$99,999 for serving on a Speakers Bureau for Biogen. Dr. Frishberg has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Sanofi. Dr. Frishberg has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Biohaven. Dr. Frishberg has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Viela-Bio. Dr. Frishberg has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Horizon. Dr. Frishberg has received personal compensation in the range of $5,000-$9,999 for serving as an officer or member of the Board of Directors for Headache Cooperative of the Pacific. Dr. Frishberg has a non-compensated relationship as a Delegate with AMA that is relevant to AAN interests or activities.
Albert Lai, MD	No disclosure on file
	No disclosure on file
Steven C. Cramer, MD, FAAN	Dr. Cramer has received personal compensation for serving as an employee of University of California. An immediate family member of Dr. Cramer has received personal compensation for serving as an employee of University of California. Dr. Cramer has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Astellas, Alevian, Bayer, BlueRock Therapeutics, BrainQ, Constant Therapeutics, Medtronic, MicroTransponder, Myomo, Myrobalan, NeuroTrauma Sciences, Simcere, and TRCare. Dr. Cramer has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for MUSC. Dr. Cramer has stock in Constant Therapeutics, Panaxium, and TRCare. The institution of Dr. Cramer has received research support from NIH; PCORI; Veterans Administration.
	No disclosure on file
Neil E. Schwartz, MD, PhD (Stanford Stroke Center)	Dr. Schwartz has nothing to disclose.
	No disclosure on file
	No disclosure on file

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