A 2nd, pre-specified interim analysis for a trial (NCT03280056) of neurotrophic factor (NTF)-secreting stem-cell therapy (NurOwn; BrainStorm Cell Therapeutics, New York, NY) for treatment of amyotrophic lateral sclerosis (ALS) has been announced. This review was done by an independent Data Safety Monitoring Board (DSMB). Review of safety outcomes for the 1st 106 participants who received repeat dosing of the therapy indicated that the phase 3 clinical trial should continue as planned.
This randomized placebo-controlled phase 3 trial is now fully enrolled with 200 participants with ALS randomly assigned 1:1 to receive the therapy or placebo. Top-line data is anticipated in the 4th quarter of 2020.
“After reviewing all of the safety data as of September 30th, the DSMB has recommended the study continue without any changes in the protocol. We did not identify any significant safety concerns,” said Carlayne Jackson, MD, FAAN professor of neurology and otolaryngology UT Health San Antonio, and the DSMB chairperson.
"We are very pleased with the DSMB recommendation that the phase 3 clinical trial continue without any protocol modification. This represents an important clinical trial advancement for BrainStorm and for the development of NurOwn as an innovative cellular therapy approach for ALS patients. Enrollment is now complete and top line data is expected in Q4 2020, which will be used to support an FDA Biologics License Application," said Ralph Kern MD, MHSc, BrainStorm chief operating officer and chief medical officer.
Neha M. Kramer, MD; Jessica Besbris, MD; and Rima M. Dafer, MD, MPH
Stefan Nicolau, MD, and Elie Naddaf, MD