A Double-Blind, Controlled Phase 2 Study of the Safety and Efficacy of Modified Stem Cells

A Double-Blind, Controlled Phase 2 Study of the Safety and Efficacy of Modified Stem Cells (SB623) in Patients With Chronic Motor Deficit From Traumatic Brain Injury (TBI)

ClinicalTrials.gov Identifier: NCT02416492

UCLA IRB #: 13-001411

Sponsor: SanBio, Inc.

Time Frame: Change from baseline in Fugl-Meyer Motor Scale (FMMS) score at Month 6 

Purpose: The primary purpose of the clinical study is to evaluate the clinical efficacy of intracranial administration of SB623 cells on patients with chronic motor deficit from Traumatic Brain Injury. A secondary purpose of the study is 1) to evaluate the effect of intracranial administration of SB623 cells on disability parameters and 2) to evaluate the safety and tolerability of intracranial administration of SB623 cells. Patients with stable, chronic motor deficits secondary to focal traumatic brain injury must be 12 months post TBI.

SB623 cells are adult bone-marrow-derived cells that are transiently transfected with a plasmid construct encoding the intracellular domain of human Notch-1. These cells produce trophic factors that protect neurons in models of ischemic insult. In a rat contusion model of TBI, implantation of SB623 around the area of the injury resulted in significant improvement of motor function. Further, safety evaluation of animal models implanted with SB623 cells have shown no adverse effects and no SB623-related clinical, laboratory, or histological abnormalities were found. A 2-year Phase 1/2A dose escalation study (NCT01287936) of SB623 stereotactically implanted into the brains of patients with chronic motor deficits due to ischemic stroke has completed.The study has shown statistically-significant improvements in motor function in each of three scales: the European Stroke Scale (ESS), the National Institute of Health Stroke Scale (NIHSS) and the Fugl-Meyer scale. The study showed no serious adverse events likely attributed to SB623, and only minor adverse events mostly grade 1 or 2 (with one grade 3) that were unrelated, unlikely related, or possibly related to SB623. No dose-limiting toxicities were observed.

Principal Investigator: Daniel Lu, MD, PhD

Contact: Maggie Lindenbaum

Phone: (310) 794-3788

Email: [email protected]