Accepted for/Published in: JMIR Research Protocols
Date Submitted: Mar 11, 2022
Date Accepted: May 31, 2022
Warning: This is an author submission that is not peer-reviewed or edited. Preprints - unless they show as "accepted" - should not be relied on to guide clinical practice or health-related behavior and should not be reported in news media as established information.
Intravenous infusion of auto serum-expanded autologous mesenchymal stem cells in chronic brain injury patients: a study protocol for a Phase II trial
ABSTRACT
Background:
Brain injury resulting from motor vehicle accidents and falls, hypoxic insult and other conditions is one of the leading causes of disability and death in the world. Current treatments are limited, but include continuous rehabilitation especially for the chronic state. Recent studies have demonstrated that intravenous infusion of mesenchymal stem cells (MSCs) have therapeutic efficacy for several neurological diseases including stroke and spinal cord injury (SCI).
Objective:
The objective of this investigator-initiated clinical trial is to assess the safety and potential efficacy of intravenous infusion of auto serum-expanded autologous MSCs for chronic brain injury patients.
Methods:
This (Phase II) trial will be a single arm, open-label trial with the primary objective to confirm the safety and efficacy of the auto serum-expanded autologous MSCs under Good Manufacturing Practice (GMP) production (STR-01) when administered to patients with chronic brain injury. Estimated enrollment is 6-20 patients with a modified Rankin Scale (mRS) grade ranging from 3 to 5 and an unlimited number of patients in mRS grade 2 during the study. Assessment of safety and the proportion of cases that the mRS grade improves one point or more at 180 days after injection of STR-01 will be measured after MSC infusion.
Results:
Recruitment of the patients started on April 20, 2018, and is underway.
Conclusions:
This Phase II study will aim to address the safety and efficacy of intravenous infusion of MSCs for patients with chronic brain injury. The use of STR-01 has been performed for patients with cerebral infarction and spinal cord injury providing encouraging results. The potential of systemic administration of auto serum-expanded autologous MSCs for therapeutic efficacy for chronic brain injury should be evaluated given the safety and promising results for stroke and SCI. Clinical Trial: JMACCT, JMA-IIA00333, https://dbcentre3.jmacct.med.or.jp/JMACTR/App/JMACTRS06/JMACTRS06.aspx?seqno=7577
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