Randomized placebo-controlled trial of CL2020, an allogenic muse cell-based product, in subacute ischemic stroke.
Kuniyasu NiizumaShin-Ichiro OsawaHidenori EndoShin-Ichi IzumiKota AtakaAkihiro HirakawaMasao IwanoTeiji TominagaPublished in: Journal of cerebral blood flow and metabolism : official journal of the International Society of Cerebral Blood Flow and Metabolism (2023)
Effective treatments for stroke after the acute phase remain elusive. Muse cells are endogenous, pluripotent, immune-privileged stem cells capable of selectively homing to damaged tissue after intravenous injection and replacing damaged/lost cells via differentiation. This randomized, double-blind, placebo-controlled trial enrolled ischemic stroke patients with modified Rankin Scale (mRS) ≥3. Randomized patients received a single intravenous injection of an allogenic Muse cell-based product, CL2020 (n = 25), or placebo (n = 10), without immunosuppressant, 14-28 days after stroke onset. Safety (primary endpoint: week 12) and efficacy (mRS, other stroke-specific measures) were assessed up to 52 weeks. Key efficacy endpoint was response rate (percentage of patients with mRS ≤2 at week 12). To week 12, 96% of patients in the CL2020 group experienced adverse events and 28% experienced adverse reactions (including one Grade 4 status epilepticus), compared with 100% and 10%, respectively, in the placebo group. Response rate was 40.0% (95% CI, 21.1-61.3) in the CL2020 group and 10.0% (0.3-44.5) in the placebo group; the lower CI in the CL2020 group exceeded the preset efficacy threshold (8.7% from registry data). This randomized placebo-controlled trial demonstrated CL2020 is a possible effective treatment for subacute ischemic stroke.Registry information: JAPIC Clinical Trials Information site (JapicCTI-184103, URL: https://www.clinicaltrials.jp/cti-user/trial/ShowDirect.jsp?japicId=JapicCTI-184103).
Keyphrases
- double blind
- placebo controlled
- clinical trial
- phase iii
- phase ii
- study protocol
- atrial fibrillation
- stem cells
- end stage renal disease
- open label
- ejection fraction
- induced apoptosis
- chronic kidney disease
- newly diagnosed
- single cell
- healthcare
- emergency department
- cell death
- cell therapy
- peritoneal dialysis
- electronic health record
- cell cycle arrest
- randomized controlled trial
- signaling pathway
- artificial intelligence
- patient reported