Design of A Phase 3B Trial of Debamestrocel in ALS (MDA 2024) - Feb 16, 2024 - Abstract #M201; Pres time: Mar 4, 2024; Location: Poster Hall; "Background: Neurotrophic factor (NTF)-secreting mesenchymal stem cells (MSC-NTF) are a novel cell-therapeutic approach aimed at effectively delivering NTFs directly to the site of damage in ALS patients. Participants who are receiving standard of care (e.g., riluzole, edaravone, sodium phenylbutyrate/taurursodiol) must be on a stable dose for at least 60 days before Screening. Outcome Measures: Participants will undergo several safety assessments and efficacy measures including the ALSFRS-R, CAFS, rate of survival, respiratory function (SVC), muscle strength (HHD), patient-reported outcomes such as ALSAQ-40, caregiver burden interview, as well as biomarkers and genetic testing."
P3 data
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Merit Cudkowicz; MD; MSC; Massachusetts General Hospital; Bob Dagher; MD; Brainstorm Cell Therapeutics; Nathan Staff; MD; Mayo Clinic College of Medicine; Jeremy Shefner; MD; Barrow Neurological Institute; Jinsy Andrews; MD; Columbia University; Jonathan Katz; MD; California Pacific Medical Center; James Berry; MD; Massachusetts General Hospital; Margaret Owegi; MD; University of Massachusetts Medical School; Catherine Douthwright; MD; University of Massachusetts Medical School; Jenny Li; PhD; Massachusetts General Hospital; Melanie Quintana; PhD; Berry Consultants; Yossef Levy; PhD; Massachusetts General Hospital; Chaim Lebovits; Massachusetts General Hospital; Robert Brown; MD; University of Massachusetts Medical School; Tony Windebank; MD; Mayo Clinic College of Medicine; Stacy Lindborg; PhD; Brainstorm Cell Therapeutics
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