Phase 1 Study of CK0801 in Treatment of Bone Marrow Failure Syndromes.

Tapan M KadiaMeixian HuangNaveen PemmarajuHussein A AbbasChristopher LyLucia MasarovaMusa YilmazMi-Ae LyuKe ZengTara SadeghiRobin CookCourtney D DiNardoNaval DaverGhayas C IssaElias JabbourGautam BorthakurNitin JainGuillermo Garcia-ManeroSimrit ParmarChristopher FlowersHagop KantarjianSrdan Verstovsek

Published in: NEJM evidence (2024)

In previously treated patients, CK0801 demonstrated no dose-limiting toxicity and showed evidence of efficacy, providing proof of concept for targeting inflammation as a therapy for bone marrow failure. (Funded by Cellenkos Inc.; Clinicaltrials.gov number, NCT03773393.).

Keyphrases

bone marrow
end stage renal disease
mesenchymal stem cells
oxidative stress
newly diagnosed
ejection fraction
chronic kidney disease
protein kinase
prognostic factors
open label
clinical trial
patient reported outcomes
patient reported
combination therapy