UCMSC-Exo for chemotherapy-induced myelosuppression in acute myeloid leukemia: a phase I clinical trial protocol

Myelosuppression induced by chemotherapy represents a critical bottleneck for patients with hematological malignancies, leading to a series of severe complications responsible for chemotherapy dose reduction, interruptions, and even treatment-related death. There exists an urgent need for strategies to repair the bone marrow microenvironment and thus promote early recovery from myelosuppression. Umbilical cord-derived mesenchymal stem cell exosomes (UCMSC-Exo), as the key effector of stem cells, exhibit multi-target characteristics, making them a promising candidate for bone marrow structural reconstruction and functional remodeling. This study is a single-arm, open-label, dose-escalation, single-center, phase I clinical trial designed to evaluate the safety, tolerability, and preliminary efficacy of UCMSC-Exo in the treatment of chemotherapy-induced myelosuppression in patients with acute myeloid leukemia. Nine to eighteen eligible participants will be enrolled sequentially into three UCMSC-Exo dose-escalation groups to receive a single infusion, after which they will be followed up for 12 months. The primary endpoints focus on safety and tolerability. Secondary endpoints include parameters assessing myelosuppression recovery, the incidence of myelosuppression-related complications, and supportive care. The study protocol has been approved by the Ethics Committee of Union Hospital, Tongji Medical College, Huazhong University of Science and Technology.Clinical trial registration: NCT06245746.