Phase I/II Trial of Intracerebral Transplantation of Autologous Bone Marrow Stromal Cells Combined With Recombinant Peptide Scaffold for Patients With Chronic Intracerebral Hemorrhage

Intracerebral hemorrhage stands as a leading global cause of death and disability, posing a significant challenge with limited treatment options, especially for chronic patients. Recent advances of stem cell therapies have opened new avenues for therapeutic potential. The investigators' previous preclinical research has demonstrated that intracerebral transplantation of bone marrow stromal cells (BMSCs) combined with a recombinant human collagen type I scaffold enables higher cell survival and engraftment, holds promising potential. The investigators present the protocol for a novel clinical trial, named "Research on Advanced Intervention using Novel Bone Marrow Stem Cells for Chronic Intracerebral Hemorrhage" (RAINBOW-Hx).

Methods and Analysis:

RAINBOW-Hx is a phase I/II, open-label, uncontrolled study with the primary objective of assessing the safety and efficacy of intracerebral transplantation of autologous BMSCs combined with the scaffold (HUFF-01) in patients with chronic intracerebral hemorrhage. Eight patients, experiencing moderate to severe neurological deficits for 12 months or longer, will be enrolled. The hemorrhage location will be limited to the basal ganglia and thalamus. Approximately 50 mL of bone marrow will be extracted from the iliac bone of each patient, and BMSCs will be cultured using autologous platelet lysate. Three days before transplantation, BMSCs will be combined with the scaffold to generate HUFF-01. Each patient will receive a 50,000 HUFF-01 dose, containing approximately 50 million BMSCs, through stereotactic transplantation into the hemorrhagic cavity. Neurological assessments, magnetic resonance imaging, 18F-fluorodeoxyglucose positron emission tomography, and 123I-Iomazenil single-photon emission computed tomography will be performed for one year after administration.