Multi-modular Chimeric Antigen Receptor T Cells tarGeting B7-H3 in Children, Teenage & Young Adult Sarcoma
MIGHTY is a multicentre, open label, phase 1 clinical trial of an Advanced Therapy Investigational Medicinal Product (ATIMP) in children, teenagers and young adults aged 1-24 with relapsed or refractory (r/r) rhabdomyosarcoma (RMS), Ewing sarcoma (ES) or desmoplastic small round cell tumour (DSRCT). The study will assess the feasibility of generating the ATIMP and administering hBRCA84D CAR T cells to patients with r/r RMS, ES or DSRCT.
• Age ≥ 1 and ≤ 24 years.
• Tissue diagnosis of RMS, ES or DSRCT
• Expression of B7-H3 in the tumour
• Relapsed or refractory disease after one or multiple lines of previous treatment.
• Measurable disease by cross sectional imaging. Patients with only bone marrow detectable disease (bone marrow aspirate or trephine) are NOT eligible for the study.
• At least 3 weeks or 5 half-lives, whichever is shorter, after treatment with agents on other early phase clinical trial.
• Performance status: Karnofsky (age ≥ 10 years) or Lansky (age \< 10) score ≥ 50%.
• Creatinine ≤1.5 x Upper Limit of Normal (ULN) for age, if higher, an estimated (calculated) creatinine clearance must be ≥ 60 ml/min/1.73 m2.
• Left ventricular ejection fraction (LVEF) ≥ 50%
⁃ Absolute lymphocyte count ≥ 0.25 x 109/L.
⁃ Women of childbearing potential (WOCBP) must have a negative pregnancy test and agree to comply with the pregnancy reporting requirements of the protocol (if applicable).
⁃ Written informed consent.