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.
Patients with only bone marrow detectable disease in the absence of measurable disease by cross sectional imaging.
Patients with active, inoperative central nervous system (CNS) disease including leptomeningeal disease.
Active hepatitis B, C or HIV infection.
Inability to tolerate leukapheresis.