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.
Single group
Biological/Vaccine: hBRCA84D CAR T cells
DrugThe hBRCA84D CAR T cells target B7-H3 positive cells.
Safety of administering the ATIMP
Incidence of grade 3-5 toxicity causally related to the ATIMP, particularly severe cytokine release syndrome and severe neurotoxicity.
Number of therapeutic products generated and the number of ATIMPs infused after successful manufacture
Feasibility of generation of the ATIMP as evaluated by the number of therapeutic products generated and the number of ATIMPs infused after successful manufacture.
Objective response rate
Based on cross-sectional imaging after the ATIMP intravenous administration
Progression Free Survival (PFS)
Progression Free Survival (PFS) after the ATIMP intravenous administration
Time to Progression (TTP)
Time to Progression (TTP) after the ATIMP intravenous administration
Overall survival
Overall Survival after the ATIMP intravenous administration
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