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FDA places clinical hold on trials of UCART123 for blood cancers
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The FDA placed a clinical hold on two phase 1 trials designed to evaluate UCART123 for the treatment of patients with acute myeloid leukemia or blastic plasmacytoid dendritic cell neoplasm.
Cellectis announced it is working with investigators and the FDA to resume evaluation of UCART123, a chimeric antigen receptor T-cell therapy, with an amended protocol that includes a reduced dose.
The FDA initiated the clinical hold after Cellectis reported a death in the phase 1 ABC study, designed to evaluate UCART123 in patients with blastic plasmacytoid dendritic cell neoplasm.
As a preconditioning regimen, patients received 30 mg/m fludarabine daily for 4 days and 1g/m cyclophosphamide daily for 3 days. Patients then received 6.25 x 105 UCART123 cells per kilogram, the first dose level explored in the protocol, without complication.
The patient who died experienced grade 2 cytokine release syndrome and grade 3 lung infection on day 5, followed by grade 5 cytokine release syndrome and grade 4 capillary leak syndrome on day 8.
Despite treatment to manage cytokine release syndrome, the patient died on day 9.
The data safety monitoring board recommended lowering the dose to 6.25 x 104 UCART123 cells per kilogram in both studies and capping cyclophosphamide to a total dose of 4 g over 3 days.