Cellectis Halts UCART123 Studies Following FDA Clinical Hold
Cellectis has received notification from the U.S. Food and Drug Administration (FDA) placing a clinical hold on its UCART123 program.
Cellectis, a biopharmaceutical company, announced it has received notification from the U.S. Food and Drug Administration (FDA) placing a clinical hold on its two ongoing Phase 1 studies for UCART123. The studies were investigating the treatment for acute myeloid leukemia (AML) and blastic plasmacytoid dendritic cell neoplasm (BPDCN).
The company is collaborating with investigators and the FDA to resume the trials with an amended protocol that includes a reduced dosage of UCART123. The clinical hold was initiated following a patient fatality in the BPDCN clinical trial. The deceased was a 78-year-old male patient who had previously undergone treatment and had relapsed/refractory BPDCN.
The patient experienced a Grade 2 Cytokine Release Syndrome (CRS) and a Grade 3 lung infection five days after receiving UCART123 cells. Despite initial improvement, the patient's condition rapidly worsened, leading to death on Day 9 due to Grade 5 CRS and Grade 4 Capillary Leak Syndrome. Cellectis reported the event to the FDA, prompting the clinical hold.
Cellectis stated that the patient had been treated with medications for CRS management, including corticosteroids and tocilizumab. The company is working closely with regulatory authorities to ensure safety and aims to resume the studies as soon as possible.