The manufacturer is working with the Food and Drug Administration to address any requests including changes to the protocol of the MELANI-01 trial to enhance patient safety.
The Food and Drug Administration (FDA) has issued an order to delay the open-label, first-in-human phase 1 MELANI-01 trial, which is evaluating the CAR T-cell product UCARTCS1A in patients with relapsed or refractory multiple myeloma, according to a press release from the product’s manufacturer.
The agency issued the clinical hold, which orders a pharmaceutical company to either delay or suspend a clinical trial, after it received a safety report that focused on a patient enrolled in the trial. The patient, who had received previous treatment with multiple lines of therapy, including autologous CAR T-cells, died after experiencing a treatment-emergent cardiac arrest.
Further evaluation of the case is ongoing and additional details as to any immediate or underlying causes of the cardiac arrest are being collected, according to the release.
“We share the FDA’s commitment to patient safety and are working collaboratively with the agency and the investigators to resolve this clinical hold,” said Dr. Carrie Brownstein, chief medical officer of Cellectis, in the release. “The safety of patients enrolled in our clinical trials is our utmost priority and we at Cellectis remain committed to safely resuming the clinical development of UCART product candidate targeting CS1 for patients with multiple myeloma and unmet medical need.”
The patient who died was receiving UCARTCS1a – an allogeneic, off-the-shelf, gene-edited T-cell product candidate designed to treat CS1/SLAMF7-expressing hematologic malignancies – at dose level two. However, prior to the FDA issuing the clinical hold, the manufacturer expanded enrollment to a smaller dose, or dose level one, which, according to the release, may be the appropriate dose moving forward.
The manufacturer, according to the release, is working with the agency to address any requests including changes to the protocol of the MELANI-01 trial to enhance patient safety.