The partial clinical hold placed by the FDA on the phase 1 chimeric antigen receptor T-cell clinical trial, MELANI-01, has now been lifted.
The partial clinical hold placed by the FDA on the phase 1 chimeric antigen receptor (CAR) T-cell clinical trial (MELANI-01, NCT04142619) has now been lifted, announced Cellectis in a press release.
A safety report on 1 patient with relapsed and refractory multiple myeloma that was submitted to the FDA was the cause of the partial clinical hold. The patient was enrolled in the second level dose cohort after failing on multiple prior lines of therapy. The study drug, UCARTCS1A, induced fatal treatment-emergent adverse event (AE) of cardiac arrest. It was previously reported by Targeted Oncology that investigators were conducted an evaluation of the death case evaluating the case, which would include more details regarding the immediate and underlying causes of death in the patients.
In effort to restart the trial, Cellectis worked closely with the FDA to adjust the study protocol in a way that would enhance patient safety. Collaboration with the study’s investigators and on-site staff is ongoing to relaunch the trial and resume recruitment of patients with relapsed/refractory multiple myeloma.
“We remain confident in the potential clinical benefit of UCARTCS1 product candidate for patients with relapsed/refractory multiple myeloma, a widely unmet medical need that Cellectis will continue to address. The safety of patients enrolled in our clinical trials remains our priority, and we are committed to resuming the clinical development of this promising program,” said Carrie Brownstein, MD, chief medical officer, Cellectis, in a statement.
With the changes recommended by the FDA, the study will enroll patients with a confirmed diagnosis of active multiple myeloma who relapsed after multiple prior therapies. Patients are required to have an ECOG performance status of 0 or 1, no prior treatment with an investigational gene targeting CS1 or CAR therapy targeting CS1, and adequate organ function.
The primary end point of the study is the safety of the CAR T-cell product UCARTCS1A as defined by incidence, nature, and severity of both adverse events (AEs) and serious AEs. The prospective completion date of the study in November 2022.
UCARTCS1 is an allogeneic, off-the-shelf, gene-edited T-cell product. The agent was designed to treat hematologic malignancies that express CS1/SLAMF7, including multiple myeloma.
FDA lifts clinical hold on MELANI-01 study evaluating Cellectis’ product candidate ucartcs1 in multiple myeloma. News release. Cellectis. November 17, 2020. Accessed November 18, 2020. https://bit.ly/2UEv8YQ