On July 6, 2020, the US Food and Drug Administration (FDA) issued a clinical hold for the phase 1 MELANI-01 trial (ClinicalTrials.gov Identifier: NCT04142619), a dose-escalation study evaluating an investigational universal chimeric antigen receptor (CAR) T-cell (CAR-T) product in patients with relapsed or refractory multiple myeloma.
Developed by Cellectis, the product is known as UCARTCS1A and consists of allogenic T-cells that are genetically engineered to express a CAR against CS1, also known as SLAMF7. CS1 is “highly” expressed in multiple myeloma, according to the company’s press release.
The trial was put on clinical hold after a safety report revealed that 1 trial participant died from cardiac arrest, which was deemed a treatment-emergent adverse event. The participant received dose level 2 of the investigational product and had prior treatment with “numerous lines” of therapy, including autologous CAR T cells.
“Clinical evaluation of the case remains ongoing and additional details as to the immediate and underlying causes of this event are being collected,” the press release stated.
Cellectis is amending the MELANI-01 clinical protocol, among other requests by the FDA.
The clinical hold does not affect phase 1 dose-escalation trials AMELI-01 and BALLI-01, which are each evaluating different universal CAR-T products from Cellectis.
The AMELI-01 trial (ClinicalTrials.gov Identifier: NCT03190278) is evaluating a CAR-T product that targets CD123 in patients with relapsed and refractory acute myeloid leukemia, and the BALLI-01 trial (ClinicalTrials.gov Identifier: NCT04150497) is evaluating a CAR-T product that targets CD22 in patients with relapsed and refractory B-cell acute lymphoblastic leukemia.
Cellectis reports clinical hold placed on MELANI-01 study [news release]. New York, NY: Cellectis Inc.; July 6, 2020. Accessed July 11, 2020.