A Safety and Efficacy Study of Defibrotide in the Prevention of Chimeric Antigen Receptor-T-cell-associated Neurotoxicity

Summary

This is a prospective, open-label, single-arm study evaluating the safety and efficacy of defibrotide for the prevention of CAR-T-associated neurotoxicity in subjects with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) receiving Yescarta.

Timeline

Start

2019-10-04

Primary completion

2020-09-18

Completion

2020-09-30

Outcome

Missed primary endpoint

Stopped: “Primary endpoint would unlikely to be met based on the unplanned interim assessment on the first 20 efficacy evaluable patients.”

paper reduction was unlikely to meet the primary end point, so the study was terminated early. PMID 37399456 ↗

Publications

• Jacobson CA, Rosenthal AC, Arnason J, Agarwal S, Zhang P, Wu W, Amber V, Yared JA. A phase 2 trial of defibrotide for the prevention of chimeric antigen receptor T-cell-associated neurotoxicity syndrome. Blood Adv. 2023 Nov 14;7(21):6790-6799. doi: 10.1182/bloodadvances.2023009961.
• Danish H, Santomasso BD. Neurotoxicity Biology and Management. Cancer J. 2021 Mar-Apr 01;27(2):126-133. doi: 10.1097/PPO.0000000000000507.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Defibrotide	Oligonucleotide (other)	2.5 mg/kg	—
Subject	Defibrotide	Oligonucleotide (other)	6.25 mg/kg	—

Indications