NCT04171843

A Dose-escalation Study to Evaluate the Safety and Clinical Activity of PBCAR269A, With or Without Nirogacestat, in Study Participants With Relapsed/Refractory Multiple Myeloma

A Phase 1/2a, Open-label, Dose-escalation, Dose-expansion Study to Evaluate the Safety and Clinical Activity of PBCAR269A, With or Without Nirogacestat, in Study Participants With Relapsed/Refractory Multiple Myeloma

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 48 (actual)
• Sponsor: Precision BioSciences, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This is a Phase 1/2a, nonrandomized, open-label, parallel assignment, single-dose, dose-escalation, and dose-expansion study to evaluate the safety and clinical activity of PBCAR269A, with or without nirogacestat, in adults with r/r MM. Study subjects in Cohort A will receive PBCAR269A and study subjects in Cohort B will receive PBCAR269A and nirogacestat. At each dose level, study subjects in Cohort A and Cohort B will receive the same dose of PBCAR269A. In Cohort B, all study subjects will follow the same dosing regimen of nirogacestat. This study was terminated prior to beginning of Phase II due to lack of sufficient therapeutic effect

Detailed description

This is a multicenter, nonrandomized, open-label, parallel assignment, single-dose, dose-escalation, and dose-expansion study to evaluate the safety and clinical activity of PBCAR269A, with or without nirogacestat, in adults with r/r MM. Before initiating the study treatment PBCAR269A, all study participants will be administered lymphodepletion chemotherapy. The initial lymphodepletion chemotherapy regimen will be composed of fludarabine and cyclophosphamide during the Screening Period. Study subjects in Cohort B will also receive nirogacestat. On Day 0 of the Treatment Period, study participants will receive a single intravenous (IV) infusion of PBCAR269A. All subjects are monitored during the treatment period through Day 28. All subjects who receive a dose of PBCAR269A, with or without nirogacestat, will be followed in a separate long-term follow-up (LTFU) study for 15 years after exiting this study.

Conditions

• Relapsed/Refractory Multiple Myeloma

Interventions

Type	Name	Description
GENETIC	PBCAR269A	Allogeneic anti-BCMA CAR T-cell
DRUG	Fludarabine	Fludarabine is used for lymphodepletion.
DRUG	Cyclophosphamide	Cyclophosphamide is used for lymphodepletion.
DRUG	Nirogacestat	Allogeneic anti-BCMA CAR T-cell

Timeline

• Start date: 2020-04-30
• Primary completion: 2022-10-19
• Completion: 2022-10-19
• First posted: 2019-11-21
• Last updated: 2023-09-21

Locations

5 sites across 1 country: United States

Regulatory

• FDA-regulated drug study