Safety and Activity Study of PSCA-Targeted CAR-T Cells (BPX-601) in Subjects With Selected Advanced Solid Tumors

Phase 1

Suspended

• Conditions: Metastatic Castration-resistant Prostate Cancer; Metastatic Prostate Cancer
• Interventions: Biological: BPX-601; Drug: Rimiducid

• Registration Number: NCT02744287
• Lead Sponsor: Bellicum Pharmaceuticals

Brief Summary

The purpose of this study is to evaluate the safety and activity of BPX-601 CAR-T cells in participants with previously treated advanced solid tumors (prostate) expressing high levels of prostate stem cell antigen (PSCA). Participants' T cells are modified to recognize and target the PSCA tumor marker on cancer cells.

Detailed Description

The goal of this study is to characterize the feasibility, safety, and clinical activity of PSCA-specific CAR-T cells, BPX-601, administered with rimiducid to subjects with previously treated, PSCA-positive advanced solid tumors (prostate). BPX-601 CAR-T cells are genetically engineered to express a chimeric antigen receptor (CAR) to target the PSCA antigen and a rimiducid-inducible signaling domain which functions as a molecular "go-switch" to enhance activation and proliferation.

Phase 1: Cell dose escalation to identify the maximum dose of BPX-601 administered with single or repeat doses of rimiducid.

Phase 2: Indication-specific dose expansion to assess the safety, pharmacodynamics (including BPX-601 persistence), and clinical activity at the recommended dose identified in Phase 1 in various PSCA-expressing solid tumors.

Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations
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Related News

Study Timeline

• Study First Submitted: 2016-04-11
• Study First Submitted QC: 2016-04-15
• Study First Posted: 2016-04-20
• Study Start: 2016-11
• Status Verified: 2023-04
• Last Update Submitted: 2023-04-18
• Last Update Posted: 2023-04-20
• Primary Completion: 2025-10
• Study Completion: 2026-02

Recruitment & Eligibility

• Status: SUSPENDED
• Sex: All
• Target Recruitment: 151

Inclusion Criteria

• Metastatic castration-resistant prostate cancer (mCRPC), with progressive disease per PCWG3 criteria during or following the direct prior line of therapy.
• Measurable disease per RECIST v1.1 at baseline; subjects with mCRPC with bone only metastases must have measurable PSA.
• Age ≥18 years.
• Life expectancy > 12 weeks.
• ECOG 0-1
• Adequate organ function.

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Exclusion Criteria

• Prostate cancer with unstable bone lesions or symptomatic/untreated coagulopathy, or history of > Grade 2 hematuria within the previous 6 months.
• Prior CAR T cell or other genetically-modified T cell therapy. Prior treatment with an immune-based therapy for the treatment of prostate cancer, including cancer vaccine therapies are allowable.
• Symptomatic, untreated, or actively progressing central nervous system metastases.
• Impaired cardiac function or clinically significant cardiac disease.
• Pregnant or breastfeeding.
• Participant requires chronic, systemic steroid therapy.
• Severe intercurrent infection.
• Known HIV positivity.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Arm 1: Phase 1 Dose Escalation	BPX-601	Participants with advanced prostate cancer will receive an intravenous infusion of BPX-601 followed by one or more intravenous infusions of rimiducid. Dose escalation of BPX-601 will continue until the recommended cell dose level is reached.
Arm 1: Phase 1 Dose Escalation	Rimiducid	Participants with advanced prostate cancer will receive an intravenous infusion of BPX-601 followed by one or more intravenous infusions of rimiducid. Dose escalation of BPX-601 will continue until the recommended cell dose level is reached.
Arm 2: Phase 2 Dose Expansion	BPX-601	Participants with advanced prostate cancer will receive an intravenous infusion of BPX-601 at the recommended cell dose level followed by one or more intravenous infusions of rimiducid.
Arm 2: Phase 2 Dose Expansion	Rimiducid	Participants with advanced prostate cancer will receive an intravenous infusion of BPX-601 at the recommended cell dose level followed by one or more intravenous infusions of rimiducid.

Primary Outcome Measures

Name	Time	Method
Dose Limiting Toxicity	4 weeks after first rimiducid infusion (i.e., Day 35)	Incidence of dose limiting toxicity
Treatment emergent adverse events (AEs) and serious AEs (SAEs)	180 days after BPX-601 treatment up to 15 years	Number of participants with adverse events (AEs) and serious AEs (SAEs) assessed for severity using NCI CTCAE v4.03
Maximum tolerated dose (MTD) and/or recommended phase 2 dose (RP2D)	through Phase 1 completion, up to 5 years	Identify the optimal dose of BPX-601 with rimiducid for Phase 2

Secondary Outcome Measures

Name	Time	Method
Antitumor activity of BPX-601	From the time of BPX-601 cell infusion until confirmed disease progression or death due to any cause, the start of new anticancer therapy, or withdrawal, whichever comes first, as assessed for up to 5 years after the last subject has been enrolled	Percentage of subjects with objective response determined by the investigator according to the Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 or the Prostate Cancer Working Group 3 (PCWG3) criteria
Pharmacodynamics (PD) of BPX-601	up to 1 year after treatment	Change from baseline in pharmacodynamic blood biomarkers - markers of BPX-601 CAR-T cells

Trial Locations

Locations (13)

Baylor Sammons Cancer Center; 🇺🇸 Dallas, Texas, United States

John Theurer Cancer Center, Hackensack University Medical Center; 🇺🇸 Hackensack, New Jersey, United States

Moffitt Cancer Center; 🇺🇸 Tampa, Florida, United States

University of Nebraska; 🇺🇸 Omaha, Nebraska, United States

Emory Winship Cancer Institute; 🇺🇸 Atlanta, Georgia, United States

Rush University Medical Center; 🇺🇸 Chicago, Illinois, United States

University of Chicago Medicine; 🇺🇸 Chicago, Illinois, United States

Karmanos Cancer Institute; 🇺🇸 Detroit, Michigan, United States

Columbia University Medical Center; 🇺🇸 New York, New York, United States

Roswell Park Cancer Institute; 🇺🇸 Buffalo, New York, United States

Duke University; 🇺🇸 Durham, North Carolina, United States

The University of Texas MD Anderson Cancer Center; 🇺🇸 Houston, Texas, United States

Thomas Jefferson University; 🇺🇸 Philadelphia, Pennsylvania, United States