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

NCT02744287 | Terminated Phase 1 FDA Drug

• 1 other identifier: BP-012
• Study Type: interventional
• Target: 52
• Locations: 1 country
• Sites: 13

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.

Conditions

Metastatic Prostate Cancer; Metastatic Castration-resistant Prostate Cancer

Keywords

prostate stem cell antigen; BPX-601; AP1903; CAR-T; PSCA-CAR; castration-resistant prostate cancer; rimiducid; prostate cancer; PSCA; CRPC; mCRPC

Outcome Measures

Primary Outcomes (3)

• Dose Limiting Toxicity

  Incidence of dose limiting toxicity

  4 weeks after first rimiducid infusion (i.e., Day 35)

• Treatment emergent adverse events (AEs) and serious AEs (SAEs)

  Number of participants with adverse events (AEs) and serious AEs (SAEs) assessed for severity using NCI CTCAE v4.03

  180 days after BPX-601 treatment up to 15 years

• Maximum tolerated dose (MTD) and/or recommended phase 2 dose (RP2D)

  Identify the optimal dose of BPX-601 with rimiducid for Phase 2

  through Phase 1 completion, up to 5 years

Secondary Outcomes (2)

• Pharmacodynamics (PD) of BPX-601

  up to 1 year after treatment

• 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

Study Arms (2)

Arm 1: Phase 1 Dose Escalation

EXPERIMENTAL

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.

Biological: BPX-601; Drug: Rimiducid

Arm 2: Phase 2 Dose Expansion

EXPERIMENTAL

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.

Biological: BPX-601; Drug: Rimiducid

Interventions

BPX-601 BIOLOGICAL

Autologous T cells genetically modified with retrovirus vector containing PSCA-specific CAR and an inducible MyD88/Cluster Designation (CD)40 (iMC) co-stimulatory domain

Arm 1: Phase 1 Dose Escalation; Arm 2: Phase 2 Dose Expansion

Rimiducid DRUG

Dimerizer infusion to activate the iMC of the BPX-601 cells for improved proliferation and persistence

Also known as: AP1903

Arm 1: Phase 1 Dose Escalation; Arm 2: Phase 2 Dose Expansion

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Regeneron Pharmaceuticals: lead

Study Sites (13)

Moffitt Cancer Center

Tampa, Florida, 33612, United States

Location

Emory Winship Cancer Institute

Atlanta, Georgia, 30322, United States

Location

Rush University Medical Center

Chicago, Illinois, 60612, United States

Location

University of Chicago Medicine

Chicago, Illinois, 60637, United States

Location

Karmanos Cancer Institute

Detroit, Michigan, 48201, United States

Location

University of Nebraska

Omaha, Nebraska, 68198, United States

Location

John Theurer Cancer Center, Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States

Location

Roswell Park Cancer Institute

Buffalo, New York, 14263, United States

Location

Columbia University Medical Center

New York, New York, 10032, United States

Location

Duke University

Durham, North Carolina, 27705, United States

Location

Thomas Jefferson University

Philadelphia, Pennsylvania, 19107, United States

Location

Baylor Sammons Cancer Center

Dallas, Texas, 75246, United States

Location

The University of Texas MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Related Publications (1)

• Stein MN, Dumbrava EE, Teply BA, Gergis US, Guiterrez ME, Reshef R, Subudhi SK, Jacquemont CF, Senesac JH, Bayle JH, Scripture CD, Chatwal MS, Bilen MA, Stadler WM, Becerra CR. PSCA-targeted BPX-601 CAR T cells with pharmacological activation by rimiducid in metastatic pancreatic and prostate cancer: a phase 1 dose escalation trial. Nat Commun. 2024 Dec 30;15(1):10743. doi: 10.1038/s41467-024-53220-6.

  PMID: 39737899 DERIVED

MeSH Terms

Conditions

Prostatic Neoplasms

Interventions

AP 1903 reagent

Condition Hierarchy (Ancestors)

Genital Neoplasms, Male; Urogenital Neoplasms; Neoplasms by Site; Neoplasms; Genital Diseases, Male; Genital Diseases; Urogenital Diseases; Prostatic Diseases; Male Urogenital Diseases

Study Officials

• Clinical Trial Management

  Regeneron Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 11, 2016
• First Posted: April 20, 2016
• Study Start: November 30, 2016
• Primary Completion: March 14, 2023
• Study Completion: March 14, 2023
• Last Updated: February 23, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (13)