A Clinical Study Evaluating the Safety and Efficacy of Local Injection of ACT#001 Chimeric Antigen Receptor T Cells in the Treatment of Castration-Resistant Prostate Cancer

NCT07240857 | Not Yet Recruiting Early Phase 1 DMC

• 1 other identifier: ACT#001
• Study Type: interventional
• Target: 18
• Locations: 0 countries
• Sites: N/A

Brief Summary

An exploratory clinical study evaluating the safety and efficacy of ACT#001 chimeric antigen receptor T-cell (CAR-T cell) local injection in the treatment of castration-resistant prostate cancer.

Conditions

Castration-Resistant Prostate Cancer (CRPC)

Keywords

CAR-T; CRPC

Outcome Measures

Primary Outcomes (1)

• DLT

  adverse events occurring within 28 days after the first infusion that are related to the study drug (definitely related, probably related, possibly related).

  28 days

Secondary Outcomes (16)

• PSA50 response rate

  6 months

• DoPSA50（duration of PSA50 response）

  6 months

• DRRPSA50（durable PSA50 response rates）

  6 months

• TTPSAP50（time to PSA50 progression )

  6 months

• PSA90 response rate

  6 Months

Other Outcomes (8)

• The proportion of immune cell subsets in peripheral blood

  28 DAYS

• The proportion of subgroups of the intestinal microbiota

  28 DAYS

• The correlation between therapeutic effect and PSA level

  6 months

Study Arms (2)

Rapid titration dose exploration phase

EXPERIMENTAL

It is 18×10⁶ CAR-T cells, and it is planned to include 1 participant,If the participant experiences no dose-limiting toxicity (DLT) or grade 2 adverse reactions, the dose will be escalated to the 5×10⁷ CAR-T cells group for the "3+3" dose escalation; if the patient develops DLT or grade 2 adverse reactions, 2 additional participants will be enrolled, and the study will switch to the traditional "3+3" design for dose exploration.

Biological: Intraprostatic or localized lesion injection of ACT#001-PSMA CAR-T cells under transrectal ultrasound (TRUS) guidance

"3+3" dose escalation phase

EXPERIMENTAL

Dose escalation will be conducted sequentially in two dose groups: 5×10⁷ CAR-T cells and 1×10⁸ CAR-T cells. In view of the particularity of cell preparations, the actual administration dose in each dose group is allowed to have a fluctuation of ±30%. Each dose group will first enroll 3 participants. Within the same dose group, the interval between cell infusions for the first 2 participants should not be less than 14 days. During dose escalation, the interval between the infusion time of the last participant in each dose level and that of the first participant in the next dose level should be at least 28 days.If no dose-limiting toxicity (DLT) is observed in all participants within the same dose group, the dose will be escalated to the next level. If 1 case of DLT occurs, 3 additional participants will be enrolled in this dose group (with a total of 6 participants enrolled). If no new DLT occurs, the dose will be escalated to the next level.

Biological: Intraprostatic or localized lesion injection of ACT#001-PSMA CAR-T cells under transrectal ultrasound (TRUS) guidance

Interventions

Intraprostatic or localized lesion injection of ACT#001-PSMA CAR-T cells under transrectal ultrasound (TRUS) guidance BIOLOGICAL

Prior to CAR-T cell infusion, subjects will receive lymphodepleting chemotherapy based on fludarabine and cyclophosphamide. Surgical method : 1)The patient is taken to the operating room, and the anesthesia method is intravenous anesthesia or local anesthesia. A digital rectal examination is performed, and the anus is dilated to accommodate three fingers. The genitals and perineum are prepared and covered in a sterile manner. An ultrasound probe is inserted into the rectum; (2) Under ultrasound guidance, CAR-T cells are injected into the prostate or localized lesions via the rectum or perineum; (3) Then, the TRUS probe is removed from the patient's rectum.

"3+3" dose escalation phase; Rapid titration dose exploration phase

Eligibility Criteria

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

You may qualify if:

• understanding of this study and voluntarily signs the informed consent form;
• Aged ≥ 18 years;
• Expected survival time of at least 3 months;
• non-metastatic CRPC (nmCRPC) (local recurrence) or metastatic CRPC (mCRPC)；
• continue GnRH analog therapy；
• at least one evaluable lesion per RECIST 1.1;
• Has received at least one type of novel endocrine therapy；
• PSMA positive expression rate \> 10%;
• Eastern Cooperative Oncology Group (ECOG) 0-1;
• Well organ function
• Left Ventricular Ejection Fraction (LVEF) \> 50%;
• Oxygen saturation \> 92% without oxygen supplementation;
• agree to use effective contraceptive measures

You may not qualify if:

• Presence of brain metastasis;
• Organ transplantation or pending organ transplantation;
• Uncontrolled large-volume serous cavity effusions；
• A history of autoimmune diseases；
• A history of receiving other cell therapies or genetically modified cell therapies (e.g., TCR-T therapy, CAR-T therapy);
• A history of receiving any PSMA-targeted therapy;
• Requirement for steroid therapy (except for physiological replacement therapy);
• A history of receiving immunotherapies;
• A history of clinically significant central nervous system (CNS) diseases (either past or present at screening);
• (12) A history of other untreated malignant tumors; (13) Participants with severe cardiovascular diseases; (14) Active infectious diseases; (15) Active hepatitis B or hepatitis C virus infection; (16) Active Epstein-Barr virus (EBV) or cytomegalovirus (CMV) infection (17) Intolerance or allergy to cyclophosphamide or fludarabine chemotherapeutic drugs; (18) No accessible injection sites; (19) Assessment by the investigator that the participant is unsuitable for participation in this clinical study.

Sponsors & Collaborators

• Zhejiang University: lead
• Orig Stone Biotech(Zhejiang) Co.,Ltd.: collaborator

MeSH Terms

Interventions

Counseling

Intervention Hierarchy (Ancestors)

Mental Health Services; Behavioral Disciplines and Activities; Community Health Services; Health Services; Health Care Facilities Workforce and Services

Central Study Contacts

Weijia Fang, MD

CONTACT

86-0571-87237587; weijiafang@zju.edu.cn

Zhou Tong, MD

CONTACT

zju_tz@zju.edu.cn

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: August 23, 2025
• First Posted: November 21, 2025
• Study Start: December 31, 2025
• Primary Completion (Estimated): October 31, 2026
• Study Completion (Estimated): October 31, 2027
• Last Updated: November 21, 2025

Record last verified: 2025-08