Castration-Resistant Prostate Cancer (CRPC)Early Phase 1NCT07240857
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
A Early Phase 1 study testing Intraprostatic or localized lesion injection of ACT#001-PSMA CAR-T cells under transrectal ultrasound (TRUS) guidance in people with Castration-Resistant Prostate Cancer (CRPC), run by Zhejiang University at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Castration-Resistant Prostate Cancer (CRPC)
- What is being tested?
- Intraprostatic or localized lesion injection of ACT#001-PSMA CAR-T cells under transrectal ultrasound (TRUS) guidance
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 1 siteChina
- Study dates
- 12 Apr 2026to 31 Oct 2027
- Phase
- Early Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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;
Show all 13Show fewer
- 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
Who cannot join
- 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;
Show all 10Show fewer
- 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);
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site
The First Affiliated Hospital of Zhejiang University
Hangzhou, Zhejiang, 310000
About this study
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.
Read the detailed description
Chimeric Antigen Receptor (CAR) T cells are genetically engineered T cells that can express the introduced CAR gene, which contains signaling molecules such as antigen recognition fragments, T cell receptor activation molecules, and co-stimulatory signals. Currently, in the field of hematological tumors, CD19-targeted CAR-T cells have been clinically proven to effectively treat B-cell malignancies. The US FDA has approved their use for treating relapsed or refractory CD19-positive B-cell malignancies, with favorable therapeutic effects. However, significant challenges remain before CAR-T therapy can be widely applied to solid tumor treatment. For example, the non-specific targeting of CAR-T cells to normal/non-malignant tissues ("on-target, off-tumor toxicity") can be fatal. In fact, off-tumor toxicity to the lungs, cerebral gray matter, and cardiac muscle has resulted in multiple deaths.
Prostate-specific membrane antigen (PSMA) is a surface antigen highly expressed in prostate cancer. Over 98% of lymph node metastases and almost all bone metastases in patients with castration-resistant prostate cancer (CRPC) highly express PSMA, making it an ideal target for prostate cancer treatment. We have developed a thermally activated and regulated FB-PSMA CAR-T cell targeting the prostate cancer antigen PSMA. In vitro, heating at 43°C can activate CAR expression without impairing cell function. After injection into the tumor site, PSMA-expressing prostate cancer cells can activate FB-PSMA CAR-T cells and, through a feedback mechanism, increase the expression level of CAR molecules, thereby enhancing the tumor-killing effect. Once tumor elimination is completed, CAR molecules will gradually degrade to provide a higher level of safety. In a mouse model of prostate cancer xenografts, FB-PSMA CAR-T cells have demonstrated significant anti-tumor effects and good safety.
Based on the above background, we plan to conduct this clinical trial, aiming to explore a new immunotherapeutic approach that can effectively control prostate cancer while maximizing the preservation of urogenital function and the control of oligometastases, thereby addressing the unmet needs in the current treatment of local prostate cancer. We hope that through this study, we can provide a safer and more effective treatment option for prostate cancer patients, while opening up new possibilities for future cancer treatment. This study is designed to evaluate the safety and efficacy of local injection of ACT#001 chimeric antigen receptor T cells (ACT#001-PSMA CAR-T) in the treatment of castration-resistant prostate cancer.
- Being tested
- Intraprostatic or localized lesion injection of ACT#001-PSMA CAR-T cells under transrectal ultrasound (TRUS) guidance
- Keywords
- CAR-T, CRPC
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Intraprostatic or localized lesion injection of ACT#001-PSMA CAR-T cells under transrectal ultrasound (TRUS) guidanceBiological
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: Biological: Intraprostatic or localized lesion injection of ACT#001-PSMA CAR-T cells under transrectal ultrasound (TRUS) guidance |
What is being measured
The primary outcome is the main question the study is designed to answer.
DLT
adverse events occurring within 28 days after the first infusion that are related to the study drug (definitely related, probably related, possibly related).
Measured: 28 days
Secondary outcomes (16)
PSA50 response rate
The proportion of participants with a reduction of ≥50% in PSA from baseline to the lowest post-baseline PSA result, which needs to be confirmed by a second consecutive PSA assessment after at least 3 weeks.
Measured: 6 months
DoPSA50（duration of PSA50 response）
The time from the date of first documented PSA50 response (subsequently confirmed) to the date of documented PSA progression per PCWG3 criteria or death from any cause in the absence of PSA progression.
Measured: 6 months
DRRPSA50（durable PSA50 response rates）
The percentage of participants with a confirmed PSA50 response lasting at least 6 months, using the number of participants in the PSA evaluable set as the denominator.
Measured: 6 months
TTPSAP50（time to PSA50 progression )
The time from the date of the first documented PSA50 response (subsequently confirmed) to the date of documented PSA progression per PCWG3 criteria.
Measured: 6 months
PSA90 response rate
The proportion of participants with a reduction of ≥90% in PSA from baseline to the lowest post-baseline PSA result, which needs to be confirmed by a second consecutive PSA assessment after at least 3 weeks.
Measured: 6 Months
DoPSA90（duration of PSA90 response）
The time from the date of first documented PSA90 response (subsequently confirmed) to the date of documented PSA progression per PCWG3 criteria or death from any cause in the absence of PSA progression.
Measured: 6 months
DRRPSA90（durable PSA90 response rates)
The percentage of participants with a confirmed PSA90 response lasting at least 6 months, using the number of participants in the PSA evaluable set as the denominator.
Measured: 6 months
TTPSAP90（time to PSA90 progression)
The time from the date of the first documented PSA90 response (subsequently confirmed) to the date of documented PSA progression per PCWG3 criteria.
Measured: 6 months
ORR
The proportion of patients achieving complete response (CR) or partial response (PR).
Measured: 6 MONTHS
DOR（Duration of Response）
The duration from the initial achievement of partial response (PR) or complete response (CR) to the occurrence of disease progression or death.
Measured: 6MONTHS
DCR（Disease Control Rate）
The proportion of patients achieving complete response (CR), partial response (PR), or stable disease (SD).
Measured: 6months
TTR（Time to Response）
The time from treatment initiation to the first achievement of partial response (PR) or complete response (CR).
Measured: 6mohths
OS（Overall Survival）
The time from treatment initiation to death from any cause.
Measured: 6 months
rPFS（Radiographic Progression-Free Survival）
The time from treatment initiation to radiologically documented disease progression or death.
Measured: 6 months
mFS
The time from treatment initiation to the development of distant metastasis or death.
Measured: 6 months
In vivo persistence time of CAR-T cells
The time from the infusion of ACT#001-PSMA CAR-T cells to the point when ACT#001-PSMA CAR-T cells can no longer be detected in the body (with 2 consecutive negative test results), which is detected by methods such as QPCR and flow cytometry.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Nov 2025
- Study start12 Apr 2026
- Primary completion31 Oct 2026 (estimated)
- Study completion31 Oct 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Castration-Resistant Prostate Cancer (CRPC)Studies of the same condition, open and under way.
- More trials run by Zhejiang UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 18 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Zhejiang University
- Sponsor type
- University, hospital or other
- Collaborators
- Orig Stone Biotech(Zhejiang) Co.,Ltd.
- Responsible party
- Principal investigator, Weijia Fang, MD, Zhejiang University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07240857
- Sponsor's protocol ID
- ACT#001
- Status last verified
- Aug 2025
- First submitted
- 23 Aug 2025
- Last updated
- 22 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT07240857
- Acronym / sponsor ID
- ACT#001
- Keywords
- CAR-T, CRPC
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Enrolling by invitation
- Enrolment
- 18 participants (estimated)
- Lead sponsor
- Zhejiang UniversityUniversity, hospital or other
- Collaborators
- Orig Stone Biotech(Zhejiang) Co.,Ltd.
- Study start
- 12 Apr 2026 (actual)
- Primary completion
- 31 Oct 2026 (estimated)
- Study completion
- 31 Oct 2027 (estimated)
- First posted
- 21 Nov 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Chimeric Antigen Receptor (CAR) T cells are genetically engineered T cells that can express the introduced CAR gene, which contains signaling molecules such as antigen recognition fragments, T cell receptor activation molecules, and co-stimulatory signals. Currently, in the field of hematological tumors, CD19-targeted CAR-T cells have been clinically proven to effectively treat B-cell malignancies. The US FDA has approved their use for treating relapsed or refractory CD19-positive B-cell malignancies, with favorable therapeutic effects. However, significant challenges remain before CAR-T therapy can be widely applied to solid tumor treatment. For example, the non-specific targeting of CAR-T cells to normal/non-malignant tissues ("on-target, off-tumor toxicity") can be fatal. In fact, off-tumor toxicity to the lungs, cerebral gray matter, and cardiac muscle has resulted in multiple deaths.
Prostate-specific membrane antigen (PSMA) is a surface antigen highly expressed in prostate cancer. Over 98% of lymph node metastases and almost all bone metastases in patients with castration-resistant prostate cancer (CRPC) highly express PSMA, making it an ideal target for prostate cancer treatment. We have developed a thermally activated and regulated FB-PSMA CAR-T cell targeting the prostate cancer antigen PSMA. In vitro, heating at 43°C can activate CAR expression without impairing cell function. After injection into the tumor site, PSMA-expressing prostate cancer cells can activate FB-PSMA CAR-T cells and, through a feedback mechanism, increase the expression level of CAR molecules, thereby enhancing the tumor-killing effect. Once tumor elimination is completed, CAR molecules will gradually degrade to provide a higher level of safety. In a mouse model of prostate cancer xenografts, FB-PSMA CAR-T cells have demonstrated significant anti-tumor effects and good safety.
Based on the above background, we plan to conduct this clinical trial, aiming to explore a new immunotherapeutic approach that can effectively control prostate cancer while maximizing the preservation of urogenital function and the control of oligometastases, thereby addressing the unmet needs in the current treatment of local prostate cancer. We hope that through this study, we can provide a safer and more effective treatment option for prostate cancer patients, while opening up new possibilities for future cancer treatment. This study is designed to evaluate the safety and efficacy of local injection of ACT#001 chimeric antigen receptor T cells (ACT#001-PSMA CAR-T) in the treatment of castration-resistant prostate cancer.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Rapid titration dose exploration phase | Experimental | Biological: Intraprostatic or localized lesion injection of ACT#001-PSMA CAR-T cells under transrectal ultrasound (TRUS) guidanceIt 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. |
| "3+3" dose escalation phase | Experimental | Biological: Intraprostatic or localized lesion injection of ACT#001-PSMA CAR-T cells under transrectal ultrasound (TRUS) guidanceDose 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- 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；
Key exclusion criteria (10 listed)
- 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);
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Intraprostatic or localized lesion injection of ACT#001-PSMA CAR-T cells under transrectal ultrasound (TRUS) guidancePrior 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. | Biological | "3+3" dose escalation phase, Rapid titration dose exploration phase |
Primary outcome
DLT
Time frame: 28 days
adverse events occurring within 28 days after the first infusion that are related to the study drug (definitely related, probably related, possibly related).
16 secondary outcomes
PSA50 response rate
Time frame: 6 months
DoPSA50（duration of PSA50 response）
Time frame: 6 months
DRRPSA50（durable PSA50 response rates）
Time frame: 6 months
TTPSAP50（time to PSA50 progression )
Time frame: 6 months
PSA90 response rate
Time frame: 6 Months
DoPSA90（duration of PSA90 response）
Time frame: 6 months
DRRPSA90（durable PSA90 response rates)
Time frame: 6 months
TTPSAP90（time to PSA90 progression)
Time frame: 6 months
ORR
Time frame: 6 MONTHS
DOR（Duration of Response）
Time frame: 6MONTHS
DCR（Disease Control Rate）
Time frame: 6months
TTR（Time to Response）
Time frame: 6mohths
OS（Overall Survival）
Time frame: 6 months
rPFS（Radiographic Progression-Free Survival）
Time frame: 6 months
mFS
Time frame: 6 months
In vivo persistence time of CAR-T cells
Time frame: 6 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 May 2026.
Related trials for Castration-Resistant Prostate Cancer (CRPC)
RecruitingPhase 2
Study of Oral MRT-2359 With Apalutamide in Prostate Cancer
Castration-Resistant Prostate Cancer (CRPC), Castration-Resistant Prostate Cancer
RecruitingPhase 3
Testing Different Dosing Schedules of the Anti-cancer Drug, Lutetium 177Lu PSMA RLT and Its Effect on Patients With Advanced Prostate Cancer, RECIPROCAL Trial
Metastatic Castration-Resistant Prostate Carcinoma, Metastatic Prostate Adenocarcinoma
RecruitingPhase 2
The COSMYC Trial (COmbined Suppression of MYC)
Metastatic Castration-resistant Prostate Cancer
RecruitingObservational study
Pluvicto Real-world Investigation in Survival in Metastatic CRPC
Prostatic Neoplasms, Castration-Resistant, Neoplasm Metastasis
RecruitingPhase 1
Pilot Study for Detection of PSMA-Low CRPC-NE Tumors With Fluciclovine PET/CT
Advanced Prostate Cancer, Metastatic Prostate Cancer
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Intraprostatic or localized lesion injection of ACT#001-PSMA CAR-T cells under transrectal ultrasound (TRUS) guidance in people with Castration-Resistant Prostate Cancer (CRPC), run by Zhejiang University at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 13 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: enrolling by invitation, as of the sponsor's last update on 22 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
DLT (time frame: 28 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 May 2026.