Bladder CancerPhase 2RETAIN-3NCT07908979
Risk-Enabled Therapy After Initiating EV/Pembro for Bladder Cancer
A Phase 2 study testing Enfortumab vedotin, Pembrolizumab and Cisplatin in people with Bladder Cancer and Urothelial Carcinoma, run by Fox Chase Cancer Center at 1 site in United States.
Trial summary
RETAIN-3 is a phase II study in patients with cT2-T3N0M0 muscle-invasive urothelial carcinoma of the bladder. Participants will receive neoadjuvant enfortumab vedotin and pembrolizumab followed by clinical and biomarker-based assessment to inform subsequent treatment management. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Bladder Cancer, Urothelial Carcinoma, Muscle-Invasive Bladder Carcinoma
- What is being tested?
- Enfortumab vedotin, Pembrolizumab and Cisplatin
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 70 participants
- Where
- 1 siteUnited States
- Study dates
- 16 Oct 2026to 1 Nov 2031
- Phase
- Phase 2
- 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
- Primary urothelial or predominantly urothelial carcinoma of the bladder confirmed from pathology report. Patients with some component of variant histology mixed with predominant urothelial carcinoma will be allowed. Upper tract urothelial carcinoma patients are not allowed.
- Urothelial carcinoma of the prostatic urethra in men is allowed
- Histologic evidence of muscularis propria invasion.
- AJCC(22) clinical stage T2-T3 N0M0.
- No radiographic evidence of lymph node positive disease as per RECIST 1.1 (≥15 mm short axis diameter). If a lymph node is greater than 1.5 cm, it must be biopsy proven negative for the patient to be eligible.
- No metastatic disease (M0).
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- ECOG performance status 0, or 1.
- Negative pregnancy test in women of child bearing potential within 72 hours of the first dose of study treatment.
- Normal organ and bone marrow function
Who cannot join
- Any component of small cell histology.
- Prior systemic chemotherapy for muscle-invasive urothelial cancer of the bladder.
- Uncontrolled diabetes (HbA1c >8.5%)
- Pre-existing sensory or motor neuropathy grade > 2
- Has a known additional malignancy that has had progression or has required active treatments in the last two years except for locally curable cancers that have been apparently cured. Exceptions include basal cell carcinoma of the skin, squamous cell carcinoma of the skin that has undergone potentially curative therapy or in situ cervical cancer. Patients with intermediate or low risk prostate cancer as defined by the National Comprehensive Cancer Network (NCCN) risk stratification guidelines may be eligible for enrollment.
- Patients who have received experimental agents within 4 weeks of study entry.
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- Patients who have received enfortumab vedotin based treatment in the past.
- Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection (defined by current oral or intravenous antibiotic therapy), symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
- Pregnant women are excluded from this study due to the potential for teratogenic or abortifacient effects of cytotoxic therapy.
- HIV-infected patients on effective anti-retroviral therapy with an undetectable viral load are eligible
- Any condition requiring systemic treatment with corticosteroids (> 10 mg daily prednisone or equivalent) or other immunosuppressive medications within 7 days prior to first dose of study drug. Inhaled or topical steroids and adrenal replacement steroid doses < 10 mg daily prednisone or equivalent are permitted in the absence of active autoimmune disease. Use of steroids as pre-medication for contrast allergy prior to PET scans or CT scans is permitted.
- History of autoimmune disease including but not limited to myasthenia gravis, myositis, autoimmune hepatitis, systemic lupus erythematosus, rheumatoid arthritis, inflammatory bowel disease, vascular thrombosis associated with antiphospholipid syndrome, Wegener's granulomatosis, Sjögren's syndrome, Guillain-Barré syndrome, multiple sclerosis, vasculitis, or glomerulonephritis. Any history of autoimmune disease not explicitly listed above should be evaluated by the Principal Investigator and may result in exclusion based on clinical judgment.
- Prior treatment with CD137 agonists, anti-programmed death-1 (PD-1), or anti-PD-L1 therapeutic antibody or pathway-targeting agents. Has had an allogenic tissue/solid organ transplant.
- Treatment with systemic immunostimulatory agents (including but not limited to interferons or interleukin [IL]-2) within 4 weeks or five half-lives of the drug, whichever is shorter, prior to enrolment.
- Allergy to EV, pembrolizumab or any excipient in the products
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Fox Chase Cancer Center - Philadelphia
Philadelphia, Pennsylvania, 19111-2497
Pooja Ghatalia, MD, Pooja.Ghatalia@tuhs.temple.edu
About this study
Official title: GU-254: A Phase II Trial of Risk-Enabled Therapy After Initiating Neoadjuvant Enfortumab Vedotin/Pembrolizumab for Bladder Cancer (RETAIN-3)
RETAIN-3 is a phase II study in patients with cT2-T3N0M0 muscle-invasive urothelial carcinoma of the bladder. Participants will receive neoadjuvant enfortumab vedotin and pembrolizumab followed by clinical and biomarker-based assessment to inform subsequent treatment management.
Read the detailed description
RETAIN-3 is a multicenter, open-label, phase II study evaluating a biomarker- and response-adapted bladder preservation strategy in patients with cT2-T3N0M0 muscle-invasive bladder cancer treated with neoadjuvant enfortumab vedotin and pembrolizumab. Following treatment, participants undergo clinical restaging and biomarker assessment to guide subsequent management, which may include active surveillance, maintenance therapy, cystectomy, or chemoradiation. The study aims to determine whether a biomarker-guided approach can preserve the bladder in selected patients while maintaining favorable oncologic outcomes.
- Conditions
- Bladder Cancer, Urothelial Carcinoma, Muscle-Invasive Bladder Carcinoma, Muscle-invasive Bladder Urothelial Carcinoma
- Being tested
- Enfortumab vedotin, Pembrolizumab, Cisplatin, Gemcitabine, Fluorouracil
- Keywords
- Enfortumab vedotin, Pembrolizumab, Muscle-Invasive Bladder Cancer, MIBC, Bladder Cancer, Urothelial Carcinoma, ctDNA, Circulating Tumor DNA, Bladder Preservation
- Medical subject headings
- Urinary Bladder Neoplasms, Carcinoma, Transitional Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Enfortumab vedotinDrug
1.25 mg/kg (maximum 125 mg) intravenously on Days 1 and 8 of each 21-day cycle for 3 cycles.
PembrolizumabDrug
200 mg intravenously on Day 1 of each 21-day cycle or subcutaneous pembrolizumab/berahyaluronidase formulation administered per protocol.
CisplatinDrug
Cisplatin administered as a radiosensitizing chemotherapy regimen in participants receiving chemoradiation.
GemcitabineDrug
Gemcitabine administered as a radiosensitizing chemotherapy regimen in participants receiving chemoradiation.
FluorouracilDrug
Fluorouracil administered as a radiosensitizing chemotherapy regimen in participants receiving chemoradiation.
Mitomycin cDrug
Mitomycin C administered in combination with fluorouracil as a radiosensitizing chemotherapy regimen in participants receiving chemoradiation.
Radical cystectomyProcedure or surgery
Surgical removal of the bladder performed according to institutional standards.
Radiation therapyProcedure or surgery
Definitive bladder-preserving treatment consisting of external beam radiation therapy administered with concurrent radiosensitizing chemotherapy according to institutional standards and protocol specifications.
| Study group | Role | What they receive |
|---|---|---|
| Arm A: Neoadjuvant Enfortumab Vedotin Plus Pembrolizumab With Restaging | Experimental | Participants receive neoadjuvant enfortumab vedotin plus pembrolizumab for 3 cycles followed by protocol-specified clinical restaging and biomarker assessment. All enrolled participants begin study treatment in this arm. Receives: Drug: Enfortumab vedotin; Drug: Pembrolizumab |
| Arm B: Additional Enfortumab Vedotin Plus Pembrolizumab for ctDNA-Positive Participants | Experimental | Participants with positive post-treatment circulating tumor DNA (ctDNA) and favorable clinical restaging findings may receive an additional 3 cycles of enfortumab vedotin plus pembrolizumab followed by repeat assessment. Receives: Drug: Enfortumab vedotin; Drug: Pembrolizumab |
| Arm C: Maintenance Pembrolizumab With Active Surveillance | Experimental | Participants meeting protocol-defined criteria may undergo active surveillance while receiving maintenance pembrolizumab and scheduled disease evaluations. Receives: Drug: Pembrolizumab |
| Arm D: Definitive Chemoradiation | Experimental | Participants requiring definitive bladder-directed treatment may receive chemoradiation with radiation therapy and investigator-selected radiosensitizing chemotherapy according to protocol specifications. Receives: Drug: Cisplatin; Drug: Gemcitabine; Drug: Fluorouracil; Drug: Mitomycin c; Procedure: Radiation therapy |
| Arm E: Radical Cystectomy | Experimental | Participants requiring definitive surgical management may undergo radical cystectomy. Receives: Procedure: Radical cystectomy |
What is being measured
The primary outcome is the main question the study is designed to answer.
2-Year Metastasis-Free Survival (MFS)
Metastasis-free survival is defined as the time from initiation of neoadjuvant therapy to metastatic urothelial carcinoma or death from any cause, whichever occurs first.
Measured: 2 years
Secondary outcomes (8)
Bladder-intact metastasis-free survival in active surveillance patients
Measured: 2 years
Pathologic complete response (pT0) rate among participants undergoing cystectomy
Measured: At cystectomy
Clinical complete response rate
Measured: Up to 5 years
Rate of urothelial carcinoma recurrence in active surveillance participants
Measured: Up to 5 years
Overall survival (OS)
Measured: Up to 5 years
Rate of distant recurrence among post-treatment ctDNA-positive participants undergoing definitive local therapy
Measured: Up to 5 years
Proportion of participants undergoing cystectomy
Measured: Up to 5 years
Bladder-intact metastasis-free survival in all evaluable participants
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Nov 2026
- Study start16 Oct 2026
- Primary completion1 Oct 2029 (estimated)
- Study completion1 Nov 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Bladder CancerStudies of the same condition, open and under way.
- More trials run by Fox Chase Cancer CenterThe 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
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Crossover
- Model description
- All participants receive neoadjuvant enfortumab vedotin and pembrolizumab followed by response- and biomarker-guided treatment management.
- Masking
- None (open label)
- Enrolment
- 70 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Fox Chase Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- Natera, Inc.
- Responsible party
- Sponsor
- Investigators
- Pooja Ghatalia, MD, Principal investigator, Fox Chase Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07908979
- Sponsor's protocol ID
- 26-1030
- Other IDs
- GU-254
- Acronym
- RETAIN-3
- Status last verified
- Oct 2026
- First submitted
- 19 Oct 2026
- Last updated
- 2 Nov 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- GU-254: A Phase II Trial of Risk-Enabled Therapy After Initiating Neoadjuvant Enfortumab Vedotin/Pembrolizumab for Bladder Cancer (RETAIN-3)
- ClinicalTrials.gov ID
- NCT07908979
- Acronym / sponsor ID
- RETAIN-3 / 26-1030
- Conditions
- Bladder Cancer, Urothelial Carcinoma, Muscle-Invasive Bladder Carcinoma, Muscle-invasive Bladder Urothelial Carcinoma
- Keywords
- Enfortumab vedotin, Pembrolizumab, Muscle-Invasive Bladder Cancer, MIBC, Bladder Cancer, Urothelial Carcinoma, ctDNA, Circulating Tumor DNA
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 70 participants (estimated)
- Lead sponsor
- Fox Chase Cancer CenterUniversity, hospital or other
- Collaborators
- Natera, Inc.
- Study start
- 16 Oct 2026 (actual)
- Primary completion
- 1 Oct 2029 (estimated)
- Study completion
- 1 Nov 2031 (estimated)
- First posted
- 2 Nov 2026 (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.
RETAIN-3 is a phase II study in patients with cT2-T3N0M0 muscle-invasive urothelial carcinoma of the bladder. Participants will receive neoadjuvant enfortumab vedotin and pembrolizumab followed by clinical and biomarker-based assessment to inform subsequent treatment management.
Detailed description
RETAIN-3 is a multicenter, open-label, phase II study evaluating a biomarker- and response-adapted bladder preservation strategy in patients with cT2-T3N0M0 muscle-invasive bladder cancer treated with neoadjuvant enfortumab vedotin and pembrolizumab. Following treatment, participants undergo clinical restaging and biomarker assessment to guide subsequent management, which may include active surveillance, maintenance therapy, cystectomy, or chemoradiation. The study aims to determine whether a biomarker-guided approach can preserve the bladder in selected patients while maintaining favorable oncologic outcomes.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 5
- Model description
- All participants receive neoadjuvant enfortumab vedotin and pembrolizumab followed by response- and biomarker-guided treatment management.
| Arm | Type | Receives |
|---|---|---|
| Arm A: Neoadjuvant Enfortumab Vedotin Plus Pembrolizumab With Restaging | Experimental | Drug: Enfortumab vedotin, Drug: PembrolizumabParticipants receive neoadjuvant enfortumab vedotin plus pembrolizumab for 3 cycles followed by protocol-specified clinical restaging and biomarker assessment. All enrolled participants begin study treatment in this arm. |
| Arm B: Additional Enfortumab Vedotin Plus Pembrolizumab for ctDNA-Positive Participants | Experimental | Drug: Enfortumab vedotin, Drug: PembrolizumabParticipants with positive post-treatment circulating tumor DNA (ctDNA) and favorable clinical restaging findings may receive an additional 3 cycles of enfortumab vedotin plus pembrolizumab followed by repeat assessment. |
| Arm C: Maintenance Pembrolizumab With Active Surveillance | Experimental | Drug: PembrolizumabParticipants meeting protocol-defined criteria may undergo active surveillance while receiving maintenance pembrolizumab and scheduled disease evaluations. |
| Arm D: Definitive Chemoradiation | Experimental | Drug: Cisplatin, Drug: Gemcitabine, Drug: Fluorouracil, Drug: Mitomycin c, Procedure: Radiation therapyParticipants requiring definitive bladder-directed treatment may receive chemoradiation with radiation therapy and investigator-selected radiosensitizing chemotherapy according to protocol specifications. |
| Arm E: Radical Cystectomy | Experimental | Procedure: Radical cystectomyParticipants requiring definitive surgical management may undergo radical cystectomy. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Primary urothelial or predominantly urothelial carcinoma of the bladder confirmed from pathology report. Patients with some component of variant histology mixed with predominant urothelial carcinoma will be allowed. Upper tract urothelial carcinoma patients are not allowed.
- Urothelial carcinoma of the prostatic urethra in men is allowed
- Histologic evidence of muscularis propria invasion.
- AJCC(22) clinical stage T2-T3 N0M0.
- No radiographic evidence of lymph node positive disease as per RECIST 1.1 (≥15 mm short axis diameter). If a lymph node is greater than 1.5 cm, it must be biopsy proven negative for the patient to be eligible.
Key exclusion criteria (15 listed)
- Any component of small cell histology.
- Prior systemic chemotherapy for muscle-invasive urothelial cancer of the bladder.
- Uncontrolled diabetes (HbA1c >8.5%)
- Pre-existing sensory or motor neuropathy grade > 2
- Has a known additional malignancy that has had progression or has required active treatments in the last two years except for locally curable cancers that have been apparently cured. Exceptions include basal cell carcinoma of the skin, squamous cell carcinoma of the skin that has undergone potentially curative therapy or in situ cervical cancer. Patients with intermediate or low risk prostate cancer as defined by the National Comprehensive Cancer Network (NCCN) risk stratification guidelines may be eligible for enrollment.
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 |
|---|---|---|
| Enfortumab vedotin1.25 mg/kg (maximum 125 mg) intravenously on Days 1 and 8 of each 21-day cycle for 3 cycles. | Drug | Arm A: Neoadjuvant Enfortumab Vedotin Plus Pembrolizumab With Restaging, Arm B: Additional Enfortumab Vedotin Plus Pembrolizumab for ctDNA-Positive Participants |
| Pembrolizumab200 mg intravenously on Day 1 of each 21-day cycle or subcutaneous pembrolizumab/berahyaluronidase formulation administered per protocol. | Drug | Arm A: Neoadjuvant Enfortumab Vedotin Plus Pembrolizumab With Restaging, Arm B: Additional Enfortumab Vedotin Plus Pembrolizumab for ctDNA-Positive Participants, Arm C: Maintenance Pembrolizumab With Active Surveillance |
| CisplatinCisplatin administered as a radiosensitizing chemotherapy regimen in participants receiving chemoradiation. | Drug | Arm D: Definitive Chemoradiation |
| GemcitabineGemcitabine administered as a radiosensitizing chemotherapy regimen in participants receiving chemoradiation. | Drug | Arm D: Definitive Chemoradiation |
| FluorouracilFluorouracil administered as a radiosensitizing chemotherapy regimen in participants receiving chemoradiation. | Drug | Arm D: Definitive Chemoradiation |
| Mitomycin cMitomycin C administered in combination with fluorouracil as a radiosensitizing chemotherapy regimen in participants receiving chemoradiation. | Drug | Arm D: Definitive Chemoradiation |
| Radical cystectomySurgical removal of the bladder performed according to institutional standards. | Procedure or surgery | Arm E: Radical Cystectomy |
| Radiation therapyDefinitive bladder-preserving treatment consisting of external beam radiation therapy administered with concurrent radiosensitizing chemotherapy according to institutional standards and protocol specifications. | Procedure or surgery | Arm D: Definitive Chemoradiation |
Primary outcome
2-Year Metastasis-Free Survival (MFS)
Time frame: 2 years
Metastasis-free survival is defined as the time from initiation of neoadjuvant therapy to metastatic urothelial carcinoma or death from any cause, whichever occurs first.
8 secondary outcomes
Bladder-intact metastasis-free survival in active surveillance patients
Time frame: 2 years
Pathologic complete response (pT0) rate among participants undergoing cystectomy
Time frame: At cystectomy
Clinical complete response rate
Time frame: Up to 5 years
Rate of urothelial carcinoma recurrence in active surveillance participants
Time frame: Up to 5 years
Overall survival (OS)
Time frame: Up to 5 years
Rate of distant recurrence among post-treatment ctDNA-positive participants undergoing definitive local therapy
Time frame: Up to 5 years
Proportion of participants undergoing cystectomy
Time frame: Up to 5 years
Bladder-intact metastasis-free survival in all evaluable participants
Time frame: 2 years
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 2 Nov 2026.
- Individual participant dataSharing planned: No
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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.
Pooja Ghatalia, MD principal investigator
Fox Chase Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Enfortumab vedotin, Pembrolizumab and Cisplatin in people with Bladder Cancer and Urothelial Carcinoma, run by Fox Chase Cancer Center at 1 site in United States.
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 9 inclusion and 15 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: looking for participants now, as of the sponsor's last update on 2 Nov 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 United States. 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?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
Contact Investigator-Sponsored Research Unit ISRU (2678388380, protocoldev@fccc.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
2-Year Metastasis-Free Survival (MFS) (time frame: 2 years). 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 2 Nov 2026.