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Neoadjuvant Enfortumab Vedotin Plus Pembrolizumab in Muscle-Invasive Bladder Cancer and Upper Tract Urothelial Carcinoma

8 giugno 2026 aggiornato da: University of Florida

Radical cystectomy remains the standard curative-intent treatment for most patients with muscle-invasive bladder cancer, but it is associated with significant morbidity and long-term quality-of-life implications. Trimodality therapy is an accepted standard-of-care alternative for carefully selected patients who wish to preserve their bladder; however, optimal patient selection remains challenging.

The combination of enfortumab vedotin plus pembrolizumab (EV-P) has demonstrated remarkable activity in urothelial carcinoma, including in the perioperative setting, with pathologic complete response rates of approximately 50-60%. These results generate the hypothesis that a subset of patients may achieve sufficiently deep responses to allow selective deferral of cystectomy. Cohort A of this trial prospectively evaluates the use of multimodal response assessment (pelvic MRI and TURBT, ctDNA) to guide individualized decisions regarding cystectomy versus bladder preservation.

Radical nephroureterectomy (RNU) remains the standard curative-intent treatment for high-risk upper tract urothelial carcinoma (UTUC), but recurrence rates after surgery alone are high. Neoadjuvant cisplatin-based chemotherapy improves pathologic outcomes and is supported by phase II data, but its delivery is constrained by baseline renal dysfunction and the further decline in glomerular filtration that follows RNU - historically, only about 20% of patients remain cisplatin-eligible postoperatively, which is the principal rationale for delivering platinum in the neoadjuvant rather than adjuvant setting. A large fraction of patients with UTUC are cisplatin-ineligible at baseline, and no level 1 evidence supports a specific neoadjuvant regimen in this population.

EV-P is not constrained by renal function and has produced unprecedented activity in urothelial carcinoma. In the EV-302 upper tract subgroup, EV-P achieved an objective response rate of 67.7% and a complete response rate of 28.6%, with survival benefit preserved relative to platinum-based chemotherapy. In the perioperative bladder cancer setting, EV-P has yielded pathologic complete response rates of approximately 50-60%. However, available data on EV-P in UTUC are restricted to the metastatic setting, and prospective evaluation in the neoadjuvant setting is lacking. Cohort B of this trial addresses this gap by prospectively evaluating neoadjuvant EV-P followed by RNU in patients with high-risk UTUC, with pathologic complete response as the primary endpoint.

Panoramica dello studio

Stato

Non ancora reclutamento

Tipo di studio

Interventistico

Iscrizione (Stimato)

39

Fase

  • Fase 2

Criteri di partecipazione

I ricercatori cercano persone che corrispondano a una certa descrizione, chiamata criteri di ammissibilità. Alcuni esempi di questi criteri sono le condizioni generali di salute di una persona o trattamenti precedenti.

Criteri di ammissibilità

Età idonea allo studio

  • Adulto
  • Adulto più anziano

Accetta volontari sani

No

Descrizione

Inclusion Criteria:

  • Adults ≥ 18 years of age.
  • ECOG Performance Status of 0-2
  • For Cohort A, a clinical or pathological diagnosis consistent with muscle-invasive urothelial carcinoma of the bladder (locoregional), with a clinical staging of T2 or higher, N0 to N2, and M0.

For Cohort B, a clinical or pathological diagnosis of upper tract urothelial carcinoma, with high-intermediate risk or higher per NCCN guidelines, planned for definitive surgery.

  • High-intermediate: High grade + Unifocal <1.5 cm
  • High: High grade + Multifocal or >1.5 cm or high-grade renal obstruction or invasion on axial imaging Any proportion of divergent differentiation is permitted across all histologies (except neuroendocrine - see exclusion criteria), as long as a urothelial component is identified.

    • Eligible to receive enfortumab vedotin plus pembrolizumab (EV-P) in the opinion of the investigators.
    • Written informed consent obtained from the subject and the subject agrees to comply with all the study-related procedures.
    • Subjects must not have more than one active malignancy at the time of enrollment.

Note: Subjects with a prior or concurrent malignancy whose natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the investigational regimen or primary endpoint (as determined by the treating physician) are eligible for this trial.

- Subjects of childbearing potential (SOCBP) must be using an adequate method of contraception to avoid pregnancy throughout the study and for at least 4 months after the last dose of EV-P to minimize the risk of pregnancy. Prior to study enrollment, subjects of childbearing potential must be advised of the importance of avoiding pregnancy during trial participation and the potential risk factors for an unintentional pregnancy.

SOCBP includes any subject who has experienced menarche and who has not undergone successful surgical sterilization (hysterectomy, bilateral tubal ligation, or bilateral oophorectomy) or who is not post-menopausal. Post-menopause is defined as:

  • Amenorrhea that has lasted for ≥ 12 consecutive months without another cause, or
  • For subjects with irregular menstrual periods who are taking hormone replacement therapy (HRT), a documented serum follicle-stimulating hormone (FSH) level of greater than 35 mIU/mL.

    • Subjects with partners of child-bearing potential must agree to use physician-approved contraceptive methods (e.g., abstinence, condoms, vasectomy) throughout the study and should avoid conceiving children for 4 months following the last dose of EV-P.

Exclusion Criteria:

  • Histology that contains any component of neuroendocrine carcinoma.
  • Any known contraindications to enfortumab vedotin plus pembrolizumab.
  • Subjects who are confirmed to be pregnant or breastfeeding.
  • History of any other disease, metabolic dysfunction, clinical examination finding, or clinical laboratory finding giving reasonable suspicion of a disease or condition that contraindicates the use of protocol therapy or that might affect the interpretation of the results of the study or that puts the subject at high risk for treatment complications, in the opinion of the treating physician.
  • Administration of a vaccine containing live virus within 30 days prior to the first dose of trial treatment. Note: Most flu vaccines are killed viruses, with the exception of the intra-nasal vainer (Flu-Mist) which is an attenuated live virus and therefore prohibited for 30 days prior to first dose.
  • Prisoners or subjects who are involuntarily incarcerated, or subjects who are compulsorily detained for treatment of either a psychiatric or physical illness.

Piano di studio

Questa sezione fornisce i dettagli del piano di studio, compreso il modo in cui lo studio è progettato e ciò che lo studio sta misurando.

Come è strutturato lo studio?

Dettagli di progettazione

  • Scopo principale: Trattamento
  • Assegnazione: Non randomizzato
  • Modello interventistico: Assegnazione parallela
  • Mascheramento: Nessuno (etichetta aperta)

Armi e interventi

Gruppo di partecipanti / Arm
Intervento / Trattamento
Sperimentale: Cohort A (neoadjuvant EV-P + response-adapted management)

Following neoadjuvant EV-P, participants in this cohort will have response-adapted management based on if they achieved a composite clinical complete response (cCR) to determine the next step in their treatment on this study. A participant has achieved cCR if ALL the following are met:

  1. ctDNA is negative,
  2. Imaging are negative for lymph nodes and distant metastases, and
  3. TURBT shows T0 or Ta low-grade only, and a negative urinary cytology.

If a participant achieves cCR, they will have a choice between:

  • receive 5 more cycles of EV-P, followed by pembrolizumab alone to complete 1 year of therapy.
  • Alternatively, if participant wishes, partial or radical cystectomy.

If a participant does not achieve cCR, they will have cystectomy.

Participants in both cohorts will first receive neoadjuvant enfortumab vedotin + pembrolizumab (EV-P) for 4 cycles. Each cycle is 21 days. Participants will be given 1.25 mg/kg enfortumab vedotin intravenously on days 1 and 8 and 200 mg pembrolizumab intravenously on day 1 of each cycle. Participants in Cohort A may then choose to receive 5 more cycles of EV-P followed by pembrolizumab alone to complete 1 year of study therapy if they have a composite complete clinical response. Participants in Cohort B will then receive 5 more cycles of EV-P followed by pembrolizumab alone to complete 1 year of study therapy following their definitive surgery.
Sperimentale: Cohort B (neoadjuvant EV-P + surgery + adjuvant EV-P)
Participants in both cohorts will first receive neoadjuvant enfortumab vedotin + pembrolizumab (EV-P) for 4 cycles. Each cycle is 21 days. Participants will be given 1.25 mg/kg enfortumab vedotin intravenously on days 1 and 8 and 200 mg pembrolizumab intravenously on day 1 of each cycle. Participants in Cohort A may then choose to receive 5 more cycles of EV-P followed by pembrolizumab alone to complete 1 year of study therapy if they have a composite complete clinical response. Participants in Cohort B will then receive 5 more cycles of EV-P followed by pembrolizumab alone to complete 1 year of study therapy following their definitive surgery.

Cosa sta misurando lo studio?

Misure di risultato primarie

Misura del risultato
Misura Descrizione
Lasso di tempo
Clinical complete response rate
Lasso di tempo: 3 weeks following completion of neoadjuvant therapy

Determine the percent of subjects in Cohort A that achieve a clinical complete response following completion of neoadjuvant EV-P. A clinical complete response is defined as having all three of the following:

  • A negative ctDNA result,
  • Imaging of the pelvis with negative lymph nodes and no distant metastasis on CT scans, AND
  • A TURBT showing T0 or low-grade Ta only, and a negative urinary cytology.
3 weeks following completion of neoadjuvant therapy
Pathologic complete response rate
Lasso di tempo: 8 weeks following completion of neoadjuvant therapy
Determine the percent of subjects in Cohort B who achieve a pathologic complete response at the time of surgery. A pathologic complete response is defined as having disease staging of pT0N0 at time of surgery.
8 weeks following completion of neoadjuvant therapy

Misure di risultato secondarie

Misura del risultato
Misura Descrizione
Lasso di tempo
Event-free survival
Lasso di tempo: 1 year from the start of EV-P
Determine the event-free survival of patients in Cohort A at 1 year from the start of EV-P
1 year from the start of EV-P
Event-free survival
Lasso di tempo: 2 years from the start of EV-P
Determine the event-free survival of patients in Cohort A at 2 years from the start of EV-P
2 years from the start of EV-P
Overall survival
Lasso di tempo: 1 year from the start of EV-P
Determine the overall survival of patients in Cohort A at 1 year from the start of EV-P
1 year from the start of EV-P
Overall survival
Lasso di tempo: 2 years from the start of EV-P
Determine the overall survival of patients in Cohort A at 2 years from the start of EV-P
2 years from the start of EV-P
Bladder-intact survival
Lasso di tempo: 1 year from the start of EV-P
Determine the bladder-intact survival of patients in Cohort A at 1 year from the start of EV-P
1 year from the start of EV-P
Bladder-intact survival
Lasso di tempo: 2 years from the start of EV-P
Determine the bladder intact survival of patients in Cohort A at 2 years from the start of EV-P
2 years from the start of EV-P
Pathological complete response
Lasso di tempo: After completion of first 4 cycles of EV-P (each cycle of EV-P is 21 days)
Determine number of subjects in cohort A who achieve a pathological complete response after first 4 cycles of EV-P.
After completion of first 4 cycles of EV-P (each cycle of EV-P is 21 days)
Pathologic downstaging
Lasso di tempo: At completion of first 4 cycles of EV-P (each cycle of EV-P is 21 days)
Determine number of patients in cohort A who achieve pathological downstaging (disease staging <pT2N0) after the first 4 cycles of EV-P
At completion of first 4 cycles of EV-P (each cycle of EV-P is 21 days)
Event-free survival
Lasso di tempo: 2 years from the start of EV-P
Determine the event-free survival of patients in Cohort B at 2 years from the start of EV-P
2 years from the start of EV-P
Overall survival
Lasso di tempo: 2 years from the start of EV-P
Determine the overall survival of patients in Cohort B at 2 years from the start of EV-P
2 years from the start of EV-P
Pathologic downstaging
Lasso di tempo: At completion of first 4 cycles of EV-P (each cycle of EV-P is 21 days)
Determine number of patients in cohort B who achieve pathological downstaging (disease staging <pT2N0) after the first 4 cycles of EV-P
At completion of first 4 cycles of EV-P (each cycle of EV-P is 21 days)

Collaboratori e investigatori

Qui è dove troverai le persone e le organizzazioni coinvolte in questo studio.

Investigatori

  • Investigatore principale: Daniel Araujo, MD, University of Florida

Studiare le date dei record

Queste date tengono traccia dell'avanzamento della registrazione dello studio e dell'invio dei risultati di sintesi a ClinicalTrials.gov. I record degli studi e i risultati riportati vengono esaminati dalla National Library of Medicine (NLM) per assicurarsi che soddisfino specifici standard di controllo della qualità prima di essere pubblicati sul sito Web pubblico.

Studia le date principali

Inizio studio (Stimato)

1 ottobre 2026

Completamento primario (Stimato)

1 febbraio 2029

Completamento dello studio (Stimato)

1 ottobre 2030

Date di iscrizione allo studio

Primo inviato

3 giugno 2026

Primo inviato che soddisfa i criteri di controllo qualità

8 giugno 2026

Primo Inserito (Effettivo)

11 giugno 2026

Aggiornamenti dei record di studio

Ultimo aggiornamento pubblicato (Effettivo)

11 giugno 2026

Ultimo aggiornamento inviato che soddisfa i criteri QC

8 giugno 2026

Ultimo verificato

1 giugno 2026

Maggiori informazioni

Termini relativi a questo studio

Informazioni su farmaci e dispositivi, documenti di studio

Studia un prodotto farmaceutico regolamentato dalla FDA degli Stati Uniti

Studia un dispositivo regolamentato dalla FDA degli Stati Uniti

No

prodotto fabbricato ed esportato dagli Stati Uniti

No

Queste informazioni sono state recuperate direttamente dal sito web clinicaltrials.gov senza alcuna modifica. In caso di richieste di modifica, rimozione o aggiornamento dei dettagli dello studio, contattare register@clinicaltrials.gov. Non appena verrà implementata una modifica su clinicaltrials.gov, questa verrà aggiornata automaticamente anche sul nostro sito web .

Prove cliniche su Carcinoma uroteliale (UC)

Prove cliniche su enfortumab vedotin and pembrolizumab (EV/P)

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