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Sacituzumab Tirumotecan vs MMAE-ADCs in Advanced Urothelial Carcinoma (FUSCC-SPARE-UC-01) (SPARE-UC-01)

2026년 6월 17일 업데이트: Ding-Wei Ye, Fudan University

A Randomized, Open-label, Phase II Study Evaluating the Neurotoxicity and Efficacy of Sacituzumab Tirumotecan (Sac-TMT) Versus MMAE-based ADCs in Patients With Advanced Urothelial Carcinoma: The SPARE-UC-01 Trial

The main goal of this clinical trial is to learn if a new targeted cancer drug called sacituzumab tirumotecan (sac-TMT) works to treat cancer while causing less nerve damage in patients with advanced urothelial carcinoma who have progressed on or could not tolerate previous treatment such as enfortumab vedotin plus pembrolizumab (EVP) or disitamab vedotin plus toripalimab (DVT).

The main question it aims to answer is: Does sac-TMT lower the risk of getting severe nerve damage, as measured together by doctors, machines, and the participants?

Researchers will compare sac-TMT to alternative MMAE-based ADC drugs (switching to a different MMAE-based ADC after the first one stopped working) to see if sac-TMT causes less nerve damage while still effectively treating the cancer. A small group of participants who had to stop their previous MMAE-based ADC treatment because of nerve damage will also receive sac-TMT to learn if the drug is safe for their nerves.

Participants will:

  1. Receive either sac-TMT or another MMAE-based ADC drug
  2. Have regular physical exams by a doctor to check their nerves
  3. Have machine tests to measure how well their nerves work
  4. Answer survey questions about their pain, numbness, and daily activities

연구 개요

상세 설명

Metastatic urothelial carcinoma (mUC) is associated with a poor prognosis, and traditional platinum-based chemotherapy offers limited clinical benefit with significant toxicity. In recent years, the emergence of antibody-drug conjugates (ADCs) has transformed the treatment landscape of mUC. Nectin-4-directed enfortumab vedotin (EV) and HER2-directed disitamab vedotin (DV) have demonstrated breakthrough survival benefits in multiple pivotal clinical studies. EV plus pembrolizumab (EVP) and DV plus toripalimab (DVT) have been approved and are increasingly adopted as first-line treatments for mUC, with growing utilization in Chinese clinical practice.

However, as patient survival extends, the long-term safety profile of ADCs has become a major clinical challenge. Both EV and DV utilize MMAE as their cytotoxic payload, a potent microtubule- targeting agent. Non-specific uptake of these ADCs may lead to the release of MMAE within peripheral nerves, potentially causing microtubule dysregulation. By interfering with microtubule dynamics and inhibiting microtubule-dependent axonal transport, MMAE is thought to contribute to the development of peripheral neuropathy. Clinical data indicate that EV and DV cause a high incidence of peripheral neuropathy. Numbness, pain, and motor dysfunction significantly impair patients' activities of daily living (ADLs). Given the absence of effective prophylactic or therapeutic agents for peripheral neuropathy, clinical management is currently limited to dose reductions or complete treatment discontinuation, which will inevitably diminish anti-tumor efficacy.

Sacituzumab tirumotecan (sac-TMT) is a novel TROP2-directed ADC delivering a topoisomerase I (Topo-I) inhibitor payload. This payload induces DNA double-strand breaks within the cell nucleus, completely avoiding interference with the cytoskeletal microtubule system. This mechanistic difference fundamentally eliminates the biological basis for axonal transport blockade. Preliminary data from the Phase 1/2 MK-2870-001/KL264-01 study showed that sac-TMT achieved an overall objective response rate (ORR) of 31% and a median overall survival (OS) of 12.1 months in previously treated mUC patients, demonstrating a favorable efficacy profile. Notably, no typical drug-related peripheral neurotoxicity events were observed, highlighting a neuroprotective advantage.

The SPARE-UC-01 study is a head-to-head, Phase II clinical trial to compare the neurotoxicity and anti-tumor efficacy of sac-TMT versus sequential MMAE-based ADC therapy. The study targets a population that has progressed on or is intolerant to prior EVP or DVT. By comparing sac-TMT to the sequential use of alternative MMAE-based ADCs, the research aims to provide an evidence-based sequential treatment strategy. The study utilizes an integrated evaluation system designed to quantitatively capture the spectrum of neurotoxicity. This includes investigator-assessed NCI-CTCAE v5.0 scoring of Peripheral Neuropathy Progression Rate (PNPR), instrument-based nerve conduction studies (NCS), and patient- reported quality-of-life outcomes (PROs). Such a three-dimensional paradigm ensures that both structural nerve damage and functional disability are accurately documented. In view of the substantial population of patients who undergo discontinuation of MMAE-ADCs due to cumulative neurotoxicity, an observational cohort has been established within this study. This cohort aims to characterize the clinical utility of sac-TMT in these patients. Given that its payload does not target the microtubule system, sac-TMT is expected to provide effective treatment without introducing additional neurotoxicity.

연구 유형

중재적

등록 (추정된)

75

단계

  • 2 단계

연락처 및 위치

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연구 연락처

연구 연락처 백업

연구 장소

    • Shanghai Municipality
      • Shanghai, Shanghai Municipality, 중국, 200032
        • Fudan University Shanghai Cancer Center
        • 수석 연구원:
          • Yao Zhu
        • 연락하다:

참여기준

연구원은 적격성 기준이라는 특정 설명에 맞는 사람을 찾습니다. 이러한 기준의 몇 가지 예는 개인의 일반적인 건강 상태 또는 이전 치료입니다.

자격 기준

공부할 수 있는 나이

  • 성인
  • 고령자

건강한 자원 봉사자를 받아들입니다

아니

설명

Inclusion Criteria

  1. Must voluntarily sign the written Institutional Review Board (IRB)/Ethics Committee (EC) approved informed consent form (ICF) prior to any screening procedures.
  2. Age > 18 years at the time of signing the ICF.
  3. Histologically or cytologically confirmed locally advanced (unresectable) or metastatic urothelial carcinoma (UC), including bladder, ureter, renal pelvis, or urethra. Participants with mixed histology are eligible provided that UC is the predominant component (> 50%).
  4. Must have received at least one prior line of systemic therapy for locally advanced or metastatic UC (e.g., Enfortumab Vedotin plus Pembrolizumab, Disitamab Vedotin plus Toripalimab, platinum-based chemotherapy, immune checkpoint inhibitors, Nectin-4 ADCs, HER2 ADCs, FGFR inhibitors, or other palliative chemotherapy regimens).
  5. Neuropathy Status:

    Cohort A/B: Baseline peripheral neuropathy (PN) Grade 0-1 (per NCI-CTCAE v5.0) with stable nerve function confirmed by Nerve Conduction Study (NCS) during screening.

    Cohort C (Observational): Baseline PN Grade 2, or a history of PN > Grade 2 where the investigator deems the patient unsuitable for MMAE-based ADC treatment.

  6. At least one measurable lesion per RECIST v1.1. (Lesions in previously irradiated areas are considered target lesions only if clear progression is documented after radiotherapy).
  7. ECOG Performance Status of 0 or 1 at screening.
  8. Expected survival > 3 months.
  9. Must have adequate organ and bone marrow function (no blood transfusion, growth factors, or albumin support within 14 days prior to screening):

    • Hematological: ANC >= 1.5 x 10^9/L; Platelets >= 75 x 10^9/L; Hemoglobin >= 90 g/L.
    • Hepatic: ALT and AST <= 2.5 x ULN (or <= 5 x ULN for patients with liver metastases); Total Bilirubin <= 1.5 x ULN (if Total Bilirubin > 1.5 x ULN, Direct Bilirubin must be <= ULN).
    • Coagulation: INR <= 1.5; APTT <= 1.5 x ULN; PT < ULN + 4 seconds.
    • Renal: Creatinine Clearance (CrCl) >= 30 mL/min, or Serum Creatinine <= 1.5 x ULN.

Exclusion Criteria

  1. Prior treatment with TROP2-targeted ADCs, topoisomerase I inhibitors (e.g., irinotecan, topotecan), or ADCs containing topoisomerase I inhibitor payloads.
  2. Patients previously treated with both Enfortumab Vedotin (EV) and Disitamab Vedotin (DV) are excluded from Cohorts A and B (eligible for Cohort C only).
  3. Treatment with any investigational anti-tumor agents, chemotherapy, immunotherapy, monoclonal antibodies, targeted therapy, or radical radiotherapy within 2 weeks or 5 half-lives (whichever is shorter) prior to the first dose. Major surgery within 4 weeks prior to the first dose.
  4. Active CNS or meningeal metastases. Patients with previously treated CNS metastases are eligible if clinically stable for ≥ 4 weeks, off systemic corticosteroids for ≥ 2 weeks (physiological replacement ≤ 10 mg/day prednisone equivalent is allowed), and no evidence of radiographic progression.
  5. History of non-infectious pneumonitis/interstitial lung disease (ILD) requiring steroids. Current ILD or suspected ILD on screening chest CT (even if asymptomatic). Severe COPD, severely impaired lung function, or requirement for long-term oxygen therapy.
  6. QTcF interval > 470 ms (females) or > 450 ms (males). Within 6 months prior to the first dose: myocardial infarction, unstable angina, severe arrhythmia requiring intervention, uncontrolled hypertension, stroke, or TIA. NYHA Class III or IV congestive heart failure.
  7. Active keratitis, corneal ulcer, or severe dry eye syndrome.
  8. Active Hepatitis B (HBsAg positive and HBV-DNA > 2000 IU/mL; patients with lower HBV-DNA must receive antiviral therapy); Active Hepatitis C (HCV antibody and RNA positive); Known HIV infection; Severe infection requiring IV antibiotics within 2 weeks prior to first dose.
  9. Hypersensitivity: Known severe hypersensitivity to sac-TMT, EV, DV, or their excipients.
  10. Any severe or uncontrolled systemic disease that, in the investigator's opinion, increases the risk to the participant.
  11. HbA1c ≥ 8% (Patients with well-controlled blood glucose, fasting glucose ≤ 10 mmol/L, and investigator approval are eligible).
  12. History of allogeneic stem cell transplant or solid organ transplant.
  13. Pregnant or breastfeeding females.

공부 계획

이 섹션에서는 연구 설계 방법과 연구가 측정하는 내용을 포함하여 연구 계획에 대한 세부 정보를 제공합니다.

연구는 어떻게 설계됩니까?

디자인 세부사항

  • 주 목적: 치료
  • 할당: 무작위
  • 중재 모델: 병렬 할당
  • 마스킹: 없음(오픈 라벨)

무기와 개입

참가자 그룹 / 팔
개입 / 치료
실험적: Experimental
Patients receive sac-TMT (a Topo-I inhibitor-payload ADC) following failure of prior MMAE-based ADC therapy.
4.0 mg/kg intravenously administered on Day 1 every 2 weeks.
다른 이름들:
  • SKB264
  • MK-2870
  • 주머니-TMT
활성 비교기: Active Comparator
Patients receive an alternative MMAE-based ADC regimen as sequential therapy following progression on or intolerance to prior MMAE-based ADC treatment.

EV (Enfortumab Vedotin): 1.25 mg/kg administered intravenously on Days 1, 8, and 15 of every 4 weeks.

DV (Disitamab Vedotin): 2.0 mg/kg administered intravenously on Day 1 of every 2 weeks.

다른 이름들:
  • DV
  • EV
다른: Observational
An observational cohort for patients who discontinued prior MMAE-ADCs due to neurotoxicity.
4.0 mg/kg intravenously administered on Day 1 every 2 weeks.
다른 이름들:
  • SKB264
  • MK-2870
  • 주머니-TMT

연구는 무엇을 측정합니까?

주요 결과 측정

결과 측정
측정값 설명
기간
Peripheral Neuropathy Progression Rate (PNPR)
기간: From randomization up to the end of treatment (approximately 24 months).

The PNPR is defined as the proportion of participants who experience their first occurrence of treatment-emergent peripheral neurotoxicity of Grade 2 or higher from the time of randomization until the end of treatment.

Neurotoxicity severity is graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 5.0, a 5-point ordinal scale ranging from Grade 1 (mild; asymptomatic or mild symptoms, clinical or diagnostic observations only) to Grade 5 (death related to adverse event). Higher grades indicate worse outcomes (i.e., more severe neurotoxicity).

The difference in PNPR between treatment arms will be analyzed using the Cochran-Mantel-Haenszel (CMH) test, adjusted for stratification factors specified in the protocol.

From randomization up to the end of treatment (approximately 24 months).

2차 결과 측정

결과 측정
측정값 설명
기간
Percent Change in NCS Parameters
기간: Baseline, and at protocol-specified time points (including the time of first occurrence of Grade ≥2 neurotoxicity symptoms) during the treatment period, up to approximately 24 months.
The NCS percentage change is calculated as the relative change in electrophysiological parameters from baseline to each post-baseline assessment. The formula used is: [(Post-baseline Value - Baseline Value) / Baseline Value] × 100%.
Baseline, and at protocol-specified time points (including the time of first occurrence of Grade ≥2 neurotoxicity symptoms) during the treatment period, up to approximately 24 months.
Cumulative Neurotoxicity Burden (CNB)
기간: From randomization up to the end of treatment (approximately 24 months).

Cumulative Neurotoxicity Burden (CNB) quantifies total peripheral neurotoxicity exposure, incorporating both severity and duration. Neurotoxicity grades are assigned using the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 5.0, a 5-point ordinal scale ranging from Grade 1 (mild) to Grade 5 (death); higher grades indicate worse outcomes.

Two complementary CNB metrics are calculated:

  1. Raw CNB: trapezoidal AUC of NCI-CTCAE grades plotted against time from randomization to end of treatment. Unit: grade-months.
  2. Time-Standardized CNB (CNB-TS): Raw CNB divided by total observation duration, expressed as mean grade per unit time. CNB-TS = AUC / T(observation). This standardizes burden across participants with differing treatment durations.

Higher values of both metrics indicate worse outcomes. Between-arm comparisons will use mixed-effects models adjusted for stratification factors.

From randomization up to the end of treatment (approximately 24 months).
Health-Related Quality of Life: EORTC QLQ-C30
기간: From randomization up to the end of treatment (approximately 24 months), assessed at baseline and at protocol-specified time points.
Health-related quality of life (HRQoL) is assessed using the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30), a 30-item validated cancer- specific questionnaire comprising 5 functional scales (physical, role, emotional, cognitive, social functioning), 3 symptom scales (fatigue, nausea/vomiting, pain), 6 single-item symptom assessments, and a global health status/quality of life scale. All scores are linearly transformed to a 0-100 scale. For functional scales and the global health status/QoL scale, higher scores indicate better functioning or better quality of life (better outcomes). For symptom scales and single items, higher scores indicate a higher level of symptom burden (worse outcomes). Mean changes from baseline in EORTC QLQ-C30 subscale scores will be compared between treatment arms using a mixed-effects model for repeated measures (MMRM), adjusted for stratification factors.
From randomization up to the end of treatment (approximately 24 months), assessed at baseline and at protocol-specified time points.
Chemotherapy-Induced Peripheral Neuropathy Symptoms: EORTC QLQ-CIPN20
기간: From randomization up to the end of treatment (approximately 24 months), assessed at baseline and at protocol-specified time points.

Patient-reported peripheral neuropathy symptoms are assessed using the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire - Chemotherapy-Induced Peripheral Neuropathy 20-item module (EORTC QLQ-CIPN20), a validated 20-item instrument designed to specifically capture sensory, motor, and autonomic symptoms of chemotherapy-induced peripheral neuropathy.

Items are rated on a 4-point Likert scale (1 = "Not at all" to 4 = "Very much"). Raw scores are linearly transformed to a 0-100 scale. Higher scores indicate a higher level of peripheral neuropathy symptom burden (worse outcomes).

Mean changes from baseline in EORTC QLQ-CIPN20 sensory, motor, and autonomic subscale scores will be compared between treatment arms using a mixed-effects model for repeated measures (MMRM), adjusted for stratification factors.

From randomization up to the end of treatment (approximately 24 months), assessed at baseline and at protocol-specified time points.
Objective Response Rate (ORR)
기간: From randomization up to the end of treatment (approximately 24 months).
The proportion of participants who achieve a Complete Response (CR) or Partial Response (PR) based on RECIST v1.1.
From randomization up to the end of treatment (approximately 24 months).
Disease Control Rate (DCR)
기간: From randomization up to the end of treatment (approximately 24 months).
The proportion of participants with CR, PR, or Stable Disease (SD) maintained for at least 6 weeks.
From randomization up to the end of treatment (approximately 24 months).
Progression-Free Survival (PFS)
기간: From randomization up to the end of treatment (approximately 24 months).
The time from randomization to the first disease progression (per RECIST v1.1) or death from any cause, whichever occurs first.
From randomization up to the end of treatment (approximately 24 months).
Overall Survival (OS)
기간: From randomization up to the end of treatment (approximately 24 months).
The time from randomization to death from any cause.
From randomization up to the end of treatment (approximately 24 months).
Duration of Response (DoR)
기간: From randomization up to the end of treatment (approximately 24 months).
The time from the first documented CR or PR until the first documented disease progression or death.
From randomization up to the end of treatment (approximately 24 months).
Adverse Events (AEs)
기간: From randomization up to the end of treatment (approximately 24 months).
Assessment of AEs graded by NCI-CTCAE v5.0 including incidence, type, severity, duration, and relationship to study drug.
From randomization up to the end of treatment (approximately 24 months).

기타 결과 측정

결과 측정
측정값 설명
기간
Serum Neurofilament Light Chain (sNfL) Levels
기간: Baseline, and at protocol-specified time points during treatment (including the time of first occurrence of Grade ≥2 neurotoxicity symptoms), up to approximately 24 months.

Serum neurofilament light chain (sNfL) is a blood-based biomarker of neuronal axonal injury. Elevated sNfL levels reflect ongoing axonal damage and have been validated as a biomarker for chemotherapy-induced peripheral neuropathy and other neurodegenerative conditions.

sNfL concentration is measured in serum using a quantitative single-molecule array (Simoa) immunoassay. Results are reported in picograms per milliliter (pg/mL). Higher sNfL levels indicate greater axonal injury (worse neurological outcomes).

Outcomes assessed include: (1) absolute sNfL concentration at each time point, (2) change from baseline in sNfL concentration, and (3) fold change in sNfL from baseline. Between-arm comparisons will be performed using mixed-effects model for repeated measures (MMRM) on log-transformed values.

Baseline, and at protocol-specified time points during treatment (including the time of first occurrence of Grade ≥2 neurotoxicity symptoms), up to approximately 24 months.

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스폰서

연구 기록 날짜

이 날짜는 ClinicalTrials.gov에 대한 연구 기록 및 요약 결과 제출의 진행 상황을 추적합니다. 연구 기록 및 보고된 결과는 공개 웹사이트에 게시되기 전에 특정 품질 관리 기준을 충족하는지 확인하기 위해 국립 의학 도서관(NLM)에서 검토합니다.

연구 주요 날짜

연구 시작 (추정된)

2026년 7월 15일

기본 완료 (추정된)

2028년 12월 1일

연구 완료 (추정된)

2029년 12월 1일

연구 등록 날짜

최초 제출

2026년 5월 10일

QC 기준을 충족하는 최초 제출

2026년 6월 17일

처음 게시됨 (실제)

2026년 6월 23일

연구 기록 업데이트

마지막 업데이트 게시됨 (실제)

2026년 6월 23일

QC 기준을 충족하는 마지막 업데이트 제출

2026년 6월 17일

마지막으로 확인됨

2026년 6월 1일

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방광암에 대한 임상 시험

Sacituzumab Tirumotecan에 대한 임상 시험

구독하다