- ICH GCP
- Registr klinických studií v USA
- Klinická studie NCT07662863
Sacituzumab Tirumotecan vs MMAE-ADCs in Advanced Urothelial Carcinoma (FUSCC-SPARE-UC-01) (SPARE-UC-01)
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:
- Receive either sac-TMT or another MMAE-based ADC drug
- Have regular physical exams by a doctor to check their nerves
- Have machine tests to measure how well their nerves work
- Answer survey questions about their pain, numbness, and daily activities
Přehled studie
Postavení
Podmínky
Detailní popis
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.
Typ studie
Zápis (Odhadovaný)
Fáze
- Fáze 2
Kontakty a umístění
Studijní kontakt
- Jméno: Yao Zhu
- Telefonní číslo: +86 13816751347
- E-mail: zhuyao@fudan.edu.cn
Studijní záloha kontaktů
- Jméno: Shanshan Wang
- Telefonní číslo: +86 13918029242
- E-mail: fuscc_wangshanshan@fudan.edu.cn
Studijní místa
-
-
Shanghai Municipality
-
Shanghai, Shanghai Municipality, Čína, 200032
- Fudan University Shanghai Cancer Center
-
Vrchní vyšetřovatel:
- Yao Zhu
-
Kontakt:
- Yao Zhu
- Telefonní číslo: 13816751347
- E-mail: zhuyao@fudan.edu.cn
-
-
Kritéria účasti
Kritéria způsobilosti
Věk způsobilý ke studiu
- Dospělý
- Starší dospělý
Přijímá zdravé dobrovolníky
Popis
Inclusion Criteria
- Must voluntarily sign the written Institutional Review Board (IRB)/Ethics Committee (EC) approved informed consent form (ICF) prior to any screening procedures.
- Age > 18 years at the time of signing the ICF.
- 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%).
- 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).
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.
- 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).
- ECOG Performance Status of 0 or 1 at screening.
- Expected survival > 3 months.
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
- Prior treatment with TROP2-targeted ADCs, topoisomerase I inhibitors (e.g., irinotecan, topotecan), or ADCs containing topoisomerase I inhibitor payloads.
- Patients previously treated with both Enfortumab Vedotin (EV) and Disitamab Vedotin (DV) are excluded from Cohorts A and B (eligible for Cohort C only).
- 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.
- 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.
- 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.
- 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.
- Active keratitis, corneal ulcer, or severe dry eye syndrome.
- 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.
- Hypersensitivity: Known severe hypersensitivity to sac-TMT, EV, DV, or their excipients.
- Any severe or uncontrolled systemic disease that, in the investigator's opinion, increases the risk to the participant.
- HbA1c ≥ 8% (Patients with well-controlled blood glucose, fasting glucose ≤ 10 mmol/L, and investigator approval are eligible).
- History of allogeneic stem cell transplant or solid organ transplant.
- Pregnant or breastfeeding females.
Studijní plán
Jak je studie koncipována?
Detaily designu
- Primární účel: Léčba
- Přidělení: Randomizované
- Intervenční model: Paralelní přiřazení
- Maskování: Žádné (otevřený štítek)
Zbraně a zásahy
Skupina účastníků / Arm |
Intervence / Léčba |
|---|---|
|
Experimentální: 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.
Ostatní jména:
|
|
Aktivní komparátor: 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.
Ostatní jména:
|
|
Jiný: 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.
Ostatní jména:
|
Co je měření studie?
Primární výstupní opatření
Měření výsledku |
Popis opatření |
Časové okno |
|---|---|---|
|
Peripheral Neuropathy Progression Rate (PNPR)
Časové okno: 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).
|
Sekundární výstupní opatření
Měření výsledku |
Popis opatření |
Časové okno |
|---|---|---|
|
Percent Change in NCS Parameters
Časové okno: 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)
Časové okno: 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:
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
Časové okno: 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
Časové okno: 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)
Časové okno: 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)
Časové okno: 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)
Časové okno: 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)
Časové okno: 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)
Časové okno: 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)
Časové okno: 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).
|
Další výstupní opatření
Měření výsledku |
Popis opatření |
Časové okno |
|---|---|---|
|
Serum Neurofilament Light Chain (sNfL) Levels
Časové okno: 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.
|
Spolupracovníci a vyšetřovatelé
Sponzor
Termíny studijních záznamů
Hlavní termíny studia
Začátek studia (Odhadovaný)
Primární dokončení (Odhadovaný)
Dokončení studie (Odhadovaný)
Termíny zápisu do studia
První předloženo
První předloženo, které splnilo kritéria kontroly kvality
První zveřejněno (Aktuální)
Aktualizace studijních záznamů
Poslední zveřejněná aktualizace (Aktuální)
Odeslaná poslední aktualizace, která splnila kritéria kontroly kvality
Naposledy ověřeno
Více informací
Termíny související s touto studií
Klíčová slova
Další relevantní podmínky MeSH
- Urogenitální onemocnění
- Nemoci nervového systému
- Urogenitální novotvary
- Novotvary podle místa
- Novotvary
- Mužská urogenitální onemocnění
- Urologická onemocnění
- Ženské urogenitální onemocnění
- Ženské urogenitální onemocnění a těhotenské komplikace
- Neuromuskulární onemocnění
- Novotvary podle histologického typu
- Novotvary, žlázové a epiteliální
- Urologické novotvary
- Karcinom
- Onemocnění močového měchýře
- Onemocnění periferního nervového systému
- Novotvary močového měchýře
- Karcinom, přechodná buňka
Další identifikační čísla studie
- FUSCC-SPARE-UC-01
Plán pro data jednotlivých účastníků (IPD)
Plánujete sdílet data jednotlivých účastníků (IPD)?
Informace o lécích a zařízeních, studijní dokumenty
Studuje lékový produkt regulovaný americkým FDA
Studuje produkt zařízení regulovaný americkým úřadem FDA
Tyto informace byly beze změn načteny přímo z webu clinicaltrials.gov. Máte-li jakékoli požadavky na změnu, odstranění nebo aktualizaci podrobností studie, kontaktujte prosím register@clinicaltrials.gov. Jakmile bude změna implementována na clinicaltrials.gov, bude automaticky aktualizována i na našem webu .
Klinické studie na Sacituzumab Tirumotecan
-
National Cancer Centre, SingaporeMerck Sharp & Dohme LLC; National Cancer Center, JapanZatím nenabírámeRecidivující adenoidní cystický karcinom | Papilární karcinom štítné žlázy | Metastazující adenoidně cystický karcinomSingapur, Japonsko, Malajsie, Jižní Korea
-
Yale UniversityJohns Hopkins University; Merck Sharp & Dohme LLC; Translational Breast Cancer...Zatím nenabírámeMetastatické triple negativní rakoviny prsuSpojené státy
-
Sichuan Kelun-Biotech Biopharmaceutical Co., Ltd.Zatím nenabíráme
-
Guangdong Association of Clinical TrialsNábor
-
Zheng MinQilu Pharmaceutical Co., Ltd.; Sichuan Kelun-Biotech Biopharmaceutical Co.,...Zatím nenabíráme
-
Sichuan Kelun-Biotech Biopharmaceutical Co., Ltd.Nábor
-
Tianjin Medical University Cancer Institute and...Southwest Hospital, ChinaZatím nenabírámeTNBC - Triple-negativní rakovina prsu | PD-L1 pozitivní
-
Tianjin Medical University Cancer Institute and...Zatím nenabírámeTriple negativní rakovina prsu (TNBC) | PD-L1 pozitivníČína
-
M.D. Anderson Cancer CenterMerck Sharp & Dohme LLCZatím nenabírámeFáze 2 | Sacituzumab Tirumotecan | Refrakterní metastatický | Neresekabilní dlaždicobuněčný | Karcinom buněk anu/konečníkuSpojené státy
-
M.D. Anderson Cancer CenterMerck Sharp & Dohme LLCZatím nenabírámeFáze II | Sacituzumab | Tirumotecan | Renální medulární karcinom s deficitem SMARCB1Spojené státy