一项评估 Vismodegib（GDC-0449，Hedgehog 通路抑制剂）在晚期基底细胞癌患者中的疗效和安全性的研究
2026年4月17日 更新者：Genentech, Inc.
一项评估 GDC-0449 在晚期基底细胞癌患者中的疗效和安全性的关键 II 期、多中心、单臂、双队列试验
这是一项 II 期、单臂、双队列多中心临床试验，评估 vismodegib (GDC-0449) 在晚期基底细胞癌患者中的疗效和安全性。
所有患者均接受 vismodegib，直至出现进展证据、最有可能归因于 vismodegib 的不可耐受毒性或退出研究。
研究概览
研究类型
介入性
注册 (实际的)
104
阶段
- 阶段2
联系人和位置
本节提供了进行研究的人员的详细联系信息，以及有关进行该研究的地点的信息。
学习地点
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Essen、德国、45122
- Universitätsklinik Essen; Klinik und Poliklinik für Dermatologie, Venerologie und Allergologie
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Kiel、德国、24105
- Universitätsklinikum Schleswig
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Tübingen、德国、72076
- Universitaets-Hautklinik Tuebingen
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Würzburg、德国、97080
- Universitatsklinikum Wurzburg; Derma
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Anderlecht、比利时、1070
- Institut Jules Bordet
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Wilrijk、比利时、2610
- Sint Augustinus Wilrijk, Apotheek
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Lille、法国、59037
- Hôpital Claude Huriez
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Nantes、法国、44093
- Hopital Hotel Dieu Et Hme; Clinique Dermatologique
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Paris、法国、75475
- Hopital Saint Louis; Dermatologie 1
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Pierre-Bénite、法国、69495
- Centre Hospitalier Lyon Sud; Dermatologie
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New South Wales
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Kogarah、New South Wales、澳大利亚、2217
- St George Hospital
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Queensland
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Woolloongabba、Queensland、澳大利亚、4102
- Princess AleXandra Hospital; Department of Medical Oncology
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Victoria
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Melbourne、Victoria、澳大利亚、3000
- Peter MacCallum Cancer Centre
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Arizona
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Scottsdale、Arizona、美国、85259
- Mayo Clinic
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California
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Los Angeles、California、美国、90025
- Angeles Clinic & Rsch Inst
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Palo Alto、California、美国、94304
- Stanford University Medical Center
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San Francisco、California、美国、94115
- Univ of Calif-San Francisco
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Colorado
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Aurora、Colorado、美国、80045
- Univ of Colorado Hlth Sci Ctr
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Florida
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Ormond Beach、Florida、美国、32174
- Advanced Derm & Cosmetic Surg
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Illinois
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Chicago、Illinois、美国、60611
- Northwestern University
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Iowa
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Sioux City、Iowa、美国、51101
- Siouxland Hematology/Oncology
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Maryland
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Baltimore、Maryland、美国、21231
- Johns Hopkins Univ Med Center
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Massachusetts
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Boston、Massachusetts、美国、02215
- Dana Farber Cancer Institute
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Boston、Massachusetts、美国、02114
- Massachusetts General Hospital.
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Michigan
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Ann Arbor、Michigan、美国、48109
- University Of Michigan Comprehensive Cancer Center
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Minnesota
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Rochester、Minnesota、美国、55905
- Mayo Clinic Rochester; Medical Oncology
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Nevada
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Las Vegas、Nevada、美国、89103
- VA Southern Nevada Healthcare
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New Hampshire
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Lebanon、New Hampshire、美国、03756
- Dartmouth Hitchcock Med Center
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New York
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New York、New York、美国、10029
- Mount Sinai School of Medicine; Dermatology
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North Carolina
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Chapel Hill、North Carolina、美国、27599-7305v
- Univ No Carolina School of Med; Physicians Office Bldg
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Ohio
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Columbus、Ohio、美国、43210
- Arthur G. James Cancer Hosp
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Pennsylvania
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Philadelphia、Pennsylvania、美国、19104
- University of Pennsylvania
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Tennessee
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Nashville、Tennessee、美国、37203
- The Sarah Cannon Research Inst
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Texas
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Houston、Texas、美国、77030-4095
- MD Anderson Cancer Center
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London、英国、SW3 6JJ
- Royal Marsden Hospital - London
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Poole、英国、BH15 2JB
- Poole Hospital; Dorset Cancer Centre
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参与标准
研究人员寻找符合特定描述的人，称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。
资格标准
适合学习的年龄
18年 及以上 (成人、年长者)
接受健康志愿者
不
描述
纳入标准：
- ≥ 18 岁的男性和女性。
- 对于转移性基底细胞癌 (BCC) 患者，远处 BCC 转移（例如，肺、肝、淋巴结或骨）的组织学确认，转移性疾病是实体瘤反应评估标准（RECIST）可使用计算机断层扫描测量（ CT）或磁共振成像（MRI）。
- 对于局部晚期 BCC 患者，经组织学证实的疾病被认为不能手术。
- 对于局部晚期 BCC 患者，除非放疗禁忌或不合适，否则必须先前对其局部晚期 BCC 进行过放疗。 对于局部晚期 BCC 接受过照射的患者，疾病一定是在照射后进展的。
- 对于有生育能力的女性，同意在研究期间和停用 vismodegib (GDC-0449) 后的 12 个月内使用两种可接受的避孕方法，包括一种屏障方法。
- 对于有生育潜力女性伴侣的男性，同意使用乳胶避孕套，并建议其女性伴侣在研究期间和停用 vismodegib 后 3 个月内使用额外的避孕方法。
排除标准：
- 先前使用 vismodegib 或其他 Hedgehog 通路抑制剂治疗。
- 怀孕或哺乳。
- 预期寿命 < 12 周。
- 浅表多灶性基底细胞癌患者，由于受累范围广，可能被认为无法切除。
- 同时进行非方案指定的抗肿瘤治疗（例如，化学疗法、其他靶向疗法、放射疗法或光动力疗法）。
- 最近、当前或计划参与实验性药物研究。
- 在本研究中用 vismodegib 治疗的第一天（第 1 天）起 3 年内有其他恶性肿瘤病史，转移或死亡风险可忽略不计的肿瘤除外，例如充分治疗的皮肤鳞状细胞癌、导管癌乳房原位癌，或子宫颈原位癌。
- 不受控制的内科疾病，例如需要静脉注射抗生素治疗的感染。
学习计划
本节提供研究计划的详细信息，包括研究的设计方式和研究的衡量标准。
研究是如何设计的？
设计细节
- 主要用途：治疗
- 分配：不适用
- 介入模型：单组作业
- 屏蔽：无（打开标签）
武器和干预
参与者组/臂 |
干预/治疗 |
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实验性的：维莫吉布 150 毫克
患者每天口服一次 vismodegib 150 mg，直至疾病进展；无法忍受的毒性，最有可能归因于 vismodegib；或退出研究。
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在硬明胶胶囊中提供 Vismodegib 150 mg。
其他名称：
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研究衡量的是什么？
主要结果指标
结果测量 |
措施说明 |
大体时间 |
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Objective Response (OR) Determined by the Independent Review Facility
大体时间：At Baseline and once every 8 weeks thereafter (responses confirmed within ≥4 weeks) until the end of study (up to the clinical cutoff date of 26 November 2010, up to 90 weeks)
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OR=complete (CR) or partial response (PR).
Metastatic-CR:Disappearance of all targets.
PR:≥30% decreased sum of longest diameter (SLD) of targets compared to baseline (B).
Locally advanced-Response=No progressive disease (PD) and ≥30% decreased SLD from baseline (radiography [R]) or ≥30% decreased SLD from B (externally visible dimension [EVD]) or completely resolved ulceration.
CR:Response with no residual BCC on tumor biopsy (otherwise response was PR).
PD:Any of ≥20% increased SLD from nadir (R or EVD), new ulceration, new lesions (R or physical exam) or non-target lesion progression by R.
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At Baseline and once every 8 weeks thereafter (responses confirmed within ≥4 weeks) until the end of study (up to the clinical cutoff date of 26 November 2010, up to 90 weeks)
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次要结果测量
结果测量 |
措施说明 |
大体时间 |
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Duration of Objective Response (OR) Determined by the Independent Review Facility
大体时间：From initial OR until the earliest documented disease progression (PD) or death (until clinical cutoff date of 26 November 2010, up to 90 weeks)
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Duration of OR was defined as the time from the initial CR or PR to the earliest documented disease progression (PD) or death.
Metastatic BCC - PD: ≥ 20% increased sum of the longest diameter (SLD) of targets from nadir, or 1 or more new lesions.
Locally advanced BCC - PD: any of: (1) ≥ 20% increased SLD from nadir (radiography or externally visible dimension); (2) new ulceration; (3) new lesions (radiography or physical exam); (4) progression of non-target lesions by radiography.
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From initial OR until the earliest documented disease progression (PD) or death (until clinical cutoff date of 26 November 2010, up to 90 weeks)
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Progression-Free Survival (PFS) Determined by the Independent Review Facility
大体时间：From the initial dose of study drug until the earliest documented disease progression (PD) or death (up to the clinical cutoff date of 28 November 2011, up to 2 years, 5.5 months)
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PFS was defined as the time from start of treatment to the earliest documented disease progression (PD) or death.
Metastatic BCC - PD: ≥ 20% increased sum of the longest diameter (SLD) of targets from nadir, or 1 or more new lesions.
Locally advanced BCC - PD: any of: (1) ≥ 20% increased SLD from nadir (radiography or externally visible dimension); (2) new ulceration; (3) new lesions (radiography or physical exam); (4) progression of non-target lesions by radiography.
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From the initial dose of study drug until the earliest documented disease progression (PD) or death (up to the clinical cutoff date of 28 November 2011, up to 2 years, 5.5 months)
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Overall Survival
大体时间：From the initial dose of study drug until death from any cause (up to the clinical cutoff date of 30 May 2013, up to 4 years)
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Overall survival was defined as the time from the initial dose of vismodegib until death from any cause.
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From the initial dose of study drug until death from any cause (up to the clinical cutoff date of 30 May 2013, up to 4 years)
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Change From Baseline in Short Form 36 (SF-36) Health Survey Scores
大体时间：Baseline, Week 12, Week 24, and at the end of the study or early termination visit (up to the clinical cutoff date of 26 November 2010, up to 90 weeks)
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The SF-36 Health Survey (Version 2) uses patient-reported symptoms on 8 subscales to assess health-related quality of life (HRQoL).
The Physical Component Summary (PCS) score summarizes the subscales Physical Functioning, Role-Physical, Bodily Pain, and General Health.
The Mental Component Summary (MCS) score summarizes the subscales Vitality, Social Functioning, Role-Emotional, and Mental Health.
Each score was scaled from 0 to 100.
A positive change score indicates better HRQoL.
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Baseline, Week 12, Week 24, and at the end of the study or early termination visit (up to the clinical cutoff date of 26 November 2010, up to 90 weeks)
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Percentage of Participants With Absence of Residual Basal Cell Carcinoma (BCC) in the Efficacy-Evaluable Locally Advanced BCC Cohort
大体时间：At Baseline and 24 weeks, and at any optional point post-baseline through end of the study (up to the clinical cutoff date of 26 November 2010, up to 90 weeks)
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In patients with locally advanced BCC, the histopathological effect of vismodegib was determined in tissue biopsies obtained at baseline (prior to study drug dosing) and at 24 weeks after the start of vismodegib treatment, if the patient was still on study without evidence of progression, or at the investigator's assessment of best clinical response (or best clinical/RECIST response), if occurring prior to 24 weeks.
At any time during a patient's participation in the study, an optional tumor biopsy might have been requested to clarify the response status of the patient.
Reported is the percentage of efficacy-evaluable patients with locally advanced BCC pathology that was confirmed in a baseline biopsy who had an absence of residual BCC post-baseline as assessed by an independent pathological review.
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At Baseline and 24 weeks, and at any optional point post-baseline through end of the study (up to the clinical cutoff date of 26 November 2010, up to 90 weeks)
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合作者和调查者
在这里您可以找到参与这项研究的人员和组织。
调查人员
- 研究主任：Jeannie Hou, M.D.、Genentech, Inc.
出版物和有用的链接
负责输入研究信息的人员自愿提供这些出版物。这些可能与研究有关。
一般刊物
- Chang AL, Arron ST, Migden MR, Solomon JA, Yoo S, Day BM, McKenna EF, Sekulic A. Safety and efficacy of vismodegib in patients with basal cell carcinoma nevus syndrome: pooled analysis of two trials. Orphanet J Rare Dis. 2016 Sep 1;11(1):120. doi: 10.1186/s13023-016-0506-z.
- Sekulic A, Migden MR, Oro AE, Dirix L, Lewis KD, Hainsworth JD, Solomon JA, Yoo S, Arron ST, Friedlander PA, Marmur E, Rudin CM, Chang AL, Low JA, Mackey HM, Yauch RL, Graham RA, Reddy JC, Hauschild A. Efficacy and safety of vismodegib in advanced basal-cell carcinoma. N Engl J Med. 2012 Jun 7;366(23):2171-9. doi: 10.1056/NEJMoa1113713.
- Sekulic A, Migden MR, Basset-Seguin N, Garbe C, Gesierich A, Lao CD, Miller C, Mortier L, Murrell DF, Hamid O, Quevedo JF, Hou J, McKenna E, Dimier N, Williams S, Schadendorf D, Hauschild A; ERIVANCE BCC Investigators. Long-term safety and efficacy of vismodegib in patients with advanced basal cell carcinoma: final update of the pivotal ERIVANCE BCC study. BMC Cancer. 2017 May 16;17(1):332. doi: 10.1186/s12885-017-3286-5.
研究记录日期
这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查，以确保它们在发布到公共网站之前符合特定的质量控制标准。
研究主要日期
学习开始 (实际的)
2009年2月10日
初级完成 (实际的)
2010年11月26日
研究完成 (实际的)
2014年4月9日
研究注册日期
首次提交
2009年1月30日
首先提交符合 QC 标准的
2009年1月30日
首次发布 (估计的)
2009年2月2日
研究记录更新
最后更新发布 (实际的)
2026年5月11日
上次提交的符合 QC 标准的更新
2026年4月17日
最后验证
2026年4月1日
更多信息
与本研究相关的术语
其他研究编号
- SHH4476g
- GO01541
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是的
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See Roche's commitment to transparency of clinical study information here: https://go.roche.com/data_sharing
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