Trial reportThe New England journal of medicine2012
Efficacy and safety of vismodegib in advanced basal-cell carcinoma.
Trial report in The New England journal of medicine, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 4 registered trials, which are not on this map. Cited by 620 papers, 6 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Pivotal Phase II, Multicenter, Single-arm, Two-cohort Trial Evaluating the Efficacy and Safety of GDC-0449 in Patients With Advanced Basal Cell Carcinoma
ML28485:Phase 2B Single-site,Open-label,Nonrandomized Study Evaluating Efficacy of Oral Vismodegib in Various Histologic Subtypes (Infiltrative/Morpheaform,Nodular and Superficial)of High Risk and/or Locally Advanced Basal Cell Carcinoma
A Single Arm, Phase II, Multicenter Study To Assess The Efficacy And Safety Of Vismodegib And Radiotherapy In Patients With High Risk Or Locally Advanced Basal Cell Carcinoma Not Amenable To Radical Surgery
Registration of Oral Hedgehog Inhibitors Vismodegib and Sonidegib in the Treatment of Advanced and Multiple Basal Cell Carcinoma in the Netherlands: a Prospective Registration Study.
Who cites it
620 citing papers in PubMed, 6 syntheses or guidelines pooled it.
- Efficacy and safety of vismodegib in periocular and orbital basal cell carcinoma: a systematic review and meta-analysis.Frontiers in medicine · 2026Pooled it
- Prevalence of genetic alterations in basal cell carcinoma patients resistant to Hedgehog pathway inhibitors: a systematic review.Annals of medicine · 2025Pooled it
- European Association of Neuro-Oncology guideline on molecular testing of meningiomas for targeted therapy selection.Neuro-oncology · 2025Guideline
- S2k guideline basal cell carcinoma of the skin (update 2023).Journal der Deutschen Dermatologischen Gesellschaft = Journal of the German Society of Dermatology : JDDG · 2024Guideline
- Efficacy and Safety of Sonic Hedgehog Inhibitors in Basal Cell Carcinomas: An Updated Systematic Review and Meta-analysis (2009-2022).American journal of clinical dermatology · 2023Pooled it
- Swiss Recommendations for Cutaneous Basal Cell Carcinoma.Dermatology (Basel, Switzerland) · 2023Guideline
- A Phase II Trial of Perioperative Oral Itraconazole for the Management of Low-Risk Basal Cell Carcinoma.Experimental dermatology · 2026Trial
- Phase II Study of Vismodegib in Patients WithJCO precision oncology · 2025Trial
- Phase II, Single-Arm Trial of Induction and Concurrent Vismodegib With Curative-Intent Radiation Therapy for Locally Advanced, Unresectable Basal Cell Carcinoma.Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2024Trial
- Alliance A071401: Phase II Trial of Focal Adhesion Kinase Inhibition in Meningiomas With SomaticJournal of clinical oncology : official journal of the American Society of Clinical Oncology · 2023Trial
- Pilot study of bempegaldesleukin in combination with nivolumab in patients with metastatic sarcoma.Nature communications · 2022Trial
- Review
- The Therapeutic Potential of Purmorphamine Across Disease Models.Journal of cellular and molecular medicine · 2026Review
- Massive Solitary Liver Metastasis From a Locally Recurrent Basal Cell Carcinoma Treated With Stereotactic Body Radiation Therapy: A Rare Manifestation of a Common Disease.Advances in radiation oncology · 2026Article
- Tumor heterogeneity: development, mechanisms, and therapeutic implications.Signal transduction and targeted therapy · 2026Review
- Transforming Outcomes in Advanced Basal Cell Carcinoma: The Evolving Role of Systemic Therapy.American journal of clinical dermatology · 2026Review
- Context-dependent synthetic lethality - an emerging precision therapeutic approach.Nature reviews. Cancer · 2026Review
- Molecular mechanisms for sustaining cancer stem cells characteristics and targeted therapeutic strategies.Acta pharmacologica Sinica · 2026Review
- Hedgehog Inhibitor and Paclitaxel: A Phase I Trial of the Oral Hedgehog inhibitor Taladegib in Combination with Weekly Paclitaxel in Patients with Advanced Solid Tumours.Investigational new drugs · 2026Article
- Use of Hedgehog Pathway Inhibitors in Combination Therapy for Basal Cell Carcinoma: A Review and Narrative Synthesis.The Journal of clinical and aesthetic dermatology · 2026Review
560 more citing papers are in PubMed but not listed here.
Corrections and comments
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Authors and funding
19 authors.
Funding
Abstract
backgroundAlterations in hedgehog signaling are implicated in the pathogenesis of basal-cell carcinoma. Although most basal-cell carcinomas are treated surgically, no effective therapy exists for locally advanced or metastatic basal-cell carcinoma. A phase 1 study of vismodegib (GDC-0449), a first-in-class, small-molecule inhibitor of the hedgehog pathway, showed a 58% response rate among patients with advanced basal-cell carcinoma.
methodsIn this multicenter, international, two-cohort, nonrandomized study, we enrolled patients with metastatic basal-cell carcinoma and those with locally advanced basal-cell carcinoma who had inoperable disease or for whom surgery was inappropriate (because of multiple recurrences and a low likelihood of surgical cure, or substantial anticipated disfigurement). All patients received 150 mg of oral vismodegib daily. The primary end point was the independently assessed objective response rate; the primary hypotheses were that the response rate would be greater than 20% for patients with locally advanced basal-cell carcinoma and greater than 10% for those with metastatic basal-cell carcinoma.
resultsIn 33 patients with metastatic basal-cell carcinoma, the independently assessed response rate was 30% (95% confidence interval [CI], 16 to 48; P=0.001). In 63 patients with locally advanced basal-cell carcinoma, the independently assessed response rate was 43% (95% CI, 31 to 56; P<0.001), with complete responses in 13 patients (21%). The median duration of response was 7.6 months in both cohorts. Adverse events occurring in more than 30% of patients were muscle spasms, alopecia, dysgeusia (taste disturbance), weight loss, and fatigue. Serious adverse events were reported in 25% of patients; seven deaths due to adverse events were noted.
conclusionsVismodegib is associated with tumor responses in patients with locally advanced or metastatic basal-cell carcinoma. (Funded by Genentech; Erivance BCC ClinicalTrials.gov number, NCT00833417.).
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.