Oncology · Clinical trials
Basal Cell Carcinoma Clinical Trials
Approved therapies, the pivotal-trial endpoints they were judged on, the magnitude of benefit — and the drugs that failed their endpoints, and why.
Indication overview
About basal cell carcinoma — and why its trials are hard
Basal cell carcinoma is the most common human cancer, a slow-growing keratinocyte malignancy of the skin driven overwhelmingly by aberrant Hedgehog signalling, most often through inactivating PTCH1 mutations or activating SMO mutations. The vast majority of cases are cured by simple surgical excision, Mohs micrographic surgery, or local destructive/topical therapies, and metastasis is rare. Systemic therapy is reserved for the small minority with locally advanced disease not amenable to surgery or radiotherapy, or with metastatic disease. For these patients the Hedgehog pathway inhibitors vismodegib and sonidegib, which block the smoothened (SMO) receptor, produce meaningful response rates and were practice-changing. Their use is limited by characteristic on-target toxicities (muscle spasms, dysgeusia, alopecia) causing frequent discontinuation, and by acquired resistance. For patients who progress on or cannot tolerate Hedgehog inhibitors, the anti-PD-1 antibody cemiplimab provides an immunotherapy option, exploiting the very high ultraviolet-induced mutational burden of these tumours. Overall prognosis for advanced BCC has improved markedly, though durable control after Hedgehog-inhibitor failure remains an area of need.
Approved therapies & pivotal evidence
What's been approved — and by how much it moved the endpoint
| Drug (brand) | Approved | Setting | Pivotal trial | Primary endpoint | Magnitude of benefit |
|---|---|---|---|---|---|
| Vismodegib (Erivedge) | 2012 | Metastatic BCC, or locally advanced BCC recurring after surgery or unsuitable for surgery/radiotherapy | ERIVANCE (phase 2) | Objective response rate | ORR ~43% in locally advanced and ~30% in metastatic BCC |
| Sonidegib (Odomzo) | 2015 | Locally advanced BCC recurring after surgery/radiotherapy or unsuitable for them | BOLT (phase 2) | Objective response rate | ORR ~56% in locally advanced BCC (investigator-assessed); durable responses |
| Cemiplimab (Libtayo) | 2021 | Locally advanced (accelerated) or metastatic BCC after Hedgehog inhibitor or when a HHI is not appropriate | Study 1620 (phase 2) | Objective response rate | ORR ~31% in locally advanced BCC after HHI therapy, with durable responses |
Choosing the right endpoint
Primary endpoints that matter in basal cell carcinoma trials
- Objective response rate (ORR) — Primary endpoint for all advanced-BCC approvals (ERIVANCE, BOLT, cemiplimab), reflecting rarity of advanced disease and single-arm designs
- Duration of response (DoR) — Critical secondary measure given on-target Hedgehog toxicities and acquired resistance limiting long-term benefit
- Progression-free survival (PFS) — Supportive endpoint in single-arm trials of advanced disease
- Complete response rate — Meaningful in locally advanced disease where complete clearance can restore surgical resectability
- Safety/tolerability and discontinuation rate — Especially important for Hedgehog inhibitors, where muscle spasms, dysgeusia and alopecia drive frequent treatment interruption
How iNGENū runs basal cell carcinoma trials
Physician-led design, built for FDA submission
Endpoint & biomarker strategy
Board-certified specialists design endpoints and patient selection aligned to current FDA guidance for this indication.
FDA-ready data
Built to ICH-GCP and 21 CFR 312.120, with direct FDA submission — data accepted by the FDA, EMA and MHRA.
Faster, lower-cost delivery
~4-week ethics via the TGA CTN scheme, up to 43.5% R&D rebate, and 80–90% below US CRO cost.
Request a fixed-milestone proposal and a tailored endpoint & feasibility summary for this indication.
Frequently asked questions
Basal Cell Carcinoma clinical trials — FAQs
Does most basal cell carcinoma need systemic drugs?
How do Hedgehog inhibitors work?
What can be done after Hedgehog inhibitors stop working?
Ready to discuss your basal cell carcinoma trial?
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