Oncology · Clinical trials
Vulvar Cancer 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 vulvar cancer — and why its trials are hard
Vulvar cancer is a rare gynecologic malignancy, most commonly squamous cell carcinoma, arising either from HPV infection or from chronic inflammatory conditions such as lichen sclerosus. Management is dominated by surgery and radiation rather than drugs: wide local excision or vulvectomy with sentinel lymph node biopsy or inguinofemoral lymphadenectomy is the mainstay for localized disease, and concurrent chemoradiation (often with cisplatin as a radiosensitizer) is used for locally advanced or unresectable tumors. There are no vulvar-specific systemic drug approvals; advanced and metastatic disease is treated by extrapolation from cervical and other squamous cancers, using platinum-based chemotherapy. The only meaningfully applicable targeted/immunotherapy option is pembrolizumab under its tissue-agnostic approvals for MSI-high/mismatch-repair-deficient tumors and for high tumor mutational burden (TMB-H, at least 10 mutations/megabase), which a subset of vulvar cancers meet. This sparse pharmacologic landscape reflects the disease's rarity and lack of dedicated trials, leaving a substantial unmet need for effective systemic therapy in advanced vulvar cancer.
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 |
|---|---|---|---|---|---|
| Pembrolizumab (Keytruda) | 2017 | Tissue-agnostic: unresectable/metastatic MSI-high or mismatch-repair-deficient (dMMR) solid tumors, including eligible vulvar cancers | KEYNOTE-016/-158/-164 (pooled) | Overall response rate (accelerated, tumor-agnostic) | ORR ~30-40% across MSI-H/dMMR tumors; vulvar-specific data limited |
| Pembrolizumab (Keytruda) | 2020 | Tissue-agnostic: unresectable/metastatic tumor mutational burden-high (TMB-H, >=10 mut/Mb) solid tumors, including eligible vulvar cancers | KEYNOTE-158 (phase 2) | Overall response rate (accelerated, tumor-agnostic) | ORR ~29% in TMB-H cohort; vulvar-specific benefit unverified |
Choosing the right endpoint
Primary endpoints that matter in vulvar cancer trials
- Overall survival (OS) — Definitive outcome for localized surgical and chemoradiation strategies
- Locoregional control / recurrence-free survival — Central to surgery and radiation trials given high rates of local and groin recurrence
- Overall response rate (ORR) — Basis for pembrolizumab's tissue-agnostic approvals applicable to biomarker-selected vulvar tumors
- Groin node status / sentinel node accuracy — Key prognostic and staging endpoint driving surgical decision-making
- Treatment-related morbidity — Important given the substantial functional and wound-healing complications of vulvar surgery and radiation
How iNGENū runs vulvar cancer 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
Vulvar Cancer clinical trials — FAQs
Are there drugs approved specifically for vulvar cancer?
How is localized vulvar cancer treated?
Why are systemic options so limited?
Ready to discuss your vulvar cancer trial?
Talk to our physician-led team about an FDA-ready, cost-efficient trial design.
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