OVARIAN CANCER · White paper
Ovarian 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 ovarian cancer — and why its trials are hard
Ovarian cancer is the fifth-leading cause of cancer death among women and the most lethal gynecologic malignancy, largely because its non-specific symptoms drive late-stage diagnosis. The median age at diagnosis is about 63, and roughly 10-15% of cases are hereditary, most commonly BRCA1/BRCA2 mutations (also Lynch syndrome). Most tumors are high-grade serous epithelial cancers. The therapeutic backbone remains cytoreductive surgery plus platinum/taxane chemotherapy (carboplatin and paclitaxel), with the anti-angiogenic antibody bevacizumab and, transformatively, PARP inhibitors (olaparib, niraparib, rucaparib) used in maintenance, especially for BRCA-mutated and homologous-recombination-deficient disease. PARP inhibitors markedly extend progression-free survival, though several later-line, non-BRCA indications were voluntarily withdrawn in 2022 after mature overall-survival data raised concerns. Trial design is complicated by high recurrence rates, tumor heterogeneity across subtypes, ascites affecting drug distribution, variable surgical timing, and reliance on surrogate endpoints such as PFS and CA-125 response. Precision-medicine stratification by BRCA/HRD status and adaptive, biomarker-driven designs are central to modern development.
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 |
|---|---|---|---|---|---|
| Carboplatin (Paraplatin) | 1989 | First-line chemotherapy backbone (with paclitaxel) for epithelial ovarian cancer | GOG/ICON platinum-taxane trials | Overall survival / progression-free survival | Platinum-taxane doublet is the established standard-of-care backbone; better tolerated than cisplatin with comparable efficacy |
| Paclitaxel (Taxol) | 1992 | First-line chemotherapy (with a platinum agent) for advanced ovarian cancer | GOG-111 / OV-10 | Overall survival and response rate | Adding paclitaxel to platinum significantly improved survival vs prior cyclophosphamide-based regimens |
| Bevacizumab (Avastin) | 2014 | Platinum-resistant recurrent disease (2014); later front-line and maintenance (VEGF inhibitor) | AURELIA / GOG-0218 | Progression-free survival | Significant PFS improvement when added to chemotherapy and continued as maintenance |
| Olaparib (Lynparza) | 2018 | First-line maintenance in BRCA-mutated advanced ovarian cancer after platinum response (PARP inhibitor; initially approved 2014 for later lines) | SOLO-1 (NCT01844986) | Progression-free survival | Substantial PFS benefit; HR ~0.30 vs placebo with a large, durable improvement in median PFS |
| Niraparib (Zejula) | 2020 | First-line maintenance for advanced ovarian cancer after platinum response regardless of biomarker status (PARP inhibitor; initially 2017 for recurrent) | PRIMA (NCT02655016) | Progression-free survival | Significant PFS improvement across the overall population, with greatest benefit in HRD-positive tumors |
Where trials have failed
Drugs that missed their endpoint — and what contributed
The most instructive lessons in ovarian cancer development come from programmes that failed the endpoint that mattered.
| Drug / trial | Endpoint outcome | What contributed |
|---|---|---|
| Batiraxcept (AXL inhibitor) — NCT04729608 | Failed to show benefit | No significant difference in median PFS between batiraxcept plus paclitaxel and paclitaxel alone |
| Motesanib diphosphate (multi-kinase inhibitor) — NCT00574951 | Trial stopped early | Halted for severe toxicity raising patient-safety concerns |
| Idronoxil / phenoxodiol — NCT00382811 | Ineffective in late-stage disease | Oral phenoxodiol, alone or with weekly carboplatin, was ineffective in late-stage ovarian cancer |
Choosing the right endpoint
Primary endpoints that matter in ovarian cancer trials
- Overall survival (OS) — Gold-standard endpoint but requires long follow-up and can be confounded by post-progression crossover and subsequent therapies
- Progression-free survival (PFS) — Primary surrogate for most maintenance approvals (bevacizumab, PARP inhibitors); depends on consistent RECIST assessment
- Objective response rate (ORR) — Useful in treatment (non-maintenance) settings; requires standardized, reproducible tumor measurement
- CA-125 response (GCIG criteria) — Biomarker of tumor burden for monitoring response and recurrence, but individual variability limits standalone use
- Quality of life / patient-reported outcomes — Increasingly required to show maintenance therapy benefit is not offset by toxicity
How iNGENū runs ovarian 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.
The full white paper: approved-product analyses, pivotal endpoints and trial-design strategy. Open-access · no sign-in.
Frequently asked questions
Ovarian Cancer clinical trials — FAQs
How have PARP inhibitors changed ovarian cancer treatment?
Why were some PARP inhibitor indications withdrawn in 2022?
What is the standard first-line treatment for ovarian cancer?
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