Biomarker-driven trials are transforming oncology research, enabling targeted therapies for genetically defined populations. Precision partnered with a biotech sponsor developing a Notch-targeted therapy to manage two concurrent Phase 2 trials—one in Adenoid Cystic Carcinoma (ACC) and one in triple-negative breast cancer (TNBC).
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Study Type |
Phase 2 |
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Therapeutic Area |
Oncology |
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Indication |
ACC with activating Notch mutations |
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Geography |
US, Canada, EU |
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Sites |
24 total (17 US/Canada, 7 EU) |
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Precision’s Role |
End-to-end operational support including site selection, biomarker strategy, NGS coordination, and regulatory guidance |
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Key Challenges |
Biomarker-driven eligibility, NGS coordination, regulatory risk determination, and minimizing screen failures |
This case study focuses on the ACC trial, which required identifying patients with activating Notch mutations through next-generation sequencing (NGS). The study was split into two parts:
- Part 1: 35+ patients enrolled over 12 months
- Part 2: 50+ additional patients targeted over the following 12 months
Strategic Design and Site Selection
Precision’s success began with identifying the right sites—those capable of performing NGS testing either internally or through a vendor. This capability was critical to minimizing screen failures and accelerating enrollment.
Key considerations included:
- Targeting Notch 1–4 mutations
- Implementing pre-screening informed consent (ICF) to reduce screen failures
- Navigating Significant Risk Determination as part of regulatory submission
- Sponsor review and approval of NGS results prior to patient enrollment
Lessons Learned and Operational Wins
Despite the complexity of biomarker-driven enrollment, the ACC trial exceeded expectations:- Enrollment rates were better than anticipated, thanks to strategic site selection and pre-screening
- NGS testing was streamlined, with vendor activation completed upfront
- Site coordination was optimized, enabling consistent sample handling and data flow
- Regulatory alignment was achieved early, avoiding delays in patient entry
Precision’s proactive approach ensured that biomarker testing did not become a bottleneck, but rather a catalyst for efficient enrollment.
Precision’s Path to Biomarker-Driven Enrollment in Rare Oncology
The ACC trial demonstrated that biomarker-driven studies can succeed with the right infrastructure, planning, and site partnerships. Precision’s experience in coordinating NGS workflows, managing regulatory complexity, and supporting site operations was instrumental in achieving strong enrollment and data quality.
