Study Snapshot: A First-in-Human ROS1 Inhibitor Trial in Rare Cancers
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Indication: |
Non-Small Cell Lung Cancer (NSCLC) and other ROS1-positive solid tumors |
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Trial Phase: |
Phase 1–2 (First-in-Human, Dose Escalation + Expansion) |
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Patient Population: |
Adults and pediatric patients with advanced ROS1-positive tumors |
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Geography: |
Australia, Belgium, Canada, France, Germany, Italy, Japan, Netherlands, Singapore, South Korea, Spain, Taiwan, United Kingdom, United States |
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Sites Activated: |
60+ sites |
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Primary Challenge: |
Global FIH trial requiring seamless transition from Phase 1 to Phase 2, rapid site activation, and high-quality data integrity across diverse geographies |
The Challenge: Coordinating a Seamless Global Trial in ROS1-Positive NSCLC
This global, first-in-human Phase 1–2 trial evaluated a novel ROS1 inhibitor in patients with NSCLC and other advanced ROS1-positive solid tumors. The study also included a pediatric cohort and was designed to transition smoothly from dose escalation to expansion.
ROS1 fusions are found in approximately 1–3% of NSCLC cases and are associated with resistance mutations and CNS involvement. The sponsor aimed to address these limitations with a compound showing strong preclinical activity, including intracranial models.
With a target enrollment of 360 patients across 60+ sites in 14 countries, the sponsor needed a partner who could manage global startup, ensure continuity between study phases, and maintain data quality. Precision for Medicine was selected to lead site strategy, operational execution, and medical engagement.
Precision’s Clinical Trial Solution: Rapid Activation and Data Integrity
Pre-Selecting Global Sites to Support Seamless Phase Transition
Precision worked closely with the sponsor to identify and pre-select sites that could support both Phase 1 and Phase 2 activities. This approach minimized delays between phases and ensured continuity in patient enrollment and data collection.
Activating Global Sites in as Little as 42 Days
Precision activated sites across 14 countries, achieving as early as 42 days from study start-to-site activation. This was made possible by leveraging Precision’s global site network and regulatory expertise.
Engaging Investigators Through Medical-to-Medical Collaboration
Precision’s Medical Monitors engaged directly with principal investigators to support patient eligibility reviews, attend SIVs and dose escalation meetings, and participate in regular PI calls. This hands-on approach ensured accurate enrollment and protocol adherence.
Achieving Zero Data Issues at Interim Data Cut
At the time of the interim data cut (23 May 2025), the study had zero missing pages, zero SDV pending, zero open queries, and zero unresolved SAE reconciliation issues—demonstrating exceptional data quality and operational oversight.
Optimizing Protocol Design with Real-Time Sponsor Feedback
Precision provided early feedback on the protocol synopsis and supported the sponsor’s interest in pursuing a tumor-agnostic cohort. Recommendations were incorporated to strengthen the study design and align with regulatory expectations.
Leveraging the Precision Site Network for Rare Oncology Trials
By leveraging its global site network, Precision ensured rapid site identification, startup, and patient recruitment. Sites were selected based on prior performance, infrastructure, and ability to manage complex oncology protocols.
Study Outcomes: Meeting Milestones and Maintaining Quality in Rare Oncology
- As few as 42 days from study start to site activation
- 109 days from site activation to first patient screened in rare population
- 360 rare patients targeted across 60+ global sites
- Zero data issues at interim data cut (no missing pages, SDV, queries, or SAE issues)
- Seamless transition from Phase 1 to Phase 2
- Strong investigator engagement through regular medical-to-medical collaboration
- Protocol enhancements based on real-time feedback and sponsor collaboration
Key Takeaways for Sponsors Running First-in-Human Oncology Trials
First-in-human oncology trials demand precision at every level—from site selection and regulatory planning to patient enrollment and data integrity. This study demonstrates how early alignment, global coordination, and medical engagement can drive success across complex, multi-phase programs.
Precision’s ability to activate sites quickly, maintain high-quality data, and support real-time decision-making helped the sponsor meet aggressive timelines and achieve a clean interim data cut. For sponsors developing targeted therapies in rare or biomarker-driven populations, this model offers a proven path to success.
Whether you're planning a tumor-agnostic expansion or navigating CNS-active compounds, Precision’s integrated approach ensures your trial stays on track and delivers the data you need.
