OPTIMISE-P
Research type
Research Study
Full title
OPTIMISE-P: incOrporating PaTIent-reported outcoMes In doSE-finding trials – Platform study
IRAS ID
340472
Contact name
Christina Yap
Contact email
Sponsor organisation
The Institute of Cancer Research
Duration of Study in the UK
1 years, 7 months, 0 days
Research summary
Phase I trials initiate clinical drug testing by administering escalating doses of Investigational Medicinal Products (IMPs) to small patient groups to identify the maximum tolerated dose (MTD) and recommended phase II dose (RP2D). Traditionally focused on physician-evaluated adverse events, toxicity assessment now includes patient-related factors for a more comprehensive evaluation.
Despite the potential of Patient-Reported Outcomes (PROs) - direct patient reports on health status - as valuable indicators of treatment tolerability, they have been underutilised in early phase trials due to concerns about data processing timeliness, patient burden, and causality with IMPs. The PRO-CTCAE system, assessing 124 items related to adverse events, shows promise in phase I trials but lacks standard reporting guidelines.
The OPTIMISE-P study addresses these gaps by developing a non-interventional PRO-platform trial to assess integrating PROs into early clinical trial decision-making. The platform provides a standardised framework for efficient data collection and analysis across multiple dose-finding oncology trials. OPTIMISE-P incorporates PRO-CTCAE (full and 30-item versions), FACT-GP5, and questions 29 and 30 of the EORTC QLQ-C30. Including an overall side effect impact measure like FACT-GP5, as recommended by the FDA, is valuable in cancer trials. The EORTC QLQ-C30 questions on overall health and quality of life offer a holistic patient perspective, providing context for interpreting adverse events and efficacy measures, and serve as baseline tools for future trials. OPTIMISE-P collects PRO data weekly during dose-limiting toxicity evaluation and monthly thereafter for up to six months, offering a dynamic understanding of patient experiences. Conducted across diverse sites and cancer types, the study introduces unique dimensions to data collection frequency, sample size, and patient diversity, contributing valuable insights to early phase oncology trials.REC name
East of England - Cambridge Central Research Ethics Committee
REC reference
25/EE/0031
Date of REC Opinion
10 Apr 2025
REC opinion
Further Information Favourable Opinion