Abstract 552P
Background
Phase I trials aim at offering new therapeutic opportunities to pre-treated patients (pts) while assessing efficacy/safety. Trial enrolment is a two-step process: first, an investigator evaluates the pts’ eligibility, then the pt is screened and undergoes new tests. This warrants that pts meet the protocol’s criteria, notably 90-day survival. We report reasons of screening (scr) failure in pts who were deemed eligible by investigators and their outcome.
Methods
We reviewed files of pts with solid tumors who consented to participate in a phase I trial yet screenfailed between 2016 and 2019 in the Clinical Research Unit of Institut Universitaire du Cancer of Toulouse, France. We collected demographic data, performance status (PS), time between progression and referral, Royal-Marsden Score (RMS) upon signature, reason of non-enrolment and survival. Trials requiring a biomarker for enrolment were excluded.
Results
One-hundred ten pts aged from 25 to 84 years failed to enrol in a phase I/Ib trial during the scr phase. Median time between progression and first evaluation by the investigator was 20 days, with 97/110 PS 0-1 pts. Reasons for scr failure were: biological criteria in 29.1% (N=32, 53% due to vital organ’s dysfunction), radiological criteria in 17.3% (N=19, 73% due to newly discovered brain metastases & 15% for non-accessible lesions for mandatory biopsy); PS deterioration in 14.5% (N=16); clinical exclusion criteria in 13.6 % (N=15), and “administrative” reasons in 16.7% (N=18) of the cases. The 90-day and 6 -month mortality rate after scr failure was of 34.5% and 56%, respectively. Scr failure due to PS deterioration (50%) or discovery of brain metastases (36%) explained most of 90-day mortality. RMS upon signature was available for 54 pts, those with an RMS⩾2 showed a significant risk of dying within 6 months (p = 0.0239) but not within 90 days.
Conclusions
Reasons of scr failure after initial evaluation are mostly biological, radiological and deterioration of PS during the 28-day period. Pts referred for phase I trials should provide brain scan and laboratory results in order to reduce scr failure rates. However, we observed that current phase I inclusion criteria exclude 44% of pts who are still alive at 6 months.
Clinical trial identification
Editorial acknowledgement
Legal entity responsible for the study
The authors.
Funding
Has not received any funding.
Disclosure
All authors have declared no conflicts of interest.