EXPECT-1
Research type
Research Study
Full title
Pilot Study to Assess Success Factors and Barriers for Preparation of a Phase 3 Study with ExPEC10V in Adults Aged 60 Years or Older and in Stable Health
IRAS ID
266775
Contact name
Christopher Butler
Contact email
Sponsor organisation
University Medical Centre Utrecht
Duration of Study in the UK
1 years, 3 months, 31 days
Research summary
Research Summary
Extraintestinal pathogenic Escherichia coli (ExPEC) is the most common and\nrising cause of bacteremia and bloodstream infections worldwide. Increasing multidrug\nresistance among ExPEC strains constitutes a major challenge for the prevention and\ntreatment of ExPEC infections. Although Invasive ExPEC Disease (IED) affects all age categories,adults aged 60 years or older have an increased risk of developing IED. To date, there is no vaccine available to prevent IED. ExPEC10V is a vaccine candidate in development by Janssen R&D for the prevention of IED in adults aged 60 years and older. A Phase 3 clinical study is planned to investigate the efficacy and safety of this vaccine. To obtain insight in the feasibility and design of the Phase 3 study, a pilot study is required.\n\nParticipants will be followed for 12 months from point of study entry. Their medical history will be recorded and participants will be contacted approximately every four months in the 12 month period to enquire about healthcare usage and any hospitalisations. Participants will also be invited to contact the research team to inform them of any hospitalisations. If hospitalised further data will be collected from their medical records and laboratory reports. At the end of the 12 month period the participant’s medical notes will be reviewed. This study will be conducted at GP practices and their associated local hospital. \n\nThis study is part of the COMBACTE NET programme funded by Innovative Medicines Initiative Joint Undertaking which are composed of financial contribution from the European Union Seventh Framework Programme (FP7/2007-2013) and EFPIA (European Federation of Pharmaceutical Industries and Association\n
Summary of Results
Overall, counting from the first participant enrolled, 27.4% of all participants (4,470) were enrolled in 3 months’ time, 78.4% in 6 months’ time, and 100.0% in 9 months’ time. This suggests that enrolling participants with or without an history of UTI in the last 10 years in a Phase 3 efficacy trial is feasible. However, the findings on the enrollment rate of this study cannot be reliably extrapolated to the ExPEC Phase 3 pivotal efficacy clinical trial due to differences in the target study population (ExPEC Phase 3 pivotal efficacy clinical trial will enroll participants with an history of UTI in the past 2 years) and in the study design
(EXPECT-1 is non-interventional, with no vaccine given, while the EXPEC Phase 3 pivotal efficacy clinical trial will be interventional, with a EXPEC vaccine administered to participants).
• Spain was able to enroll more than the target of 750 participants and enrolled 988 participants in 3 months’ time. Japan enrolled 732 participants in 3 months’ time but reached the target of
750 participants in 6 months’ time.
• Eighty-three (1.9%) participants discontinued from the study prematurely. The reasons for discontinuation were withdrawal by subject for 42 (50.6%) participants, death for 32 (38.6%) participants, decision by the investigator for 3 (3.6%) participants, and lost to follow-up, screen failure, and discontinuation due to other reasons for 2 (2.4%) participants each. In total,
32 participants died during the study; no participants died due to IED. Notably, 1 participant died due to epileptic crisis/urinary sepsis and 1 participant died due to septic shock. Two deaths were COVID-19 related.
• The majority of participants reported a medical encounter during the 1-year follow-up period:
3,759 (84.1%) when reported by the GP/PC center and 3,480 (77.9%) when reported by the participants. The most common types of medical encounters (>10% of participants) were GP/family doctor, hospital outpatient department, (para-)medical services, emergency room, and hospital inpatient department. In general, the frequencies of the different types reported over all participants were similar when reported by the GP/PC centers versus reported by the participants. The values of kappa of the medical encounters (5 types) varied from 0.31 to 0.65, further indicating a fair to moderate agreement between the medical encounters reported by the GP/PC center and the participant.
• The number of hospital admissions during the 1-year follow-up period were similar when reported by the GP/PC centers versus the participants: The number of hospital admissions was
2,529 (56.6%) participants reported by the GP/PC center and 2,177 (48.7%) participants reported by the participants.REC name
Yorkshire & The Humber - Leeds East Research Ethics Committee
REC reference
19/YH/0264
Date of REC Opinion
8 Aug 2019
REC opinion
Further Information Favourable Opinion