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Updated: Jul 9, 2025
Generation of Human Nasal Epithelial Cell Spheroids for Individualized Cystic Fibrosis Transmembrane Conductance Regulator Study
Published on: April 11, 2018
How representative are clinical trial cohorts of the general CF population? Implications for trial planning
Rebecca Dobra1, Sophie Pinnell1, Andy Jones1
1Imperial College London, United Kingdom.
Abstract:
Understanding the number of patients eligible to participate in research is important to design protocols and define research priorities. We reviewed the records of all patients with CF, age 12+, who receive care at our centre. We assessed their eligibility for trial participation based on common trial inclusion/exclusion criteria. 643 patients were included in the analysis, 31 were modulator ineligible(MI). Only 198(31 %) of the total cohort and 7(23 %) of the MI cohort were eligible for participation based on the hypothetical criteria. The most common reason for ineligibility was ppFEV1 ≥90 % followed by clinical instability, complex comorbidity and anticipated inability to adhere to the protocol. We suggest this would be a useful exercise for centres planning to either participate in, or refer subjects into, upcoming trials to undertake for their own cohort. We also make suggestions for protocol designs that optimise the number of patients who are eligible to participate.
Insights
Few cystic fibrosis (CF) patients aged 12+ are eligible for clinical trials. Eligibility criteria, like lung function and clinical stability, significantly limit participation, impacting research design.
Area of Science:
- Medical Research
- Clinical Trial Design
- Cystic Fibrosis Therapeutics
Background:
- Patient eligibility is crucial for designing effective clinical trials and setting research priorities.
- Understanding eligibility rates in cystic fibrosis (CF) research is essential for protocol development.
Purpose of the Study:
- To assess the eligibility of CF patients aged 12 and above for clinical trial participation.
- To identify key factors influencing ineligibility and suggest improvements for trial protocols.
Main Methods:
- Reviewed medical records of 643 CF patients (age 12+) at a single center.
- Assessed eligibility based on common clinical trial inclusion/exclusion criteria.
- Analyzed reasons for ineligibility, including lung function (ppFEV1), clinical stability, comorbidities, and adherence.
Main Results:
- Only 198 (31%) of the total cohort and 7 (23%) of modulator-ineligible patients met hypothetical trial criteria.
- Primary reasons for ineligibility included high ppFEV1 (≥90%), clinical instability, complex comorbidities, and anticipated poor adherence.
- A small subset of 31 patients were identified as modulator ineligible (MI).
Conclusions:
- A significant proportion of CF patients, even those not meeting modulator eligibility, are not eligible for clinical trials.
- Protocol design and eligibility criteria require optimization to increase patient participation in CF research.
- Centers should evaluate their patient cohorts for trial eligibility to inform research planning and subject referral.
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