Response Requested: Soliciting Interest and Input for Biospecimen Collection in Support of New Methodologies to Detect Bacteria in the Lung

 

This is a time-sensitive Request for Information (RFI) directed at obtaining input prior to finalizing an amendment to the PROMISE study protocol to expand biospecimen collection at the final study visit occurring in 2024. In this well-characterized study cohort, the biospecimens to be collected will be used to enable researchers developing and testing promising novel detection methods focused on identifying pathogenic bacteria present in the lungs. Individuals or groups interested in conducting this research are additionally encouraged to submit descriptions of their proposed work as described below.

 

Obtaining sputum samples in clinics for culture has been increasingly difficult in individuals using elexacaftor/tezacaftor/ivacaftor (ETI or Trikafta®) and has long been challenging in children and adolescents. PROMISE (NCT04038047) is a longitudinal, observational study in adolescents and adults with CF ages 12y and older that was funded through the CFF’s collaborative CRA+ Program. Nearly 500 individuals were enrolled shortly before starting ETI by clinical indication and will be followed for research purposes at multiple timepoints into ETI use. The final study visits (4.5 years after starting ETI) will occur beginning in March 2024 as part of an optional extension of the original study. Over 350 individuals are expected to complete this final visit, and over half of participants will have sputum collected for traditional bacterial culture at the time of the visit.  The bacterial culture results collected within PROMISE, including but not limited to the final visit, will be available along with linkage to the US CFF National Patient Registry Data for all participants regardless of their ability to provide sputum samples for culture during the PROMISE study. This is a highly characterized cohort with research-grade data collection over several years and the potential to link to clinical care data through the national patient registry. This unique opportunity affords ancillary studies the ability to test and compare promising novel detection methods that may reduce the dependence on sputum collection in outpatient clinical settings.

PROMISE has already collected biospecimens (see list below) from participants. These are stored in the CF Foundation Biorepository and will be made available for request by investigators after the primary study conduct and publications are completed. The investigators and sponsor have agreed to expand biospecimen collection at the final visit, if necessary, and prioritize ancillary studies of detection methods, including limited data sharing prior to publication of the final visit results. Requests for remote (home) sample collection will also be considered as part of this RFI. Existing sputum collection is limited in this cohort, and it is unlikely that sputum will be available in sufficient quantity to support informative ancillary research beyond the preplanned microbiology research, but comments are welcome regarding sputum as a necessary biomaterial. Sample types besides sputum that are being collected in PROMISE are listed below, and alternative processing or aliquoting requests will be considered. Additional sample types beyond this list will be considered but may be more challenging to include in an active study protocol. Scientific justification for adding new procedures or sample collection types will be important.

Biospecimens currently collected and stored in some or all participants in PROMISE (≥12y old):
serum, EDTA-plasma, buffy coat, urine, sputum, nasal airway epithelial cells, stool