A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function (The PROMISE Study)

Grants and Contracts Details

Description

Human Clinical Trial - The primary objective of this study is to collect physiological and clinical data plus biospecimens from CF patients who are prescribed new CFTR modulator TCT before and after treatment with that modulator. This is a prospective, multi-center observational study. The study is designed to collect specimens from and perform functional measurements on CF subjects who have been prescribed the CFTR modulator TCT. Subjects in the study will have one “before TCT” visit within 30 days before initiation of the therapy and five “after TCT” visits over a 24 month follow-up period. All subjects should be fasting at all study visits. Most participating sites will be divided into sub-study groups; each sub-study group will have specific non-optional procedures conducted in addition to the “Core” procedures. In addition, there are four optional procedures (pH Pill, Transient Elastography, chest CT, and Nasal Cell procurement) that will be offered to subjects at sites with the capability of performing the procedure independent of which sub-study group the site is participating in. (Refer to Study Schematic in protocol section 4.1 and the Schedule of Events in Appendix 1). The duration of participation for each subject is 25 months. Total duration of the study is expected to be 4 years.
StatusActive
Effective start/end date11/1/186/14/23

Funding

  • Seattle Childrens Research Institute: $74,338.00

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