Trikafta in Cystic Fibrosis Patients
Summary
This clinical study will enroll 42 participants without the F508del mutation, carrying partial function or N1303K mutations not approved for Trikafta, and who are not expected to be approved for CFTR modulator treatment in the immediate future. Each participant will be given Trikafta for approximately four weeks. The study researchers will monitor clinical endpoints that include forced expiratory volume (FEV1) and sweat chloride. Additionally, the researchers will obtain skin biopsy material and/or blood sample from each subject so that induced pluripotent stem (iPS) cells can be modified into airway cell monolayers and tested for response to Trikafta. In this way, the study will evaluate an emerging and readily accessible in vitro endpoint as a predictor of clinical response. This study will serve as a pilot/test case for other clinical protocols relevant to patients with rare CFTR variants who do not currently receive modulator therapies.
Timeline
- Start
- 2019-09-04
- Primary completion
- 2024-02-13
- Completion
- 2024-02-13
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Ivacaftor | Other / unclassified | 75 mg | Oral |
| Subject | Ivacaftor | Other / unclassified | 150 mg | Oral |
| Subject | elexacaftor | Small molecule | 100 mg | Oral |
| Subject | tezacaftor | Other / unclassified | 50 mg | Oral |