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NCT03506061 · ClinicalTrials.gov registry record · Phase 2
Trikafta in Cystic Fibrosis Patients
A Phase 2 study, sponsored by Emory University.
- Completed
- Registry status
- Phase 2
- Development phase
- 42
- Enrollment target
NCT03506061: Completed Phase 2 study, sponsored by Emory University.
NCT03506061 is a Phase 2 study that has completed, run by Emory University. The registered enrollment target is 42 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (68% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03506061, a Phase 2 study, has completed, sponsored by Emory University.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 42 participants
- Enrollment target
Study 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.
Primary Outcome
FEV1 provides a direct measurement of patient health among individuals with cystic fibrosis and declines in FEV1 are associated with poor outcomes among those with CF. FEV1 is measured by spirometry and is the maximum amount of air the participant can blow out in one second.
Interventions
- DRUG Trikafta
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 42 participants |
| Start Date | 2019-09-04 |
| Est. Completion | 2024-02-13 |
| Phase | Phase 2 |
What the finished NCT03506061 record still lists
NCT03506061 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 42 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (68% lower).
The record links to 0 conditions, and to 1 intervention - of which Trikafta is the first listed.
NCT03506061 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT03506061 about?
NCT03506061 is a clinical study titled "Trikafta in Cystic Fibrosis Patients". 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 approximate...
What is the current status of trial NCT03506061?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 42 participants. The study started on 2019-09-04. Estimated completion is 2024-02-13.
What interventions are being tested in trial NCT03506061?
The interventions under investigation include: Trikafta (DRUG).
Who is sponsoring clinical trial NCT03506061?
This trial is sponsored by Emory University, which has 1,208 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT03506061's enrollment target sits among peer trials
42 1339th of 2000 higher than 649 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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