Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor for 24 Weeks or Longer in People with Cystic Fibrosis and One or More <i>F508del</i> Alleles: Interim Results of an Open-Label Phase 3 Clinical Trial
Bibliographic record
Abstract
Safety results from this interim analysis were consistent with the initial 24-week placebo-controlled F/MF pivotal study, with similar or lower exposure-adjusted event rates observed in the OLE (Table 1) (5). ELX/TEZ/IVA was generally safe and well tolerated. Most AEs were consistent with common manifestations of CF and were not treatment limiting (3, 10). In participants who received ELX/TEZ/IVA in parent studies, improvements in efficacy and PD measures, including ppFEV1, SwCl concentration, BMI, CFQ-R RD score, and PEx event rate, were maintained or continued to improve further over 24 weeks (F/MF genotypes) or 36 weeks (F/F genotype) of additional treatment. These results validate the durability of ELX/TEZ/IVA efficacy responses, with no emerging safety concerns. Among participants who had received placebo or TEZ/IVA in the respective parent studies, initiation of ELX/TEZ/IVA rapidly led to marked improvements in these efficacy measures that were consistent with the results seen in the ELX/TEZ/IVA arms of those parent studies. Thus, the results of this combined-group interim analysis demonstrate the safety and sustained efficacy of long-term ELX/TEZ/IVA treatment in pwCF 12 years old or older with one or more F508del alleles.
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How this classification was reachedexpand
Full frame distilled prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.
Codex and Gemma teacher scores by category
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.002 | 0.018 |
| Meta-epidemiology (narrow) | 0.001 | 0.000 |
| Meta-epidemiology (broad) | 0.004 | 0.000 |
| Bibliometrics | 0.000 | 0.001 |
| Science and technology studies | 0.000 | 0.005 |
| Scholarly communication | 0.000 | 0.000 |
| Open science | 0.001 | 0.000 |
| Research integrity | 0.000 | 0.002 |
| Insufficient payload (model declined to judge) | 0.000 | 0.000 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one teacher head, not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".