Long-term Safety of Lumacaftor/Ivacaftor in Participants With Cystic Fibrosis Who Are Homozygous for F508del and 12 to <24 Months of Age at Treatment Initiation

Model scores of the 29,894 trials active now.

Model score 11% for any early termination, 1% for termination due to slow enrollment. Past trials like this one (phase, sponsor type and disease area, started 2008–2016) ended terminated 6% of the time (528 trials).

Finished trial that started in 2015 or later: the model never saw it during training.

Cumulative chance of ending terminated (red) or completed (green) by each year from its start; it actually ended on 2023-08-22. The rest of the way to 100% is still running.

From the competing-risks survival model (termination vs completion, still-running trials censored), fit to trials that started 2008–2014 and recalibrated to termination rates over 2022–2024, since termination has become more common. Checked on past years, it still ran a little low when the rise continued.

• Accepts healthy volunteers: No (raises risk) · Who can join
• Sponsor's past termination rate: 12.3% (raises risk) · Sponsor's track record
• Phase: 3 (lowers risk) · Design
• Drug's FAERS reports with a death: 2.1% (lowers risk) · Drug's FDA history
• Has a data monitoring committee: not given (lowers risk) · Design
• Design: single group (raises risk) · Design
• Drug's FAERS reports marked serious: 61.4% (lowers risk) · Drug's FDA history
• Trials the sponsor started in the prior 2 years: 24 (lowers risk) · Sponsor's track record
• Sponsor type: industry (lowers risk) · Sponsor's track record
• Wording of the registration (title, summary, criteria) (raises risk) · Wording Phrases that raised it: transplantationkeylong termterm safetythe long
  Phrases that lowered it: participantsopen labelevaluate theto evaluateinterfere with
• Disease area, cancer: No (lowers risk) · Disease area
• Primary purpose: treatment (raises risk) · Design
• Disease area, neurology: No (lowers risk) · Disease area
• Disease area, respiratory: Yes (raises risk) · Disease area
• Sponsor's past trials: 106 (lowers risk) · Sponsor's track record

Every input that moved this score, largest first: SHAP contributions in log-odds, red raising risk and green lowering it. They describe the model, not causes of termination. Phrases are traced from the text features back to this registration's words, approximately.

• Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor NCT02544451 · started 2015 · scored riskier than 24% of active trials completed
• A Phase 3 Rollover Study of Lumacaftor in Combination With Ivacaftor in Subjects 12 Years and Older With Cystic Fibrosis NCT01931839 · started 2013 · scored riskier than 17% of active trials completed
• A Rollover Safety Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation NCT03125395 · started 2017 · scored riskier than 22% of active trials completed
• A Study to Explore the Impact of Lumacaftor/Ivacaftor on Disease Progression in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508del NCT03625466 · started 2018 · scored riskier than 22% of active trials completed
• Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Participants 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del NCT03601637 · started 2018 · scored riskier than 16% of active trials completed

Closest by meaning of the registration text (PubMedBERT embeddings), among finished trials. How they ended is context, not an input to this trial's score.