A Study to Evaluate Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)

Model scores of the 29,894 trials active now.

Model score 15% 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 10% of the time (139 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 2020-10-09. 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.

• Wording of the registration (title, summary, criteria) (raises risk) · Wording Phrases that raised it: placebowomenmonths priorsubjectsinjury
  Phrases that lowered it: participantsstem celldosedaily28 days
• Accepts healthy volunteers: No (raises risk) · Who can join
• Phase: 3 (lowers risk) · Design
• Has a data monitoring committee: Yes (raises risk) · Design
• Blinding: triple (raises risk) · Design
• Design: parallel (raises risk) · Design
• Drug's FAERS reports with a death: no FAERS history (raises risk) · Drug's FDA history
• Disease area, cancer: No (lowers risk) · Disease area
• Sponsor type: industry (lowers risk) · Sponsor's track record
• Minimum age: 18 (raises risk) · Who can join
• Drug's FAERS reports marked serious: no FAERS history (raises risk) · Drug's FDA history
• Primary purpose: treatment (raises risk) · Design
• Number of arms: 4 (lowers risk) · Design
• Sponsor's past termination rate: 7.7% (lowers risk) · Sponsor's track record
• Trials the sponsor started in the prior 2 years: 6 (raises risk) · Sponsor's track record
• Sponsor's past trials: 13 (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.

• A Study Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD) NCT03559699 · started 2018 · scored riskier than 23% of active trials completed
• A Study of AG-348 in Adult Participants With Pyruvate Kinase (PK) Deficiency NCT02476916 · started 2015 · scored riskier than 87% of active trials completed
• Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007 NCT03853798 · started 2019 · scored riskier than 51% of active trials completed
• A Safety and Tolerability Study of AG-519 in Healthy Subjects NCT02630927 · started 2015 · scored riskier than 1% of active trials terminated
• A Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-946 in Healthy Volunteers and in Participants With Sickle Cell Disease NCT04536792 · started 2020 · scored riskier than 9% 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.