Gene Therapy for Pyruvate Kinase Deficiency (PKD)

Model scores of the 29,894 trials active now.

Model score 20% for any early termination, 3% for termination due to slow enrollment. Past trials like this one (phase, sponsor type and disease area, started 2008–2016) ended terminated 14% of the time (117 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 2025-06-09. 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: mriimaginganemiairondl
  Phrases that lowered it: evaluate thesafety ofto evaluatetrial toopen label
• Accepts healthy volunteers: No (raises risk) · Who can join
• Sponsor's past termination rate: no earlier finished trials (lowers risk) · Sponsor's track record
• Phase: 1 (lowers risk) · Design
• Has a data monitoring committee: Yes (raises risk) · Design
• Trials the sponsor started in the prior 2 years: 4 (raises risk) · Sponsor's track record
• Design: sequential (lowers risk) · Design
• Disease area, cancer: No (lowers risk) · Disease area
• Primary purpose: treatment (raises risk) · Design
• Drug's FAERS reports with a death: no FAERS history (raises risk) · Drug's FDA history
• Number of arms: 1 (raises risk) · Design
• Maximum age: 50 (lowers risk) · Who can join
• Drug's FAERS reports marked serious: no FAERS history (raises risk) · Drug's FDA history

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 of AG-348 in Adult Participants With Pyruvate Kinase (PK) Deficiency NCT02476916 · started 2015 · scored riskier than 87% of active trials completed
• 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
• Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Three-times Weekly Dosing of GSK1278863 in Hemodialysis-dependent Subjects With Anemia Associated With Chronic Kidney Disease Who Are Switched From a Stable Dose of an Erythropoiesis-stimulating Agent NCT02689206 · started 2016 · scored riskier than 44% of active trials completed
• An Efficacy and Safety Study of Fedratinib Compared to Best Available Therapy in Subjects With DIPSS-intermediate or High-risk Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis, or Post-essential Thrombocythemia Myelofibrosis and Previously Treated With Ruxolitinib NCT03952039 · started 2019 · scored riskier than 62% of active trials completed
• Sorafenib and Nivolumab in Treating Participants With Unresectable, Locally Advanced or Metastatic Liver Cancer NCT03439891 · started 2018 · scored riskier than 84% 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.