Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN) and Patients With Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM)

Model scores of the 29,894 trials active now.

Model score 16% for any early termination, 2% for termination due to slow enrollment. Past trials like this one (phase, sponsor type and disease area, started 2008–2016) ended terminated 7% of the time (318 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 2025-09-12. 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: heartsafety tolerabilityfailuretolerability pharmacokineticscardiomyopathy
  Phrases that lowered it: open labelpatientsevaluatepharmacokineticslabel single
• 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
• Drug's FAERS reports with a death: no FAERS history (raises risk) · Drug's FDA history
• Disease area, neurology: Yes (raises risk) · Disease area
• Number of arms: 5 (lowers risk) · Design
• Drug's FAERS reports marked serious: no FAERS history (raises risk) · Drug's FDA history
• Minimum age: 18 (raises risk) · Who can join
• Primary purpose: treatment (raises risk) · Design
• Maximum age: 90 (lowers risk) · Who can join
• Disease area, cancer: No (lowers risk) · Disease area
• Sponsor's past trials: 0 (lowers risk) · Sponsor's track record
• Trials the sponsor started in the prior 2 years: 0 (raises risk) · Sponsor's track record
• Sponsor type: industry (lowers risk) · Sponsor's track record
• Disease area, cardiovascular: Yes (raises risk) · Disease area
• Collaborators: 0 (raises risk) · Design

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.

• First-in-Human Study of NI006 in Patients With Amyloid Transthyretin Cardiomyopathy NCT04360434 · started 2020 · scored riskier than 93% of active trials completed
• NEURO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy NCT04136184 · started 2019 · scored riskier than 19% of active trials completed
• Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Cardiomyopathy NCT03860935 · started 2019 · scored riskier than 85% of active trials completed
• A Study of PRX004 in Subjects With Amyloid Transthyretin (ATTR) Amyloidosis NCT03336580 · started 2018 · scored riskier than 86% of active trials terminated
• 24 Month Open Label Study of the Tolerability and Efficacy of Inotersen in TTR Amyloid Cardiomyopathy Patients NCT03702829 · started 2019 · scored riskier than 98% of active trials terminated

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.