Extension Study Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP)

Model scores of the 29,894 trials active now.

Model score 23% 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 15% of the time (734 trials).

Finished trial that started 2008–2014: scored by a model trained without any trial from its start year, so its own outcome was never used.

Cumulative chance of ending terminated (red) or completed (green) by each year from its start; it actually ended on 2021-01-07. 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: term safetylong termextension studythe longlabel extension
  Phrases that lowered it: efficacysafetystudy tolabeldosing
• Accepts healthy volunteers: No (raises risk) · Who can join
• Phase: 3 (lowers risk) · Design
• Sponsor's past termination rate: 9.5% (raises risk) · Sponsor's track record
• Has a data monitoring committee: Yes (raises risk) · Design
• Disease area, neurology: Yes (raises risk) · Disease area
• Design: parallel (raises risk) · Design
• Drug's FAERS reports with a death: no FAERS history (raises risk) · Drug's FDA history
• Drug's FAERS reports marked serious: no FAERS history (raises risk) · Drug's FDA history
• Minimum age: 18 (raises risk) · Who can join
• Trials the sponsor started in the prior 2 years: 7 (lowers risk) · Sponsor's track record
• Blinding: none (raises risk) · Design
• Disease area, cancer: No (lowers risk) · Disease area
• Sponsor type: industry (lowers risk) · Sponsor's track record
• Primary purpose: treatment (raises risk) · Design
• Sponsor's past trials: 21 (lowers risk) · Sponsor's track record
• Keywords listed: 5 (lowers risk) · Design
• Drug's FAERS reports before start: 0 (raises risk) · Drug's FDA history
• Responsible party: sponsor (lowers 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.

• The Study of ALN-TTR02 (Patisiran) for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Who Have Already Been Treated With ALN-TTR02 (Patisiran) NCT01961921 · started 2013 · scored riskier than 90% of active trials completed
• Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy NCT01737398 · started 2013 · scored riskier than 37% of active trials completed
• The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Participants Who Have Already Been Treated With ALN-TTR02 (Patisiran) NCT02510261 · started 2015 · scored riskier than 71% of active trials completed
• The Effects of Fx-1006A on Transthyretin Stabilization and Clinical Outcome Measures in Patients With Non-V30M Transthyretin Amyloidosis NCT00630864 · started 2008 · scored riskier than 82% 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

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.