Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy

Model scores of the 29,894 trials active now.

Model score 14% 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 21% of the time (63 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 2017-11-07. 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: type diabetesscreeningplacebodouble blinddiabetes mellitus
  Phrases that lowered it: and safetyefficacy andsafety ofparticipants withthe efficacy
• Accepts healthy volunteers: No (raises risk) · Who can join
• Sponsor's past termination rate: 6.2% (lowers risk) · Sponsor's track record
• Minimum age: 18 (raises risk) · Who can join
• Has a data monitoring committee: Yes (raises risk) · Design
• Blinding: quadruple (lowers risk) · Design
• Disease area, cancer: No (lowers risk) · Disease area
• Disease area, neurology: Yes (raises risk) · Disease area
• Design: parallel (raises risk) · Design
• Maximum age: 82 (lowers risk) · Who can join
• Sponsor's past trials: 16 (lowers risk) · Sponsor's track record
• Primary purpose: treatment (raises risk) · Design
• Trials the sponsor started in the prior 2 years: 6 (raises risk) · Sponsor's track record
• Number of arms: 2 (raises risk) · Design
• Sponsor type: industry (lowers risk) · Sponsor's track record
• Drug's FAERS reports marked serious: no FAERS history (raises risk) · Drug's FDA history
• 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.

• 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
• Extension Study Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP) NCT02175004 · started 2014 · scored riskier than 84% of active trials completed
• 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
• APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis NCT01960348 · started 2013 · scored riskier than 85% of active trials completed
• Study of SOM0226 in Familial Amyloid Polyneuropathy NCT02191826 · started 2014 · scored riskier than 7% 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.