A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy

Model scores of the 29,894 trials active now.

Model score 10% 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 19% of the time (731 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 2026-05-04. 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) (lowers risk) · Wording Phrases that raised it: first doseinfantslifelongerwhichever is
  Phrases that lowered it: and safetythe pharmacokineticssafety ofblood pressurescreening
• Accepts healthy volunteers: No (raises risk) · Who can join
• Sponsor's past termination rate: 11.6% (raises risk) · Sponsor's track record
• Phase: 2 (raises risk) · Design
• Has a data monitoring committee: not given (lowers risk) · Design
• Sponsor's past trials: 1342 (raises risk) · Sponsor's track record
• Drug's FAERS reports marked serious: 50.7% (lowers risk) · Drug's FDA history
• Disease area, neurology: Yes (raises risk) · Disease area
• Design: single group (raises risk) · Design
• Trials the sponsor started in the prior 2 years: 71 (lowers risk) · Sponsor's track record
• Drug's FAERS reports with a death: 10.3% (lowers risk) · Drug's FDA history
• Disease area, cancer: No (lowers risk) · Disease area
• Minimum age: not given (raises risk) · Who can join
• Sponsor type: industry (lowers risk) · Sponsor's track record
• Primary purpose: treatment (raises risk) · Design
• Maximum age: 0.05 (raises risk) · Who can join
• Blinding: none (lowers risk) · Design
• Drug's FAERS reports before start: 2213 (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 to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants NCT02908685 · started 2016 · scored riskier than 47% of active trials completed
• Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy NCT02913482 · started 2016 · scored riskier than 44% of active trials completed
• A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy NCT03032172 · started 2017 · scored riskier than 39% of active trials completed
• An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA) NCT02268552 · started 2015 · scored riskier than 92% of active trials completed
• An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) NCT01494701 · started 2011 · scored riskier than 81% 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.