BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)

Model scores of the 29,894 trials active now.

Model score 15% 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 10% of the time (1,463 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 2016-09-12. 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: screeningonsetsubjectsmonthsuse
  Phrases that lowered it: and safetyefficacy andpatientssafety ofmoderate
• Accepts healthy volunteers: No (raises risk) · Who can join
• Sponsor's past termination rate: 0% (lowers risk) · Sponsor's track record
• Phase: 3 (lowers risk) · Design
• Has a data monitoring committee: Yes (raises risk) · Design
• Design: single group (raises risk) · Design
• Disease area, cancer: No (lowers risk) · Disease area
• Minimum age: 18 (raises risk) · Who can join
• Drug's FAERS reports marked serious: no FAERS history (raises risk) · Drug's FDA history
• Sponsor type: industry (lowers risk) · Sponsor's track record
• Drug's FAERS reports with a death: no FAERS history (raises risk) · Drug's FDA history
• Primary purpose: treatment (raises risk) · Design
• Trials the sponsor started in the prior 2 years: 13 (raises risk) · Sponsor's track record
• Disease area, neurology: No (lowers risk) · Disease area
• Blinding: none (raises risk) · Design
• Sponsor's past trials: 23 (lowers risk) · Sponsor's track record

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.

• Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease NCT01230801 · started 2011 · scored riskier than 41% of active trials completed
• Extension Study for Patients Who Have Participated in a BMN 701 Study NCT01435772 · started 2011 · scored riskier than 42% of active trials terminated
• A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease NCT03729362 · started 2018 · scored riskier than 58% of active trials completed
• A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD) NCT04138277 · started 2019 · scored riskier than 78% of active trials completed
• First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221 NCT02675465 · started 2016 · 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.