A Phase 2 Study to Evaluate the Dose and Pharmacodynamic Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy

Model scores of the 29,894 trials active now.

Model score 16% 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 13% of the time (1,304 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 2013-11-30. 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: greaterriskpotentialwillingprocedures
  Phrases that lowered it: kneebodyscreeningsubjectsprior
• Accepts healthy volunteers: No (raises risk) · Who can join
• Sponsor's past termination rate: no earlier finished trials (lowers risk) · Sponsor's track record
• Blinding: quadruple (lowers risk) · Design
• Phase: 2 (raises risk) · Design
• Has a data monitoring committee: Yes (raises risk) · Design
• Design: parallel (raises risk) · Design
• Disease area, cancer: No (lowers risk) · Disease area
• Minimum age: 18 (raises risk) · Who can join
• Drug's FAERS reports with a death: no FAERS history (raises risk) · Drug's FDA history
• Number of arms: 3 (lowers risk) · Design
• Drug's FAERS reports marked serious: no FAERS history (raises risk) · Drug's FDA history
• Sponsor type: industry (lowers risk) · Sponsor's track record
• Disease area, neurology: No (lowers risk) · Disease area
• Primary purpose: treatment (raises risk) · Design
• Collaborators: 0 (raises risk) · Design
• Sponsor's past trials: 0 (lowers risk) · Sponsor's track record
• Maximum age: 65 (lowers risk) · Who can join

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.

• Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM) NCT02377921 · started 2015 · scored riskier than 19% of active trials completed
• Efficacy Confirmation Study of NPC-09 NCT04671472 · started 2021 · scored riskier than 12% of active trials completed
• A Study to Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER; UX001) Tablets in Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy (GNEM) (Also Known as Hereditary Inclusion Body Myopathy [HIBM]) Patients With Severe Ambulatory Impairment NCT02731690 · started 2016 · scored riskier than 32% of active trials terminated
• Safety and Pharmacokinetics of Sialic Acid Tables in Patients With Hereditary Inclusion Body Myopathy (HIBM) NCT01359319 · started 2011 · scored riskier than 40% of active trials completed
• An Open Label Phase 2 Extension Study of Higher Dose Sialic Acid-Extended Release (SA-ER) Tablets and Sialic Acid-Immediate Release (SA-IR) Capsules in Patients With Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy NCT01830972 · started 2013 · scored riskier than 77% 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.