An Open Label Phase 2 Study of ManNAc in Subjects With GNE Myopathy

Model scores of the 29,894 trials active now.

Model score 20% for any early termination, 6% for termination due to slow enrollment. Past trials like this one (phase, sponsor type and disease area, started 2008–2016) ended terminated 22% of the time (102 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 2018-11-15. 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: subject hasequal toor equalcellcells
  Phrases that lowered it: predictedkneehepaticpulmonaryupper
• Accepts healthy volunteers: No (raises risk) · Who can join
• Phase: 2 (raises risk) · Design
• Design: single group (raises risk) · Design
• Has a data monitoring committee: Yes (raises risk) · Design
• Sponsor's past termination rate: 7.7% (raises risk) · Sponsor's track record
• Disease area, cancer: No (lowers risk) · Disease area
• Minimum age: 18 (raises risk) · Who can join
• Maximum age: 60 (lowers risk) · Who can join
• Drug's FAERS reports with a death: no FAERS history (raises risk) · Drug's FDA history
• Primary purpose: treatment (raises risk) · Design
• Drug's FAERS reports marked serious: no FAERS history (raises risk) · Drug's FDA history
• Collaborators: 1 (raises risk) · Design
• Trials the sponsor started in the prior 2 years: 2 (raises risk) · Sponsor's track record
• Number of arms: 2 (raises risk) · Design
• Sponsor type: government (lowers risk) · Sponsor's track record
• Responsible party: sponsor (lowers risk) · Design
• Blinding: none (raises risk) · Design
• Sponsor's past trials: 13 (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.

• Phase I Clinical Trial of ManNAc in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM) NCT01634750 · started 2012 · scored riskier than 16% of active trials completed
• Efficacy Confirmation Study of NPC-09 NCT04671472 · started 2021 · scored riskier than 12% of active trials completed
• 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 NCT01517880 · started 2012 · scored riskier than 46% of active trials completed
• 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
• 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

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.