Phase I Clinical Trial of ManNAc in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)

Model scores of the 29,894 trials active now.

Model score 10% for any early termination, 2% for termination due to slow enrollment. Past trials like this one (phase, sponsor type and disease area, started 2008–2016) ended terminated 7% of the time (106 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 2013-05-29. 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.

• Sponsor's past termination rate: 0% (lowers risk) · Sponsor's track record
• Accepts healthy volunteers: No (raises risk) · Who can join
• Phase: 1 (lowers risk) · Design
• Wording of the registration (title, summary, criteria) (raises risk) · Wording Phrases that raised it: subject hasdiagnosissubject isto randomizationdisease
  Phrases that lowered it: participantsevaluate theto evaluatepharmacokineticssafety of
• Has a data monitoring committee: not given (lowers risk) · Design
• Collaborators: 2 (lowers risk) · Design
• Design: parallel (raises risk) · Design
• Disease area, cancer: No (lowers risk) · Disease area
• Minimum age: 18 (raises risk) · Who can join
• Primary purpose: treatment (raises risk) · Design
• Sponsor's past trials: 10 (raises risk) · Sponsor's track record
• Maximum age: 70 (lowers risk) · Who can join
• Drug's FAERS reports marked serious: no FAERS history (raises risk) · Drug's FDA history
• Trials the sponsor started in the prior 2 years: 2 (raises risk) · Sponsor's track record
• Blinding: double (raises risk) · Design
• Drug's FAERS reports with a death: no FAERS history (raises risk) · Drug's FDA history
• Disease area, neurology: No (lowers risk) · Disease area
• Sponsor type: government (raises 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.

• An Open Label Phase 2 Study of ManNAc in Subjects With GNE Myopathy NCT02346461 · started 2015 · scored riskier than 73% 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.