Efficacy and Safety of HPN-100 for the Treatment of Adults With Urea Cycle Disorders

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 15% of the time (734 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 2010-09-30. 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.

• Design: crossover (lowers risk) · Design
• Accepts healthy volunteers: No (raises risk) · Who can join
• Wording of the registration (title, summary, criteria) (raises risk) · Wording Phrases that raised it: subjectstablepotentialscreeningto day
  Phrases that lowered it: and safetyefficacy andsafety of14 dayscycle
• Phase: 3 (lowers risk) · Design
• Sponsor's past termination rate: 6.2% (lowers risk) · Sponsor's track record
• Drug's FAERS reports with a death: 43.8% (raises risk) · Drug's FDA history
• Disease area, neurology: Yes (raises risk) · Disease area
• Has a data monitoring committee: Yes (raises risk) · Design
• Trials the sponsor started in the prior 2 years: 66 (lowers risk) · Sponsor's track record
• Disease area, cancer: No (lowers risk) · Disease area
• Sponsor type: industry (lowers risk) · Sponsor's track record
• Drug's FAERS reports marked serious: 93.8% (raises risk) · Drug's FDA history
• Minimum age: 18 (raises risk) · Who can join
• Primary purpose: treatment (raises risk) · Design
• Sponsor's past trials: 162 (lowers risk) · Sponsor's track record
• Number of arms: 2 (raises risk) · Design
• Responsible party: sponsor (lowers risk) · Design
• Drug's FAERS reports before start: 16 (lowers 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.

• Study of the Safety and Tolerability of HPN-100 Compared to Sodium Phenylbutyrate in Children With Urea Cycle Disorders NCT00947544 · started 2010 · scored riskier than 61% of active trials completed
• Study of the Safety of HPN (Hyperion)-100 for the Long-Term Treatment of Urea Cycle Disorders (Treat UCD) NCT00947297 · started 2009 · scored riskier than 62% of active trials completed
• Study of the Safety, Pharmacokinetics and Efficacy of HPN-100, in Pediatric Subjects With Urea Cycle Disorders (UCDs) NCT01347073 · started 2011 · scored riskier than 48% of active trials completed
• Arginine and Buphenyl in Patients With Argininosuccinic Aciduria (ASA), a Urea Cycle Disorder NCT00345605 · started 2008 · scored riskier than 46% of active trials completed
• UNLOCKED: A Phase 2, Open-label Trial With KB195 in Subjects With a Urea Cycle Disorder NCT03933410 · started 2019 · scored riskier than 21% 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.