Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease

Model scores of the 29,894 trials active now.

Model score 11% 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 21% of the time (63 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 2022-04-11. 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.

• Accepts healthy volunteers: No (raises risk) · Who can join
• Wording of the registration (title, summary, criteria) (lowers risk) · Wording Phrases that raised it: priorprotocoldiseasein subjectsrandomized double
  Phrases that lowered it: participanttypeparticipantshas receivedto evaluate
• Sponsor's past termination rate: no earlier finished trials (lowers risk) · Sponsor's track record
• Disease area, neurology: Yes (raises risk) · Disease area
• Disease area, cancer: No (lowers risk) · Disease area
• Drug's FAERS reports with a death: 0% (lowers risk) · Drug's FDA history
• Design: single group (raises risk) · Design
• Has a data monitoring committee: Yes (raises risk) · Design
• Phase: 2/3 (raises risk) · Design
• Primary purpose: treatment (raises risk) · Design
• Disease area, psychiatry: Yes (raises risk) · Disease area
• Sponsor type: industry (lowers risk) · Sponsor's track record
• Trials the sponsor started in the prior 2 years: 1 (raises risk) · Sponsor's track record
• Number of arms: 1 (raises risk) · Design
• Sponsor's past trials: 0 (lowers risk) · Sponsor's track record
• Minimum age: 4 (lowers risk) · Who can join
• Drug's FAERS reports marked serious: 100% (raises risk) · Drug's FDA history
• Maximum age: 21 (raises risk) · Who can join
• Responsible party: sponsor (raises risk) · Design
• Blinding: none (raises risk) · Design

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.

• Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1 NCT03879655 · started 2019 · scored riskier than 53% of active trials terminated
• VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease NCT02534844 · started 2015 · scored riskier than 67% of active trials completed
• Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System NCT03643562 · started 2018 · scored riskier than 31% of active trials terminated
• Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1) NCT02939547 · started 2017 · scored riskier than 44% of active trials completed
• Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C NCT02612129 · started 2016 · scored riskier than 30% 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.