Tolerability and Safety Study of Recombinant Human Acid Sphingomyelinase in Acid Sphingomyelinase Deficiency Patients

Model scores of the 29,894 trials active now.

Model score 17% 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 5% of the time (4,470 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 2014-01-31. 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
• Sponsor's past termination rate: 14.3% (raises risk) · Sponsor's track record
• Wording of the registration (title, summary, criteria) (raises risk) · Wording Phrases that raised it: patient hastolerabilitythe patientascendingdays prior
  Phrases that lowered it: and safetysafety ofopen labelevaluate thepatients
• Phase: 1 (lowers risk) · Design
• Has a data monitoring committee: Yes (raises risk) · Design
• Minimum age: 18 (raises risk) · Who can join
• Design: single group (raises risk) · Design
• Disease area, cancer: No (lowers risk) · Disease area
• Sponsor type: industry (lowers risk) · Sponsor's track record
• Primary purpose: treatment (raises risk) · Design
• Sponsor's past trials: 182 (lowers risk) · Sponsor's track record
• Drug's FAERS reports marked serious: no FAERS history (raises risk) · Drug's FDA history
• Disease area, neurology: No (lowers risk) · Disease area
• Drug's FAERS reports with a death: no FAERS history (raises risk) · Drug's FDA history
• Trials the sponsor started in the prior 2 years: 14 (lowers risk) · Sponsor's track record
• Number of arms: 1 (raises risk) · Design
• Keywords listed: 1 (raises risk) · Design
• Collaborators: 0 (raises risk) · Design
• Responsible party: sponsor (lowers 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.

• A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency NCT02004704 · started 2013 · scored riskier than 45% of active trials completed
• Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency NCT02004691 · started 2015 · scored riskier than 37% of active trials completed
• Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency NCT02292654 · started 2015 · scored riskier than 64% of active trials completed
• A Study to Evaluate Safety and Tolerability of Olipudase Alfa in Pediatric and Adult Participants With Acid Sphingomyelinase Deficiency (ASMD) Who Completed the DFI12712 or the LTS13632 Study in France NCT06949358 · started 2021 · scored riskier than 47% of active trials completed
• Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency NCT01757184 · started 2013 · scored riskier than 69% 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.