A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)

Model scores of the 29,894 trials active now.

Model score 22% 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 9% of the time (803 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 2019-01-14. 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: the subjectdonortransplantsubjectslong term
  Phrases that lowered it: safety ofto evaluateevaluate thetreatment naïveaged
• Accepts healthy volunteers: No (raises risk) · Who can join
• Sponsor's past termination rate: 0% (lowers risk) · Sponsor's track record
• Phase: 3 (lowers risk) · Design
• Design: single group (raises risk) · Design
• 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: 8 (raises risk) · Sponsor's track record
• Has a data monitoring committee: No (lowers risk) · Design
• Drug's FAERS reports marked serious: no FAERS history (raises risk) · Drug's FDA history
• Disease area, cancer: No (lowers risk) · Disease area
• Primary purpose: treatment (raises risk) · Design
• Number of arms: 1 (raises risk) · Design
• Disease area, neurology: No (lowers risk) · Disease area
• Sponsor type: industry (lowers risk) · Sponsor's track record
• Sponsor's past trials: 5 (raises risk) · Sponsor's track record
• Maximum age: not given (raises risk) · Who can join
• Drug's FAERS reports in the prior 12 months: 0 (raises 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.

• An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of Study Drug UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7) NCT01856218 · started 2013 · scored riskier than 76% of active trials completed
• A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7) NCT02230566 · started 2014 · scored riskier than 19% of active trials completed
• Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age NCT02418455 · started 2015 · scored riskier than 75% of active trials completed
• A Study in MPS VI to Assess Safety and Efficacy of Odiparcil NCT03370653 · started 2017 · scored riskier than 46% of active trials completed
• Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease NCT02060526 · started 2014 · scored riskier than 95% 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.