A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)

Model scores of the 29,894 trials active now.

Model score 11% 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 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 2016-05-31. 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.

• Wording of the registration (title, summary, criteria) (raises risk) · Wording Phrases that raised it: subjectsubjectstimedonormajor
  Phrases that lowered it: crossoverefficacyand safetyparticipantspulmonary
• Design: crossover (lowers risk) · Design
• Sponsor's past termination rate: 0% (lowers risk) · Sponsor's track record
• Accepts healthy volunteers: No (raises risk) · Who can join
• Phase: 3 (lowers risk) · Design
• Has a data monitoring committee: Yes (raises risk) · Design
• Blinding: quadruple (lowers risk) · Design
• Disease area, cancer: No (lowers risk) · Disease area
• Number of arms: 4 (lowers risk) · Design
• Sponsor type: industry (lowers risk) · Sponsor's track record
• Drug's FAERS reports marked serious: no FAERS history (raises risk) · Drug's FDA history
• 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: 5 (raises risk) · Sponsor's track record
• Disease area, dermatology: Yes (raises risk) · Disease area
• Primary purpose: treatment (raises risk) · Design
• Disease area, neurology: No (lowers risk) · Disease area

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 Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) NCT02432144 · started 2015 · scored riskier than 79% 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
• A Study of JR-141 in Patients With Mucopolysaccharidosis II (BR21) NCT03359213 · started 2018 · scored riskier than 18% 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.