Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU

Model scores of the 29,894 trials active now.

Model score 13% for any early termination, 1% 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 2019-02-05. 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: 0% (lowers risk) · Sponsor's track record
• Disease area, neurology: Yes (raises risk) · Disease area
• Has a data monitoring committee: Yes (raises risk) · Design
• Design: parallel (raises risk) · Design
• Phase: 3 (lowers risk) · Design
• Disease area, cancer: No (lowers risk) · Disease area
• Number of arms: 4 (lowers risk) · Design
• Wording of the registration (title, summary, criteria) (raises risk) · Wording Phrases that raised it: stablescalesubjectto screeningtransplantation
  Phrases that lowered it: efficacyand safetysafety ofevaluate theto evaluate
• Minimum age: 18 (raises risk) · Who can join
• Blinding: triple (raises risk) · Design
• Primary purpose: treatment (raises risk) · Design
• Sponsor's past trials: 22 (lowers risk) · Sponsor's track record
• Sponsor type: industry (lowers risk) · Sponsor's track record
• Drug's FAERS reports with a death: 14.3% (lowers risk) · Drug's FDA history
• Trials the sponsor started in the prior 2 years: 11 (raises risk) · Sponsor's track record
• Drug's FAERS reports marked serious: 76.7% (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.

• An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165 NCT01819727 · started 2013 · scored riskier than 39% of active trials completed
• Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU NCT03694353 · started 2018 · scored riskier than 73% of active trials completed
• Safety and Tolerability of SYNB1618 in Healthy Adult Volunteers and Adult Subjects With Phenylketonuria NCT03516487 · started 2018 · scored riskier than 5% of active trials completed
• A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks NCT01560286 · started 2012 · scored riskier than 13% of active trials completed
• Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU NCT00925054 · started 2009 · 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.