Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1

Model scores of the 29,894 trials active now.

Model score 18% for any early termination, 4% for termination due to slow enrollment. Past trials like this one (phase, sponsor type and disease area, started 2008–2016) ended terminated 8% of the time (544 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 2017-12-15. 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: treatment ofpressurethe followingpurpose ofmonths of
  Phrases that lowered it: trialpatientsdaypositivemg
• Accepts healthy volunteers: No (raises risk) · Who can join
• Phase: 1 (lowers risk) · Design
• Sponsor's past termination rate: no earlier finished trials (lowers risk) · Sponsor's track record
• Design: single group (raises risk) · Design
• Disease area, neurology: Yes (raises risk) · Disease area
• Maximum age: 0.5 (raises risk) · Who can join
• Has a data monitoring committee: Yes (raises risk) · Design
• Trials the sponsor started in the prior 2 years: 0 (raises risk) · Sponsor's track record
• Disease area, cancer: No (lowers risk) · Disease area
• Primary purpose: treatment (raises 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
• Blinding: none (raises risk) · Design
• Number of arms: 2 (raises risk) · Design
• Minimum age: not given (lowers risk) · Who can join
• Keywords listed: 6 (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.

• Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1 NCT03461289 · started 2018 · scored riskier than 27% of active trials completed
• Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1 NCT03306277 · started 2017 · scored riskier than 31% of active trials completed
• Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies NCT03837184 · started 2019 · scored riskier than 29% of active trials completed
• Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy NCT03381729 · started 2017 · scored riskier than 71% of active trials terminated
• Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2 NCT03505099 · started 2018 · scored riskier than 44% 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.