Gene Therapy in Patients With Mucopolysaccharidosis Disease

Model scores of the 29,894 trials active now.

Model score 17% 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 12% of the time (56 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 2024-07-16. 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: subjectadministrationchronicsubjects within subjects
  Phrases that lowered it: evaluate theto evaluatethe aimefficacyaim of
• Accepts healthy volunteers: No (raises risk) · Who can join
• Phase: 1/2 (raises risk) · Design
• Sponsor's past termination rate: no earlier finished trials (lowers risk) · Sponsor's track record
• Design: single group (raises risk) · Design
• Has a data monitoring committee: not given (lowers 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
• Sponsor type: academic or other (raises risk) · Sponsor's track record
• Drug's FAERS reports with a death: no FAERS history (raises risk) · Drug's FDA history
• Primary purpose: treatment (raises risk) · Design
• Number of arms: 1 (raises risk) · Design
• Drug's FAERS reports marked serious: no FAERS history (raises risk) · Drug's FDA history
• Disease area, dermatology: Yes (raises risk) · Disease area
• Blinding: none (raises risk) · Design
• Maximum age: 65 (lowers risk) · Who can join
• Sponsor's past trials: 0 (lowers risk) · Sponsor's track record
• Disease area, neurology: No (lowers risk) · Disease area
• Collaborators: 0 (raises 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.

• Gene Transfer Clinical Trial for Mucopolysaccharidosis (MPS) IIIB NCT03315182 · started 2017 · scored riskier than 60% of active trials terminated
• Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease NCT04088734 · started 2019 · scored riskier than 71% of active trials terminated
• Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1 NCT03306277 · started 2017 · scored riskier than 31% of active trials completed
• Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I NCT02702115 · started 2017 · scored riskier than 20% of active trials terminated
• Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II NCT03041324 · started 2017 · scored riskier than 31% of active trials terminated

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.