Long-Term Evaluation of BIIB067 (Tofersen)

Model scores of the 29,894 trials active now.

Model score 28% 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 10% of the time (1,463 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 2024-08-12. 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: sclerosistolerabilitykeylongterm safety
  Phrases that lowered it: plateletcountparticipantbleedinginvestigator
• Accepts healthy volunteers: No (raises risk) · Who can join
• Sponsor's past termination rate: 18.8% (raises risk) · Sponsor's track record
• Drug's FAERS reports with a death: no FAERS history (raises risk) · Drug's FDA history
• Has a data monitoring committee: Yes (raises risk) · Design
• Trials the sponsor started in the prior 2 years: 48 (lowers risk) · Sponsor's track record
• Phase: 3 (lowers risk) · Design
• Drug's FAERS reports marked serious: no FAERS history (raises risk) · Drug's FDA history
• Minimum age: 18 (raises risk) · Who can join
• Primary purpose: treatment (raises risk) · Design
• Sponsor's past trials: 223 (lowers risk) · Sponsor's track record
• Drug's FAERS reports before start: 0 (raises risk) · Drug's FDA history
• Design: sequential (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 Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of BIIB067 (Tofersen) in Adults With Inherited Amyotrophic Lateral Sclerosis (ALS) NCT02623699 · started 2016 · scored riskier than 39% of active trials completed
• Study to Assess the Safety, Tolerability, Pharmacokinetics, and Effect on Disease Progression of BIIB078 Administered to Previously Treated Adults C9ORF72-Associated Amyotrophic Lateral Sclerosis (ALS) NCT04288856 · started 2020 · scored riskier than 88% of active trials terminated
• A Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB078 in Adults With C9ORF72-Associated Amyotrophic Lateral Sclerosis NCT03626012 · started 2018 · scored riskier than 40% of active trials completed
• A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BIIB100 Administered Orally to Adults With Amyotrophic Lateral Sclerosis NCT03945279 · started 2019 · scored riskier than 46% of active trials completed
• A Study to Assess the Safety, Tolerability, and Effect on Disease Progression of BIIB105 in Participants With Amyotrophic Lateral Sclerosis (ALS) and Participants With the ALS Ataxin-2 (ATXN2) Genetic Mutation NCT04494256 · started 2020 · scored riskier than 70% 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.