3478 Baseline characteristics in the tolebrutinib phase 3 primary progressive multiple sclerosis perseus clinical trial
Bibliographic record
Abstract
Background Tolebrutinib, an oral, brain-penetrant, bioactive Bruton’s tyrosine kinase inhibitor that modulates persistent immune activation within the central nervous system, demonstrated a significant benefit on disability accumulation versus placebo in a non-relapsing secondary progressive MS population. Here, we present baseline characteristics of participants in the PERSEUS phase 3 trial ( NCT04458051), evaluating the efficacy, safety, and tolerability of tolebrutinib in primary progressive MS (PPMS).Methods PERSEUS is a randomized, double-blind, placebo-controlled, parallel-group, event-driven trial. Enrollment criteria included age 18–55 years, diagnosis of PPMS according to the 2017 McDonald criteria, Expanded Disability Status Scale (EDSS) score of 2.0–6.5 at screening, positive cerebrospinal fluid findings (oligoclonal bands and/or elevated immunoglobulin G index), and, in some countries early in study enrollment with subsequent global implementation, either no access to ocrelizumab (due to being unavailable nationally or not being reimbursed for the approved indication) or intolerance (due to adverse effects or safety reasons) or treatment failure to ocrelizumab. Participants were randomized 2:1 to receive tolebrutinib 60 mg or placebo, once daily.Results 767 participants with a mean age of 45.3 years (54% male and 83% white) were enrolled. At baseline, mean EDSS was 4.9 (median, 5.0; n=766), mean time since symptom onset was 7.7 years, and mean time since PPMS diagnosis was 4.2 years. 59% participants were treatment naïve. On MRI, 89% of participants had no gadolinium-enhancing lesions, and median T2 lesion volume was 10.8 cm 3.Conclusion This trial (results anticipated in 2025) will comprehensively assess tolebrutinib’s efficacy and safety in the PPMS population.
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How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.001 | 0.002 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.001 | 0.001 |
| Bibliometrics | 0.000 | 0.000 |
| Science and technology studies | 0.000 | 0.000 |
| Scholarly communication | 0.001 | 0.001 |
| Open science | 0.000 | 0.000 |
| Research integrity | 0.001 | 0.001 |
| Insufficient payload (model declined to judge) | 0.007 | 0.001 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".