P.059 Results From the Randomized and Open-Label Periods of the CENTAUR Trial of Sodium Phenylbutyrate and Ursodoxicoltaurine in Amyotrophic Lateral Sclerosis (ALS)
Bibliographic record
Abstract
Background: An oral, fixed-dose sodium phenylbutyrate-ursodoxicoltaurine (PB-TURSO) coformulation was evaluated in a multicenter ALS trial (CENTAUR). Methods: Adults with definite ALS, ≤18 months from symptom onset, (N=137) were randomized 2:1 to PB-TURSO or placebo for 6 months. Completing participants were eligible to receive PB-TURSO in the open-label extension (OLE) (≤30 months). The primary efficacy endpoint in both periods was rate of ALS Functional Rating Scale–Revised (ALSFRS-R) total score decline. All-cause survival was analyzed July 2020 (longest follow-up, 35 months). Safety was assessed in both periods. Results: Over 6-month randomized treatment, mean ALSFRS-R total score decline was slower with PB-TURSO vs placebo (difference, 0.42 points/month; P=0.03). Participants receiving PB-TURSO in the OLE (continued or crossover from placebo) maintained or initiated functional benefit beyond 6 months of therapy. Mean hazard of death was 44% lower (P=0.02) in the original PB-TURSO group. Overall adverse event (AE) incidence was similar, though early (week ≤3) gastrointestinal AEs were more frequent during initial exposure to PB-TURSO (randomized period or OLE). Conclusions: PB-TURSO resulted in superior retention of function in the randomized period. Long-term OLE results support functional benefits of early vs delayed therapy and of sustained treatment. Survival was longer in the original PB-TURSO group after nearly 3 years.
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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 | 0.003 | 0.004 |
| Meta-epidemiology (narrow) | 0.001 | 0.000 |
| Meta-epidemiology (broad) | 0.001 | 0.001 |
| Bibliometrics | 0.000 | 0.000 |
| Science and technology studies | 0.000 | 0.001 |
| Scholarly communication | 0.001 | 0.001 |
| Open science | 0.000 | 0.001 |
| Research integrity | 0.001 | 0.002 |
| Insufficient payload (model declined to judge) | 0.026 | 0.002 |
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".