RAISE-XT: interim analysis of an open-label extension study of zilucoplan in patients with myasthenia gravis
Bibliographic record
Abstract
Background Long-term data from RAISE-XT (NCT04225871), a Phase 3, multicentre, open-label extension study, will enhance our understanding of the safety and efficacy of the C5 inhibitor zilucoplan in patients with gMG. Design/Methods Adults (≥18–<75 years) with gMG who completed a qualifying zilucoplan study (NCT03315130/NCT04115293) self-administered daily subcutaneous injections of zilucoplan 0.3 mg/kg. Primary outcome was incidence of TEAEs. Secondary outcomes included change from qualifying study baseline to Extension Week 12 (Week E12) in MG-ADL score and responder rates for MG-ADL (reduction of ≥3 points without rescue therapy). Results Among 199 patients enrolled in RAISE-XT, 104 continued zilucoplan from their parent study (zilu- coplan group) and 95 switched to zilucoplan from placebo (placebo-switch). Median exposure at data cut-off was 253 (range 29–1434) days. TEAEs occurred in 169 (84.9%) patients; 46 (23.1%) patients experienced a serious TEAE. At Week E12, LS mean (95% CI) changes in MG-ADL score for the zilucoplan and placebo-switch groups were −6.30 (−7.44, −5.15) and −6.32 (−8.00, −4.65). MG-ADL responder rates improved from extension study baseline through Week E12 (zilucoplan: 75.0% to 80.3% of patients; pla- cebo-switch: 52.2% to 79.7%). Conclusion In this interim analysis, zilucoplan demonstrated a favourable long-term safety profile and sustained efficacy. Funding: UCB Pharma.
Fetched live from OpenAlex and de-inverted. Abstracts are not stored in this database: the inverted indexes are 8.6 GB of the frame’s 9.3 GB of text, and the host has 13 GB free.
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 | 0.009 | 0.004 |
| Meta-epidemiology (narrow) | 0.001 | 0.000 |
| Meta-epidemiology (broad) | 0.002 | 0.003 |
| Bibliometrics | 0.000 | 0.000 |
| Science and technology studies | 0.000 | 0.001 |
| Scholarly communication | 0.001 | 0.001 |
| Open science | 0.001 | 0.001 |
| Research integrity | 0.001 | 0.002 |
| Insufficient payload (model declined to judge) | 0.003 | 0.000 |
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".