2603 Safety and tolerability of zilucoplan in RAISE-XT: a multicenter, open-label extension study in patients with myasthenia gravis
Bibliographic record
Abstract
Objective To evaluate the safety and efficacy of zilucoplan in an interim analysis of RAISE-XT (NCT04225871). Collating long-term clinical data will contribute to an increased understanding of the safety profile of zilucoplan in generalized myasthenia gravis (gMG). Methods RAISE-XT, a Phase 3, multicenter, open-label extension study, recruited patients with gMG who participated in randomized Phase 2 (NCT03315130) and Phase 3 (NCT04115293) zilucoplan studies. All patients self-administered daily subcutaneous injections of 0.3 mg/kg zilucoplan. Primary outcome was incidence of treatment-emergent adverse events (TEAEs). Key secondary outcomes included Myasthenia Gravis Activities of Daily Living (MG-ADL) score. Results 199 patients enrolled in RAISE-XT; 104 continued zilucoplan from their qualifying study (zilucoplan group) and 95 switched to zilucoplan from placebo (placebo-switch group). Median exposure at data cut-off was 253.0 (range 29–765) days. 169 (84.9%) patients experienced a TEAE; 46 (23.1%) patients experienced a serious TEAE. Most common TEAEs were headache and worsening of MG, both in 33 (16.6%) patients. At extension study Week 12, after 24 weeks of zilucoplan, the zilucoplan group achieved a least square mean change in MG-ADL score from double-blind study baseline of −6.30 (95% CI: −7.44, −5.15). MG-ADL reduction from baseline for the placebo-switch group, after 12 weeks of zilucoplan, was −6.32 (95% CI: −8.00, −4.65). Conclusion In this interim analysis of RAISE-XT, zilucoplan demonstrated a favorable long-term safety profile. Efficacy in patients who had previously received zilucoplan continued to improve and was demonstrated for those who switched from placebo. The study is ongoing. 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 | 0.002 | 0.001 |
| 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 | 0.000 |
| Scholarly communication | 0.000 | 0.000 |
| Open science | 0.000 | 0.000 |
| Research integrity | 0.001 | 0.001 |
| 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".