Pegcetacoplan for 52 Weeks Maintains Proteinuria Reduction and eGFR Stabilization in Pediatric Patients: Phase 3 VALIANT Subgroup Analysis
Bibliographic record
Abstract
Background: C3 glomerulopathy (C3G) and primary immune-complex mediated membranoproliferative glomerulonephritis (IC-MPGN) are often diagnosed in childhood. In the 26-week randomized controlled period (RCP) of VALIANT (phase 3; NCT05067127), pegcetacoplan (C3/C3b inhibitor) for C3G and primary IC-MPGN led to significant proteinuria reduction vs placebo and estimated glomerular filtration rate (eGFR) stabilization for adolescents (12–17 years) and the overall population. In the following 26-week open-label period (OLP), all patients received pegcetacoplan. Methods: 28 and 27 adolescents were randomized to pegcetacoplan or placebo, respectively; 27 and 25 adolescents, respectively, entered the OLP. Efficacy end points included changes from baseline in proteinuria (measured as urine protein-to-creatinine ratio [UPCR]) and eGFR. Treatment-emergent adverse events were noted. Results: Adolescents who received pegcetacoplan for 52 weeks had sustained proteinuria decrease (mean [95% CI] UPCR change from baseline: week 26, –73.6% [–83.1, –58.6]; week 52, –71.8% [–82.9, –53.6]) (Figure). Results for placebo-to-pegcetacoplan patients in the OLP were consistent with proteinuria decreases in the RCP for the pegcetacoplan group. Adolescents achieved stable eGFR throughout VALIANT (least squares mean [SE] change from baseline: week 26, +0.7 [3.6] mL/min/1.73 m2; week 52, +3.2 [4.1] mL/min/1.73 m2). No new safety signals were identified. Conclusion: Clinical efficacy of 52 weeks of pegcetacoplan treatment for adolescents was consistent with the overall population of VALIANT, confirming its sustained benefit in broad patient populations. Funding: Commercial Support - This study was funded by Apellis Pharmaceuticals, Inc and Sobi (Swedish Orphan Biovitrum AB).
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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 | 0.003 |
| Meta-epidemiology (narrow) | 0.001 | 0.000 |
| Meta-epidemiology (broad) | 0.003 | 0.010 |
| Bibliometrics | 0.000 | 0.001 |
| Science and technology studies | 0.000 | 0.000 |
| Scholarly communication | 0.001 | 0.001 |
| Open science | 0.001 | 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".