Trial results for the MERIDIAN study (NCT04579666) investigating pegcetacoplan in adults with amyotrophic lateral sclerosis (ALS) were posted on ClinicalTrials.gov on 2025-04-24. The Phase 2 trial showed no significant difference in the Combined Assessment of Function and Survival (CAFS) rank score between pegcetacoplan and placebo (LS mean difference -3.0, p=0.7205 at Week 52), alongside higher rates of treatment-emergent adverse events.
Background
Pegcetacoplan was investigated for amyotrophic lateral sclerosis (ALS), a progressive neurodegenerative disease also known as motor neuron disease.
Trial design
The MERIDIAN study (NCT04579666) was a Phase 2, multicenter, randomized, double-blind, placebo-controlled study. It enrolled 249 participants with amyotrophic lateral sclerosis (ALS) or motor neuron disease. The trial aimed to evaluate the efficacy and safety of pegcetacoplan compared to placebo.
Key results
For the Combined Assessment of Function and Survival (CAFS) rank score at Week 52, the least squares mean for the pegcetacoplan group was 123.0 (Standard Error: 4.71), compared to 126.0 (Standard Error: 6.89) for the placebo group. An ANCOVA analysis showed a difference in least squares mean of -3.0 (95.0% CI: -19.5 to 13.5), with a p-value of 0.7205, indicating no significant difference.
Safety outcomes included:
- Treatment-emergent adverse events (TEAEs): 137 participants in pegcetacoplan group vs. 61 in placebo group.
- Treatment-emergent serious adverse events (TESAEs): 57 participants in pegcetacoplan group vs. 27 in placebo group.
- Positive response to Columbia-Suicide Severity Rating Scale (C-SSRS) up to Week 52: 16 participants in pegcetacoplan group vs. 6 in placebo group.
What this means
The MERIDIAN trial results indicate that pegcetacoplan did not show a significant benefit in improving the CAFS rank score in adults with ALS compared to placebo. The higher incidence of adverse events and serious adverse events in the pegcetacoplan group, without efficacy, suggests potential safety concerns. These findings contribute to understanding pegcetacoplan's profile in ALS, especially given the trial's terminated status.
Source
The information regarding these trial results was obtained from ClinicalTrials.gov. The results for study NCT04579666, titled "MERIDIAN: A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Adults With Amyotrophic Lateral Sclerosis (ALS)," were posted on 2025-04-24 on clinicaltrials.gov.