Trial results for pegcetacoplan in patients with Cold Agglutinin Disease (CAD) were posted on ClinicalTrials.gov on 2025-10-30. The Phase 3 study demonstrated that patients treated with pegcetacoplan achieved a least squares mean increase in hemoglobin of 2.85 g/dL from baseline at Week 24, compared to 1.36 g/dL in the placebo group.
Background
Pegcetacoplan (also known as Syfovre or Empaveli) is being investigated for its efficacy and safety in patients with Cold Agglutinin Disease (CAD).
Trial design
This completed Phase 3 trial (NCT05096403) enrolled 24 participants with Cold Agglutinin Disease (CAD). The study aimed to evaluate the efficacy and safety of pegcetacoplan administration compared to placebo in increasing hemoglobin levels from baseline and avoiding transfusions in participants with primary CAD.
Key results
The key results from the study at Week 24 included:
- Response Rate: 8 Participants in the pegcetacoplan group achieved a response, compared to 2 Participants in the placebo group.
- Hemoglobin Level Change: Patients treated with pegcetacoplan showed a least squares mean change from baseline of 2.85 g/dL (Standard Error 0.306) in hemoglobin, while the placebo group showed a change of 1.36 g/dL (Standard Error 0.278).
- Transfusion Avoidance: 12 Participants in the pegcetacoplan group achieved transfusion avoidance from Week 5 to Week 24, versus 5 Participants in the placebo group.
- Quality of Life (FACT-An Scale): The pegcetacoplan group had a mean change of 20.67 Units on a scale (Standard Deviation 19.175) from baseline, compared to 16.40 Units on a scale (Standard Deviation 37.246) for placebo.
- Packed Red Blood Cell Transfusions: The mean number of transfusions received from Week 5 to Week 24 was 1.5 (Standard Deviation 3.62) for pegcetacoplan and 1.1 (Standard Deviation 2.80) for placebo.
- LDH Levels: The pegcetacoplan group showed a mean change of -251.73 U/L (Standard Deviation 211.312) in LDH levels, compared to -82.00 U/L (Standard Deviation 138.477) for placebo.
What this means
The trial results suggest that pegcetacoplan may offer benefits for patients with Cold Agglutinin Disease, particularly in improving hemoglobin levels and reducing the need for transfusions compared to placebo. The observed increase in response rate and favorable changes in LDH levels further support these findings. These outcomes indicate a potential positive impact on disease management and patient quality of life for individuals with CAD.
Source
The information for these trial results was obtained from ClinicalTrials.gov, a public database of clinical studies. The results for study NCT05096403, titled "A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With Cold Agglutinin Disease (CAD)," were posted on 2025-10-30 on clinicaltrials.gov.