drugset / Trial / NCT04085601
A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNH
RandomizedParallel-groupOpen-labelTreatment
Summary
Evaluation of the Efficacy and Safety of Pegcetacoplan in Patients with Paroxysmal Nocturnal Hemoglobinuria .
Timeline
- Start
- 2019-08-27
- Primary completion
- 2021-06-23
- Completion
- 2021-06-23
Outcome
Met primary endpoint
registry analysis (superiority test); Pegcetacoplan vs Standard of Care; p <0.0001; Difference 0.7311 (95% CI 0.572 to 0.8902) NCT04085601 ↗
registry analysis (superiority test); Pegcetacoplan vs Standard of Care; p <0.0001; LS mean difference -1470.38 (95% CI -2113.44 to -827.32) NCT04085601 ↗
release “PRINCE phase 3 study in treatment-naïve patients with PNH showed that treatment with pegcetacoplan resulted in sustained and superior improvements in the co-primary endpoints” sobi.com ↗
Publications
- de Castro C, Kelly RJ, Griffin M, Patriquin CJ, Mulherin B, Hochsmann B, Selvaratnam V, Wong RSM, Hillmen P, Horneff R, Uchendu UO, Zhang Y, Surova E, Szamosi J, de Latour RP. Efficacy and Safety Maintained up to 3 Years in Adults with Paroxysmal Nocturnal Hemoglobinuria Receiving Pegcetacoplan. Adv Ther. 2025 Sep;42(9):4641-4658. doi: 10.1007/s12325-025-03310-8. Epub 2025 Jul 28.
- Panse J, Daguindau N, Okuyama S, Peffault de Latour R, Schafhausen P, Straetmans N, Al-Adhami M, Persson E, Wong RSM. Improvements in hematologic markers and decreases in fatigue with pegcetacoplan for patients with paroxysmal nocturnal hemoglobinuria and mild or moderate anemia (hemoglobin >/=10 g/dL) who had received eculizumab or were naive to complement inhibitors. PLoS One. 2024 Jul 29;19(7):e0306407. doi: 10.1371/journal.pone.0306407. eCollection 2024.
- Mulherin BP, Yeh M, Al-Adhami M, Dingli D. Normalization of Hemoglobin, Lactate Dehydrogenase, and Fatigue in Patients with Paroxysmal Nocturnal Hemoglobinuria Treated with Pegcetacoplan. Drugs R D. 2024 Jun;24(2):169-177. doi: 10.1007/s40268-024-00463-9. Epub 2024 May 10.
- Fishman J, Wilson K, Drzewiecka A, Pochopien M, Dingli D. The cost-effectiveness of pegcetacoplan in complement treatment-naive adults with paroxysmal nocturnal hemoglobinuria in the USA. J Comp Eff Res. 2023 Oct;12(10):e230055. doi: 10.57264/cer-2023-0055. Epub 2023 Sep 1.
- Wojciechowski P, Wdowiak M, Hakimi Z, Wilson K, Fishman J, Nazir J, Toumi M. Mapping the EORTC QLQ-C30 onto the EQ-5D-5L index for patients with paroxysmal nocturnal hemoglobinuria in France. J Comp Eff Res. 2023 May;12(5):e220178. doi: 10.57264/cer-2022-0178. Epub 2023 Apr 13.
- Wong RSM, Navarro-Cabrera JR, Comia NS, Goh YT, Idrobo H, Kongkabpan D, Gomez-Almaguer D, Al-Adhami M, Ajayi T, Alvarenga P, Savage J, Deschatelets P, Francois C, Grossi F, Dumagay T. Pegcetacoplan controls hemolysis in complement inhibitor-naive patients with paroxysmal nocturnal hemoglobinuria. Blood Adv. 2023 Jun 13;7(11):2468-2478. doi: 10.1182/bloodadvances.2022009129.
- Wong R, Fishman J, Wilson K, Yeh M, Al-Adhami M, Zion A, Yee CW, Huynh L, Duh MS. Comparative Effectiveness of Pegcetacoplan Versus Ravulizumab and Eculizumab in Complement Inhibitor-Naive Patients with Paroxysmal Nocturnal Hemoglobinuria: A Matching-Adjusted Indirect Comparison. Adv Ther. 2023 Apr;40(4):1571-1589. doi: 10.1007/s12325-023-02438-9. Epub 2023 Feb 7.
- Del Pozo Martin Y. 2021 ASH annual meeting. Lancet Haematol. 2022 Feb;9(2):e92-e93. doi: 10.1016/S2352-3026(21)00384-7. Epub 2021 Dec 16. No abstract available.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Pegcetacoplan | Peptide | 1080 mg | Subcutaneous |