Paroxysmal Nocturnal Hemoglobinuria (PNH)
ACTIVE, RECRUITING
PIONEER: A Phase 2 study evaluating the safety, pharmacokinetics, and biologic activity of pegcetacoplan in pediatric patients with PNH
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COMPLETED
PEGASUS: A Phase 3 study to evaluate the efficacy and safety of pegcetacoplan (APL‑2) in patients with paroxysmal nocturnal hemoglobinuria
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ACTIVE, NOT RECRUITING
PRINCE: A Phase 3, randomized, multicenter, open-label, controlled study to evaluate the efficacy and safety of pegcetacoplan (APL‑2) in patients with PNH
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COMPLETED
PADDOCK: Pilot study to assess safety, preliminary efficacy and pharmacokinetics of subcutaneous pegcetacoplan (APL‑2) in patients with PNH
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COMPLETED
PALOMINO: A Phase 2a, open-label study to evaluate the efficacy and safety of pegcetacoplan (APL‑2) in patients with PNH who have never been treated with eculizumab
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COMPLETED
PHAROAH: A Phase 1 study to assess the safety of pegcetacoplan (APL‑2) as an add-on to standard of care in patients with PNH
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ENROLLING BY INVITATION
An open-label, nonrandomized, multicenter extension study to evaluate the long term safety and efficacy of pegcetacoplan (APL‑2) in patients with PNH
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Geographic Atrophy (GA)
ACTIVE, NOT RECRUITING
DERBY: A Phase 3 study to evaluate the efficacy and safety of pegcetacoplan (APL-2) in patients with GA secondary to age-related macular degeneration
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ACTIVE, NOT RECRUITING
OAKS: A Phase 3 study to evaluate the efficacy and safety of pegcetacoplan (APL-2) in patients with GA secondary to age-related macular degeneration
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ENROLLING BY INVITATION
GALE: An open-label extension study evaluating the long-term safety and efficacy of pegcetacoplan (APL-2) in adults with GA secondary to age-related macular degeneration (AMD)
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Amyotrophic Lateral Sclerosis (ALS)
ACTIVE, RECRUITING
MERIDIAN: A Phase 2 study evaluating the efficacy and safety of pegcetacoplan in patients with sporadic ALS
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C3 glomerulopathy (C3G) and Immune-Complex Membranoproliferative Glomerulonephritis (IC-MPGN)
ACTIVE, RECRUITING
VALIANT: Phase 3 study assessing efficacy and safety of pegcetacoplan (APL‑2) in C3G and IC-MPGN
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ACTIVE, RECRUITING
NOBLE: A Phase 2 study evaluating the safety and efficacy of pegcetacoplan in the treatment of post-transplant recurrence C3G or IC-MPGN
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ACTIVE, NOT RECRUITING
DISCOVERY: Phase 2 study assessing safety and efficacy of pegcetacoplan (APL‑2) in glomerulopathies
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Cold Agglutinin Disease (CAD)
ACTIVE, RECRUITING
A Phase 3, randomized, placebo-controlled study to determine the efficacy and safety of pegcetacoplan in patients with primary CAD.
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Hematopoietic Stem Cell Transplant-Associated Thrombotic Microangiopathy (HSCT-TMA)
ACTIVE, RECRUITING
A Phase 2 study to assess pharmacokinetics (PK), pharmacodynamics (PD) and safety of pegcetacoplan in patients with TA-TMA after HSCT.
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Autoimmune Hemolytic Anemia (AIHA)
ACTIVE, NOT RECRUITING
PLAUDIT: A Phase 2, prospective, open-label study to assess the safety, tolerability, preliminary efficacy, pharmacokinetics and pharmacodynamics of multiple doses of pegcetacoplan (APL‑2) in patients with autoimmune hemolytic anemia (AIHA).