Clinical Trials

At Apellis, we are committed to developing transformative therapies for people living with a broad range of debilitating diseases by controlling complement.

Visit clinicaltrials.gov to learn more about study participation including eligibility, risks and benefits participation, the informed consent process, questions to ask when considering study participation.

If you have questions about our clinical trials, please email us at clinicaltrials@apellis.com. Learn more about ongoing, Apellis-sponsored clinical trials below.

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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PNHpioneer.com

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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clinicaltrials.gov

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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clinicaltrials.gov

COMPLETED

PADDOCK: Pilot study to assess safety, preliminary efficacy and pharmacokinetics of subcutaneous pegcetacoplan (APL‑2) in patients with PNH

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clinicaltrials.gov

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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clinicaltrials.gov

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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clinicaltrials.gov

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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clinicaltrials.gov

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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clinicaltrials.gov

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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clinicaltrials.gov

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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clinicaltrials.gov

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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MERIDIANTrial.com

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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TheVALIANTStudy.com

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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TheNOBLEStudy.com

ACTIVE, NOT RECRUITING

DISCOVERY: Phase 2 study assessing safety and efficacy of pegcetacoplan (APL‑2) in glomerulopathies

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clinicaltrials.gov

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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clinicaltrials.gov

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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clinicaltrials.gov

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).

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clinicaltrials.gov