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, and 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

PNHpioneer.com »
COMPLETED

PEGASUS: A Phase 3 study to evaluate the efficacy and safety of pegcetacoplan (APL‑2) in patients with paroxysmal nocturnal hemoglobinuria

clinicaltrials.gov »
COMPLETED

PRINCE: A Phase 3, randomized, multicenter, open-label, controlled study to evaluate the efficacy and safety of pegcetacoplan (APL‑2) in patients with PNH

clinicaltrials.gov »
COMPLETED

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

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

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

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

clinicaltrials.gov »

Geographic Atrophy
(GA)

COMPLETED

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

clinicaltrials.gov »
COMPLETED

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

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)

clinicaltrials.gov »

Amyotrophic Lateral Sclerosis (ALS)

ACTIVE, NOT RECRUITING

MERIDIAN: A Phase 2 study evaluating the efficacy and safety of pegcetacoplan in patients with sporadic ALS

clinicaltrials.gov

Immune-Complex Membranoproliferative Glomerulonephritis
(IC-MPGN) and C3 Glomerulopathy (C3G)

ACTIVE, RECRUITING

VALIANT: Phase 3 study assessing efficacy and safety of pegcetacoplan (APL‑2) in IC-MPGN and C3G

TheVALIANTStudy.com
ACTIVE, RECRUITING

NOBLE: A Phase 2 study evaluating the safety and efficacy of pegcetacoplan in the treatment of post-transplant recurrence IC-MPGN or C3G

TheNOBLEStudy.com »
ACTIVE, NOT RECRUITING

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

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

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

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)

clinicaltrials.gov »