A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNH

A Phase 3, Randomized, Multicenter, Open-Label, Controlled Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Evaluation of the Efficacy and Safety of Pegcetacoplan in Patients with Paroxysmal Nocturnal Hemoglobinuria .

Inclusion Criteria:

• Be at least 18 years old (inclusive).
• Have LDH ≥1.5 x ULN at the screening visit.
• Have PNH diagnosis, confirmed by high sensitivity flow cytometry (granulocyte or monocyte clone >10%).
• Have Hb less than the lower limit of normal (LLN) at the screening visit.
• Have ferritin greater than/equal to the LLN, or total iron binding capacity (TIBC) less than/equal to ULN at the screening visit, based on central laboratory reference ranges. If a subject is receiving iron supplements at screening, the Investigator must ensure that the subject's dose has been stable for 4 weeks prior to screening, and it must be maintained throughout the study. Subjects not receiving iron at screening must not start iron supplementation during the course of the study.
• Body mass index (BMI) ≤ 35 kg/m2 at the screening visit.
• Have a platelet count of >50,000/mm3 at the screening visit.
• Have an absolute neutrophil count >500/mm3 at the screening visit.

Exclusion Criteria:

• Treatment with any complement inhibitor (eg, eculizumab) within 3 months prior to screening.

• Hereditary complement deficiency.

• History of bone marrow transplantation.

• Concomitant use of any of the following medications is prohibited if not on a stable regimen for the time period indicated below prior to screening:

  • Erythropoietin or immunosuppressants for at least 8 weeks
  • Systemic corticosteroids for at least 4 weeks
  • Vitamin K antagonists (eg, warfarin) with a stable international normalized ratio (INR) for at least 4 weeks
  • Iron supplements, vitamin B12, or folic acid for at least 4 weeks
  • Low-molecular-weight heparin for at least 4 weeks