Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China

• Status: Not yet recruiting
• Trial end date: 2025-04-30
• Target enrollment: 0
• Participant gender: All

Summary

This is a Phase 3b, single-arm, open-label, multicenter study to evaluate the efficacy, safety, pharmacokinetics (PK), pharmacodynamics (PD), and immunogenicity of eculizumab in adult participants with paraxysmal noturnal hemoglobinuria (PNH) in China.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Alexion
• Collaborator: AstraZeneca
• Treatments: Eculizumab

Criteria

Inclusion Criteria:

• Adult C5 inhibitor naïve PNH patients (age>=18), which is confirmed by flow cytometry evaluation.

• Must be vaccinated against N meningitidis.

Exclusion Criteria:

• Meningitidis infection or unresolved meningococcal disease

• Significant bone marrow failure

• Other significant systemic diseases that might have impact on efficacy and safety assessment