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

Alexion Pharmaceuticals

Status and phase: Completed; Phase 3

Conditions: Paroxysmal Nocturnal Hemoglobinuria

Treatments: Drug: Eculizumab

Study type: Interventional

Funder types: Industry

Identifiers

NCT05886244

D7414C00001

Details and patient eligibility

About

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.

Full description

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 PNH in China who previously have not been treated with complement inhibitors. Approximately 25 eligible participants in China will be enrolled.

Enrollment: 25 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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25 participants in 1 patient group

Eculizumab

Experimental group

Description:

Eculizumab will be administered by IV infusion.

Treatment:

Drug: Eculizumab