Clinical trial

Open-Label, Multicenter Study to Assess the Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Eculizumab in Complement Inhibitor Treatment Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China

Name

D7414C00001

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 paraxysmal noturnal hemoglobinuria (PNH) in China.

Trial arms

Trial start

2023-07-05

Estimated PCD

2025-06-13

Trial end

2025-06-13

Status

Active (not recruiting)

Phase

Early phase I

Treatment

Eculizumab

Participants will receive 600 milligrams (mg) once a week on Day 1, 8, 15, and 22 followed by 900 mg every 2 weeks from Day 29 to Day 435.

Arms:

Eculizumab

Size

Primary endpoint

Percentage change from baseline in Lactate Dehydrogenase (LDH) at Week 12

Baseline, Week 12

Eligibility 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

Protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'interventionModelDescription': 'open label', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 15, 'type': 'ACTUAL'}}

Updated at

2024-05-07

1 organization

1 product

1 indication

Organization

Alexion Pharmaceuticals

Product

Eculizumab

Indication

Paroxysmal Nocturnal Hemoglobinuria