Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Chinese Adults Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Alexion Pharmaceuticals

Status and phase: Completed; Phase 3

Conditions: PNH; Paroxysmal Nocturnal Hemoglobinuria

Treatments: Drug: Ravulizumab

Study type: Interventional

Funder types: Industry

Identifiers

NCT06578949

D9289C00008

ALXN1210-PNH-323 (Other Identifier)

Details and patient eligibility

About

The primary objective of this study is to evaluate the efficacy of ravulizumab in adult participants with PNH.

Enrollment: 18 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria:

• Adult C5 inhibitor naive PNH patients (age>=18), which is confirmed by flow cytometry evaluation.
• Must be vaccinated againast N meningitidis.

Exclusion Criteria

• Meningitidis infection or unresolved meningococcal disease
• History of bone marrow transplantation
• 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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18 participants in 1 patient group

Ravulizumab

Experimental group

Description:

During the Primary Treatment Period, participants will receive a weight-based loading dose of ravulizumab on Day 1 followed by weight-based maintenance dose of ravulizumab on Day 15 and once every 8 weeks (q8w) thereafter for a total of 26 weeks. On Day 183, all participants will enter a 32-week Extension Treatment Period and receive ravulizumab. Beginning on Day 183, participants will receive a maintenance dose of ravulizumab q8w for an additional 32 weeks.

Treatment:

Drug: Ravulizumab