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

NCT06578949 | Completed Phase 3 FDA Drug

• 2 other identifiers: D9289C00008ALXN1210-PNH-323
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 8

Brief Summary

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

Conditions

Paroxysmal Nocturnal Hemoglobinuria; PNH

Keywords

Paroxysmal Nocturnal Hemoglobinuria; PNH; Ravulizumab

Outcome Measures

Primary Outcomes (1)

• Percentage Change in Lactate Dehydrogenase (LDH) From Baseline to Day 183 (Week 26)

  Baseline, Day 183 (Week 26)

Secondary Outcomes (5)

• Number of Participants Achieving LDH <1.5 × Upper Limit of Normal (ULN) at Day 183 (Week 26)

  Day 183 (Week 26)

• Number of Participants Achieving Transfusion Avoidance (TA) Through Day 183 (Week 26)

  Day 183 (Week 26)

• Number of Participants Experiencing Breakthrough Hemolysis Through Day 183 (Week 26)

  Day 183 (Week 26)

• Change in Functional Assessment of Chronic Illness Therapy-Fatigue Scale (FACIT-Fatigue) Score From Baseline to Day 183 (Week 26)

  Day 183 (Week 26)

• Change in Hemoglobin (Hgb) From Baseline to Day 183 (Week 26)

  Baseline, Day 183 (Week 26)

Study Arms (1)

Ravulizumab

EXPERIMENTAL

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.

Drug: Ravulizumab

Interventions

Ravulizumab DRUG

Ravulizumab will be administered by intravenous (IV) infusion.

Also known as: ALXN1210

Ravulizumab

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• Meningitidis infection or unresolved meningococcal disease
• History of bone marrow transplantation
• Other significant systemic diseases that might have impact on efficacy and safety assessment

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead

Study Sites (8)

Research Site

Beijing, CN-100730, China

Location

Research Site

Guangzhou, 510100, China

Location

Research Site

Hangzhou, 310003, China

Location

Research Site

Nantong, 226001, China

Location

Research Site

Shanghai, 200040, China

Location

Research Site

Tianjin, 300020, China

Location

Research Site

Tianjin, 300050, China

Location

Research Site

Wuhan, 430022, China

Location

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Interventions

ravulizumab

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 28, 2024
• First Posted: August 30, 2024
• Study Start: October 10, 2024
• Primary Completion: May 15, 2025
• Study Completion: December 22, 2025
• Last Updated: January 21, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR

Locations

CN (8)