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

Paroxysmal Nocturnal Hemoglobinuria Clinical Trial

— Soliris  

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05886244
• Other study ID #: D7414C00001
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: July 5, 2023
• Est. completion date: June 13, 2025

Study information

• Verified date: April 2024
• Source: Alexion Pharmaceuticals, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 15
• Est. completion date: June 13, 2025
• Est. primary completion date: June 13, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Hemoglobinuria
• Hemoglobinuria, Paroxysmal
• Paroxysmal Nocturnal Hemoglobinuria

Intervention

Drug:
• 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.

Locations

Country	Name	City	State
China	Research Site	Beijing
China	Research Site	Hangzhou
China	Research Site	Nantong
China	Research Site	Shanghai
China	Research Site	Tianjian
China	Research Site	Tianjin
China	Research Site	Wuhan

Sponsors (2)

Lead Sponsor	Collaborator
Alexion Pharmaceuticals, Inc.	AstraZeneca

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Percentage change from baseline in Lactate Dehydrogenase (LDH) at Week 12	To assess efficacy of eculizumab in participants with PNH	Baseline, Week 12
Secondary	Number(%) of participants with Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)	To assess the safety and tolerability of eculizumab in participants with PNH	Baseline through Week 64
Secondary	Pharmacokinetics (PK): Serum Eculizumab Concentration	To characterize the pharmacokinetics of eculizumab in participants with PNH	Baseline through Week 64 (predose and postdose)
Secondary	Change from baseline in Serum Free and total Complement 5 (C5) Concentration	To characterize the pharmacodynamics of eculizumab in participants with PNH	Baseline through week 64 (predose and postdose)
Secondary	Number(%) of participants with Antidrug Antibodies (ADAs) to Eculizumab	To characterize the immunogenicity of eculizumab in participants with PNH	Baseline through Week 64
Secondary	Change from baseline in functional assessment of Chronic Illness Therapy (FACIT)-Fatigue Score at Week 64	To assess the efficacy of eculizumab in participants with PNH	Baseline through Week 64
Secondary	Number(%) of participants with Breakthrough Hemolysis	To assess the efficacy of eculizumab in participants with PNH	Baseline through Week 64
Secondary	Number(%) of participants achieving LDH Normalization	To assess the efficacy of eculizumab in participants with PNH	Baseline through Week 64
Secondary	Number(%) of participants needing Blood Transfusion	To assess the efficacy of eculizumab in participants with PNH	Baseline through Week 64
Secondary	Number(%) of participants changes from baseline in vital signs and laboratory parameters at all scheduled visits.	To characterize the safety profile of eculizumab in participants with PNH	Baseline through Week 64