Clinical trial NCT05886244: Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Paroxysmal Nocturnal Hemoglobinuria
Keywords: paroxysmal nocturnal hemoglobinuria
PNH
Lead sponsor: Alexion Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
Collaborators: name: AstraZeneca
First posted: 2023-06-02
Enrollment: 25
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: open label
Primary purpose: TREATMENT

Brief 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.

Detailed 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.

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

Primary outcomes:
measure: Percentage change from baseline in Lactate Dehydrogenase (LDH) at Week 12
timeFrame: Baseline, Week 12
Secondary outcomes:
measure: Number(%) of participants with Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
timeFrame: Baseline through Week 64
measure: Pharmacokinetics (PK): Serum Eculizumab Concentration
timeFrame: Baseline through Week 64 (predose and postdose)
measure: Change from baseline in Serum Free and total Complement 5 (C5) Concentration
timeFrame: Baseline through week 64 (predose and postdose)
measure: Number(%) of participants with Antidrug Antibodies (ADAs) to Eculizumab
timeFrame: Baseline through Week 64
measure: Change from baseline in functional assessment of Chronic Illness Therapy (FACIT)-Fatigue Score at Week 64
timeFrame: Baseline through Week 64
measure: Number(%) of participants with Breakthrough Hemolysis
timeFrame: Baseline through Week 64
measure: Number(%) of participants achieving LDH Normalization
timeFrame: Baseline through Week 64
measure: Number(%) of participants needing Blood Transfusion
timeFrame: Baseline through Week 64
measure: Number(%) of participants changes from baseline in vital signs and laboratory parameters at all scheduled visits.
timeFrame: Baseline through Week 64
Other outcomes:
Locations:
city: Beijing
country: China
facility: Research Site
city: Hangzhou
country: China
facility: Research Site
city: Nantong
country: China
facility: Research Site
city: Shanghai
country: China
facility: Research Site
city: Tianjin
country: China
facility: Research Site
city: Tianjin
country: China
facility: Research Site
city: Wuhan
country: China
facility: Research Site
Study officials:
References: