Soliris: Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China
Study Details
Study Description
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
| Phase 3 |
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.
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Eculizumab Eculizumab will be administered by IV infusion. |
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.
|
Outcome Measures
Primary Outcome Measures
- Percentage change from baseline in Lactate Dehydrogenase (LDH) at Week 12 [Baseline, Week 12]
To assess efficacy of eculizumab in participants with PNH
Secondary Outcome Measures
- Number(%) of participants with Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) [Baseline through Week 64]
To assess the safety and tolerability of eculizumab in participants with PNH
- Pharmacokinetics (PK): Serum Eculizumab Concentration [Baseline through Week 64 (predose and postdose)]
To characterize the pharmacokinetics of eculizumab in participants with PNH
- Change from baseline in Serum Free and total Complement 5 (C5) Concentration [Baseline through week 64 (predose and postdose)]
To characterize the pharmacodynamics of eculizumab in participants with PNH
- Number(%) of participants with Antidrug Antibodies (ADAs) to Eculizumab [Baseline through Week 64]
To characterize the immunogenicity of eculizumab in participants with PNH
- Change from baseline in functional assessment of Chronic Illness Therapy (FACIT)-Fatigue Score at Week 64 [Baseline through Week 64]
To assess the efficacy of eculizumab in participants with PNH
- Number(%) of participants with Breakthrough Hemolysis [Baseline through Week 64]
To assess the efficacy of eculizumab in participants with PNH
- Number(%) of participants achieving LDH Normalization [Baseline through Week 64]
To assess the efficacy of eculizumab in participants with PNH
- Number(%) of participants needing Blood Transfusion [Baseline through Week 64]
To assess the efficacy of eculizumab in participants with PNH
- Number(%) of participants changes from baseline in vital signs and laboratory parameters at all scheduled visits. [Baseline through Week 64]
To characterize the safety profile of eculizumab in participants with PNH
Eligibility Criteria
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
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Significant bone marrow failure
-
Other significant systemic diseases that might have impact on efficacy and safety assessment
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Research Site | Beijing | China | CN-100730 | |
| 2 | Research Site | Hangzhou | China | 310003 | |
| 3 | Research Site | Nantong | China | 226001 | |
| 4 | Research Site | Shanghai | China | 200040 | |
| 5 | Research Site | Tianjian | China | 300020 | |
| 6 | Research Site | Tianjin | China | 300052 | |
| 7 | Research Site | Wuhan | China | 430022 |
Sponsors and Collaborators
- Alexion
- AstraZeneca
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- D7414C00001