Phase 3
Completed N=25
Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China
Source: ClinicalTrials.gov NCT05886244 ↗Enrolled (actual)
25
Serious AEs
36.0%
Results posted
Jun 2026
Primary outcomePrimary: Percentage Change From Baseline in LDH at Week 12 — -76.5 percentage change — p=<0.001
◆ Published Evidence
No publication linked
No peer-reviewed publication reporting this trial's results has been linked yet. This can indicate results are unpublished — a known publication-bias signal. We re-check periodically.
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.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage Change From Baseline in LDH at Week 12 |
-76.5 | <0.001 sig |
| SECONDARY Number of Participants With Treatment-emergent Adverse Events (TEAEs) |
23 | — |
| SECONDARY Serum Concentration of Eculizumab |
248.81; 491.96; 507.62; 479.04; 531.03; 530.18 | — |
| SECONDARY Change From Baseline in Serum Free Complement 5 (C5) Concentration |
-80.6911; -80.6911; -80.6911; -80.6911; -80.6911; -81.5460 | — |
| SECONDARY Change From Baseline in Serum Total C5 Concentration |
-13.3422; 49.5372; 52.8607; 50.9998; 61.3600; 60.9581 | — |
| SECONDARY Number of Participants With Treatment-emergent Antidrug Antibodies (ADAs) to Eculizumab |
— | — |
| SECONDARY Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Score at Week 12 |
5.6 | — |
| SECONDARY Percentage of Participants With Breakthrough Hemolysis |
4.0 | — |
| SECONDARY Percentage of Participants Achieving LDH Normalization |
36.0 | — |
| SECONDARY Percentage of Participants Achieving Transfusion Avoidance |
96.0 | — |
| SECONDARY Percentage of Participants With LDH ≤1.5* ULN at Week 12 |
80.0 | — |
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
Data sourced from ClinicalTrials.gov (NCT05886244). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.