Phase 2
Completed N=3
Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients Who Have an Inadequate Response to Eculizumab
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Source: ClinicalTrials.gov NCT03030183 ↗
Enrolled (actual)
3
Serious AEs
0.0%
Results posted
Feb 2020
Primary outcomePrimary: Change-from-baseline in Serum Lactate Dehydrogenase (LDH) Levels. — 100.3 U/L
Summary
The purpose of the study is to evaluate the safety and efficacy of RA101495 in patients with paroxysmal nocturnal hemoglobinuria (PNH) who have an inadequate response to eculizumab. Patients will be treated with RA101495 for 12 weeks.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change-from-baseline in Serum Lactate Dehydrogenase (LDH) Levels. |
100.3 | — |
| SECONDARY Change-from-baseline Bilirubin Values |
-3.3; -4.7; -4.3; -0.3; -6.7; -2.3 | — |
| SECONDARY Change-from-baseline Total Hemoglobin Values |
5.7; 3.3; 0.3; -0.3; 0.7; -3.3 | — |
| SECONDARY Change-from-baseline Free Hemoglobin Values |
-145.90; -140.60; -147.70; -138.40; -147.30; -148.10 | — |
| SECONDARY Change-from-baseline Haptoglobin Values |
0.000; 0.000; 0.000; 0.133; 0.000; 0.000 | — |
| SECONDARY Change-from-baseline Reticulocyte Values |
-0.0190; 0.0090; 0.0430; 0.0477; 0.0310; 0.0253 | — |
| SECONDARY Change-from-baseline Hemoglobinuria Values |
0.0; 0.5; 0.5; 1.0; 0.5; 2.0 | — |
Eligibility Criteria
Inclusion Criteria
- Diagnosis of PNH by flow cytometry
- Inadequate response to eculizumab defined as having received eculizumab for at least 6 months plus a documented LDH level ≥ 1.5 x the upper limit of normal (ULN) and/or the presence of a known C5 mutation conferring resistance to eculizumab
Exclusion Criteria
- History of meningococcal disease
- Current systemic infection or suspicion of active bacterial infection
Data sourced from ClinicalTrials.gov (NCT03030183). 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.