Phase 2
Completed N=26
Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Source: ClinicalTrials.gov NCT03078582 ↗
Enrolled (actual)
26
Serious AEs
11.5%
Results posted
Mar 2020
Primary outcomePrimary: Change-from-baseline in Serum Lactate Dehydrogenase (LDH) Level. — -695.2; 216.7 U/L
Summary
The purpose of the study is to evaluate the safety and efficacy of zilucoplan (RA101495) in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). There will be two groups of patients in the study: the first group will include patients who have never received eculizumab for treatment of PNH. The second group will include patients who have received treatment with eculizumab for at least 6 months prior to the study. Patients will be treated with RA101495 for 12 weeks.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change-from-baseline in Serum Lactate Dehydrogenase (LDH) Level. |
-695.2; 216.7 | — |
| SECONDARY Changes From Baseline in Bilirubin Values |
1.3; 1.9; 3.1; 4.4; 4.4; 6.8 | — |
| SECONDARY Total Hemoglobin |
-1.5; -0.7; 1.6; 0.9; 4.3; -1.1 | — |
| SECONDARY Changes From Baseline in Free Hemoglobin Values |
-7.84; 3.55; -8.31; -1.00; -5.65; -0.21 | — |
| SECONDARY Haptoglobin Values |
0.090; 0.010; 0.080; -0.003; 0.006; -0.003 | — |
| SECONDARY Reticulocyte Values |
-0.0113; -0.0147; -0.0157; 0.0022; -0.0099; 0.0063 | — |
| SECONDARY Hemoglobinuria Values |
-0.2; 0.8; 0.4; 0.1; 0.3; 0.7 | — |
Eligibility Criteria
Inclusion Criteria
- Diagnosis of PNH by flow cytometry
- For treatment naive patients: subjects must not have received treatment with eculizumab prior to or during the Screening Period and must have a lactate dehydrogenase (LDH) level ≥2 times the upper limit of normal (xULN) during Screening
- For patients who previously received eculizumab: subjects must have received treatment with eculizumab for at least 6 months prior to Screening
Exclusion Criteria
- History of meningococcal disease
- Current systemic infection or suspicion of active bacterial infection
Data sourced from ClinicalTrials.gov (NCT03078582). 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.