Phase 3
Completed N=187
Extension Study of Eculizumab in Patients With Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH)
Paroxysmal Hemoglobinuria, Nocturnal
Source: ClinicalTrials.gov NCT00122317 ↗
Enrolled (actual)
187
Serious AEs
30.5%
Results posted
Jun 2017
Primary outcomePrimary: Incidence of Treatment-emergent Adverse Events — 182 Participants
◆ Published Evidence
Highly cited
579citations · ~30 / year
Effect of the complement inhibitor eculizumab on thromboembolism in patients with paroxysmal nocturnal hemoglobinuria.
Summary
The purpose of this study is to evaluate the long-term safety of eculizumab in patients with transfusion dependent hemolytic PNH.
Linked Publications
-
Effect of the complement inhibitor eculizumab on thromboembolism in patients with paroxysmal nocturnal hemoglobinuria.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Incidence of Treatment-emergent Adverse Events |
182 | — |
| SECONDARY Hemolysis as Measured by Change From Baseline in LDH Area Under the Curve |
-365185.8 | — |
| SECONDARY Quality of Life as Measured by FACIT-Fatigue Scale Change From Baseline |
9.24 | — |
| SECONDARY Incidence of Thrombosis After Eculizumab Infusion |
7.82; 1.99 | — |
Eligibility Criteria
Inclusion Criteria
- Patients who have fully completed the TRIUMPH, SHEPHERD, or X03-001 studies
- TRIUMPH patients who have discontinued receiving investigational drug prior to the last visit of the study due to lack of efficacy or exacerbation of symptoms of PNH and have completed all monthly safety and efficacy procedures
- Patient must be willing and able to give written informed consent
- Patient must avoid conception during the trial
Exclusion Criteria
- Patients who have terminated early from the SHEPHERD or X03-001 studies
- Patients who have terminated early from the TRIUMPH study due to an adverse event
- Female who is pregnant, breast feeding, or intending to conceive during the course of the study
- Any condition that could increase the patient's risk by participating in the study or could confound the outcome of the study
Data sourced from ClinicalTrials.gov (NCT00122317) and the linked publication. 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.