Phase 2
Completed N=27
E07-001: Safety and Efficacy Extension Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients
Source: ClinicalTrials.gov NCT01194804 ↗Enrolled (actual)
27
Serious AEs
44.4%
Results posted
Jul 2019
Primary outcomePrimary: Change From Baseline in Lactate Dehydrogenase — -15.9 Units/Liter — p=<0.001
Summary
The objective of this study was to assess the long-term safety and efficacy of eculizumab in hemolytic PNH patients who completed the 4-week screening and 12-week treatment period of the C07-001 study. In addition, pharmacokinetic and pharmacodynamic assessments of eculizumab were conducted.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline in Lactate Dehydrogenase |
-15.9 | <0.001 sig |
| SECONDARY Change From Baseline in FACIT-Fatigue Scale Total Score |
8.0 | — |
| SECONDARY Change From Baseline in PNH Red Blood Cell (RBC) Count |
0.51 | — |
| SECONDARY Change From Baseline in Number of Units of Packed RBCs Transfused |
-3.5 | — |
| SECONDARY Change From Baseline in Plasma Free Hemoglobin |
-13.5 | — |
Eligibility Criteria
Inclusion Criteria
- Patients must have fully completed C07-001 study 12-week treatment period
Exclusion Criteria
- Patients who terminated early from the C07-001 study
Data sourced from ClinicalTrials.gov (NCT01194804). 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.