Phase 2 N=27 Treatment

E07-001: Safety and Efficacy Extension Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients

Paroxysmal Nocturnal Hemoglobinuria

Bottom Line

View on ClinicalTrials.gov: NCT01194804 ↗

Enrolled (actual)

Serious AEs

44.4%

Results posted

Jul 2019

Primary outcome: Primary: Change From Baseline in Lactate Dehydrogenase — -15.9 Units/Liter — p=<0.001

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Eculizumab (Drug)
Age: Pediatric, Adult, Older Adult · 12+ yrs
Sex: All
Sponsor: Alexion Pharmaceuticals, Inc.
Primary completion: Sep 2010

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	—

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.

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

View full record on ClinicalTrials.gov →

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.