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

Phase 2

Completed

• Conditions: Paroxysmal Nocturnal Hemoglobinuria
• Interventions: Drug: Eculizumab

• Registration Number: NCT01194804
• Lead Sponsor: Alexion Pharmaceuticals, Inc.

Brief 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.

Detailed Description

Not available

Study Timeline

• Study Start: 2008-04
• Study First Submitted: 2010-08-30
• Primary Completion: 2010-09
• Study First Submitted QC: 2010-09-02
• Study First Posted: 2010-09-03
• Study Completion: 2011-03
• Results First Submitted: 2017-11-06
• Results First Submitted QC: 2019-04-30
• Results First Posted: 2019-07-12
• Status Verified: 2019-09
• Last Update Submitted: 2019-09-13
• Last Update Posted: 2019-09-17

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 27

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Eculizumab	Eculizumab	Treatment with eculizumab for patients with PNH who have successfully completed the C07-001 protocol

Primary Outcome Measures

Name	Time	Method
Change From Baseline in Lactate Dehydrogenase	52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)

Secondary Outcome Measures

Name	Time	Method
Change From Baseline in FACIT-Fatigue Scale Total Score	52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)	The FACIT-Fatigue scale, Version 4.0, is a collection of quality of life questionnaires pertaining to the management of fatigue symptoms due to a chronic illness. The FACIT-Fatigue is a 13-item questionnaire that assesses self-reported fatigue and its impact upon daily activities and function over the preceding 7 days. Patients score each item on a 5-point scale: 0 (Not at all) to 4 (Very much). Total scores range from 0 to 52, with higher score indicating better quality of life.
Change From Baseline in PNH Red Blood Cell (RBC) Count	52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)
Change From Baseline in Number of Units of Packed RBCs Transfused	52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)	Baseline is defined as the number of units transfused in 3 months prior to baseline
Change From Baseline in Plasma Free Hemoglobin	52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)