Back to resultsE07-001: Safety and Efficacy Extension Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients
Primary Purpose
Paroxysmal Nocturnal Hemoglobinuria
Status
Completed
Phase
Phase 2
Locations
Study Type
Interventional
Intervention
Eculizumab
Sponsored by
About this trial
This is an interventional treatment trial for Paroxysmal Nocturnal Hemoglobinuria focused on measuring Hemolytic Paroxysmal Nocturnal Hemoglobinuria, Eculizumab, PNH
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
Sites / Locations
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Eculizumab
Arm Description
Treatment with eculizumab for patients with PNH who have successfully completed the C07-001 protocol
Outcomes
Primary Outcome Measures
Change From Baseline in Lactate Dehydrogenase
Secondary Outcome Measures
Change From Baseline in FACIT-Fatigue Scale Total Score
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
Change From Baseline in Number of Units of Packed RBCs Transfused
Baseline is defined as the number of units transfused in 3 months prior to baseline
Change From Baseline in Plasma Free Hemoglobin
Full Information
NCT ID
NCT01194804
First Posted
August 30, 2010
Last Updated
September 13, 2019
Sponsor
Alexion
Collaborators
CMIC Co, Ltd. Japan
1. Study Identification
Unique Protocol Identification Number
NCT01194804
Brief Title
E07-001: Safety and Efficacy Extension Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients
Official Title
Phase II Clinical Study of Eculizumab (h5G1.1-mAb) in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients: Extension Study to C07-001 Protocol
Study Type
Interventional
2. Study Status
Record Verification Date
September 2019
Overall Recruitment Status
Completed
Study Start Date
April 2008 (undefined)
Primary Completion Date
September 2010 (Actual)
Study Completion Date
March 2011 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Alexion
Collaborators
CMIC Co, Ltd. Japan
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Paroxysmal Nocturnal Hemoglobinuria
Keywords
Hemolytic Paroxysmal Nocturnal Hemoglobinuria, Eculizumab, PNH
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
27 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Eculizumab
Arm Type
Experimental
Arm Description
Treatment with eculizumab for patients with PNH who have successfully completed the C07-001 protocol
Intervention Type
Drug
Intervention Name(s)
Eculizumab
Other Intervention Name(s)
Soliris
Intervention Description
Each vial contains 30 mL of 10 mg/mL eculizumab; dose of 900 mg intravenous every 14 days.
Primary Outcome Measure Information:
Title
Change From Baseline in Lactate Dehydrogenase
Time Frame
52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)
Secondary Outcome Measure Information:
Title
Change From Baseline in FACIT-Fatigue Scale Total Score
Description
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.
Time Frame
52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)
Title
Change From Baseline in PNH Red Blood Cell (RBC) Count
Time Frame
52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)
Title
Change From Baseline in Number of Units of Packed RBCs Transfused
Description
Baseline is defined as the number of units transfused in 3 months prior to baseline
Time Frame
52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)
Title
Change From Baseline in Plasma Free Hemoglobin
Time Frame
52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)
10. Eligibility
Sex
All
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
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
12. IPD Sharing Statement
Plan to Share IPD
No
Citations:
PubMed Identifier
23934275
Citation
Kanakura Y, Ohyashiki K, Shichishima T, Okamoto S, Ando K, Ninomiya H, Kawaguchi T, Nakao S, Nakakuma H, Nishimura J, Kinoshita T, Bedrosian CL, Ozawa K, Omine M. Long-term efficacy and safety of eculizumab in Japanese patients with PNH: AEGIS trial. Int J Hematol. 2013 Oct;98(4):406-16. doi: 10.1007/s12185-013-1404-y. Epub 2013 Aug 11.
Results Reference
result
Learn more about this trial
E07-001: Safety and Efficacy Extension Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients
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