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

Study Overview
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Brief Summary
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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.

Official Title
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Phase II Clinical Study of Eculizumab (h5G1.1-mAb) in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients: Extension Study to C07-001 Protocol

Conditions
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Paroxysmal Nocturnal Hemoglobinuria

Intervention / Treatment
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* Drug: Eculizumab


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 12 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Eculizumab<br>Treatment with eculizumab for patients with PNH who have successfully completed the C07-001 protocol | Drug: Eculizumab<br>* Each vial contains 30 mL of 10 mg/mL eculizumab; dose of 900 mg intravenous every 14 days.<br>* Other names: Soliris;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | 52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study) | 
| 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 | Baseline is defined as the number of units transfused in 3 months prior to baseline | 52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study) | 
| 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) | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Hemolytic Paroxysmal Nocturnal Hemoglobinuria, Eculizumab, PNH