Extension Study of Eculizumab in Patients With Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH)

Study Overview
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Brief Summary
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The purpose of this study is to evaluate the long-term safety of eculizumab in patients with transfusion dependent hemolytic PNH.

Detailed Description
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An open-label extension study to evaluate long-term safety of eculizumab in PNH patients who had completed the TRIUMPH (C04-001), SHEPHERD (C04-002), and X03-001 studies.

Official Title
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Open Label Extension Study of Eculizumab in Patients With Transfusion Dependent PNH

Conditions
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Paroxysmal Hemoglobinuria, Nocturnal

Intervention / Treatment
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* Drug: eculizumab


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients who have fully completed the TRIUMPH, SHEPHERD, or X03-001 studies TRIUMPH patients who have discontinued receiving investigational drug prior to the last visit of the study due to lack of efficacy or exacerbation of symptoms of PNH and have completed all monthly safety and efficacy procedures Patient must be willing and able to give written informed consent Patient must avoid conception during the trial Exclusion Criteria: Patients who have terminated early from the SHEPHERD or X03-001 studies Patients who have terminated early from the TRIUMPH study due to an adverse event Female who is pregnant, breast feeding, or intending to conceive during the course of the study Any condition that could increase the patient's risk by participating in the study or could confound the outcome of the study

Ages Eligible for Study
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Minimum Age: 18 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>600 mg intravenous infusion every week x 4 then 900 mg iv every two weeks | Drug: eculizumab<br> <br> * Other names: Soliris;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of Treatment-emergent Adverse Events |  | From time of consent to a maximum of 2.5 years of study treatment | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Hemolysis as Measured by Change From Baseline in LDH Area Under the Curve |  | From time of first infusion through 24 months of study treatment | 
| Quality of Life as Measured by FACIT-Fatigue Scale Change From Baseline | The FACIT-Fatigue scale 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. | From time of first infusion through 24 months of study treatment | 
| Incidence of Thrombosis After Eculizumab Infusion | Thrombosis was defined as occurrence of major adverse vascular events | From time of first ever dose through last dose (up to 24 months of study treatment) | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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transfusion dependent, paroxysmal nocturnal hemoglobinuria, hemolytic