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

Phase 3

Completed

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

Detailed Description: 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.

Recruitment & Eligibility

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

Arm && Interventions

Group	Intervention	Description
Eculizumab	eculizumab	600 mg intravenous infusion every week x 4 then 900 mg iv every two weeks

Primary Outcome Measures

Name	Time	Method
Incidence of Treatment-emergent Adverse Events	From time of consent to a maximum of 2.5 years of study treatment

Secondary Outcome Measures

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

Locations (40): Stanford University Medical Center, Division of Hematology
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Stanford, California, United States
Hartford Hospital, Cancer Clinical Research Office
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Hartford, Connecticut, United States
Cleveland Clinic Florida, Department of Clinical Research
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Weston, Florida, United States
Indianapolis University Cancer Center
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Indianapolis, Indiana, United States
Johns Hopkins University Medical Center
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Baltimore, Maryland, United States
National Heart, Lung, and Blood Institute, National Institutes of Health
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Bethesda, Maryland, United States
Massachusetts General Hospital
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Boston, Massachusetts, United States
Mayo Clinic, Divison of Hematology
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Rochester, Minnesota, United States
Washington University, Department of Internal Medicine/Division of Hematology
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Saint Louis, Missouri, United States
NYU Clinical Cancer Center
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New York, New York, United States
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Stanford University Medical Center, Division of Hematology
🇺🇸Stanford, California, United States