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

Alexion Pharmaceuticals, Inc.187 enrolled

Overview

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

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

187

Conditions

Paroxysmal Hemoglobinuria, Nocturnal

Interventions

eculizumabDRUG

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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

Locations (40)

Outcomes

Primary Outcomes

Incidence of Treatment-emergent Adverse Events

Time frame: From time of consent to a maximum of 2.5 years of study treatment

Secondary Outcomes

Hemolysis as Measured by Change From Baseline in LDH Area Under the Curve

Time frame: 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.

Time frame: 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

Time frame: From time of first ever dose through last dose (up to 24 months of study treatment)

Data from ClinicalTrials.gov

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