NCT01194804 - E07-001: Safety and Efficacy Extension Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients | Crick | Crick

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

Phase 2CompletedNCT01194804

Alexion Pharmaceuticals, Inc.27 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

NA

Purpose

TREATMENT

Masking

NONE

Enrollment

27

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Interventions

Each vial contains 30 mL of 10 mg/mL eculizumab; dose of 900 mg intravenous every 14 days.

Eligibility

Sex: ALLMin age: 12 Years

Medical Language ↔ Plain English

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

Outcomes

Primary Outcomes

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 Outcomes

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.

Time frame: 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

Time frame: 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

Time frame: 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

Time frame: 52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)

Data from ClinicalTrials.gov

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