Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients

Phase 2CompletedNCT01192399

Alexion Pharmaceuticals, Inc.29 enrolled

Overview

A study to assess the safety and efficacy of eculizumab in Japanese patients with hemolytic PNH.

The objective of this study was to assess the safety and efficacy of eculizumab in Japanese patients with hemolytic PNH. In addition, the pharmacokinetics (PK) and pharmacodynamics (PD) of eculizumab in Japanese patients were assessed and compared to that of non-Japanese patients who had participated earlier studies of eculizumab treatment in hemolytic PNH.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Interventions

EculizumabBIOLOGICAL

Eligibility

Sex: ALLMin age: 12 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Japanese individuals at least 12 years of age * Diagnosis of PNH \> 6 months * At least one transfusion in the past 2 years for anemia or anemia-related symptoms * LDH level ≥ 1.5 x upper limit of normal within 12 weeks * Presence of a glycosylphosphatidylinositol (GPI)-deficient red blood cell (RBC) clone (type III cells) by flow cytometry of ≥10% * Negative serum pregnancy test for women of child-bearing potential Exclusion Criteria: * Platelet count \< 30,000/µL * Absolute neutrophil count ≤ 500/µL * Known or suspected hereditary complement deficiency * History of hematopoietic stem cell transplant * History of meningococcal disease

Outcomes

Primary Outcomes

Change From Baseline in Lactate Dehydrogenase

Time frame: Baseline, Week 12

Secondary Outcomes

Change From Baseline in Functional Assessment of Chronic Illness Therapy (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: Baseline, Week 12

Change From Baseline in Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Count

Time frame: Baseline, Week 12

Number of Units of Packed Red Blood Cells (pRBCs) Transfused

Comparison of number of units of pRBCs transfused in the 12 weeks prior to the first dose of eculizumab, and between baseline and 12 weeks after the first dose of eculizumab

Time frame: 12 weeks pre-treatment, baseline, 12 weeks post-treatment

Change From Baseline in Lactate Dehydrogenase (LDH) Area Under the Curve (AUC)

Time frame: Baseline to Week 12

Change From Baseline in Plasma Free Hemoglobin

Time frame: Baseline, Week 12

Change From Baseline in European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire (QLQ)-C30 Score (Global Health Status)

The European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire (QLQ) incorporates 5 functional scales (physical, role, cognitive, emotional, and social), 3 symptom scales (fatigue, pain, and nausea and vomiting), a global health status scale, and a number of single items assessing additional symptoms commonly reported by cancer patients (dyspnoea, loss of appetite, insomnia, constipation and diarrhea) and perceived financial impact of the disease. All of the scales and single-item measures range in score from 0 to 100. For global health status, a high score represents a high quality of life.

Data from ClinicalTrials.gov

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