Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Interventions
- Biological: Eculizumab
- Registration Number
- NCT01192399
- Lead Sponsor
- Alexion Pharmaceuticals, Inc.
- Brief Summary
A study to assess the safety and efficacy of eculizumab in Japanese patients with hemolytic PNH.
- Detailed Description
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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 29
- 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
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Eculizumab Eculizumab Eculizumab intravenous infusions every week x 4 doses, then 900 mg 1 week later for 1 dose, then 900 mg every 2 weeks for 4 doses
- Primary Outcome Measures
Name Time Method Change From Baseline in Lactate Dehydrogenase Baseline, Week 12
- Secondary Outcome Measures
Name Time Method Change From Baseline in Plasma Free Hemoglobin Baseline, Week 12 Change From Baseline in Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Count Baseline, Week 12 Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Total Score Baseline, Week 12 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.
Number of Units of Packed Red Blood Cells (pRBCs) Transfused 12 weeks pre-treatment, baseline, 12 weeks post-treatment 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
Change From Baseline in Lactate Dehydrogenase (LDH) Area Under the Curve (AUC) Baseline to 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) Baseline, Week 12 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.