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Completed

NCT Number: NCT01192399

Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients

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

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Key information

Conditions

Paroxysmal Nocturnal Hemoglobinuria Anemia Anemia, Hemolytic Bone Marrow Diseases Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinuria, Paroxysmal Myelodysplastic Syndromes

Age range

12 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Eculizumab

Biological

Other names: Soliris

Primary outcomes

  1. Change From Baseline in Lactate Dehydrogenase

    Time frame: Baseline, Week 12

Secondary outcomes

  1. Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Total Score

    Time frame: 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.

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

    Time frame: Baseline, Week 12

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

    Time frame: 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

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

    Time frame: Baseline to Week 12

  5. Change From Baseline in Plasma Free Hemoglobin

    Time frame: Baseline, Week 12

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

    Time frame: 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.

Sponsors and collaborators

Lead sponsor

Alexion Pharmaceuticals, Inc.

Industry

Registry information

Official study title

Phase II Clinical Study of Eculizumab (h5G1.1-mAb) in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients

Important dates

Study start
2007
Primary completion
2008
Study completion
2008
First posted
Sep 1, 2010
Registry last updated
Mar 13, 2018

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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