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Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01192399
Recruitment Status : Completed
First Posted : September 1, 2010
Results First Posted : March 13, 2018
Last Update Posted : March 13, 2018
Information provided by (Responsible Party):
Alexion Pharmaceuticals

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

Condition or disease Intervention/treatment Phase
Paroxysmal Nocturnal Hemoglobinuria Biological: Eculizumab Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 29 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase II Clinical Study of Eculizumab (h5G1.1-mAb) in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients
Study Start Date : November 2007
Actual Primary Completion Date : June 2008
Actual Study Completion Date : June 2008

Arm Intervention/treatment
Experimental: 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
Biological: Eculizumab
Other Name: Soliris

Primary Outcome Measures :
  1. Change From Baseline in Lactate Dehydrogenase [ Time Frame: Baseline, Week 12 ]

Secondary Outcome Measures :
  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.

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01192399

Sponsors and Collaborators
Alexion Pharmaceuticals
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Study Director: Stephen Squinto, PhD Alexion Pharmaceuticals
Publications of Results:
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Responsible Party: Alexion Pharmaceuticals Identifier: NCT01192399    
Other Study ID Numbers: C07-001
First Posted: September 1, 2010    Key Record Dates
Results First Posted: March 13, 2018
Last Update Posted: March 13, 2018
Last Verified: February 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
Keywords provided by Alexion Pharmaceuticals:
Hemolytic Paroxysmal Nocturnal Hemoglobinuria
Additional relevant MeSH terms:
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Hemoglobinuria, Paroxysmal
Urination Disorders
Urologic Diseases
Urological Manifestations
Signs and Symptoms
Anemia, Hemolytic
Hematologic Diseases
Myelodysplastic Syndromes
Bone Marrow Diseases