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Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients

Primary Purpose

Paroxysmal Nocturnal Hemoglobinuria

Status
Completed
Phase
Phase 2
Locations
Study Type
Interventional
Intervention
Eculizumab
Sponsored by
Alexion
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Paroxysmal Nocturnal Hemoglobinuria focused on measuring Hemolytic Paroxysmal Nocturnal Hemoglobinuria, Eculizumab, PNH, Japan

Eligibility Criteria

12 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

    Arms of the Study

    Arm 1

    Arm Type

    Experimental

    Arm Label

    Eculizumab

    Arm Description

    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

    Outcomes

    Primary Outcome Measures

    Change From Baseline in Lactate Dehydrogenase

    Secondary Outcome Measures

    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.
    Change From Baseline in Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Count
    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
    Change From Baseline in Lactate Dehydrogenase (LDH) Area Under the Curve (AUC)
    Change From Baseline in Plasma Free Hemoglobin
    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.

    Full Information

    First Posted
    August 30, 2010
    Last Updated
    February 11, 2018
    Sponsor
    Alexion
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    1. Study Identification

    Unique Protocol Identification Number
    NCT01192399
    Brief Title
    Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients
    Official Title
    Phase II Clinical Study of Eculizumab (h5G1.1-mAb) in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    February 2018
    Overall Recruitment Status
    Completed
    Study Start Date
    November 2007 (undefined)
    Primary Completion Date
    June 2008 (Actual)
    Study Completion Date
    June 2008 (Actual)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Alexion

    4. Oversight

    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
    Data Monitoring Committee
    No

    5. Study Description

    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.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Paroxysmal Nocturnal Hemoglobinuria
    Keywords
    Hemolytic Paroxysmal Nocturnal Hemoglobinuria, Eculizumab, PNH, Japan

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 2
    Interventional Study Model
    Single Group Assignment
    Masking
    None (Open Label)
    Allocation
    N/A
    Enrollment
    29 (Actual)

    8. Arms, Groups, and Interventions

    Arm Title
    Eculizumab
    Arm Type
    Experimental
    Arm Description
    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
    Intervention Type
    Biological
    Intervention Name(s)
    Eculizumab
    Other Intervention Name(s)
    Soliris
    Primary Outcome Measure Information:
    Title
    Change From Baseline in Lactate Dehydrogenase
    Time Frame
    Baseline, Week 12
    Secondary Outcome Measure Information:
    Title
    Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Total Score
    Description
    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
    Title
    Change From Baseline in Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Count
    Time Frame
    Baseline, Week 12
    Title
    Number of Units of Packed Red Blood Cells (pRBCs) Transfused
    Description
    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
    Title
    Change From Baseline in Lactate Dehydrogenase (LDH) Area Under the Curve (AUC)
    Time Frame
    Baseline to Week 12
    Title
    Change From Baseline in Plasma Free Hemoglobin
    Time Frame
    Baseline, Week 12
    Title
    Change From Baseline in European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire (QLQ)-C30 Score (Global Health Status)
    Description
    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.
    Time Frame
    Baseline, Week 12

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    12 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    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
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Stephen Squinto, PhD
    Organizational Affiliation
    Alexion
    Official's Role
    Study Director

    12. IPD Sharing Statement

    Plan to Share IPD
    No
    Citations:
    PubMed Identifier
    21222185
    Citation
    Kanakura Y, Ohyashiki K, Shichishima T, Okamoto S, Ando K, Ninomiya H, Kawaguchi T, Nakao S, Nakakuma H, Nishimura J, Kinoshita T, Bedrosian CL, Valentine ME, Khursigara G, Ozawa K, Omine M. Safety and efficacy of the terminal complement inhibitor eculizumab in Japanese patients with paroxysmal nocturnal hemoglobinuria: the AEGIS clinical trial. Int J Hematol. 2011 Jan;93(1):36-46. doi: 10.1007/s12185-010-0748-9. Epub 2011 Jan 12.
    Results Reference
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