Back to resultsSafety 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
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
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
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.
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Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients
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