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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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12 December 2020 |
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Main ID: |
NCT01192399 |
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Date of registration:
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30/08/2010 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients
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Scientific title:
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Phase II Clinical Study of Eculizumab (h5G1.1-mAb) in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients |
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Date of first enrolment:
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November 2007 |
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Target sample size:
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29 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT01192399 |
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Study type:
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Interventional |
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Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 2
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Contacts
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Name:
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Stephen Squinto, PhD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Alexion Pharmaceuticals |
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Key inclusion & exclusion criteria
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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
Age minimum:
12 Years
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Paroxysmal Nocturnal Hemoglobinuria
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Intervention(s)
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Biological: Eculizumab
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Primary Outcome(s)
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Change From Baseline in Lactate Dehydrogenase
[Time Frame: Baseline, Week 12]
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Secondary Outcome(s)
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Change From Baseline in Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Count
[Time Frame: Baseline, Week 12]
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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]
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Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Total Score
[Time Frame: Baseline, Week 12]
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Change From Baseline in Plasma Free Hemoglobin
[Time Frame: Baseline, Week 12]
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Change From Baseline in Lactate Dehydrogenase (LDH) Area Under the Curve (AUC)
[Time Frame: Baseline to Week 12]
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Number of Units of Packed Red Blood Cells (pRBCs) Transfused
[Time Frame: 12 weeks pre-treatment, baseline, 12 weeks post-treatment]
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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