- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01192399
Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients
February 11, 2018 updated by: Alexion Pharmaceuticals
Phase II Clinical Study of Eculizumab (h5G1.1-mAb) in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients
A study to assess the safety and efficacy of eculizumab in Japanese patients with hemolytic PNH.
Study Overview
Status
Completed
Conditions
Intervention / Treatment
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.
Study Type
Interventional
Enrollment (Actual)
29
Phase
- Phase 2
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
12 years and older (ADULT, OLDER_ADULT, CHILD)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
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
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: TREATMENT
- Allocation: NA
- Interventional Model: SINGLE_GROUP
- Masking: NONE
Arms and Interventions
Participant Group / 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
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Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Change From Baseline in Lactate Dehydrogenase
Time Frame: Baseline, Week 12
|
Baseline, Week 12
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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.
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Baseline, Week 12
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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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Baseline, 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
|
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
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12 weeks pre-treatment, baseline, 12 weeks post-treatment
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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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Baseline to Week 12
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Change From Baseline in Plasma Free Hemoglobin
Time Frame: Baseline, Week 12
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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
|
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.
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Baseline, Week 12
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Study Director: Stephen Squinto, PhD, Alexion Pharmaceuticals
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
November 1, 2007
Primary Completion (ACTUAL)
June 1, 2008
Study Completion (ACTUAL)
June 1, 2008
Study Registration Dates
First Submitted
August 30, 2010
First Submitted That Met QC Criteria
August 30, 2010
First Posted (ESTIMATE)
September 1, 2010
Study Record Updates
Last Update Posted (ACTUAL)
March 13, 2018
Last Update Submitted That Met QC Criteria
February 11, 2018
Last Verified
February 1, 2018
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Urologic Diseases
- Urological Manifestations
- Bone Marrow Diseases
- Hematologic Diseases
- Urination Disorders
- Anemia
- Proteinuria
- Anemia, Hemolytic
- Myelodysplastic Syndromes
- Hemoglobinuria
- Hemoglobinuria, Paroxysmal
- Physiological Effects of Drugs
- Immunosuppressive Agents
- Immunologic Factors
- Complement Inactivating Agents
- Eculizumab
Other Study ID Numbers
- C07-001
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
NO
Drug and device information, study documents
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
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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