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Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients

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ClinicalTrials.gov Identifier: NCT01192399
Recruitment Status : Completed
First Posted : September 1, 2010
Results First Posted : March 13, 2018
Last Update Posted : March 13, 2018
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Paroxysmal Nocturnal Hemoglobinuria
Intervention Biological: Eculizumab
Enrollment 29
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Eculizumab
Hide Arm/Group 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
Period Title: Overall Study
Started 29
Completed 27
Not Completed 2
Reason Not Completed
Lack of Efficacy             2
Arm/Group Title Eculizumab
Hide Arm/Group 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
Overall Number of Baseline Participants 29
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 29 participants
47.0  (12.39)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 29 participants
Female
15
  51.7%
Male
14
  48.3%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Japanese Number Analyzed 29 participants
29
 100.0%
Region of Enrollment  
Measure Type: Count of Participants
Unit of measure:  Participants
Japan Number Analyzed 29 participants
29
 100.0%
1.Primary Outcome
Title Change From Baseline in Lactate Dehydrogenase
Hide Description [Not Specified]
Time Frame Baseline, Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Eculizumab
Hide Arm/Group 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
Overall Number of Participants Analyzed 29
Mean (Standard Error)
Unit of Measure: Units/Liter
Baseline 1845.1  (115.34)
Week 12 398.7  (99.15)
Change from Baseline at Week 12 -1446.4  (138.68)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Eculizumab
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Sign test
Comments [Not Specified]
2.Secondary Outcome
Title Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Total Score
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Eculizumab
Hide Arm/Group 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
Overall Number of Participants Analyzed 29
Mean (Standard Error)
Unit of Measure: units on a scale
Baseline 38.5  (1.88)
Change from Baseline at Week 12 4.1  (2.25)
3.Secondary Outcome
Title Change From Baseline in Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Count
Hide Description [Not Specified]
Time Frame Baseline, Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Eculizumab
Hide Arm/Group 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
Overall Number of Participants Analyzed 29
Mean (Standard Error)
Unit of Measure: cells x 10^12/L
Baseline 1.3  (0.14)
Week 12 1.8  (0.19)
Change from Baseline at Week 12 0.5  (0.10)
4.Secondary Outcome
Title Number of Units of Packed Red Blood Cells (pRBCs) Transfused
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Eculizumab
Hide Arm/Group 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
Overall Number of Participants Analyzed 29
Mean (Standard Error)
Unit of Measure: Units
12 Weeks Pre-Dose 5.2  (1.04)
12 Weeks Post-Dose 1.5  (0.67)
Difference Post - Pre -3.7  (1.12)
5.Secondary Outcome
Title Change From Baseline in Lactate Dehydrogenase (LDH) Area Under the Curve (AUC)
Hide Description [Not Specified]
Time Frame Baseline to Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Eculizumab
Hide Arm/Group 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
Overall Number of Participants Analyzed 29
Mean (Standard Error)
Unit of Measure: U/L x Day
-113540.5  (10539.33)
6.Secondary Outcome
Title Change From Baseline in Plasma Free Hemoglobin
Hide Description [Not Specified]
Time Frame Baseline, Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Eculizumab
Hide Arm/Group 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
Overall Number of Participants Analyzed 29
Mean (Standard Error)
Unit of Measure: mg/dL
Baseline 22.6  (2.61)
Change from Baseline at Week 12 -19.8  (2.71)
7.Secondary Outcome
Title Change From Baseline in European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire (QLQ)-C30 Score (Global Health Status)
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Eculizumab
Hide Arm/Group 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
Overall Number of Participants Analyzed 28
Least Squares Mean (Standard Error)
Unit of Measure: units on a scale
8.9  (6.39)
Time Frame Adverse events were collected over the full study period (12 weeks).
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Eculizumab
Hide Arm/Group Description 600 mg of eculizumab as intravenous (IV) infusion once a week for 4 doses, followed by 900 mg eculizumab IV infusion 1 week later for 1 dose, then 900 mg eculizumab IV infusion every 2 weeks for 4 doses.
All-Cause Mortality
Eculizumab
Affected / at Risk (%)
Total   0/29 (0.00%) 
Show Serious Adverse Events Hide Serious Adverse Events
Eculizumab
Affected / at Risk (%)
Total   1/29 (3.45%) 
General disorders   
Pyrexia  1  1/29 (3.45%) 
Investigations   
C-Reactive Protein Increased  1  1/29 (3.45%) 
1
Term from vocabulary, MedDRA (11.0)
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Eculizumab
Affected / at Risk (%)
Total   28/29 (96.55%) 
Blood and lymphatic system disorders   
Anaemia  1  2/29 (6.90%) 
Leukopenia  1  2/29 (6.90%) 
Ear and labyrinth disorders   
Tinnitis  1  2/29 (6.90%) 
Eye disorders   
Conjunctival haemorrhage  1  2/29 (6.90%) 
Gastrointestinal disorders   
Nausea  1  6/29 (20.69%) 
Diarrhoea  1  4/29 (13.79%) 
Vomiting  1  3/29 (10.34%) 
General disorders   
Chest discomfort  1  2/29 (6.90%) 
Fatigue  1  2/29 (6.90%) 
Pyrexia  1  2/29 (6.90%) 
Infections and infestations   
Nasopharyngitis  1  12/29 (41.38%) 
Investigations   
Blood alkaline phosphatase increased  1  2/29 (6.90%) 
Blood bilirubin increased  1  2/29 (6.90%) 
Nervous system disorders   
Headache  1  15/29 (51.72%) 
Respiratory, thoracic and mediastinal disorders   
Upper respiratory tract inflammation  1  2/29 (6.90%) 
Skin and subcutaneous tissue disorders   
Eczema  1  3/29 (10.34%) 
1
Term from vocabulary, MedDRA (11.0)
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Alexion Pharmaceuticals, Inc
Organization: Alexion Pharmaceuticals, Inc
EMail: clinicaltrials@alexion.com
Layout table for additonal information
Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01192399     History of Changes
Other Study ID Numbers: C07-001
First Submitted: August 30, 2010
First Posted: September 1, 2010
Results First Submitted: November 6, 2017
Results First Posted: March 13, 2018
Last Update Posted: March 13, 2018