Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu

Extension Study of Eculizumab in Patients With Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00122317
Recruitment Status : Completed
First Posted : July 22, 2005
Results First Posted : June 5, 2017
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 Hemoglobinuria, Nocturnal
Intervention Drug: eculizumab
Enrollment 187
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Eculizumab
Hide Arm/Group Description eculizumab: 600 mg intravenous infusion every week x 4 then 900 mg iv every two weeks
Period Title: Overall Study
Started 187
Completed 176
Not Completed 11
Reason Not Completed
Death             3
Adverse Event             4
Physician Decision             2
patient non-compliance             1
Withdrawal by Subject             1
Arm/Group Title Eculizumab
Hide Arm/Group Description eculizumab: 600 mg intravenous infusion every week x 4 then 900 mg iv every two weeks
Overall Number of Baseline Participants 187
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 187 participants
42.1  (14.1)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 187 participants
Female
101
  54.0%
Male
86
  46.0%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 187 participants
Asian
6
   3.2%
Black
7
   3.7%
Caucasian
168
  89.8%
Other
6
   3.2%
1.Primary Outcome
Title Incidence of Treatment-emergent Adverse Events
Hide Description [Not Specified]
Time Frame From time of consent to a maximum of 2.5 years of study treatment
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Eculizumab
Hide Arm/Group Description:
eculizumab: 600 mg intravenous infusion every week x 4 then 900 mg iv every two weeks
Overall Number of Participants Analyzed 187
Measure Type: Count of Participants
Unit of Measure: Participants
182
  97.3%
2.Secondary Outcome
Title Hemolysis as Measured by Change From Baseline in LDH Area Under the Curve
Hide Description [Not Specified]
Time Frame From time of first infusion through 24 months of study treatment
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Eculizumab
Hide Arm/Group Description:
eculizumab: 600 mg intravenous infusion every week x 4 then 900 mg iv every two weeks
Overall Number of Participants Analyzed 184
Mean (Standard Deviation)
Unit of Measure: U/L x Day
-365185.8  (210407.1)
3.Secondary Outcome
Title Quality of Life as Measured by FACIT-Fatigue Scale Change From Baseline
Hide Description The FACIT-Fatigue scale 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 From time of first infusion through 24 months of study treatment
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Eculizumab
Hide Arm/Group Description:
eculizumab: 600 mg intravenous infusion every week x 4 then 900 mg iv every two weeks
Overall Number of Participants Analyzed 184
Mean (Standard Deviation)
Unit of Measure: units on a scale
9.24  (12.42)
4.Secondary Outcome
Title Incidence of Thrombosis After Eculizumab Infusion
Hide Description Thrombosis was defined as occurrence of major adverse vascular events
Time Frame From time of first ever dose through last dose (up to 24 months of study treatment)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Eculizumab
Hide Arm/Group Description:
eculizumab: 600 mg intravenous infusion every week x 4 then 900 mg iv every two weeks
Overall Number of Participants Analyzed 184
Measure Type: Number
Unit of Measure: Number of events per 100 patient-years
Prior to first eculizumab dose 7.82
From first dose of eculizumab through last dose 1.99
Time Frame From time of consent to a maximum of 2.5 years of study treatment
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Eculizumab
Hide Arm/Group Description eculizumab: 600 mg intravenous infusion every week x 4 then 900 mg iv every two weeks
All-Cause Mortality
Eculizumab
Affected / at Risk (%)
Total   3/187 (1.60%) 
Show Serious Adverse Events Hide Serious Adverse Events
Eculizumab
Affected / at Risk (%)
Total   57/187 (30.48%) 
Blood and lymphatic system disorders   
Agranulocytosis  1  1/187 (0.53%) 
Anaemia  1  3/187 (1.60%) 
Aplastic anaemia  1  2/187 (1.07%) 
Haemolysis  1  5/187 (2.67%) 
Neutropenia  1  1/187 (0.53%) 
Thrombocytopenia  1  1/187 (0.53%) 
Cardiac disorders   
Cardiac Failure  1  1/187 (0.53%) 
Eye disorders   
Retinal vein thrombosis  1  1/187 (0.53%) 
Gastrointestinal disorders   
Abdominal pain  1  5/187 (2.67%) 
Ascites  1  1/187 (0.53%) 
Colitis  1  1/187 (0.53%) 
Diarrhoea  1  1/187 (0.53%) 
Gastrointestinal hemorrhage  1  1/187 (0.53%) 
Sigmoiditis  1  1/187 (0.53%) 
General disorders   
Chest pain  1  1/187 (0.53%) 
Pyrexia  1  5/187 (2.67%) 
Hepatobiliary disorders   
Bile duct stone  1  1/187 (0.53%) 
Cholecystitis  1  3/187 (1.60%) 
Cholecystitis acute  1  1/187 (0.53%) 
Cholelithiasis  1  2/187 (1.07%) 
Hepatic cirrhosis  1  1/187 (0.53%) 
Hepatic vein thrombosis  1  1/187 (0.53%) 
Portal vein thrombosis  1  2/187 (1.07%) 
Immune system disorders   
Endocarditis  1  1/187 (0.53%) 
Infections and infestations   
Bacterial sepsis  1  1/187 (0.53%) 
Bronchitis  1  1/187 (0.53%) 
Cellulitis  1  2/187 (1.07%) 
Cellulitis gangrenous  1  1/187 (0.53%) 
Empyema  1  1/187 (0.53%) 
Enterococcal sepsis  1  1/187 (0.53%) 
Gallbladder abscess  1  1/187 (0.53%) 
Gastroenteritis  1  1/187 (0.53%) 
Gastroenteritis viral  1  2/187 (1.07%) 
Haemophilus infection  1  1/187 (0.53%) 
Infection  1  1/187 (0.53%) 
Liver abscess  1  1/187 (0.53%) 
Lower respiratory tract infection  1  2/187 (1.07%) 
Meningococcal sepsis  1  2/187 (1.07%) 
Necrotising fasciitis  1  1/187 (0.53%) 
Penile infection  1  1/187 (0.53%) 
Pneumonia  1  2/187 (1.07%) 
Respiratory tract infection  1  2/187 (1.07%) 
Sepsis  1  1/187 (0.53%) 
Septic shock  1  2/187 (1.07%) 
Staphylococcal Infection  1  1/187 (0.53%) 
Upper Respiratory Tract Infection  1  1/187 (0.53%) 
Urinary Tract Infection  1  2/187 (1.07%) 
Viral Infection  1  3/187 (1.60%) 
Viral Upper Respiratory Tract Infection  1  1/187 (0.53%) 
Injury, poisoning and procedural complications   
Drug Toxicity  1  1/187 (0.53%) 
Wrist Fracture  1  1/187 (0.53%) 
Investigations   
Haemoglobin Decreased  1  1/187 (0.53%) 
Metabolism and nutrition disorders   
Hypokalaemia  1  1/187 (0.53%) 
Hyponatraemia  1  1/187 (0.53%) 
Musculoskeletal and connective tissue disorders   
Intervertebral Disc Protrusion  1  1/187 (0.53%) 
Tenosynovitis Stenosans  1  1/187 (0.53%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
Adenocarcinoma  1  1/187 (0.53%) 
Chronic Myelomonocytic Leukaemia  1  1/187 (0.53%) 
Malignant Melanoma  1  1/187 (0.53%) 
Metastases to Bone  1  1/187 (0.53%) 
Nervous system disorders   
Cerebral Haemorrhage  1  1/187 (0.53%) 
Convulsion  1  1/187 (0.53%) 
Headache  1  1/187 (0.53%) 
Metabolic Encephalopathy  1  1/187 (0.53%) 
Psychiatric disorders   
Suicidal Ideation  1  1/187 (0.53%) 
Renal and urinary disorders   
Paroxysmal Nocturnal Haemoglobinuria  1  4/187 (2.14%) 
Calculus Urinary  1  1/187 (0.53%) 
Nephrolithiasis  1  2/187 (1.07%) 
Renal Failure Acute  1  2/187 (1.07%) 
Renal Impairment  1  1/187 (0.53%) 
Reproductive system and breast disorders   
Erectile Dysfunction  1  1/187 (0.53%) 
Ovarian Cyst Ruptured  1  1/187 (0.53%) 
Respiratory, thoracic and mediastinal disorders   
Pleurisy  1  1/187 (0.53%) 
Vascular disorders   
Hypotension  1  1/187 (0.53%) 
Phlebothrombosis  1  1/187 (0.53%) 
Thrombophlebitis  1  1/187 (0.53%) 
Thrombosis  1  1/187 (0.53%) 
1
Term from vocabulary, MedDRA 6.1
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   180/187 (96.26%) 
Gastrointestinal disorders   
Diarrhoea  1  44/187 (23.53%) 
Nausea  1  39/187 (20.86%) 
Vomiting  1  38/187 (20.32%) 
Abdominal Pain  1  25/187 (13.37%) 
Abdominal Pain Upper  1  18/187 (9.63%) 
Constipation  1  16/187 (8.56%) 
Dyspepsia  1  14/187 (7.49%) 
General disorders   
Influenza like Illness  1  29/187 (15.51%) 
Pyrexia  1  18/187 (9.63%) 
Oedema Peripheral  1  12/187 (6.42%) 
Fatigue  1  11/187 (5.88%) 
Chest Pain  1  10/187 (5.35%) 
Infections and infestations   
Nasopharyngitis  1  74/187 (39.57%) 
Upper Respiratory Tract Infection  1  58/187 (31.02%) 
Urinary Tract Infection  1  20/187 (10.70%) 
Viral Infection  1  17/187 (9.09%) 
Sinusitis  1  15/187 (8.02%) 
Gastroenteritis  1  12/187 (6.42%) 
Herpes Simplex  1  12/187 (6.42%) 
Bronchitis  1  10/187 (5.35%) 
Influenza  1  10/187 (5.35%) 
Injury, poisoning and procedural complications   
Contusion  1  22/187 (11.76%) 
Musculoskeletal and connective tissue disorders   
Arthralgia  1  33/187 (17.65%) 
Back Pain  1  32/187 (17.11%) 
Pain in Extremity  1  25/187 (13.37%) 
Myalgia  1  17/187 (9.09%) 
Muscle Cramp  1  10/187 (5.35%) 
Nervous system disorders   
Headache  1  61/187 (32.62%) 
Dizziness  1  21/187 (11.23%) 
Psychiatric disorders   
Insomnia  1  11/187 (5.88%) 
Respiratory, thoracic and mediastinal disorders   
Cough  1  29/187 (15.51%) 
Pharyngolaryngeal Pain  1  25/187 (13.37%) 
Epistaxis  1  14/187 (7.49%) 
Skin and subcutaneous tissue disorders   
Rash  1  12/187 (6.42%) 
Pruritus  1  11/187 (5.88%) 
Vascular disorders   
Haematoma  1  12/187 (6.42%) 
1
Term from vocabulary, MedDRA 6.1
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.
Participation in this multicenter study involved a commitment to publish the data from the study in a cooperative publication prior to release of study results on an individual basis.
Results Point of Contact
Name/Title: Alexion Pharmaceuticals, Inc.
Organization: Alexion Pharmaceuticals, Inc.
Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT00122317     History of Changes
Other Study ID Numbers: E05-001
First Submitted: July 20, 2005
First Posted: July 22, 2005
Results First Submitted: April 24, 2017
Results First Posted: June 5, 2017
Last Update Posted: March 13, 2018