Trial Outcomes & Findings for Evaluation of Potential Predictors of Disease Progression in Participants With Atypical Hemolytic Uremic Syndrome (aHUS) Including Genetics, Biomarkers, and Treatment (NCT NCT02614898)
NCT ID: NCT02614898
Last Updated: 2020-01-02
Results Overview
A TMA manifestation was defined as one of following: Hematologic or renal events due to aHUS; extra-renal clinical signs and symptoms of aHUS; tissue (for example, kidney transplant) biopsy demonstrating TMA due to aHUS. The study was terminated with only 20% of the planned enrollment and due to the subsequent loss of funding for the program, the samples collected for outcome measure assessment could not be analyzed to generate summary-level data. All participants enrolled in ECU-aHUS-403 were concurrently enrolled in protocol M11-001, and their data will be included in the analyses for M11-001.
Recruitment status
TERMINATED
Target enrollment
67 participants
Primary outcome timeframe
Baseline, 24 Months
Results posted on
2020-01-02
Participant Flow
Sixty-seven participants were enrolled in this study (15 pediatric and 52 adults). Treatment information was missing for 1 adult participant.
Participant milestones
| Measure |
Pediatric
Participants with atypical hemolytic uremic syndrome (aHUS) who were less than 18 years at baseline and who initiated treatment with eculizumab prior to study entry. Dosing regimen changed solely at the discretion of the treating physician.
|
Adult
Participants with aHUS who were 18 years or older at baseline and who initiated treatment with eculizumab prior to study entry. Dosing regimen changed solely at the discretion of the treating physician.
|
|---|---|---|
|
Overall Study
STARTED
|
15
|
52
|
|
Overall Study
Safety Population
|
15
|
52
|
|
Overall Study
COMPLETED
|
0
|
1
|
|
Overall Study
NOT COMPLETED
|
15
|
51
|
Reasons for withdrawal
| Measure |
Pediatric
Participants with atypical hemolytic uremic syndrome (aHUS) who were less than 18 years at baseline and who initiated treatment with eculizumab prior to study entry. Dosing regimen changed solely at the discretion of the treating physician.
|
Adult
Participants with aHUS who were 18 years or older at baseline and who initiated treatment with eculizumab prior to study entry. Dosing regimen changed solely at the discretion of the treating physician.
|
|---|---|---|
|
Overall Study
Participated until study termination
|
10
|
26
|
|
Overall Study
Lost to Follow-up
|
0
|
2
|
|
Overall Study
Physician Decision
|
1
|
8
|
|
Overall Study
Study Terminated by Sponsor
|
3
|
0
|
|
Overall Study
Withdrawal by Subject
|
0
|
2
|
|
Overall Study
Withdrawal by Parent/Guardian
|
1
|
1
|
|
Overall Study
Other-Additional Information Unavailable
|
0
|
11
|
|
Overall Study
Missing
|
0
|
1
|
Baseline Characteristics
Evaluation of Potential Predictors of Disease Progression in Participants With Atypical Hemolytic Uremic Syndrome (aHUS) Including Genetics, Biomarkers, and Treatment
Baseline characteristics by cohort
| Measure |
Pediatric
n=15 Participants
Participants with aHUS who were less than 18 years at baseline and who initiated treatment with eculizumab prior to study entry. Dosing regimen changed solely at the discretion of the treating physician.
|
Adult
n=52 Participants
Participants with aHUS who were 18 years or older at baseline and who initiated treatment with eculizumab prior to study entry. Dosing regimen changed solely at the discretion of the treating physician.
|
Total
n=67 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
7.9 years
STANDARD_DEVIATION 3.46 • n=5 Participants
|
43.3 years
STANDARD_DEVIATION 17.26 • n=7 Participants
|
35.4 years
STANDARD_DEVIATION 21.33 • n=5 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=5 Participants
|
34 Participants
n=7 Participants
|
40 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
9 Participants
n=5 Participants
|
18 Participants
n=7 Participants
|
27 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Caucasian
|
12 Participants
n=5 Participants
|
44 Participants
n=7 Participants
|
56 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Asian
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Black
|
1 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Other
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline, 24 MonthsPopulation: All enrolled participants. The study was terminated with only 20% of the planned enrollment; because the program lost funding, samples collected could not be analyzed to generate summary-level data. All participants enrolled in ECU-aHUS-403 were concurrently enrolled in protocol M11-001, and their data will be included in the analyses for M11-001.
A TMA manifestation was defined as one of following: Hematologic or renal events due to aHUS; extra-renal clinical signs and symptoms of aHUS; tissue (for example, kidney transplant) biopsy demonstrating TMA due to aHUS. The study was terminated with only 20% of the planned enrollment and due to the subsequent loss of funding for the program, the samples collected for outcome measure assessment could not be analyzed to generate summary-level data. All participants enrolled in ECU-aHUS-403 were concurrently enrolled in protocol M11-001, and their data will be included in the analyses for M11-001.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline, 24 MonthsPopulation: All enrolled participants. The study was terminated with only 20% of the planned enrollment; because the program lost funding, samples collected could not be analyzed to generate summary-level data. All participants enrolled in ECU-aHUS-403 were concurrently enrolled in protocol M11-001, and their data will be included in the analyses for M11-001.
Change in estimated eGFR over time using the chronic kidney disease-epidemiology (CKD-EPI) formula. The study was terminated with only 20% of the planned enrollment and due to the subsequent loss of funding for the program, the samples collected for outcome measure assessment could not be analyzed to generate summary-level data. All participants enrolled in ECU-aHUS-403 were concurrently enrolled in protocol M11-001, and their data will be included in the analyses for M11-001.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline, 24 MonthsPopulation: All enrolled participants. The study was terminated with only 20% of the planned enrollment; because the program lost funding, samples collected could not be analyzed to generate summary-level data. All participants enrolled in ECU-aHUS-403 were concurrently enrolled in protocol M11-001, and their data will be included in the analyses for M11-001.
The number of occurrences of PE/PI per participant-years was calculated and summarized by treatment status. The study was terminated with only 20% of the planned enrollment and due to the subsequent loss of funding for the program, the samples collected for outcome measure assessment could not be analyzed to generate summary-level data. All participants enrolled in ECU-aHUS-403 were concurrently enrolled in protocol M11-001, and their data will be included in the analyses for M11-001.
Outcome measures
Outcome data not reported
Adverse Events
Pediatric
Serious events: 1 serious events
Other events: 6 other events
Deaths: 0 deaths
Adult
Serious events: 14 serious events
Other events: 31 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Pediatric
n=15 participants at risk
Participants with aHUS who were less than 18 years at baseline and who initiated treatment with eculizumab prior to study entry. Dosing regimen changed solely at the discretion of the treating physician.
|
Adult
n=52 participants at risk
Participants with aHUS who were 18 years or older at baseline and who initiated treatment with eculizumab prior to study entry. Dosing regimen changed solely at the discretion of the treating physician.
|
|---|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
0.00%
0/15 • Day 0 through Month 24
|
3.8%
2/52 • Day 0 through Month 24
|
|
Blood and lymphatic system disorders
Neutropenia
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Endocrine disorders
Hyperparathyroidism
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Gastrointestinal disorders
Abdominal pain upper
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Gastrointestinal disorders
Diverticulum
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Gastrointestinal disorders
Duodenitis
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Gastrointestinal disorders
Gastrointestinal haemorrhage
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Gastrointestinal disorders
Melaena
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
General disorders
Chest pain
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
General disorders
Pyrexia
|
6.7%
1/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Hepatobiliary disorders
Liver injury
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Infections and infestations
Anal abscess
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Infections and infestations
Cellulitis streptococcal
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Infections and infestations
Cytomegalovirus viraemia
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Infections and infestations
Device related sepsis
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Infections and infestations
Diverticulitis
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Infections and infestations
Escherichia urinary tract infection
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Infections and infestations
Gastroenteritis
|
0.00%
0/15 • Day 0 through Month 24
|
3.8%
2/52 • Day 0 through Month 24
|
|
Infections and infestations
Influenza
|
6.7%
1/15 • Day 0 through Month 24
|
0.00%
0/52 • Day 0 through Month 24
|
|
Infections and infestations
Metapneumovirus infection
|
6.7%
1/15 • Day 0 through Month 24
|
0.00%
0/52 • Day 0 through Month 24
|
|
Infections and infestations
Rhinovirus infection
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Infections and infestations
Sepsis
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Infections and infestations
Tonsillitis
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Infections and infestations
Upper respiratory tract infection
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Infections and infestations
Urosepsis
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Infections and infestations
Viral infection
|
6.7%
1/15 • Day 0 through Month 24
|
0.00%
0/52 • Day 0 through Month 24
|
|
Injury, poisoning and procedural complications
Arteriovenous fistula occlusion
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Investigations
Liver function test abnormal
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Metabolism and nutrition disorders
Decreased appetite
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Metabolism and nutrition disorders
Fluid overload
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Metabolism and nutrition disorders
Gout
|
0.00%
0/15 • Day 0 through Month 24
|
3.8%
2/52 • Day 0 through Month 24
|
|
Metabolism and nutrition disorders
Hypercalcaemia
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Metabolism and nutrition disorders
Malnutrition
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Nervous system disorders
Altered state of consciousness
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Nervous system disorders
Encephalopathy
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Nervous system disorders
Headache
|
0.00%
0/15 • Day 0 through Month 24
|
3.8%
2/52 • Day 0 through Month 24
|
|
Nervous system disorders
Hemiparesis
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Nervous system disorders
Hepatic encephalopathy
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Nervous system disorders
Seizure
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Psychiatric disorders
Drug abuse
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Psychiatric disorders
Major depression
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Psychiatric disorders
Suicidal ideation
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Renal and urinary disorders
Acute kidney injury
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Renal and urinary disorders
Haematuria
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Renal and urinary disorders
Renal impairment
|
0.00%
0/15 • Day 0 through Month 24
|
3.8%
2/52 • Day 0 through Month 24
|
|
Respiratory, thoracic and mediastinal disorders
Acute lung injury
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Vascular disorders
Hypertension
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
Other adverse events
| Measure |
Pediatric
n=15 participants at risk
Participants with aHUS who were less than 18 years at baseline and who initiated treatment with eculizumab prior to study entry. Dosing regimen changed solely at the discretion of the treating physician.
|
Adult
n=52 participants at risk
Participants with aHUS who were 18 years or older at baseline and who initiated treatment with eculizumab prior to study entry. Dosing regimen changed solely at the discretion of the treating physician.
|
|---|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
6.7%
1/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Blood and lymphatic system disorders
Increased tendency to bruise
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Blood and lymphatic system disorders
Nephrogenic anaemia
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Blood and lymphatic system disorders
Neutropenia
|
0.00%
0/15 • Day 0 through Month 24
|
3.8%
2/52 • Day 0 through Month 24
|
|
Cardiac disorders
Bradycardia
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Cardiac disorders
Palpitations
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Cardiac disorders
Tachycardia
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Ear and labyrinth disorders
Ear discomfort
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Ear and labyrinth disorders
Inner ear inflammation
|
6.7%
1/15 • Day 0 through Month 24
|
0.00%
0/52 • Day 0 through Month 24
|
|
Ear and labyrinth disorders
Vertigo
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Eye disorders
Blindness unilateral
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Eye disorders
Eye colour change
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Eye disorders
Eye disorder
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Eye disorders
Ocular icterus
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Eye disorders
Orbital oedema
|
6.7%
1/15 • Day 0 through Month 24
|
0.00%
0/52 • Day 0 through Month 24
|
|
Gastrointestinal disorders
Abdominal distension
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/15 • Day 0 through Month 24
|
11.5%
6/52 • Day 0 through Month 24
|
|
Gastrointestinal disorders
Abdominal pain upper
|
0.00%
0/15 • Day 0 through Month 24
|
3.8%
2/52 • Day 0 through Month 24
|
|
Gastrointestinal disorders
Abdominal tenderness
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Gastrointestinal disorders
Anal incontinence
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Gastrointestinal disorders
Anorectal discomfort
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Gastrointestinal disorders
Bowel movement irregularity
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Gastrointestinal disorders
Chronic gastritis
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Gastrointestinal disorders
Colitis
|
0.00%
0/15 • Day 0 through Month 24
|
3.8%
2/52 • Day 0 through Month 24
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Gastrointestinal disorders
Crohn's disease
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Gastrointestinal disorders
Dental caries
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/15 • Day 0 through Month 24
|
13.5%
7/52 • Day 0 through Month 24
|
|
Gastrointestinal disorders
Duodenal ulcer
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Gastrointestinal disorders
Enterocolitis
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Gastrointestinal disorders
Faeces discoloured
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Gastrointestinal disorders
Gastric ulcer
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Gastrointestinal disorders
Gastritis
|
0.00%
0/15 • Day 0 through Month 24
|
3.8%
2/52 • Day 0 through Month 24
|
|
Gastrointestinal disorders
Gastrointestinal disorder
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Gastrointestinal disorders
Gastrointestinal erosion
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
0.00%
0/15 • Day 0 through Month 24
|
3.8%
2/52 • Day 0 through Month 24
|
|
Gastrointestinal disorders
Large intestine polyp
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Gastrointestinal disorders
Malabsorption
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Gastrointestinal disorders
Mouth haemorrhage
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/15 • Day 0 through Month 24
|
21.2%
11/52 • Day 0 through Month 24
|
|
Gastrointestinal disorders
Pancreatitis
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Gastrointestinal disorders
Rectal haemorrhage
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/15 • Day 0 through Month 24
|
7.7%
4/52 • Day 0 through Month 24
|
|
General disorders
Asthenia
|
0.00%
0/15 • Day 0 through Month 24
|
13.5%
7/52 • Day 0 through Month 24
|
|
General disorders
Catheter site erythema
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
General disorders
Chest discomfort
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
General disorders
Chest pain
|
0.00%
0/15 • Day 0 through Month 24
|
3.8%
2/52 • Day 0 through Month 24
|
|
General disorders
Extravasation
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
General disorders
Face oedema
|
6.7%
1/15 • Day 0 through Month 24
|
0.00%
0/52 • Day 0 through Month 24
|
|
General disorders
Fatigue
|
0.00%
0/15 • Day 0 through Month 24
|
11.5%
6/52 • Day 0 through Month 24
|
|
General disorders
Feeling hot
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
General disorders
Hypothermia
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
General disorders
Malaise
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
General disorders
Oedema peripheral
|
0.00%
0/15 • Day 0 through Month 24
|
3.8%
2/52 • Day 0 through Month 24
|
|
General disorders
Peripheral swelling
|
0.00%
0/15 • Day 0 through Month 24
|
5.8%
3/52 • Day 0 through Month 24
|
|
General disorders
Pyrexia
|
6.7%
1/15 • Day 0 through Month 24
|
5.8%
3/52 • Day 0 through Month 24
|
|
General disorders
Temperature intolerance
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Hepatobiliary disorders
Hepatic steatosis
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Infections and infestations
Aeromonas infection
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Infections and infestations
Bronchitis
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Infections and infestations
Catheter site infection
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Infections and infestations
Cellulitis
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Infections and infestations
Cytomegalovirus infection
|
0.00%
0/15 • Day 0 through Month 24
|
3.8%
2/52 • Day 0 through Month 24
|
|
Infections and infestations
Escherichia urinary tract infection
|
0.00%
0/15 • Day 0 through Month 24
|
3.8%
2/52 • Day 0 through Month 24
|
|
Infections and infestations
Gastroenteritis
|
6.7%
1/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Infections and infestations
Gastroenteritis viral
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Infections and infestations
Herpes simplex
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Infections and infestations
Lower respiratory tract infection
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Infections and infestations
Nasopharyngitis
|
0.00%
0/15 • Day 0 through Month 24
|
5.8%
3/52 • Day 0 through Month 24
|
|
Infections and infestations
Oral herpes
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Infections and infestations
Pseudomonas infection
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Infections and infestations
Rhinitis
|
6.7%
1/15 • Day 0 through Month 24
|
0.00%
0/52 • Day 0 through Month 24
|
|
Infections and infestations
Sinusitis
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Infections and infestations
Staphylococcal infection
|
0.00%
0/15 • Day 0 through Month 24
|
3.8%
2/52 • Day 0 through Month 24
|
|
Infections and infestations
Tonsillitis
|
0.00%
0/15 • Day 0 through Month 24
|
3.8%
2/52 • Day 0 through Month 24
|
|
Infections and infestations
Upper respiratory tract infection
|
6.7%
1/15 • Day 0 through Month 24
|
21.2%
11/52 • Day 0 through Month 24
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/15 • Day 0 through Month 24
|
5.8%
3/52 • Day 0 through Month 24
|
|
Infections and infestations
Varicella zoster virus infection
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Infections and infestations
Viral pharyngitis
|
6.7%
1/15 • Day 0 through Month 24
|
0.00%
0/52 • Day 0 through Month 24
|
|
Infections and infestations
Vulvovaginal candidiasis
|
0.00%
0/6 • Day 0 through Month 24
|
5.9%
2/34 • Day 0 through Month 24
|
|
Injury, poisoning and procedural complications
Anal injury
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Injury, poisoning and procedural complications
Contusion
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Injury, poisoning and procedural complications
Corneal abrasion
|
6.7%
1/15 • Day 0 through Month 24
|
0.00%
0/52 • Day 0 through Month 24
|
|
Injury, poisoning and procedural complications
Ligament sprain
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Injury, poisoning and procedural complications
Procedural dizziness
|
6.7%
1/15 • Day 0 through Month 24
|
0.00%
0/52 • Day 0 through Month 24
|
|
Injury, poisoning and procedural complications
Procedural nausea
|
6.7%
1/15 • Day 0 through Month 24
|
0.00%
0/52 • Day 0 through Month 24
|
|
Injury, poisoning and procedural complications
Procedural pain
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Injury, poisoning and procedural complications
Skin abrasion
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Injury, poisoning and procedural complications
Wound
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Investigations
Blood calcium increased
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Investigations
Blood creatinine increased
|
0.00%
0/15 • Day 0 through Month 24
|
9.6%
5/52 • Day 0 through Month 24
|
|
Investigations
Blood lactate dehydrogenase increased
|
0.00%
0/15 • Day 0 through Month 24
|
3.8%
2/52 • Day 0 through Month 24
|
|
Investigations
Blood magnesium decreased
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Investigations
Bone density decreased
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Investigations
Faecal calprotectin increased
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Investigations
Faecal elastase concentration decreased
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Investigations
Haemoglobin decreased
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Investigations
Haptoglobin decreased
|
6.7%
1/15 • Day 0 through Month 24
|
0.00%
0/52 • Day 0 through Month 24
|
|
Investigations
Liver function test increased
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Investigations
Red blood cell schistocytes present
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Investigations
Weight decreased
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Metabolism and nutrition disorders
Gout
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Metabolism and nutrition disorders
Hypercalcaemia
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Metabolism and nutrition disorders
Hypernatraemia
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Metabolism and nutrition disorders
Hypoalbuminaemia
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Metabolism and nutrition disorders
Hypocalcaemia
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
0.00%
0/15 • Day 0 through Month 24
|
5.8%
3/52 • Day 0 through Month 24
|
|
Metabolism and nutrition disorders
Hypomagnesaemia
|
0.00%
0/15 • Day 0 through Month 24
|
3.8%
2/52 • Day 0 through Month 24
|
|
Metabolism and nutrition disorders
Hypophosphataemia
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Metabolism and nutrition disorders
Malnutrition
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Metabolism and nutrition disorders
Metabolic acidosis
|
0.00%
0/15 • Day 0 through Month 24
|
5.8%
3/52 • Day 0 through Month 24
|
|
Metabolism and nutrition disorders
Vitamin B12 deficiency
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Metabolism and nutrition disorders
Vitamin D deficiency
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
0.00%
0/15 • Day 0 through Month 24
|
3.8%
2/52 • Day 0 through Month 24
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.00%
0/15 • Day 0 through Month 24
|
3.8%
2/52 • Day 0 through Month 24
|
|
Musculoskeletal and connective tissue disorders
Flank pain
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Musculoskeletal and connective tissue disorders
Joint swelling
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Musculoskeletal and connective tissue disorders
Muscular weakness
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal stiffness
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Musculoskeletal and connective tissue disorders
Osteopenia
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
0.00%
0/15 • Day 0 through Month 24
|
5.8%
3/52 • Day 0 through Month 24
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Angiolipoma
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Skin cancer
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Nervous system disorders
Dizziness
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Nervous system disorders
Dizziness postural
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Nervous system disorders
Headache
|
6.7%
1/15 • Day 0 through Month 24
|
19.2%
10/52 • Day 0 through Month 24
|
|
Nervous system disorders
Hypotonia
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Nervous system disorders
Lethargy
|
0.00%
0/15 • Day 0 through Month 24
|
3.8%
2/52 • Day 0 through Month 24
|
|
Nervous system disorders
Migraine
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Nervous system disorders
Poor quality sleep
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Nervous system disorders
Somnolence
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Nervous system disorders
Tremor
|
0.00%
0/15 • Day 0 through Month 24
|
3.8%
2/52 • Day 0 through Month 24
|
|
Psychiatric disorders
Anxiety
|
0.00%
0/15 • Day 0 through Month 24
|
5.8%
3/52 • Day 0 through Month 24
|
|
Psychiatric disorders
Confusional state
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Psychiatric disorders
Depression
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Psychiatric disorders
Insomnia
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Psychiatric disorders
Irritability
|
0.00%
0/15 • Day 0 through Month 24
|
9.6%
5/52 • Day 0 through Month 24
|
|
Renal and urinary disorders
Dysuria
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Renal and urinary disorders
Proteinuria
|
0.00%
0/15 • Day 0 through Month 24
|
3.8%
2/52 • Day 0 through Month 24
|
|
Renal and urinary disorders
Renal impairment
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Renal and urinary disorders
Renal tubular acidosis
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Reproductive system and breast disorders
Amenorrhoea
|
0.00%
0/6 • Day 0 through Month 24
|
2.9%
1/34 • Day 0 through Month 24
|
|
Reproductive system and breast disorders
Breast cyst
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
0.00%
0/15 • Day 0 through Month 24
|
9.6%
5/52 • Day 0 through Month 24
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
13.3%
2/15 • Day 0 through Month 24
|
0.00%
0/52 • Day 0 through Month 24
|
|
Respiratory, thoracic and mediastinal disorders
Nasal discomfort
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
0.00%
0/15 • Day 0 through Month 24
|
3.8%
2/52 • Day 0 through Month 24
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Skin and subcutaneous tissue disorders
Acne
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Skin and subcutaneous tissue disorders
Actinic keratosis
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Skin and subcutaneous tissue disorders
Blister
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Skin and subcutaneous tissue disorders
Neurodermatitis
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Skin and subcutaneous tissue disorders
Onychoclasis
|
6.7%
1/15 • Day 0 through Month 24
|
0.00%
0/52 • Day 0 through Month 24
|
|
Skin and subcutaneous tissue disorders
Rash
|
6.7%
1/15 • Day 0 through Month 24
|
0.00%
0/52 • Day 0 through Month 24
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Vascular disorders
Hypertension
|
6.7%
1/15 • Day 0 through Month 24
|
11.5%
6/52 • Day 0 through Month 24
|
|
Vascular disorders
Hypotension
|
0.00%
0/15 • Day 0 through Month 24
|
9.6%
5/52 • Day 0 through Month 24
|
|
Vascular disorders
Thrombophlebitis
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
|
Vascular disorders
Venous thrombosis limb
|
0.00%
0/15 • Day 0 through Month 24
|
1.9%
1/52 • Day 0 through Month 24
|
Additional Information
Alexion Pharmaceuticals, Inc.
Alexion Pharmaceuticals, Inc.
Phone: 475-230-2596
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place