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Study of Ravulizumab in Children and Adolescents With Atypical Hemolytic Uremic Syndrome (aHUS)

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ClinicalTrials.gov Identifier: NCT03131219
Recruitment Status : Active, not recruiting
First Posted : April 27, 2017
Results First Posted : September 16, 2020
Last Update Posted : September 16, 2020
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Atypical Hemolytic Uremic Syndrome (aHUS)
Intervention Biological: Ravulizumab
Enrollment 31
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Complement Inhibitor Treatment Naïve Eculizumab Experienced
Hide Arm/Group Description Complement inhibitor treatment-naïve participants received weight-based doses of ravulizumab during the 26-week Initial Evaluation Period. After the Initial Evaluation Period, participants rolled over into an Extension Period in which all participants continued their weight-based maintenance dose of ravulizumab. Eculizumab-experienced participants received weight-based doses of ravulizumab during the 26-week Initial Evaluation Period. After the Initial Evaluation Period, participants rolled over into an Extension Period in which all participants continued their weight-based maintenance dose of ravulizumab.
Period Title: Initial Evaluation Period
Started 21 10
Received At Least 1 Dose of Study Drug 21 10
Completed 17 10
Not Completed 4 0
Reason Not Completed
Adverse Event             1             0
Protocol Violation             1             0
Deemed ineligible post treatment             2             0
Period Title: Extension Period
Started 17 10
Received At Least 1 Dose of Study Drug 17 10
Ongoing [1] 16 10
Completed 0 0
Not Completed 17 10
[1]
Extension Period ongoing
Arm/Group Title Complement Inhibitor Treatment Naïve Eculizumab Experienced Total
Hide Arm/Group Description Complement inhibitor treatment-naïve participants received weight-based doses of ravulizumab during the 26-week Initial Evaluation Period. After the Initial Evaluation Period, participants rolled over into an Extension Period in which all participants continued their weight-based maintenance dose of ravulizumab. Eculizumab-experienced participants received weight-based doses of ravulizumab during the 26-week Initial Evaluation Period. After the Initial Evaluation Period, participants rolled over into an Extension Period in which all participants continued their weight-based maintenance dose of ravulizumab. Total of all reporting groups
Overall Number of Baseline Participants 21 10 31
Hide Baseline Analysis Population Description
Safety: All participants who received at least 1 dose of study drug.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 21 participants 10 participants 31 participants
6.7  (4.78) 11.0  (4.97) 8.1  (5.17)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 21 participants 10 participants 31 participants
Female
11
  52.4%
1
  10.0%
12
  38.7%
Male
10
  47.6%
9
  90.0%
19
  61.3%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 21 participants 10 participants 31 participants
Hispanic or Latino
2
   9.5%
1
  10.0%
3
   9.7%
Not Hispanic or Latino
19
  90.5%
9
  90.0%
28
  90.3%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 21 participants 10 participants 31 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
7
  33.3%
4
  40.0%
11
  35.5%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
3
  14.3%
1
  10.0%
4
  12.9%
White
9
  42.9%
5
  50.0%
14
  45.2%
More than one race
1
   4.8%
0
   0.0%
1
   3.2%
Unknown or Not Reported
1
   4.8%
0
   0.0%
1
   3.2%
Weight at Time of First Infusion  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 21 participants 10 participants 31 participants
≥5 to <10 kilograms
3
  14.3%
1
  10.0%
4
  12.9%
≥10 to <20 kilograms
9
  42.9%
1
  10.0%
10
  32.3%
≥20 to <30 kilograms
3
  14.3%
1
  10.0%
4
  12.9%
≥30 to <40 kilograms
3
  14.3%
1
  10.0%
4
  12.9%
≥40 to <60 kilograms
2
   9.5%
5
  50.0%
7
  22.6%
≥60 to <100 kilograms
1
   4.8%
1
  10.0%
2
   6.5%
Dialysis at Baseline  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 21 participants 10 participants 31 participants
6
  28.6%
0
   0.0%
6
  19.4%
Baseline Estimated Glomerular Filtration Rate (eGFR)   [1] 
Mean (Standard Deviation)
Unit of measure:  mL/min/1.73 m^2
Number Analyzed 18 participants 10 participants 28 participants
26.4  (21.17) 104.90  (29.545) NA [2]   (NA)
[1]
Measure Analysis Population Description: Full Analysis Set (FAS): all participants who received at least 1 dose of ravulizumab, had at least 1 post-baseline efficacy assessment, and met pre-specified eligibility criteria.
[2]
This value was not calculated.
Baseline Chronic Kidney Disease (CKD) Stage   [1] [2] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 18 participants 10 participants 28 participants
Stage 1
0
   0.0%
8
  80.0%
8
  28.6%
Stage 2
2
  11.1%
1
  10.0%
3
  10.7%
Stage 3a
1
   5.6%
1
  10.0%
2
   7.1%
Stage 3b
1
   5.6%
0
   0.0%
1
   3.6%
Stage 4
8
  44.4%
0
   0.0%
8
  28.6%
Stage 5
6
  33.3%
0
   0.0%
6
  21.4%
[1]
Measure Description: CKD stage was classified by eGFR (milliliters/minute/1.73 meters squared [mL/min/1.73 m^2]): eGFR ≥90 = Stage 1 (normal or above normal renal function); eGFR 60-89 = Stage 2 (mildly decreased renal function); eGFR 30-59 = Stage 3 (moderately decreased renal function); eGFR 15-29 = Stage 4 (severely decreased renal function); eGFR <15 = Stage 5 (kidney failure).
[2]
Measure Analysis Population Description: Full Analysis Set (FAS): all participants who received at least 1 dose of ravulizumab, had at least 1 post-baseline efficacy assessment, and met pre-specified eligibility criteria.
1.Primary Outcome
Title Proportion Of Complement Inhibitor Treatment-naïve Participants With Complete Thrombotic Microangiopathy (TMA) Response
Hide Description Complete TMA response during the 26-week Initial Evaluation Period is a composite outcome measure that required normalization of hematological parameters (platelet count and lactate dehydrogenase [LDH]) and improvement in kidney function (≥25% reduction in serum creatinine from baseline); for participants on dialysis, baseline was established at least 6 days after the end of dialysis. Participants had to meet these criteria for 2 separate assessments obtained at least 4 weeks (28 days) apart, and any measurement in between. To be considered a responder during the 26-week Initial Evaluation Period, the latest time point a participant could first meet the response criteria was 28 days before the Week 26 (Day 183) assessment. Formal statistical comparison analyses were not planned for this study. Proportion based on the responders among treated participants. Confidence interval (CI) based on exact confidence limits using the Clopper Pearson method.
Time Frame Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): all participants who received at least 1 dose of ravulizumab, had at least 1 post-baseline efficacy assessment, and met pre-specified eligibility criteria.
Arm/Group Title Complement Inhibitor Treatment Naïve
Hide Arm/Group Description:
Complement inhibitor treatment-naïve participants received weight-based doses of ravulizumab during the 26-week Initial Evaluation Period. After the Initial Evaluation Period, participants rolled over into an Extension Period in which all participants continued their weight-based maintenance dose of ravulizumab.
Overall Number of Participants Analyzed 18
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: proportion of participants
0.778
(0.524 to 0.936)
2.Secondary Outcome
Title Time To Complete TMA Response In Complement Inhibitor Treatment-naïve Participants
Hide Description Participants that did not have a response were censored at the date of last visit or study discontinuation at the time when the analysis was performed. The time to complete TMA Response is reported in days. The time of the event of a confirmed complete TMA response was considered the first time point at which all the criteria for complete TMA response were met. Participants had to meet all complete TMA response criteria at 2 separate assessments obtained at least 4 weeks (28 days) apart, and any measurement in between.
Time Frame Baseline through at least Week 52 and up to Week 111
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): all participants who received at least 1 dose of ravulizumab, had at least 1 post-baseline efficacy assessment, and met pre-specified eligibility criteria.
Arm/Group Title Complement Inhibitor Treatment Naïve
Hide Arm/Group Description:
Complement inhibitor treatment-naïve participants received weight-based doses of ravulizumab during the 26-week Initial Evaluation Period. After the Initial Evaluation Period, participants rolled over into an Extension Period in which all participants continued their weight-based maintenance dose of ravulizumab.
Overall Number of Participants Analyzed 18
Median (Inter-Quartile Range)
Unit of Measure: days
30.0
(22.0 to 88.0)
3.Secondary Outcome
Title Participants Who Do Not Require Dialysis
Hide Description For participants requiring dialysis within 5 days prior to ALXN1210 treatment initiation, the number of participants no longer requiring dialysis is reported.
Time Frame Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): all participants who received at least 1 dose of ravulizumab, had at least 1 post-baseline efficacy assessment, met pre-specified eligibility criteria, and had evaluable data at specified timepoint.
Arm/Group Title Complement Inhibitor Treatment Naïve Eculizumab Experienced
Hide Arm/Group Description:
Complement inhibitor treatment-naïve participants received weight-based doses of ravulizumab during the 26-week Initial Evaluation Period. After the Initial Evaluation Period, participants rolled over into an Extension Period in which all participants continued their weight-based maintenance dose of ravulizumab.
Eculizumab-experienced participants received weight-based doses of ravulizumab during the 26-week Initial Evaluation Period. After the Initial Evaluation Period, participants rolled over into an Extension Period in which all participants continued their weight-based maintenance dose of ravulizumab.
Overall Number of Participants Analyzed 6 0
Measure Type: Count of Participants
Unit of Measure: Participants
5
  83.3%
0
4.Secondary Outcome
Title Proportion Of Complement Inhibitor Treatment-naïve Participants With Complete TMA Response Through All Available Follow-Up
Hide Description The proportion of participants considered responders, along with a 2-sided 95% CI based on exact confidence limits using the Clopper Pearson method through all available follow-up (up to Week 111), is reported.
Time Frame At least 52 weeks and up to a maximum of 111 weeks of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): all participants who received at least 1 dose of ravulizumab, had at least 1 post-baseline efficacy assessment, met pre-specified eligibility criteria, and had evaluable data at specified timepoint.
Arm/Group Title Complement Inhibitor Treatment Naïve
Hide Arm/Group Description:
Complement inhibitor treatment-naïve participants received weight-based doses of ravulizumab during the 26-week Initial Evaluation Period. After the Initial Evaluation Period, participants rolled over into an Extension Period in which all participants continued their weight-based maintenance dose of ravulizumab.
Overall Number of Participants Analyzed 18
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: proportion of participants
0.944
(0.727 to 0.999)
5.Secondary Outcome
Title Observed Value And Change From Baseline In eGFR At Week 26
Hide Description Kidney function evaluated by eGFR was summarized at baseline and the Week 26 time point using descriptive statistics for continuous variables for the observed value, as well as the change from baseline. The baseline value was defined as the average of the values from the assessments performed prior to the first study drug infusion (these could include results from Screening and the Day 1 visit). A value of 10 mL/min/1.73 m^2 for eGFR was imputed for participants requiring dialysis for acute kidney injury. The observed value and change from baseline are reported in mL/min/1.73 m^2. An increase indicated improvement in kidney function.
Time Frame Baseline, Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): all participants who received at least 1 dose of ravulizumab, had at least 1 post-baseline efficacy assessment, met pre-specified eligibility criteria, and had evaluable data at specified timepoint (Week 26).
Arm/Group Title Complement Inhibitor Treatment Naïve Eculizumab Experienced
Hide Arm/Group Description:
Complement inhibitor treatment-naïve participants received weight-based doses of ravulizumab during the 26-week Initial Evaluation Period. After the Initial Evaluation Period, participants rolled over into an Extension Period in which all participants continued their weight-based maintenance dose of ravulizumab.
Eculizumab-experienced participants received weight-based doses of ravulizumab during the 26-week Initial Evaluation Period. After the Initial Evaluation Period, participants rolled over into an Extension Period in which all participants continued their weight-based maintenance dose of ravulizumab.
Overall Number of Participants Analyzed 18 10
Mean (Standard Deviation)
Unit of Measure: mL/min/1.73 m^2
Observed Value Number Analyzed 17 participants 10 participants
108.5  (56.87) 94.00  (35.131)
Change From Baseline Number Analyzed 17 participants 10 participants
85.4  (54.33) -10.90  (30.584)
6.Secondary Outcome
Title Participants With Change From Baseline In CKD Stage At Week 26
Hide Description The CKD stage is presented as the change from baseline in the participants that Improved (excluding those with Stage 1 [normal renal function] at baseline as they cannot improve), Worsened (excluding those with Stage 5 at baseline as they cannot worsen), and Stayed the Same, compared to the CKD stage at baseline. Baseline was derived based on the last available eGFR before starting treatment. Stage 5 was considered the worst category, while Stage 1 was considered the best category. A 2-sided 95% CI for the proportion, based on exact confidence limits using the Clopper-Pearson method, was provided for each category. The CKD stage was classified based on the National Kidney Foundation Chronic Kidney Disease Stage.
Time Frame Baseline, Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): all participants who received at least 1 dose of ravulizumab, had at least 1 post-baseline efficacy assessment, met pre-specified eligibility criteria, and had evaluable data at specified timepoint (Week 26).
Arm/Group Title Complement Inhibitor Treatment Naïve Eculizumab Experienced
Hide Arm/Group Description:
Complement inhibitor treatment-naïve participants received weight-based doses of ravulizumab during the 26-week Initial Evaluation Period. After the Initial Evaluation Period, participants rolled over into an Extension Period in which all participants continued their weight-based maintenance dose of ravulizumab.
Eculizumab-experienced participants received weight-based doses of ravulizumab during the 26-week Initial Evaluation Period. After the Initial Evaluation Period, participants rolled over into an Extension Period in which all participants continued their weight-based maintenance dose of ravulizumab.
Overall Number of Participants Analyzed 17 10
Measure Type: Count of Participants
Unit of Measure: Participants
Improved
15
  88.2%
0
   0.0%
Worsened
0
   0.0%
3
  30.0%
Stayed the Same
2
  11.8%
7
  70.0%
7.Secondary Outcome
Title Change From Baseline In Platelet Count At Week 26
Hide Description The hematologic TMA parameter of platelet count was summarized at baseline and at Week 26 using descriptive statistics for continuous variables for the change from baseline. Results are reported in platelets*10^9/liter (L) blood.
Time Frame Baseline, Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): all participants who received at least 1 dose of ravulizumab, had at least 1 post-baseline efficacy assessment, met pre-specified eligibility criteria, and had evaluable data at specified timepoint (Week 26).
Arm/Group Title Complement Inhibitor Treatment Naïve Eculizumab Experienced
Hide Arm/Group Description:
Complement inhibitor treatment-naïve participants received weight-based doses of ravulizumab during the 26-week Initial Evaluation Period. After the Initial Evaluation Period, participants rolled over into an Extension Period in which all participants continued their weight-based maintenance dose of ravulizumab.
Eculizumab-experienced participants received weight-based doses of ravulizumab during the 26-week Initial Evaluation Period. After the Initial Evaluation Period, participants rolled over into an Extension Period in which all participants continued their weight-based maintenance dose of ravulizumab.
Overall Number of Participants Analyzed 17 10
Mean (Standard Deviation)
Unit of Measure: platelets*10^9/L
245.59  (91.827) 6.70  (55.202)
8.Secondary Outcome
Title Change From Baseline In LDH At Week 26
Hide Description The hematologic TMA parameter of serum LDH was summarized at baseline and at Week 26 using descriptive statistics for continuous variables for the change from baseline. Results are reported in units (U)/L serum.
Time Frame Baseline, Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): all participants who received at least 1 dose of ravulizumab, had at least 1 post-baseline efficacy assessment, met pre-specified eligibility criteria, and had evaluable data at specified timepoint (Week 26).
Arm/Group Title Complement Inhibitor Treatment Naïve Eculizumab Experienced
Hide Arm/Group Description:
Complement inhibitor treatment-naïve participants received weight-based doses of ravulizumab during the 26-week Initial Evaluation Period. After the Initial Evaluation Period, participants rolled over into an Extension Period in which all participants continued their weight-based maintenance dose of ravulizumab.
Eculizumab-experienced participants received weight-based doses of ravulizumab during the 26-week Initial Evaluation Period. After the Initial Evaluation Period, participants rolled over into an Extension Period in which all participants continued their weight-based maintenance dose of ravulizumab.
Overall Number of Participants Analyzed 16 10
Mean (Standard Deviation)
Unit of Measure: U/L
-2044.13  (1328.059) -3.40  (30.261)
9.Secondary Outcome
Title Change From Baseline In Hemoglobin At Week 26
Hide Description The hematologic TMA parameter of hemoglobin level was summarized at baseline and at Week 26 using descriptive statistics for continuous variables for the change from baseline. Results are reported in grams (g)/L blood.
Time Frame Baseline, Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): all participants who received at least 1 dose of ravulizumab, had at least 1 post-baseline efficacy assessment, met pre-specified eligibility criteria, and had evaluable data at specified timepoint (Week 26).
Arm/Group Title Complement Inhibitor Treatment Naïve Eculizumab Experienced
Hide Arm/Group Description:
Complement inhibitor treatment-naïve participants received weight-based doses of ravulizumab during the 26-week Initial Evaluation Period. After the Initial Evaluation Period, participants rolled over into an Extension Period in which all participants continued their weight-based maintenance dose of ravulizumab.
Eculizumab-experienced participants received weight-based doses of ravulizumab during the 26-week Initial Evaluation Period. After the Initial Evaluation Period, participants rolled over into an Extension Period in which all participants continued their weight-based maintenance dose of ravulizumab.
Overall Number of Participants Analyzed 17 10
Mean (Standard Deviation)
Unit of Measure: g/L
46.50  (16.743) -4.10  (7.086)
10.Secondary Outcome
Title Proportion Of Complement Inhibitor Treatment-naïve Participants With An Increase From Baseline In Hemoglobin ≥20 g/L Through Week 26
Hide Description The proportion of participants with an increase from baseline in hemoglobin ≥20 g/L, observed at 2 separate assessments obtained at least 4 weeks (28 days) apart, and any measurement in between, was assessed through Week 26 and is presented as the proportion of responders, along with a 2-sided 95% CI. The 95% CIs are based on exact confidence limits using the Clopper-Pearson method. To be considered a responder during the 26-week Initial Evaluation Period, the latest time point a participant could first meet the response criteria was 28 days before the Week 26 (Day 183) assessment (components of the response maintained for at least 28 days).
Time Frame Baseline through Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): all participants who received at least 1 dose of ravulizumab, had at least 1 post-baseline efficacy assessment, met pre-specified eligibility criteria, and had evaluable data at specified timepoint (Week 26).
Arm/Group Title Complement Inhibitor Treatment Naïve
Hide Arm/Group Description:
Complement inhibitor treatment-naïve participants received weight-based doses of ravulizumab during the 26-week Initial Evaluation Period. After the Initial Evaluation Period, participants rolled over into an Extension Period in which all participants continued their weight-based maintenance dose of ravulizumab.
Overall Number of Participants Analyzed 18
Median (95% Confidence Interval)
Unit of Measure: proportion of participants
0.889
(0.653 to 0.986)
11.Secondary Outcome
Title Change From Baseline In Quality Of Life As Measured By The Functional Assessment Of Chronic Illness Therapy (FACIT)-Fatigue Version 4 Questionnaire (Participants ≥5 Years Of Age) At Week 26
Hide Description Quality of life was assessed in participants >5 years of age by the Pediatric FACIT-Fatigue Questionnaire (reported by participants who were ≥8 years of age at the time of enrollment; caregiver reported or caregiver assistance for participants who were 5 to <8 years of age at the time of enrollment). The FACIT Fatigue data were summarized at baseline and each post baseline time point using descriptive statistics for continuous variables for the observed value as well as the change from baseline. The FACIT Fatigue Version 4 questionnaire at baseline and each post-infusion time point was scored using standard scoring algorithms. The score ranges from 0 to 52, with a higher score indicating less fatigue. An increase in score indicated an improvement in quality of life.
Time Frame Baseline, Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): all participants who received at least 1 dose of ravulizumab, had at least 1 post-baseline efficacy assessment, met pre-specified eligibility criteria, and had evaluable data at specified timepoint (Week 26).
Arm/Group Title Complement Inhibitor Treatment Naïve Eculizumab Experienced
Hide Arm/Group Description:
Complement inhibitor treatment-naïve participants received weight-based doses of ravulizumab during the 26-week Initial Evaluation Period. After the Initial Evaluation Period, participants rolled over into an Extension Period in which all participants continued their weight-based maintenance dose of ravulizumab.
Eculizumab-experienced participants received weight-based doses of ravulizumab during the 26-week Initial Evaluation Period. After the Initial Evaluation Period, participants rolled over into an Extension Period in which all participants continued their weight-based maintenance dose of ravulizumab.
Overall Number of Participants Analyzed 9 8
Mean (Standard Deviation)
Unit of Measure: units on a scale
16.78  (14.704) 0.00  (2.268)
Time Frame From the beginning of the initial evaluation period (Day 1) through data cutoff (at least 52 weeks and up to a maximum of 111 weeks of treatment, representing 36.2 patient-years of exposure).
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Complement Inhibitor Treatment Naïve Eculizumab Experienced
Hide Arm/Group Description Complement inhibitor treatment-naïve participants received weight-based doses of ravulizumab during the 26-week Initial Evaluation Period. After the Initial Evaluation Period, participants rolled over into an Extension Period in which all participants continued their weight-based maintenance dose of ravulizumab. Eculizumab-experienced participants received weight-based doses of ravulizumab during the 26-week Initial Evaluation Period. After the Initial Evaluation Period, participants rolled over into an Extension Period in which all participants continued their weight-based maintenance dose of ravulizumab.
All-Cause Mortality
Complement Inhibitor Treatment Naïve Eculizumab Experienced
Affected / at Risk (%) Affected / at Risk (%)
Total   0/21 (0.00%)   0/10 (0.00%) 
Hide Serious Adverse Events
Complement Inhibitor Treatment Naïve Eculizumab Experienced
Affected / at Risk (%) Affected / at Risk (%)
Total   14/21 (66.67%)   1/10 (10.00%) 
Blood and lymphatic system disorders     
Anaemia  1  1/21 (4.76%)  0/10 (0.00%) 
Congenital, familial and genetic disorders     
Atrial septal defect  1  1/21 (4.76%)  0/10 (0.00%) 
Gastrointestinal disorders     
Abdominal pain  1  2/21 (9.52%)  0/10 (0.00%) 
Diarrhoea  1  1/21 (4.76%)  0/10 (0.00%) 
Pancreatitis  1  1/21 (4.76%)  0/10 (0.00%) 
Vomiting  1  1/21 (4.76%)  0/10 (0.00%) 
General disorders     
Pyrexia  1  1/21 (4.76%)  0/10 (0.00%) 
Infections and infestations     
Gastroenteritis viral  1  2/21 (9.52%)  0/10 (0.00%) 
Bronchitis  1  1/21 (4.76%)  1/10 (10.00%) 
Cytomegalovirus enteritis  1  1/21 (4.76%)  0/10 (0.00%) 
Escherichia bacteraemia  1  1/21 (4.76%)  0/10 (0.00%) 
Escherichia pyelonephritis  1  1/21 (4.76%)  0/10 (0.00%) 
Gastroenteritis  1  1/21 (4.76%)  0/10 (0.00%) 
Gastroenteritis rotavirus  1  1/21 (4.76%)  0/10 (0.00%) 
Human bocavirus infection  1  1/21 (4.76%)  0/10 (0.00%) 
Pharyngitis  1  1/21 (4.76%)  0/10 (0.00%) 
Pneumonia  1  1/21 (4.76%)  1/10 (10.00%) 
Viral infection  1  1/21 (4.76%)  0/10 (0.00%) 
Viral pharyngitis  1  1/21 (4.76%)  0/10 (0.00%) 
Upper respiratory tract infection  1  0/21 (0.00%)  1/10 (10.00%) 
Injury, poisoning and procedural complications     
Subdural haematoma  1  1/21 (4.76%)  0/10 (0.00%) 
Metabolism and nutrition disorders     
Decreased appetite  1  1/21 (4.76%)  0/10 (0.00%) 
Musculoskeletal and connective tissue disorders     
Myalgia  1  1/21 (4.76%)  0/10 (0.00%) 
Nervous system disorders     
Headache  1  1/21 (4.76%)  0/10 (0.00%) 
Posterior reversible encephalopathy syndrome  1  1/21 (4.76%)  0/10 (0.00%) 
Respiratory, thoracic and mediastinal disorders     
Pulmonary haemorrhage  1  1/21 (4.76%)  0/10 (0.00%) 
Vascular disorders     
Hypertension  1  1/21 (4.76%)  0/10 (0.00%) 
Hypertensive crisis  1  1/21 (4.76%)  0/10 (0.00%) 
1
Term from vocabulary, MedDRA 21.0
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Complement Inhibitor Treatment Naïve Eculizumab Experienced
Affected / at Risk (%) Affected / at Risk (%)
Total   21/21 (100.00%)   10/10 (100.00%) 
Blood and lymphatic system disorders     
Lymphadenopathy  1  2/21 (9.52%)  1/10 (10.00%) 
Anaemia  1  2/21 (9.52%)  0/10 (0.00%) 
Eye disorders     
Lacrimation increased  1  0/21 (0.00%)  1/10 (10.00%) 
Photophobia  1  0/21 (0.00%)  1/10 (10.00%) 
Gastrointestinal disorders     
Abdominal pain  1  5/21 (23.81%)  1/10 (10.00%) 
Diarrhoea  1  6/21 (28.57%)  1/10 (10.00%) 
Dyspepsia  1  0/21 (0.00%)  1/10 (10.00%) 
Lip dry  1  0/21 (0.00%)  1/10 (10.00%) 
Vomiting  1  7/21 (33.33%)  1/10 (10.00%) 
Constipation  1  4/21 (19.05%)  0/10 (0.00%) 
Nausea  1  4/21 (19.05%)  0/10 (0.00%) 
Abdominal distension  1  2/21 (9.52%)  0/10 (0.00%) 
General disorders     
Fatigue  1  2/21 (9.52%)  1/10 (10.00%) 
Pyrexia  1  10/21 (47.62%)  0/10 (0.00%) 
Infections and infestations     
Upper respiratory tract infection  1  3/21 (14.29%)  4/10 (40.00%) 
Nasopharyngitis  1  7/21 (33.33%)  2/10 (20.00%) 
Otitis media  1  0/21 (0.00%)  2/10 (20.00%) 
Pharyngitis  1  2/21 (9.52%)  2/10 (20.00%) 
Viral upper respiratory tract infection  1  2/21 (9.52%)  2/10 (20.00%) 
Bronchitis  1  0/21 (0.00%)  1/10 (10.00%) 
Hand-foot-and-mouth disease  1  0/21 (0.00%)  1/10 (10.00%) 
Impetigo  1  0/21 (0.00%)  1/10 (10.00%) 
Influenza  1  0/21 (0.00%)  1/10 (10.00%) 
Pneumonia  1  2/21 (9.52%)  1/10 (10.00%) 
Viral infection  1  0/21 (0.00%)  1/10 (10.00%) 
Tonsilitis  1  3/21 (14.29%)  0/10 (0.00%) 
Conjunctivitis  1  2/21 (9.52%)  0/10 (0.00%) 
Sinusitis  1  2/21 (9.52%)  0/10 (0.00%) 
Injury, poisoning and procedural complications     
Contusion  1  4/21 (19.05%)  0/10 (0.00%) 
Head injury  1  2/21 (9.52%)  0/10 (0.00%) 
Skin abrasion  1  2/21 (9.52%)  0/10 (0.00%) 
Investigations     
Influenza A virus test positive  1  0/21 (0.00%)  1/10 (10.00%) 
Vitamin D decreased  1  3/21 (14.29%)  0/10 (0.00%) 
Metabolism and nutrition disorders     
Dehydration  1  0/21 (0.00%)  1/10 (10.00%) 
Iron deficiency  1  3/21 (14.29%)  0/10 (0.00%) 
Musculoskeletal and connective tissue disorders     
Back pain  1  2/21 (9.52%)  1/10 (10.00%) 
Osteochondrosis  1  0/21 (0.00%)  1/10 (10.00%) 
Pain in extremity  1  2/21 (9.52%)  1/10 (10.00%) 
Tenosynovitis  1  0/21 (0.00%)  1/10 (10.00%) 
Myalgia  1  3/21 (14.29%)  0/10 (0.00%) 
Nervous system disorders     
Headache  1  7/21 (33.33%)  1/10 (10.00%) 
Dizziness  1  2/21 (9.52%)  0/10 (0.00%) 
Product Issues     
Device occlusion  1  2/21 (9.52%)  0/10 (0.00%) 
Respiratory, thoracic and mediastinal disorders     
Oropharyngeal pain  1  0/21 (0.00%)  3/10 (30.00%) 
Epistaxis  1  0/21 (0.00%)  1/10 (10.00%) 
Rhinitis allergic  1  0/21 (0.00%)  1/10 (10.00%) 
Sinus disorder  1  0/21 (0.00%)  1/10 (10.00%) 
Cough  1  5/21 (23.81%)  1/10 (10.00%) 
Rhinorrhoea  1  4/21 (19.05%)  0/10 (0.00%) 
Nasal congestion  1  3/21 (14.29%)  0/10 (0.00%) 
Dyspnoea  1  2/21 (9.52%)  0/10 (0.00%) 
Productive cough  1  2/21 (9.52%)  0/10 (0.00%) 
Skin and subcutaneous tissue disorders     
Asteatosis  1  0/21 (0.00%)  1/10 (10.00%) 
Erythema  1  0/21 (0.00%)  1/10 (10.00%) 
Rash  1  4/21 (19.05%)  0/10 (0.00%) 
Dermatitis diaper  1  2/21 (9.52%)  0/10 (0.00%) 
Vascular disorders     
Hypertension  1  6/21 (28.57%)  1/10 (10.00%) 
Hypotension  1  2/21 (9.52%)  0/10 (0.00%) 
1
Term from vocabulary, MedDRA 21.0
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT 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.
Phone: 855-752-2356
EMail: clinicaltrials@alexion.com
Layout table for additonal information
Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03131219    
Other Study ID Numbers: ALXN1210-aHUS-312
2016-002499-29 ( EudraCT Number )
First Submitted: April 24, 2017
First Posted: April 27, 2017
Results First Submitted: July 27, 2020
Results First Posted: September 16, 2020
Last Update Posted: September 16, 2020