Clinical Trial Results:
Prospective, Single-Arm, Multicenter Study to Evaluate the Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Eculizumab in Complement Inhibitor Treatment-Naïve Pediatric and Adult Participants with Atypical Hemolytic Uremic Syndrome (aHUS) in Chin
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Summary
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EudraCT number |
2025-000162-29 |
Trial protocol |
Outside EU/EEA |
Global end of trial date |
07 May 2025
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Results information
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Results version number |
v1(current) |
This version publication date |
22 Nov 2025
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First version publication date |
22 Nov 2025
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Other versions |
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Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
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Trial identification
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Sponsor protocol code |
ECU-aHUS-302
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Additional study identifiers
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ISRCTN number |
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US NCT number |
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WHO universal trial number (UTN) |
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Sponsors
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Sponsor organisation name |
Alexion Pharmaceuticals Inc.
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Sponsor organisation address |
121 Seaport Boulevard, Boston, MA, United States, 02210
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Public contact |
Alexion Pharmaceuticals Inc., European Clinical Trial Information, +35 3874162507, clinicaltrials.eu@alexion.com
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Scientific contact |
European Clinical Trial Information, Alexion Pharmaceuticals Inc., +35 +35 3874162507, clinicaltrials.eu@alexion.com
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
No
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Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
07 May 2025
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Is this the analysis of the primary completion data? |
No
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Global end of trial reached? |
Yes
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Global end of trial date |
07 May 2025
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Was the trial ended prematurely? |
No
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General information about the trial
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Main objective of the trial |
The main objective of the trial was to assess the efficacy of eculizumab in the treatment of participants with aHUS.
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Protection of trial subjects |
This study was conducted in accordance with the protocol and consensus ethical principles derived from international guidelines including the Declaration of Helsinki, Council for International Organizations of Medical Sciences (CIOMS) International Ethical Guidelines, applicable International Council for Harmonisation (ICH) Good Clinical Practice (GCP) Guidelines, and other applicable laws and regulations.
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Background therapy |
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Evidence for comparator |
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Actual start date of recruitment |
14 Jul 2023
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
No
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Population of trial subjects
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Number of subjects enrolled per country |
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Country: Number of subjects enrolled |
China: 25
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Worldwide total number of subjects |
25
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EEA total number of subjects |
0
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Number of subjects enrolled per age group |
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In utero |
0
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Preterm newborn - gestational age < 37 wk |
0
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Newborns (0-27 days) |
0
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Infants and toddlers (28 days-23 months) |
2
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Children (2-11 years) |
7
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Adolescents (12-17 years) |
2
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Adults (18-64 years) |
14
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From 65 to 84 years |
0
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85 years and over |
0
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Recruitment
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Recruitment details |
- | ||||||||||||||
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Pre-assignment
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Screening details |
After providing informed consent/assent, participants were screened for eligibility for the study during the 7-day Screening Period. | ||||||||||||||
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Period 1
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Period 1 title |
Overall Study (overall period)
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Is this the baseline period? |
Yes | ||||||||||||||
Allocation method |
Not applicable
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Blinding used |
Not blinded | ||||||||||||||
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Arms
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Arm title
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Eculizumab | ||||||||||||||
Arm description |
Participants received eculizumab at a dose and schedule according to body weight for 26 weeks. | ||||||||||||||
Arm type |
Experimental | ||||||||||||||
Investigational medicinal product name |
Eculizumab
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Solution for infusion
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Routes of administration |
Intravenous use
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Dosage and administration details |
Administered as an intravenous (IV) infusion.
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Baseline characteristics reporting groups
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Reporting group title |
Eculizumab
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Reporting group description |
Participants received eculizumab at a dose and schedule according to body weight for 26 weeks. | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
Eculizumab
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Reporting group description |
Participants received eculizumab at a dose and schedule according to body weight for 26 weeks. | ||
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End point title |
Percentage of Participants with a Complete Thrombotic Microangiopathy (TMA) Response [1] | ||||||||
End point description |
The criteria for complete TMA response were:
1. Normalization of platelet count (defined as platelet count ≥ 150000/microliter (ul).
2. Normalization of lactate dehydrogenase (LDH, defined as LDH ≤ upper limit of normal [ULN]).
3. ≥ 25% improvement in serum creatinine from baseline.
Full Analysis Set: Included all participants who received at least 1 dose of study intervention and had at least 1 efficacy assessment post first dose.
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End point type |
Primary
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End point timeframe |
Up to Week 26
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| Notes [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: No comparative statistical analyses were planned. |
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| No statistical analyses for this end point | |||||||||
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End point title |
Number of Participants with an Adverse Event (AE) | ||||||||||
End point description |
An AE was defined as any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and which did not necessarily have a causal relationship with this treatment.
A serious AE (SAE) was defined as any untoward medical occurrence that, at any dose:
- resulted in death,
- was life-threatening,
- required inpatient hospitalization or prolongation of existing hospitalization,
- resulted in persistent disability/incapacity,
- was a congenital anomaly/birth defect, or
- was an important medical event.
A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the ‘Reported Adverse Events’ Section.
Safety Set: Included all participants who received at least 1 dose of study intervention.
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End point type |
Secondary
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End point timeframe |
Up to Week 34
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| No statistical analyses for this end point | |||||||||||
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End point title |
Mean Serum Concentration of Eculizumab | ||||||||||||||||||||||||||||||
End point description |
Pharmacokinetic (PK) Analysis Set: Included all participants who received at least 1 dose of study intervention and had evaluable pharmacokinetic data. N = the number of participants evaluable at the specific timepoint.
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End point type |
Secondary
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End point timeframe |
Pre-dose and post-dose at Days 1, 8, 29, 85 and 141; Pre-dose at Day 183
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| No statistical analyses for this end point | |||||||||||||||||||||||||||||||
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End point title |
Change from Baseline in Serum Free Complement 5 (C5) | ||||||||||||||||||||||||||||
End point description |
Pharmacodynamic (PD) Analysis Set: Included all participants who received at least 1 dose of study intervention and had evaluable PD data. N = the number of participants evaluable at the specific timepoint.
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End point type |
Secondary
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End point timeframe |
Baseline (Day 1 pre-dose) to Days 1, 8, 29, 85 and 141 (pre-dose and post-dose) and pre-dose at Day 183
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| No statistical analyses for this end point | |||||||||||||||||||||||||||||
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End point title |
Change from Baseline in Serum Total C5 | ||||||||||||||||||||||||||||
End point description |
PD Analysis Set: Included all participants who received at least 1 dose of study intervention and had evaluable PD data. N = the number of participants evaluable at the specific timepoint.
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End point type |
Secondary
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End point timeframe |
Baseline (Day 1 pre-dose) to Days 1, 8, 29, 85 and 141 (pre-dose and post-dose) and pre-dose at Day 183
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| No statistical analyses for this end point | |||||||||||||||||||||||||||||
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End point title |
Number of Participants with an Anti-drug Antibody (ADA) Response | ||||||
End point description |
An ADA response was defined as a positive ADA sample at any time during the study.
Safety Set: Included all participants who received at least 1 dose of study intervention.
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End point type |
Secondary
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End point timeframe |
Up to Week 26
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| No statistical analyses for this end point | |||||||
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End point title |
Time to Complete TMA Response | ||||||||
End point description |
Time to complete TMA response was defined as the time from first infusion to the first time point at which all criteria for complete TMA response was met.
The criteria for complete TMA response were:
1. Normalization of platelet count (defined as platelet count ≥ 150000/ul.
2. Normalization of LDH, defined as LDH ≤ ULN).
3. ≥ 25% improvement in serum creatinine from baseline.
Participants who did not have a response were censored at the date of last visit or study discontinuation at the time when the analysis was performed.
99999 = Upper limit was not reached due to limited number of events.
Full Analysis Set: Included all participants who received at least 1 dose of study intervention and had at least 1 efficacy assessment post first dose.
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End point type |
Secondary
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End point timeframe |
Up to Week 26
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| No statistical analyses for this end point | |||||||||
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End point title |
Proportion of Participants On or Off Dialysis at Each Timepoint | ||||||||||||||||||||||||||||||||||||||||||||
End point description |
Participants were considered as 'off' dialysis at a specific time point if they were dialysis free for more than 5 days prior to that time point. Participants were considered as 'on' dialysis at a specific time point if they were dialysis free to 5 days or less up prior to that time point. N = number of participants evaluable at the specific timepoint.
Full Analysis Set: Included all participants who received at least 1 dose of study intervention and had at least 1 efficacy assessment post first dose.
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End point type |
Secondary
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End point timeframe |
Baseline and Days 22, 43, 71, 99, 113, 127, 155 and 183
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| No statistical analyses for this end point | |||||||||||||||||||||||||||||||||||||||||||||
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End point title |
Change from Baseline in Estimated Glomerular Filtration Rate (eGFR) at Each Scheduled Visit | ||||||||||||||||||||||||
End point description |
Expressed in milliliter per minute per 1.73 square meters of body surface area.
Full Analysis Set: Included all participants who received at least 1 dose of study intervention and had at least 1 efficacy assessment post first dose. N = number of participants evaluable at the specific timepoint.
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End point type |
Secondary
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End point timeframe |
Baseline, Days 22, 43, 71, 99, 113, 127, 155 and 183
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| No statistical analyses for this end point | |||||||||||||||||||||||||
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End point title |
Proportion of Participants with a Chronic Kidney Disease (CKD) Stage Shift Categorized as "Improved", "Stable", or "Worsened" at each Scheduled Visit Compared to Baseline | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point description |
CKD stage was classified based on the National Kidney Foundation Chronic Kidney Disease Stage where Stage 5 represents the most severe disease and Stage 1 represents the least severe disease.
Full Analysis Set: Included all participants who received at least 1 dose of study intervention and had at least 1 efficacy assessment post first dose. N = the number of participants evaluable at the specific timepoint. "Improved" excluded participants with Stage 1 at baseline as there was no room for improvement. "Worsened" excludes participants with Stage 5 at baseline as there was no room to worsen.
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End point type |
Secondary
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End point timeframe |
Baseline to Days 22, 43, 71, 99, 113, 127, 155 and 183
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| No statistical analyses for this end point | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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End point title |
Change from Baseline in Platelets | ||||||||||||||||||||||||
End point description |
Platelet values obtained from the day of a blood transfusion of platelets through 3 days after the transfusion are excluded from all analysis.
Full Analysis Set: Included all participants who received at least 1 dose of study intervention and had at least 1 efficacy assessment post first dose. N = the number of participants evaluable at the specific timepoint.
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End point type |
Secondary
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End point timeframe |
Baseline, Days 22, 43, 71, 99, 113, 127, 155, and 183
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| No statistical analyses for this end point | |||||||||||||||||||||||||
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End point title |
Change from Baseline in LDH | ||||||||||||||||||||||||
End point description |
Full Analysis Set: Included all participants who received at least 1 dose of study intervention and had at least 1 efficacy assessment post first dose. N = the number of participants evaluable at the specific timepoint.
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End point type |
Secondary
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End point timeframe |
Baseline, Days 22, 43, 71, 99, 113, 127, 155, and 183
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| No statistical analyses for this end point | |||||||||||||||||||||||||
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End point title |
Change from Baseline in Hemoglobin | ||||||||||||||||||||||||
End point description |
Hemoglobin values obtained from the day of a blood transfusion of either whole blood or packed red blood cells through 7 days after the transfusion are excluded from all analysis.
Full Analysis Set: Included all participants who received at least 1 dose of study intervention and had at least 1 efficacy assessment post first dose. N = number of participants evaluable at the specific timepoint.
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End point type |
Secondary
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End point timeframe |
Baseline, Days 22, 43, 71, 99, 113, 127, 155, and 183
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| No statistical analyses for this end point | |||||||||||||||||||||||||
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Adverse events information
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Timeframe for reporting adverse events |
Up to Week 34
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Adverse event reporting additional description |
Safety Set: Included all participants who received at least 1 dose of study intervention.
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Assessment type |
Systematic | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
27.1
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Reporting groups
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Reporting group title |
Eculizumab
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Reporting group description |
Participants received eculizumab at a dose and schedule according to body weight for 26 weeks. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Frequency threshold for reporting non-serious adverse events: 5% | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Substantial protocol amendments (globally) |
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| Were there any global substantial amendments to the protocol? Yes | |||
Date |
Amendment |
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20 Jun 2022 |
This amendment was initiated mainly to update exclusion criteria and pregnancy language. |
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26 Apr 2023 |
The primary reason for this amendment was to clarify activities to be followed when a change in weight causes a participant to move to a different weight cohort during the study period.
Additionally, updates to estimand descriptions for endpoints were also made. |
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Interruptions (globally) |
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| Were there any global interruptions to the trial? No | |||
Limitations and caveats |
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| Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
| None reported | |||
