Trial Outcomes & Findings for Eculizumab in Pediatric and Adult Participants With Atypical Hemolytic Uremic Syndrome (aHUS) in China (NCT NCT05876351)

Last Updated: 2025-12-08

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

25 participants

Primary outcome timeframe

Up to Week 26

Results posted on

2025-12-08

Participant Flow

After providing informed consent/assent, participants were screened for eligibility for the study during the 7-day Screening Period.

Participant milestones

Participant milestones
Measure	Eculizumab Participants received eculizumab as an intravenous (IV) infusion at a dose and schedule according to body weight for 26 weeks.
Overall Study STARTED	25
Overall Study Received at Least 1 Dose of Study Drug	25
Overall Study COMPLETED	22
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Reasons for withdrawal
Measure	Eculizumab Participants received eculizumab as an intravenous (IV) infusion at a dose and schedule according to body weight for 26 weeks.
Overall Study Other than specified	2
Overall Study Study specific discontinuation criteria	1

Baseline Characteristics

Eculizumab in Pediatric and Adult Participants With Atypical Hemolytic Uremic Syndrome (aHUS) in China

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Eculizumab n=25 Participants Participants received eculizumab as an IV infusion at a dose and schedule according to body weight for 26 weeks.
Age, Continuous	23.40 years STANDARD_DEVIATION 15.95 • n=37 Participants
Age, Customized In utero	0 Participants n=37 Participants
Age, Customized Preterm newborn infants (gestational age < 37 wks)	0 Participants n=37 Participants
Age, Customized Newborns (0-27 days)	0 Participants n=37 Participants
Age, Customized Infants and toddlers (28 days-23 months)	2 Participants n=37 Participants
Age, Customized Children (2-11 years)	7 Participants n=37 Participants
Age, Customized Adolescents (12-17 years)	2 Participants n=37 Participants
Age, Customized Adults (18-64 years)	14 Participants n=37 Participants
Age, Customized From 65-84 years	0 Participants n=37 Participants
Age, Customized 85 years and over	0 Participants n=37 Participants
Sex: Female, Male Female	6 Participants n=37 Participants
Sex: Female, Male Male	19 Participants n=37 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=37 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	24 Participants n=37 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=37 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=37 Participants
Race (NIH/OMB) Asian	25 Participants n=37 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=37 Participants
Race (NIH/OMB) Black or African American	0 Participants n=37 Participants
Race (NIH/OMB) White	0 Participants n=37 Participants
Race (NIH/OMB) More than one race	0 Participants n=37 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=37 Participants

PRIMARY outcome

Timeframe: Up to Week 26

Population: 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.

Outcome measures

Outcome measures
Measure	Eculizumab n=25 Participants Participants received eculizumab as an IV infusion at a dose and schedule according to body weight for 26 weeks.
Percentage of Participants With a Complete Thrombotic Microangiopathy (TMA) Response	64.0 percentage of participants 95% Confidence Interval 42.5 • Interval 42.5 to 82.0

SECONDARY outcome

Timeframe: Up to Week 34

Population: Safety Set: Included all participants who received at least 1 dose of study intervention.

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 'Adverse Events' Section.

Outcome measures

Outcome measures
Measure	Eculizumab n=25 Participants Participants received eculizumab as an IV infusion at a dose and schedule according to body weight for 26 weeks.
Number of Participants With an Adverse Event (AE) Any AE	24 Participants
Number of Participants With an Adverse Event (AE) Any SAE	8 Participants

SECONDARY outcome

Timeframe: Pre-dose and post-dose at Days 1, 8, 29, 85, and 141; Pre-dose at Day 183

Population: Pharmacokinetic (PK) Analysis Set: Included all participants who received at least 1 dose of study intervention and had evaluable pharmacokinetic data. 'Number Analyzed' = number of participants evaluable at specified timepoint.

Outcome measures

Outcome measures
Measure	Eculizumab n=25 Participants Participants received eculizumab as an IV infusion at a dose and schedule according to body weight for 26 weeks.
Mean Serum Concentration of Eculizumab Day 1: Pre-dose	4.690 micrograms per milliliter (ug/mL) Geometric Coefficient of Variation 0.00
Mean Serum Concentration of Eculizumab Day 1: Post-dose	373.423 micrograms per milliliter (ug/mL) Geometric Coefficient of Variation 47.59
Mean Serum Concentration of Eculizumab Day 8: Pre-dose	153.190 micrograms per milliliter (ug/mL) Geometric Coefficient of Variation 69.69
Mean Serum Concentration of Eculizumab Day 8: Post-dose	498.262 micrograms per milliliter (ug/mL) Geometric Coefficient of Variation 41.18
Mean Serum Concentration of Eculizumab Day 29: Pre-dose	353.726 micrograms per milliliter (ug/mL) Geometric Coefficient of Variation 36.96
Mean Serum Concentration of Eculizumab Day 29: Post-dose	727.862 micrograms per milliliter (ug/mL) Geometric Coefficient of Variation 32.22
Mean Serum Concentration of Eculizumab Day 85: Pre-dose	360.070 micrograms per milliliter (ug/mL) Geometric Coefficient of Variation 43.89
Mean Serum Concentration of Eculizumab Day 85: Post-dose	728.624 micrograms per milliliter (ug/mL) Geometric Coefficient of Variation 42.04
Mean Serum Concentration of Eculizumab Day 141: Pre-dose	433.737 micrograms per milliliter (ug/mL) Geometric Coefficient of Variation 41.32
Mean Serum Concentration of Eculizumab Day 141: Post-dose	883.675 micrograms per milliliter (ug/mL) Geometric Coefficient of Variation 38.74
Mean Serum Concentration of Eculizumab Day 183: Pre-dose	434.739 micrograms per milliliter (ug/mL) Geometric Coefficient of Variation 38.66

SECONDARY outcome

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

Population: Pharmacodynamic (PD) Analysis Set: Included all participants who received at least 1 dose of study intervention and had evaluable PD data. 'Number Analyzed' = number of participants evaluable at specified timepoint.

Outcome measures

Outcome measures
Measure	Eculizumab n=25 Participants Participants received eculizumab as an IV infusion at a dose and schedule according to body weight for 26 weeks.
Change From Baseline in Serum Free Complement 5 (C5) Day 1: Post-dose	-79.8842 ug/mL Standard Deviation 17.5197
Change From Baseline in Serum Free Complement 5 (C5) Day 8: Pre-dose	-79.6539 ug/mL Standard Deviation 17.8589
Change From Baseline in Serum Free Complement 5 (C5) Day 8: Post-dose	-79.6636 ug/mL Standard Deviation 17.8610
Change From Baseline in Serum Free Complement 5 (C5) Day 29: Pre-dose	-80.4277 ug/mL Standard Deviation 17.1241
Change From Baseline in Serum Free Complement 5 (C5) Day 29: Post-dose	-80.4284 ug/mL Standard Deviation 17.1241
Change From Baseline in Serum Free Complement 5 (C5) Day 85: Pre-dose	-80.0632 ug/mL Standard Deviation 17.3489
Change From Baseline in Serum Free Complement 5 (C5) Day 85: Post-dose	-80.0632 ug/mL Standard Deviation 17.3489
Change From Baseline in Serum Free Complement 5 (C5) Day 141: Pre-dose	-80.0625 ug/mL Standard Deviation 17.3485
Change From Baseline in Serum Free Complement 5 (C5) Day 141: Post-dose	-80.0632 ug/mL Standard Deviation 17.3489
Change From Baseline in Serum Free Complement 5 (C5) Day 183: Pre-dose	-80.0632 ug/mL Standard Deviation 17.3489

SECONDARY outcome

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

Population: PD Analysis Set: Included all participants who received at least 1 dose of study intervention and had evaluable PD data. 'Number Analyzed' = number of participants evaluable at specified timepoint.

Outcome measures

Outcome measures
Measure	Eculizumab n=25 Participants Participants received eculizumab as an IV infusion at a dose and schedule according to body weight for 26 weeks.
Change From Baseline in Serum Total C5 Day 183: Pre-dose	78.5497 ug/mL Standard Deviation 27.1158
Change From Baseline in Serum Total C5 Day 1: Post-dose	-12.0267 ug/mL Standard Deviation 6.1396
Change From Baseline in Serum Total C5 Day 8: Pre-dose	37.9293 ug/mL Standard Deviation 21.3162
Change From Baseline in Serum Total C5 Day 8: Post-dose	40.2831 ug/mL Standard Deviation 23.0175
Change From Baseline in Serum Total C5 Day 29: Pre-dose	65.9100 ug/mL Standard Deviation 25.0734
Change From Baseline in Serum Total C5 Day 29: Post-dose	64.4783 ug/mL Standard Deviation 22.7695
Change From Baseline in Serum Total C5 Day 85: Pre-dose	71.2418 ug/mL Standard Deviation 25.7350
Change From Baseline in Serum Total C5 Day 85: Post-dose	66.5267 ug/mL Standard Deviation 26.7232
Change From Baseline in Serum Total C5 Day 141: Pre-dose	71.8284 ug/mL Standard Deviation 25.3689
Change From Baseline in Serum Total C5 Day 141: Post-dose	67.0693 ug/mL Standard Deviation 23.1231

SECONDARY outcome

Timeframe: Up to Week 26

Population: Safety Set: Included all participants who received at least 1 dose of study intervention.

An ADA response was defined as a positive ADA sample at any time during the study.

Outcome measures

Outcome measures
Measure	Eculizumab n=25 Participants Participants received eculizumab as an IV infusion at a dose and schedule according to body weight for 26 weeks.
Number of Participants With an Anti-drug Antibody (ADA) Response	0 Participants

SECONDARY outcome

Timeframe: Up to Week 26

Population: 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.

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.

Outcome measures

Outcome measures
Measure	Eculizumab n=25 Participants Participants received eculizumab as an IV infusion at a dose and schedule according to body weight for 26 weeks.
Time to Complete TMA Response	75.0 days Interval 22.0 to Upper limit was not reached due to limited number of events.

SECONDARY outcome

Timeframe: Baseline and Days 22, 43, 71, 99, 113, 127, 155 and 183

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.

Outcome measures

Outcome measures
Measure	Eculizumab n=25 Participants Participants received eculizumab as an IV infusion at a dose and schedule according to body weight for 26 weeks.
Proportion of Participants On or Off Dialysis at Each Timepoint Day 127: Off Dialysis	0.889 proportion of participants Interval 0.653 to 0.986
Proportion of Participants On or Off Dialysis at Each Timepoint Day 155: On Dialysis	0.167 proportion of participants Interval 0.036 to 0.414
Proportion of Participants On or Off Dialysis at Each Timepoint Day 155: Off Dialysis	0.833 proportion of participants Interval 0.586 to 0.964
Proportion of Participants On or Off Dialysis at Each Timepoint Day 183: On Dialysis	0.167 proportion of participants Interval 0.036 to 0.414
Proportion of Participants On or Off Dialysis at Each Timepoint Day 183: Off Dialysis	0.833 proportion of participants Interval 0.586 to 0.964
Proportion of Participants On or Off Dialysis at Each Timepoint Baseline: On Dialysis	0.440 proportion of participants Interval 0.244 to 0.651
Proportion of Participants On or Off Dialysis at Each Timepoint Baseline: Off Dialysis	0.560 proportion of participants Interval 0.349 to 0.756
Proportion of Participants On or Off Dialysis at Each Timepoint Day 22: On Dialysis	0.261 proportion of participants Interval 0.102 to 0.484
Proportion of Participants On or Off Dialysis at Each Timepoint Day 22: Off Dialysis	0.739 proportion of participants Interval 0.516 to 0.898
Proportion of Participants On or Off Dialysis at Each Timepoint Day 43: On Dialysis	0.235 proportion of participants Interval 0.068 to 0.499
Proportion of Participants On or Off Dialysis at Each Timepoint Day 43: Off Dialysis	0.765 proportion of participants Interval 0.501 to 0.932
Proportion of Participants On or Off Dialysis at Each Timepoint Day 71: On Dialysis	0.211 proportion of participants Interval 0.061 to 0.456
Proportion of Participants On or Off Dialysis at Each Timepoint Day 71: Off Dialysis	0.789 proportion of participants Interval 0.544 to 0.939
Proportion of Participants On or Off Dialysis at Each Timepoint Day 99: On Dialysis	0.176 proportion of participants Interval 0.038 to 0.434
Proportion of Participants On or Off Dialysis at Each Timepoint Day 99: Off Dialysis	0.824 proportion of participants Interval 0.566 to 0.962
Proportion of Participants On or Off Dialysis at Each Timepoint Day 113: On Dialysis	0.167 proportion of participants Interval 0.036 to 0.414
Proportion of Participants On or Off Dialysis at Each Timepoint Day 113: Off Dialysis	0.833 proportion of participants Interval 0.586 to 0.964
Proportion of Participants On or Off Dialysis at Each Timepoint Day 127: On Dialysis	0.111 proportion of participants Interval 0.014 to 0.347

SECONDARY outcome

Timeframe: Baseline, Days 22, 43, 71, 99, 113, 127, 155 and 183

Population: 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. 'Number Analyzed' = number of participants evaluable at the specific timepoint. 'Overall number of participants analyzed' = participants evaluable for this outcome measure. 'Number Analyzed' = number of participants evaluable at the specific timepoint.

Expressed in milliliters per minute per 1.73 square meters of body surface area.

Outcome measures

Outcome measures
Measure	Eculizumab n=22 Participants Participants received eculizumab as an IV infusion at a dose and schedule according to body weight for 26 weeks.
Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Each Scheduled Visit Day 22	39.82 mL/min/1.73^2 Standard Deviation 50.83
Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Each Scheduled Visit Day 43	32.72 mL/min/1.73^2 Standard Deviation 44.75
Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Each Scheduled Visit Day 71	40.33 mL/min/1.73^2 Standard Deviation 47.38
Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Each Scheduled Visit Day 99	37.53 mL/min/1.73^2 Standard Deviation 47.17
Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Each Scheduled Visit Day 127	35.85 mL/min/1.73^2 Standard Deviation 48.01
Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Each Scheduled Visit Day 155	43.33 mL/min/1.73^2 Standard Deviation 49.32
Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Each Scheduled Visit Day 183	36.29 mL/min/1.73^2 Standard Deviation 44.21
Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Each Scheduled Visit Day 113	35.88 mL/min/1.73^2 Standard Deviation 45.38

SECONDARY outcome

Timeframe: Baseline to Days 22, 43, 71, 99, 113, 127, 155 and 183

Population: 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. 'Overall number of participants analyzed' = participants evaluable for this outcome measure. 'Number Analyzed' = number of participants evaluable at specified timepoint.

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. "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.

Outcome measures

Outcome measures
Measure	Eculizumab n=22 Participants Participants received eculizumab as an IV infusion at a dose and schedule according to body weight for 26 weeks.
Proportion of Participants With a Chronic Kidney Disease (CKD) Stage Shift Categorized as "Improved", "Stable", or "Worsened" at Each Scheduled Visit Compared to Baseline Day 22: Improved	0.545 proportion of participants Interval 0.322 to 0.756
Proportion of Participants With a Chronic Kidney Disease (CKD) Stage Shift Categorized as "Improved", "Stable", or "Worsened" at Each Scheduled Visit Compared to Baseline Day 22: Stable	0.455 proportion of participants Interval 0.244 to 0.678
Proportion of Participants With a Chronic Kidney Disease (CKD) Stage Shift Categorized as "Improved", "Stable", or "Worsened" at Each Scheduled Visit Compared to Baseline Day 22: Worsened	0 proportion of participants Interval 0.0 to 0.41
Proportion of Participants With a Chronic Kidney Disease (CKD) Stage Shift Categorized as "Improved", "Stable", or "Worsened" at Each Scheduled Visit Compared to Baseline Day 43: Improved	0.688 proportion of participants Interval 0.413 to 0.89
Proportion of Participants With a Chronic Kidney Disease (CKD) Stage Shift Categorized as "Improved", "Stable", or "Worsened" at Each Scheduled Visit Compared to Baseline Day 43: Stable	0.313 proportion of participants Interval 0.11 to 0.587
Proportion of Participants With a Chronic Kidney Disease (CKD) Stage Shift Categorized as "Improved", "Stable", or "Worsened" at Each Scheduled Visit Compared to Baseline Day 43: Worsened	0 proportion of participants Interval 0.0 to 0.602
Proportion of Participants With a Chronic Kidney Disease (CKD) Stage Shift Categorized as "Improved", "Stable", or "Worsened" at Each Scheduled Visit Compared to Baseline Day 71: Improved	0.722 proportion of participants Interval 0.465 to 0.903
Proportion of Participants With a Chronic Kidney Disease (CKD) Stage Shift Categorized as "Improved", "Stable", or "Worsened" at Each Scheduled Visit Compared to Baseline Day 71: Stable	0.278 proportion of participants Interval 0.097 to 0.535
Proportion of Participants With a Chronic Kidney Disease (CKD) Stage Shift Categorized as "Improved", "Stable", or "Worsened" at Each Scheduled Visit Compared to Baseline Day 71: Worsened	0 proportion of participants Interval 0.0 to 0.522
Proportion of Participants With a Chronic Kidney Disease (CKD) Stage Shift Categorized as "Improved", "Stable", or "Worsened" at Each Scheduled Visit Compared to Baseline Day 99: Improved	0.750 proportion of participants Interval 0.476 to 0.927
Proportion of Participants With a Chronic Kidney Disease (CKD) Stage Shift Categorized as "Improved", "Stable", or "Worsened" at Each Scheduled Visit Compared to Baseline Day 99: Stable	0.250 proportion of participants Interval 0.073 to 0.524
Proportion of Participants With a Chronic Kidney Disease (CKD) Stage Shift Categorized as "Improved", "Stable", or "Worsened" at Each Scheduled Visit Compared to Baseline Day 99: Worsened	0 proportion of participants Interval 0.0 to 0.522
Proportion of Participants With a Chronic Kidney Disease (CKD) Stage Shift Categorized as "Improved", "Stable", or "Worsened" at Each Scheduled Visit Compared to Baseline Day 113: Improved	0.765 proportion of participants Interval 0.501 to 0.932
Proportion of Participants With a Chronic Kidney Disease (CKD) Stage Shift Categorized as "Improved", "Stable", or "Worsened" at Each Scheduled Visit Compared to Baseline Day 113: Stable	0.235 proportion of participants Interval 0.068 to 0.499
Proportion of Participants With a Chronic Kidney Disease (CKD) Stage Shift Categorized as "Improved", "Stable", or "Worsened" at Each Scheduled Visit Compared to Baseline Day 113: Worsened	0 proportion of participants Interval 0.0 to 0.459
Proportion of Participants With a Chronic Kidney Disease (CKD) Stage Shift Categorized as "Improved", "Stable", or "Worsened" at Each Scheduled Visit Compared to Baseline Day 127: Improved	0.765 proportion of participants Interval 0.501 to 0.932
Proportion of Participants With a Chronic Kidney Disease (CKD) Stage Shift Categorized as "Improved", "Stable", or "Worsened" at Each Scheduled Visit Compared to Baseline Day 127: Stable	0.235 proportion of participants Interval 0.068 to 0.499
Proportion of Participants With a Chronic Kidney Disease (CKD) Stage Shift Categorized as "Improved", "Stable", or "Worsened" at Each Scheduled Visit Compared to Baseline Day 127: Worsened	0 proportion of participants Interval 0.0 to 0.459
Proportion of Participants With a Chronic Kidney Disease (CKD) Stage Shift Categorized as "Improved", "Stable", or "Worsened" at Each Scheduled Visit Compared to Baseline Day 155: Improved	0.875 proportion of participants Interval 0.617 to 0.984
Proportion of Participants With a Chronic Kidney Disease (CKD) Stage Shift Categorized as "Improved", "Stable", or "Worsened" at Each Scheduled Visit Compared to Baseline Day 155: Stable	0.125 proportion of participants Interval 0.016 to 0.383
Proportion of Participants With a Chronic Kidney Disease (CKD) Stage Shift Categorized as "Improved", "Stable", or "Worsened" at Each Scheduled Visit Compared to Baseline Day 155: Worsened	0 proportion of participants Interval 0.0 to 0.522
Proportion of Participants With a Chronic Kidney Disease (CKD) Stage Shift Categorized as "Improved", "Stable", or "Worsened" at Each Scheduled Visit Compared to Baseline Day 183: Improved	0.824 proportion of participants Interval 0.566 to 0.962
Proportion of Participants With a Chronic Kidney Disease (CKD) Stage Shift Categorized as "Improved", "Stable", or "Worsened" at Each Scheduled Visit Compared to Baseline Day 183: Stable	0.176 proportion of participants Interval 0.038 to 0.434
Proportion of Participants With a Chronic Kidney Disease (CKD) Stage Shift Categorized as "Improved", "Stable", or "Worsened" at Each Scheduled Visit Compared to Baseline Day 183: Worsened	0 proportion of participants Interval 0.0 to 0.522

SECONDARY outcome

Timeframe: Baseline, Days 22, 43, 71, 99, 113, 127, 155, and 183

Platelet values obtained from the day of a blood transfusion of platelets through 3 days after the transfusion are excluded from all analysis.

Outcome measures

Outcome measures
Measure	Eculizumab n=23 Participants Participants received eculizumab as an IV infusion at a dose and schedule according to body weight for 26 weeks.
Change From Baseline in Platelets Day 22	76.5 10^9 platelets/liter (L) Standard Deviation 117.7
Change From Baseline in Platelets Day 43	65.5 10^9 platelets/liter (L) Standard Deviation 90.1
Change From Baseline in Platelets Day 71	71.3 10^9 platelets/liter (L) Standard Deviation 97.9
Change From Baseline in Platelets Day 99	79.4 10^9 platelets/liter (L) Standard Deviation 68.1
Change From Baseline in Platelets Day 113	60.1 10^9 platelets/liter (L) Standard Deviation 82.7
Change From Baseline in Platelets Day 127	61.8 10^9 platelets/liter (L) Standard Deviation 86.1
Change From Baseline in Platelets Day 155	76.8 10^9 platelets/liter (L) Standard Deviation 98.2
Change From Baseline in Platelets Day 183	80.3 10^9 platelets/liter (L) Standard Deviation 98.0

SECONDARY outcome

Timeframe: Baseline, Days 22, 43, 71, 99, 113, 127, 155, and 183

Outcome measures

Outcome measures
Measure	Eculizumab n=23 Participants Participants received eculizumab as an IV infusion at a dose and schedule according to body weight for 26 weeks.
Change From Baseline in LDH Day 22	-9.445 microkatal per liter (ukat/L) Standard Deviation 12.459
Change From Baseline in LDH Day 43	-6.147 microkatal per liter (ukat/L) Standard Deviation 10.175
Change From Baseline in LDH Day 71	-7.083 microkatal per liter (ukat/L) Standard Deviation 10.099
Change From Baseline in LDH Day 99	-5.861 microkatal per liter (ukat/L) Standard Deviation 8.356
Change From Baseline in LDH Day 113	-5.769 microkatal per liter (ukat/L) Standard Deviation 8.041
Change From Baseline in LDH Day 127	-5.444 microkatal per liter (ukat/L) Standard Deviation 8.314
Change From Baseline in LDH Day 155	-5.848 microkatal per liter (ukat/L) Standard Deviation 8.478
Change From Baseline in LDH Day 183	-5.680 microkatal per liter (ukat/L) Standard Deviation 8.172

SECONDARY outcome

Timeframe: Baseline, Days 22, 43, 71, 99, 113, 127, 155, and 183

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.

Outcome measures

Outcome measures
Measure	Eculizumab n=23 Participants Participants received eculizumab as an IV infusion at a dose and schedule according to body weight for 26 weeks.
Change From Baseline in Hemoglobin Day 183	36.2 grams per liter (g/L) Standard Deviation 15.8
Change From Baseline in Hemoglobin Day 22	17.0 grams per liter (g/L) Standard Deviation 18.3
Change From Baseline in Hemoglobin Day 43	25.7 grams per liter (g/L) Standard Deviation 20.1
Change From Baseline in Hemoglobin Day 71	30.5 grams per liter (g/L) Standard Deviation 22.6
Change From Baseline in Hemoglobin Day 99	30.3 grams per liter (g/L) Standard Deviation 19.3
Change From Baseline in Hemoglobin Day 113	29.8 grams per liter (g/L) Standard Deviation 17.8
Change From Baseline in Hemoglobin Day 127	25.2 grams per liter (g/L) Standard Deviation 16.4
Change From Baseline in Hemoglobin Day 155	30.5 grams per liter (g/L) Standard Deviation 14.9

Adverse Events

Eculizumab

Serious events: 8 serious events

Other events: 24 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Eculizumab n=25 participants at risk Participants received eculizumab as an IV infusion at a dose and schedule according to body weight for 26 weeks.
Infections and infestations Bacterial infection	4.0% 1/25 • Number of events 1 • Up to Week 34 Safety Set: Included all participants who received at least 1 dose of study intervention.
Infections and infestations Clostridium difficile infection	4.0% 1/25 • Number of events 1 • Up to Week 34 Safety Set: Included all participants who received at least 1 dose of study intervention.
Infections and infestations Infection	4.0% 1/25 • Number of events 1 • Up to Week 34 Safety Set: Included all participants who received at least 1 dose of study intervention.
Infections and infestations Pneumonia	8.0% 2/25 • Number of events 2 • Up to Week 34 Safety Set: Included all participants who received at least 1 dose of study intervention.
Infections and infestations Rotavirus infection	4.0% 1/25 • Number of events 1 • Up to Week 34 Safety Set: Included all participants who received at least 1 dose of study intervention.
Gastrointestinal disorders Retching	4.0% 1/25 • Number of events 1 • Up to Week 34 Safety Set: Included all participants who received at least 1 dose of study intervention.
Gastrointestinal disorders Upper gastrointestinal haemorrhage	4.0% 1/25 • Number of events 1 • Up to Week 34 Safety Set: Included all participants who received at least 1 dose of study intervention.
Hepatobiliary disorders Hepatic function abnormal	4.0% 1/25 • Number of events 1 • Up to Week 34 Safety Set: Included all participants who received at least 1 dose of study intervention.
Musculoskeletal and connective tissue disorders Synovial cyst	4.0% 1/25 • Number of events 1 • Up to Week 34 Safety Set: Included all participants who received at least 1 dose of study intervention.

Other adverse events

Other adverse events
Measure	Eculizumab n=25 participants at risk Participants received eculizumab as an IV infusion at a dose and schedule according to body weight for 26 weeks.
Metabolism and nutrition disorders Hyperkalaemia	28.0% 7/25 • Number of events 9 • Up to Week 34 Safety Set: Included all participants who received at least 1 dose of study intervention.
Infections and infestations Bronchitis	8.0% 2/25 • Number of events 2 • Up to Week 34 Safety Set: Included all participants who received at least 1 dose of study intervention.
Infections and infestations Infection	12.0% 3/25 • Number of events 4 • Up to Week 34 Safety Set: Included all participants who received at least 1 dose of study intervention.
Infections and infestations Influenza	8.0% 2/25 • Number of events 2 • Up to Week 34 Safety Set: Included all participants who received at least 1 dose of study intervention.
Infections and infestations Respiratory tract infection	16.0% 4/25 • Number of events 5 • Up to Week 34 Safety Set: Included all participants who received at least 1 dose of study intervention.
Infections and infestations Upper respiratory tract infection	20.0% 5/25 • Number of events 8 • Up to Week 34 Safety Set: Included all participants who received at least 1 dose of study intervention.
Blood and lymphatic system disorders Anaemia	8.0% 2/25 • Number of events 2 • Up to Week 34 Safety Set: Included all participants who received at least 1 dose of study intervention.
Blood and lymphatic system disorders Leukopenia	8.0% 2/25 • Number of events 2 • Up to Week 34 Safety Set: Included all participants who received at least 1 dose of study intervention.
Metabolism and nutrition disorders Electrolyte imbalance	8.0% 2/25 • Number of events 2 • Up to Week 34 Safety Set: Included all participants who received at least 1 dose of study intervention.
Metabolism and nutrition disorders Hyperphosphataemia	12.0% 3/25 • Number of events 3 • Up to Week 34 Safety Set: Included all participants who received at least 1 dose of study intervention.
Metabolism and nutrition disorders Hypokalaemia	8.0% 2/25 • Number of events 2 • Up to Week 34 Safety Set: Included all participants who received at least 1 dose of study intervention.
Vascular disorders Hypertension	8.0% 2/25 • Number of events 2 • Up to Week 34 Safety Set: Included all participants who received at least 1 dose of study intervention.
Respiratory, thoracic and mediastinal disorders Cough	8.0% 2/25 • Number of events 2 • Up to Week 34 Safety Set: Included all participants who received at least 1 dose of study intervention.
Gastrointestinal disorders Diarrhoea	8.0% 2/25 • Number of events 2 • Up to Week 34 Safety Set: Included all participants who received at least 1 dose of study intervention.
Gastrointestinal disorders Vomiting	20.0% 5/25 • Number of events 5 • Up to Week 34 Safety Set: Included all participants who received at least 1 dose of study intervention.
Hepatobiliary disorders Hepatic function abnormal	16.0% 4/25 • Number of events 5 • Up to Week 34 Safety Set: Included all participants who received at least 1 dose of study intervention.
General disorders Pyrexia	12.0% 3/25 • Number of events 3 • Up to Week 34 Safety Set: Included all participants who received at least 1 dose of study intervention.

Additional Information

Alexion Pharmaceuticals Inc.

European Clinical Trial Information

Phone: +1.855.752.2356

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place