Trial Outcomes & Findings for Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China (NCT NCT05886244)
NCT ID: NCT05886244
Last Updated: 2026-06-01
Results Overview
Blood samples were collected for measurement of serum LDH at specified timepoints. Statistical analysis was performed using a mixed model for repeated measures (MMRM), including the percentage change from baseline of LDH at the scheduled visits from Week 1 to Week 12 as the dependent variable, with the fixed categorical effect of visit, fixed continuous effect of LDH baseline value as covariates, and participant as random effect. Assessed at baseline, 1, 2, 3, 4, 6, 8, 10, and 12 weeks, Week 12 reported. A decrease indicated a reduction in disease activity.
COMPLETED
PHASE3
25 participants
Baseline, Week 12
2026-06-01
Participant Flow
Participant milestones
| Measure |
Eculizumab
Participants received eculizumab by intravenous (IV) infusion.
|
|---|---|
|
Primary Treatment Period (12 Weeks)
STARTED
|
25
|
|
Primary Treatment Period (12 Weeks)
Received at Least 1 Dose of Study Drug
|
25
|
|
Primary Treatment Period (12 Weeks)
COMPLETED
|
25
|
|
Primary Treatment Period (12 Weeks)
NOT COMPLETED
|
0
|
|
Long-Term Extension Period (52 Weeks)
STARTED
|
25
|
|
Long-Term Extension Period (52 Weeks)
Received at Least 1 Dose of Study Drug
|
25
|
|
Long-Term Extension Period (52 Weeks)
COMPLETED
|
20
|
|
Long-Term Extension Period (52 Weeks)
NOT COMPLETED
|
5
|
Reasons for withdrawal
| Measure |
Eculizumab
Participants received eculizumab by intravenous (IV) infusion.
|
|---|---|
|
Long-Term Extension Period (52 Weeks)
Adverse Event
|
1
|
|
Long-Term Extension Period (52 Weeks)
Other than specified
|
1
|
|
Long-Term Extension Period (52 Weeks)
Withdrawal by Subject
|
3
|
Baseline Characteristics
Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China
Baseline characteristics by cohort
| Measure |
Eculizumab
n=25 Participants
Participants received eculizumab by IV infusion.
|
|---|---|
|
Age, Continuous
|
40.5 years
STANDARD_DEVIATION 13.4 • n=24 Participants
|
|
Sex: Female, Male
Female
|
10 Participants
n=24 Participants
|
|
Sex: Female, Male
Male
|
15 Participants
n=24 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=24 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
25 Participants
n=24 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=24 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=24 Participants
|
|
Race (NIH/OMB)
Asian
|
25 Participants
n=24 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=24 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=24 Participants
|
|
Race (NIH/OMB)
White
|
0 Participants
n=24 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=24 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=24 Participants
|
|
Baseline Lactate Dehydrogenase (LDH)
|
1657.50 U/L
STANDARD_DEVIATION 575.93 • n=24 Participants
|
PRIMARY outcome
Timeframe: Baseline, Week 12Population: Full Analysis Set: all participants who received at least 1 dose of study intervention and had at least 1 efficacy assessment post first dose.
Blood samples were collected for measurement of serum LDH at specified timepoints. Statistical analysis was performed using a mixed model for repeated measures (MMRM), including the percentage change from baseline of LDH at the scheduled visits from Week 1 to Week 12 as the dependent variable, with the fixed categorical effect of visit, fixed continuous effect of LDH baseline value as covariates, and participant as random effect. Assessed at baseline, 1, 2, 3, 4, 6, 8, 10, and 12 weeks, Week 12 reported. A decrease indicated a reduction in disease activity.
Outcome measures
| Measure |
Eculizumab
n=25 Participants
Participants received eculizumab by IV infusion.
|
|---|---|
|
Percentage Change From Baseline in LDH at Week 12
|
-76.5 percentage change
Interval -87.8 to -65.2
|
SECONDARY outcome
Timeframe: Baseline through Week 64Population: Safety Set: all participants who received at least 1 dose of study intervention.
An adverse event (AE) was any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product that did not necessarily have to have a causal relationship with the study intervention. A TEAE was any AE that started during or after the first infusion of the study intervention. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.
Outcome measures
| Measure |
Eculizumab
n=25 Participants
Participants received eculizumab by IV infusion.
|
|---|---|
|
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
|
23 Participants
|
SECONDARY outcome
Timeframe: Day 1, Day 29, Day 85, Day 169, Day 253, Day 337, Day 421, Day 449Population: Pharmacokinetic Analysis Set: all participants who receive at least 1 dose of study intervention and who have evaluable pharmacokinetic data. Here, 'Number Analyzed' signifies those participants evaluable for this outcome measure at specified timepoints.
Blood samples were collected for measurement of serum concentrations of eculizumab at specified timepoints. No study intervention administered on Day 449. Results reported as micrograms/milliliter (ug/mL).
Outcome measures
| Measure |
Eculizumab
n=25 Participants
Participants received eculizumab by IV infusion.
|
|---|---|
|
Serum Concentration of Eculizumab
Day 169
|
479.04 ug/mL
Geometric Coefficient of Variation 33.3
|
|
Serum Concentration of Eculizumab
Day 253
|
531.03 ug/mL
Geometric Coefficient of Variation 31.1
|
|
Serum Concentration of Eculizumab
Day 337
|
530.18 ug/mL
Geometric Coefficient of Variation 41.3
|
|
Serum Concentration of Eculizumab
Day 421
|
594.32 ug/mL
Geometric Coefficient of Variation 35.2
|
|
Serum Concentration of Eculizumab
Day 449
|
231.97 ug/mL
Geometric Coefficient of Variation 48.6
|
|
Serum Concentration of Eculizumab
Day 85
|
507.62 ug/mL
Geometric Coefficient of Variation 33.8
|
|
Serum Concentration of Eculizumab
Day 1
|
248.81 ug/mL
Geometric Coefficient of Variation 22.6
|
|
Serum Concentration of Eculizumab
Day 29
|
491.96 ug/mL
Geometric Coefficient of Variation 31.3
|
SECONDARY outcome
Timeframe: Day 1, Day 29, Day 85, Day 169, Day 253, Day 337, Day 421, Day 449Population: Pharmacodynamic Analysis Set: all participants who received at least 1 dose of study intervention and who have evaluable pharmacodynamic data. Here, 'Number Analyzed' signifies those participants evaluable for this outcome measure at specified timepoints.
Blood samples were collected for measurement of serum concentration of free C5 at specified timepoints. No study intervention administered on Day 449. A decrease indicated a reduction in disease activity.
Outcome measures
| Measure |
Eculizumab
n=25 Participants
Participants received eculizumab by IV infusion.
|
|---|---|
|
Change From Baseline in Serum Free Complement 5 (C5) Concentration
Day 169
|
-80.6911 ug/mL
Standard Deviation 21.2241
|
|
Change From Baseline in Serum Free Complement 5 (C5) Concentration
Day 253
|
-80.6911 ug/mL
Standard Deviation 21.2241
|
|
Change From Baseline in Serum Free Complement 5 (C5) Concentration
Day 337
|
-81.5460 ug/mL
Standard Deviation 21.1775
|
|
Change From Baseline in Serum Free Complement 5 (C5) Concentration
Day 421
|
-82.2563 ug/mL
Standard Deviation 21.3936
|
|
Change From Baseline in Serum Free Complement 5 (C5) Concentration
Day 1
|
-80.6911 ug/mL
Standard Deviation 21.2241
|
|
Change From Baseline in Serum Free Complement 5 (C5) Concentration
Day 29
|
-80.6911 ug/mL
Standard Deviation 21.2241
|
|
Change From Baseline in Serum Free Complement 5 (C5) Concentration
Day 85
|
-80.6911 ug/mL
Standard Deviation 21.2241
|
|
Change From Baseline in Serum Free Complement 5 (C5) Concentration
Day 449
|
-82.4700 ug/mL
Standard Deviation 21.8728
|
SECONDARY outcome
Timeframe: Day 1, Day 29, Day 85, Day 169, Day 253, Day 337, Day 421, Day 449Population: Pharmacodynamic Analysis Set: all participants who received at least 1 dose of study intervention and who have evaluable pharmacodynamic data. Here, 'Number Analyzed' signifies those participants evaluable for this outcome measure at specified timepoints.
Blood samples were collected for measurement of serum concentration of total C5 at specified timepoints. No study intervention administered on Day 449. A decrease indicated a reduction in disease activity.
Outcome measures
| Measure |
Eculizumab
n=25 Participants
Participants received eculizumab by IV infusion.
|
|---|---|
|
Change From Baseline in Serum Total C5 Concentration
Day 85
|
52.8607 ug/mL
Standard Deviation 17.8070
|
|
Change From Baseline in Serum Total C5 Concentration
Day 1
|
-13.3422 ug/mL
Standard Deviation 6.3902
|
|
Change From Baseline in Serum Total C5 Concentration
Day 29
|
49.5372 ug/mL
Standard Deviation 15.2890
|
|
Change From Baseline in Serum Total C5 Concentration
Day 169
|
50.9998 ug/mL
Standard Deviation 17.9024
|
|
Change From Baseline in Serum Total C5 Concentration
Day 253
|
61.3600 ug/mL
Standard Deviation 24.0315
|
|
Change From Baseline in Serum Total C5 Concentration
Day 337
|
60.9581 ug/mL
Standard Deviation 24.3898
|
|
Change From Baseline in Serum Total C5 Concentration
Day 421
|
62.8651 ug/mL
Standard Deviation 19.0970
|
|
Change From Baseline in Serum Total C5 Concentration
Day 449
|
64.4332 ug/mL
Standard Deviation 22.4364
|
SECONDARY outcome
Timeframe: Baseline through Week 64Population: Immunogenicity Analysis Set: all participants who received at least 1 dose of eculizumab and with an ADA result at baseline and at least 1 post-baseline assessment.
Blood samples were collected for measurement of treatment-emergent ADAs to eculizumab at specified timepoints.
Outcome measures
| Measure |
Eculizumab
n=25 Participants
Participants received eculizumab by IV infusion.
|
|---|---|
|
Number of Participants With Treatment-emergent Antidrug Antibodies (ADAs) to Eculizumab
|
0 Participants
|
SECONDARY outcome
Timeframe: Baseline, Week 12Population: Full Analysis Set: all participants who received at least 1 dose of study intervention and had at least 1 efficacy assessment post first dose.
The FACIT-Fatigue is a 13-item questionnaire that assesses self-reported fatigue and its impact upon daily activities and function over the preceding 7 days. The FACIT-Fatigue scale is a collection of quality of life (QoL) questionnaires pertaining to the management of fatigue symptoms due to a chronic illness. Participants scored each item on a 5-point scale: 0 (Not at all) to 4 (Very much). Total scores were calculated as the sum from all 13 items and ranged from 0 to 52, with higher scores indicating an increased QoL. Analysis was performed using a MMRM, including FACIT-Fatigue score change from baseline values at the scheduled visits from Week 1 to Week 12 as the dependent variable, with the fixed categorical effect of study visit, fixed continuous effect of baseline value of FACIT-Fatigue, and participant as random effect. Assessed at baseline, 1, 2, 3, 4, 6, 8, 10, and 12 weeks, Week 12 reported.
Outcome measures
| Measure |
Eculizumab
n=25 Participants
Participants received eculizumab by IV infusion.
|
|---|---|
|
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Score at Week 12
|
5.6 score on a scale
Interval 2.3 to 8.9
|
SECONDARY outcome
Timeframe: Baseline through Week 12Population: Full Analysis Set: all participants who received at least 1 dose of study intervention and had at least 1 efficacy assessment post first dose.
Breakthrough hemolysis was defined as at least 1 new or worsening symptom or sign of intravascular hemolysis (fatigue, hemoglobinuria, abdominal pain, shortness of breath \[dyspnea\], anemia \[hemoglobin \<10 grams/deciliter\], major adverse vascular event \[including thrombosis\], dysphagia, or erectile dysfunction) in the presence of elevated LDH ≥2 times (\*) upper limit of normal (ULN), after prior LDH reduction to \<1.5\* ULN on therapy.
Outcome measures
| Measure |
Eculizumab
n=25 Participants
Participants received eculizumab by IV infusion.
|
|---|---|
|
Percentage of Participants With Breakthrough Hemolysis
|
4.0 percentage of participants
Interval 0.1 to 20.4
|
SECONDARY outcome
Timeframe: Baseline through Week 12Population: Full Analysis Set: all participants who received at least 1 dose of study intervention and had at least 1 efficacy assessment post first dose.
Blood samples were collected for measurement of LDH at specified timepoints. LDH normalization was defined as LDH ≤ULN.
Outcome measures
| Measure |
Eculizumab
n=25 Participants
Participants received eculizumab by IV infusion.
|
|---|---|
|
Percentage of Participants Achieving LDH Normalization
|
36.0 percentage of participants
Interval 18.0 to 57.5
|
SECONDARY outcome
Timeframe: Baseline through Week 12Population: Full Analysis Set: all participants who received at least 1 dose of study intervention and had at least 1 efficacy assessment post first dose.
Transfusion avoidance was defined as remaining transfusion free (that is, had not received any transfusion) and did not require transfusion as per protocol.
Outcome measures
| Measure |
Eculizumab
n=25 Participants
Participants received eculizumab by IV infusion.
|
|---|---|
|
Percentage of Participants Achieving Transfusion Avoidance
|
96.0 percentage of participants
Interval 79.6 to 99.9
|
SECONDARY outcome
Timeframe: Baseline through Week 12Population: Full Analysis Set: all participants who received at least 1 dose of study intervention and had at least 1 efficacy assessment post first dose.
Blood samples were collected for measurement of LDH at specified timepoints.
Outcome measures
| Measure |
Eculizumab
n=25 Participants
Participants received eculizumab by IV infusion.
|
|---|---|
|
Percentage of Participants With LDH ≤1.5* ULN at Week 12
|
80.0 percentage of participants
Interval 59.3 to 93.2
|
Adverse Events
Eculizumab
Serious adverse events
| Measure |
Eculizumab
n=25 participants at risk
Participants received eculizumab by IV infusion.
|
|---|---|
|
Infections and infestations
Complicated appendicitis
|
4.0%
1/25 • Baseline through Week 64
All reported safety data based upon Safety Set: all participants who received at least 1 dose of eculizumab. All deaths regardless of causality or whether they were reported as a reason for study discontinuation are reported in the All-Cause Mortality section.
|
|
Infections and infestations
Endocarditis
|
4.0%
1/25 • Baseline through Week 64
All reported safety data based upon Safety Set: all participants who received at least 1 dose of eculizumab. All deaths regardless of causality or whether they were reported as a reason for study discontinuation are reported in the All-Cause Mortality section.
|
|
Infections and infestations
Febrile infection
|
4.0%
1/25 • Baseline through Week 64
All reported safety data based upon Safety Set: all participants who received at least 1 dose of eculizumab. All deaths regardless of causality or whether they were reported as a reason for study discontinuation are reported in the All-Cause Mortality section.
|
|
Infections and infestations
Hepatitis A
|
4.0%
1/25 • Baseline through Week 64
All reported safety data based upon Safety Set: all participants who received at least 1 dose of eculizumab. All deaths regardless of causality or whether they were reported as a reason for study discontinuation are reported in the All-Cause Mortality section.
|
|
Infections and infestations
Influenza
|
4.0%
1/25 • Baseline through Week 64
All reported safety data based upon Safety Set: all participants who received at least 1 dose of eculizumab. All deaths regardless of causality or whether they were reported as a reason for study discontinuation are reported in the All-Cause Mortality section.
|
|
Infections and infestations
Pneumonia
|
12.0%
3/25 • Baseline through Week 64
All reported safety data based upon Safety Set: all participants who received at least 1 dose of eculizumab. All deaths regardless of causality or whether they were reported as a reason for study discontinuation are reported in the All-Cause Mortality section.
|
|
Infections and infestations
Urinary tract infection
|
4.0%
1/25 • Baseline through Week 64
All reported safety data based upon Safety Set: all participants who received at least 1 dose of eculizumab. All deaths regardless of causality or whether they were reported as a reason for study discontinuation are reported in the All-Cause Mortality section.
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
4.0%
1/25 • Baseline through Week 64
All reported safety data based upon Safety Set: all participants who received at least 1 dose of eculizumab. All deaths regardless of causality or whether they were reported as a reason for study discontinuation are reported in the All-Cause Mortality section.
|
|
Nervous system disorders
Cerebral infarction
|
4.0%
1/25 • Baseline through Week 64
All reported safety data based upon Safety Set: all participants who received at least 1 dose of eculizumab. All deaths regardless of causality or whether they were reported as a reason for study discontinuation are reported in the All-Cause Mortality section.
|
|
Cardiac disorders
Myocardial infarction
|
4.0%
1/25 • Baseline through Week 64
All reported safety data based upon Safety Set: all participants who received at least 1 dose of eculizumab. All deaths regardless of causality or whether they were reported as a reason for study discontinuation are reported in the All-Cause Mortality section.
|
|
Hepatobiliary disorders
Cholecystitis
|
4.0%
1/25 • Baseline through Week 64
All reported safety data based upon Safety Set: all participants who received at least 1 dose of eculizumab. All deaths regardless of causality or whether they were reported as a reason for study discontinuation are reported in the All-Cause Mortality section.
|
|
Hepatobiliary disorders
Cholecystitis acute
|
4.0%
1/25 • Baseline through Week 64
All reported safety data based upon Safety Set: all participants who received at least 1 dose of eculizumab. All deaths regardless of causality or whether they were reported as a reason for study discontinuation are reported in the All-Cause Mortality section.
|
|
Hepatobiliary disorders
Jaundice cholestatic
|
4.0%
1/25 • Baseline through Week 64
All reported safety data based upon Safety Set: all participants who received at least 1 dose of eculizumab. All deaths regardless of causality or whether they were reported as a reason for study discontinuation are reported in the All-Cause Mortality section.
|
|
Investigations
Mycoplasma test positive
|
4.0%
1/25 • Baseline through Week 64
All reported safety data based upon Safety Set: all participants who received at least 1 dose of eculizumab. All deaths regardless of causality or whether they were reported as a reason for study discontinuation are reported in the All-Cause Mortality section.
|
|
Investigations
Platelet count decreased
|
4.0%
1/25 • Baseline through Week 64
All reported safety data based upon Safety Set: all participants who received at least 1 dose of eculizumab. All deaths regardless of causality or whether they were reported as a reason for study discontinuation are reported in the All-Cause Mortality section.
|
|
Injury, poisoning and procedural complications
Rib fracture
|
4.0%
1/25 • Baseline through Week 64
All reported safety data based upon Safety Set: all participants who received at least 1 dose of eculizumab. All deaths regardless of causality or whether they were reported as a reason for study discontinuation are reported in the All-Cause Mortality section.
|
Other adverse events
| Measure |
Eculizumab
n=25 participants at risk
Participants received eculizumab by IV infusion.
|
|---|---|
|
Infections and infestations
COVID-19
|
12.0%
3/25 • Baseline through Week 64
All reported safety data based upon Safety Set: all participants who received at least 1 dose of eculizumab. All deaths regardless of causality or whether they were reported as a reason for study discontinuation are reported in the All-Cause Mortality section.
|
|
Infections and infestations
H1N1 influenza
|
8.0%
2/25 • Baseline through Week 64
All reported safety data based upon Safety Set: all participants who received at least 1 dose of eculizumab. All deaths regardless of causality or whether they were reported as a reason for study discontinuation are reported in the All-Cause Mortality section.
|
|
Infections and infestations
Nasopharyngitis
|
8.0%
2/25 • Baseline through Week 64
All reported safety data based upon Safety Set: all participants who received at least 1 dose of eculizumab. All deaths regardless of causality or whether they were reported as a reason for study discontinuation are reported in the All-Cause Mortality section.
|
|
Infections and infestations
Upper respiratory tract infection
|
28.0%
7/25 • Baseline through Week 64
All reported safety data based upon Safety Set: all participants who received at least 1 dose of eculizumab. All deaths regardless of causality or whether they were reported as a reason for study discontinuation are reported in the All-Cause Mortality section.
|
|
Metabolism and nutrition disorders
Hyperuricaemia
|
12.0%
3/25 • Baseline through Week 64
All reported safety data based upon Safety Set: all participants who received at least 1 dose of eculizumab. All deaths regardless of causality or whether they were reported as a reason for study discontinuation are reported in the All-Cause Mortality section.
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
12.0%
3/25 • Baseline through Week 64
All reported safety data based upon Safety Set: all participants who received at least 1 dose of eculizumab. All deaths regardless of causality or whether they were reported as a reason for study discontinuation are reported in the All-Cause Mortality section.
|
|
Nervous system disorders
Headache
|
20.0%
5/25 • Baseline through Week 64
All reported safety data based upon Safety Set: all participants who received at least 1 dose of eculizumab. All deaths regardless of causality or whether they were reported as a reason for study discontinuation are reported in the All-Cause Mortality section.
|
|
Hepatobiliary disorders
Hepatic function abnormal
|
8.0%
2/25 • Baseline through Week 64
All reported safety data based upon Safety Set: all participants who received at least 1 dose of eculizumab. All deaths regardless of causality or whether they were reported as a reason for study discontinuation are reported in the All-Cause Mortality section.
|
|
General disorders
Pyrexia
|
16.0%
4/25 • Baseline through Week 64
All reported safety data based upon Safety Set: all participants who received at least 1 dose of eculizumab. All deaths regardless of causality or whether they were reported as a reason for study discontinuation are reported in the All-Cause Mortality section.
|
|
Investigations
Neutrophil count decreased
|
12.0%
3/25 • Baseline through Week 64
All reported safety data based upon Safety Set: all participants who received at least 1 dose of eculizumab. All deaths regardless of causality or whether they were reported as a reason for study discontinuation are reported in the All-Cause Mortality section.
|
|
Investigations
White blood cell count decreased
|
8.0%
2/25 • Baseline through Week 64
All reported safety data based upon Safety Set: all participants who received at least 1 dose of eculizumab. All deaths regardless of causality or whether they were reported as a reason for study discontinuation are reported in the All-Cause Mortality section.
|
Additional Information
Alexion Pharmaceuticals, Inc.
Alexion Pharmaceuticals, Inc.
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: OTHER