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| ID | Type | Description | Link |
|---|---|---|---|
| ECU-aHUS-302 | Other Identifier | Alexion | |
| 2025-000162-29 | EudraCT Number |
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| Name | Class |
|---|---|
| AstraZeneca | INDUSTRY |
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This is a Phase 3b, open-label, single-arm, multicenter study to evaluate the efficacy and safety of eculizumab in participants with atypical hemolytic uremic syndrome (aHUS) in China
This is a Phase 3b, open-label, single-arm, multicenter study to evaluate the efficacy and safety of eculizumab in participants with aHUS in China. The study will be conducted in participants of any age who weigh ≥ 5 kg and who previously have not been treated with complement inhibitors. The study consists of an up to 7-day Screening Period and a 26-week Treatment Period. An 8-week Safety Follow-up Phone Call will be required only for participants who discontinue eculizumab treatment during the study or for participants who will not receive continued access to eculizumab after completing study treatment. Approximately 25 eligible participants in China will be enrolled.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Eculizumab | Experimental | Participants will receive Eculizumab in a single dose vial. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Eculizumab | Drug | Weight-based doses of Eculizumab will be administered intravenously as an induction dose followed by maintenance dose at Day 8, 15, or 29 depending on weight; then every 2 or 3 weeks, depending upon weight. |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants With a Complete Thrombotic Microangiopathy (TMA) Response | The criteria for complete TMA response were:
| Up to Week 26 |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With an Adverse Event (AE) | 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:
A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Adverse Events' Section. |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Research Site | Beijing | 100034 | China | |||
| Research Site |
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| Label | URL |
|---|---|
| Related Info | View source |
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Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal.
All request will be evaluated as per the AZ disclosure commitment:
https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure. Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.
AstraZeneca will meet or exceed data availability as per the commitments made to the EFPIA Pharma Data Sharing Principles. For details of our timelines, please refer to our disclosure commitment at https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.
When a request has been approved AstraZeneca will provide access to the deidentified individual patient-level data in an approved sponsored tool . Signed Data Sharing Agreement (non-negotiable contract for data accessors) must be in place before accessing requested information. Additionally, all users will need to accept the terms and conditions of the SAS MSE to gain access. For additional details, please review the Disclosure Statements at https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.
After providing informed consent/assent, participants were screened for eligibility for the study during the 7-day Screening Period.
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| ID | Title | Description |
|---|---|---|
| FG000 | Eculizumab | Participants received eculizumab as an intravenous (IV) infusion at a dose and schedule according to body weight for 26 weeks. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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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| ID | Title | Description |
|---|---|---|
| BG000 | Eculizumab | Participants received eculizumab as an IV infusion at a dose and schedule according to body weight for 26 weeks. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Percentage of Participants With a Complete Thrombotic Microangiopathy (TMA) Response | The criteria for complete TMA response were:
| 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. | Posted | Number | 95% Confidence Interval | percentage of participants | Up to Week 26 |
|
Up to Week 34
Safety Set: Included all participants who received at least 1 dose of study intervention.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Eculizumab | Participants received eculizumab as an IV infusion at a dose and schedule according to body weight for 26 weeks. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Bacterial infection | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Bronchitis | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Alexion Pharmaceuticals Inc. | European Clinical Trial Information | +1.855.752.2356 | clinicaltrials@alexion.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Apr 26, 2023 | Nov 5, 2025 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | May 20, 2025 | Nov 5, 2025 | SAP_001.pdf |
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| ID | Term |
|---|---|
| C481642 | eculizumab |
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open-label study
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| Up to Week 34 |
| Mean Serum Concentration of Eculizumab | Pre-dose and post-dose at Days 1, 8, 29, 85, and 141; Pre-dose at Day 183 |
| Change From Baseline in Serum Free Complement 5 (C5) | Baseline (Day 1 pre-dose) to Days 1, 8, 29, 85 and 141 (pre-dose and post-dose) and pre-dose at Day 183 |
| Change From Baseline in Serum Total C5 | Baseline (Day 1 pre-dose) to Days 1, 8, 29, 85 and 141 (pre-dose and post-dose) and pre-dose at Day 183 |
| Number of Participants With an Anti-drug Antibody (ADA) Response | An ADA response was defined as a positive ADA sample at any time during the study. | Up to Week 26 |
| Time to Complete TMA Response | 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:
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. | Up to Week 26 |
| Proportion of Participants On or Off Dialysis at Each Timepoint | 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. | Baseline and Days 22, 43, 71, 99, 113, 127, 155 and 183 |
| Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Each Scheduled Visit | Expressed in milliliters per minute per 1.73 square meters of body surface area. | Baseline, Days 22, 43, 71, 99, 113, 127, 155 and 183 |
| Proportion of Participants With a Chronic Kidney Disease (CKD) Stage Shift Categorized as "Improved", "Stable", or "Worsened" at Each Scheduled Visit Compared to Baseline | 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. | Baseline to Days 22, 43, 71, 99, 113, 127, 155 and 183 |
| Change From Baseline in Platelets | Platelet values obtained from the day of a blood transfusion of platelets through 3 days after the transfusion are excluded from all analysis. | Baseline, Days 22, 43, 71, 99, 113, 127, 155, and 183 |
| Change From Baseline in LDH | Baseline, Days 22, 43, 71, 99, 113, 127, 155, and 183 |
| Change From Baseline in Hemoglobin | 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. | Baseline, Days 22, 43, 71, 99, 113, 127, 155, and 183 |
| Beijing |
| 100045 |
| China |
| Research Site | Changsha | 410007 | China |
| Research Site | Qingdao | 110016 | China |
| Research Site | Taiyuan | 030012 | China |
| Research Site | Wuhan | 430030 | China |
| years |
|
| Age, Customized | Count of Participants | Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Units | Counts |
|---|---|
| Participants |
|
|
| Secondary | Number of Participants With an Adverse Event (AE) | 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:
A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Adverse Events' Section. | Safety Set: Included all participants who received at least 1 dose of study intervention. | Posted | Count of Participants | Participants | Up to Week 34 |
|
|
|
| Secondary | Mean Serum Concentration of Eculizumab | 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. | Posted | Geometric Mean | Geometric Coefficient of Variation | micrograms per milliliter (ug/mL) | Pre-dose and post-dose at Days 1, 8, 29, 85, and 141; Pre-dose at Day 183 |
|
|
|
| Secondary | Change From Baseline in Serum Free Complement 5 (C5) | 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. | Posted | Mean | Standard Deviation | ug/mL | Baseline (Day 1 pre-dose) to Days 1, 8, 29, 85 and 141 (pre-dose and post-dose) and pre-dose at Day 183 |
|
|
|
| Secondary | Change From Baseline in Serum Total C5 | 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. | Posted | Mean | Standard Deviation | ug/mL | Baseline (Day 1 pre-dose) to Days 1, 8, 29, 85 and 141 (pre-dose and post-dose) and pre-dose at Day 183 |
|
|
|
| Secondary | Number of Participants With an Anti-drug Antibody (ADA) Response | 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. | Posted | Count of Participants | Participants | Up to Week 26 |
|
|
|
| Secondary | Time to Complete TMA Response | 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:
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. | 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. | Posted | Median | 95% Confidence Interval | days | Up to Week 26 |
|
|
|
| Secondary | Proportion of Participants On or Off Dialysis at Each Timepoint | 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. | 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 specified timepoint. | Posted | Number | 95% Confidence Interval | proportion of participants | Baseline and Days 22, 43, 71, 99, 113, 127, 155 and 183 |
|
|
|
| Secondary | Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Each Scheduled Visit | Expressed in milliliters 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. '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. | Posted | Mean | Standard Deviation | mL/min/1.73^2 | Baseline, Days 22, 43, 71, 99, 113, 127, 155 and 183 |
|
|
|
| Secondary | Proportion of Participants With a Chronic Kidney Disease (CKD) Stage Shift Categorized as "Improved", "Stable", or "Worsened" at Each Scheduled Visit Compared to Baseline | 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. | 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. | Posted | Number | 95% Confidence Interval | proportion of participants | Baseline to Days 22, 43, 71, 99, 113, 127, 155 and 183 |
|
|
|
| Secondary | Change From Baseline in Platelets | 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. 'Overall number of participants analyzed' = participants evaluable for this outcome measure. 'Number Analyzed' = number of participants evaluable at specified timepoint. | Posted | Mean | Standard Deviation | 10^9 platelets/liter (L) | Baseline, Days 22, 43, 71, 99, 113, 127, 155, and 183 |
|
|
|
| Secondary | Change From Baseline in LDH | 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. | Posted | Mean | Standard Deviation | microkatal per liter (ukat/L) | Baseline, Days 22, 43, 71, 99, 113, 127, 155, and 183 |
|
|
|
| Secondary | Change From Baseline in Hemoglobin | 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. 'Overall number of participants analyzed' = participants evaluable for this outcome measure. 'Number Analyzed' = number of participants evaluable at the specific timepoint. | Posted | Mean | Standard Deviation | grams per liter (g/L) | Baseline, Days 22, 43, 71, 99, 113, 127, 155, and 183 |
|
|
|
| 0 |
| 25 |
| 8 |
| 25 |
| 24 |
| 25 |
| Clostridium difficile infection | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
|
| Infection | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
|
| Pneumonia | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
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| Rotavirus infection | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
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| Retching | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
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| Upper gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
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| Hepatic function abnormal | Hepatobiliary disorders | MedDRA 27.1 | Systematic Assessment |
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| Synovial cyst | Musculoskeletal and connective tissue disorders | MedDRA 27.1 | Systematic Assessment |
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| Infection | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
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| Influenza | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
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| Respiratory tract infection | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
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| Upper respiratory tract infection | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
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| Anaemia | Blood and lymphatic system disorders | MedDRA 27.1 | Systematic Assessment |
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| Leukopenia | Blood and lymphatic system disorders | MedDRA 27.1 | Systematic Assessment |
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| Electrolyte imbalance | Metabolism and nutrition disorders | MedDRA 27.1 | Systematic Assessment |
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| Hyperkalaemia | Metabolism and nutrition disorders | MedDRA 27.1 | Systematic Assessment |
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| Hyperphosphataemia | Metabolism and nutrition disorders | MedDRA 27.1 | Systematic Assessment |
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| Hypokalaemia | Metabolism and nutrition disorders | MedDRA 27.1 | Systematic Assessment |
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| Hypertension | Vascular disorders | MedDRA 27.1 | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 27.1 | Systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA 27.1 | Systematic Assessment |
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| Hepatic function abnormal | Hepatobiliary disorders | MedDRA 27.1 | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA 27.1 | Systematic Assessment |
|
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| Day 8: Pre-dose |
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| Day 8: Post-dose |
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| Day 29: Pre-dose |
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| Day 29: Post-dose |
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| Day 85: Pre-dose |
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| Day 85: Post-dose |
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| Day 141: Pre-dose |
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| Day 141: Post-dose |
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| Day 183: Pre-dose |
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| Day 8: Post-dose |
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| Day 29: Pre-dose |
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| Day 29: Post-dose |
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| Day 85: Pre-dose |
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| Day 85: Post-dose |
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| Day 141: Pre-dose |
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| Day 141: Post-dose |
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| Day 183: Pre-dose |
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| Day 8: Post-dose |
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| Day 29: Pre-dose |
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| Day 29: Post-dose |
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| Day 85: Pre-dose |
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| Day 85: Post-dose |
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| Day 141: Pre-dose |
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| Day 141: Post-dose |
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| Day 183: Pre-dose |
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| Day 22: On Dialysis |
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| Day 22: Off Dialysis |
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| Day 43: On Dialysis |
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| Day 43: Off Dialysis |
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| Day 71: On Dialysis |
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| Day 71: Off Dialysis |
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| Day 99: On Dialysis |
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| Day 99: Off Dialysis |
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| Day 113: On Dialysis |
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| Day 113: Off Dialysis |
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| Day 127: On Dialysis |
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| Day 127: Off Dialysis |
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| Day 155: On Dialysis |
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| Day 155: Off Dialysis |
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| Day 183: On Dialysis |
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| Day 183: Off Dialysis |
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| Day 71 |
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| Day 99 |
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| Day 113 |
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| Day 127 |
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| Day 155 |
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| Day 183 |
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| Day 22: Worsened |
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| Day 43: Improved |
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| Day 43: Stable |
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| Day 43: Worsened |
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| Day 71: Improved |
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| Day 71: Stable |
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| Day 71: Worsened |
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| Day 99: Improved |
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| Day 99: Stable |
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| Day 99: Worsened |
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| Day 113: Improved |
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| Day 113: Stable |
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| Day 113: Worsened |
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| Day 127: Improved |
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| Day 127: Stable |
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| Day 127: Worsened |
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| Day 155: Improved |
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| Day 155: Stable |
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| Day 155: Worsened |
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| Day 183: Improved |
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| Day 183: Stable |
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| Day 183: Worsened |
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| Day 71 |
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| Day 99 |
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| Day 113 |
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| Day 127 |
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| Day 155 |
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| Day 183 |
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| Day 71 |
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| Day 99 |
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| Day 113 |
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| Day 127 |
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| Day 155 |
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| Day 183 |
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| Day 71 |
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| Day 99 |
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| Day 113 |
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| Day 127 |
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| Day 155 |
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| Day 183 |
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