Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| ECU-PNH-301 | Other Identifier | Alexion |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Class |
|---|---|
| AstraZeneca | INDUSTRY |
Not provided
Not provided
Not provided
Not provided
This is a Phase 3b, single-arm, open-label, multicenter study to evaluate the efficacy, safety, pharmacokinetics (PK), pharmacodynamics (PD), and immunogenicity of eculizumab in adult participants with paraxysmal noturnal hemoglobinuria (PNH) in China.
This is a Phase 3b, single-arm, open-label, multicenter study to evaluate the efficacy, safety, pharmacokinetics (PK), pharmacodynamics (PD), and immunogenicity of eculizumab in adult participants with PNH in China who previously have not been treated with complement inhibitors. Approximately 25 eligible participants in China will be enrolled.
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Eculizumab | Experimental | Eculizumab will be administered by IV infusion. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Eculizumab | Drug | Participants will receive 600 milligrams (mg) once a week on Day 1, 8, 15, and 22 followed by 900 mg every 2 weeks from Day 29 to Day 435. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Percentage Change From Baseline in LDH at Week 12 | 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. | Baseline, Week 12 |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) | 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. |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Research Site | Beijing | CN-100730 | China | |||
| Research Site |
Not provided
| Label | URL |
|---|---|
| Redacted CSR Synopsis | View source |
Not provided
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.
Not provided
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Eculizumab | Participants received eculizumab by intravenous (IV) infusion. |
| Title | Milestones | Reasons Not Completed | |||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary Treatment Period (12 Weeks) |
| ||||||||||||||||
| Long-Term Extension Period (52 Weeks) |
|
Full Analysis Set: all participants who received at least 1 dose of study intervention and had at least 1 efficacy assessment post first dose.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Eculizumab | Participants received eculizumab by IV infusion. |
| 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 Change From Baseline in LDH at Week 12 | 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. | Full Analysis Set: all participants who received at least 1 dose of study intervention and had at least 1 efficacy assessment post first dose. | Posted | Least Squares Mean | 95% Confidence Interval | percentage change | Baseline, Week 12 |
|
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.
Not provided
| 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 by IV infusion. | 1 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Complicated appendicitis | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| COVID-19 | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Alexion Pharmaceuticals, Inc. | Alexion Pharmaceuticals, Inc. | 1-855-752-2356 | clinicaltrials@alexion.com |
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Nov 6, 2023 | Mar 26, 2026 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | May 8, 2025 | Mar 26, 2026 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D006457 | Hemoglobinuria, Paroxysmal |
| ID | Term |
|---|---|
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C481642 | eculizumab |
Not provided
Not provided
Not provided
open label
Not provided
Not provided
Not provided
Not provided
| Baseline through Week 64 |
| Serum Concentration of Eculizumab | 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). | Day 1, Day 29, Day 85, Day 169, Day 253, Day 337, Day 421, Day 449 |
| Change From Baseline in Serum Free Complement 5 (C5) Concentration | 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. | Day 1, Day 29, Day 85, Day 169, Day 253, Day 337, Day 421, Day 449 |
| Change From Baseline in Serum Total C5 Concentration | 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. | Day 1, Day 29, Day 85, Day 169, Day 253, Day 337, Day 421, Day 449 |
| Number of Participants With Treatment-emergent Antidrug Antibodies (ADAs) to Eculizumab | Blood samples were collected for measurement of treatment-emergent ADAs to eculizumab at specified timepoints. | Baseline through Week 64 |
| Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Score at Week 12 | 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. | Baseline, Week 12 |
| Percentage of Participants With Breakthrough Hemolysis | 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. | Baseline through Week 12 |
| Percentage of Participants Achieving LDH Normalization | Blood samples were collected for measurement of LDH at specified timepoints. LDH normalization was defined as LDH ≤ULN. | Baseline through Week 12 |
| Percentage of Participants Achieving Transfusion Avoidance | Transfusion avoidance was defined as remaining transfusion free (that is, had not received any transfusion) and did not require transfusion as per protocol. | Baseline through Week 12 |
| Percentage of Participants With LDH ≤1.5* ULN at Week 12 | Blood samples were collected for measurement of LDH at specified timepoints. | Baseline through Week 12 |
| Hangzhou |
| 310003 |
| China |
| Research Site | Nantong | 226001 | China |
| Research Site | Shanghai | 200040 | China |
| Research Site | Tianjin | 300020 | China |
| Research Site | Tianjin | 300050 | China |
| Research Site | Wuhan | 430022 | China |
| NOT COMPLETED |
|
|
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Baseline Lactate Dehydrogenase (LDH) | LDH reported in units/liter (U/L). | Mean | Standard Deviation | U/L |
|
|
|
|
| Secondary | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | 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. | Safety Set: all participants who received at least 1 dose of study intervention. | Posted | Count of Participants | Participants | Baseline through Week 64 |
|
|
|
| Secondary | Serum Concentration of Eculizumab | 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). | 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. | Posted | Geometric Mean | Geometric Coefficient of Variation | ug/mL | Day 1, Day 29, Day 85, Day 169, Day 253, Day 337, Day 421, Day 449 |
|
|
|
| Secondary | Change From Baseline in Serum Free Complement 5 (C5) Concentration | 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. | 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. | Posted | Mean | Standard Deviation | ug/mL | Day 1, Day 29, Day 85, Day 169, Day 253, Day 337, Day 421, Day 449 |
|
|
|
| Secondary | Change From Baseline in Serum Total C5 Concentration | 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. | 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. | Posted | Mean | Standard Deviation | ug/mL | Day 1, Day 29, Day 85, Day 169, Day 253, Day 337, Day 421, Day 449 |
|
|
|
| Secondary | Number of Participants With Treatment-emergent Antidrug Antibodies (ADAs) to Eculizumab | Blood samples were collected for measurement of treatment-emergent ADAs to eculizumab at specified timepoints. | 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. | Posted | Count of Participants | Participants | Baseline through Week 64 |
|
|
|
| Secondary | Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Score at Week 12 | 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. | Full Analysis Set: all participants who received at least 1 dose of study intervention and had at least 1 efficacy assessment post first dose. | Posted | Least Squares Mean | 95% Confidence Interval | score on a scale | Baseline, Week 12 |
|
|
|
| Secondary | Percentage of Participants With Breakthrough Hemolysis | 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. | Full Analysis Set: 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 | Baseline through Week 12 |
|
|
|
| Secondary | Percentage of Participants Achieving LDH Normalization | Blood samples were collected for measurement of LDH at specified timepoints. LDH normalization was defined as LDH ≤ULN. | Full Analysis Set: 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 | Baseline through Week 12 |
|
|
|
| Secondary | Percentage of Participants Achieving Transfusion Avoidance | Transfusion avoidance was defined as remaining transfusion free (that is, had not received any transfusion) and did not require transfusion as per protocol. | Full Analysis Set: 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 | Baseline through Week 12 |
|
|
|
| Secondary | Percentage of Participants With LDH ≤1.5* ULN at Week 12 | Blood samples were collected for measurement of LDH at specified timepoints. | Full Analysis Set: 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 | Baseline through Week 12 |
|
|
|
| 25 |
| 9 |
| 25 |
| 21 |
| 25 |
| Endocarditis | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
|
| Febrile infection | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
|
| Hepatitis A | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
|
| Influenza | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
|
| Pneumonia | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
|
| Urinary tract infection | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
|
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA 27.1 | Systematic Assessment |
|
| Cerebral infarction | Nervous system disorders | MedDRA 27.1 | Systematic Assessment |
|
| Myocardial infarction | Cardiac disorders | MedDRA 27.1 | Systematic Assessment |
|
| Cholecystitis | Hepatobiliary disorders | MedDRA 27.1 | Systematic Assessment |
|
| Cholecystitis acute | Hepatobiliary disorders | MedDRA 27.1 | Systematic Assessment |
|
| Jaundice cholestatic | Hepatobiliary disorders | MedDRA 27.1 | Systematic Assessment |
|
| Mycoplasma test positive | Investigations | MedDRA 27.1 | Systematic Assessment |
|
| Platelet count decreased | Investigations | MedDRA 27.1 | Systematic Assessment |
|
| Rib fracture | Injury, poisoning and procedural complications | MedDRA 27.1 | Systematic Assessment |
|
| H1N1 influenza | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA 27.1 | Systematic Assessment |
|
| Hyperuricaemia | Metabolism and nutrition disorders | MedDRA 27.1 | Systematic Assessment |
|
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA 27.1 | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA 27.1 | Systematic Assessment |
|
| Hepatic function abnormal | Hepatobiliary disorders | MedDRA 27.1 | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA 27.1 | Systematic Assessment |
|
| Neutrophil count decreased | Investigations | MedDRA 27.1 | Systematic Assessment |
|
| White blood cell count decreased | Investigations | MedDRA 27.1 | Systematic Assessment |
|
Not provided
| D009190 |
| Myelodysplastic Syndromes |
| D001855 | Bone Marrow Diseases |
|
| Day 85 |
|
|
| Day 169 |
|
|
| Day 253 |
|
|
| Day 337 |
|
|
| Day 421 |
|
|
| Day 449 |
|
|
|
| Day 85 |
|
|
| Day 169 |
|
|
| Day 253 |
|
|
| Day 337 |
|
|
| Day 421 |
|
|
| Day 449 |
|
|
|
| Day 85 |
|
|
| Day 169 |
|
|
| Day 253 |
|
|
| Day 337 |
|
|
| Day 421 |
|
|
| Day 449 |
|
|