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| ID | Type | Description | Link |
|---|---|---|---|
| ALXN1210-PNH-323 | Other Identifier | Alexion |
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The primary objective of this study is to evaluate the efficacy of ravulizumab in adult participants with PNH.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Ravulizumab | Experimental | During the Primary Treatment Period, participants will receive a weight-based loading dose of ravulizumab on Day 1 followed by weight-based maintenance dose of ravulizumab on Day 15 and once every 8 weeks (q8w) thereafter for a total of 26 weeks. On Day 183, all participants will enter a 32-week Extension Treatment Period and receive ravulizumab. Beginning on Day 183, participants will receive a maintenance dose of ravulizumab q8w for an additional 32 weeks. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Ravulizumab | Drug | Ravulizumab will be administered by intravenous (IV) infusion. |
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| Measure | Description | Time Frame |
|---|---|---|
| Percentage Change in Lactate Dehydrogenase (LDH) From Baseline to Day 183 (Week 26) | LDH is an indicator of intravascular hemolysis that occurs in participants with paroxysmal nocturnal hemoglobinuria. A decrease in LDH indicated reduction (improvement) in hemolysis. Baseline was defined as the average of all available on-study assessments prior to the first dose of study drug. The percent change in LDH was analyzed using a mixed-effect model for repeated measures (MMRM) with the fixed categorical effect of visit, fixed continuous effect of the LDH baseline value as covariates, and participant as random effect. | Baseline, Day 183 (Week 26) |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants Achieving LDH <1.5 * Upper Limit of Normal (ULN) at Day 183 (Week 26) | LDH is an indicator of intravascular hemolysis that occurs in participants with paroxysmal nocturnal hemoglobinuria. A decrease in LDH indicated reduction (improvement) in hemolysis. Baseline was defined as the average of all available on-study assessments prior to the first dose of study drug. | Day 183 (Week 26) |
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Inclusion Criteria:
Exclusion Criteria
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Research Site | Beijing | CN-100730 | China | |||
| Research Site |
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| Label | URL |
|---|---|
| Related Info | View source |
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Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.
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The study included a 26-week Primary Treatment Period, and an additional 32-week Extension Treatment Period. The results for the Primary Treatment Period have been reported. Final analysis data will be reported after completion of the 32-week Extension Treatment Period.
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| ID | Title | Description |
|---|---|---|
| FG000 | Ravulizumab | Participants received a weight-based loading dose of ravulizumab on Day 1 followed by weight-based maintenance dose of ravulizumab on Day 15 and once every 8 weeks (q8w) thereafter for a total of 26 weeks in Primary Treatment Period. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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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 | Mar 12, 2024 | May 11, 2026 |
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| Percentage of Participants Achieving Transfusion Avoidance Through Day 183 (Week 26) | Transfusion avoidance was defined as the percentage of participants who remained transfusion free and did not require a transfusion per protocol-specified guidelines (hemoglobin value of ≤9 grams (g)/deciliter (dL) with signs or symptoms of sufficient severity to warrant a transfusion, or a hemoglobin value of ≤7 g/dL regardless of presence of clinical signs or symptoms) through Day 183. | Day 183 (Week 26) |
| Percentage of Participants Experiencing Breakthrough Hemolysis Through Day 183 (Week 26) | Breakthrough hemolysis was defined as at least one new or worsening symptom or sign of intravascular hemolysis (fatigue, hemoglobinuria, abdominal pain, dyspnea, anemia [hemoglobin <10 g/dL], major adverse vascular event [including thrombosis], dysphagia, or erectile dysfunction) in the presence of elevated LDH ≥2 times the ULN. | Day 183 (Week 26) |
| Change in Functional Assessment of Chronic Illness Therapy-Fatigue Scale (FACIT-Fatigue) Score From Baseline to Day 183 (Week 26) | 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. Participants scored each item on a 5-point scale: 0 (Not at all) to 4 (Very much). Total scores range from 0 to 52, with a higher score indicating better quality of life. Analysis was using a mixed-effect model for repeated measures (MMRM) with the fixed categorical effect of visit, fixed continuous effect of the baseline value of FACIT-Fatigue score as covariates, and participant as random effect. | Baseline, Day 183 (Week 26) |
| Change in Hemoglobin (Hgb) From Baseline to Day 183 (Week 26) | Analysis was performed using MMRM with the fixed categorical effect of visit, fixed continuous effect of the Hgb baseline value as covariates, and participant as random effect. | Baseline, Day 183 (Week 26) |
| Guangzhou |
| 510100 |
| China |
| Research Site | 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 |
| Received at Least 1 Dose of Study Drug |
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| COMPLETED | Completed Primary Treatment Period |
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| NOT COMPLETED |
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Full Analysis Set (FAS) included all enrolled participants who received at least 1 dose (full or partial) of the study intervention.
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| ID | Title | Description |
|---|---|---|
| BG000 | Ravulizumab | Participants received a weight-based loading dose of ravulizumab on Day 1 followed by weight-based maintenance dose of ravulizumab on Day 15 and once every 8 weeks (q8w) thereafter for a total of 26 weeks in Primary Treatment Period. |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| 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 in Lactate Dehydrogenase (LDH) From Baseline to Day 183 (Week 26) | LDH is an indicator of intravascular hemolysis that occurs in participants with paroxysmal nocturnal hemoglobinuria. A decrease in LDH indicated reduction (improvement) in hemolysis. Baseline was defined as the average of all available on-study assessments prior to the first dose of study drug. The percent change in LDH was analyzed using a mixed-effect model for repeated measures (MMRM) with the fixed categorical effect of visit, fixed continuous effect of the LDH baseline value as covariates, and participant as random effect. | FAS included all enrolled participants who received at least 1 dose (full or partial) of the study intervention. 'Overall number of participants analyzed' = participants evaluable for this outcome measure. | Posted | Least Squares Mean | 95% Confidence Interval | percent change | Baseline, Day 183 (Week 26) |
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| Secondary | Percentage of Participants Achieving LDH <1.5 * Upper Limit of Normal (ULN) at Day 183 (Week 26) | LDH is an indicator of intravascular hemolysis that occurs in participants with paroxysmal nocturnal hemoglobinuria. A decrease in LDH indicated reduction (improvement) in hemolysis. Baseline was defined as the average of all available on-study assessments prior to the first dose of study drug. | FAS included all enrolled participants who received at least 1 dose (full or partial) of the study intervention. 'Overall number of participants analyzed' = participants evaluable for this outcome measure. | Posted | Number | 95% Confidence Interval | percentage of participants | Day 183 (Week 26) |
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| Secondary | Percentage of Participants Achieving Transfusion Avoidance Through Day 183 (Week 26) | Transfusion avoidance was defined as the percentage of participants who remained transfusion free and did not require a transfusion per protocol-specified guidelines (hemoglobin value of ≤9 grams (g)/deciliter (dL) with signs or symptoms of sufficient severity to warrant a transfusion, or a hemoglobin value of ≤7 g/dL regardless of presence of clinical signs or symptoms) through Day 183. | FAS included all enrolled participants who received at least 1 dose (full or partial) of the study intervention. | Posted | Number | 95% Confidence Interval | percentage of participants | Day 183 (Week 26) |
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| Secondary | Percentage of Participants Experiencing Breakthrough Hemolysis Through Day 183 (Week 26) | Breakthrough hemolysis was defined as at least one new or worsening symptom or sign of intravascular hemolysis (fatigue, hemoglobinuria, abdominal pain, dyspnea, anemia [hemoglobin <10 g/dL], major adverse vascular event [including thrombosis], dysphagia, or erectile dysfunction) in the presence of elevated LDH ≥2 times the ULN. | FAS included all enrolled participants who received at least 1 dose (full or partial) of the study intervention. | Posted | Number | 95% Confidence Interval | percentage of participants | Day 183 (Week 26) |
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| Secondary | Change in Functional Assessment of Chronic Illness Therapy-Fatigue Scale (FACIT-Fatigue) Score From Baseline to Day 183 (Week 26) | 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. Participants scored each item on a 5-point scale: 0 (Not at all) to 4 (Very much). Total scores range from 0 to 52, with a higher score indicating better quality of life. Analysis was using a mixed-effect model for repeated measures (MMRM) with the fixed categorical effect of visit, fixed continuous effect of the baseline value of FACIT-Fatigue score as covariates, and participant as random effect. | FAS included all enrolled participants who received at least 1 dose (full or partial) of the study intervention. | Posted | Least Squares Mean | 95% Confidence Interval | units on a scale | Baseline, Day 183 (Week 26) |
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| Secondary | Change in Hemoglobin (Hgb) From Baseline to Day 183 (Week 26) | Analysis was performed using MMRM with the fixed categorical effect of visit, fixed continuous effect of the Hgb baseline value as covariates, and participant as random effect. | FAS included all enrolled participants who received at least 1 dose (full or partial) of the study intervention. | Posted | Least Squares Mean | 95% Confidence Interval | g/liter (L) | Baseline, Day 183 (Week 26) |
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Baseline up to Week 26
Safety Set included all enrolled participants who received at least 1 dose (full or partial) of the 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 | Ravulizumab | Participants received a weight-based loading dose of ravulizumab on Day 1 followed by weight-based maintenance dose of ravulizumab on Day 15 and once every 8 weeks (q8w) thereafter for a total of 26 weeks in Primary Treatment Period. | 0 | 18 | 2 | 18 | 16 | 18 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Liver injury | Hepatobiliary disorders | MedDRA 28.0 | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA 28.0 | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Influenza | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
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| Periodontitis | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
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| Sinusitis | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
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| Tinea pedis | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
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| Upper respiratory tract infection | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
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| Decreased appetite | Metabolism and nutrition disorders | MedDRA 28.0 | Systematic Assessment |
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| Hyperuricaemia | Metabolism and nutrition disorders | MedDRA 28.0 | Systematic Assessment |
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| Hypoalbuminaemia | Metabolism and nutrition disorders | MedDRA 28.0 | Systematic Assessment |
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| Hypokalaemia | Metabolism and nutrition disorders | MedDRA 28.0 | Systematic Assessment |
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| Insomnia | Psychiatric disorders | MedDRA 28.0 | Systematic Assessment |
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| Dizziness | Nervous system disorders | MedDRA 28.0 | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA 28.0 | Systematic Assessment |
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| Dry eye | Eye disorders | MedDRA 28.0 | Systematic Assessment |
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| Middle ear inflammation | Ear and labyrinth disorders | MedDRA 28.0 | Systematic Assessment |
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| Sinus bradycardia | Cardiac disorders | MedDRA 28.0 | Systematic Assessment |
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| Tachycardia | Cardiac disorders | MedDRA 28.0 | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 28.0 | Systematic Assessment |
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| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 28.0 | Systematic Assessment |
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| Chronic gastritis | Gastrointestinal disorders | MedDRA 28.0 | Systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDRA 28.0 | Systematic Assessment |
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| Toothache | Gastrointestinal disorders | MedDRA 28.0 | Systematic Assessment |
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| Eczema | Skin and subcutaneous tissue disorders | MedDRA 28.0 | Systematic Assessment |
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| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 28.0 | Systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 28.0 | Systematic Assessment |
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| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 28.0 | Systematic Assessment |
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| Blood glucose increased | Investigations | MedDRA 28.0 | Systematic Assessment |
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| Electrocardiogram QT prolonged | Investigations | MedDRA 28.0 | Systematic Assessment |
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| Electrocardiogram ST-T segment abnormal | Investigations | MedDRA 28.0 | Systematic Assessment |
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| Weight decreased | Investigations | MedDRA 28.0 | Systematic Assessment |
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| Weight increased | Investigations | MedDRA 28.0 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Alexion Pharmaceuticals Inc. | Alexion Pharmaceuticals Inc. | +1 855-752-2356 | clinicaltrials@alexion.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jul 2, 2025 | May 11, 2026 | SAP_001.pdf |
| ID | Term |
|---|---|
| D006457 | Hemoglobinuria, Paroxysmal |
| ID | Term |
|---|---|
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D009190 | Myelodysplastic Syndromes |
| D001855 | Bone Marrow Diseases |
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| ID | Term |
|---|---|
| C000629409 | ravulizumab |
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| Unknown or Not Reported |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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