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| ID | Type | Description | Link |
|---|---|---|---|
| 2017-002820-26 | EudraCT Number |
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The purpose of this study was to assess the pharmacokinetics (PK), pharmacodynamics (PD), safety, and efficacy of ravulizumab in pediatric participants with paroxysmal nocturnal hemoglobinuria (PNH).
The study consists of a 4-week Screening Period, a 26-week Primary Evaluation Period, and an Extension Period of up to 4 years (with the exception of any country-specific mandates), whichever occurs first.
Efficacy and safety data are reported for the 26-week Primary Evaluation Period only. Analyses were conducted separately for complement inhibitor treatment-naïve participants and eculizumab-experienced participants.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Ravulizumab | Experimental | Complement inhibitor treatment-naïve and eculizumab-experienced participants received ravulizumab. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Ravulizumab | Biological | Single intravenous (IV) loading dose on Day 1, followed by regular IV maintenance dosing beginning on Day 15, based on weight. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Maximum Observed Serum Concentration (Cmax) Of Ravulizumab | Blood samples for determination of ravulizumab Cmax were collected before and after administration of study drug at designated time points. Results are reported in micrograms/milliliter (μg/mL). | Week 1 (Day 1), Week 2 (Day 15), Week 10 (Day 71), and Week 18 (Day 127) |
| Trough Serum Concentration (Ctrough) Of Ravulizumab | Blood samples for determination of ravulizumab Ctrough were collected before and after administration of study drug at designated time points. Trough serum concentration was measured at end of dosing interval at steady state. Results are reported in μg/mL. | Week 2 (Day 15), Week 10 (Day 71), Week 18 (Day 127), Week 26 (Day 183) |
| Mean Accumulation Ratio For Cmax Of Ravulizumab Following The Last Maintenance Dose Relative To The First Maintenance Dose | Blood samples for determination of ravulizumab accumulation ratio for Cmax were collected before and after administration of study drug at designated time points. The accumulation ratio was calculated as Cmax from the last maintenance dose (Week 18) divided by Cmax from the first maintenance dose (Week 2). | Week 18 |
| Mean Accumulation Ratio For Ctrough Of Ravulizumab Following The Last Maintenance Dose Relative To The First Maintenance Dose | Blood samples for determination of ravulizumab accumulation ratio for Ctrough were collected before and after administration of study drug at designated time points. The accumulation ratio was calculated as Ctrough from the last maintenance dose (Week 18) divided by Ctrough from the first maintenance dose (Week 2). | Week 18 |
| Change In Free Complement Component C5 (C5) Concentrations Over Time | Blood samples for determination of free C5 were collected before and after administration of study drug at designated time points. |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage Change From Baseline At Week 26 In Lactate Dehydrogenase (LDH) Levels | Blood and urine samples for determination of LDH levels were collected at designated time points. Baseline was defined as the average of all assessments analyzed by the central laboratory prior to first study drug administration. | Baseline, Week 26 |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Clinical Trial Site | Atlanta | Georgia | 30329 | United States | ||
| Clinical Trial Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| Result | Kulagin A, Chonat S, Maschan A, Bartels M, Buechner J, Punzalan R, Richards M, Ogawa M, Hicks E, Yu J, Baruchel A, Kulasekararaj AG. Pharmacokinetics, pharmacodynamics, efficacy, and safety of ravulizumab in children and adolescents with paroxysmal nocturnal hemoglobinuria: interim analysis of a phase 3, open-label study. Presented at the European Hematology Association 2021 Virtual Congress, June 9-17, 2021. | ||
| 38551806 | Derived | Chonat S, Kulagin A, Maschan A, Bartels M, Buechner J, Punzalan R, Richards M, Ogawa M, Hicks E, Yu J, Baruchel A, Kulasekararaj AG. Pharmacokinetics, pharmacodynamics, efficacy, and safety of ravulizumab in pediatric paroxysmal nocturnal hemoglobinuria. Blood Adv. 2024 Jun 11;8(11):2813-2824. doi: 10.1182/bloodadvances.2023012267. |
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Thirteen participants, from birth to < 18 years, were planned to be enrolled to ensure at least 10 evaluable participants would complete the 26-week Primary Evaluation Period. Participants were recruited from 9 sites across 6 countries (United States, United Kingdom, France, Netherlands, Russia, and Norway).
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| ID | Title | Description |
|---|---|---|
| FG000 | Treatment Naïve | Complement inhibitor treatment-naïve participants received weight-based doses of ravulizumab. |
| FG001 | Eculizumab Experienced | Eculizumab-experienced participants received weight-based doses of ravulizumab. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Primary Evaluation Period |
|
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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 22, 2020 | Feb 23, 2023 |
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|
| Baseline, Weeks 2, 10, 18, and 26 (end of infusion) |
| Change In Chicken Red Blood Cell (cRBC) Hemolytic Activity Over Time | Blood samples for determination of cRBC hemolytic activity were collected before and after administration of study drug at designated time points. | Baseline, Weeks 2, 10, 18, and 26 |
| Percentage Of Participants Who Achieved Transfusion Avoidance (TA) |
Transfusion avoidance was defined as the proportion of participants who remained transfusion-free and did not require a transfusion according to protocol-specified guidelines. Point estimates and 2-sided 95% exact confidence intervals (CIs) were computed. Participants who withdrew from the study due to lack of efficacy during the Primary Evaluation Period were considered as non-responders and were counted in the group needing transfusion. For participants who withdrew from the study for any other reason during the Primary Evaluation Period, their data up to the time of withdrawal was used to assess TA. |
| Week 26 |
| Change In Quality Of Life (QoL) From Baseline To Week 26 | Quality of life was measured by Pediatric Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue Questionnaire (participants ≥ 5 years of age), a 13-item questionnaire that assesses fatigue and its impact upon daily activities and function over the preceding 7 days. Each item is scored on a 5-point scale, and total scores range from 0 to 52, with a higher score indicating better QoL. The scoring guideline for the Pediatric FACIT-Fatigue instrument was used to calculate a FACIT-Fatigue score. Changes from baseline in FACIT-Fatigue scores were summarized at baseline and at the study visits where this assessment was collected up to Day 183 (Week 26). At each study visit, the proportion of participants who showed an improvement of at least 3 points for the Pediatric FACIT-Fatigue scores were summarized by point estimates and 2-sided 95% exact CIs. | Baseline, Week 26 |
| Percentage Of Participants With Stabilized Hemoglobin At Week 26 | Stabilized hemoglobin was defined as avoidance of a ≥ 2 g/dL decrease in hemoglobin level from baseline in the absence of transfusion through Week 26. Point estimates and 2-sided 95% exact CIs were computed. Participants who withdrew from the study due to lack of efficacy during the Primary Evaluation Period were considered as non-responders and were counted in the group who did not meet the stabilized hemoglobin definition. For participants who withdrew from the study for any other reason during the Primary Evaluation Period, their data up to the time of withdrawal were used to assess stabilized hemoglobin. | Week 26 |
| Percentage Change In Free Hemoglobin From Baseline To Week 26 | Percentage change from baseline in free hemoglobin was summarized at all study visits up to Day 183 (Week 26). Baseline was defined as the last non-missing assessment value prior to the first study drug infusion. | Baseline, Week 26 |
| Percentage Of Participants With Breakthrough Hemolysis (BTH) At Week 26 | Breakthrough hemolysis was defined as at least one new or worsening symptom or sign of intravascular hemolysis (fatigue, hemoglobinuria, abdominal pain, shortness of breath [dyspnea], anemia, major adverse vascular event [including thrombosis], dysphagia, or erectile dysfunction) in the presence of elevated LDH as follows: for participants who entered the study naïve to complement inhibitor treatment, elevated LDH ≥ 2 × the upper limit of normal (ULN) after prior LDH reduction to < 1.5 × ULN on therapy; for participants who entered the study stabilized on eculizumab treatment, elevated LDH ≥ 2 × ULN. Participants who withdrew from the study due to lack of efficacy during the Primary Evaluation Period were considered as non-responders and were counted in the group with BTH. For participants who withdrew from the study for any other reason during the Primary Evaluation Period, their data up to the time of withdrawal were used to assess BTH. No participants experienced BTH. | Week 26 |
| Milwaukee |
| Wisconsin |
| 53226 |
| United States |
| Clinical Trial Site | Paris | France |
| Clinical Trial Site | Utrecht | Netherlands |
| Clinical Trial Site | Oslo | Norway |
| Clinical Trial Site | Moscow | Russia |
| Clinical Trial Site | Saint Petersburg | Russia |
| Clinical Trial Site | Leeds | United Kingdom |
| Clinical Trial Site | London | United Kingdom |
| Received at Least 1 Dose of Study Drug |
|
| COMPLETED |
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| NOT COMPLETED |
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| Extension Period |
|
|
Full Analysis: all participants who received at least 1 dose of ravulizumab and had at least 1 efficacy assessment after the first infusion of ravulizumab.
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| ID | Title | Description |
|---|---|---|
| BG000 | Treatment Naïve | Complement inhibitor treatment-naïve participants received weight-based doses of ravulizumab. |
| BG001 | Eculizumab Experienced | Eculizumab-experienced participants received weight-based doses of ravulizumab. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Age at first infusion | Mean | Standard Deviation | years |
| ||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Race/Ethnicity, Customized | Specific information pertaining to race and ethnicity undisclosed by Sponsor to ensure participant privacy. | Count of Participants | Participants |
|
| 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 | Maximum Observed Serum Concentration (Cmax) Of Ravulizumab | Blood samples for determination of ravulizumab Cmax were collected before and after administration of study drug at designated time points. Results are reported in micrograms/milliliter (μg/mL). | Pharmacokinetic (PK): Participants enrolled into the study who received at least 1 dose of ravulizumab and who had evaluable interim PK data. The PK set was used for all PK analyses. | Posted | Mean | Standard Deviation | μg/mL | Week 1 (Day 1), Week 2 (Day 15), Week 10 (Day 71), and Week 18 (Day 127) |
|
|
| ||||||||||||||||||||||||||||
| Primary | Trough Serum Concentration (Ctrough) Of Ravulizumab | Blood samples for determination of ravulizumab Ctrough were collected before and after administration of study drug at designated time points. Trough serum concentration was measured at end of dosing interval at steady state. Results are reported in μg/mL. | Pharmacokinetic (PK): participants enrolled into the study who received at least 1 dose of ravulizumab and who had evaluable interim PK data. The PK set was used for all PK analyses. | Posted | Mean | Standard Deviation | μg/mL | Week 2 (Day 15), Week 10 (Day 71), Week 18 (Day 127), Week 26 (Day 183) |
|
| |||||||||||||||||||||||||||||
| Primary | Mean Accumulation Ratio For Cmax Of Ravulizumab Following The Last Maintenance Dose Relative To The First Maintenance Dose | Blood samples for determination of ravulizumab accumulation ratio for Cmax were collected before and after administration of study drug at designated time points. The accumulation ratio was calculated as Cmax from the last maintenance dose (Week 18) divided by Cmax from the first maintenance dose (Week 2). | Pharmacokinetic (PK): participants enrolled into the study who received at least 1 dose of ravulizumab and who had evaluable interim PK data. The PK set was used for all PK analyses. | Posted | Mean | Standard Deviation | Ratio | Week 18 |
|
| |||||||||||||||||||||||||||||
| Primary | Mean Accumulation Ratio For Ctrough Of Ravulizumab Following The Last Maintenance Dose Relative To The First Maintenance Dose | Blood samples for determination of ravulizumab accumulation ratio for Ctrough were collected before and after administration of study drug at designated time points. The accumulation ratio was calculated as Ctrough from the last maintenance dose (Week 18) divided by Ctrough from the first maintenance dose (Week 2). | Pharmacokinetic (PK): participants enrolled into the study who received at least 1 dose of ravulizumab and who had evaluable interim PK data. The PK set was used for all PK analyses. | Posted | Mean | Standard Deviation | Ratio | Week 18 |
|
| |||||||||||||||||||||||||||||
| Primary | Change In Free Complement Component C5 (C5) Concentrations Over Time | Blood samples for determination of free C5 were collected before and after administration of study drug at designated time points. | Pharmacodynamic: all participants who received at least 1 dose of ravulizumab and who had evaluable pharmacodynamic data. | Posted | Mean | Standard Deviation | ug/mL | Baseline, Weeks 2, 10, 18, and 26 (end of infusion) |
|
| |||||||||||||||||||||||||||||
| Primary | Change In Chicken Red Blood Cell (cRBC) Hemolytic Activity Over Time | Blood samples for determination of cRBC hemolytic activity were collected before and after administration of study drug at designated time points. | Pharmacodynamic: all participants who received at least 1 dose of ravulizumab and who had evaluable pharmacodynamic data. | Posted | Mean | Standard Deviation | percentage of hemolysis | Baseline, Weeks 2, 10, 18, and 26 |
|
| |||||||||||||||||||||||||||||
| Secondary | Percentage Change From Baseline At Week 26 In Lactate Dehydrogenase (LDH) Levels | Blood and urine samples for determination of LDH levels were collected at designated time points. Baseline was defined as the average of all assessments analyzed by the central laboratory prior to first study drug administration. | Full Analysis: all participants who received at least 1 dose of ravulizumab and had at least 1 efficacy assessment after the first infusion of ravulizumab. | Posted | Mean | Standard Deviation | Percent Change | Baseline, Week 26 |
|
| |||||||||||||||||||||||||||||
| Secondary | Percentage Of Participants Who Achieved Transfusion Avoidance (TA) | Transfusion avoidance was defined as the proportion of participants who remained transfusion-free and did not require a transfusion according to protocol-specified guidelines. Point estimates and 2-sided 95% exact confidence intervals (CIs) were computed. Participants who withdrew from the study due to lack of efficacy during the Primary Evaluation Period were considered as non-responders and were counted in the group needing transfusion. For participants who withdrew from the study for any other reason during the Primary Evaluation Period, their data up to the time of withdrawal was used to assess TA. | Full Analysis: all participants who received at least 1 dose of ravulizumab and had at least 1 efficacy assessment after the first infusion of ravulizumab. | Posted | Number | 95% Confidence Interval | Percentage of Participants | Week 26 |
|
| |||||||||||||||||||||||||||||
| Secondary | Change In Quality Of Life (QoL) From Baseline To Week 26 | Quality of life was measured by Pediatric Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue Questionnaire (participants ≥ 5 years of age), a 13-item questionnaire that assesses fatigue and its impact upon daily activities and function over the preceding 7 days. Each item is scored on a 5-point scale, and total scores range from 0 to 52, with a higher score indicating better QoL. The scoring guideline for the Pediatric FACIT-Fatigue instrument was used to calculate a FACIT-Fatigue score. Changes from baseline in FACIT-Fatigue scores were summarized at baseline and at the study visits where this assessment was collected up to Day 183 (Week 26). At each study visit, the proportion of participants who showed an improvement of at least 3 points for the Pediatric FACIT-Fatigue scores were summarized by point estimates and 2-sided 95% exact CIs. | Full Analysis: all participants who received at least 1 dose of ravulizumab and had at least 1 efficacy assessment after the first infusion of ravulizumab. | Posted | Mean | Standard Deviation | units on a scale | Baseline, Week 26 |
| ||||||||||||||||||||||||||||||
| Secondary | Percentage Of Participants With Stabilized Hemoglobin At Week 26 | Stabilized hemoglobin was defined as avoidance of a ≥ 2 g/dL decrease in hemoglobin level from baseline in the absence of transfusion through Week 26. Point estimates and 2-sided 95% exact CIs were computed. Participants who withdrew from the study due to lack of efficacy during the Primary Evaluation Period were considered as non-responders and were counted in the group who did not meet the stabilized hemoglobin definition. For participants who withdrew from the study for any other reason during the Primary Evaluation Period, their data up to the time of withdrawal were used to assess stabilized hemoglobin. | Full Analysis: all participants who received at least 1 dose of ravulizumab and had at least 1 efficacy assessment after the first infusion of ravulizumab. | Posted | Number | 95% Confidence Interval | Percentage of Participants | Week 26 |
|
| |||||||||||||||||||||||||||||
| Secondary | Percentage Change In Free Hemoglobin From Baseline To Week 26 | Percentage change from baseline in free hemoglobin was summarized at all study visits up to Day 183 (Week 26). Baseline was defined as the last non-missing assessment value prior to the first study drug infusion. | Full Analysis: all participants who received at least 1 dose of ravulizumab and had at least 1 efficacy assessment after the first infusion of ravulizumab. | Posted | Mean | Standard Deviation | Percentage Change | Baseline, Week 26 |
|
| |||||||||||||||||||||||||||||
| Secondary | Percentage Of Participants With Breakthrough Hemolysis (BTH) At Week 26 | Breakthrough hemolysis was defined as at least one new or worsening symptom or sign of intravascular hemolysis (fatigue, hemoglobinuria, abdominal pain, shortness of breath [dyspnea], anemia, major adverse vascular event [including thrombosis], dysphagia, or erectile dysfunction) in the presence of elevated LDH as follows: for participants who entered the study naïve to complement inhibitor treatment, elevated LDH ≥ 2 × the upper limit of normal (ULN) after prior LDH reduction to < 1.5 × ULN on therapy; for participants who entered the study stabilized on eculizumab treatment, elevated LDH ≥ 2 × ULN. Participants who withdrew from the study due to lack of efficacy during the Primary Evaluation Period were considered as non-responders and were counted in the group with BTH. For participants who withdrew from the study for any other reason during the Primary Evaluation Period, their data up to the time of withdrawal were used to assess BTH. No participants experienced BTH. | Full Analysis: all participants who received at least 1 dose of ravulizumab and had at least 1 efficacy assessment after the first infusion of ravulizumab. | Posted | Count of Participants | Participants | Week 26 |
|
Day 1 through End of Study (Up to Day 1610)
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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 | Treatment Naïve | Complement inhibitor treatment-naïve participants received weight-based doses of ravulizumab. | 0 | 5 | 2 | 5 | 4 | 5 |
| EG001 | Eculizumab Experienced | Eculizumab-experienced participants received weight-based doses of ravulizumab. | 0 | 8 | 2 | 8 | 7 | 8 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Device related thrombosis | General disorders | MedDRA 24.0 | Systematic Assessment |
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| Multiple organ dysfunction syndrome | General disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Device related sepsis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Septic shock | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Staphylococcal infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Aplastic anaemia | Blood and lymphatic system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Breakthrough haemolysis | Blood and lymphatic system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Ear pain | Ear and labyrinth disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Cushing's syndrome | Endocrine disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Dysphagia | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Serum sickness | Immune system disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Cystitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Hordeolum | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Paronychia | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Electric shock | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Sunburn | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Wound | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Blood pressure increased | Investigations | MedDRA 25.1 | Systematic Assessment |
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| Decreased appetite | Metabolism and nutrition disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Rhinitis allergic | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Anaemia | Blood and lymphatic system disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Rectal hemorrhage | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Hyperbilirubinemia | Hepatobiliary disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Hematoma | Vascular disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
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| Ear infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Infected bite | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
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| Respiratory tract infection viral | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Tooth fracture | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Toxicity to various agents | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Transfusion reaction | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Blood bilirubin increased | Investigations | MedDRA 25.1 | Systematic Assessment |
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| Blood creatinine increased | Investigations | MedDRA 25.1 | Systematic Assessment |
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| Iron deficiency | Metabolism and nutrition disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Arthritis | Musculoskeletal and connective tissue disorders | MedDRA 25.1 | Systematic Assessment |
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| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 25.1 | Systematic Assessment |
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| Juvenile idiopathic arthritis | Musculoskeletal and connective tissue disorders | MedDRA 25.1 | Systematic Assessment |
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| Kyphosis | Musculoskeletal and connective tissue disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
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| Acne | Skin and subcutaneous tissue disorders | MedDRA 25.1 | Systematic Assessment |
| |
| In growing nail | Skin and subcutaneous tissue disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Aplastic anaemia | Blood and lymphatic system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Lymphadenitis | Blood and lymphatic system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Administration site pain | General disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Cholelithiasis | Hepatobiliary disorders | MedDRA 25.1 | Systematic Assessment |
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| Pallor | Vascular disorders | MedDRA 25.1 | Systematic Assessment |
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| Dysmenorrhoea | Reproductive system and breast disorders | MedDRA 25.1 | Systematic Assessment | The N for this adverse event has been adjusted to the number of females in the study as it is a sex-specific event. |
|
| Asthenia | General disorders | MedDRA 25.1 | Systematic Assessment |
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| Catheter site pain | General disorders | MedDRA 25.1 | Systematic Assessment |
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| Chest pain | General disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Non-cardiac chest pain | General disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Chromosomal deletion | Congenital, familial and genetic disorders | MedDRA 25.1 | Systematic Assessment |
|
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Alexion Pharmaceuticals, Inc. | Alexion Pharmaceuticals, Inc. | 1.855.752.2356 | clinicaltrials@alexion.com |
| Prot_002.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | May 8, 2020 | Sep 15, 2021 | 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 |
| D009190 | Myelodysplastic Syndromes |
| D001855 | Bone Marrow Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C000629409 | ravulizumab |
Not provided
Not provided
Not provided
| Male |
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| Ethnicity - Undisclosed |
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| Day 15: End of Infusion |
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| Day 71: End of Infusion |
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| Day 127: End of Infusion |
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| Participants |
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| Participants |
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