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| ID | Type | Description | Link |
|---|---|---|---|
| 2020-000761-16 | EudraCT Number |
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This study will evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of ravulizumab administered by intravenous infusion to pediatric participants, from 1 month to < 18 years of age, with HSCT-TMA. The treatment period is 26 weeks, followed by a 26-week off-treatment follow-up period.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Ravulizumab plus Best Supportive Care | Experimental | Participants will receive ravulizumab plus Best Supportive Care as background therapy. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Ravulizumab | Drug | Weight-based doses of ravulizumab will be administered intravenously as a loading dose regimen followed by maintenance dosing every 4 or 8 weeks, depending upon weight. |
| Measure | Description | Time Frame |
|---|---|---|
| Participants With Thrombotic Microangiopathy (TMA) Response | The criteria for TMA response were: 1. Normalization of platelet count (defined as platelet count ≥ 50000 mm^3 or >=50% increase in platelet count) without transfusion support during the prior 7 days. 2. Normalization of lactate dehydrogenase (LDH, defined as LDH ≤ upper limit of normal [ULN]) and absence of schistocytes. 3. At least 50% reduction in protein/creatinine ratio from baseline. Participants must meet each TMA criterion at 2 separate assessments obtained at least 24 hours apart, with no criteria failures or more than 1 missed scheduled visit in between. Additionally, all intervals in which the criteria were met must overlap for at least 1 day. | Up to Week 26 |
| Measure | Description | Time Frame |
|---|---|---|
| Time to TMA Response During the 26-Week Treatment Period | Time to TMA response was defined as the time from first infusion to the first time point at which all criteria for TMA response was met. Participants were assigned as responders at the time of their TMA response and were censored at the earlier of last assessment with all 3 TMA response components available (including measurements collected after treatment discontinuation), or death if they did not respond by then. TMA response required the following: 1. Normalization of platelet count (defined as platelet count ≥ 50000 mm^3 or >=50% increase in platelet count) without transfusion support during the prior 7 days. 2. Normalization of LDH (defined as LDH ≤ULN) and absence of schistocytes. 3. At least 50% reduction in protein/creatinine ratio from baseline. |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Research Site | Tucson | Arizona | 85724 | United States | ||
| Research Site |
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| Label | URL |
|---|---|
| Related Info | View source |
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| ID | Title | Description |
|---|---|---|
| FG000 | Ravulizumab | Participants received weight-based dosages of ravulizumab intravenously for 26 weeks during the Treatment Period. Participants received a loading dose of ravulizumab intravenously on Days 1, 5 and 10 followed by maintenance dosing on Day 15 and once every 8 weeks or every 4 weeks thereafter depending upon their body weight. After the completion of the treatment period, participants were followed for 26 weeks in Follow-up period (no study drug was administered during this period). |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Treatment Period (26 Weeks) |
|
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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 | Sep 2, 2024 | Nov 25, 2025 |
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| Best Supportive Care | Other | Participants will receive medications, therapies, and interventions per standard hospital treatment protocols (unless specifically prohibited by the protocol). |
|
| Day 1 through Week 26 |
| Participants With Hematologic Response | Hematologic response required the following: (1) Normalization of platelet count (defined as platelet count ≥ 50000 mm^3 or >=50% increase in platelet count) without transfusion support during the prior 7 days and (2) Normalization of LDH (defined as LDH ≤ULN) and absence of schistocytes. | Up to Week 26 |
| Time to Hematologic Response During the 26-Week Treatment Period | Time to Hematologic response was defined as the time from first infusion to the first time point at which all criteria for hematologic response was met. Participants were assigned as responders at the time of their response and were censored at their discontinuation time or at the end of available follow-up if they did not respond by then. Hematologic response required the following: (1) Normalization of platelet count (defined as platelet count ≥ 50000 mm^3 or >=50% increase in platelet count) without transfusion support during the prior 7 days and (2) Normalization of LDH (defined as LDH ≤ULN) and absence of schistocytes. | Day 1 through Week 26 |
| Participants With Hemoglobin Response | Hemoglobin response was defined as the ability to maintain hemoglobin ≥ 10 g/dL without RBC transfusion support. The criterion must have been met at 2 separate assessments obtained at least 24 hours apart, and any measurement in between, and without RBC transfusion support during the prior 7 days. | Up to Week 26 |
| Participants With Platelet Response | Normalization of platelet count was defined as baseline platelet count ≤ 50000 mm^3 or > 50000 mm^3, absolute platelet count > 50000 mm^3 or >=50% increase in platelet count without transfusion support during the prior 7 days. | Up to Week 26 |
| Participants With Partial TMA Response | Partial response was defined as a participant meeting at least 1, but not all, criteria for TMA response. TMA response required the following: 1. Normalization of platelet count (defined as platelet count ≥ 50000 mm^3 or >=50% increase in platelet count) without transfusion support during the prior 7 days. 2. Normalization of LDH, (defined as LDH ≤ULN) and absence of schistocytes. 3. At least 50% reduction in protein/creatinine ratio from baseline. | Up to Week 26 |
| Participants With Loss of TMA Response | Loss of response occurred when a participant who had previously achieved a TMA response failed to meet criteria for one or more components of TMA response at a subsequent visit in treatment period. At least one parameter must fail to meet response criteria at 2 separate assessments obtained at least 24 hours apart, and any measurement in between. TMA response required the following: 1. Normalization of platelet count (defined as platelet count ≥ 50000 mm^3 or >=50% increase in platelet count) without transfusion support during the prior 7 days. 2. Normalization of LDH (defined as LDH ≤ULN) and absence of schistocytes. 3. At least 50% reduction in protein/creatinine ratio from baseline. | Up to Week 26 |
| Duration of TMA Response Through Week 52 | This analysis includes data for each participant after TMA response through final follow-up. Participants with TMA response who do not experience these events are censored at their end of study date. TMA response required following: 1. Normalization of platelet count (defined as platelet count ≥ 50000 mm^3 or >=50% increase in platelet count) without transfusion support during the prior 7 days. 2. Normalization of LDH (defined as LDH ≤ULN) and absence of schistocytes. 3. At least 50% reduction in protein/creatinine ratio from baseline. The estimate is calculated based on Kaplan-Meier method. | Day 1 through Week 52 |
| Participants With TMA Relapse | For participants that meet criteria for TMA response during 26-week Treatment Period, TMA relapse is defined as evidence of worsening hematologic and renal dysfunction due to TMA during post-treatment Follow-up Period that requires treatment intervention, as determined by Investigator. TMA response required the following: 1. Normalization of platelet count (defined as platelet count ≥ 50000 mm^3 or >=50% increase in platelet count) without transfusion support during the prior 7 days. 2. Normalization of LDH (defined as LDH ≤ULN) and absence of schistocytes. 3. At least 50% reduction in protein/creatinine ratio from baseline. | Up to Week 52 |
| Overall Survival | The Kaplan-Meier method was used to measure overall survival estimate. Overall survival was calculated from date of treatment start to date of a documented death event (death due to any cause) or date of censoring. Participants who survived were censored at the earliest of an additional hematopoietic stem cell transplant (HSCT), Week 52, or their last known date alive. | Day 1 through Week 52 |
| Non-relapse Mortality During the 52-Week Treatment Period | Cumulative incidence was estimated using a competing risk model. Non-relapse mortality was defined as a participant's death due to any cause during the study, with the exception of death due to underlying disease progression or relapse. | Day 1 through Week 52 |
| Aurora |
| Colorado |
| 80045 |
| United States |
| Research Site | Atlanta | Georgia | 30322 | United States |
| Research Site | Chicago | Illinois | 60611 | United States |
| Research Site | Portland | Oregon | 97239 | United States |
| Research Site | Dallas | Texas | 75235 | United States |
| Research Site | Salt Lake City | Utah | 84108 | United States |
| Research Site | Madison | Wisconsin | 53792 | United States |
| Research Site | Haifa | 91096 | Israel |
| Research Site | Jerusalem | 91120 | Israel |
| Research Site | Petah Tikva | 4920235 | Israel |
| Research Site | Ramat Gan | 5265601 | Israel |
| Research Site | Roma | 00165 | Italy |
| Research Site | Fukushima | 960-1295 | Japan |
| Research Site | Kobe | 650-0047 | Japan |
| Research Site | Nagoya | 466-8560 | Japan |
| Research Site | Osaka | 534-0021 | Japan |
| Research Site | Seoul | 03080 | South Korea |
| Research Site | Seoul | 5505 | South Korea |
| Research Site | Barcelona | 08041 | Spain |
| Research Site | Esplugues de Llobregat | 8950 | Spain |
| Research Site | Madrid | 28046 | Spain |
| Research Site | Salamanca | 37007 | Spain |
| Research Site | Birmingham | B4 6NH | United Kingdom |
| Research Site | Bristol | BS2 8BJ | United Kingdom |
| Research Site | Newcastle upon Tyne | NE1 4LP | United Kingdom |
| Received at Least 1 Dose of Study Drug |
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| COMPLETED |
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| NOT COMPLETED |
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|
| Follow-Up Period (26 Weeks) |
|
|
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
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| ID | Title | Description |
|---|---|---|
| BG000 | Ravulizumab | Participants received weight-based dosages of ravulizumab intravenously for 26 weeks during the Treatment Period. Participants received a loading dose of ravulizumab intravenously on Days 1, 5 and 10 followed by maintenance dosing on Day 15 and once every 8 weeks or every 4 weeks thereafter depending upon their body weight. After the completion of the treatment period, participants were followed for 26 weeks in Follow-up period (no study drug was administered during this 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 |
| |||||||||||||||||
| 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 | Participants With Thrombotic Microangiopathy (TMA) Response | The criteria for TMA response were: 1. Normalization of platelet count (defined as platelet count ≥ 50000 mm^3 or >=50% increase in platelet count) without transfusion support during the prior 7 days. 2. Normalization of lactate dehydrogenase (LDH, defined as LDH ≤ upper limit of normal [ULN]) and absence of schistocytes. 3. At least 50% reduction in protein/creatinine ratio from baseline. Participants must meet each TMA criterion at 2 separate assessments obtained at least 24 hours apart, with no criteria failures or more than 1 missed scheduled visit in between. Additionally, all intervals in which the criteria were met must overlap for at least 1 day. | Full analysis set included all participants who signed the informed consent and received at least 1 dose of ravulizumab, excluding participants who enrolled prior to availability of shiga toxin-related hemolytic uremic syndrome (ST-HUS) and a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13 (ADAMTS13) laboratory results and are subsequently found to be ineligible after enrollment. | Posted | Count of Participants | Participants | Up to Week 26 |
|
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| Secondary | Time to TMA Response During the 26-Week Treatment Period | Time to TMA response was defined as the time from first infusion to the first time point at which all criteria for TMA response was met. Participants were assigned as responders at the time of their TMA response and were censored at the earlier of last assessment with all 3 TMA response components available (including measurements collected after treatment discontinuation), or death if they did not respond by then. TMA response required the following: 1. Normalization of platelet count (defined as platelet count ≥ 50000 mm^3 or >=50% increase in platelet count) without transfusion support during the prior 7 days. 2. Normalization of LDH (defined as LDH ≤ULN) and absence of schistocytes. 3. At least 50% reduction in protein/creatinine ratio from baseline. | Full analysis set included all participants who signed the informed consent and received at least 1 dose of ravulizumab, excluding participants who enrolled prior to availability of ST-HUS and ADAMTS13 laboratory results and are subsequently found to be ineligible after enrollment. | Posted | Median | 95% Confidence Interval | days | Day 1 through Week 26 |
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| Secondary | Participants With Hematologic Response | Hematologic response required the following: (1) Normalization of platelet count (defined as platelet count ≥ 50000 mm^3 or >=50% increase in platelet count) without transfusion support during the prior 7 days and (2) Normalization of LDH (defined as LDH ≤ULN) and absence of schistocytes. | Full analysis set included all participants who signed the informed consent and received at least 1 dose of ravulizumab, excluding participants who enrolled prior to availability of ST-HUS and ADAMTS13 laboratory results and are subsequently found to be ineligible after enrollment. | Posted | Count of Participants | Participants | Up to Week 26 |
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| |||||||||||||||||||||||||||
| Secondary | Time to Hematologic Response During the 26-Week Treatment Period | Time to Hematologic response was defined as the time from first infusion to the first time point at which all criteria for hematologic response was met. Participants were assigned as responders at the time of their response and were censored at their discontinuation time or at the end of available follow-up if they did not respond by then. Hematologic response required the following: (1) Normalization of platelet count (defined as platelet count ≥ 50000 mm^3 or >=50% increase in platelet count) without transfusion support during the prior 7 days and (2) Normalization of LDH (defined as LDH ≤ULN) and absence of schistocytes. | Full analysis set included all participants who signed the informed consent and received at least 1 dose of ravulizumab, excluding participants who enrolled prior to availability of ST-HUS and ADAMTS13 laboratory results and are subsequently found to be ineligible after enrollment. Here, overall number of participants analyzed signifies those participants who were evaluable for this outcome measure. | Posted | Median | 95% Confidence Interval | days | Day 1 through Week 26 |
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| Secondary | Participants With Hemoglobin Response | Hemoglobin response was defined as the ability to maintain hemoglobin ≥ 10 g/dL without RBC transfusion support. The criterion must have been met at 2 separate assessments obtained at least 24 hours apart, and any measurement in between, and without RBC transfusion support during the prior 7 days. | Full analysis set included all participants who signed the informed consent and received at least 1 dose of ravulizumab, excluding participants who enrolled prior to availability of ST-HUS and ADAMTS13 laboratory results and are subsequently found to be ineligible after enrollment. | Posted | Count of Participants | Participants | Up to Week 26 |
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| Secondary | Participants With Platelet Response | Normalization of platelet count was defined as baseline platelet count ≤ 50000 mm^3 or > 50000 mm^3, absolute platelet count > 50000 mm^3 or >=50% increase in platelet count without transfusion support during the prior 7 days. | Full analysis set included all participants who signed the informed consent and received at least 1 dose of ravulizumab, excluding participants who enrolled prior to availability of ST-HUS and ADAMTS13 laboratory results and are subsequently found to be ineligible after enrollment. | Posted | Count of Participants | Participants | Up to Week 26 |
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| Secondary | Participants With Partial TMA Response | Partial response was defined as a participant meeting at least 1, but not all, criteria for TMA response. TMA response required the following: 1. Normalization of platelet count (defined as platelet count ≥ 50000 mm^3 or >=50% increase in platelet count) without transfusion support during the prior 7 days. 2. Normalization of LDH, (defined as LDH ≤ULN) and absence of schistocytes. 3. At least 50% reduction in protein/creatinine ratio from baseline. | Full analysis set included all participants who signed the informed consent and received at least 1 dose of ravulizumab, excluding participants who enrolled prior to availability of ST-HUS and ADAMTS13 laboratory results and are subsequently found to be ineligible after enrollment. | Posted | Count of Participants | Participants | Up to Week 26 |
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| Secondary | Participants With Loss of TMA Response | Loss of response occurred when a participant who had previously achieved a TMA response failed to meet criteria for one or more components of TMA response at a subsequent visit in treatment period. At least one parameter must fail to meet response criteria at 2 separate assessments obtained at least 24 hours apart, and any measurement in between. TMA response required the following: 1. Normalization of platelet count (defined as platelet count ≥ 50000 mm^3 or >=50% increase in platelet count) without transfusion support during the prior 7 days. 2. Normalization of LDH (defined as LDH ≤ULN) and absence of schistocytes. 3. At least 50% reduction in protein/creatinine ratio from baseline. | Full analysis set included all participants who signed the informed consent and received at least 1 dose of ravulizumab, excluding participants who enrolled prior to availability of ST-HUS and ADAMTS13 laboratory results and are subsequently found to be ineligible after enrollment. Here, overall number of participants analyzed signifies those participants who were evaluable for this outcome measure. | Posted | Count of Participants | Participants | Up to Week 26 |
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| Secondary | Duration of TMA Response Through Week 52 | This analysis includes data for each participant after TMA response through final follow-up. Participants with TMA response who do not experience these events are censored at their end of study date. TMA response required following: 1. Normalization of platelet count (defined as platelet count ≥ 50000 mm^3 or >=50% increase in platelet count) without transfusion support during the prior 7 days. 2. Normalization of LDH (defined as LDH ≤ULN) and absence of schistocytes. 3. At least 50% reduction in protein/creatinine ratio from baseline. The estimate is calculated based on Kaplan-Meier method. | Full analysis set included all participants who signed the informed consent and received at least 1 dose of ravulizumab, excluding participants who enrolled prior to availability of ST-HUS and ADAMTS13 laboratory results and are subsequently found to be ineligible after enrollment. Here, overall number of participants analyzed signifies those participants who were evaluable for this outcome measure. | Posted | Median | 95% Confidence Interval | days | Day 1 through Week 52 |
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| Secondary | Participants With TMA Relapse | For participants that meet criteria for TMA response during 26-week Treatment Period, TMA relapse is defined as evidence of worsening hematologic and renal dysfunction due to TMA during post-treatment Follow-up Period that requires treatment intervention, as determined by Investigator. TMA response required the following: 1. Normalization of platelet count (defined as platelet count ≥ 50000 mm^3 or >=50% increase in platelet count) without transfusion support during the prior 7 days. 2. Normalization of LDH (defined as LDH ≤ULN) and absence of schistocytes. 3. At least 50% reduction in protein/creatinine ratio from baseline. | Full analysis set included all participants who signed the informed consent and received at least 1 dose of ravulizumab, excluding participants who enrolled prior to availability of ST-HUS and ADAMTS13 laboratory results and are subsequently found to be ineligible after enrollment. Here, overall number of participants analyzed signifies those participants who were evaluable for this outcome measure. | Posted | Count of Participants | Participants | Up to Week 52 |
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| Secondary | Overall Survival | The Kaplan-Meier method was used to measure overall survival estimate. Overall survival was calculated from date of treatment start to date of a documented death event (death due to any cause) or date of censoring. Participants who survived were censored at the earliest of an additional hematopoietic stem cell transplant (HSCT), Week 52, or their last known date alive. | Full analysis set included all participants who signed the informed consent and received at least 1 dose of ravulizumab, excluding participants who enrolled prior to availability of ST-HUS and ADAMTS13 laboratory results and are subsequently found to be ineligible after enrollment. | Posted | Number | 95% Confidence Interval | proportion of participants | Day 1 through Week 52 |
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| Secondary | Non-relapse Mortality During the 52-Week Treatment Period | Cumulative incidence was estimated using a competing risk model. Non-relapse mortality was defined as a participant's death due to any cause during the study, with the exception of death due to underlying disease progression or relapse. | Full analysis set included all participants who signed the informed consent and received at least 1 dose of ravulizumab, excluding participants who enrolled prior to availability of ST-HUS and ADAMTS13 laboratory results and are subsequently found to be ineligible after enrollment. | Posted | Number | 95% Confidence Interval | proportion of participants | Day 1 through Week 52 |
|
|
Day 1 up to Week 52
Safety set included all participants who signed the informed consent and received at least 1 dose of ravulizumab.
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 | Treatment Period: Ravulizumab | Participants received weight-based dosages of ravulizumab intravenously for 26 weeks during the Treatment Period. Participants received a loading dose of ravulizumab intravenously on Days 1, 5 and 10 followed by maintenance dosing on Day 15 and once every 8 weeks or every 4 weeks thereafter depending upon their body weight. | 6 | 41 | 30 | 41 | 41 | 41 |
| EG001 | Follow-up Period: Ravulizumab | After the completion of the treatment period, participants were followed for 26 Weeks in Follow-up period (no study drug was administered during this period). | 4 | 29 | 15 | 29 | 23 | 29 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Febrile neutropenia | Blood and lymphatic system disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Pancytopenia | Blood and lymphatic system disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Thrombotic microangiopathy | Blood and lymphatic system disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Pericardial effusion | Cardiac disorders | MedDRA 28.0 | Systematic Assessment |
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| Arrhythmia | Cardiac disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Cardiac arrest | Cardiac disorders | MedDRA 28.0 | Systematic Assessment |
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| Cardiac failure | Cardiac disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Cardiac tamponade | Cardiac disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Phimosis | Congenital, familial and genetic disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Mydriasis | Eye disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Uveitis | Eye disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Colitis | Gastrointestinal disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Gastric haemorrhage | Gastrointestinal disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Pneumatosis intestinalis | Gastrointestinal disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Rectal haemorrhage | Gastrointestinal disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Multiple organ dysfunction syndrome | General disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Venoocclusive liver disease | Hepatobiliary disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Graft versus host disease | Immune system disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Graft versus host disease in gastrointestinal tract | Immune system disorders | MedDRA 28.0 | Systematic Assessment |
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| COVID-19 | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
| |
| Gastroenteritis clostridial | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
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| Septic shock | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
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| Acinetobacter sepsis | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
| |
| Adenovirus infection | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
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| Appendicitis | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
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| Bacteraemia | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
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| Device related infection | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
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| Gastroenteritis Escherichia coli | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
| |
| Gastroenteritis rotavirus | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
| |
| Gastroenteritis salmonella | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
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| Herpes zoster | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
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| Infection | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
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| Klebsiella infection | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
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| Mucormycosis | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
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| Norovirus infection | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
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| Pneumonia | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
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| Pseudomonal bacteraemia | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
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| Sepsis | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
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| Stenotrophomonas infection | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
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| Vaginal abscess | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
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| Dehydration | Metabolism and nutrition disorders | MedDRA 28.0 | Systematic Assessment |
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| Hyperkalaemia | 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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| Metabolic acidosis | Metabolism and nutrition disorders | MedDRA 28.0 | Systematic Assessment |
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| Soft tissue necrosis | Musculoskeletal and connective tissue disorders | MedDRA 28.0 | Systematic Assessment |
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| Seizure | Nervous system disorders | MedDRA 28.0 | Systematic Assessment |
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| Depressed level of consciousness | Nervous system 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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| Loss of consciousness | Nervous system disorders | MedDRA 28.0 | Systematic Assessment |
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| Neurological symptom | Nervous system disorders | MedDRA 28.0 | Systematic Assessment |
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| Partial seizures | Nervous system disorders | MedDRA 28.0 | Systematic Assessment |
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| Syncope | Nervous system disorders | MedDRA 28.0 | Systematic Assessment |
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| Acute kidney injury | Renal and urinary disorders | MedDRA 28.0 | Systematic Assessment |
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| Renal failure | Renal and urinary disorders | MedDRA 28.0 | Systematic Assessment |
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| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Atelectasis | Respiratory, thoracic and mediastinal disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Interstitial lung disease | Respiratory, thoracic and mediastinal disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Respiratory distress | Respiratory, thoracic and mediastinal disorders | MedDRA 28.0 | Systematic Assessment |
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| Stem cell transplant | Surgical and medical procedures | MedDRA 28.0 | Systematic Assessment |
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| Microangiopathy | Vascular disorders | MedDRA 28.0 | Systematic Assessment |
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| Haematemesis | Gastrointestinal disorders | MedDRA 28.0 | Systematic Assessment |
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| Haemoperitoneum | Gastrointestinal disorders | MedDRA 28.0 | Systematic Assessment |
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| Device related bacteraemia | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
| |
| Bacterial sepsis | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
| |
| Device related sepsis | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
| |
| Gonococcal infection | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
| |
| Pseudomonas infection | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
| |
| Staphylococcal infection | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
| |
| Streptococcal sepsis | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA 28.0 | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Hypoglycaemia | Metabolism and nutrition disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Neuroblastoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 28.0 | Systematic Assessment |
| |
| Cerebral haemorrhage | Nervous system disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Superficial vein thrombosis | Vascular disorders | MedDRA 28.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Neutropenia | Blood and lymphatic system disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Pericardial effusion | Cardiac disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Bradycardia | Cardiac disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Hypothyroidism | Endocrine disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Adrenal insufficiency | Endocrine disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Ocular hypertension | Eye disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Pancreatitis | Gastrointestinal disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Pain | General disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Graft versus host disease in skin | Immune system disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Hypogammaglobulinaemia | Immune system disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Clostridium difficile infection | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
| |
| Staphylococcal infection | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
| |
| Respiratory syncytial virus infection | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
| |
| Rhinovirus infection | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
| |
| Candida infection | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 28.0 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA 28.0 | Systematic Assessment |
| |
| Weight decreased | Investigations | MedDRA 28.0 | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA 28.0 | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Hyperkalaemia | Metabolism and nutrition disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Hypophosphataemia | Metabolism and nutrition disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Hyperglycaemia | Metabolism and nutrition disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Dysuria | Renal and urinary disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Proteinuria | Renal and urinary disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Dry skin | Skin and subcutaneous tissue disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Erythema | Skin and subcutaneous tissue disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA 28.0 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA 28.0 | Systematic Assessment |
|
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Alexion Pharmaceuticals, Inc. | Alexion Pharmaceuticals, Inc. | 855-752-2356 | clinicaltrials@alexion.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Oct 9, 2024 | Nov 25, 2025 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D057049 | Thrombotic Microangiopathies |
| ID | Term |
|---|---|
| D013921 | Thrombocytopenia |
| D001791 | Blood Platelet Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D000095542 | Cytopenia |
Not provided
Not provided
| ID | Term |
|---|---|
| C000629409 | ravulizumab |
Not provided
Not provided
Not provided
| Unknown or Not Reported |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
|
|
|
|
|
|
|
| Participants |
|
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
|
|
|
|