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| ID | Type | Description | Link |
|---|---|---|---|
| 2016-002027-29 | EudraCT Number |
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The purpose of the study is to assess the safety and efficacy of ravulizumab to control disease activity in adolescent and adult participants with aHUS who had not previously used a complement inhibitor.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Ravulizumab | Experimental | Participants were administered weight-based doses of ravulizumab every 8 weeks for 26 weeks in the Initial Evaluation Period. After the Initial Evaluation Period, participants could enter an Extension Period and receive ravulizumab until the product registration or approval (in accordance with country-specific regulations) or for up to 4.5 years, whichever occurs first. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Ravulizumab | Biological | Single loading dose on Day 1, followed by regular maintenance dosing beginning on Day 15, based on weight: ≥ 40 to < 60 kilograms (kg), 2400 milligrams (mg) loading, then 3000 mg every 8 weeks; ≥ 60 to < 100 kg, 2700 mg loading, then 3300 mg every 8 weeks; ≥ 100 kg, 3000 mg loading, then 3600 mg every 8 weeks. |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage Of Participants With Complete Thrombotic Microangiopathy (TMA) Response at Week 26 | Complete TMA response during the 26-week Initial Evaluation Period is a composite outcome measure that required normalization of hematological parameters (platelet count and lactate dehydrogenase [LDH]) and improvement in kidney function (≥25% reduction in serum creatinine from baseline); for participants on dialysis, baseline was established at least 6 days after the end of dialysis. Participants had to meet these criteria for 2 separate assessments obtained at least 4 weeks (28 days) apart, and any measurement in between. To be considered a responder during the 26-week Initial Evaluation Period, the latest time point a participant could first meet the response criteria was 28 days before the Week 26 (Day 183) assessment. Formal statistical comparison analyses were not planned for this study. The percentage was based on the responders among treated participants. The 95% confidence interval (CI) was based on the asymptotic Gaussian approximation method with a continuity correction. | Week 26 |
| Measure | Description | Time Frame |
|---|---|---|
| Time To Complete TMA Response | Participants that did not have a response were censored at the date of last visit or study discontinuation at the time when the analysis was performed. The time to complete TMA Response is reported in days. The time of the event of a confirmed complete TMA response was considered the first time point at which all the criteria for complete TMA response were met. | Baseline through Week 114 |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Clinical Trial Site | Fort Wayne | Indiana | 46804 | United States | ||
| Clinical Trial Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 34169200 | Result | Barbour T, Scully M, Ariceta G, Cataland S, Garlo K, Heyne N, Luque Y, Menne J, Miyakawa Y, Yoon SS, Kavanagh D; 311 Study Group Members. Long-Term Efficacy and Safety of the Long-Acting Complement C5 Inhibitor Ravulizumab for the Treatment of Atypical Hemolytic Uremic Syndrome in Adults. Kidney Int Rep. 2021 Mar 24;6(6):1603-1613. doi: 10.1016/j.ekir.2021.03.884. eCollection 2021 Jun. | |
| 39468592 |
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| ID | Title | Description |
|---|---|---|
| FG000 | Ravulizumab | Participants received weight-based dosages of ravulizumab for 26 weeks during the Initial Evaluation Period. Participants received a loading dose of ravulizumab intravenously on Day 1, followed by maintenance dosing on Day 15 and once every 8 weeks thereafter. After the Initial Evaluation Period, participants entered an Extension Period and received ravulizumab until the product registration or approval (in accordance with country-specific regulations) or for up to 4.5 years, whichever occurs first. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Initial Evaluation 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 | May 7, 2019 | Nov 25, 2019 |
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|
|
| Participants Who Do Not Require Dialysis at Weeks 26 and 52 | For participants requiring dialysis within 5 days prior to ALXN1210 treatment initiation, the number of participants no longer requiring dialysis is reported | Week 26 and Week 52 |
| Proportion Of Participants With Complete TMA Response At Week 52 | The proportion of participants considered responders, along with a 2-sided 95% CI for the Week 52 time point, is reported. To be considered a responder during the 26-week Initial Evaluation Period, the latest time point a participant could first meet the response criteria was 28 days before the Week 26 (Day 183) assessment (components of the response maintained for at least 28 days). | Week 52 |
| Change From Baseline In Estimated Glomerular Filtration Rate (eGFR) At Weeks 26 and 52 | Kidney function evaluated by eGFR was summarized at baseline and the Week 26 and Week 52 time points using descriptive statistics for continuous variables for the observed value, as well as the change from baseline. The baseline value was defined as the average of the values from the assessments performed prior to the first study drug infusion (these could include results from Screening and the Day 1 visit). A value of 10 milliliters (mL)/minute (min)/1.73 meters squared (m^2) for eGFR was imputed for participants requiring dialysis for acute kidney injury. The observed value and change from baseline are reported in mL/min/1.73 m^2. An increase indicated improvement in kidney function. | Baseline, Week 26 and Week 52 |
| Participants With Change From Baseline In CKD Stage At Weeks 26 and 52 | The CKD stage is presented as the change from baseline in the participants that Improved (excluding those with Stage 1 [normal renal function] at baseline as they cannot improve), Worsened (excluding those with Stage 5 at baseline as they cannot worsen), and Stayed the Same, compared to the CKD stage at baseline. Baseline was derived based on the last available eGFR before starting treatment. Stage 5 was considered the worst category, while Stage 1 was considered the best category. A 2-sided 95% CI for the proportion, based on exact confidence limits using the Clopper-Pearson method, was provided for each category. The CKD stage was classified based on the National Kidney Foundation Chronic Kidney Disease Stage. | Baseline, Week 26, and Week 52 |
| Change From Baseline In Platelet Count At Weeks 26 and 52 | The hematologic TMA parameter of platelet count was summarized at baseline and at Week 26 and Week 52 using descriptive statistics for continuous variables for the change from baseline. Results are reported in platelets*10^9/liter (L) blood. | Baseline, Week 26 and Week 52 |
| Change From Baseline In LDH At Weeks 26 and 52 | The hematologic TMA parameter of serum LDH was summarized at baseline and at Week 26 and Week 52 using descriptive statistics for continuous variables for the change from baseline. Results are reported in units (U)/L. | Baseline, Week 26 and Week 52 |
| Change From Baseline In Hemoglobin At Weeks 26 and 52 | The hematologic TMA parameter of hemoglobin level was summarized at baseline and at Week 26 and Week 52 using descriptive statistics for continuous variables for the change from baseline. Results are reported in grams (g)/L. | Baseline, Week 26 and Week 52 |
| Percentage Of Complement Inhibitor Treatment-naïve Participants With An Increase From Baseline In Hemoglobin ≥20 g/L Through Week 26 and Week 52 | The percentage of participants with an increase from baseline in hemoglobin ≥20 g/L, observed at 2 separate assessments obtained at least 4 weeks (28 days) apart, and any measurement in between, was assessed through Week 26 and Week 52 and is presented as the percentage of responders, along with a 2-sided 95% CI. The 95% CIs are based on exact confidence limits using the Clopper-Pearson method. To be considered a responder during the 26-week and 52-week Extension Periods, the latest time point a participant could first meet the response criteria was 28 days before the respective Week 26 and Week 52 assessments (components of the response maintained for at least 28 days). | Baseline through Week 26 and through Week 52 |
| Change From Baseline In Quality Of Life As Measured By The EuroQol 5-Dimension 3-Level (EQ-5D-3L) At Weeks 26 and 52 | The EQ-5D-3L is a participant-answered questionnaire that scores 5 dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression (scored as a 1, 2, or 3, with 3 being the worst health state), as well as health state on a visual analogue scale (0 to 100, with 100 representing the best health state). From these scores, a summary index score is derived using the time trade-off valuation set for the United States and ranges from -1 to 1, where a score above 0.94 indicates full health. An increase in score from baseline indicates improvement in quality of life. | Baseline, Week 26 and Week 52 |
| Change From Baseline In Quality Of Life As Measured By The Functional Assessment Of Chronic Illness Therapy (FACIT)-Fatigue Version 4 Questionnaire At Weeks 26 and 52 | Quality of life was evaluated in part using FACIT Fatigue Version 4. The data were summarized at baseline and at the Week 26 and Week 52 time point using descriptive statistics for continuous variables. The FACIT Fatigue Version 4 questionnaire at baseline and the Week 52 timepoint was scored using standard scoring algorithms. The score ranges from 0-52, with a higher score indicating less Fatigue. An increase in score indicated an improvement in quality of life. | Baseline, Week 26 and Week 52 |
| Fort Wayne |
| Indiana |
| 46845 |
| United States |
| Clinical Trial Site | Durham | North Carolina | 27705 | United States |
| Clinical Trial Site | Winston-Salem | North Carolina | 27103 | United States |
| Clinical Trial Site | Columbus | Ohio | 43210 | United States |
| Clinical Trial Site | Clayton | Australia |
| Clinical Trial Site | Geelong | Australia |
| Clinical Trial Site | Parkville | Australia |
| Clinical Trial Site | Vienna | Austria |
| Clinical Trial Site | Brussels | Belgium |
| Clinical Trial Site | London | Canada |
| Clinical Trial Site | Bordeaux | France |
| Clinical Trial Site | Clermont-Ferrand | France |
| Clinical Trial Site | Lille | France |
| Clinical Trial Site | Montpellier | France |
| Clinical Trial Site | Nice | France |
| Clinical Trial Site | Paris | France |
| Clinical Trial Site | Aachen | Germany |
| Clinical Trial Site | Essen | Germany |
| Clinical Trial Site | Hanover | Germany |
| Clinical Trial Site | München | Germany |
| Clinical Trial Site | Tübingen | Germany |
| Clinical Trial Site | Bologna | Italy |
| Clinical Trial Site | Florence | Italy |
| Clinical Trial Site | Saitama | Japan |
| Clinical Trial Site | Tokyo | Japan |
| Clinical Trial Site | Moscow | Russia |
| Clinical Trial Site | Saint Petersburg | Russia |
| Clinical Trial Site | Gyeonggi-do | South Korea |
| Clinical Trial Site | Seoul | South Korea |
| Clinical Trial Site | Barcelona | Spain |
| Clinical Trial Site | Madrid | Spain |
| Clinical Trial Site | Valencia | Spain |
| Clinical Trial Site | Taichung | Taiwan |
| Clinical Trial Site | Taipei | Taiwan |
| Clinical Trial Site | Cardiff | United Kingdom |
| Clinical Trial Site | London | United Kingdom |
| Derived |
| Matsumoto M, Shimono A, Yokosawa J, Hirose K, Wang E, Maruyama S. Correlation between a 2-week change in platelet count and clinical outcomes after the initiation of ravulizumab treatment in adult patients with atypical hemolytic uremic syndrome: post-hoc analysis of the phase III trial. Thromb J. 2024 Oct 28;22(1):93. doi: 10.1186/s12959-024-00652-1. |
| 36329366 | Derived | Cammett TJ, Garlo K, Millman EE, Rice K, Toste CM, Faas SJ. Exploratory Prognostic Biomarkers of Complement-Mediated Thrombotic Microangiopathy (CM-TMA) in Adults with Atypical Hemolytic Uremic Syndrome (aHUS): Analysis of a Phase III Study of Ravulizumab. Mol Diagn Ther. 2023 Jan;27(1):61-74. doi: 10.1007/s40291-022-00620-3. Epub 2022 Nov 4. |
| 33783815 | Derived | Pugh D, O'Sullivan ED, Duthie FA, Masson P, Kavanagh D. Interventions for atypical haemolytic uraemic syndrome. Cochrane Database Syst Rev. 2021 Mar 23;3(3):CD012862. doi: 10.1002/14651858.CD012862.pub2. |
| 33407224 | Derived | Gackler A, Schonermarck U, Dobronravov V, La Manna G, Denker A, Liu P, Vinogradova M, Yoon SS, Praga M. Efficacy and safety of the long-acting C5 inhibitor ravulizumab in patients with atypical hemolytic uremic syndrome triggered by pregnancy: a subgroup analysis. BMC Nephrol. 2021 Jan 6;22(1):5. doi: 10.1186/s12882-020-02190-0. |
| Received At Least 1 Dose of Study Drug |
|
| COMPLETED | Completed the Initial Evaluation Period |
|
| NOT COMPLETED |
|
|
| Extension Period (Up to 4.5 Years) |
|
|
Full Analysis Set (FAS): all participants who received at least 1 dose of ravulizumab, had at least 1 post-baseline efficacy assessment, and met pre-specified eligibility criteria.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Ravulizumab | Participants received weight-based dosages of ravulizumab for 26 weeks during the Initial Evaluation Period. Participants received a loading dose of ravulizumab intravenously on Day 1, followed by maintenance dosing on Day 15 and once every 8 weeks thereafter. After the Initial Evaluation Period, participants entered an Extension Period and received ravulizumab until the product registration or approval (in accordance with country-specific regulations) or for up to 4.5 years, whichever occurs first. |
| Units | Counts |
|---|---|
| Participants |
|
| 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 |
| ||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Race (NIH/OMB) | 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 | Percentage Of Participants With Complete Thrombotic Microangiopathy (TMA) Response at Week 26 | Complete TMA response during the 26-week Initial Evaluation Period is a composite outcome measure that required normalization of hematological parameters (platelet count and lactate dehydrogenase [LDH]) and improvement in kidney function (≥25% reduction in serum creatinine from baseline); for participants on dialysis, baseline was established at least 6 days after the end of dialysis. Participants had to meet these criteria for 2 separate assessments obtained at least 4 weeks (28 days) apart, and any measurement in between. To be considered a responder during the 26-week Initial Evaluation Period, the latest time point a participant could first meet the response criteria was 28 days before the Week 26 (Day 183) assessment. Formal statistical comparison analyses were not planned for this study. The percentage was based on the responders among treated participants. The 95% confidence interval (CI) was based on the asymptotic Gaussian approximation method with a continuity correction. | Full Analysis Set (FAS): all participants who received at least 1 dose of ravulizumab, had at least 1 post-baseline efficacy assessment, and met pre-specified eligibility criteria. | Posted | Number | 95% Confidence Interval | percentage of participants | Week 26 |
|
|
| ||||||||||||||||||||||||||||||||||
| Secondary | Time To Complete TMA Response | Participants that did not have a response were censored at the date of last visit or study discontinuation at the time when the analysis was performed. The time to complete TMA Response is reported in days. The time of the event of a confirmed complete TMA response was considered the first time point at which all the criteria for complete TMA response were met. | Full Analysis Set (FAS): all participants who received at least 1 dose of ravulizumab, had at least 1 post-baseline efficacy assessment, and met pre-specified eligibility criteria. | Posted | Median | Inter-Quartile Range | days | Baseline through Week 114 |
|
| |||||||||||||||||||||||||||||||||||
| Secondary | Participants Who Do Not Require Dialysis at Weeks 26 and 52 | For participants requiring dialysis within 5 days prior to ALXN1210 treatment initiation, the number of participants no longer requiring dialysis is reported | Full Analysis Set (FAS): all participants who received at least 1 dose of ravulizumab, had at least 1 post-baseline efficacy assessment, met pre-specified eligibility criteria, and had evaluable data at specified timepoint. Here, Overall Number of Participants analyzed signifies those who were evaluable for this outcome measure. | Posted | Count of Participants | Participants | Week 26 and Week 52 |
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Proportion Of Participants With Complete TMA Response At Week 52 | The proportion of participants considered responders, along with a 2-sided 95% CI for the Week 52 time point, is reported. To be considered a responder during the 26-week Initial Evaluation Period, the latest time point a participant could first meet the response criteria was 28 days before the Week 26 (Day 183) assessment (components of the response maintained for at least 28 days). | Full Analysis Set (FAS): all participants who received at least 1 dose of ravulizumab, had at least 1 post-baseline efficacy assessment, met pre-specified eligibility criteria, and had evaluable data at specified timepoint (Week 52). | Posted | Number | 95% Confidence Interval | proportion of participants | Week 52 |
|
| |||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline In Estimated Glomerular Filtration Rate (eGFR) At Weeks 26 and 52 | Kidney function evaluated by eGFR was summarized at baseline and the Week 26 and Week 52 time points using descriptive statistics for continuous variables for the observed value, as well as the change from baseline. The baseline value was defined as the average of the values from the assessments performed prior to the first study drug infusion (these could include results from Screening and the Day 1 visit). A value of 10 milliliters (mL)/minute (min)/1.73 meters squared (m^2) for eGFR was imputed for participants requiring dialysis for acute kidney injury. The observed value and change from baseline are reported in mL/min/1.73 m^2. An increase indicated improvement in kidney function. | Full Analysis Set (FAS): all participants who received at least 1 dose of ravulizumab, had at least 1 post-baseline efficacy assessment, met pre-specified eligibility criteria, and had evaluable data at the specified timepoint (Week 26 or Week 52). | Posted | Median | Full Range | mL/min/1.73 m^2 | Baseline, Week 26 and Week 52 |
|
| |||||||||||||||||||||||||||||||||||
| Secondary | Participants With Change From Baseline In CKD Stage At Weeks 26 and 52 | The CKD stage is presented as the change from baseline in the participants that Improved (excluding those with Stage 1 [normal renal function] at baseline as they cannot improve), Worsened (excluding those with Stage 5 at baseline as they cannot worsen), and Stayed the Same, compared to the CKD stage at baseline. Baseline was derived based on the last available eGFR before starting treatment. Stage 5 was considered the worst category, while Stage 1 was considered the best category. A 2-sided 95% CI for the proportion, based on exact confidence limits using the Clopper-Pearson method, was provided for each category. The CKD stage was classified based on the National Kidney Foundation Chronic Kidney Disease Stage. | Full Analysis Set (FAS): all participants who received at least 1 dose of ravulizumab, had at least 1 post-baseline efficacy assessment, met pre-specified eligibility criteria, and had evaluable data at the specified timepoint (Week 26 or Week 52). Here, Overall 'Number of Participants Analyzed' signifies those who were evaluable for this outcome measure and 'Number Analyzed' signifies participants evaluable for specified categories. | Posted | Count of Participants | Participants | Baseline, Week 26, and Week 52 |
| |||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline In Platelet Count At Weeks 26 and 52 | The hematologic TMA parameter of platelet count was summarized at baseline and at Week 26 and Week 52 using descriptive statistics for continuous variables for the change from baseline. Results are reported in platelets*10^9/liter (L) blood. | Full Analysis Set (FAS): all participants who received at least 1 dose of ravulizumab, had at least 1 post-baseline efficacy assessment, met pre-specified eligibility criteria, and had evaluable data at the specified timepoints (Week 26 or 52). | Posted | Median | Full Range | platelets*10^9/L | Baseline, Week 26 and Week 52 |
|
| |||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline In LDH At Weeks 26 and 52 | The hematologic TMA parameter of serum LDH was summarized at baseline and at Week 26 and Week 52 using descriptive statistics for continuous variables for the change from baseline. Results are reported in units (U)/L. | Full Analysis Set (FAS): all participants who received at least 1 dose of ravulizumab, had at least 1 post-baseline efficacy assessment, met pre-specified eligibility criteria, and had evaluable data at the specified timepoints (Week 26 or Week 52). | Posted | Median | Full Range | U/L | Baseline, Week 26 and Week 52 |
|
| |||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline In Hemoglobin At Weeks 26 and 52 | The hematologic TMA parameter of hemoglobin level was summarized at baseline and at Week 26 and Week 52 using descriptive statistics for continuous variables for the change from baseline. Results are reported in grams (g)/L. | Full Analysis Set (FAS): all participants who received at least 1 dose of ravulizumab, had at least 1 post-baseline efficacy assessment, met pre-specified eligibility criteria, and had evaluable data at the specified timepoints (Week 26 or Week 52). | Posted | Median | Full Range | g/L | Baseline, Week 26 and Week 52 |
|
| |||||||||||||||||||||||||||||||||||
| Secondary | Percentage Of Complement Inhibitor Treatment-naïve Participants With An Increase From Baseline In Hemoglobin ≥20 g/L Through Week 26 and Week 52 | The percentage of participants with an increase from baseline in hemoglobin ≥20 g/L, observed at 2 separate assessments obtained at least 4 weeks (28 days) apart, and any measurement in between, was assessed through Week 26 and Week 52 and is presented as the percentage of responders, along with a 2-sided 95% CI. The 95% CIs are based on exact confidence limits using the Clopper-Pearson method. To be considered a responder during the 26-week and 52-week Extension Periods, the latest time point a participant could first meet the response criteria was 28 days before the respective Week 26 and Week 52 assessments (components of the response maintained for at least 28 days). | Full Analysis Set (FAS): all participants who received at least 1 dose of ravulizumab, had at least 1 post-baseline efficacy assessment, met pre-specified eligibility criteria, and had evaluable data at the specified timepoints (Week 26 or Week 52). Here, Overall Number of Participants analyzed signifies those who were evaluable for this outcome measure. | Posted | Number | 95% Confidence Interval | percentage of participants | Baseline through Week 26 and through Week 52 |
| ||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline In Quality Of Life As Measured By The EuroQol 5-Dimension 3-Level (EQ-5D-3L) At Weeks 26 and 52 | The EQ-5D-3L is a participant-answered questionnaire that scores 5 dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression (scored as a 1, 2, or 3, with 3 being the worst health state), as well as health state on a visual analogue scale (0 to 100, with 100 representing the best health state). From these scores, a summary index score is derived using the time trade-off valuation set for the United States and ranges from -1 to 1, where a score above 0.94 indicates full health. An increase in score from baseline indicates improvement in quality of life. | Full Analysis Set (FAS): all participants who received at least 1 dose of ravulizumab, had at least 1 post-baseline efficacy assessment, met pre-specified eligibility criteria, and had evaluable data at the specified timepoint (Week 26 or Week 52). | Posted | Median | Full Range | units on a scale | Baseline, Week 26 and Week 52 |
|
| |||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline In Quality Of Life As Measured By The Functional Assessment Of Chronic Illness Therapy (FACIT)-Fatigue Version 4 Questionnaire At Weeks 26 and 52 | Quality of life was evaluated in part using FACIT Fatigue Version 4. The data were summarized at baseline and at the Week 26 and Week 52 time point using descriptive statistics for continuous variables. The FACIT Fatigue Version 4 questionnaire at baseline and the Week 52 timepoint was scored using standard scoring algorithms. The score ranges from 0-52, with a higher score indicating less Fatigue. An increase in score indicated an improvement in quality of life. | Full Analysis Set (FAS): all participants who received at least 1 dose of ravulizumab, had at least 1 post-baseline efficacy assessment, met pre-specified eligibility criteria, and had evaluable data at the specified timepoints (Week 26 and Week 52). | Posted | Median | Full Range | units on a scale | Baseline, Week 26 and Week 52 |
|
|
From the beginning of the initial evaluation period (Day1) up to 4.5 years
Not provided
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 | Ravulizumab | Participants received weight-based dosages of ravulizumab for 26 weeks during the Initial Evaluation Period. Participants received a loading dose of ravulizumab intravenously on Day 1, followed by maintenance dosing on Day 15 and once every 8 weeks thereafter. After the Initial Evaluation Period, participants entered an Extension Period and received ravulizumab until the product registration or approval (in accordance with country-specific regulations) or for up to 4.5 years, whichever occurs first. | 4 | 58 | 38 | 58 | 56 | 58 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pneumonia | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
| |
| Septic shock | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Device related infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Escherichia pyelonephritis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Fungaemia | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Gastrointestinal infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Peritonitis bacterial | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Chronic kidney disease | Renal and urinary disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Hydronephrosis | Renal and urinary disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Nephrolithiasis | Renal and urinary disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Renal failure | Renal and urinary disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Renal haematoma | Renal and urinary disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Renal impairment | Renal and urinary disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Renal pseudoaneurysm | Renal and urinary disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Atypical haemolytic uraemic syndrome | Blood and lymphatic system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Autoimmune haemolytic anaemia | Blood and lymphatic system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Febrile neutropenia | Blood and lymphatic system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Malignant hypertension | Vascular disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Hypertensive crisis | Vascular disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Lower gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Toothache | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Haemorrhage intracranial | Nervous system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Lacunar infarction | Nervous system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Loss of consciousness | Nervous system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Seizure | Nervous system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Acute pulmonary oedema | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Pulmonary oedema | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Respiratory disorder | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Hypersensitivity | Immune system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Kidney transplant rejection | Immune system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Seroma | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Shunt occlusion | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Biopsy kidney | Investigations | MedDRA 25.1 | Systematic Assessment |
| |
| Troponin increased | Investigations | MedDRA 25.1 | Systematic Assessment |
| |
| Hypervolaemia | Metabolism and nutrition disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Vertigo positional | Ear and labyrinth disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Vitreous haemorrhage | Eye disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Device leakage | Product Issues | MedDRA 25.1 | Systematic Assessment |
| |
| Systemic inflammatory response syndrome | General disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Nephrectomy | Surgical and medical procedures | MedDRA 25.1 | Systematic Assessment |
| |
| Arteriovenous fistula | Vascular disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Pancreatitis acute | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| End stage renal disease | Renal and urinary disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Enterocolitis infectious | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Peritonitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Stenotrophomonas infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Endophthalmitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Enterococcal infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Hantaviral infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Large intestine infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Pseudomonal sepsis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Pyelonephritis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Rhinovirus infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Staphylococcal sepsis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Leukopenia | Blood and lymphatic system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Pancreatitis relapsing | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Retroperitoneal haematoma | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Shunt stenosis | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Femoral neck fracture | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Post procedural complication | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Shunt thrombosis | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Vascular access site thrombosis | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Milk allergy | Immune system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Transplant rejection | Immune system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Cervicobrachial syndrome | Nervous system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Cardiac failure acute | Cardiac disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Pericardial effusion | Cardiac disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Ventricular arrhythmia | Cardiac disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Vitreous disorder | Eye disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Cholecystitis | Hepatobiliary disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Dermatitis bullous | Skin and subcutaneous tissue disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Dysuria | Renal and urinary disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Immune thrombocytopenia | Blood and lymphatic system disorders | MedDRA 25.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Diarrhoea | Gastrointestinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Somnolence | Nervous system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Oral herpes | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Pain | General disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Face oedema | General disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Hypocalcaemia | Metabolism and nutrition disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Vitamin D deficiency | Metabolism and nutrition disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Alopecia | Skin and subcutaneous tissue disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Dry Skin | Skin and subcutaneous tissue disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 25.1 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA 25.1 | Systematic Assessment |
| |
| Anaemia | Blood and lymphatic system disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| End stage renal disease | Renal and urinary disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Vision blurred | Eye disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Palpitations | Cardiac disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Drug hypersensitivity | Immune system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Hypoaesthesia | Nervous system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Device related infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Catheter site pain | General disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Haemorrhoids | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Paraesthesia | Nervous system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Presyncope | Nervous system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Iron deficiency | Metabolism and nutrition disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Infusion related reaction | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Skin abrasion | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA 25.1 | Systematic Assessment |
| |
| Weight decreased | Investigations | MedDRA 25.1 | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Dysuria | Renal and urinary disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Delirium | Psychiatric disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Intermenstrual bleeding | Reproductive system and breast disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Ovarian cyst | Reproductive system and breast disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Cystitis | Infections and infestations | MedDRA 25.1 | 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 | May 23, 2018 | Jun 30, 2022 | SAP_002.pdf |
| ID | Term |
|---|---|
| D065766 | Atypical Hemolytic Uremic Syndrome |
| ID | Term |
|---|---|
| D006463 | Hemolytic-Uremic Syndrome |
| D014511 | Uremia |
| D007674 | Kidney Diseases |
| D014570 | Urologic Diseases |
| D052776 | Female Urogenital Diseases |
| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D052801 | Male Urogenital Diseases |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D057049 | Thrombotic Microangiopathies |
| D013921 | Thrombocytopenia |
| D001791 | Blood Platelet Disorders |
| D000095542 | Cytopenia |
Not provided
Not provided
| ID | Term |
|---|---|
| C000629409 | ravulizumab |
Not provided
Not provided
Not provided
| Withdrawal by Subject |
|
| Other than specified |
|
| Unknown or Not Reported |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
|
| ≥ 25% improvement in serum creatinine from baseline |
|
|
|
|
| Participants |
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
|
|
|