Trial · NCT03131219
Study of Ravulizumab in Children and Adolescents With Atypical Hemolytic Uremic Syndrome (aHUS)
NCT03131219, Phase 3, Completed, Alexion Pharmaceuticals, Inc.; results first posted 2020-09-16
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study of Ravulizumab in Children and Adolescents With Atypical Hemolytic Uremic Syndrome (aHUS). Trials, record NCT03131219. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03131219
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of Ravulizumab in Children and Adolescents With Atypical Hemolytic Uremic Syndrome (aHUS)
- NCT number
- NCT03131219
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 34 participants, actual
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Conditions
- Atypical Hemolytic Uremic Syndrome (aHUS)
- Interventions
- Ravulizumab
- First posted
- 2017-04-27
- Last updated
- 2024-03-15
- Other registries' IDs
- eudract:2016-002499-29
- Results first posted
- 2020-09-16
Source ClinicalTrials.gov
Registered outcomes11
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage Of Complement Inhibitor Treatment-naïve Participants With Complete Thrombotic Microangiopathy (TMA) Response at Week 26 | 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. Percentage based on the responders among treated participants. Confidence interval (CI) based on exact confidence limits using the Clopper Pearson method. |
| Secondary | Time To Complete TMA Response In Complement Inhibitor Treatment-naïve Participants | Baseline through at least Week 52 and up to Week 111 | 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. Participants had to meet all complete TMA response criteria at 2 separate assessments obtained at least 4 weeks (28 days) apart, and any measurement in between. |
| Secondary | Proportion Of Complement Inhibitor Treatment-naïve Participants With Complete TMA Response At Week 52 | Week 52 | The proportion of participants considered responders, along with a 2-sided 95% CI based on exact confidence limits using the Clopper Pearson method is reported. |
| Secondary | Participants Who Do Not Require Dialysis at Weeks 26 and 52 | Week 26 and Week 52 | For participants requiring dialysis within 5 days prior to ALXN1210 treatment initiation, the number of participants no longer requiring dialysis is reported. |
| Secondary | Change From Baseline In eGFR At Weeks 26 and 52 | Baseline, Week 26 and Week 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 mL/min/1.73 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. |
| Secondary | Participants With Change From Baseline In CKD Stage At Weeks 26 and 52 | Baseline, Week 26, and Week 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. |
| Secondary | Change From Baseline In Platelet Count At Weeks 26 and 52 | Baseline, Week 26 and Week 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. |
| Secondary | Change From Baseline In LDH At Weeks 26 and 52 | Baseline, Week 26 and Week 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 serum. |
| Secondary | Change From Baseline In Hemoglobin At Weeks 26 and 52 | Baseline, Week 26 and Week 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 blood. |
| Secondary | Percentage Of Complement Inhibitor Treatment-naïve Participants With An Increase From Baseline In Hemoglobin ≥20 g/L Through Week 26 and Week 52 | Baseline through Week 26 and through 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). |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 registered outcomesOutcomes11
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage Of Complement Inhibitor Treatment-naïve Participants With Complete Thrombotic Microangiopathy (TMA) Response at Week 26 | Week 26 | 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. Here, Overall 'Number of Participants Analyzed' signifies those who were evaluable for this outcome measure. | percentage of participants | NUMBER |
| SECONDARY | Time To Complete TMA Response In Complement Inhibitor Treatment-naïve Participants | Baseline through at least Week 52 and up to Week 111 | 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. | days | MEDIAN |
| SECONDARY | Proportion Of Complement Inhibitor Treatment-naïve Participants With Complete TMA Response At Week 52 | Week 52 | 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 and 'Number Analyzed' signifies participants evaluable for specified categories. | proportion of participants | NUMBER |
| SECONDARY | Participants Who Do Not Require Dialysis at Weeks 26 and 52 | Week 26 and Week 52 | 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 and 'Number Analyzed' signifies participants evaluable for specified categories. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline In eGFR At Weeks 26 and 52 | Baseline, Week 26 and Week 52 | 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. | mL/min/1.73 m^2 | MEDIAN |
| SECONDARY | Participants With Change From Baseline In CKD Stage At Weeks 26 and 52 | Baseline, Week 26, and Week 52 | 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. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline In Platelet Count At Weeks 26 and 52 | Baseline, Week 26 and Week 52 | 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 time point (Week 26 or Week 52). Here, 'Number Analyzed' signifies participants evaluable for specified categories. | platelets*10^9/L | MEDIAN |
| SECONDARY | Change From Baseline In LDH At Weeks 26 and 52 | Baseline, Week 26 and Week 52 | 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. | U/L | MEDIAN |
| SECONDARY | Change From Baseline In Hemoglobin At Weeks 26 and 52 | Baseline, Week 26 and Week 52 | 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, 'Number Analyzed' signifies participants evaluable for specified categories. | g/L | MEDIAN |
| SECONDARY | Percentage Of Complement Inhibitor Treatment-naïve Participants With An Increase From Baseline In Hemoglobin ≥20 g/L Through Week 26 and Week 52 | Baseline through Week 26 and through Week 52 | 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. | percentage of participants | NUMBER |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 outcomesResults by group57
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage Of Complement Inhibitor Treatment-naïve Participants With Complete Thrombotic Microangiopathy (TMA) Response at Week 26 | Complement Inhibitor Treatment Naïve | Complete TMA response | 77.8 | 95% Confidence Interval 52.4 to 93.6 | percentage of participants |
| Percentage Of Complement Inhibitor Treatment-naïve Participants With Complete Thrombotic Microangiopathy (TMA) Response at Week 26 | Complement Inhibitor Treatment Naïve | Platelet count normalization | 94.4 | 95% Confidence Interval 72.7 to 99.9 | percentage of participants |
| Percentage Of Complement Inhibitor Treatment-naïve Participants With Complete Thrombotic Microangiopathy (TMA) Response at Week 26 | Complement Inhibitor Treatment Naïve | LDH normalization | 88.9 | 95% Confidence Interval 65.3 to 98.6 | percentage of participants |
| Percentage Of Complement Inhibitor Treatment-naïve Participants With Complete Thrombotic Microangiopathy (TMA) Response at Week 26 | Complement Inhibitor Treatment Naïve | ≥ 25% improvement in serum creatinine from baseline | 83.3 | 95% Confidence Interval 58.6 to 96.4 | percentage of participants |
| Time To Complete TMA Response In Complement Inhibitor Treatment-naïve Participants | Complement Inhibitor Treatment Naïve | 30.0 | Inter-Quartile Range 22.0 to 88.0 | days | |
| Proportion Of Complement Inhibitor Treatment-naïve Participants With Complete TMA Response At Week 52 | Complement Inhibitor Treatment Naïve | Complete TMA responder | 0.882 | 95% Confidence Interval 0.636 to 0.985 | proportion of participants |
| Proportion Of Complement Inhibitor Treatment-naïve Participants With Complete TMA Response At Week 52 | Complement Inhibitor Treatment Naïve | Platelet count normalization | 0.882 | 95% Confidence Interval 0.636 to 0.985 | proportion of participants |
| Proportion Of Complement Inhibitor Treatment-naïve Participants With Complete TMA Response At Week 52 | Complement Inhibitor Treatment Naïve | LDH normalization | 1.000 | 95% Confidence Interval 0.805 to 1.000 | proportion of participants |
| Proportion Of Complement Inhibitor Treatment-naïve Participants With Complete TMA Response At Week 52 | Complement Inhibitor Treatment Naïve | ≥25% improvement in serum creatinine from baseline | 1.000 | 95% Confidence Interval 0.805 to 1.000 | proportion of participants |
| Participants Who Do Not Require Dialysis at Weeks 26 and 52 | Complement Inhibitor Treatment Naïve | Week 26 | 5 | Participants |
1 to 10 of 57Source ClinicalTrials.gov
See all 57 results by groupParticipant flow16
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Initial Evaluation Period | STARTED | Complement Inhibitor Treatment Naïve | 24 | |
| Initial Evaluation Period | STARTED | Eculizumab Experienced | 10 | |
| Initial Evaluation Period | Received At Least 1 Dose of Study Drug | Complement Inhibitor Treatment Naïve | 24 | |
| Initial Evaluation Period | Received At Least 1 Dose of Study Drug | Eculizumab Experienced | 10 | |
| Initial Evaluation Period | COMPLETED | Complement Inhibitor Treatment Naïve | 17 | |
| Initial Evaluation Period | COMPLETED | Eculizumab Experienced | 10 | |
| Initial Evaluation Period | NOT COMPLETED | Complement Inhibitor Treatment Naïve | 7 | |
| Initial Evaluation Period | NOT COMPLETED | Eculizumab Experienced | 0 | |
| Extension Period | STARTED | Complement Inhibitor Treatment Naïve | 17 | |
| Extension Period | STARTED | Eculizumab Experienced | 10 |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 participant flowAdverse events178
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Upper respiratory tract infection | Infections and infestations | Complement Inhibitor Treatment Naïve | 3 | 24 | 4 |
| other | Upper respiratory tract infection | Infections and infestations | Eculizumab Experienced | 4 | 10 | 16 |
| other | Nasopharyngitis | Infections and infestations | Complement Inhibitor Treatment Naïve | 7 | 24 | 13 |
| other | Nasopharyngitis | Infections and infestations | Eculizumab Experienced | 2 | 10 | 2 |
| other | Otitis media | Infections and infestations | Complement Inhibitor Treatment Naïve | 0 | 24 | 0 |
| other | Otitis media | Infections and infestations | Eculizumab Experienced | 2 | 10 | 2 |
| other | Pharyngitis | Infections and infestations | Complement Inhibitor Treatment Naïve | 2 | 24 | 2 |
| other | Pharyngitis | Infections and infestations | Eculizumab Experienced | 2 | 10 | 2 |
| other | Viral upper respiratory tract infection | Infections and infestations | Complement Inhibitor Treatment Naïve | 2 | 24 | 4 |
| other | Viral upper respiratory tract infection | Infections and infestations | Eculizumab Experienced | 2 | 10 | 2 |
1 to 10 of 178Source ClinicalTrials.gov
See all 178 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Complement Inhibitor Treatment Naïve | 0 | 24 |
| Total, all-cause mortality | Eculizumab Experienced | 0 | 10 |
| Total, other adverse events | Complement Inhibitor Treatment Naïve | 21 | 24 |
| Total, other adverse events | Eculizumab Experienced | 10 | 10 |
| Total, serious adverse events | Complement Inhibitor Treatment Naïve | 16 | 24 |
| Total, serious adverse events | Eculizumab Experienced | 1 | 10 |
Source ClinicalTrials.gov
Papers4
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Complement Inhibitor Treatment Naïve vs Eculizumab Experienced: deaths from any cause | Trial comparisons |
| Complement Inhibitor Treatment Naïve vs Eculizumab Experienced: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03131219
GET https://conductscience.org/api/trials/v1/NCT03131219/results?page=2