A Phase 3 interventional study of Ravulizumab in Atypical Hemolytic Uremic Syndrome (aHUS), sponsored by Alexion Pharmaceuticals, Inc.. Completed at 38 sites in 14 countries. Per ClinicalTrials.gov, last updated 2024-02-20.
Sponsored by Alexion Pharmaceuticals, Inc. (part of AstraZeneca) · Phase 3, Interventional, and Treatment
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Among 19 studies in Atypical Hemolytic Uremic Syndrome by AstraZeneca — 4 open to participants now, 5 under way, none of them phase 3. Atypical Hemolytic Uremic Syndrome · AstraZeneca.
Among the 19 studies in Atypical Hemolytic Uremic Syndrome by AstraZeneca, this one has the largest enrollment of the 10 interventional ones that give a count, is one of 2 that started in 2017, is one of 2 in phase 3, and was the 13th registered.
45 studies on the registry are indexed under Atypical Hemolytic Uremic Syndrome; 14 are open to participants now.
Browse Atypical Hemolytic Uremic Syndrome studies →4 of the 19 studies in Atypical Hemolytic Uremic Syndrome by AstraZeneca are open now, in 12 countries. Recruiting first, the newest first:
AstraZeneca · Sites in Poland
Ages 18+
Alexion Pharmaceuticals, Inc. · Sites in Japan
Ages 18+
Alexion Pharmaceuticals, Inc. · Sites in United States, Australia, France and 4 more countries
Women only
Alexion Pharmaceuticals, Inc. · Sites in United States, Australia, Belgium and 14 more countries
By country, each count listing its studies:
Of the 12 Atypical Hemolytic Uremic Syndrome studies recruiting now, 10 (83%) set no upper age limit and none (0%) stop at 75 or younger; 6 take children under 18; 2 take healthy volunteers.
This study: all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
19 studies; the count open now beside each. This study's row is marked.
First in 2009; this study’s year is marked. 2 started in 2017.
This study's enrollment of 58 is above the median of 34 across 26 interventional studies indexed under Atypical Hemolytic Uremic Syndrome.
Median enrollment 18 across the 10 interventional studies, 80 across the 9 observational. Of the 14 that completed or were terminated, 9 (64%) have results posted.
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This study is registered under Alexion Pharmaceuticals, Inc., one of the names counted as AstraZeneca. Under that name alone:
Alexion Pharmaceuticals, Inc. is the lead sponsor of 255 studies on the registry; 23 are open to participants now.
Of its 101 completed or terminated interventional studies of FDA-regulated products, 75 (74%) have results posted.
1 of the studies in Atypical Hemolytic Uremic Syndrome by AstraZeneca that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT07308574Alexion Pharmaceuticals, Inc.Post-Marketing Clinical Study of Ravulizumab in Participants With Clinical aHUS | 2027-06-25 |
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Among the 19 studies in Atypical Hemolytic Uremic Syndrome by AstraZeneca, from their registry updates:
Nothing recorded among them since 2026-09-25.
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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.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Biological: Ravulizumab
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.
Also known as: ALXN1210, Ultomiris
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.
Time frame: Week 26
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.
Time frame: Baseline through Week 114
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
Time frame: 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).
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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).
Time frame: 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.
Time frame: 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.
Time frame: Baseline, Week 26 and Week 52
| Milestone | Ravulizumab |
|---|---|
| Started | 58 |
| Received at least 1 dose of study drug | 58 |
| Completed | 49 |
| Not completed | 9 |
| Withdrew: Failed to meet eligibility criteria | 2 |
| Withdrew: Adverse event | 3 |
| Withdrew: Death | 2 |
| Withdrew: Physician decision | 1 |
| Withdrew: Protocol violation | 1 |
| Milestone | Ravulizumab |
|---|---|
| Started | 49 |
| Received at least 1 dose of study drug | 47 |
| Completed | 28 |
| Not completed | 21 |
| Withdrew: Death | 1 |
| Withdrew: Physician decision | 5 |
| Withdrew: Protocol violation | 1 |
| Withdrew: Withdrawal by subject | 9 |
| Withdrew: Other than specified | 5 |
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.
| percentage of participants | Ravulizumab |
|---|---|
| Complete TMA Response | 53.6 (39.6 to 67.5) |
| Platelet count normalization | 83.9 (73.4 to 94.4) |
| LDH normalization | 76.8 (64.8 to 88.7) |
| ≥ 25% improvement in serum creatinine from baseline | 58.9 (45.2 to 72.7) |
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.
| days | Ravulizumab |
|---|---|
| Time To Complete TMA Response | 86.0 (42.0 to NA) |
For participants requiring dialysis within 5 days prior to ALXN1210 treatment initiation, the number of participants no longer requiring dialysis is reported
| Participants | Ravulizumab |
|---|---|
| Week 26 | 16 |
| Week 52 | 16 |
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).
| proportion of participants | Ravulizumab |
|---|---|
| Complete TMA Response | 0.500 (0.346 to 0.654) |
| Platelet Count Normalization | 0.909 (0.783 to 0.975) |
| LDH Normalization | 0.750 (0.597 to 0.868) |
| ≥25% improvement in serum creatinine from baseline | 0.659 (0.501 to 0.795) |
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.
| mL/min/1.73 m^2 | Ravulizumab |
|---|---|
| Baseline | 10.00 (4 to 80) |
| Change From Baseline at Week 26 | 29.00 (-13 to 108) |
| Change From Baseline at Week 52 | 23.00 (-13 to 95) |
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.
| Participants | Ravulizumab |
|---|---|
| Week 26, Improved | 32 |
| Week 26, Worsened | 2 |
| Week 26, Stayed the Same | 13 |
| Week 52, Improved | 30 |
| Week 52, Worsened | 2 |
| Week 52, Stayed the Same | 11 |
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.
| platelets*10^9/L | Ravulizumab |
|---|---|
| Baseline | 95.25 (18 to 473) |
| Change From Baseline at Week 26 | 125.00 (-126 to 338) |
| Change From Baseline at Week 52 | 126.25 (-51.5 to 335) |
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.
| U/L | Ravulizumab |
|---|---|
| Baseline | 508.00 (229.5 to 3249) |
| Change From Baseline at Week 26 | -310.75 (-3072 to 8.5) |
| Change From Baseline at Week 52 | -293.75 (-3107 to 81) |
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.
| g/L | Ravulizumab |
|---|---|
| Baseline | 85.00 (60.5 to 140) |
| Change From Baseline at Week 26 | 35.00 (-9 to 69) |
| Change From Baseline at Week 52 | 41.75 (-25 to 83.5) |
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).
| percentage of participants | Ravulizumab |
|---|---|
| Week 26 | 75.5 (61.1 to 86.7) |
| Week 52 | 86.4 (72.6 to 94.8) |
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.
| units on a scale | Ravulizumab |
|---|---|
| Baseline | 0.59 (-0.113 to 0.753) |
| Change from Baseline at Week 26 | 0.15 (-0.138 to 0.723) |
| Change from Baseline at Week 52 | 0.26 (-0.143 to 0.707) |
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.
| units on a scale | Ravulizumab |
|---|---|
| Baseline | 24.00 (0 to 51) |
| Change From Baseline at Week 26 | 20.00 (-16 to 48) |
| Change From Baseline at Week 52 | 16.50 (-17 to 50) |
Collected over From the beginning of the initial evaluation period (Day1) up to 4.5 years. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Ravulizumab | 4/58 (6.9%) | 38/58 (65.5%) | 56/58 (96.6%) |
| Event | Ravulizumab |
|---|---|
| PneumoniaInfections and infestations | 5/58 |
| HypertensionVascular disorders | 3/58 |
| End stage renal diseaseRenal and urinary disorders | 3/58 |
| Septic shockInfections and infestations | 2/58 |
| Urinary tract infectionInfections and infestations | 2/58 |
| Acute kidney injuryRenal and urinary disorders | 2/58 |
| HydronephrosisRenal and urinary disorders | 2/58 |
| Atypical haemolytic uraemic syndromeBlood and lymphatic system disorders | 2/58 |
| Malignant hypertensionVascular disorders | 2/58 |
| Hypertensive crisisVascular disorders | 2/58 |
| Event | Ravulizumab |
|---|---|
| HeadacheNervous system disorders | 25/58 |
| DiarrhoeaGastrointestinal disorders | 21/58 |
| VomitingGastrointestinal disorders | 17/58 |
| NauseaGastrointestinal disorders | 17/58 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 16/58 |
| PyrexiaGeneral disorders | 13/58 |
| HypertensionVascular disorders | 13/58 |
| Urinary tract infectionInfections and infestations | 12/58 |
| CoughRespiratory, thoracic and mediastinal disorders | 12/58 |
| Oedema peripheralGeneral disorders | 11/58 |
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.
| Age, Continuous(years) | Ravulizumab |
|---|---|
| Mean | 42.2 ± 14.98 |
| Sex: Female, Male(Participants) | Ravulizumab |
|---|---|
| Female | 37 |
| Male | 19 |
| Ethnicity (NIH/OMB)(Participants) | Ravulizumab |
|---|---|
| Hispanic or Latino | 3 |
| Not Hispanic or Latino | 41 |
| Unknown or Not Reported | 12 |
| Race (NIH/OMB)(Participants) | Ravulizumab |
|---|---|
| American Indian or Alaska Native | 1 |
| Asian | 15 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 2 |
| White | 29 |
| More than one race | 0 |
| Unknown or Not Reported | 9 |
PDFs hosted by the registry, ClinicalTrials.gov.
Ran from Jan 2017 to primary completion in Jan 2023, 72 months.
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Studies of the drug tested here: Ravulizumab
Atypical Hemolytic Uremic Syndrome →
Alexion Pharmaceuticals, Inc. →