Trial · NCT04543591
Ravulizumab in Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplant
NCT04543591, Phase 3, Completed, Alexion Pharmaceuticals, Inc.
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Ravulizumab in Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplant. Trials, record NCT04543591. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04543591
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Ravulizumab in Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplant
- NCT number
- NCT04543591
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 148 participants, actual
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Conditions
- Thrombotic Microangiopathy
- Interventions
- Ravulizumab, Placebo, Best supportive care
- First posted
- 2020-09-10
- Last updated
- 2026-11-04
- Other registries' IDs
- eudract:2020-000144-61
Source ClinicalTrials.gov
Registered outcomes25
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Stage 2: Proportion of Participants With Event-free Survival During the 26-week Treatment Period | Up to Week 26 | Event-free survival was defined as the time from randomization until the first of the 2 following events: death (due to any cause) and clinical worsening up through Week 26. Clinical worsening was defined as meeting 2 or more of the following criteria: doubling of serum creatinine from baseline or new requirement of dialysis; increase in lactate dehydrogenase (LDH) \>25% compared to baseline; reduction in platelets of \>25% compared to baseline; reduction in hemoglobin \>10% compared to baseline; increased requirement for platelets or red blood cells at any time. The criteria for clinical worsening had to be confirmed by 2 independent samples collected at least 8 hours apart (ideally 24 hours apart). |
| Secondary | Stage 2: Overall Survival | Up to Day 100 (Treatment Period), Week 26 (Treatment Period), and Week 52 (Follow-up Period) | The Kaplan-Meier method was used to measure overall survival estimate. For Day 100 and Week 26, overall survival was calculated from randomization to death (due to any cause), up to Day 100/Week 26. Participants who survived were censored at the earliest of an additional hematopoietic stem cell transplant (HSCT), Day 100/Week 26, or their last known date alive. For Week 52, overall survival was calculated from randomization to death (due to any cause), up to Week 52. Participants who survived were censored at the earlier of an additional HSCT, or their last known date alive up to Week 52. |
| Secondary | Stage 2: Non-relapse Mortality | Baseline through Day 100 (Treatment Period), Week 26 (Treatment Period), and Week 52 (Follow-up Period) | Cumulative incidence for each group was estimated using a competing risks 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 (malignant or non-malignant reason for HSCT) progression or relapse. |
| Secondary | Stage 2: Mean Number of Thrombotic Microangiopathy (TMA) Response Criteria Met During the 26-week Treatment Period | Baseline through Week 26 | The TMA response parameters included: platelet response, normalization of LDH and absence of schistocytes, and renal response. A participant could have met 0, 1, 2, or 3 TMA response criteria during the 26-week Treatment Period. If the participants met more than 1 TMA response criterion, the number of responses overlapping for at least 24 hours was counted. Least squares mean determined from a general linear model adjusted by acute GVHD status and geographic region, assuming normal data distribution. |
| Secondary | Stage 2: Percentage of Participants With Hematologic Response During the 26-week Treatment Period | Baseline through Week 26 | Clinical laboratory assessments were performed according to the protocol and assessed against the following predetermined criteria for hematologic response: if baseline platelet count was ≤50000/cubic millimeter (mm\^3), then the absolute platelet count had to be \>50000/mm\^3 without platelet transfusion support during the prior 7 days; if the baseline platelet count had to be \>50000/mm\^3, then there must be a ≥50% increase in platelet count compared to baseline value without platelet transfusion support during the prior 7 days; normalization of LDH and absence of schistocytes (defined as ≤1% or a result of "none", per the International Council for Standardization in Hematology recommendations for schistocyte counting). |
| Secondary | Stage 2: Change From Baseline in the Estimated Glomerular Filtration Rate (eGFR) at Week 26 and Week 52 | Baseline, Week 26 (Treatment Period), Week 52 (Follow-up Period) | The eGFR was calculated based on serum creatinine using the Chronic Kidney Disease Epidemiology Collaboration (CKD EPI) formula. Serum creatinine was collected according to the protocol. Data reported as milliliters/minute/1.73 square meters (mL/min/1.73 m\^2). |
| Secondary | Stage 2: Percentage of Participants With TMA Response During the 26-week Treatment Period | Baseline through Week 26 | The criteria for TMA response included a hematological response and a renal response. Hematological Response: if baseline platelet count was ≤50000/mm\^3, then the absolute platelet count had to be \>50000/mm\^3 without platelet transfusion support during the prior 7 days; if the baseline platelet count was \>50000/mm\^3, then there had to be a ≥50% increase in platelet count compared to baseline value without platelet transfusion support during the prior 7 days; normalization of LDH and absence of schistocytes. Renal response: at least 50% reduction of proteinuria from baseline. Participants must have met each of the response criteria, with each parameter meeting the requirements at 2 separate assessments obtained at least 24 hours apart, and any measurement in between. A participant could meet 0, 1, 2, or 3 TMA response criteria during the treatment period. If the participant met more than 1 TMA response criterion, the number of responses overlapping for at least 24 hours was counted. |
| Secondary | Stage 2: Cumulative Proportion of Participants Who Experienced a Complete TMA Response During the 26-week Treatment Period | Baseline through Week 26 | Cumulative proportion of participants who experienced a complete TMA response was estimated using a competing risks model based on the time (days) to complete TMA response. Time to complete TMA response was defined as the days from randomization to the first day during the 26-week Treatment Period on which a participant met all criteria for a complete TMA response. The date of complete TMA response was defined as the first date on which all individual response criteria were met simultaneously. |
| Secondary | Stage 2: Cumulative Proportion of Participants Experiencing Each Individual Component of TMA During the 26-week Treatment Period | Baseline through Week 26 | Cumulative proportion of participants experiencing individual TMA components included those for platelet count, LDH, and protein/creatinine ratio. For platelet count: if baseline platelet count was ≤50000/mm\^3, then the absolute platelet count had to be \>50000/mm\^3 without platelet transfusion support during the prior 7 days; if the baseline platelet count was \>50000/mm\^3, then there had to be a ≥50% increase in platelet count compared to baseline value without platelet transfusion support during the prior 7 days. For LDH: normalization of LDH and absence of schistocytes. For protein/creatinine ratio: at least 50% reduction of proteinuria from baseline. |
| Secondary | Stage 2: Cumulative Proportion of Participants Experiencing a Hematologic Response During the 26-week Treatment Period | Baseline through Week 26 | Clinical laboratory assessments were performed according to the protocol and assessed against the following predetermined criteria for hematologic response: if baseline platelet count was ≤50000/mm\^3, then the absolute platelet count had to be \>50000/mm\^3 without platelet transfusion support during the prior 7 days; if the baseline platelet count was \>50000/mm\^3, then there had to be a ≥50% increase in platelet count compared to baseline value without platelet transfusion support during the prior 7 days. |
1 to 10 of 25Source ClinicalTrials.gov
See all 25 registered outcomesOutcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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 comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
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
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The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04543591
GET https://conductscience.org/api/trials/v1/NCT04543591/registered?page=2