Trial · NCT04743804
Ravulizumab in Thrombotic Microangiopathy Associated With a Trigger
NCT04743804, Phase 3, Terminated, Alexion Pharmaceuticals, Inc.; results first posted 2023-12-12
3 sourcestrials-2026-11-12-0.5.0
Download JSON
Cite this record
ConductScience Library. Ravulizumab in Thrombotic Microangiopathy Associated With a Trigger. Trials, record NCT04743804. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04743804
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Ravulizumab in Thrombotic Microangiopathy Associated With a Trigger
- NCT number
- NCT04743804
- Status
- Terminated
- Phases
- Phase 3
- Enrollment
- 16 participants, actual
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Conditions
- Thrombotic Microangiopathy
- Interventions
- Ravulizumab, Placebo, Best Supportive Care
- First posted
- 2021-02-08
- Last updated
- 2024-10-01
- Other registries' IDs
- eudract:2020-005328-13
- Results first posted
- 2023-12-12
- Limitations and caveats
Due to continued enrollment challenges, Alexion decided to terminate this study prematurely. There were no safety or efficacy concerns throughout the course of study. The planned interim analysis for sample size re-estimation and the planned primary analysis as specified in Protocol Amendment 2 were not conducted due to early termination of the study. No hypothesis testing or inferential statistical analysis for the purpose of treatment comparisons was performed due to small sample size.
Source ClinicalTrials.gov
Registered outcomes6
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Complete Thrombotic Microangiopathy (TMA) Response at Week 26 | Week 26 | TMA response required the following: 1) Normalization of platelet count without transfusion support during the prior 7 days. 2) Normalization of LDH. 3) Improvement in glomerular filtration rate (eGFR) of \>= 30% compared to baseline. Participants must meet each TMA criterion at 2 separate assessments obtained at least 24 hours apart, and any measurement in between. |
| Secondary | Time to Complete TMA Response | Baseline through Week 26 | The Kaplan-Meier estimate of time to event of complete TMA response is reported. TMA response required the following: 1) Normalization of platelet count without transfusion support during the prior 7 days. 2) Normalization of LDH. 3) Improvement in eGFR of \>= 30% compared to baseline. Participants must meet each TMA criterion at 2 separate assessments obtained at least 24 hours apart, and any measurement in between. Participants who did not have a response were censored at the date of last visit or study discontinuation at the time when the analysis was performed. |
| Secondary | Number of Participants With Hematologic Response at Week 26 | Week 26 | Hematologic response required the following: (1) Normalization of platelet count without transfusion support during the prior 7 days, and (2) normalization of LDH. |
| Secondary | Number of Participants With Renal Response at Week 26 | Week 26 | Renal response is improvement in eGFR of \>= 30% compared to baseline. If a participant is on dialysis ≤5 days prior to the date of eGFR assessment, the eGFR will be set to 10 milliliter/minute/1.73 meter square (mL/min/1.73 m\^2) for that assessment. If a participant is on dialysis during the entire 26 week randomized Treatment Period, or through early discontinuation of study drug, then the change in eGFR was not calculated. |
| Secondary | Number of Participants On Dialysis at Week 26 | Week 26 | |
| Secondary | Change From Baseline in eGFR at Week 26 | Baseline, Week 26 | If a participant is on dialysis during the entire 26 week randomized Treatment Period, or through early discontinuation of study drug, then the change in eGFR was not calculated. |
Source ClinicalTrials.gov
Outcomes6
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Complete Thrombotic Microangiopathy (TMA) Response at Week 26 | Week 26 | The Modified Intent-to-Treat Analysis Set included all randomized participants, excluding participants who enrolled prior to availability of ST-HUS and ADAMTS13 central laboratory results and were subsequently found to be ineligible after randomization. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Time to Complete TMA Response | Baseline through Week 26 | The Modified Intent-to-Treat Analysis Set included all randomized participants, excluding participants who enrolled prior to availability of ST-HUS and ADAMTS13 central laboratory results and were subsequently found to be ineligible after randomization. | days | MEDIAN |
| SECONDARY | Number of Participants With Hematologic Response at Week 26 | Week 26 | The Modified Intent-to-Treat Analysis Set included all randomized participants, excluding participants who enrolled prior to availability of ST-HUS and ADAMTS13 central laboratory results and were subsequently found to be ineligible after randomization. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Renal Response at Week 26 | Week 26 | The Modified Intent-to-Treat Analysis Set included all randomized participants, excluding participants who enrolled prior to availability of ST-HUS and ADAMTS13 central laboratory results and were subsequently found to be ineligible after randomization. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants On Dialysis at Week 26 | Week 26 | The Modified Intent-to-Treat Analysis Set included all randomized participants, excluding participants who enrolled prior to availability of ST-HUS and ADAMTS13 central laboratory results and were subsequently found to be ineligible after randomization. Here, Number of Participants analyzed signifies those who were evaluable for this outcome measure. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline in eGFR at Week 26 | Baseline, Week 26 | The Modified Intent-to-Treat Analysis Set included all randomized participants, excluding participants who enrolled prior to availability of ST-HUS and ADAMTS13 central laboratory results and were subsequently found to be ineligible after randomization. Here, Number of Participants analyzed signifies those who were evaluable for this outcome measure. | milliliter/minute/1.73 meter^2 | MEAN |
Source ClinicalTrials.gov
Results by group12
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Complete Thrombotic Microangiopathy (TMA) Response at Week 26 | Ravulizumab | 2 | Participants | ||
| Number of Participants With Complete Thrombotic Microangiopathy (TMA) Response at Week 26 | Placebo | 3 | Participants | ||
| Time to Complete TMA Response | Ravulizumab | NA | 95% Confidence Interval 21.0 to NA | days | |
| Time to Complete TMA Response | Placebo | NA | 95% Confidence Interval 16.0 to NA | days | |
| Number of Participants With Hematologic Response at Week 26 | Ravulizumab | 5 | Participants | ||
| Number of Participants With Hematologic Response at Week 26 | Placebo | 6 | Participants | ||
| Number of Participants With Renal Response at Week 26 | Ravulizumab | 4 | Participants | ||
| Number of Participants With Renal Response at Week 26 | Placebo | 4 | Participants | ||
| Number of Participants On Dialysis at Week 26 | Ravulizumab | 1 | Participants | ||
| Number of Participants On Dialysis at Week 26 | Placebo | 0 | Participants |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 results by groupParticipant flow8
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Ravulizumab | 9 | |
| Overall Study | STARTED | Placebo | 7 | |
| Overall Study | Received at Least 1 Dose of Study Drug | Ravulizumab | 9 | |
| Overall Study | Received at Least 1 Dose of Study Drug | Placebo | 7 | |
| Overall Study | COMPLETED | Ravulizumab | 4 | |
| Overall Study | COMPLETED | Placebo | 1 | |
| Overall Study | NOT COMPLETED | Ravulizumab | 5 | |
| Overall Study | NOT COMPLETED | Placebo | 6 |
Source ClinicalTrials.gov
Adverse events236
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 | Nausea | Gastrointestinal disorders | Ravulizumab | 2 | 9 | |
| other | Nausea | Gastrointestinal disorders | Placebo | 4 | 7 | |
| other | Vomiting | Gastrointestinal disorders | Ravulizumab | 2 | 9 | |
| other | Vomiting | Gastrointestinal disorders | Placebo | 3 | 7 | |
| other | Diarrhoea | Gastrointestinal disorders | Ravulizumab | 1 | 9 | |
| other | Diarrhoea | Gastrointestinal disorders | Placebo | 2 | 7 | |
| other | Abdominal discomfort | Gastrointestinal disorders | Ravulizumab | 0 | 9 | |
| other | Abdominal discomfort | Gastrointestinal disorders | Placebo | 1 | 7 | |
| other | Abdominal pain | Gastrointestinal disorders | Ravulizumab | 1 | 9 | |
| other | Abdominal pain | Gastrointestinal disorders | Placebo | 0 | 7 |
1 to 10 of 236Source ClinicalTrials.gov
See all 236 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Ravulizumab | 3 | 9 |
| Total, all-cause mortality | Placebo | 0 | 7 |
| Total, other adverse events | Ravulizumab | 9 | 9 |
| Total, other adverse events | Placebo | 7 | 7 |
| Total, serious adverse events | Ravulizumab | 6 | 9 |
| Total, serious adverse events | Placebo | 3 | 7 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 37094330 | DERIVED |
Source PubMed
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 comparisons6
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Ravulizumab vs Placebo: Number of Participants With Renal Response at Week 26 | Trial comparisons |
| Ravulizumab vs Placebo: Number of Participants On Dialysis at Week 26 | Trial comparisons |
| Ravulizumab vs Placebo: Number of Participants With Hematologic Response at Week 26 | Trial comparisons |
| Ravulizumab vs Placebo: Number of Participants With Complete Thrombotic Microangiopathy (TMA) Response at Week 26 | Trial comparisons |
| Ravulizumab vs Placebo: deaths from any cause | Trial comparisons |
| Ravulizumab vs Placebo: 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/NCT04743804
GET https://conductscience.org/api/trials/v1/NCT04743804/events?page=2