Study of Ravulizumab in Pediatric Participants With HSCT-TMA

December 23, 2025 updated by: Alexion Pharmaceuticals, Inc.

A Phase 3, Open-label, Single Arm, Multicenter Study of Ravulizumab in Addition to Best Supportive Care in Pediatric Participants With Thrombotic Microangiopathy (TMA) After Hematopoietic Stem Cell Transplantation (HSCT)

This study will evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of ravulizumab administered by intravenous infusion to pediatric participants, from 1 month to < 18 years of age, with HSCT-TMA. The treatment period is 26 weeks, followed by a 26-week off-treatment follow-up period.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
41

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • Israel
      • Haifa, Israel, 91096
        • Research Site
      • Jerusalem, Israel, 91120
        • Research Site
      • Petah Tikva, Israel, 4920235
        • Research Site
      • Ramat Gan, Israel, 5265601
        • Research Site
  • Italy
      • Roma, Italy, 00165
        • Research Site
  • Japan
      • Fukushima, Japan, 960-1295
        • Research Site
      • Kobe, Japan, 650-0047
        • Research Site
      • Nagoya, Japan, 466-8560
        • Research Site
      • Osaka, Japan, 534-0021
        • Research Site
  • South Korea
      • Seoul, South Korea, 03080
        • Research Site
      • Seoul, South Korea, 5505
        • Research Site
  • Spain
      • Barcelona, Spain, 08041
        • Research Site
      • Esplugues de Llobregat, Spain, 8950
        • Research Site
      • Madrid, Spain, 28046
        • Research Site
      • Salamanca, Spain, 37007
        • Research Site
  • United Kingdom
      • Birmingham, United Kingdom, B4 6NH
        • Research Site
      • Bristol, United Kingdom, BS2 8BJ
        • Research Site
      • Newcastle upon Tyne, United Kingdom, NE1 4LP
        • Research Site
  • United States
    • Arizona
      • Tucson, Arizona, United States, 85724
        • Research Site
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Research Site
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Research Site
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Research Site
    • Oregon
      • Portland, Oregon, United States, 97239
        • Research Site
    • Texas
      • Dallas, Texas, United States, 75235
        • Research Site
    • Utah
      • Salt Lake City, Utah, United States, 84108
        • Research Site
    • Wisconsin
      • Madison, Wisconsin, United States, 53792
        • Research Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

3 years to 13 years (Child)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. ≥ 28 days of age up to < 18 years of age at the time of signing the informed consent.
  2. Received HSCT within the past 12 months.
  3. Diagnosis of TMA that persists for at least 72 hours after initial management of any triggering agent/condition.
  4. A TMA diagnosis based on meeting the laboratory-based criteria during the Screening Period and/or ≤14 days prior to the Screening Period.
  5. Body weight ≥ 5 kilograms at Screening or ≤7 days prior to the start of the Screening Period (date of consent).
  6. Female participants of childbearing potential and male participants with female partners of childbearing potential must use highly effective contraception.
  7. Participants must be vaccinated against meningococcal infections if clinically feasible. Participants who cannot receive meningococcal vaccine should receive antibiotic prophylaxis. Participants <18 years of age must be re-vaccinated against Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae if clinically feasible.
  8. Participants or their legally authorized representative must be capable of giving signed informed consent or assent.

Exclusion Criteria:

  1. Thrombotic thrombocytopenic purpura (TTP) evidenced by ADAMTS13 deficiency.
  2. Known Shiga toxin-related hemolytic uremic syndrome as demonstrated by positive test.
  3. Positive direct Coombs test indicative of a clinically significant immune-mediated hemolysis not due to TMA.
  4. Clinical diagnosis of disseminated intravascular coagulation (DIC).
  5. Known bone marrow/graft failure for the current HSCT.
  6. Diagnosis of veno-occlusive disease (VOD) which is unresolved at the time of Screening.
  7. Human immunodeficiency virus (HIV) infection.
  8. Unresolved meningococcal disease.
  9. Presence of sepsis requiring vasopressor support.
  10. Pregnancy or breastfeeding.
  11. Hypersensitivity to murine proteins or to 1 of the excipients of Ravulizumab.
  12. Any ongoing or history of medical or psychological conditions unrelated to HSCT-TMA that could increase the risk to the participant or confound the outcome of the study.
  13. Respiratory failure requiring mechanical ventilation.
  14. Previously or currently treated with a complement inhibitor.
  15. Participation in an interventional treatment study of any therapy for TMA.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Ravulizumab plus Best Supportive Care
Participants will receive ravulizumab plus Best Supportive Care as background therapy.
Other: Best Supportive Care
Participants will receive medications, therapies, and interventions per standard hospital treatment protocols (unless specifically prohibited by the protocol).
Drug: Ravulizumab
Weight-based doses of ravulizumab will be administered intravenously as a loading dose regimen followed by maintenance dosing every 4 or 8 weeks, depending upon weight.
Other Names:
  • Ultomiris

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Participants With Thrombotic Microangiopathy (TMA) Response
Time Frame: Up to Week 26
The criteria for TMA response were: 1. Normalization of platelet count (defined as platelet count ≥ 50000 mm^3 or >=50% increase in platelet count) without transfusion support during the prior 7 days. 2. Normalization of lactate dehydrogenase (LDH, defined as LDH ≤ upper limit of normal [ULN]) and absence of schistocytes. 3. At least 50% reduction in protein/creatinine ratio from baseline. Participants must meet each TMA criterion at 2 separate assessments obtained at least 24 hours apart, with no criteria failures or more than 1 missed scheduled visit in between. Additionally, all intervals in which the criteria were met must overlap for at least 1 day.
Up to Week 26

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Time to TMA Response During the 26-Week Treatment Period
Time Frame: Day 1 through Week 26
Time to TMA response was defined as the time from first infusion to the first time point at which all criteria for TMA response was met. Participants were assigned as responders at the time of their TMA response and were censored at the earlier of last assessment with all 3 TMA response components available (including measurements collected after treatment discontinuation), or death if they did not respond by then. TMA response required the following: 1. Normalization of platelet count (defined as platelet count ≥ 50000 mm^3 or >=50% increase in platelet count) without transfusion support during the prior 7 days. 2. Normalization of LDH (defined as LDH ≤ULN) and absence of schistocytes. 3. At least 50% reduction in protein/creatinine ratio from baseline.
Day 1 through Week 26
Participants With Hematologic Response
Time Frame: Up to Week 26
Hematologic response required the following: (1) Normalization of platelet count (defined as platelet count ≥ 50000 mm^3 or >=50% increase in platelet count) without transfusion support during the prior 7 days and (2) Normalization of LDH (defined as LDH ≤ULN) and absence of schistocytes.
Up to Week 26
Time to Hematologic Response During the 26-Week Treatment Period
Time Frame: Day 1 through Week 26
Time to Hematologic response was defined as the time from first infusion to the first time point at which all criteria for hematologic response was met. Participants were assigned as responders at the time of their response and were censored at their discontinuation time or at the end of available follow-up if they did not respond by then. Hematologic response required the following: (1) Normalization of platelet count (defined as platelet count ≥ 50000 mm^3 or >=50% increase in platelet count) without transfusion support during the prior 7 days and (2) Normalization of LDH (defined as LDH ≤ULN) and absence of schistocytes.
Day 1 through Week 26
Participants With Hemoglobin Response
Time Frame: Up to Week 26
Hemoglobin response was defined as the ability to maintain hemoglobin ≥ 10 g/dL without RBC transfusion support. The criterion must have been met at 2 separate assessments obtained at least 24 hours apart, and any measurement in between, and without RBC transfusion support during the prior 7 days.
Up to Week 26
Participants With Platelet Response
Time Frame: Up to Week 26
Normalization of platelet count was defined as baseline platelet count ≤ 50000 mm^3 or > 50000 mm^3, absolute platelet count > 50000 mm^3 or >=50% increase in platelet count without transfusion support during the prior 7 days.
Up to Week 26
Participants With Partial TMA Response
Time Frame: Up to Week 26
Partial response was defined as a participant meeting at least 1, but not all, criteria for TMA response. TMA response required the following: 1. Normalization of platelet count (defined as platelet count ≥ 50000 mm^3 or >=50% increase in platelet count) without transfusion support during the prior 7 days. 2. Normalization of LDH, (defined as LDH ≤ULN) and absence of schistocytes. 3. At least 50% reduction in protein/creatinine ratio from baseline.
Up to Week 26
Participants With Loss of TMA Response
Time Frame: Up to Week 26
Loss of response occurred when a participant who had previously achieved a TMA response failed to meet criteria for one or more components of TMA response at a subsequent visit in treatment period. At least one parameter must fail to meet response criteria at 2 separate assessments obtained at least 24 hours apart, and any measurement in between. TMA response required the following: 1. Normalization of platelet count (defined as platelet count ≥ 50000 mm^3 or >=50% increase in platelet count) without transfusion support during the prior 7 days. 2. Normalization of LDH (defined as LDH ≤ULN) and absence of schistocytes. 3. At least 50% reduction in protein/creatinine ratio from baseline.
Up to Week 26
Duration of TMA Response Through Week 52
Time Frame: Day 1 through Week 52
This analysis includes data for each participant after TMA response through final follow-up. Participants with TMA response who do not experience these events are censored at their end of study date. TMA response required following: 1. Normalization of platelet count (defined as platelet count ≥ 50000 mm^3 or >=50% increase in platelet count) without transfusion support during the prior 7 days. 2. Normalization of LDH (defined as LDH ≤ULN) and absence of schistocytes. 3. At least 50% reduction in protein/creatinine ratio from baseline. The estimate is calculated based on Kaplan-Meier method.
Day 1 through Week 52
Participants With TMA Relapse
Time Frame: Up to Week 52
For participants that meet criteria for TMA response during 26-week Treatment Period, TMA relapse is defined as evidence of worsening hematologic and renal dysfunction due to TMA during post-treatment Follow-up Period that requires treatment intervention, as determined by Investigator. TMA response required the following: 1. Normalization of platelet count (defined as platelet count ≥ 50000 mm^3 or >=50% increase in platelet count) without transfusion support during the prior 7 days. 2. Normalization of LDH (defined as LDH ≤ULN) and absence of schistocytes. 3. At least 50% reduction in protein/creatinine ratio from baseline.
Up to Week 52
Overall Survival
Time Frame: Day 1 through Week 52
The Kaplan-Meier method was used to measure overall survival estimate. Overall survival was calculated from date of treatment start to date of a documented death event (death due to any cause) or date of censoring. Participants who survived were censored at the earliest of an additional hematopoietic stem cell transplant (HSCT), Week 52, or their last known date alive.
Day 1 through Week 52
Non-relapse Mortality During the 52-Week Treatment Period
Time Frame: Day 1 through Week 52
Cumulative incidence was estimated using a competing risk 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 progression or relapse.
Day 1 through Week 52

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Alexion Pharmaceuticals, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
December 7, 2020

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
November 26, 2024

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
May 27, 2025

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
September 2, 2020

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 18, 2020

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
September 22, 2020

Study Record Updates

Last Update Posted (Estimated)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
January 12, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 23, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
December 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • ALXN1210-TMA-314
  • 2020-000761-16 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

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