Study of Ravulizumab in Pediatric Participants With HSCT-TMA
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|ClinicalTrials.gov Identifier: NCT04557735|
Recruitment Status : Recruiting
First Posted : September 22, 2020
Last Update Posted : May 6, 2023
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|Condition or disease||Intervention/treatment||Phase|
|Thrombotic Microangiopathy||Drug: Ravulizumab Other: Best Supportive Care||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||40 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||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)|
|Actual Study Start Date :||November 6, 2020|
|Estimated Primary Completion Date :||June 28, 2024|
|Estimated Study Completion Date :||August 11, 2025|
Experimental: Ravulizumab plus Best Supportive Care
Participants will receive ravulizumab plus Best Supportive Care as background therapy.
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 Name: Ultomiris
Other: Best Supportive Care
Participants will receive medications, therapies, and interventions per standard hospital treatment protocols (unless specifically prohibited by the protocol).
- TMA Response [ Time Frame: 26 weeks (treatment period) ]
- Time To TMA Response [ Time Frame: 26 weeks (treatment period) and through 52 weeks (includes treatment period and off-treatment follow- up period) ]
- TMA Relapse [ Time Frame: Follow Up period (183-365 Days after start of study medication) ]
- Overall Survival [ Time Frame: 26 weeks and 52 weeks ]
- Hematologic Response [ Time Frame: 26 weeks and 52 weeks ]
Hematologic Response as assessed by blood tests to measure lactate dehydrogenase (LDH) and platelet count.
If baseline platelet count ≤ 50,000/mm3, all of the following criteria must be met:
- Absolute platelet count > 50,000/mm3 without platelet transfusion support during the prior 7 days [or]
If baseline platelet count > 50,000/mm3, all of the following criteria must be met:
- ≥ 50% increase in platelet count compared to baseline value
- Normalization of LDH and absence of schistocytes
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
|Ages Eligible for Study:||28 Days to 17 Years (Child)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- ≥ 28 days of age up to < 18 years of age at the time of signing the informed consent.
- Received HSCT within the past 12 months.
- Diagnosis of TMA that persists for at least 72 hours despite initial management.
- A TMA diagnosis based on meeting the select criteria during the Screening Period and/or <=14 days prior to the Screening Period.
- Body weight ≥ 5 kilograms at Screening or ≤7 days prior to the start of the Screening Period (date of consent).
- Female participants of childbearing potential and male participants with female partners of childbearing potential must use highly effective contraception.
- Participants must be vaccinated against meningococcal infections if clinically feasible. Participants who cannot receive meningococcal vaccine should receive antibiotic prophylaxis.
- Participants or their legally authorized representative must be capable of giving signed informed consent or assent
- Thrombotic thrombocytopenic purpura (TTP) evidenced by ADAMTS13 deficiency.
- Shiga toxin producing Escherichia coli infection.
- Positive direct Coombs test
- Clinical diagnosis of disseminated intravascular coagulation (DIC)
- Known bone marrow/graft failure.
- Diagnosis of veno-occlusive disease (VOD), regardless of severity.
- Human immunodeficiency virus (HIV) infection
- Unresolved meningococcal disease.
- Presence or suspicion of sepsis (treated or untreated).
- Pregnancy or breastfeeding.
- Respiratory failure requiring mechanical ventilation
- Previously or currently treated with a complement inhibitor
- Participation in an interventional treatment study of any therapy for TMA
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04557735
|Contact: Alexion Pharmaceuticals Inc.||firstname.lastname@example.org|
|Other Study ID Numbers:||
2020-000761-16 ( EudraCT Number )
|First Posted:||September 22, 2020 Key Record Dates|
|Last Update Posted:||May 6, 2023|
|Last Verified:||May 2023|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||No|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
Thrombotic Microangiopathy (TMA)
Hematopoietic Stem Cell Transplant
Blood Platelet Disorders
Complement Inactivating Agents
Physiological Effects of Drugs