Study of Ravulizumab in Pediatric Participants With HSCT-TMA

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

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

JPRN

Registry ID

JPRN-jRCT2071200070

Enrollment

Registered

2020-12-21

Start date

2020-12-11

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Thrombotic Microangiopathy (TMA) after Hematopoietic Stem Cell Transplantation (HSCT) Thrombotic Microangiopathy TMA

Interventions

The ravulizumab will be administered via IV infusion as below dosage regimen

In cae Patient Body Weight (kg) is 5 to 10, 600mg at Day1, 300mg at at Day5 and Day10 and 300mg at every 4 weeks after Day15 In cae Patient Body Weight (kg) is 10 to 20, 600mg at Day1, 300mg at at Day

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. = or >28 days of age up to 5 kilograms at Screening. 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. 8. Participants or their legally authorized representative must be capable of giving signed informed consent or assent.

Exclusion criteria

Exclusion criteria: 1. Thrombotic thrombocytopenic purpura (TTP) evidenced by ADAMTS13 deficiency. 2. Shiga toxin producing Escherichia coli infection. 3. Positive direct Coombs test 4. Clinical diagnosis of disseminated intravascular coagulation (DIC) 5. Known bone marrow/graft failure. 6. Diagnosis of veno-occlusive disease (VOD), regardless of severity. 7. Human immunodeficiency virus (HIV) infection 8. Unresolved meningococcal disease. 9. Presence or suspicion of sepsis (treated or untreated). 10. Pregnancy or breastfeeding. 11. Respiratory failure requiring mechanical ventilation 12. Previously or currently treated with a complement inhibitor 13. Participation in an interventional treatment study of any therapy for TMA

Design outcomes

Primary

Measure	Time frame
TMA Response [ Time Frame: 26 weeks (treatment period) ]	—

Secondary

Measure	Time frame
1.Time to TMA response [ Time Frame: 26 weeks (treatment period) and 52 weeks (includes treatment period and off-treatment follow-up period) ] 2.TMA Relapse [ Time Frame: Follow-up period) ] 3.Overall Survival [ Time Frame: 26 weeks and 52 weeks] 4. Hematologic response [ Time Frame: 26 weeks and 52 weeks ]	—

Countries

France, Israel, Italy, Japan, North America, South Korea, Spain, United Kingdom

Contacts

Public ContactYuko Sugita

Alexion Pharma GK

JPDept-DevOps-PMCO@alexion.com+81-3-3457-9559

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026