Ravulizumab in Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplant

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Ravulizumab in Adult and Adolescent Participants who have Thrombotic Microangiopathy (TMA) after Hematopoietic Stem Cell Transplant (HSCT)

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

JPRN

Registry ID

JPRN-jRCT2071200071

Enrollment

184

Registered

2020-12-21

Start date

2020-12-17

Completion date

Unknown

Last updated

2026-05-25

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 30 to 40, 1200mg at Day1, 300mg at at Day5 and Day10 and 2700mg at every 8 weeks after Day15 In cae Patient Body Weight (kg) is 40 to 60, 2400mg at Day1, 600mg at at

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1.12 years of age or older at time of consent/assent. 2.Received HSCT within the past 12 months. 3.Diagnosis of TMA that persists for at least 72 hours despite initial management. 4.A TMA diagnosis based on meeting the select criteria during the Screening Period and/or /=30 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. 7.Human immunodeficiency virus (HIV) infection. 8.Unresolved meningococcal disease. 9.Presence of sepsis requiring vasopressor support. 10.Pregnancy or breastfeeding. 11.Previously or currently treated with a complement inhibitor. 12.Respiratory failure requiring mechanical ventilation. 13.Acute and/or chronic heart failure. 14.Participation in an interventional treatment study of any therapy for TMA.

Design outcomes

Primary

Measure	Time frame
TMA Response [ Time Frame: 26 weeks ]	—

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.Loss of TMA Response [ Time Frame: 26 weeks (treatment period)] 3. Improvement in Organ Dysfunction[Time Frame: eGFR at 26wks (tx period) & 52wks (includes tx period & off-tx f/up period); TMA-associated organ dysfunction in renal, cardiovascular, pulmonary, CNS & GI systems through 26wks (tx period) & 52wks (includes tx period & off-tx f/up period) ] 4.TMA Relapse[ Time Frame:During the Follow-up Period ] 5. Overall Survival [ Time Frame: 26 weeks (treatment period) and 52 weeks (includes treatment period and off-treatment follow-up period) ] 6.Non-relapse Mortality [ Time Frame: 26 weeks (treatment period) and 52 weeks (includes treatment period and off-treatment follow-up period) ] 7.Platelet Response [ Time Frame: 26 weeks (treatment period) and 52 weeks (includes treatment period and off-treatment follow-up period)] 8.Hematologic Response [ Time Frame: 26 weeks (treatment period) and 52 weeks (includes treatment period and off-treatment follow-up period)]	—

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.Loss of TMA Response [ Time Frame: 26 weeks (treatment period)] 3. Improvement in Organ Dysfunction[Time Frame: eGFR at 26wks (tx period) & 52wks (includes tx period & off-tx f/up period); TMA-associated organ dysfunction in renal, cardiovascular, pulmonary, CNS & GI systems through 26wks (tx period) & 52wks (includes tx period & off-tx f/up period) ] 4.TMA Relapse[ Time Frame:During the Follow-up Period ] 5. Overall Survival [ Time Frame: 26 weeks (treatment period) and 52 weeks (includes treatment period and off-treatment follow-up period) ] 6.Non-relapse Mortality [ Time Frame: 26 weeks (treatment period) and 52 weeks (includes treatment period and off-treatment follow-up period) ] 7.Platelet Response [ Time Frame: 26 weeks (treatment period) and 52 weeks (includes treatment period and off-treatment follow-up period)] 8.Hematologic Response [ Time Frame: 26 weeks (treatment period) and 52 weeks (includes treatment period and off-treatment follow-up period)]

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Countries

Belgium, Canada, France, Italy, Japan, North America, South Korea, Spain

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: May 29, 2026