Study of Ravulizumab in Pediatric Participants With HSCT-TMA

JRCT ID: jRCT2071200070

Registered date:21/12/2020

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Thrombotic Microangiopathy (TMA) after Hematopoietic Stem Cell Transplantation (HSCT)
• Date of first enrollment: 11/12/2020
• Target sample size: 40
• Countries of recruitment: North America,Japan,France,Japan,Italy,Japan,Spain,Japan,Israel,Japan,United Kingdom,Japan,South Korea,Japan
• Study type: Interventional

Intervention(s)

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 Day5 and Day10 and 600mg at every 4 weeks after Day15
In cae Patient Body Weight (kg) is 20 to 30, 900mg at Day1, 300mg at at Day5 and Day10 and 2100mg at every 8 weeks after Day15
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 Day5 and Day10 and 3000mg at every 8 weeks after Day15
In cae Patient Body Weight (kg) is 60 to 100, 2700mg at Day1, 900mg at at Day5, Day10 and 3300mg at every 8 weeks after Day15
In cae Patient Body Weight (kg) is over 100, 3000mg at Day1, 900mg at at Day5, Day10 and 3600mg at every 8 weeks after Day15

Outcome(s)

• Primary Outcome: TMA Response [ Time Frame: 26 weeks (treatment period) ]

Secondary Outcome

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 ]

Key inclusion & exclusion criteria

• Age minimum: >= 4weeks old
• Age maximum: < 18age old
• Gender: Both

Include criteria

1. = or >28 days of age up to <18 years 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 despite initial management.
4. A TMA diagnosis based on meeting the select criteria during the Screening Period and/or = or <14 days prior to the Screening Period.
5. Body weight = or > 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.

Exclude 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

Related Information

• Primary Sponsor: Sugita Yuko
• Secondary ID(s): NCT04557735