臨床研究等提出・公開システム

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)

Study of Ravulizumab in Pediatric Participants With HSCT-TMA

Sugita Yuko

Alexion Pharma GK

3-1-1, Shibaura, Minato-ku, Tokyo 108-0023, Japan

JPDept-DevOps-PMCO@alexion.com

Sugita Yuko

Alexion Pharma GK

3-1-1, Shibaura, Minato-ku, Tokyo 108-0023, Japan

+81-3-3457-9559

JPDept-DevOps-PMCO@alexion.com

Not Recruiting

Dec. 11, 2020

Interventional

single arm study

open(masking not used)

uncontrolled control

single assignment

treatment purpose

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.

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

Both

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

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

Thrombotic Microangiopathy TMA

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

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 ]

+81-92-691-3881

Sept. 23, 2020

Yes

Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.

North America/France/Italy/Spain/Israel/ United Kingdom/South Korea