Ravulizumab in Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplant

JRCT ID: jRCT2071200071

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: 17/12/2020
• Target sample size: 184
• Countries of recruitment: North America,Japan,France,Japan,Italy,Japan,Belgium,Japan,South Korea,Japan,Spain,Japan,Canada,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 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 ]

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.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)]

Key inclusion & exclusion criteria

• Age minimum: >= 12age old
• Age maximum: Not applicable
• Gender: Both

Include 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 </=14 days prior to the Screening Period.
5.Body weight >/=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.

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.
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.

Related Information

• Primary Sponsor: Sugita Yuko
• Secondary ID(s): NCT04543591