Study of Ravulizumab in Pediatric Participants With HSCT-TMA
- Conditions
- Thrombotic Microangiopathy (TMA) after Hematopoietic Stem Cell Transplantation (HSCT)Thrombotic Microangiopathy TMA
- Registration Number
- JPRN-jRCT2071200070
- Lead Sponsor
- Yokosawa Jun
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 40
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
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method TMA Response [ Time Frame: 26 weeks (treatment period) ]
- Secondary Outcome Measures
Name Time Method 1.Time to TMA response [ Time Frame: 26 weeks (treatment period) and 52 weeks (includes treatment period and off-treatment follow-up period) ] <br>2.TMA Relapse [ Time Frame: Follow-up period) ]<br>3.Overall Survival [ Time Frame: 26 weeks and 52 weeks]<br>4. Hematologic response [ Time Frame: 26 weeks and 52 weeks ]