Phase 3 Study of Ravulizumab in Thrombotic Microangiopathy Associated With a Trigger

JRCT ID: jRCT2031210494

Registered date:17/12/2021

Basic Information

• Recruitment status: Suspended
• Health condition(s) or Problem(s) studied: thrombotic microangiopathy (TMA) associated with a trigger
• Date of first enrollment: 18/01/2022
• Target sample size: 100
• Countries of recruitment: US,Japan,Spain,Japan,Korea,Japan,Belgium,Japan,France,Japan,Italy,Japan,UK,Japan,Taiwan,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 and 2700mg at every 8 weeks after Day15
In cae Patient Body Weight (kg) is 40 to 60, 2400mg at Day1 and 3000mg at every 8 weeks after Day15
In cae Patient Body Weight (kg) is 60 to 100, 2700mg at Day1 and 3300mg at every 8 weeks after Day15
In cae Patient Body Weight (kg) is over 100, 3000mg at Day1 and 3600mg at every 8 weeks after Day15

Outcome(s)

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

Secondary Outcome

1.Time to Complete TMA Response [ Time Frame: 26 weeks (treatment period) ]
2.Hematologic Response [ Time Frame: 26 weeks (treatment period) and 52 weeks (includes treatment period and off-treatment follow-up ]
3.Renal Response [ Time Frame: 26 weeks (treatment period) and 52 weeks (includes treatment period and off-treatment follow-up ]
4.TMA Response Duration [ Time Frame: 26 weeks (treatment period) and 52 weeks (includes treatment period and off-treatment follow-up ]
5.Change in Kidney Function as measured by estimated glomerular filtration rate (eGFR) in mL/min/1.73m^2 [ Time Frame: 26 weeks (treatment period) and 52 weeks (includes treatment period and off-treatment follow-up ]

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

1.18 years of age or older
2.Body weight >= 30 kilograms
3.Female participants of childbearing potential and male participants with female partners of childbearing potential must use highly effective contraception starting at screening and continuing until at least 8 months after the last dose of ravulizumab
4.Diagnosis of TMA associated with a trigger
5.Vaccinated against meningococcal infection (Neisseria meningitidis), within 3 years prior to, or at the time of, randomization. Participants who initiate study drug treatment less than 2 weeks after receiving a meningococcal vaccine must receive appropriate prophylactic antibiotics for at least 2 weeks after the vaccination. If participant cannot receive the meningococcal vaccine, then participant must be willing to receive antibiotic prophylaxis coverage against N. meningitidis during the entire Treatment Period and for 8 months following the final dose of study drug. Additional vaccination (Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae) may be considered based on individual patient condition.

Exclude criteria

1.Any known gene mutation that causes atypical hemolytic uremic syndrome (aHUS)
2.Postpartum aHUS
3.Known chronic kidney disease with estimated glomerular filtration rate <= 45 mL/min/1.73 m2 due to any cause
4.TMA due to hematopoietic stem cell transplantation <= 12 months of Screening
5.Primary and secondary glomerular diseases other than lupus
6.Diagnosis of primary antiphospholipid antibody syndrome
7.Shiga toxin-producing Escherichia coli infections including but not limited to Shiga toxin-related hemolytic uremic syndrome
8.Known familial or acquired 'a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13' (ADAMTS13) deficiency (activity < 5%)
9.Positive direct Coombs test
10.Diagnosis of disseminated intravascular coagulation (DIC)
11.Presence of sepsis according within 7 days prior to or during Screening
12.Presence of monoclonal gammopathy including but not limited to multiple myeloma
13.Known bone marrow insufficiency or failure evidenced by cytopenias
14.Unresolved N. meningitidis infection
15.History of malignancy within 5 years of Screening with the exception of nonmelanoma skin cancer or carcinoma in situ of the cervix that has been treated with no evidence of recurrence
16.Use of any complement inhibitors within the past 3 years

Related Information

• Primary Sponsor: Yokosawa Jun
• Secondary ID(s): NCT04743804