A Phase 3, Randomized, Double-blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Ravulizumab in Adult Participants Who Have Thrombotic Microangiopathy Associated With a Trigger
- Conditions
- thrombotic microangiopathy associated with a triggerTMA with a trigger10064477
- Registration Number
- NL-OMON51930
- Lead Sponsor
- PPD
- Brief Summary
Trial never started
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Withdrawn
- Sex
- Not specified
- Target Recruitment
- 4
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.TMA (platelet count, LDH, and acute kidney injury) associated with a trigger
such as autoimmune, solid organ transplant or drug induced
5.Vaccinated against meningococcal infection (N 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
1.Any known gene mutation that causes aHUS
2.Postpartum aHUS
3.Known CKD
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.Known 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.Clinical diagnosis of disseminated intravascular coagulation (DIC)
11.Presence of sepsis
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
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Complete TMA Response during the 26-week randomized<br /><br>Treatment Period</p><br>
- Secondary Outcome Measures
Name Time Method