thrombotic microangiopathy associated with a trigger MedDRA version: 20.0 Level: PT Classification code 10043645 Term: Thrombotic microangiopathy System Organ Class: 10005329 - Blood and lymphatic system disorders MedDRA version: 20.0 Level: PT Classification code 10062198 Term: Microangiopathy System Organ Class: 10047065 - Vascular disorders
Conditions
Interventions
Trade Name: Ultomiris
Product Name: ravulizumab
Product Code: ALXN1210
Pharmaceutical Form: Concentrate for solution for injection
INN or Proposed INN: RAVULIZUMAB
CAS Number: 1803171-55-2
Current S
Sponsors
Alexion Pharmaceuticals, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion 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 (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 Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 79 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 21
Exclusion criteria
Exclusion criteria: 1.Any known gene mutation that causes aHUS 2.Postpartum aHUS 3.Known CKD with eGFR = 45 mL/min/1.73 m2 by CKD-EPI equation (Levey, 2009) 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the efficacy of ravulizumab versus placebo ;Secondary Objective: To assess Safety and tolerability of ravulizumab and additional efficacy measures;Primary end point(s): Complete TMA response ;Timepoint(s) of evaluation of this end point: throughout week 26 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Time to complete TMA repsonse 2. Hematologic response 3. Renal response 4. TMA response duration and TMA relapse 5. Change in kidney function ;Timepoint(s) of evaluation of this end point: Week 26 and Week 52 | — |
Countries
Belgium, Canada, France, Germany, Italy, Japan, Korea, Republic of, Netherlands, Spain, Taiwan, United Kingdom, United States
Contacts
Public ContactEuropean Clinical Trial Information
Alexion Europe SAS
Outcome results
None listed