This study will investigate the efficacy and safety of ravulizumab compared to placebo in adult participants with thrombotic microangiopathy (TMA) associated with a trigger. Participants will be randomized to receive either ravulizumab plus best supportive care or placebo plus best supportive care. The treatment period is 26 weeks followed by a 26-week off-treatment follow-up period.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
16
Body weight-based doses of ravulizumab will be administered intravenously as loading dose regimen followed by maintenance dosing every 8 weeks.
Matching placebo
Participants will receive medications, therapies, and interventions per standard hospital treatment protocols (unless specifically prohibited by the protocol).
Number of Participants With Complete Thrombotic Microangiopathy (TMA) Response at Week 26
TMA response required the following: 1) Normalization of platelet count without transfusion support during the prior 7 days. 2) Normalization of LDH. 3) Improvement in glomerular filtration rate (eGFR) of \>= 30% compared to baseline. Participants must meet each TMA criterion at 2 separate assessments obtained at least 24 hours apart, and any measurement in between.
Time frame: Week 26
Time to Complete TMA Response
The Kaplan-Meier estimate of time to event of complete TMA response is reported. TMA response required the following: 1) Normalization of platelet count without transfusion support during the prior 7 days. 2) Normalization of LDH. 3) Improvement in eGFR of \>= 30% compared to baseline. Participants must meet each TMA criterion at 2 separate assessments obtained at least 24 hours apart, and any measurement in between. Participants who did not have a response were censored at the date of last visit or study discontinuation at the time when the analysis was performed.
Time frame: Baseline through Week 26
Number of Participants With Hematologic Response at Week 26
Hematologic response required the following: (1) Normalization of platelet count without transfusion support during the prior 7 days, and (2) normalization of LDH.
Time frame: Week 26
Number of Participants With Renal Response at Week 26
Renal response is improvement in eGFR of \>= 30% compared to baseline. If a participant is on dialysis ≤5 days prior to the date of eGFR assessment, the eGFR will be set to 10 milliliter/minute/1.73 meter square (mL/min/1.73 m\^2) for that assessment. If a participant is on dialysis during the entire 26 week randomized Treatment Period, or through early discontinuation of study drug, then the change in eGFR was not calculated.
Time frame: Week 26
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Research Site
Tucson, Arizona, United States
Research Site
Orange, California, United States
Research Site
Washington D.C., District of Columbia, United States
Research Site
Gainesville, Florida, United States
Research Site
Lexington, Kentucky, United States
Research Site
Louisville, Kentucky, United States
Research Site
Boston, Massachusetts, United States
Research Site
Boston, Massachusetts, United States
Research Site
Boston, Massachusetts, United States
Research Site
Rochester, Minnesota, United States
...and 87 more locations
Number of Participants On Dialysis at Week 26
Time frame: Week 26
Change From Baseline in eGFR at Week 26
If a participant is on dialysis during the entire 26 week randomized Treatment Period, or through early discontinuation of study drug, then the change in eGFR was not calculated.
Time frame: Baseline, Week 26