Fetal and neonatal alloimmune thrombocytopenia (FNAIT) is the focus of a new international clinical trial that could change how doctors treat pregnancies at risk. Patients are currently being recruited for the trial.
Researchers are testing whether the drug nipocalimab can reduce life-threatening complications in babies, such as severe bleeding, very low platelet counts, or even death. Current standard treatment uses intravenous immunoglobulin (IVIG), but questions remain about safety, cost,and effectiveness.
This study, sponsored by Janssen Research & Development, opened earlier this year and is expected to finish in late 2029. Recruitment is underway at hospitals in the United States, including the Hospital of the University of Pennsylvania in Philadelphia, as well as in Austria, Germany, Poland and the United Kingdom. About 50 pregnant women are expected to enroll.
Participants are randomly assigned to one of two treatment arms. In the first, mothers with certain antibodies linked to FNAIT receive nipocalimab intravenously starting between weeks 13 and 18 of pregnancy. In the second, mothers are treated with IVIG, beginning as early as week 12 for high-risk pregnancies or week 20 for standard-risk cases. Prednisone may also be used alongside IVIG, with tapering after delivery depending on tolerance.
The trial’s main outcome will measure whether newborns die, experience major bleeding, or are born with platelet levels below 30 billion per liter in the first week of life. Secondary measures include platelet counts at birth, need for transfusions, and developmental progress using standardized infant testing. Researchers will also track adverse events in mothers and babies, including infections and other complications.
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For patients and families, the study offers hope of advancing care for a rare but serious condition. FNAIT occurs when a mother’s immune system makes antibodies against her baby’s platelets, leading to dangerously low platelet levels. Even when pregnancies appear healthy, affected newborns can face sudden bleeding problems, including brain hemorrhage.
If nipocalimab proves safer or more effective than IVIG, it could expand treatment options and improve outcomes for mothers and babies. Until results are available, experts stress the importance of early testing and enrollment in clinical trials for families affected by FNAIT.
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