Fetal and neonatal alloimmune thrombocytopenia (FNAIT) is an extraordinarily rare disease, and as is the case for most rare diseases, there is still much that the scientific community does not understand about it, from the mechanisms underpinning the disorder to the treatments that work best to slow disease progression.
Clinical research in the field of FNAIT is ongoing, and current and future trials show promise in helping clinicians understand the disease in a deeper way, potentially opening the door to significant therapeutic breakthroughs in the future. Here are some clinical trials that are currently being carried out that may help clinicians further unravel the scientific mysteries of FNAIT.
Investigating the Merits of Nipocalimab
An ongoing trial (NCT06449651) that is expected to end in 2029 is assessing the use of the experimental therapy nipocalimab in patients with at least one prior pregnancy affected by FNAIT.
An estimated 39 participants will either receive nipocalimab intravenously or a placebo. The main outcome measure is fetal/newborn death, or the presence of severe bleeding up to the first week after birth, or a platelet count (platelets being necessary to control bleeding) under 30 x 109/L, which is extremely low for a human child.
This study has a wide international scope, including countries across South America and Europe.
Another similar study (NCT06533098) also assesses the use of nipocalimab in FNAIT, but it does so by comparing its efficacy and safety with that of intravenous immunoglobulin (IVIG), which is already a standard therapy used in treating FNAIT. The main outcome measures are identical to that of the study above.
This study aims to recruit patients with at least one prior pregnancy affected by FNAIT, with an estimated gestational age between 13 and 16 weeks upon the first visit. This study is conducted in Birmingham, UK.
Assessing Occurence of HPA-1a Alloimmunization
This study (NCT05345561), conducted across 27 locations across the globe and has an estimated completion date of 2027, intends to assess if FNAIT risk is affected by race and ethnicity. By gaining a better understanding of the global spread of FNAIT risk, healthcare authorities are better able to allocate adequate resources to ensure that this disorder is dealt with equitably.
The primary outcome measures of this study is the frequency of anti-human platelet antigen (HPA)-1a alloimmunization, which is associated with FNAIT risk. In addition, researchers are interested in pregnancy outcomes, such as live births, premature births, or abortions. They will also seek to keep track of the occurrence of neonatal thrombocytopenia among the study cohort.
Exploring the Safety of RLYB212
An ongoing study (NCT06435845) seeks to assess the efficacy and safety of RLYB212, which is an experimental therapy that is administered subcutaneously. It is a human monoclonal anti-HPa-1a immunoglobulin G antibody that can potentially be used to treat FNAIT.
There are two primary outcomes in this study. The first is to quantify the number of participants who experience treatment-related adverse events as a result of the administration of RLYB212; this data will help inform clinicians about the relative safety of this therapy. The second is exposure to RLYB212 as measured in the mother’s blood; this will help clinicians understand how much of this therapy remains in the blood instead of being metabolized.
In addition, researchers are interested in a number of secondary outcome measures. This include newborn exposure to RLYB212 as measured in the umbilical cord blood, pregnancy outcomes, and overall newborn health and well-being four to six weeks after delivery.
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