Nipocalimab for Hemolytic Disease of the Fetus and Newborn

(AZALEA Trial)

This trial tests whether nipocalimab (an anti-FcRn monoclonal antibody) can prevent fetal anemia (low red blood cell count in a developing baby) in pregnant women at high risk for severe hemolytic disease of the fetus and newborn. Participants will receive either nipocalimab or a placebo to evaluate the treatment's effectiveness. Pregnant women who have experienced fetal anemia or related conditions in past pregnancies and have specific antibodies in their blood during this pregnancy may qualify for this study. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to potentially groundbreaking treatment advancements.

The trial does not specify if you need to stop taking your current medications, but you cannot participate if you are currently taking systemic corticosteroids or other immunosuppressants for conditions unrelated to the pregnancy.

Research has shown that nipocalimab is generally well-tolerated. Administered through an IV (intravenous) treatment, it has been tested in pregnant women and their babies. These studies focused on safety and found promising results, with no major safety concerns reported.

In earlier studies, participants did not experience significant side effects, indicating that serious issues were uncommon. Nipocalimab has received "Breakthrough Therapy Designation" from the FDA, highlighting its potential for conditions like severe hemolytic disease of the fetus and newborn, where new options are needed.

Nipocalimab is unique because it offers a new approach to treating Hemolytic Disease of the Fetus and Newborn (HDFN). Unlike traditional treatments that primarily focus on managing symptoms, nipocalimab targets the underlying cause by blocking the activity of antibodies responsible for the disease. This mechanism of action is different from current options like intrauterine transfusions, which temporarily alleviate symptoms. Researchers are excited about nipocalimab because it has the potential to provide a more effective and lasting solution by addressing the root of the problem, potentially reducing the need for invasive procedures.

Research has shown that nipocalimab, which participants in this trial may receive, may help delay or prevent anemia in unborn babies. Studies have found that nipocalimab can reduce the need for blood transfusions given to the baby before birth. In earlier trials, 54% of participants who received nipocalimab had a live birth, which is encouraging for those at risk of severe hemolytic disease of the fetus and newborn, a condition where the baby's red blood cells break down too quickly. These findings suggest that nipocalimab could effectively reduce risks associated with this condition.¹²⁵⁶⁷