Nipocalimab + IVIG for Neonatal Alloimmune Thrombocytopenia

(FREESIA-3 Trial)

Recruiting at 19 trial locations

Overseen By Study Contact

Age: 18 - 65

Sex: Female

Trial Phase: Phase 3

Sponsor: Janssen Research & Development, LLC

Must not be taking: IVIG, Prednisone

Disqualifiers: Multiple gestations, Severe preeclampsia, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: Up to delivery

50 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the effectiveness and safety of nipocalimab in reducing the risk of severe fetal and neonatal alloimmune thrombocytopenia (FNAIT), a condition where a mother's antibodies attack the baby's platelets, which help with clotting. The study compares nipocalimab to intravenous immunoglobulins (IVIG), with or without the steroid medication prednisone. It targets pregnant women who have had a previous pregnancy affected by FNAIT and possess specific antibodies that could harm their current pregnancy. Participants should be between 13 and 18 weeks pregnant and maintain a stable health status. 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.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the trial team or your doctor.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that nipocalimab has been safe in earlier studies. One study demonstrated that it helped delay or prevent issues like fetal anemia without causing major side effects, suggesting it might be well-tolerated by pregnant individuals.

For intravenous immunoglobulins (IVIG), research indicates they are generally safe and often used to prevent serious pregnancy issues, such as low platelet counts that can cause bleeding. IVIG is a standard treatment with a good safety record for both mothers and babies.

Both nipocalimab and IVIG have shown positive safety results in studies, providing some confidence in their use. However, ongoing studies aim to further confirm these findings. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Unlike the standard of care for neonatal alloimmune thrombocytopenia, which often involves administering intravenous immunoglobulins (IVIG) and steroids, nipocalimab offers a fresh approach by targeting a different mechanism. Nipocalimab works by blocking the FcRn receptor, which plays a key role in preventing harmful antibodies from crossing the placenta and attacking fetal platelets. This targeted action could potentially offer a more effective and safer alternative for managing this condition during pregnancy. Researchers are excited about nipocalimab because it might reduce the need for steroids, which can have significant side effects, and offer a more streamlined treatment for high-risk pregnancies.

What evidence suggests that this trial's treatments could be effective for neonatal alloimmune thrombocytopenia?

This trial will compare Nipocalimab and Intravenous Immunoglobulins (IVIG) for managing neonatal alloimmune thrombocytopenia. Studies have shown that Nipocalimab, which participants in this trial may receive, can help prevent or delay issues like fetal anemia, a condition where the baby lacks enough healthy red blood cells. This is crucial for making pregnancy safer and improving outcomes for the baby. Research indicates that Nipocalimab works by blocking a specific receptor, reducing certain risks during pregnancy.

IVIG, another treatment option in this trial, has shown positive results in many pregnancies with fetal and neonatal alloimmune thrombocytopenia, where the baby's platelet count is low due to the mother's antibodies. In some studies, IVIG treatment led to a median platelet count at birth of 172,000 per microliter, considered a strong response. Both treatments have shown promise in managing these conditions, but their effectiveness can vary from case to case. ^{3 6 7 8 9}

Who Is on the Research Team?

Janssen Research & Development, LLC Clinical Trial

Principal Investigator

Janssen Research & Development, LLC

Are You a Good Fit for This Trial?

This trial is for pregnant women between weeks 13 to 16 with a history of FNAIT in previous pregnancies. They must have specific antibodies and their fetus must have the corresponding genotype. Participants should be healthy as determined by medical exams and agree not to join other studies until this one ends.

Inclusion Criteria

My health is stable as confirmed by recent medical exams and tests.

I am pregnant and between 13 to 16 weeks along.

I am pregnant and have specific antibodies that could affect my baby.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Maternal participants receive either nipocalimab or IVIG starting at gestational age week 12 to 20 until before delivery

Up to delivery

Follow-up

Participants are monitored for safety and effectiveness after treatment, including maternal and neonatal outcomes

Up to 104 weeks post birth

What Are the Treatments Tested in This Trial?

Interventions

• Nipocalimab
• Prednisone

Trial Overview The study is testing nipocalimab's effectiveness compared to IVIG in preventing severe FNAIT, which affects platelets in newborns. It involves administering these treatments during pregnancy and observing outcomes.

How Is the Trial Designed?

Group I: Arm 2: Intravenous Immunoglobins (IVIG) Experimental Treatment 2 Interventions

Maternal participants with alloantibodies against HPA-1a and/or HPA-5b will be randomized to receive IVIG from GA week 12 for high-risk pregnancies or GA week 20 for standard-risk pregnancies. Additionally, prednisone will be added per study protocol. Participants will be gradually tapered off prednisone after delivery as per investigator judgement or maternal participant tolerance.

Group II: Arm 1: Nipocalimab Experimental Treatment 1 Intervention

Maternal participants with alloantibodies against HPA-1a and/or HPA-5b will be randomized to receive nipocalimab starting at gestational age (GA) week 13 to 18 until before delivery.

Who Is Running the Clinical Trial?

Janssen Research & Development, LLC

Lead Sponsor

Trials

1,022

Recruited

6,408,000+

Joaquin Duato

Janssen Research & Development, LLC

Chief Executive Officer since 2022

MBA from ESADE, Master of International Management from Thunderbird School of Global Management

Dr. Jijo James, MD

Janssen Research & Development, LLC

Chief Medical Officer since 2014

MD from St. Johns Medical College, MPH from Columbia University

Citations

1. obgyn.onlinelibrary.wiley.com obgyn.onlinelibrary.wiley.com/doi/10.1002/uog.70105

Effectiveness of antenatal intravenous immunoglobulin ... This study demonstrates that a successful response to IVIG treatment in one pregnancy with FNAIT does not guarantee the same outcome in ...

2. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/41097884/

Effectiveness of antenatal intravenous immunoglobulin ... Among the IVIG-treated pregnancies, 100/121 (82.6%) exhibited a good response to IVIG, with a median platelet count at birth of 172 000/μL, ...

3. ajog.org ajog.org/article/S0002-9378(22)00325-8/fulltext

Antenatal intravenous immunoglobulins in pregnancies at ... Neonatal outcome in alloimmune thrombocytopenia after maternal treatment with intravenous immunoglobulin. Blood Transfus. 2015; 13:66-71.

4. ashpublications.org ashpublications.org/blood/article/124/4/654/33312/Prediction-of-IVIG-treatment-efficiency-in-fetal

Prediction of IVIG treatment efficiency in fetal/neonatal ... Prediction of IVIG treatment efficiency in fetal/neonatal alloimmune thrombocytopenia Available ; <23 IU/mL, 5, 30 ; ≥23 IU/mL, 16, 8 ; Gestation = ...

5. sciencedirect.com sciencedirect.com/science/article/pii/S0002937822004380

Long-term neurodevelopmental outcome in children after ... Intravenous immunoglobulin seems effective in vastly reducing the risk of fetal or neonatal bleeding complications. However, information on long-term ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC4317092/

Neonatal outcome in alloimmune thrombocytopenia after ... Our results suggest that antenatal maternal IVIG and, if necessary, postnatal matched platelet transfusions, are effective and safe for the treatment of FNAIT.

7. sciencedirect.com sciencedirect.com/science/article/abs/pii/S1473050219302836

The use of IVIg in fetal and neonatal alloimmune ... Up to 20 % of FNAIT neonates [1] are affected by ICH and about 30 % of cases are fatal [5]. Non-fatal cases can be affected by life-long sequela including ...

8. obgyn.onlinelibrary.wiley.com obgyn.onlinelibrary.wiley.com/doi/10.1002/pd.6854

Role of Biologics in Fetal Hematologic Conditions: HDFN and ... IVIG is the current standard of care to prevent fetal/neonatal thrombocytopenia and intracranial hemorrhage in cases of platelet ...

9. clinicaltrials.gov clinicaltrials.gov/study/NCT06533098

NCT06533098 | A Study of Nipocalimab or Intravenous ... The purpose of this study is to assess the efficacy and safety of nipocalimab in reducing the risk of severe fetal and neonatal alloimmune thrombocytopenia ...