Double-blind, Randomized, Placebo-controlled Study Evaluating the Safety and Efficacy of Nipocalimab in Reducing the Risk of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT) in At-risk Pregnancies

Janssen Research & Development, LLC41 sites in 12 countries39 target enrollmentNovember 11, 2024

ConditionsThrombocytopenia, Neonatal Alloimmune

InterventionsNipocalimab Placebo

DrugsNipocalimab Placebo

Overview

Phase: Phase 3
Intervention: Nipocalimab
Conditions: Thrombocytopenia, Neonatal Alloimmune
Sponsor: Janssen Research & Development, LLC
Enrollment: 39
Locations: 41
Primary Endpoint: Fetus/Neonate with Outcome of Death or Adjudicated Severe Bleeding or Platelet Count Less Than (<) 30*10^9/L
Status: Recruiting
Last Updated: 19 days ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

The purpose of this study is to evaluate the effectiveness of nipocalimab compared with placebo in reducing the risk of severe fetal and neonatal alloimmune thrombocytopenia (FNAIT).

Registry

clinicaltrials.gov

Start Date

November 11, 2024

End Date

December 5, 2029

Last Updated

19 days ago

Study Type

Interventional

Study Design

Parallel

Sex

Female

Investigators

Sponsor

Janssen Research & Development, LLC

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Pregnant and an estimated gestational age (GA; based on ultrasound dating) from Week 13 to 18 at randomization
•Has a history of greater than or equal to (\>=) 1 prior pregnancy with fetal and neonatal alloimmune thrombocytopenia (FNAIT) (including neonatal platelet count less than (\<) 150×10\^9/Liter) with none of them affected by fetal/neonatal intracranial hemorrhage (ICH) or severe hemorrhage based on medical records
•Current pregnancy with presence of maternal anti- human platelet antigen (HPA)-1a alloantibody and positive fetal HPA-1a genotype as confirmed by cell-free fetal deoxyribonucleic acid (DNA) in maternal blood
•Health status considered stable by the investigator based on physical examination, medical history, vital signs, 12-lead Electrocardiogram (ECG), and clinical laboratory tests performed at screening
•For maternal participant and neonate/infant, willing to forego participation in another clinical study of an investigational therapy until the last follow-up visit

Exclusion Criteria

•Currently pregnant with multiple gestations (twins or more)
•History of severe preeclampsia in a previous pregnancy
•History of myocardial infarction, unstable ischemic heart disease, or stroke
•Known allergies, hypersensitivity, or intolerance to nipocalimab or its excipients (refer to the Investigator Brochure (IB))
•Has any confirmed or suspected clinical immunodeficiency syndrome or has a family history of congenital or hereditary immunodeficiency unless confirmed absent in the participant

Arms & Interventions

Nipocalimab

Maternal participants will receive nipocalimab Intravenously (IV).

Intervention: Nipocalimab

Placebo

Maternal participants will receive placebo IV.

Intervention: Placebo

Outcomes

Primary Outcomes

Fetus/Neonate with Outcome of Death or Adjudicated Severe Bleeding or Platelet Count Less Than (<) 30*10^9/L

Time Frame: Up to 1 week post birth

Outcome of fetus/neonate death or adjudicated severe bleeding up to the first week post birth or platelet count \<30\*10\^9/L will be reported.

Secondary Outcomes

Neonate/Fetus With Adjudicated Bleeding(Up to 1 Week post birth)
Platelet Count at Birth in a Neonate(At birth)
Neonate/Fetus with Outcome of Death(Up to 1 Week post birth)
Platelet Count at Birth <10×10^9/L in a Neonate(At birth)
Number of Platelet Transfusion(s) in Neonate/Fetus(Up to 1 Week post birth)
Platelet Count at Birth <30×10^9/ L In a Neonate(At birth)
Platelet Count at Birth <50×10^9/L In a Neonate(At birth)
Platelet Count at Birth <150×10^9/L In a Neonate(At birth)
Nadir Platelet Count of a Neonate Over the First Week Post Birth(Upto 1 Week post birth)
Neonate/Fetus Requiring Platelet Transfusion(s)(Up to 1 Week post birth)
Number of Donor Exposures for Platelet Transfusion(s) in Neonate/Fetus(Up to 1 Week post birth)
Neonate/Fetus With Adjudicated Severe Bleeding(Up to 1 week post birth)
Neonates With Postnatal Intravenous Immunoglobulin (IVIG) for The Treatment of Thrombocytopenia(Up to 1 Week post birth)
Maternal Participants With Treatment-Emergent Adverse Event (TEAE)(From randomization up to 24 weeks postpartum)
Maternal Participants With Serious Adverse Event (SAE)(From randomization up to 24 weeks postpartum)
Maternal Participants With Adverse Event of Special Interest (AESI)(From randomization up to 24 weeks postpartum)
Maternal Participants with TEAE Leading to Discontinuation of Study Intervention(Up to Week 104)
Neonate/Infant with TEAE(Up to Week 104)
Neonate/Infant with SAE(Up to Week 104)
Neonate/Infant with AESI(Up to Week 104)
Fetus/Neonate With TEAE of Bleeding(Up to Week 104)
Neonate With TEAE of Infection(Up to Week 104)
Infant Development as Measured by Bayley Scales at Week 52 and Week 104(At Week 52 and 104)
Maternal Participants With Antibodies to Nipocalimab Including Neutralizing Antibodies in Maternal Serum During Pregnancy and Postpartum(Up to Week 24)