A Study of Nipocalimab or Intravenous Immunoglobulin (IVIG) in Pregnancies At Risk of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT) (FREESIA-3)

Multicenter, Open-Label, Randomized Study of Nipocalimab or IVIG in Pregnancies At Risk of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT)

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 (FNAIT).

Study Overview

• Status: Recruiting
• Conditions: Thrombocytopenia, Neonatal Alloimmune
• Intervention / Treatment: Drug: Nipocalimab; Drug: Prednisone; Drug: Intravenous immunoglobulins (IVIG)

• Study Type: Interventional
• Enrollment (Estimated): 50
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Study Contact; Phone Number: 844-434-4210; Email: Participate-In-This-Study1@its.jnj.com

Study Locations

• Austria
  • Graz, Austria, 8036
    • Recruiting
    • Medizinische Universitaet Graz
  • Vienna, Austria, 1090
    • Recruiting
    • Medical University Vienna
• Germany
  • Giessen, Germany, 35392
    • Recruiting
    • Universitaetsklinikum Giessen Und Marburg Gmbh
  • Hanover, Germany, 30625
    • Recruiting
    • Medizinische Hochschule Hannover
  • Jena, Germany, 07747
    • Recruiting
    • Universitatsklinikum Jena
  • Tübingen, Germany, 72076
    • Recruiting
    • Universitaetsklinikum Tuebingen
• Netherlands
  • Leiden, Netherlands, 2333 ZA
    • Recruiting
    • Leiden University Medical Center
• Poland
  • Lodz, Poland, 93 338
    • Recruiting
    • Instytut Centrum Zdrowia Matki Polki
  • Warsaw, Poland, 01 211
    • Recruiting
    • Instytut Matki i Dziecka
  • Warsaw, Poland, 02 507
    • Recruiting
    • Panstwowy Instytut Medyczny MSWiA w Warszawie
• United Kingdom
  • Birmingham, United Kingdom, B15 2TG
    • Recruiting
    • Birmingham Women's Hospital
  • Liverpool, United Kingdom, L8 7SS
    • Recruiting
    • Liverpool Women's NHS Foundation Trust - Liverpool Women's Hospital
  • London, United Kingdom, W12 0HS
    • Recruiting
    • Queen Charlotte's and Chelsea Hospital
  • Oxford, United Kingdom, OX3 9DU
    • Recruiting
    • John Radcliffe Hospital
• United States
  • Alabama
    • Birmingham, Alabama, United States, 35205
      • Recruiting
      • The University of Alabama at Birmingham
  • California
    • Sacramento, California, United States, 95817
      • Recruiting
      • UC Davis School of Medicine
  • Colorado
    • Aurora, Colorado, United States, 80045
      • Recruiting
      • Childrens Hospital Colorado
  • Illinois
    • Park Ridge, Illinois, United States, 60068
      • Recruiting
      • Advocate Children's Hospital
  • Iowa
    • Iowa City, Iowa, United States, 52242
      • Recruiting
      • University of Iowa Hospital and Clinics
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27599
      • Recruiting
      • University of North Carolina at Chapel Hill
  • Ohio
    • Cincinnati, Ohio, United States, 45267
      • Recruiting
      • University of Cincinnati
  • Oregon
    • Portland, Oregon, United States, 97239
      • Recruiting
      • Oregon Health and Science University
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Recruiting
      • Hospital of the University of Pennsylvania
  • Texas
    • Galveston, Texas, United States, 77555
      • Recruiting
      • University of Texas Medical Branch
  • Utah
    • Salt Lake City, Utah, United States, 84108
      • Recruiting
      • University of Utah

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Pregnant and an estimated gestational age (GA) from week 13* to 18 at visit 1

  *Randomization for high-risk FNAIT participants to occur at GA Week 12

• Has a history of greater than or equal to (>=) 1 prior pregnancy with FNAIT based on medical records including: a) neonatal platelet count less than (<) 150*10^9/Liter with no fetal/neonatal intracranial hemorrhage (ICH) or severe fetal/neonatal hemorrhage (standard-risk) OR b) fetus/neonate with ICH or severe hemorrhage in a fetus/neonate (high-risk)
• Current pregnancy with presence of maternal anti-HPA-1a and/or anti-HPA-5b alloantibody and positive fetal HPA-1a and/or HPA-5b genotype as confirmed by cell-free fetal 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, to IVIG or to prednisone
• Has any confirmed or suspected clinical immunodeficiency syndrome or has a family history of congenital or hereditary immunodeficiency unless confirmed absent in the participant

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Arm 1: Nipocalimab; 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.	Drug: Nipocalimab; Nipocalimab will be administered intravenously.; Other Names: JNJ-80202135; M281; JNJ-86507083
Experimental: Arm 2: Intravenous Immunoglobins (IVIG); 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.	Drug: Prednisone; Prednisone will be administered orally.; Drug: Intravenous immunoglobulins (IVIG); IVIG will be administered intravenously.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Fetus/Neonate with Outcome of Death or Adjudicated Severe Bleeding or Platelet Count Less Than (<) 30*10^9/L	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.	Up to 1 Week post birth

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Platelet Count at Birth in a Neonate	Platelet count at birth in a neonate will be reported.	At birth
Neonate/Fetus with Outcome of Death	Fetus/neonate with outcome of death will be reported.	Up to 1 Week post birth
Neonate with Platelet Count at Birth <10*10^9/L	Platelet count at birth <10*10^9/L in a neonate will be reported.	At birth
Neonate with Platelet Count at Birth <30*10^9/L	Platelet count at birth <30*10^9/L in a neonate will be reported.	At birth
Neonate with Platelet Count at Birth <50*10^9/L	Platelet count at birth <50*10^9/L in a neonate will be reported.	At birth
Neonate with Platelet Count at Birth <150*10^9/L	Platelet count at birth <150*10^9/L in a neonate will be reported.	At birth
Neonate Requiring Platelet Transfusion	Neonate who require at least one platelet transfusion will be reported.	Up to 1 Week post birth
Number of Platelet Transfusions in Neonate	Number of platelet transfusions per neonate will be reported.	Up to 1 Week post birth
Number of Donor Exposures for Platelet Transfusions in Neonate	Number of donor exposures for a neonate who received at least one platelet transfusion will be reported.	Up to 1 Week post birth
Neonate/Fetus With Adjudicated Bleeding	Neonate/Fetus with adjudicated bleeding will be reported.	Up to 1 Week post birth
Neonate Requiring Postnatal Intravenous Immunoglobulin (IVIG) for the Treatment of Thrombocytopenia	Neonate requiring IVIG for the treatment of thrombocytopenia will be reported.	Up to 1 Week post birth
Nadir Platelet Count in a Neonate	Nadir platelet count in a neonate will be reported.	Up to 1 Week post birth
Maternal Participant with Treatment-Emergent Adverse Events (TEAE), Serious Adverse Events (SAE) and Adverse Event of Special Interest (AESI)	Maternal participant with TEAE, SAE and AESI will be reported. An Adverse event (AE) is any untoward medical occurrence in a participant participating in a clinical study that does not necessarily have a causal relationship with the intervention under study.	Up to Week 24
Maternal Participant with TEAE Leading to Discontinuation of Study Intervention	Maternal participant with TEAE leading to discontinuation of study intervention will be reported.	Up to Week 24
Neonate/Infant With TEAE, SAE and AESI	Neonate/infant with TEAE, SAE and AESI will be reported. An AE is any untoward medical occurrence in a participant participating in a clinical study that does not necessarily have a causal relationship with the intervention under study.	From Day of birth to Week 104
Maternal Participants with Incidence of Antibodies to Nipocalimab	Incidence of antibodies to nipocalimab including neutralizing antibodies in maternal serum during pregnancy and postpartum will be reported.	Up to Week 4
Fetus/Neonate with a TEAE of Bleeding	Fetus/Neonate with a TEAE of bleeding will be reported.	From Day of birth to Week 104
Neonate with a TEAE of Infection	Neonate with a TEAE of infection will be reported.	From Day of birth to Week 104
Bayley Scales Assessment for Infant Development	The Bayley Scales of infant development is considered the standard assessment of early child development and includes cognition, language, motor skills, social emotional, and adaptive behavior will be reported. The Bayley Scales are reference standards that measure infant and toddler development in five areas: cognition, language, motor skills, social-emotional and adaptive behavior. The cognition, language and motor skills scales are directly administered to the infant, while social-emotional, and adaptive behavior scales are caregiver questionnaires. The scores are standardized using norm reference samples with representative demographics and age adjusted for prematurity. Higher scores in the Bayley Scales indicate better outcomes.	At Week 52 and Week 104

Collaborators and Investigators

• Sponsor: Janssen Research & Development, LLC
• Investigators: Study Director: Janssen Research & Development, LLC Clinical Trial, Janssen Research & Development, LLC

Publications and helpful links

General Publications

• Bussel J, Stegmann B, Baker P, Oey A, Jiang Y, Zaha R, Van Valkenburgh H, Keshinro B. Design of a Phase 3, Multicenter, Randomized, Open-Label Study of Nipocalimab or IVIG and Prednisone in Pregnancies at Risk for Fetal and Neonatal Alloimmune Thrombocytopenia. Am J Perinatol. 2025 Dec 12. doi: 10.1055/a-2753-9323. Online ahead of print.

Study record dates

Study Major Dates

• Study Start (Actual): February 10, 2025
• Primary Completion (Estimated): December 15, 2027
• Study Completion (Estimated): December 5, 2029

Study Registration Dates

• First Submitted: July 30, 2024
• First Submitted That Met QC Criteria: July 30, 2024
• First Posted (Actual): August 1, 2024

Study Record Updates

• Last Update Posted (Actual): May 27, 2026
• Last Update Submitted That Met QC Criteria: May 22, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Cytopenia; Infant, Newborn, Diseases; Hematologic Diseases; Blood Platelet Disorders; Thrombocytopenia; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hemic and Lymphatic Diseases; Thrombocytopenia, Neonatal Alloimmune; Amino Acids, Peptides, and Proteins; Proteins; Polycyclic Compounds; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Serum Globulins; Globulins; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Pregnadienediols; Immunoglobulin Isotypes; Immunoglobulin G; Prednisone; Immunoglobulins, Intravenous
• Other Study ID Numbers: 80202135FNAIT3003 (Janssen Research & Development, LLC); 2023-509434-19-00 (Registry Identifier: EUCT number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: The data sharing policy of Johnson & Johnson Innovative Medicine is available at innovativemedicine.jnj.com/our-innovation/clinical-trials/transparency. As noted on this site, requests for access to the study data can be submitted through Yale Open Data Access (YODA) Project site at yoda.yale.edu

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No