A Study of Nipocalimab in Reducing the Risk of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT)

Official title

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

Overview

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).

Interventions

• Drug Nipocalimab
  Nipocalimab will be administered intravenously.
• Drug Placebo
  Placebo will be administered intravenously.

Primary outcome measures

• 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]

Secondary outcome measures (12)

• Neonate/Fetus With Adjudicated Bleeding [Time frame: Up to 1 Week post birth]
• Platelet Count at Birth in a Neonate [Time frame: At birth]
• Neonate/Fetus with Outcome of Death [Time frame: Up to 1 Week post birth]
• Platelet Count at Birth <10×10^9/L in a Neonate [Time frame: At birth]
• Platelet Count at Birth <30×10^9/ L In a Neonate [Time frame: At birth]
• Platelet Count at Birth <50×10^9/L In a Neonate [Time frame: At birth]
• Platelet Count at Birth <150×10^9/L In a Neonate [Time frame: At birth]
• Nadir Platelet Count of a Neonate Over the First Week Post Birth [Time frame: Upto 1 Week post birth]
• Neonate/Fetus Requiring Platelet Transfusion(s) [Time frame: Up to 1 Week post birth]
• Number of Platelet Transfusion(s) in Neonate/Fetus [Time frame: Up to 1 Week post birth]
• Number of Donor Exposures for Platelet Transfusion(s) in Neonate/Fetus [Time frame: Up to 1 Week post birth]
• Neonate/Fetus With Adjudicated Severe Bleeding [Time frame: Up to 1 week post birth]

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 a clinical review of the 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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

Randomized

Model

Parallel assignment

Masking

Double blind

Primary purpose

Treatment

Study locations

Norway · 4 centers

• Haukeland University Hospital — Bergen
• Oslo University Hospital HF Ulleval sykehus — Oslo
• Universitetssykehuset Nord-Norge HF — Tromsø
• St. Olavs Hospital — Trondheim

Brazil · 3 centers

• Instituto de Medicina Integral Professor Fernando Figueira — Recife
• Instituto D Or de Pesquisa e Ensino IDOR — Rio de Janeiro
• Hospital Das Clinicas Da Faculdade De Medicina Da USP — São Paulo

Slovakia · 3 centers

• Univerzitna nemocnica L. Pasteura Kosice — Košice
• Univerzitná nemocnica Martin — Martin
• Fakultna nemocnica s poliklinikou Nove Zamky — Nové Zámky

France · 2 centers

• CHRU Lille — Lille
• Hopital trousseau- APHP — Paris

Italy · 2 centers

• Mangiagalli Clinic IRCCS Ca Granda Foundation Ospedale Maggiore Policlinico — Milan
• Fondazione Policlinico Universitario A Gemelli IRCCS — Rome

Belgium · 1 center

• Universitair Ziekenhuis Leuven — Leuven

Hungary · 1 center

• Semmelweis Egyetem — Budapest

Israel · 1 center

• Sheba Medical Center — Ramat Gan

Slovenia · 1 center

• Univerzitetni klinicni center Ljubljana — Ljubljana

Spain · 1 center

• Hosp. Virgen Del Rocio — Seville

Sweden · 1 center

• Karolinska Universitetssjukhuset Huddinge — Stockholm

Switzerland · 1 center

• Centre Hospitalier Universitaire Vaudois CHUV — Lausanne

Publications

• Tiller H, Tiblad E, Baker P, Van Valkenburgh H, Heerwegh D, Keshinro B. Design of a Phase 3, Multicenter, Randomized, Placebo-Controlled, Double-Blind Study of Nipocalimab in Pregnancies at Risk for Fetal and Neonatal Alloimmune Thrombocytopenia. Am J Perinatol. 2026 Apr;43(5):648-656. doi: 10.1055/a-2666-5642. Epub 2025 Jul 28. PMID 40720970

Identifiers

NCT: NCT06449651 · 80202135FNAIT3001 · 80202135FNAIT3001 · 2023-504307-88-00

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗