Clinical trial NCT06449651: 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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Thrombocytopenia, Neonatal Alloimmune
Lead sponsor: Janssen Research & Development, LLC
Sponsor class: INDUSTRY
First posted: 2024-06-10
Enrollment: 39
Sex: FEMALE
Minimum age (years): 18
Maximum age (years): 45
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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).


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

Primary outcomes:
measure: Fetus/Neonate with Outcome of Death or Adjudicated Severe Bleeding or Platelet Count Less Than (<) 30*10^9/L
timeFrame: Up to 1 week post birth
Secondary outcomes:
measure: Neonate/Fetus With Adjudicated Bleeding
timeFrame: Up to 1 Week post birth
measure: Platelet Count at Birth in a Neonate
timeFrame: At birth
measure: Neonate/Fetus with Outcome of Death
timeFrame: Up to 1 Week post birth
measure: Platelet Count at Birth <10×10^9/L in a Neonate
timeFrame: At birth
measure: Platelet Count at Birth <30×10^9/ L In a Neonate
timeFrame: At birth
measure: Platelet Count at Birth <50×10^9/L In a Neonate
timeFrame: At birth
measure: Platelet Count at Birth <150×10^9/L In a Neonate
timeFrame: At birth
measure: Nadir Platelet Count of a Neonate Over the First Week Post Birth
timeFrame: Upto 1 Week post birth
measure: Neonate/Fetus Requiring Platelet Transfusion(s)
timeFrame: Up to 1 Week post birth
measure: Number of Platelet Transfusion(s) in Neonate/Fetus
timeFrame: Up to 1 Week post birth
measure: Number of Donor Exposures for Platelet Transfusion(s) in Neonate/Fetus
timeFrame: Up to 1 Week post birth
measure: Neonate/Fetus With Adjudicated Severe Bleeding
timeFrame: Up to 1 week post birth
measure: Neonates With Postnatal Intravenous Immunoglobulin (IVIG) for The Treatment of Thrombocytopenia
timeFrame: Up to 1 Week post birth
measure: Maternal Participants With Treatment-Emergent Adverse Event (TEAE)
timeFrame: From randomization up to 24 weeks postpartum
measure: Maternal Participants With Serious Adverse Event (SAE)
timeFrame: From randomization up to 24 weeks postpartum
measure: Maternal Participants With Adverse Event of Special Interest (AESI)
timeFrame: From randomization up to 24 weeks postpartum
measure: Maternal Participants with TEAE Leading to Discontinuation of Study Intervention
timeFrame: Up to Week 104
measure: Neonate/Infant with TEAE
timeFrame: Up to Week 104
measure: Neonate/Infant with SAE
timeFrame: Up to Week 104
measure: Neonate/Infant with AESI
timeFrame: Up to Week 104
measure: Fetus/Neonate With TEAE of Bleeding
timeFrame: Up to Week 104
measure: Neonate With TEAE of Infection
timeFrame: Up to Week 104
measure: Infant Development as Measured by Bayley Scales at Week 52 and Week 104
timeFrame: At Week 52 and 104
measure: Maternal Participants With Antibodies to Nipocalimab Including Neutralizing Antibodies in Maternal Serum During Pregnancy and Postpartum
timeFrame: Up to Week 24
Other outcomes:
Locations:
city: Leuven
country: Belgium
facility: Universitair Ziekenhuis Leuven
city: Recife
country: Brazil
facility: Instituto de Medicina Integral Professor Fernando Figueira
city: Rio de Janeiro
country: Brazil
facility: Instituto D Or de Pesquisa e Ensino IDOR
city: Sao Paulo
country: Brazil
facility: Hospital Das Clinicas Da Faculdade De Medicina Da USP
city: Lille
country: France
facility: CHRU Lille
city: Paris
country: France
facility: Hopital trousseau- APHP
city: Budapest
country: Hungary
facility: Semmelweis Egyetem
city: Ramat Gan
country: Israel
facility: Sheba Medical Center
city: Rome
country: Italy
facility: Fondazione Policlinico Universitario A Gemelli IRCCS
city: Bergen
country: Norway
facility: Haukeland University Hospital
city: Oslo
country: Norway
facility: Oslo University Hospital HF Ulleval sykehus
city: Tromso
country: Norway
facility: Universitetssykehuset Nord-Norge HF
city: Trondheim
country: Norway
facility: St. Olavs Hospital
city: Kosice
country: Slovakia
facility: Univerzitna nemocnica L. Pasteura Kosice
city: Martin
country: Slovakia
facility: Univerzitná nemocnica Martin
city: Nove Zamky
country: Slovakia
facility: Fakultna nemocnica s poliklinikou Nove Zamky
city: Ljubljana
country: Slovenia
facility: Univerzitetni klinicni center Ljubljana
city: Sevilla
country: Spain
facility: Hosp. Virgen Del Rocio
city: Stockholm
country: Sweden
facility: Karolinska Universitetssjukhuset Huddinge
city: Lausanne
country: Switzerland
facility: Centre Hospitalier Universitaire Vaudois CHUV
Study officials:
affiliation: Janssen Research & Development, LLC
name: Janssen Research & Development, LLC Clinical Trial
role: STUDY_DIRECTOR
References: