A Study of Nipocalimab or Intravenous Immunoglobulin (IVIG) in Pregnancies At Risk of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT)
- Conditions
- Thrombocytopenia, Neonatal Alloimmune
- Interventions
- Registration Number
- NCT06533098
- Lead Sponsor
- Janssen Research & Development, LLC
- Brief Summary
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).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- Female
- Target Recruitment
- 50
- Pregnant and an estimated gestational age from week 13 to 18 at visit 1
- 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
- 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 & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Arm 1: Nipocalimab 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. Arm 2: Intravenous Immunoglobins (IVIG) Intravenous immunoglobulins (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. Arm 2: Intravenous Immunoglobins (IVIG) Prednisone 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.
- Primary Outcome Measures
Name Time Method Fetus/Neonate with Outcome of Death or Adjudicated Severe Bleeding or Platelet Count Less Than (<) 30*10^9/L 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 Outcome Measures
Name Time Method Platelet Count at Birth in a Neonate At birth Platelet count at birth in a neonate will be reported.
Neonate/Fetus with Outcome of Death Up to 1 Week post birth Fetus/neonate with outcome of death will be reported.
Neonate with Platelet Count at Birth <10*10^9/L At birth Platelet count at birth \<10\*10\^9/L in a neonate will be reported.
Neonate with Platelet Count at Birth <30*10^9/L At birth Platelet count at birth \<30\*10\^9/L in a neonate will be reported.
Neonate with Platelet Count at Birth <50*10^9/L At birth Platelet count at birth \<50\*10\^9/L in a neonate will be reported.
Neonate with Platelet Count at Birth <150*10^9/L At birth Platelet count at birth \<150\*10\^9/L in a neonate will be reported.
Nadir Platelet Count in a Neonate Up to 1 Week post birth Nadir platelet count in a neonate will be reported.
Neonate Requiring Platelet Transfusion Up to 1 Week post birth Neonate who require at least one platelet transfusion will be reported.
Number of Platelet Transfusions in Neonate Up to 1 Week post birth Number of platelet transfusions per neonate will be reported.
Number of Donor Exposures for Platelet Transfusions in Neonate Up to 1 Week post birth Number of donor exposures for a neonate who received at least one platelet transfusion will be reported.
Neonate/Fetus With Adjudicated Bleeding Up to 1 Week post birth Neonate/Fetus with adjudicated bleeding will be reported.
Neonate Requiring Postnatal Intravenous Immunoglobulin (IVIG) for the Treatment of Thrombocytopenia Up to 1 Week post birth Neonate requiring IVIG for the treatment of thrombocytopenia will be reported.
Maternal Participant with TEAE Leading to Discontinuation of Study Intervention Up to Week 24 Maternal participant with TEAE leading to discontinuation of study intervention will be reported.
Neonate/Infant With TEAE, SAE and AESI From Day of birth to Week 104 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.
Fetus/Neonate with a TEAE of Bleeding From Day of birth to Week 104 Fetus/Neonate with a TEAE of bleeding will be reported.
Neonate with a TEAE of Infection From Day of birth to Week 104 Neonate with a TEAE of infection will be reported.
Maternal Participant with Treatment-Emergent Adverse Events (TEAE), Serious Adverse Events (SAE) and Adverse Event of Special Interest (AESI) Up to Week 24 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.
Bayley Scales Assessment for Infant Development At Week 52 and Week 104 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 (3rd edition) 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.
Maternal Participants with Incidence of Antibodies to Nipocalimab Up to Week 4 Incidence of antibodies to nipocalimab including neutralizing antibodies in maternal serum during pregnancy and postpartum will be reported.
Trial Locations
- Locations (12)
Universitaetsklinikum Tuebingen
🇩🇪Tuebingen, Germany
John Radcliffe Hospital
🇬🇧Oxford, United Kingdom
Medizinische Universitaet Graz
🇦🇹Graz, Austria
Medical University Vienna
🇦🇹Vienna, Austria
Universitaetsklinikum Giessen und Marburg GmbH
🇩🇪Giessen, Germany
Universitatsklinikum Jena
🇩🇪Jena, Germany
Instytut Centrum Zdrowia Matki Polki
🇵🇱Lodz, Poland
Instytut Matki i Dziecka
🇵🇱Warszawa, Poland
Panstwowy Instytut Medyczny MSWiA w Warszawie
🇵🇱Warszawa, Poland
Birmingham Women's Hospital
🇬🇧Birmingham, United Kingdom
Liverpool Women's NHS Foundation Trust - Liverpool Women's Hospital
🇬🇧Liverpool, United Kingdom
Queen Charlotte's and Chelsea Hospital
🇬🇧London, United Kingdom