Thrombocytopenia, Neonatal Alloimmune
Conditions
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).
Interventions
DRUGNipocalimab
Nipocalimab will be administered intravenously.
IVIG will be administered intravenously.
DRUGPrednisone
Prednisone will be administered orally.
Sponsors
Janssen Research & Development, LLC
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
FEMALE
Age
18 Years to 45 Years
Healthy volunteers
No
Inclusion criteria
* 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
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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 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. |
| 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. |
| 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 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. |
Countries
Austria, Germany, Netherlands, Poland, United Kingdom, United States
Contacts
CONTACTStudy Contact
STUDY_DIRECTORJanssen Research & Development, LLC Clinical Trial
Janssen Research & Development, LLC
Outcome results
None listed