Multicenter, Open-Label, Randomized Study of Nipocalimab or IVIG in Pregnancies At Risk of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT)
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 50
- 试验地点
- 35
- 主要终点
- Fetus/Neonate with Outcome of Death or Adjudicated Severe Bleeding or Platelet Count Less Than (<) 30*10^9/L
研究概览
简要总结
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).
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 45 Years（Adult）
- 性别
- Female
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
研究组 & 干预措施
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.
干预措施: Prednisone (Drug)
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.
干预措施: Nipocalimab (Drug)
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.
干预措施: Intravenous immunoglobulins (IVIG) (Drug)
结局指标
主要结局
Fetus/Neonate with Outcome of Death or Adjudicated Severe Bleeding or Platelet Count Less Than (<) 30*10^9/L
时间窗: 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.
次要结局
- Platelet Count at Birth in a Neonate(At birth)
- Neonate/Fetus with Outcome of Death(Up to 1 Week post birth)
- Neonate with Platelet Count at Birth <10*10^9/L(At birth)
- Neonate with Platelet Count at Birth <50*10^9/L(At birth)
- Nadir Platelet Count in a Neonate(Up to 1 Week post birth)
- Neonate Requiring Platelet Transfusion(Up to 1 Week post birth)
- Number of Platelet Transfusions in Neonate(Up to 1 Week post birth)
- Number of Donor Exposures for Platelet Transfusions in Neonate(Up to 1 Week post birth)
- Neonate Requiring Postnatal Intravenous Immunoglobulin (IVIG) for the Treatment of Thrombocytopenia(Up to 1 Week post birth)
- Neonate/Infant With TEAE, SAE and AESI(From Day of birth to Week 104)
- Fetus/Neonate with a TEAE of Bleeding(From Day of birth to Week 104)
- Bayley Scales Assessment for Infant Development(At Week 52 and Week 104)
- Maternal Participants with Incidence of Antibodies to Nipocalimab(Up to Week 4)
- Neonate/Fetus with Outcome of Death(Up to 1 Week post birth)
- Platelet Count at Birth in a Neonate(At birth)
- Neonate with Platelet Count at Birth <10*10^9/L(At birth)
- Neonate with Platelet Count at Birth <30*10^9/L(At birth)
- Neonate with Platelet Count at Birth <50*10^9/L(At birth)
- Neonate with Platelet Count at Birth <150*10^9/L(At birth)
- Nadir Platelet Count in a Neonate(Up to 1 Week post birth)
- Neonate Requiring Platelet Transfusion(Up to 1 Week post birth)
- Number of Platelet Transfusions in Neonate(Up to 1 Week post birth)
- Number of Donor Exposures for Platelet Transfusions in Neonate(Up to 1 Week post birth)
- Neonate/Fetus With Adjudicated Bleeding(Up to 1 Week post birth)
- Neonate Requiring Postnatal Intravenous Immunoglobulin (IVIG) for the Treatment of Thrombocytopenia(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)(Up to Week 24)
- Maternal Participant with TEAE Leading to Discontinuation of Study Intervention(Up to Week 24)
- Neonate/Infant With TEAE, SAE and AESI(From Day of birth to Week 104)
- Fetus/Neonate with a TEAE of Bleeding(From Day of birth to Week 104)
- Neonate with a TEAE of Infection(From Day of birth to Week 104)
- Bayley Scales Assessment for Infant Development(At Week 52 and Week 104)
- Maternal Participants with Incidence of Antibodies to Nipocalimab(Up to Week 4)