A Study on Hemolytic Disease of the Fetus and Newborn (HDFN) Through Global Registry
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexFemale
Age18+
SponsorJanssen Research & Development, LLC
About this trial
The purpose of this non-interventional study is to prospectively evaluate the risk of anemia (decreased red blood cells) in fetuses (baby before birth) and neonates (baby just after birth) of pregnant participants who are at risk for hemolytic disease of the fetus and newborn (HDFN) and receiving standard of care (SoC). HDFN is a blood disease that occurs in babies before birth or just after birth when the blood types of the pregnant individual and babies are incompatible, thus resulting in fast breakdown of red blood cells (RBCs) of the fetus/baby.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Pregnant with an estimated gestational age (GA) (based on ultrasound dating) up to week 24
History of a previous alloimmunized pregnancy that included at least one of the following: Fetal anemia diagnosed by middle cerebral artery (MCA) doppler ultrasound; Received greater than or equal to (>=) 1 intrauterine transfusion (IUT) as a result of hemolytic disease of the fetus and newborn (HDFN); Fetal hydrops; Stillbirth or fetal demise with fetal or placental pathology indicative of HDFN; Neonatal exchange transfusion due to HDFN; Neonatal simple transfusion due to HDFN; Neonatal hyperbilirubinemia due to HDFN; Positive direct antiglobulin test (DAT) in neonate
Documented presence of maternal alloantibody based on local laboratory results during current pregnancy
Evidence of an antigen-positive fetus corresponding to the current maternal alloantibody: Fetal antigen status confirmed by cell-free fetal DNA (cffDNA); OR Fetal antigen status confirmed by amniocentesis; OR Paternal genotype confirmed
Disqualifiers
Participant actively participating in an interventional trial of an investigational agent
At risk for HDFN due to ABO being the sole alloimmunization antigen in the current pregnancy (that is, ABO plus another antigen is permissible)
Trial population
The study population will include pregnant participants with a risk for hemolytic disease of the fetus and newborn (HDFN) in their current pregnancy by virtue of having a previous alloimmunized pregnancy with a gestation that included fetal anemia, received intrauterine transfusion (IUT), fetal hydrops, stillbirth with fetal or placental pathology indicative of HDFN, neonatal exchange transfusion, neonatal hyperbilirubinemia due to HDFN, or positive direct antiglobulin test (DAT) in the neonate.
Trial design
Design model
Cohort
Time perspective
Prospective
Treatments tested in this trial
Standard of Care
Other interventionNo study treatment will be administered as part of this study. Participants will receive standard of care therapy as per local clinical practice.
Treatment groups
175 Participants
are divided into 1 treatment groupGroup A: Pregnant Participants at Risk for Hemolytic Disease of the Fetus and Newborn (HDFN)1 intervention
Trial outcomes
Primary outcomes
1
Percentage of Pregnancies That did not Result in Fetal Loss, Intrauterine Transfusion (IUT), Hydrops Fetalis, or Neonatal Death During the Neonatal Period
Percentage of pregnancies that did not result in fetal loss (due to any reason), IUT, hydrops fetalis, or neonatal death (due to any reason) during the neonatal period will be reported. Hydrops fetalis is defined as the presence of greater than or equal to (\>=) 2 abnormal fluid collections in the fetus or neonate, such as ascites, pleural effusions, pericardial effusion, and generalized skin edema (skin thickness greater than \[\>\] 5 millimeter \[mm\]). Post-menstrual age (PMA) is the time elapsed between the first day of the last menstrual period and birth (gestational age) plus the time elapsed after birth (chronological age).
Time frame
From conception through 4 weeks of age or 41 weeks PMA, whichever is laterSecondary outcomes
1
Number of Participants With Hemolytic Disease of the Fetus and Newborn (HDFN) by Severity
Number of participants in each HDFN severity index category will be reported. The severity of HDFN is defined as: 5 (fatal): fetal or neonatal death due to any reason; 4 (severe): hydrops fetalis (in fetus or newborn) or receiving IUT during pregnancy as a result of HDFN but not 5 (fatal); 3 (moderate): neonatal exchange transfusions received as a result of HDFN related hemolysis and jaundice but not 4 (severe) or 5 (fatal); 2 (mild): neonatal simple transfusions received due to HDFN after birth, with or without phototherapy, but not 3 (moderate), 4 (severe), or 5 (fatal); and 1 (minimal or none): not in 2 (mild), 3 (moderate), 4 (severe), or 5 (fatal) as described above. HDFN requiring phototherapy will be classified in this category.
Time frame
Through 4 weeks of age or 41 weeks PMA, whichever is later2
Time to First Occurrence of IUT or Hydrops Fetalis
Time to first occurrence of IUT or hydrops fetalis will be reported.
Time frame
From conception to delivery date (up to maximum of 42 weeks)3
Modified Neonatal Morbidity and Mortality Index (mNMMI) in Live Newborn Neonates
The mNMMI will be assessed with the following categories: fatal: neonatal death; major morbidity: any of intraventricular hemorrhage grade 3/4, seizures, hypoxic-ischemic encephalopathy, necrotizing enterocolitis stage 2/3, respiratory distress syndrome requiring mechanical ventilation, bronchopulmonary dysplasia requiring oxygen support, or persistent pulmonary hypertension; Minor morbidity: anemia requiring simple transfusion, hyperbilirubinemia requiring an exchange transfusion, hypotension requiring treatment, intraventricular hemorrhage grade 1/2, necrotizing enterocolitis stage 1, or respiratory distress syndrome not requiring mechanical ventilation; None: no major or minor morbidities described above. Hyperbilirubinemia requiring phototherapy will be classified in this category.
Time frame
Through 38 weeks PMA or at discharge if earlier than 38 weeks PMA4
Number of IUTs Received During the Pregnancy
Number of IUT's received during the pregnancy will be reported.
Time frame
From conception to delivery date (up to maximum of 42 weeks)Sponsors and contacts
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Source ClinicalTrials.gov