Hemolytic Disease of the Newborn

Researchers are evaluating nipocalimab to reduce the risk of fetal anemia in pregnant individuals at risk for severe hemolytic disease of the fetus and newborn (HDFN). This condition involves a decrease in the baby's red blood cell volume during pregnancy. The study is a Phase 3 randomized, placebo-controlled trial aiming to compare nipocalimab's effectiveness to placebo in protecting live neonates from complications related to HDFN. Participants will receive either nipocalimab or a placebo through weekly intravenous infusions starting from randomization until gestational week 35. The study is double-blind and multicenter, ensuring that neither participants nor researchers know which treatment is given. The trial monitors pregnancies from early gestation through delivery and the neonatal period. Participants will be closely followed with assessments during pregnancy and after birth including monitoring for fetal loss, intrauterine transfusions, hydrops fetalis, neonatal death, and other health outcomes up to 104 weeks for the infant. The study also collects data on maternal health, pregnancy complications, infant development, and quality of life. Safety evaluations continue through 24 weeks postpartum, with detailed tracking of treatment effects and infant well-being.

Researchers are evaluating nipocalimab compared with a placebo to see if it can reduce the risk of severe fetal and neonatal alloimmune thrombocytopenia (FNAIT) in pregnant women at risk. This randomized, double-blind, placebo-controlled Phase 3 study focuses on pregnancies affected by FNAIT, a condition where the mother's immune system attacks fetal platelets, leading to low platelet counts and bleeding risks. Pregnant participants will be randomly assigned to receive either nipocalimab or a placebo, both administered intravenously. The study includes maternal dosing during pregnancy, with treatments given between gestational weeks 13 to 18. The trial monitors mothers and their babies closely throughout pregnancy and after birth to assess safety and effects. Participants will undergo health assessments including physical exams, medical histories, vital signs, ECGs, and laboratory tests. Researchers will measure outcomes such as fetal and neonatal death, severe bleeding, platelet counts at birth and during the first week post-birth, and the need for platelet transfusions. Safety monitoring for both mothers and infants continues up to 104 weeks after birth, including evaluation of infant development and maternal antibody responses to nipocalimab.

Researchers are evaluating nipocalimab and intravenous immunoglobulin (IVIG) as treatments in pregnancies at risk of fetal and neonatal alloimmune thrombocytopenia (FNAIT), a condition where the mother's immune system attacks the baby's platelets. This Phase 3 study aims to assess the effectiveness and safety of nipocalimab in reducing severe complications such as bleeding or low platelet counts in newborns. The study is sponsored by Janssen Research & Development, LLC. Pregnant women with specific antibodies (anti-HPA-1a and/or anti-HPA-5b) will be randomly assigned to receive either nipocalimab or IVIG along with prednisone. Nipocalimab will be given intravenously from gestational week 13 to 18 until delivery. IVIG is administered intravenously starting at week 12 for high-risk pregnancies or week 20 for standard-risk pregnancies, with oral prednisone added and tapered off after delivery based on tolerance. Participants will be closely monitored through pregnancy and after birth, with evaluations including blood tests for platelet counts, assessments of bleeding events, and developmental testing of the infant up to two years old. Researchers will track safety outcomes for both mother and baby, including adverse events and antibody responses. The study involves follow-up visits up to one year after birth, with the main outcome measured as death, severe bleeding, or very low platelet count in the newborn within one week after birth.

Researchers are studying the risk of anemia in babies before and just after birth whose mothers are at risk for hemolytic disease of the fetus and newborn (HDFN). HDFN happens when the blood types of the pregnant participant and baby do not match, causing the baby's red blood cells to break down quickly. This non-interventional study aims to better understand this risk by following pregnant participants with HDFN risk and their babies over time. Pregnant participants who are at risk for HDFN during their current pregnancy will be enrolled and receive standard medical care according to their local practice. The study does not provide any treatment but collects data throughout pregnancy and for two years after birth from medical records and participant-reported outcomes. Data collection will occur at medical centers experienced in diagnosing and treating HDFN. Participants will provide medical information during pregnancy and after birth, including clinical outcomes and questionnaires about health status. Researchers will measure the percentage of pregnancies without fetal loss, intrauterine transfusions, hydrops fetalis, or neonatal death up to four weeks after birth. They will also track the severity of HDFN, timing of complications, and transfusions received. The study follows participants from conception through two years after delivery to gather long-term data.

Researchers are evaluating KLN-1010, a novel gene therapy, in patients with relapsed or refractory multiple myeloma to assess its safety, tolerability, and to determine the recommended Phase 2 dose. The trial is a Phase 1 study sponsored by Kelonia Therapeutics, Inc., focusing on patients who have previously undergone multiple treatments for their condition. Participants receive a single specified dose of KLN-1010, which is designed to generate anti-B Cell Maturation Antigen (anti-BCMA) CAR-T cells in the body. This gene therapy is administered once during the study, and its effects, including safety and pharmacokinetics, are closely monitored over time. During the study, participants undergo assessments for treatment-emergent adverse events and dose-limiting toxicities for up to 15 years after dosing. Pharmacokinetic evaluations of KLN-1010 and generated CAR-T cells occur up to two years post-infusion. Multiple myeloma status is monitored from dosing until disease progression or up to 15 years, with comprehensive safety and disease assessments throughout the follow-up period.

Researchers are evaluating the safety, pharmacokinetics, pharmacodynamics, and preliminary effectiveness of an anti-GPRC5D CAR-T cell product called OriCAR-017 in adults with relapsed or refractory multiple myeloma. This Phase I/II open-label study is the first clinical trial of OriCAR-017 in the United States by OriCell Therapeutics Co., Ltd., aiming to find suitable dosing and assess early treatment results in this patient group. The study includes a Phase I dose escalation stage with three different doses given as a single intravenous infusion to up to 18 participants. This is followed by a dose expansion stage with 10-15 participants and then a Phase II stage that may include up to 48 participants. Each participant receives one infusion of OriCAR-017 to evaluate its effects and safety. Participants will be closely monitored for up to two years after treatment. Researchers will assess the maximum tolerated dose and dose-limiting toxicities within 28 days after infusion. They will also study how the drug moves through and affects the body, measure response duration, progression-free survival, overall survival, and other response rates. Regular evaluations include laboratory tests, clinical assessments, and safety monitoring throughout the study period.

Researchers are evaluating the safety and clinical activity of different doses of belantamab mafodotin combined with lenalidomide, dexamethasone, and nirogacestat in patients with newly diagnosed multiple myeloma who are not able to undergo a transplant. This phase 1/2, open-label study aims to find the recommended dose for future studies and to explore how to manage eye-related side effects. About 36 participants will be enrolled, with follow-up lasting up to 3 to 4 years after enrollment ends. Belantamab mafodotin is given intravenously every other 28-day cycle at doses ranging from 1.0 to 1.9 mg/kg. Lenalidomide is taken orally daily on days 1 to 21 of each 28-day cycle. Dexamethasone is given either orally or intravenously on days 1, 8, 15, and 22 of each cycle, with dose adjustments based on age. Nirogacestat is taken twice daily starting three days before the first dose and on the same days as belantamab mafodotin. The study has two parts: dose finding to establish the best dose, followed by dose expansion with random assignment to different dose modification guidelines for eye side effects. Participants will undergo regular assessments to monitor side effects, including eye exams, and overall response to treatment. Researchers will track dose-limiting toxicities, adverse events, ocular toxicity, and response rates over up to four years. Additional evaluations include measuring drug levels, disease progress, and survival. Participants must provide consent and will be closely monitored throughout the study period, which is expected to last approximately four years in total.

Researchers are evaluating the safety and feasibility of lung transplants from blood group A2 donors to blood group O recipients. This study aims to increase the donor pool for blood group O lung transplant candidates, who often face longer wait times and higher mortality on the waitlist. It is the first prospective study worldwide to assess ABO-incompatible lung transplants in this way. The intervention involves lung transplant surgery using lungs from blood group A2 donors transplanted into blood group O recipients who have low anti-A antibody levels and a negative virtual crossmatch at the time of transplant. The study monitors the recipients closely after surgery to assess immune response and transplant success. Participants will be monitored through various evaluations including antibody levels on post-operative days 5 or 12, primary graft dysfunction grading in the first three days, and assessment for rejection or infection over the following years. Long-term follow-up up to 10 years will evaluate graft function, chronic lung problems, infections, malignancies, and survival outcomes. The total participation time varies depending on individual follow-up and outcomes.

This research aims to evaluate a new advanced wireless skin sensor system designed to monitor vital signs in healthy newborn infants of at least 35 weeks gestational age. The study focuses on assessing the feasibility, safety, and accuracy of this wireless system compared to the standard wired monitoring during the first two hours after birth, especially during unsupervised parental care in the obstetrical center. The goal is to improve early detection and prevention of Sudden Unexpected Postnatal Collapse (SUPC), a rare but serious condition affecting newborns shortly after delivery. Participants will have both the wireless monitoring system and the standard wired system placed on their chest and limb. For vaginal births, the wireless system is placed first, followed by the wired system after 15-20 minutes; for C-section births, the order of placement is randomized. Both monitoring systems remain in place for two hours to continuously record vital signs such as heart rate, respiratory rate, oxygen saturation, and skin temperature. During the study, newborns' vital signs will be recorded and compared between the two systems for up to two hours immediately after delivery. Researchers will evaluate the feasibility by checking for gaps in data and user satisfaction, assess safety through skin and pain scores, and measure accuracy using statistical comparisons. The study also includes monitoring the time between sensor placement and data display. Participation lasts only the initial two-hour period after birth, with monitoring done under real-world conditions in the obstetrical center.

This research aims to find out if giving plasma from a donor who recently had Chikungunya infection to newborns whose mothers have peripartum Chikungunya infection can improve the chances of newborns surviving without brain inflammation or damage within the first five days of life. The study will compare these results with a group of newborns who did not receive plasma transfusion due to delays in diagnosis or transfer, or parental refusal. The trial is led by the Centre Hospitalier Universitaire de la Réunion and includes newborns up to 2 days old. Participants in the treatment group will receive a plasma transfusion from donors who declared Chikungunya infection less than six months ago. This treatment is given to newborns whose mothers were diagnosed with Chikungunya infection around the time of birth. Newborns will be treated within 12 hours of birth or diagnosis depending on timing. There is also a parallel observational group of newborns who will not receive plasma but will be monitored as part of standard care. During the study, newborns will have daily medical and biological tests from day 1 to day 7, and a follow-up visit between 1 and 3 months of age. For the observational group, data collected during routine care up to 3 months will be used for research. Researchers will measure the effectiveness of early plasma administration in preventing neonatal brain inflammation or damage within five days after treatment. Safety and health will be monitored throughout the trial period, which is planned to end in late 2027.