Izumi

Researchers are evaluating whether an investigational drug called OHB-607 can prevent Bronchopulmonary Dysplasia (BPD), a common chronic lung disease, in extremely premature infants. The study compares infants receiving OHB-607 alongside standard neonatal care to those receiving standard care alone to reduce the burden of this lung condition. This is a Phase 2b, multicenter, randomized, open-label study focused on safety and clinical efficacy. Participants will receive an intravenous infusion of OHB-607 from birth until reaching a postmenstrual age (PMA) of 29 weeks and 6 days. The study includes two arms: one group receives the investigational drug plus standard care, while the other group receives only standard neonatal care. The treatment period ends at 29 weeks plus 6 days PMA, after which infants are monitored. Throughout the study, researchers will track the incidence of severe BPD or death up to 36 weeks PMA, whichever occurs first. Assessments will include clinical evaluations and monitoring for safety and any side effects. The study also involves long-term follow-up to observe the infants' health outcomes beyond the treatment period. Participation involves consent from parents and collection of birth and medical history information.

This trial studies participants with relapsed Small Cell Lung Cancer who have received prior treatment. It aims to compare the effectiveness and safety of a new drug called ZL-1310 with therapies chosen by investigators, such as Topotecan, Lurbinectedin, or Amrubicin. This is a Phase 3, randomized, open-label study evaluating outcomes including tumor response rate and overall survival over a period of up to 27 months. Participants receive either ZL-1310 alone or one of the Investigator's Choice therapies as treatment. The study includes screening to confirm measurable disease and eligibility. Treatments are administered according to the study protocol, and tumor biopsies or archived tissue samples are collected at screening. Both treatment groups are monitored for response and side effects throughout the study period. During the trial, participants undergo regular assessments including tumor imaging evaluated by an independent review, and survival status is tracked for up to 27 months. Researchers monitor safety, treatment adherence, and disease progression. Participants are expected to comply with study visits and procedures for the entire duration of the trial to help evaluate the benefits and risks of the treatments.

Researchers are collecting detailed information about very premature infants born at 22 to 23 weeks of gestation and their mothers to better understand outcomes and treatments in neonatal intensive care units. This registry aims to gather data from multiple hospitals to track and evaluate the health, care, and progress of these extremely premature infants. The study gathers baseline observational data on mothers and infants, including demographics, maternal health, labor and delivery details, infant health, medical treatments, and clinical outcomes. Participating hospitals use this information to improve quality of care, analyze relationships between patient characteristics and treatments, and observe trends in disease and therapy. Participants include all local births within the specified gestational age range and outborn infants admitted to NICUs at these gestational ages. Data collection continues longitudinally through January 2028, providing ongoing reporting and tracking of outcomes for these infants and their mothers.

Researchers are evaluating the safety and effectiveness of ION582 in children and adults with Angelman syndrome caused by a deletion or mutation of the UBE3A gene. This Phase 3 study includes two groups: pediatric participants aged 2 to under 18 years and adult participants aged 18 to 50 years. The study aims to measure changes in expressive communication abilities using the Bayley Scales for Infant and Toddler Development-4 over one year in the pediatric group. Participants will be randomly assigned to receive either 80 mg of ION582 or a placebo through intrathecal injections during a double-blind treatment period lasting about 60 weeks. After this, all participants who complete the placebo-controlled phase may enter a long-term extension period of approximately 25 months, during which everyone will receive ION582. Following the extension, there is an additional 8-month follow-up period. The study started with three dosing groups but was amended to two arms: 80 mg ION582 and placebo. Throughout the study, participants and caregivers will attend visits for assessments, including developmental tests without caregiver input. Researchers will monitor safety, treatment effects, and adherence. Consent and compliance with study rules, including restrictions on sharing personal medical information publicly, are required. The total participation spans screening, treatment, extension, and follow-up phases lasting over three years in total.

Researchers are evaluating eltrombopag in children aged 6 to under 18 years old who have aplastic anemia and have not previously received Anti-Thymocyte Globulin (ATG) treatment. This multicenter, non-interventional study aims to confirm the safety and effectiveness of eltrombopag in this group. The study focuses on pediatric patients who are new to treatment with ATG combined with eltrombopag. Eltrombopag is given according to the latest dosage instructions provided in the package insert. This is an observational study without random treatment allocation. Patients who meet the eligibility requirements are registered and monitored as they receive eltrombopag. The observation period lasts for one year (364 days) from the start of eltrombopag treatment, unless the patient undergoes hematopoietic stem cell transplantation within that year, in which case the observation ends on the transplantation date. During the study, researchers will track serious adverse events and monitor safety and efficacy. Patients will be followed up through regular assessments during the one-year observation period. This monitoring helps ensure thorough data collection on how eltrombopag performs in children with aplastic anemia who have not previously been treated with ATG.

This research observes patients with Paroxysmal Nocturnal Hemoglobinuria who are treated with Fabhalta capsules. It is a multicenter, single-arm, non-interventional study designed to monitor drug use and safety over time. The study uses a central registration and all-case surveillance system to collect data. Participants will be observed for 48 weeks after starting Fabhalta treatment. If treatment stops within this period, any adverse events and use of other medications will be tracked up to 30 days after the last treatment day. There are no additional interventions or comparison groups in this study. During the study, researchers will monitor the occurrence of infections and other adverse events through case report forms. Participants' health and drug usage will be recorded throughout the observation period. The total participation lasts for 48 weeks, focusing on safety and drug use in real-world settings.

This research investigates the safety of tisagenlecleucel (CTL019) that does not meet the usual commercial release standards, focusing on patients treated within the approved label by the Japan Health Authority. The study includes pediatric and young adult patients with relapsed or refractory B-cell acute lymphoblastic leukemia (pALL) and adults with relapsed or refractory large B-cell lymphoma (LBCL) and related types for Part 1. Part 2 includes patients with relapsed or refractory acute lymphoblastic leukemia and non-Hodgkin's lymphomas. The study is a Phase IIIb, open-label, multicenter trial assessing both safety and key efficacy for Part 1, with safety the main focus in Part 2. Participants receive a single intravenous infusion of CTL019, which consists of CAR-positive viable T cells. Part 1 participants are followed for 3 months after infusion, while Part 2 participants are followed for 1 day. Patients are included if their tisagenlecleucel batch does not meet commercial release standards but is individually approved by Novartis teams for safety. The study excludes those with certain infections, CNS lymphoma, hypersensitivity, or uncontrolled infections in Part 1, while Part 2 follows the product’s approved guidelines. During the study, participants undergo safety monitoring to record adverse events from screening through follow-up periods (3 months for Part 1 and 1 day for Part 2). Investigators carefully assess each patient's response and safety outcomes related to the tisagenlecleucel treatment. Informed consent is required before participation, and patients are observed for any side effects or reactions connected to the investigational infusion.

Researchers are evaluating whether the drug zilebesiran can reduce the risk of major cardiovascular events such as cardiovascular death, nonfatal heart attacks, strokes, or heart failure in adults who have hypertension that is not well controlled and who either have established cardiovascular disease or are at high risk for it. This Phase 3 global study is designed to continue until enough cardiovascular events have occurred to assess the treatment's effect. Participants will be randomly assigned to receive either zilebesiran or a placebo, both given as injections under the skin (subcutaneous administration). All participants will continue with their standard care, which includes treatment with at least two antihypertensive medications, one of which must be a diuretic such as a thiazide or loop diuretic. The study is double-blind, so neither participants nor researchers know who is receiving the active drug or placebo. During the study, participants will be closely monitored for cardiovascular events including heart attacks, strokes, heart failure hospitalizations, and cardiovascular deaths over approximately five years. Researchers will collect data on these events to determine the time until the first occurrence of any of these outcomes. Safety assessments and standard clinical evaluations will also be performed throughout the study period to ensure participant well-being.