Belton

Researchers are evaluating the effectiveness, safety, and tolerability of mavorixafor in people aged 12 and older who have congenital or acquired primary autoimmune and idiopathic chronic neutropenic disorders. These participants experience repeated and/or serious infections due to low neutrophil levels. The study aims to show clinical benefit by increasing circulating neutrophils and reducing infection rates. Participants will continue their existing treatments, which may include granulocyte-colony stimulating factor (G-CSF), immunoglobulin replacement, prophylactic antibiotics, or observation without active treatment. They will be randomly assigned to receive either mavorixafor or a placebo, with both drugs given according to a set schedule. The study is double-blind and placebo-controlled, conducted across multiple centers. During the study, researchers will monitor participants for up to 52 weeks, focusing on the annual infection rate and the number of participants achieving a positive response in absolute neutrophil count. Participants will undergo regular assessments, including blood tests to measure neutrophil levels and evaluations for infections. The study includes safety monitoring and requires participants to maintain stable doses of their background therapies unless safety concerns arise.

Researchers are studying the safety and effectiveness of AlloNK, a non-genetically modified natural killer (NK) cell therapy derived from cord blood, combined with rituximab. This open-label Phase 2a trial focuses on adults with relapsing forms of B-cell dependent rheumatologic diseases, including refractory rheumatoid arthritis, Sjf6gren's disease, idiopathic inflammatory myopathies, and systemic sclerosis. The goal is to evaluate how this combination treatment works in these difficult-to-treat conditions. Participants receive AlloNK following a conditioning regimen along with rituximab. AlloNK is an off-the-shelf, cryopreserved NK cell therapy given to support the immune response. The study does not mention a placebo group, and all participants receive this combination treatment. The trial monitors participants for up to 104 weeks to assess safety and treatment effects. Throughout the study, participants undergo regular assessments including clinical evaluations, laboratory tests, and disease activity measurements specific to their condition. Researchers will monitor safety from enrollment until the end of treatment at week 104. The study aims to better understand the treatment's impact on disease symptoms and progression over this two-year period.

Researchers are studying children aged 0 to 2 years who have been hospitalized for bronchiolitis, a common lung infection. The study compares two different strategies designed to improve how doctors prescribe follow-up care that families can use as needed after hospital discharge. This is important because many automatic follow-up visits may not be necessary, and the study aims to reduce unnecessary visits while supporting families' preferences. The study tests two approaches: a moderate-resource strategy and a high-resource strategy. The moderate approach provides education for doctors and families, review of doctors' performance, and materials to help with clinical decisions. The high-resource strategy includes all these components plus additional small-group sessions and expert coaching to support doctors in follow-up care decisions. Participants will be monitored to see how well these strategies increase as-needed follow-up prescriptions within 7 days after leaving the hospital. Families and doctors will be supported with educational and decision-making materials. The study focuses on improving care after hospital discharge while minimizing unnecessary clinic visits, aiming for better, more personalized follow-up care for children with bronchiolitis.

Researchers are evaluating a new quantitative HER2 test to see if it can accurately identify which patients with HER2-low metastatic breast cancer respond to treatment with T-DXd (Enhertu). The study focuses on patients with metastatic breast cancer showing low HER2 expression (1+ level by immunohistochemistry) and aims to improve how response to therapy is predicted. Participants will have leftover tumor tissue from routine biopsies sent for analysis using the CE-10-IVD diagnostic test. The treatment involved is T-DXd, given at the FDA-approved dose and schedule as the next line of therapy. Patients may already be receiving T-DXd if enrolled within 30 days of starting therapy. The study uses tissue samples to measure HER2 levels through immunofluorescence and/or RT-qPCR methods. During the study, patients will be monitored for their response to T-DXd treatment over a period of up to 100 months from their first dose. Assessments include measuring tumor response through imaging and biomarker analysis of tumor tissue. The main outcome is the objective response rate, which tracks how well tumors shrink or disappear. Participants must be able to provide informed consent and have measurable disease by imaging, including bone-only disease or active brain metastases.

Researchers are evaluating the safety, tolerability, and characteristics of a drug called Rina-S in people with various advanced solid tumors that have spread or cannot be removed by surgery. This includes cancers such as ovarian, endometrial, breast, non-small cell lung cancer, and mesothelioma. The study is a Phase 1/2 trial designed to assess how the drug works and its effects on these cancers. Participants will receive intravenous infusions of Rina-S alone or in combination with other drugs like Carboplatin, Bevacizumab, or Pembrolizumab. The study is divided into multiple parts focusing on different tumor types and treatment combinations, including monotherapy and combination therapy cohorts. Treatments continue until disease progression, unacceptable side effects, or other reasons for stopping treatment. During the study, participants will undergo various assessments including safety monitoring for side effects and dose-limiting toxicities, tumor response evaluations using imaging criteria, and heart monitoring with electrocardiograms for some participants. Researchers will track treatment-emergent adverse events for up to about one year. Participants remain involved through treatment cycles and follow-up evaluations to understand the drug's effects and safety profile.