Belton

Researchers are evaluating mavorixafor, a drug being studied for people aged 12 and older who have congenital or acquired primary autoimmune and idiopathic chronic neutropenic disorders. These conditions cause low neutrophil levels and lead to recurrent or serious infections. The study aims to show if mavorixafor can improve clinical outcomes and increase neutrophil counts, while also assessing its safety and tolerability. Participants will continue their current treatment during the study, which may include therapies like granulocyte-colony stimulating factor G-CSF, immunoglobulin replacement, antibiotics, or no active treatment. They will be randomly assigned to receive either mavorixafor or a placebo orally once daily for up to 52 weeks. The study uses a quadruple-blind design to compare these groups in parallel. During the trial, participants will have regular assessments to monitor infection rates and severity, neutrophil counts, antibiotic use, oral ulcers, and fatigue levels using questionnaires. An independent committee will review infections to ensure accuracy. Safety and treatment effects will be followed throughout the 52-week treatment period. The total participation may last until the study ends in late 2027.

Researchers are evaluating the safety and effectiveness of AlloNK, a non-genetically modified allogeneic natural killer NK cell therapy derived from cord blood, combined with rituximab, in people with relapsing forms of B-cell dependent rheumatologic diseases. These diseases include refractory rheumatoid arthritis, Sjgrens disease, idiopathic inflammatory myopathies, and systemic sclerosis. This open-label Phase 2a study aims to explore treatment options for patients whose conditions have not responded well to previous therapies. Participants receive AlloNK after a conditioning regimen, followed by rituximab. AlloNK is an off-the-shelf, cryopreserved NK cell therapy given in combination with rituximab. The treatment period lasts up to 104 weeks, during which participants are monitored for safety and clinical response. The study includes one treatment group receiving this combination therapy. Throughout the study, participants undergo regular assessments to evaluate safety from enrollment until the end of treatment at Week 104. Researchers measure disease-specific outcomes at Week 52, including disease activity scores for rheumatoid arthritis, Sjgrens disease activity indices, improvement scores for inflammatory myopathies, and response indices for systemic sclerosis. The study involves clinical evaluations, laboratory tests including inflammatory markers, and joint assessments to monitor treatment effects and safety over the extended period.

Researchers are studying children hospitalized with bronchiolitis to compare two different strategies aimed at improving follow-up care after hospital discharge. The study focuses on increasing the use of as-needed follow-up visits prescribed by hospital physicians and reducing unnecessary routine follow-up visits, which research has shown are often not needed. This trial evaluates how these strategies may impact care and family experience for young children recovering from bronchiolitis. The study compares a moderate-resource implementation strategy with a high-resource implementation strategy. The moderate approach includes educational outreach with materials for families, clinician performance reviews, and clinical decision support tools. The high-resource strategy adds small-group facilitation and expert coach-led support to the components of the moderate strategy. These strategies are designed to encourage appropriate follow-up care decisions by hospitalists. Participants are children diagnosed primarily with bronchiolitis, discharged from general inpatient care outside intensive or emergency settings. Researchers will measure how often as-needed follow-up is prescribed and sustained within seven days after hospital discharge. Families attendance at follow-up visits will be tracked to assess if unnecessary visits decrease. The study will monitor outcomes related to follow-up care practices and their impact through the hospital stay and shortly after discharge, with participation lasting through the immediate post-hospital period.

Researchers are evaluating whether a quantitative HER2 assay can accurately distinguish responders from non-responders among patients with HER2-low metastatic breast cancer receiving T-Dxd. This observational study focuses on improving measurement methods for HER2 expression levels to better understand treatment response. Participants provide leftover tumor tissue from routine biopsies for analysis using quantitative immunofluorescence and RT-qPCR assays. The study measures HER2 expression via these tests and correlates results with real-world treatment responses and progression over up to 100 months following the start of T-Dxd therapy. During the study, researchers collect data on tumor HER2 levels and track objective response rates and progression-free survival. Imaging and biomarker assessments are used to evaluate measurable disease, including bone and brain metastases. Participants are monitored throughout their treatment with T-Dxd, with up to 100 months of follow-up to assess outcomes and associations with HER2 assay results.

Researchers are studying Rina-S, a drug designed to target folate receptor alpha, in people with advanced solid tumors that have either spread or cannot be removed by surgery. This Phase 12 trial aims to evaluate the safety, how the drug behaves in the body, and its antitumor activity. The study includes several parts focusing on specific cancers such as ovarian, endometrial, breast, lung cancers, mesothelioma, and cervical cancer, reflecting different patient groups and treatment approaches. Participants may receive Rina-S alone or combined with other drugs like carboplatin, bevacizumab, or pembrolizumab. Treatment continues until disease progression, unacceptable side effects, or other reasons such as withdrawal or study end. The study involves multiple cohorts, including those with platinum-resistant or platinum-sensitive ovarian cancer and endometrial cancer, with dosing and combination therapies tailored to these groups. During the trial, participants undergo regular assessments including imaging scans evaluated by standardized criteria, safety monitoring for side effects, and blood tests to study drug levels and immune responses. Outcomes measured include treatment side effects, tumor response rates, progression-free and overall survival, and pharmacokinetics. Participants may be followed for up to approximately two years to evaluate long-term effects and treatment responses.