Charleston

Researchers are investigating the drug bezuclastinib in an open-label, two-part Phase 2 study for patients with Advanced Systemic Mastocytosis (AdvSM), including Aggressive Systemic Mastocytosis (ASM), Systemic Mastocytosis with an Associated Hematologic Neoplasm (SM-AHN), and Mast Cell Leukemia (MCL). The study aims to evaluate the safety, effectiveness, and how the drug behaves in the body for these serious conditions. Bezuclastinib is given orally as tablets taken continuously in 28-day cycles. The study has two parts: Part I focuses on identifying safe and tolerable doses of bezuclastinib over 18 months, while Part II evaluates its effectiveness by measuring the objective response rate and confirming the relationship between drug exposure and response during another 18-month period. Participants will undergo assessments including clinical evaluations, laboratory tests, and monitoring of their disease status to determine treatment effects and safety. Researchers will track the drug's impact on the disease and patient health throughout the study, which involves continuous treatment and follow-up over the specified time frames.

Researchers are comparing two types of maxillary expanders used to widen the upper jaw in children aged 8 to 13 years who have maxillary transverse deficiency. The study aims to evaluate whether the newer 3D-printed expander, designed on a computer and printed in metal, works as well as the traditional laboratory-made expander, while potentially providing greater comfort to patients. This randomized controlled clinical trial involves participants from orthodontic clinics at the Medical University of South Carolina and the University of Pittsburgh School of Dental Medicine. Participants will be randomly assigned to receive either the conventional hand-made laboratory expander or the 3D-printed expander. Both devices are used to expand the upper jaw, with the 3D-printed version incorporating advanced digital design and printing technology. The treatment will be monitored over a period of 6 months to assess the effects of each expander. During the study, participants will attend regular clinical follow-up visits where photos, X-rays, and dental photo scans will be taken to observe the progress of the maxillary expansion. Additionally, participants will complete online questionnaires with help from their parents or guardians to report their quality of life and any pain or discomfort experienced at various times. The main outcome measured is the dimension of the anterior midpalatal suture opening two weeks after starting treatment. This study also monitors safety and comfort throughout the 6-month period.

Researchers are evaluating 4D-710, an investigational gene therapy, in adults with cystic fibrosis (CF) lung disease who cannot use or tolerate CFTR modulator therapy. This Phase 1/2, multicenter, open-label trial also includes a sub-study assessing 4D-710 in adults with advanced CF lung disease or frequent lung flare-ups despite using CFTR modulators. The study aims to assess the safety, tolerability, and early effectiveness of this gene therapy in these populations. The trial involves a single dose of 4D-710, which is a gene therapy using a specialized virus to deliver a modified CFTR gene. Participants receive this treatment once, and those in the sub-study must be on a stable CFTR modulator regimen for at least 60 days before screening and continue it during a 24-month observation period. The study monitors participants with CF lung disease ranging from moderate to advanced stages. During the study, participants undergo regular evaluations including lung function tests, oxygen level checks, and monitoring for adverse effects. Researchers track the occurrence and severity of any side effects over a 60-month period. The study also includes assessments of lung health, medication adherence, and clinical status to understand the therapy's impact and safety over time.

Researchers are evaluating the safety and effectiveness of tenapanor in adults with Chronic Idiopathic Constipation (CIC) in this 26-week phase 3 study. The study is randomized, double-blind, and placebo-controlled, involving multiple centers. It aims to compare three doses of tenapanor (5 mg, 25 mg, and 50 mg taken twice daily) against a placebo, with a focus on improving spontaneous bowel movements. Participants will first undergo a 2-week screening where their eligibility is assessed through medical history, physical exams, lab tests, ECG, and self-reported constipation symptoms using an electronic diary (eDiary). Eligible patients will then be randomly assigned to receive one of the three doses of tenapanor or placebo twice daily for 26 weeks. During this treatment period, patients will continue daily and weekly symptom reporting via the eDiary and attend regular safety visits at weeks 2, 4, 8, 12, 16, 20, and 26. After completing the 26-week treatment, patients enter a 4-week treatment-free safety follow-up period to monitor any adverse events. A final visit occurs at the end of this follow-up to assess safety. The main outcome measured is the durable complete spontaneous bowel movements response over 12 weeks. Overall, the study involves careful monitoring of symptoms, safety, and treatment effects over approximately 32 weeks.

Researchers are evaluating a new brief intervention for emerging adults aged 18 to 25 who frequently use cannabis. The study aims to compare two different brief interventions to address potentially problematic cannabis use and improve outcomes related to Cannabis Use Disorder. Given the rising use and decreasing perceived risks of cannabis among young adults, this research seeks to test the feasibility, acceptability, and initial effectiveness of these approaches using rigorous methods. Participants will be randomly assigned to one of two 2-session interventions, each session lasting 40 to 50 minutes and spaced about one week apart. The Health Education intervention provides information on improving sleep, nutrition, physical activity, stress management, and social connection. The Bloom intervention focuses on personal values, reasons for cannabis use, personalized feedback, and reflection on lifestyle choices aligned with those values. During the study, participants will complete detailed cannabis assessments including biological samples collected at baseline, after the intervention, and at 1-, 3-, and 6-month follow-ups. Saliva samples will be tested for THC and CBD levels at each time point. Researchers will use self-reported cannabis use measures and biological data to track changes over time and assess intervention impact. The total study involvement includes the two intervention sessions plus four follow-up assessments over six months.

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.

Researchers are investigating new treatments for people with high-risk, early-stage breast cancer, specifically targeting triple-negative breast cancer (TNBC) and hormone receptor (HR)-low positive/HER2-negative breast cancer. These types have little or no HER2 protein and involve hormones like estrogen or progesterone. The study aims to evaluate if the addition of sacituzumab tirumotecan (sac-TMT), a targeted therapy, combined with pembrolizumab and chemotherapy can improve outcomes compared to pembrolizumab with chemotherapy alone. Participants receive treatments including sacituzumab tirumotecan, pembrolizumab, and chemotherapy drugs such as carboplatin and paclitaxel, all given by intravenous infusion. Rescue medications like antihistamines, acetaminophen, dexamethasone, or steroid mouthwash may be used as needed. The study is randomized and open-label, comparing sac-TMT followed by chemotherapy plus pembrolizumab to chemotherapy and pembrolizumab without sac-TMT. During the study, researchers will monitor participants up to about 30 weeks to assess the percentage of people with no remaining cancer cells at surgery. They will also follow participants for up to approximately 92 months to track event-free survival, meaning time without cancer growth, spread, or return. Participants will undergo imaging, clinical assessments, and laboratory tests to evaluate treatment effects and safety throughout the study.

Researchers are evaluating sotatercept as a potential treatment for pulmonary arterial hypertension (PAH), a condition where blood vessels in the lungs thicken and narrow, causing high blood pressure in the lungs and overworking the heart. PAH symptoms include difficulty breathing and reduced ability to be active. Current standard treatments address symptoms but do not stop disease progression. This Phase 3 study focuses on the long-term safety and tolerability of sotatercept when added to standard PAH therapy. Participants in this long-term follow-up study receive sotatercept through subcutaneous injections every three weeks. Only individuals who completed prior sotatercept PAH studies without early discontinuation may join. This study continues the observation and assessment of participants over an extended period to learn about the effects and safety of sotatercept combined with background PAH treatments. During the study, participants will be regularly monitored for adverse events, treatment discontinuations, and the presence of anti-drug antibodies for up to approximately 90 months. Laboratory tests will evaluate blood components such as platelets, hemoglobin, creatinine, bilirubin, and liver enzymes. Changes from baseline in body weight, blood pressure, and electrocardiogram readings will also be tracked. The study involves adherence to visit schedules and compliance with study procedures to ensure comprehensive long-term safety data collection.

Researchers are looking for ways to treat germinal center B-cell-like diffuse large B-cell lymphoma (GCB DLBCL). DLBCL is a fast-growing blood cancer that affects B-cells. GCB is a type of DLBCL that affects young B-cells that are still maturing. The goal of this study is to learn if more people who receive zilovertamab vedotin (MK-2140) and R-CHP have the cancer respond (go away) than those who receive polatuzumab vedotin and R-CHP.

Researchers are evaluating the best ways to help smokers who have difficulty quitting with medications by testing adaptive treatment plans. This study involves smokers from Alabama and South Carolina who want to quit and aims to improve decision-making about treatments using FDA-approved medications or harm reduction products. The study tracks smoking cessation success over 12 weeks of treatment and includes follow-up at 24 weeks to assess outcomes. Participants will start with a 4-week course of either varenicline or a combination of nicotine replacement therapies (patch and lozenge). Those who show early success continue with the same treatment for another 4 weeks. Smokers who do not show early success at 4 or 8 weeks will be randomized to either continue the same medication or switch to a different FDA-approved option, including switching to an e-cigarette at Week 8. The treatment phase lasts 12 weeks with all products provided free of charge. All study visits and assessments will be conducted remotely through 8 surveys over six months. After the 12-week treatment, no more products are provided, but participants will be contacted at 6 months from the first call to complete a final survey. Researchers will monitor treatment response, smoking cessation success, and other outcomes, with safety and progress tracked throughout the study duration.