Galveston

This observational study focuses on individuals with Recessive Dystrophic Epidermolysis Bullosa RDEB who were treated with prademagene zamikeracel pz-cel, a gene therapy made from the participants own skin cells and applied surgically to treat wounds. The study aims to evaluate the long-term safety of pz-cel, the durability of wound healing, and the occurrence of any malignancies after treatment in the post-marketing setting. Participants who have received pz-cel treatment will be followed for up to 15 years after their treatment. Assessments will primarily be collected remotely on an annual basis, but if the participants healthcare provider deems it necessary after the first year, some data may be collected during in-person visits. Annual evaluations will include reviewing adverse events, serious adverse events, hospitalizations, evidence of malignancies, potential retroviral infections, wound healing status based on caregiver reports, and any changes in medications or procedures. Throughout the study, participants will provide data annually for up to 15 years post-treatment. Researchers will monitor the number and incidence of treatment-related malignancies, serious adverse events including wound-specific events, and any positive retroviral infection test results. This long-term follow-up aims to understand the safety profile and wound healing durability of pz-cel, with participants contributing data primarily through remote means and occasional in-person assessments as needed.

Researchers are evaluating changes in bone mineral density in premenopausal women with heavy menstrual bleeding caused by uterine fibroids or moderate-to-severe pain from endometriosis. This Phase 3B, open-label study looks at the effects of continuous treatment with a relugolix combination tablet for up to 48 months 4 years, followed by a 1-year period to monitor bone health after stopping treatment. Participants will take a daily oral relugolix combination tablet containing relugolix 40 mg, estradiol 1 mg, and norethindrone acetate 0.5 mg for 4 years. Bone mineral density will be measured every 6 months using dual-energy X-ray absorptiometry DXA. Some women who have completed a previous related study may join to complete 3 years of treatment. After treatment ends, bone density will be checked again at 6 months and 12 months during the follow-up year. Women in the study will have regular visits for bone density scans and health assessments, including physical and gynecological exams, lab tests, and vital signs. Researchers will track changes in bone density at the spine, hip, and femoral neck throughout treatment and follow-up. They will also monitor for any fractures or adverse events during the 4 years of treatment and the 1-year post-treatment period. Total participation can last up to 5 years including the follow-up.

Researchers are evaluating the safety and immune response of OCU500, a chimpanzee adenovirus-based COVID-19 vaccine targeting the Omicron XBB1.5 spike protein. This phase 1 trial involves healthy adults aged 18 to 64 who have already completed their primary COVID-19 vaccination series and received at least one booster. The study aims to assess how safe and reactive a single dose of OCU500 is when given to these previously vaccinated adults. Participants are assigned randomly to one of four groups to receive a single dose of OCU500. The vaccine is given either via inhalation or intranasally at two different dose levels 1x1010 or 5x1010 viral particles. Each participant receives only one dose on Day 1. The trial includes 80 participants, with 20 individuals in each group, to compare the effects of the different doses and delivery methods. During the study, participants undergo safety monitoring for adverse events through Day 29 and up to 6 months after vaccination. Researchers also measure immune responses including anti-Spike antibodies in blood and nasal mucosa over 181 days. Participants complete study visits and lab tests to monitor health and immune reactions. The total study duration for each participant extends through 6 months following vaccine administration to evaluate both short- and longer-term safety and immunogenicity.

Researchers are evaluating the combination of adagrasib, pembrolizumab, and platinum-doublet chemotherapy compared to placebo plus pembrolizumab and platinum-doublet chemotherapy in adults with previously untreated, locally advanced or metastatic non-squamous non-small cell lung cancer NSCLC carrying the KRAS G12C mutation. This Phase 3 trial aims to assess the efficacy, safety, and tolerability of these treatment combinations in this specific patient group. Participants will receive either adagrasib plus pembrolizumab combined with platinum-doublet chemotherapy or placebo plus pembrolizumab and platinum-doublet chemotherapy. Treatments involve specified doses administered on scheduled days, with the chemotherapy consisting of carboplatin or cisplatin along with pemetrexed. Participants are randomly assigned to one of the two study groups and treatments are blinded to ensure unbiased assessment. Throughout the study, participants will undergo regular evaluations including imaging scans to measure tumor response and progression-free survival, as well as assessments of overall survival. Safety is closely monitored by recording adverse events for up to 90 days after the last dose. Quality of life and symptom assessments are also conducted using validated questionnaires. The study duration includes follow-up for up to seven years to gather comprehensive data on treatment outcomes and participant health.

Researchers are evaluating nipocalimab to reduce the risk of fetal anemia and other serious complications in pregnancies at high risk for severe Hemolytic Disease of the Fetus and Newborn HDFN. The study compares nipocalimab to a placebo in pregnant participants to see if it can decrease risks like fetal loss, the need for intrauterine transfusions, hydrops fetalis, or neonatal death. This phase 3 trial focuses on pregnancies with maternal alloantibody presence and previous severe HDFN history. Participants receive either nipocalimab or a matching placebo through weekly intravenous infusions starting at randomization until gestational week 35. The study is randomized and triple-masked, meaning neither participants nor researchers know who receives the drug or placebo. The treatment period covers the pregnancy phase where risk is highest, with careful monitoring throughout. During the study, participants undergo various assessments including lab tests, antibody titers, fetal antigen testing, and physical exams to monitor health. Researchers track pregnancy outcomes through delivery and up to 4 weeks after birth or 41 weeks postmenstrual age for newborns. Long-term infant health, including development and complications related to HDFN, is followed for up to 104 weeks. Safety and maternal outcomes are also closely observed until 24 weeks postpartum.

Researchers are evaluating the efficacy and safety of nipocalimab compared to intravenous immunoglobulin IVIG in pregnant women at risk of fetal and neonatal alloimmune thrombocytopenia FNAIT, a condition where maternal antibodies attack fetal platelets. This Phase 3 trial aims to reduce the risk of severe bleeding or low platelet counts in affected fetuses and newborns. The study is sponsored by Janssen Research & Development, LLC and focuses on pregnancies complicated by specific maternal alloantibodies and fetal genotypes. Participants are randomly assigned to receive either nipocalimab or IVIG treatments during pregnancy. Nipocalimab is given intravenously starting between 13 to 18 weeks of gestation until delivery. Those receiving IVIG start at gestational week 12 for high-risk pregnancies or week 20 for standard-risk pregnancies, with oral prednisone added per protocol and tapered off after delivery based on tolerance and investigator judgment. Both treatments are studied for their impact on fetal and neonatal outcomes. During the study, participants undergo assessments including physical exams, vital signs, ECGs, and laboratory tests to monitor health and response. Researchers will track outcomes such as fetal or neonatal death, severe bleeding, and platelet counts up to one week after birth. Long-term follow-up includes developmental assessments at 52 and 104 weeks and monitoring for treatment-emergent adverse events in mothers and infants. The overall participation period extends through delivery and includes postnatal evaluations for both mother and child.

Researchers are evaluating DMX-200 repagermanium, a drug that blocks a receptor involved in inflammation, in patients with focal segmental glomerulosclerosis FSGS who are also receiving an angiotensin II receptor blocker ARB. This Phase 3 study aims to assess the safety and effectiveness of DMX-200 compared to placebo over two years in adults and adolescents aged 12 to 17 years. The study is led by Dimerix Bioscience Pty Ltd and includes a double-blind period followed by an open-label extension to observe long-term effects. Participants receive either 120 mg of DMX-200 or a matching placebo capsule twice daily for 104 weeks during the double-blind treatment phase. Afterward, those who complete this phase may enter a two-year open-label extension where all participants receive DMX-200 twice daily. The study includes a screening and qualification period lasting 6 to 14 weeks, a possible titration phase, a stabilization phase, and a follow-up period after treatments. Throughout the trial, patients will undergo assessments including urine proteincreatinine ratio and kidney function tests like estimated glomerular filtration rate eGFR at multiple time points up to week 104 and during the extension. Safety and tolerability are closely monitored through regular evaluations, adverse event tracking, and follow-up visits. Total participation may last about 230 weeks, covering all study phases and follow-up periods.

Researchers are studying vaccines to prevent illnesses caused by Streptococcus pneumoniae bacteria, which have many different types called serotypes. This trial focuses on a new vaccine called V118E and compares it with an existing vaccine, PREVNAR 20, to learn how safe and well-tolerated V118E is in healthy adults aged 18 to 49. The study is a phase 1 trial designed to evaluate the safety, tolerability, and immune response to V118 formulation E. Participants will receive injections in a two-dose schedule. One group will get the V118E vaccine by intramuscular injection on Day 1 and Day 29. The comparison group will receive one dose of PREVNAR 20 vaccine on Day 1 and a saline injection on Day 29. Both vaccines are given by intramuscular injection, and the study is randomized and double-blind. During the study, participants will be monitored for any side effects, including injection-site reactions and other adverse events up to 12 months after the last vaccination. Researchers will also measure immune responses by checking antibody levels before and after the first vaccination. The study involves regular visits and safety assessments to understand how well participants tolerate the vaccines and to track any medical issues that may arise over the year-long follow-up period.

Researchers are evaluating Sulbactam-Durlobactam SUL-DUR in hospitalized pediatric patients from birth to under 18 years old who have suspected or confirmed infections caused by Acinetobacter baumannii-calcoaceticus complex ABC. This phase 1b, open-label study aims to collect pharmacokinetic and safety data to determine appropriate dosing for different pediatric age groups. The study is sponsored by Innoviva Specialty Therapeutics and focuses on understanding how the drugs behave in childrens bodies and their tolerability. The study includes several groups based on age, ranging from birth to less than 18 years. Different dosing regimens of Sulbactam and Durlobactam are given intravenously every 6 to 12 hours depending on the patients age and weight, with specific doses for preterm and term infants. The trial is non-randomized and involves multiple experimental cohorts receiving Sulbactam-Durlobactam at various dosages tailored to their developmental stage. Participants will be monitored closely through blood tests to assess drug levels on days 1 and 3, as well as safety assessments over 28 days. These assessments include liver and kidney function, blood counts, vital signs, and recording any adverse events. The study tracks treatment-related effects and changes in health markers to evaluate safety and drug processing in the body. The total study period involves initial dosing followed by regular monitoring to ensure participant safety and gather thorough data on the treatments effects.

Researchers are evaluating the safety and effectiveness of induction therapy using Afimkibart also called RO7790121 in people with moderately to severely active ulcerative colitis UC. This Phase III study is designed as a multicenter, double-blind, placebo-controlled trial to compare Afimkibart with a placebo. The study aims to understand how well Afimkibart works to induce remission in UC and its safety profile. Participants will be randomly assigned to one of two groups. One group will receive Afimkibart through an intravenous IV infusion followed by a subcutaneous SC injection, while the other group will receive matching placebo infusions and injections. The treatment period lasts 12 weeks, during which researchers will assess the effects of the therapies. Throughout the study, participants will undergo various assessments including evaluations of clinical remission, endoscopic improvement, histologic changes, and symptom severity at specified time points such as baseline, Week 2, and Week 12. Safety will be monitored by tracking adverse events for up to 30 weeks after starting treatment. The total participation duration spans the treatment and follow-up periods to gather comprehensive data on outcomes and safety.