Lafayette

Researchers are studying new treatment options for people with high-risk non-muscle invasive bladder cancer HR NMIBC, including cases with carcinoma in situ CIS. HR NMIBC affects the lining of the bladder but has not spread to muscle or beyond. The study aims to learn if adding intismeran autogene V940 to the standard Bacillus Calmette-Guerin BCG immunotherapy can improve outcomes by helping the immune system attack the cancer more effectively. Participants are divided into groups receiving different treatments. One group Cohort A receives both intismeran autogene via intramuscular injection every 3 weeks for 9 doses and BCG instillations weekly in specific weeks over about 75 weeks. Another group receives only BCG following the same weekly schedule. A third group Cohort B receives intismeran autogene alone every 3 weeks for 9 doses. The study evaluates these treatments over several years. During the study, participants will have regular treatments and follow-up visits where researchers will monitor cancer progression, recurrence, and survival for up to approximately 5 years. Assessments include event-free survival, recurrence-free survival, overall survival, response rates, time to cystectomy, and safety outcomes such as adverse events and treatment discontinuation. The study is randomized and open-label, with detailed long-term monitoring planned.

This research aims to evaluate the safety and effectiveness of the FloStent, a medical device used for treating men experiencing symptoms of Benign Prostatic Hyperplasia BPH. The study compares the FloStent implant procedure to a sham procedure without deploying the device. It involves men aged 45 and older with symptomatic BPH to better understand how this device may impact urinary symptoms. Participants will undergo a flexible cystoscopy procedure. Those randomized to the treatment group will receive the FloStent implant during this procedure, while those in the control group will have the cystoscopy without the implant deployment, serving as a sham treatment. The study is randomized, double-blind, and includes parallel groups to compare outcomes over time. Throughout the study, participants will be assessed on changes in their International Prostate Symptom Score IPSS over 12 months. Researchers will monitor symptom improvements at 3, 6, and 12 months to evaluate response rates. Participants will complete study visits and follow the protocol to help researchers gather data on the devices safety and impact on urinary symptoms.

This research aims to evaluate the effects of different doses of vosoritide and compare the therapeutic dose of vosoritide to human growth hormone hGH in children diagnosed with idiopathic short stature ISS. The study is a Phase 2, randomized, controlled trial that seeks to understand how these treatments influence growth in affected children. Participants will first undergo a minimum 6-month observation period to assess their baseline growth. Then, those assigned to the vosoritide and placebo groups will receive randomized treatment for at least 6 months, with placebo limited to a maximum of 6 months. After this, open-label vosoritide treatment will continue until participants reach near-final adult height or at least 16 years for females and 18 years for males, whichever is later. Participants randomized to the hGH group will receive open-label hGH treatment for a minimum of 4 years. Study treatments involve daily injections. Throughout the study, participants will attend regular visits for clinical and imaging assessments, including evaluations of hips and lower extremities. Researchers will monitor safety concerns such as hypotension, fractures, and slipped capital femoral epiphysis, with oversight from an independent Data Monitoring Committee. The main outcomes measured include changes in annualized growth velocity at 6 months and height changes over 4 years. Follow-up assessments will continue as needed, including safety monitoring, until study completion, which could last up to 15 years.

Researchers are evaluating multiple investigational therapies in adult participants with moderately to severely active Crohns Disease or Ulcerative Colitis, which are types of Inflammatory Bowel Disease IBD. This Phase 2 platform study aims to assess the safety, effectiveness, how the body processes the drugs, and their effects on the disease. The study is sponsored by Mirador Therapeutics, Inc. and is designed to explore several oral or intravenous experimental treatments. Participants will be assigned to receive one of several treatments including MT-501 tablets or multiple intravenous doses of MT-201 combined with standard care. Different groups will receive these therapies to compare their effects. The treatment period lasts up to 13 weeks, during which participants will be closely monitored for responses and side effects. During the study, participants will undergo assessments including endoscopy and clinical evaluations to measure disease activity and response. Laboratory tests will monitor safety, and pharmacokinetics will track how the drugs are absorbed and processed. Researchers will observe treatment side effects and disease improvement using specific clinical and endoscopic measures over 12 to 13 weeks. The total participation duration corresponds to the treatment and monitoring period outlined.

Researchers are evaluating the safety and immune response of a second dose of the RSVpreF vaccine in pregnant women during later pregnancies. The study also aims to understand how long the protection lasts from a single dose given during a previous pregnancy by examining blood samples from nonpregnant participants who had the vaccine before. This Phase 3 trial involves pregnant participants and their unborn babies, as well as nonpregnant participants previously vaccinated. Pregnant participants are divided into two groups one group who previously received RSVpreF in a Pfizer trial will get a second dose, and another group will be randomly assigned to receive either RSVpreF or a placebo if no prior antibody data exists. Infants born to these participants will be followed for six months to monitor safety and antibody levels. Nonpregnant participants who received RSVpreF in an earlier trial will provide blood samples to assess the durability of their immune response without receiving additional vaccine doses. Participants will undergo safety monitoring including tracking local and systemic reactions, adverse events, and serious events during pregnancy and after birth for infants. Blood tests will measure neutralizing antibodies against RSV types A and B at birth, 3 months, and 6 months. The study includes consent procedures for both pregnant participants and infants, and nonpregnant participants will have scheduled visits for blood sample collection. The total study duration extends through infant follow-up and ongoing immune response assessments.

Researchers are evaluating Rezpegaldesleukin, a drug being studied for patients aged 12 years and older with moderate to severe atopic dermatitis. This Phase 3, randomized, double-blind study compares Rezpegaldesleukin to a placebo to assess its effect on the condition. The study is designed as a parallel group trial and aims to understand treatment outcomes over approximately 15 months. Participants receive Rezpegaldesleukin injections subcutaneously during an induction period every 2 weeks and then during a maintenance period at varying intervals of every 4 or 12 weeks. Some participants will receive placebo injections on a similar schedule during the induction and maintenance periods. An open-label escape phase allows dosing frequency to be adjusted by the investigator. The study includes both blinded and open-label phases to monitor responses. During the study, participants will undergo regular assessments including Investigators Global Assessment IGA scores, Eczema Area and Severity Index EASI scores, and symptom rating scales for itch, skin pain, and sleep impact. Researchers will also track asthma control and sinusitis symptoms in applicable patients. Safety is monitored through reports of adverse events throughout the study. The total participation time is about 15 months, with key evaluations at weeks 0, 6, 24, and ongoing safety follow-ups.

Researchers are investigating two different doses of inhaled Glycopyrronium GP delivered by metered-dose inhaler MDI as add-on treatments to Budesonide and Formoterol Fumarate BFF MDI in children aged 4 to less than 12 years with asthma. This Phase II, multi-center, randomized, double-blind, crossover study aims to assess how these doses affect lung function compared to a placebo MDI added to BFF MDI. The study is designed to better understand the potential benefits and safety of these treatments in this young population with asthma. Participants will undergo a 3-week run-in period followed by three 3-week treatment periods. During the treatment periods, each participant will receive three treatments in different sequences BFF MDI combined with GP MDI Dose A, BFF MDI combined with GP MDI Dose B, and BFF MDI combined with placebo MDI. GP MDI and BFF MDI are both administered twice daily by oral inhalation. The study concludes with a safety follow-up visit 12 to 16 days after the last dose. Throughout the study, lung function will be closely monitored, with the primary outcome being the change from baseline in forced expiratory volume in one second FEV1 at 1-hour post-dose at the end of each 3-week treatment period. Additional measures include morning pre-dose trough FEV1 changes. Participants will attend scheduled visits for assessments, including lung function tests and safety evaluations, ensuring detailed monitoring of treatment effects and participant well-being during the study period.

Researchers are conducting a Phase 3 pediatric study to create a framework for evaluating the safety and effectiveness of drugs for managing obesity or overweight conditions in children and teens. The study focuses on participants who have struggled to lose weight despite structured diet and exercise programs. The goal is to assess treatments for long-term weight management in this young population. Participants will receive either the study drug Orforglipron or a placebo orally. The interventions will be detailed in specific substudies, called ISAs, which may start independently as new treatments become available. Results from all ISAs will be reported once all are completed, providing insights into the treatments effects on pediatric obesity or overweight. During the study, participants will be evaluated from baseline to week 72, with the primary outcome measuring the number of participants assigned to each ISA. Participants will undergo assessments including body mass index evaluations based on age- and gender-specific growth charts, and monitoring for weight-related health conditions. Safety and efficacy data will be collected throughout the study duration, which extends to March 2027.

Researchers are evaluating efruxifermin EFX in a phase 3, randomized, double-blind, placebo-controlled study involving adults with compensated cirrhosis caused by NASH Nonalcoholic Steatohepatitis or MASH Metabolic Dysfunction-Associated Steatohepatitis. This study aims to assess the safety and effectiveness of EFX in preventing significant clinical events such as disease progression and liver decompensation over a period of up to 5 years. Participants are randomly assigned to receive either efruxifermin 50 mg or a placebo, both given by subcutaneous injection. The study includes two cohorts one with biopsy-proven compensated cirrhosis and specific metabolic scores, and another with biopsy-proven or non-invasive diagnosis of compensated cirrhosis. The study treatment and monitoring extend up to 5 years, with evaluations at 96 weeks and long-term follow-up to track liver fibrosis, markers of liver injury, insulin sensitivity, glycemic control, body weight, and safety outcomes. During the trial, participants undergo regular assessments including laboratory tests, ECGs, ultrasounds, and vital sign monitoring. Researchers will measure changes in liver fibrosis, steatohepatitis resolution, and metabolic markers throughout the study. Safety and tolerability are closely tracked by documenting adverse events and exposure duration. The study duration allows for long-term observation of treatment effects and disease progression, with participant involvement lasting up to 5 years.

Researchers are evaluating the effects of a triple therapy inhaler combining budesonide, glycopyrronium, and formoterol fumarate BGF MDI 32014.49.6 g compared to a dual therapy inhaler with glycopyrronium and formoterol fumarate GFF MDI 14.49.6 g on heart and lung outcomes in adults with Chronic Obstructive Pulmonary Disease COPD who have a higher risk for heart and lung events. This Phase III study is randomized, double-blind, and conducted at multiple centers, focusing on participants with COPD and elevated cardiopulmonary risk. Participants will receive either the triple therapy inhaler or the dual therapy inhaler, both administered twice daily. The study compares these two inhalers over a period of up to three years, monitoring for serious cardiac or COPD events. The trial includes careful evaluation of various heart and lung-related health events during this period. During the study, participants will be closely monitored through regular visits, assessments, and tests to measure lung function, heart events, and COPD exacerbations. Researchers will track the time until the first severe cardiac or COPD event and evaluate other cardiovascular and respiratory outcomes over up to three years. Participants will also be assessed for their ability to properly use the inhaler and adherence to the study protocol throughout the trial.