Farmington

Researchers are evaluating the efficacy and safety of rilvegostomig compared to pembrolizumab as first-line treatments for patients with metastatic non-small cell lung cancer (mNSCLC) whose tumors have high PD-L1 expression. This Phase III, randomized, double-blind, and global study focuses on participants with stage IV mNSCLC who do not have certain genetic mutations or rearrangements and are eligible for systemic therapy. Participants receive either rilvegostomig or pembrolizumab intravenously on Day 1 of each 21-day cycle. The study compares these two biological treatments given as monotherapy. Both groups will be monitored over time to assess treatment impact and safety. Throughout the study, participants undergo evaluations including tumor measurements by CT or MRI, performance status assessments, and organ function tests. Researchers will measure overall survival and progression-free survival for up to approximately five years. Tumor samples are collected before treatment for central testing, and participants’ health and treatment responses are closely followed during the trial period.

Researchers are evaluating the feasibility and preliminary effectiveness of a new digital health tool called Prioritize Personalize Prescribe EXercise (P3-EX) for physicians to use when prescribing exercise to patients with cardiovascular disease (CVD) risk factors such as obesity, hypertension, dyslipidemia, and diabetes. This pilot randomized controlled trial will involve 24 physicians and 48 patients, aiming to compare P3-EX with the American College of Sports Medicine Physical Activity Vital Sign (ACSM-PAVS) method. The study addresses the challenge that many patients with CVD risk factors do not receive exercise advice due to barriers faced by physicians, including lack of time, training, and tools. Physicians will each prescribe exercise to two patients, one receiving the P3-EX exercise prescription and the other receiving the ACSM-PAVS prescription, in a randomized crossover design. Patients will follow a 12-week unsupervised exercise program with virtual weekly support from University of Connecticut Graduate Research Assistants. Participants will receive a detailed exercise information packet, record their daily exercise in a diary, and attend two virtual guidance visits during the intervention. Weekly progressive exercise goals and summary reports will be emailed to patients to support adherence. Participants will attend multiple in-person visits for assessments before and after the exercise program, including cardiovascular risk factor measurements, physical activity monitoring using accelerometers, blood tests, and questionnaires on exercise adherence and satisfaction. Physicians and patients will also complete usability and satisfaction surveys within 48 hours after receiving the exercise prescriptions. The study will monitor exercise adherence through diaries and virtual oversight over the 12 weeks, aiming to inform the feasibility and user satisfaction of P3-EX and explore its potential to improve cardiovascular health and physical activity levels.

Researchers are conducting a Phase 1/2, multicenter, open-label study to assess the safety, tolerability, and effectiveness of BEAM-301 in adults with Glycogen Storage Disease Type Ia (GSDIa) who have specific genetic variants (homozygous or compound heterozygous for the G6PC1 c.247C>T (p.R83C) variant). The study aims to find the best biological dose of this treatment for these patients. BEAM-301 is designed to correct the faulty gene by changing a specific DNA base pair, restoring the enzyme G6Pase-α activity. Participants will receive a single intravenous dose of BEAM-301. The study evaluates this treatment's safety and tolerability by monitoring adverse events, including serious side effects and dose-limiting toxicities, through 24 months after administration. This approach helps researchers explore how the body responds to different doses and determine the optimal treatment level. During the study, participants will be closely monitored for the occurrence of treatment-emergent adverse events and other safety concerns. Genetic testing confirms eligibility, and assessments include monitoring episodes of low blood sugar and liver health. The total participation duration includes follow-up through month 24 to evaluate the safety and effects of BEAM-301.

Researchers are investigating whether producing sperm after a very short abstinence period of 1 hour can improve the rate of embryos with normal chromosomes during in vitro fertilization (IVF). This study focuses on couples experiencing infertility, including male factor infertility, and aims to compare embryo chromosome normality rates when using sperm collected after ultrashort abstinence versus the standard 2-5 days of abstinence. The study is motivated by earlier findings suggesting that shorter abstinence may enhance sperm quality and IVF outcomes. During the IVF process, participants will provide two sperm samples on the day of egg retrieval: one after 2-5 days of abstinence (standard) and a second one 1 hour later (ultrashort abstinence). Eggs retrieved will be randomly divided so that half are fertilized with sperm from the standard abstinence sample and half with sperm from the ultrashort abstinence sample. The goal is to assess which sperm collection timing leads to a higher rate of embryos with normal chromosomes. Both types of sperm samples will be used in fertilization procedures, either standard insemination or intracytoplasmic sperm injection (ICSI). Participants will be monitored through IVF treatment and embryo testing, including genetic screening for chromosomal normality (euploidy). Researchers will collect data on embryo quality, pregnancy rates after embryo transfer, and the rate of chromosomally normal embryos within 2 to 4 weeks of the IVF cycle. The study requires participants to comply with all procedures, including providing informed consent and meeting specific criteria about age, egg retrieval numbers, and sperm sample production. Safety and pregnancy outcomes will also be tracked during the study period.

Researchers are evaluating the long-term safety and effectiveness of etavopivat, a new oral medicine being developed to treat inherited blood disorders such as sickle cell disease and thalassemia. These disorders affect hemoglobin, the protein responsible for carrying oxygen in the body. This phase 3 study aims to monitor how well etavopivat works and its safety profile over an extended period. Participants will receive one of three forms of etavopivat (A, B, or C) as oral doses. The study is open-label and multicenter, involving adults, adolescents, and children who have previously completed treatment in an etavopivat parent study and continue to benefit clinically. The treatment period can last up to 264 weeks but may end earlier if etavopivat is approved in the participant's country. During the study, researchers will track the number of treatment-emergent adverse events and adverse reactions for each participant by indication and age group from baseline through the end of the study, which can last up to 316 weeks. Participants' safety and response to long-term treatment will be closely monitored throughout this period.

Researchers are evaluating how well the drug JNJ-79635322 works compared to an anti-B-cell maturation antigen (BCMA)xCD3 bispecific antibody in adults with relapsed or refractory multiple myeloma. This phase 3 study includes participants who have received at least three prior treatments including a proteasome inhibitor, an immunomodulatory drug, and an anti-CD38 antibody. The study aims to compare the effectiveness of these two treatments in this patient population. The study involves two treatment groups receiving either JNJ-79635322 or Teclistamab, both given as subcutaneous injections. Participants must have measurable disease and evidence of disease progression or lack of response to their most recent therapy. The study excludes those with certain infections, central nervous system involvement, allergies to the study drugs, recent major surgery, or recent live vaccine receipt. Participants will be monitored for overall response rate and progression-free survival for up to five years and four months. Throughout the study, performance status will be assessed, and participants will be regularly evaluated for safety and treatment response. The total duration of participation and follow-up allows for long-term evaluation of treatment effects and disease progression.

This research aims to understand the safety, effectiveness, and overall treatment experience of participants prescribed BRIUMVI4 (ublituximab-xiiy) in a real-world setting. The study focuses on people living with relapsing multiple sclerosis (RMS), a form of multiple sclerosis characterized by episodes of new or increasing neurological symptoms. It is designed to gather detailed insights from actual use outside of controlled clinical trials. Participants in this study are those who have been prescribed BRIUMVI4 but have not yet received their first infusion at the start of the study. There is no intervention assigned by the study itself; instead, it observes the outcomes and experiences of patients treated with BRIUMVI4 as part of their routine care over time. Throughout the study, researchers will track the annualized relapse rate (ARR) up to week 96 to measure disease activity. Participants' safety, treatment adherence, and experiences will be evaluated through regular monitoring, including any adverse events. The total duration of participation covers up to 96 weeks, allowing for a comprehensive understanding of long-term treatment effects and patient-reported outcomes.

Researchers are evaluating the safety and effectiveness of two doses of inhaled pirfenidone (called AP01) compared to a placebo in people with progressive pulmonary fibrosis (PPF). This Phase 2b study is randomized, double-blind, and placebo-controlled, involving up to 300 participants who will continue their standard care during the 52-week trial. The goal is to see how well AP01 works and how safe it is when added to usual treatments for PPF. Participants will be randomly assigned to one of three groups: high-dose AP01, low-dose AP01, or placebo. All treatments are given as an oral inhalation solution twice daily. The study will last for 52 weeks, during which researchers will monitor and compare the effects of these treatments on lung function and disease progression. During the study, participants will undergo various assessments including lung function tests and clinical evaluations to track their respiratory health. Researchers will check for changes in lung capacity and symptoms and monitor safety throughout the treatment period. The main outcome measured is the impact of AP01 doses compared to placebo after 52 weeks of treatment.

Researchers are evaluating the safety and effectiveness of Dostarlimab compared to a placebo in adults with locally advanced unresected Head and Neck Squamous Cell Carcinoma (HNSCC). This phase 3 randomized, double-blind, placebo-controlled study focuses on patients who have completed chemoradiation therapy with cisplatin and radiation and have no distant metastatic disease. The study requires confirmation of PD-L1 positive tumor status and specific testing for oropharyngeal carcinoma cases. Participants will receive either Dostarlimab or a placebo as an intravenous infusion following their chemoradiation treatment. The study monitors these treatments as sequential therapy to assess their impact on disease progression. Treatments are administered in a controlled, blinded manner to compare outcomes between the two groups effectively. During the study, participants will be followed for up to approximately five years to measure event-free survival, with evaluations conducted by blinded independent central review. Assessments will include monitoring for safety, disease status, and any adverse events throughout the study period. This long-term follow-up aims to provide comprehensive data on the effectiveness and safety of Dostarlimab as post-chemoradiation therapy in this patient population.

Researchers are evaluating whether adding JNJ-90301900 to standard concurrent platinum-based doublet chemotherapy with radiation therapy followed by consolidation immunotherapy can improve the objective response rate in participants with locally advanced and unresectable stage III non-small cell lung cancer. This phase 2, randomized, open-label study focuses on participants diagnosed recently with NSCLC and eligible for this combined treatment approach. Participants will receive JNJ-90301900 injected directly into tumors or lymph nodes alongside concurrent chemo/radiation therapy, which includes carboplatin and paclitaxel administered intravenously and radiation delivered by intensity modulated radiation therapy. Following this, consolidation immunotherapy with intravenous durvalumab will be given. The study evaluates this combined treatment sequence against standard care approaches. During the study, participants will undergo assessments including imaging to measure tumor response according to RECIST criteria, with the primary outcome being the objective response rate over up to 2 years and 2 months. Researchers will monitor safety and treatment effects throughout, ensuring participants meet performance status criteria and have lesions suitable for injections and radiation. The study involves ongoing evaluations guided by independent central review.