Farmington

Researchers are evaluating rilvegostomig compared with pembrolizumab monotherapy as a first-line treatment for people with metastatic non-small cell lung cancer mNSCLC whose tumors have high PD-L1 expression. This Phase III, global, randomized, and double-blind study aims to assess the efficacy and safety of these treatments in this patient population. The trial is sponsored by AstraZeneca and focuses on patients with specific tumor characteristics and no certain genetic mutations. Participants will receive either rilvegostomig or pembrolizumab intravenously on Day 1 of each 21-day cycle. The study has two treatment arms one for the investigational drug rilvegostomig and one for the active comparator pembrolizumab. Both treatments are given as monotherapy to understand their effects as initial therapy in this cancer setting. During the study, participants will be monitored for overall survival and progression-free survival for up to approximately 5 years. Additional assessments include tumor response, duration of response, time to second progression or death, drug pharmacokinetics, immunogenicity, and patient-reported outcomes related to physical function, quality of life, and lung cancer symptoms. The study involves regular evaluations to track treatment effects and safety over an extended period.

Researchers are evaluating a new digital health tool called Prioritize Personalize Prescribe EXercise P3-EX designed to help physicians prescribe personalized exercise plans to adults with cardiovascular disease CVD risk factors such as obesity, hypertension, dyslipidemia, and diabetes. This pilot randomized controlled trial aims to test the usability and satisfaction of P3-EX compared to a standard exercise prescription method ACSM Physical Activity Vital Sign among physicians and their patients. The study addresses barriers physicians face in prescribing exercise, including lack of time, training, and tools. Physicians recruited for the study will each recruit two patients with CVD risk factors. One patient receives a personalized exercise prescription using P3-EX, and the other receives a generic exercise program based on ACSM guidelines, delivered in a random crossover design. Patients will follow their assigned exercise plans unsupervised for 12 weeks with virtual weekly support from graduate research assistants. The P3-EX tool assesses cardiovascular risk factors, prioritizes the greatest risk, and creates a tailored Frequency, Intensity, Time, and Type FITT exercise prescription. Patients will record their exercise activities in a diary and receive weekly progress reports and guidance. Participants will attend several in-person visits for assessments including cardiovascular risk factors, physical activity levels measured by accelerometry, and blood tests. Physicians and patients will rate the feasibility and acceptability of each prescription method shortly after delivery. Patient exercise adherence and changes in cardiovascular health and physical activity will be monitored over 12 weeks. The study includes virtual visits for guidance and ends with follow-up assessments to evaluate patient satisfaction and health outcomes. The total participation time for patients covers baseline and post-intervention visits plus the 12-week exercise period.

Researchers are studying BEAM-301, an investigational treatment, in adults with Glycogen Storage Disease Type Ia GSDIa who have a specific genetic variant G6PC1 c.247CT. This Phase 12 trial aims to evaluate the safety, tolerability, and effectiveness of BEAM-301 and to find the best dose for treatment. The study is open-label and involves multiple centers. Participants will receive a single intravenous dose of BEAM-301, which contains messenger RNA and guide RNA formulated in lipid nanoparticles designed to correct the genetic mutation causing GSDIa. This dose-exploration study will assess how the treatment behaves in the body and its effects over a 24-month period. During the study, participants will be monitored for treatment-emergent adverse events and dose-limiting toxicities. Researchers will also evaluate changes in episodes of low blood sugar, metabolic stability during fasting, starch supplementation needs, and blood levels of glucose and other metabolic markers. Pharmacokinetic parameters such as peak concentration, area under the curve, and half-life of BEAM-301 will be measured. The total study duration for each participant spans up to 24 months after treatment.

Researchers are investigating whether producing sperm after a very short abstinence period of just 1 hour can improve the rates of embryos with normal chromosomes during in vitro fertilization IVF treatment. The study aims to compare this ultrashort abstinence with the current standard abstinence period of 2 to 5 days to see if it leads to better embryo quality and pregnancy outcomes. This research is focused on couples experiencing infertility, particularly male factor infertility, and is sponsored by UConn Health. Participants will provide two sperm samples on the day of egg retrieval one after the standard 2-5 days of abstinence and another after only 1 hour of abstinence. Their eggs will be randomly divided into two groups, with half fertilized using sperm from the standard abstinence sample and the other half fertilized using sperm from the ultrashort abstinence sample. Both groups will be part of the IVF process involving insemination or injection of sperm into the eggs. The study will assess embryo chromosome normality and other IVF outcomes. During the study, participants will undergo egg retrieval and fertilization procedures, with close monitoring of embryo development. Researchers will evaluate the rate of chromosomally normal embryos within 2 to 4 weeks of the IVF cycle, as well as pregnancy rates after embryo transfer, fertilization rates, and blastulation rates. Participation lasts through these IVF cycle stages, with data collected to understand how sperm abstinence timing affects embryo quality and pregnancy chances.

Researchers are studying etavopivat, a new oral medicine being developed to treat inherited blood disorders such as sickle cell disease and thalassaemia. These conditions affect haemoglobin, the protein that carries oxygen in the blood. This phase 3 open-label study aims to assess the long-term safety and effectiveness of etavopivat in adults, adolescents, and children who have completed treatment in an earlier etavopivat study. The study is sponsored by Novo Nordisk AS and may last up to 264 weeks unless etavopivat is approved earlier in the participants country. Participants will receive an oral dose of etavopivat, with dosing varying by age and condition. Those aged 12 years or older will receive either Etavopivat A or C, while children under 12 years old will receive Etavopivat B. The study includes groups with sickle cell disease or thalassaemia, some of whom may be transfusion-dependent. The treatment will be continuous throughout the study period, aiming to observe long-term effects and safety. During the study, participants will be closely monitored for treatment-emergent adverse events and adverse reactions. Researchers will track clinical outcomes such as vaso-occlusive crisis rates, hemoglobin levels, hospitalizations, and red blood cell transfusions, both at baseline and throughout treatment. Data will be collected regularly to assess safety and treatment impact across different age groups and disease types, with the total study duration potentially extending to over six years.

Researchers are evaluating JNJ-79635322 compared with an anti-B-cell maturation antigen BCMAxCD3 bispecific antibody in people with relapsed or refractory multiple myeloma who have received at least three prior treatments including a proteasome inhibitor, an immunomodulatory drug, and an anti-CD38 antibody. This phase 3 randomized study aims to understand how well these treatments work in this patient population by comparing responses and progression outcomes over time. Participants will receive either JNJ-79635322 or teclistamab, both given as subcutaneous injections. Treatment continues until the disease progresses or side effects become intolerable. The study involves two treatment arms, one for each drug, and participants are randomly assigned to one of these groups. Both treatments are administered regularly as injections under the skin. During the study, participants will undergo various assessments including response evaluations, progression-free survival checks, and monitoring of side effects for up to five years and four months. Researchers will also track quality of life, symptom changes, and laboratory results. The study includes long-term follow-up on treatment effects, survival, and time to next therapy, with careful recording of any adverse events and immune responses to the drugs.

Researchers are evaluating the real-world experience of participants treated with BRIUMVI4 ublituximab-xiiy for relapsing multiple sclerosis RMS. The study aims to assess the safety, effectiveness, and overall treatment experience of this medication in everyday clinical practice. The trial is observational, focusing on patients who have been prescribed BRIUMVI and tracking their outcomes over time. Participants in this study will receive BRIUMVI4 as an intravenous IV infusion specifically for RMS treatment. The study includes participants who have not previously received BRIUMVI infusions before the study begins, allowing observation from the start of their treatment. This approach helps understand how the medication performs outside of controlled clinical trial settings. During the study, researchers will monitor participants for up to 96 weeks, measuring the annualized relapse rate ARR to evaluate disease activity. They will also track adverse events, serious adverse events, and infusion-related reactions at each infusion visit. Participants will undergo regular assessments to capture their health status and any side effects, providing a comprehensive view of treatment safety and patient experience over nearly two years.

Researchers are evaluating the safety and effectiveness of two doses of inhaled pirfenidone AP01 compared to a placebo, alongside standard care, in people with progressive pulmonary fibrosis PPF over a 52-week period. This randomized, double-blind, placebo-controlled Phase 2b study aims to better understand treatment options for individuals with PPF, a condition characterized by worsening lung fibrosis despite existing therapies. Participants will be randomly assigned to one of three groups a high dose of AP01 twice daily, a low dose of AP01 twice daily, or a placebo inhalation twice daily. The study involves taking inhaled treatments using a pirfenidone solution or placebo, with the goal to evaluate the impact of these treatments on lung function and disease progression over one year. Throughout the study, participants will undergo regular assessments including lung function tests, imaging scans, and quality of life evaluations. Researchers will monitor the effect of the treatments on lung fibrosis, respiratory symptoms, and safety measures. The total participation lasts for 52 weeks, during which participants will attend scheduled visits and complete various tests to track their health and response to the study treatments.

Researchers are evaluating Dostarlimab compared to a placebo in adults with locally advanced unresected Head and Neck Squamous Cell Carcinoma HNSCC. This phase 3 trial aims to assess the safety and effectiveness of Dostarlimab as a sequential therapy following chemoradiation treatment in participants with this type of cancer. Participants are randomly assigned to receive either Dostarlimab or a placebo, both given as intravenous infusions. The study is double-blind, meaning neither the participants nor the researchers know which treatment is being given. The treatments follow completion of chemoradiation with cisplatin and radiotherapy intended to cure the cancer. During the study, participants will be monitored for up to approximately 5 years. Researchers will evaluate event-free survival and overall survival, with safety assessments including treatment-emergent adverse events and laboratory tests. Blood samples will be taken to measure drug levels and immune responses. This long-term follow-up will help understand the effects and safety of Dostarlimab after chemoradiation therapy.

Researchers are evaluating whether adding JNJ-90301900 to standard treatment can improve the response rate in people with locally advanced and unresectable stage III non-small cell lung cancer NSCLC. The study focuses on combining this investigational drug with platinum-based chemotherapy and radiation therapy, followed by immunotherapy consolidation. This phase 2 trial aims to assess both the safety and effectiveness of this combination approach compared to standard therapy alone. Participants receive JNJ-90301900 injected directly into tumors or nearby lymph nodes at varying volumes 22 or 33 of gross tumor volume along with concurrent chemotherapy and radiation therapy cCRT. Following this, they undergo consolidation immunotherapy with durvalumab. The study includes experimental groups receiving these injections plus cCRT and consolidation immunotherapy, as well as a control group treated with cCRT and consolidation immunotherapy without JNJ-90301900. During the trial, participants will be closely monitored with independent central reviews assessing objective response rates up to 2 years and 2 months. Additional measures include progression-free survival, duration of response, and safety evaluations such as treatment-emergent adverse events and laboratory tests. The study duration allows detailed tracking of tumor response, disease control, and participant health throughout and after treatment.