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Researchers are studying the effects of the drug leflutrozole on semen quality in men aged 18 to 49 years with low testosterone levels. This phase 2 clinical trial aims to find out whether leflutrozole can improve semen quality and what side effects participants may experience compared to a placebo. The trial is sponsored by ReproNovo Aps and focuses on testicular function and hormone levels. Participants will be randomly assigned to receive one of three doses of leflutrozole 0.05 mg, 0.1 mg, or 0.3 mg or a placebo. The medication is taken orally once a week for 16 weeks. The study is double-blind, meaning neither the participants nor the researchers know who receives the drug or placebo. Checkups and tests occur every 4 weeks during the treatment period. Men in the trial will provide semen samples to measure sperm count, motility, and other quality markers. Blood tests will monitor hormone levels such as testosterone and estradiol, as well as safety markers including prostate-specific antigen and ECG readings. Questionnaires will assess changes in libido and energy. Researchers will track adverse events throughout the 16 weeks to evaluate safety and effectiveness.

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 conducting an observational study to collect baseline growth data in children diagnosed with Idiopathic Short Stature ISS. The study aims to better understand growth patterns by measuring various growth-related factors over time in this pediatric population aged between 2 and 16 years, with a focus on children experiencing significant short stature. This long-term study is sponsored by BioMarin Pharmaceutical to help characterize how children with ISS grow compared to typical growth standards. Participants will be observed without receiving any study treatments. Growth measurements such as annualized growth velocity, height Z-score, standing height, body mass index BMI, and BMI Z-score will be collected every six months. The study will also record medical events and explore relationships between genetic variants and growth velocity. This observational approach allows tracking changes over time without intervention. Families will be involved in regular assessments every six months for up to 15 years. These evaluations include physical growth measurements and monitoring of medical events related to short stature. Data from historic growth hormone hGH stimulation tests and treatment status will also be considered. The studys primary outcomes focus on changes in growth metrics, while secondary outcomes assess medical event rates and genetic associations. Participation requires ongoing cooperation for scheduled measurements and consent from parents or guardians.

This research investigates patient preference and the safety and eye-related effectiveness of a new treatment method called Dextenza combined with an intracameral antibiotic and NSAID, compared to standard eye drop therapy after cataract surgery. The study involves patients undergoing surgery in both eyes and aims to control pain and inflammation following the procedures. Participants will be randomly assigned to receive either the experimental treatment or the standard topical therapy in their first eye surgery. The experimental group receives a Dextenza dexamethasone intracanalicular insert placed in the lower punctum during surgery, along with intracameral ketorolac and moxifloxacin administered during and after the procedure. The control group receives standard topical drops including moxifloxacin, ketorolac, and prednisolone acetate at scheduled intervals before and after surgery. Two weeks later, the second eye will be operated on and treated with the alternate treatment. During the study, participants will attend several visits for assessments including patient preference measured 28 days after the second eye surgery, ocular inflammation scores, post-operative pain ratings, visual acuity tests, physician evaluations of insertion, and monitoring for adverse events. These evaluations occur at various time points up to two months following the second surgery, enabling researchers to compare the treatments comprehensively and monitor safety and effectiveness over time.

The BEATRIX study focuses on healthy pregnant women aged 49 or younger between 24 and 36 weeks of pregnancy to evaluate the safety and immune response of a group B streptococcus GBS vaccine. Researchers aim to understand how this vaccine works in pregnant women and their babies, assessing various safety measures and immune responses related to GBS. This study is a Phase 3, randomized, placebo-controlled, double-blinded trial sponsored by Pfizer. Participants will receive a single injection of either the GBS vaccine or a placebo saline. After birth, a subset of infants will receive routine vaccines according to each countrys immunization schedule, including vaccines for diphtheria, pneumococcal disease, and others. Some infants will have blood samples taken after completing their primary and toddler vaccine doses to evaluate immune responses. Pregnant participants will visit the study site at least three to four times, with some visits possibly conducted by phone, and may stay involved for up to 14 months, including six months after delivery. Their babies will be followed for about 12 months, with a subset participating for up to 19 months. Researchers will monitor local and systemic reactions, adverse events, and antibody levels in both mothers and infants to assess safety and immunogenicity of the vaccine throughout the study period.

Researchers are studying pancreatic cystic lesions PCL to improve diagnosis using confocal laser endomicroscopy nCLE combined with endoscopic ultrasound EUS and cyst fluid analysis. This observational study involves patients referred for EUS evaluation of PCL at academic centers, aiming to assess the accuracy of diagnosing PCL and the risk stratification of intraductal papillary mucinous neoplasms IPMNs over 48 months. Participants undergo EUS-guided nCLE imaging followed by EUS-guided fine needle aspiration FNA to collect cyst fluid. The fluid is tested for carcinoembryonic antigen CEA, cytology, and molecular markers as part of standard care. Based on these results and multidisciplinary tumor board recommendations, surgery may be performed, with surgical histopathology serving as the definitive diagnosis. During the study, patients provide consent and receive EUS procedures with imaging and fluid sampling. Researchers measure diagnostic accuracy and imaging quality over 48 months. Most participants are expected to have surgical resection after EUS. The study monitors the accuracy of diagnosis and risk assessment, guiding future care for pancreatic cysts.

Researchers are evaluating the use of Cxbladder, a non-invasive urine-based test, for detecting urothelial carcinoma bladder cancer in patients with microscopic hematuria, which means blood in the urine not visible to the naked eye. The study addresses concerns about the current invasive standard procedure, cystoscopy, which can cause discomfort and anxiety, especially since the risk of bladder cancer in this group is relatively low. This observational study aims to determine if Cxbladder can help identify patients who need further investigation and reduce unnecessary invasive procedures. This multicenter study will enroll up to 1000 subjects with either microscopic or gross hematuria from US sites. Participants are patients referred to urology and scheduled for cystoscopy. During the study, participants will provide an additional urine sample either at home or in the clinic, collected within 90 days before or up to 10 weeks after cystoscopy. The study will compare Cxbladder test results with standard diagnostic procedures to validate its performance in detecting urothelial carcinoma and stratifying risk. Participants will undergo standard care tests and cystoscopy as scheduled. They will complete forms about urine sample collection dates. Researchers will collect data professionally according to clinical standards. The main outcome is to validate Cxbladders accuracy in identifying patients at low risk who might avoid invasive testing and those at higher risk needing further care. The study does not share Cxbladder results with patients or doctors. Participation involves urine sample collection and data recording, with the study lasting through the scheduled cystoscopy and sample evaluations.

Researchers are studying the unique natural history of blood sugar control, insulin sensitivity, and beta-cell function in obese adolescents at risk for developing type 2 diabetes. This observational study aims to understand how these factors and their causes change as youth transition through puberty, providing important information that could help design future treatments and prevention strategies for youth-onset type 2 diabetes. The study will carefully examine a large group of young people at risk for type 2 diabetes using detailed biochemical, clinical, and psychosocial assessments. Participants aged 8 to 15 years who meet specific criteria related to obesity, puberty stage, and blood sugar levels will be observed over an average of 2.5 years. No treatments or medications are being tested instead, the study focuses on tracking natural changes and risk factors over time. Throughout the study, participants will undergo regular evaluations to monitor blood sugar control, beta-cell function, insulin sensitivity, and fatty acid metabolism. These measures will be collected along with clinical and psychosocial data to better understand the progression toward type 2 diabetes. The research team will review these outcomes continuously during the study period, aiming to improve knowledge about diabetes risk in youth and inform future care approaches.

Researchers are evaluating the safety and effectiveness of Halneuron, given as a series of subcutaneous injections, for patients experiencing chemotherapy-induced neuropathic pain CINP. This randomized, double-blind, placebo-controlled Phase 2 study aims to compare Halneuron with placebo injections in managing pain caused by chemotherapy drugs such as platinum and taxane. Participants may have the option to continue in an open-label extension phase where all will receive Halneuron. Participants will be randomly assigned to receive either Halneuron or placebo by subcutaneous injection. After completing the randomized phase, those who choose to continue may enter an open-label extension where every participant receives Halneuron. The study includes careful monitoring during both phases to evaluate the treatments effects and safety. Throughout the study, participants will be closely monitored for pain levels using the Numeric Rating Scale NRS and safety assessments over a four-week period. Additional measurements include patient impressions of change, fatigue, sleep quality, and overall quality of life using standardized questionnaires. The total participation time includes the randomized phase and optional extension, providing ongoing evaluation of Halneurons impact on chemotherapy-induced neuropathic pain.

This research aims to develop and validate a single-gene Non-Invasive Prenatal Test sgNIPT to detect serious health conditions like cystic fibrosis, spinal muscular atrophy, sickle cell disease, and thalassemias in unborn babies. It focuses on pregnant people with higher risk pregnancies due to carrier status or affected conditions, including cases without reproductive partner screening. The study will gather blood samples and medical information from pregnant participants and, when applicable, their partners and newborns. Participants will undergo the investigational sgNIPT, which is designed for pregnant people whose fetus is at increased risk for a single-gene disorder. This includes situations where there is no partner screening, positive partner screening but no prenatal diagnostic testing, or ultrasound findings suggesting a single-gene disorder regardless of carrier status. The study will collect newborn cheek swabs and health data within six months after delivery as part of the research. During the study, participants will provide blood samples after nine weeks of pregnancy, and researchers will collect medical and genetic information from participants and their partners. Newborn health information and cheek swabs will be collected post-delivery to assess the tests performance. The primary outcome is the accuracy of the sgNIPT in detecting four main autosomal recessive disorders approximately two years after study launch, followed by evaluation of other single gene disorders about six months later. Participation involves consenting to these procedures and ongoing information sharing throughout the study period.