Sahinbey

This research aims to investigate the frequency of Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) in children aged 2 to 6 who exhibit nonspecific neurological symptoms such as idiopathic seizures, speech disorders, and motor dysfunctions. The study targets children without hypoxic ischemic encephalopathy, head trauma, or developmental brain anomalies who visit Pediatric Metabolism, Neurology, and Developmental Pediatrics clinics over a 12-month enrollment period. It is a multicenter, non-drug screening study designed to better understand CLN2 disease among this population. During the study, demographic data, medical and family history will be collected at the first visit. Assessments will include seizure frequency, cognitive and language development evaluations, physical exams focusing on muscle strength, gait, and coordination, as well as neurological tests such as EEG and MRI scans. Children showing specific neurological signs or imaging findings will have blood samples taken for Tripeptidyl Peptidase 1 enzyme measurement. Those with low enzyme activity will undergo genetic testing to investigate CLN2 disease. Participants will be monitored for disease frequency over one year. Researchers will measure clinical and demographic characteristics including speech impairment, motor symptoms, EEG responses, and brain imaging changes. Safety monitoring includes obtaining informed consent and following up on enzyme and genetic test results. Total participation spans from initial screening through diagnostic testing and data collection during the 12-month study period.

Researchers are investigating the similarity in pharmacokinetic (PK) profile, effectiveness, safety, and immune response of HLX17 compared to US-sourced Keytruda® in patients who have had surgery for non-small cell lung cancer, melanoma, or renal cell carcinoma. This Phase I, multicenter, randomized, double-blind study aims to compare these two treatments in people with these resected solid tumors to better understand their performance and safety profiles. Participants will receive either HLX17 or US-sourced Keytruda®, each given at a dose of 200 mg on the first day of every 3-week cycle. The study is designed with parallel groups, where each participant receives one of the treatments across multiple cycles. The dosing schedule continues through six cycles, and the two treatments are directly compared under controlled conditions. Throughout the study, participants will be monitored closely with various assessments including laboratory tests and evaluations of organ function to ensure safety. The main outcomes measured are drug exposure over time from the first dose to 21 days after the initial and sixth doses. Participants are expected to have a performance status of 0 and a life expectancy of at least 12 weeks. Safety and immunogenicity will also be evaluated, with follow-up to monitor any side effects or immune responses during and after treatment.

Researchers are investigating the effectiveness, safety, and tolerability of combining baxdrostat with dapagliflozin compared to dapagliflozin alone in people with chronic kidney disease (CKD) and high blood pressure. This Phase III, international, multicenter, double-blind, placebo-controlled study aims to see if this combination reduces risks such as significant kidney function decline, kidney failure, heart failure events, or cardiovascular death. The study includes a 4-week run-in period where participants not previously treated with SGLT2 inhibitors receive dapagliflozin alone. After this, participants are randomly assigned to receive either baxdrostat plus dapagliflozin or placebo plus dapagliflozin in a double-blinded manner. Study visits occur frequently initially (at 2, 4, 8, 16, 34, and 52 weeks after randomization) and then approximately every 4 months. If participants stop the blinded treatment early, they continue dapagliflozin alone unless specific criteria require its discontinuation. Participants will undergo regular assessments including blood pressure monitoring and laboratory tests related to kidney function and cardiovascular health. The primary outcome measures the reduction in risk of major kidney and heart events over up to 37 months. Even if participants stop the study treatment, they will continue follow-up visits and data collection to ensure comprehensive safety and efficacy evaluation throughout the study duration.

Researchers are evaluating the safety and effects of different doses of a new medicine called NNC0519-0130 on kidney function in adults with chronic kidney disease, some of whom may also have type 2 diabetes, and who are living with overweight or obesity. The study compares NNC0519-0130 with semaglutide, an existing medicine, and a placebo, which is a "dummy" treatment. This is a Phase 2 proof-of-concept and dose-finding study aimed at understanding how these treatments may reduce kidney damage. Participants will be randomly assigned to one of three groups receiving either NNC0519-0130, semaglutide, or placebo. All treatments are given by subcutaneous injection once weekly. The study treatment phase lasts up to 36 weeks, with assessments at weeks 12, 24, and 36 to monitor changes in kidney damage by measuring the urinary albumin-to-creatinine ratio. The overall study duration can be up to 43 weeks. During the study, participants will be regularly monitored through laboratory tests and clinical evaluations to assess kidney function and safety. Researchers will measure changes from the start of the study in the urinary albumin-to-creatinine ratio at multiple time points. Participants will also need to have stable doses of certain blood pressure medications before joining. Safety and treatment effects will be assessed throughout the study period.

This research aims to evaluate the effectiveness and safety of two different dose schedules of pegozafermin compared to a placebo in adults with metabolic dysfunction-associated steatohepatitis (MASH) who have liver fibrosis at stage F2 or F3. This phase 3 study focuses on improving liver fibrosis and steatohepatitis in this patient group, which involves chronic liver disease associated with metabolic dysfunction. Participants will receive either pegozafermin or a placebo through subcutaneous injections. The study compares two doses of pegozafermin to assess their impact on liver fibrosis and steatohepatitis. The treatment period lasts up to 52 weeks, with outcomes measured at this time point. During the study, participants will be monitored for improvements in liver fibrosis and resolution of steatohepatitis without worsening fibrosis by week 52. Researchers will also track the time until any disease progression occurs, up to 5 years. Throughout the trial, safety and efficacy will be carefully assessed through clinical evaluations and laboratory tests to ensure participant well-being.

Researchers are evaluating how well elacestrant works compared to standard endocrine therapy in adults with node-positive, Estrogen Receptor-positive (ER+), Human Epidermal Growth Factor-2 negative (HER2-) early breast cancer who are at high risk of the cancer returning. This is a Phase 3 global, multicenter, randomized, open-label study focusing on participants who have had early invasive breast cancer removed and meet specific receptor and risk criteria. The study aims to understand which treatment better prevents invasive breast cancer over up to five years. Participants will receive either elacestrant or one of several standard endocrine therapies, including anastrozole, letrozole, exemestane, or tamoxifen, all given as oral tablets. Treatments will be administered according to the study plan, with careful monitoring throughout the trial. The study includes adults who have already received between 24 and 60 months of prior endocrine therapy, with or without certain inhibitors, and who have completed or stopped these treatments as required. During the study, participants will be monitored for invasive breast cancer-free survival for up to five years. Researchers will perform regular assessments to track treatment effects, side effects, and cancer recurrence. The study also includes safety monitoring and may involve additional tests or evaluations as needed to ensure participant well-being throughout the trial.

Researchers are evaluating the effectiveness of Saruparib (AZD5305) compared to placebo when added to a standard radiation therapy (RT) and androgen deprivation therapy (ADT) regimen in men with high-risk and very high-risk localized or locally advanced prostate cancer who have a BRCA gene mutation. This phase III study aims to assess whether Saruparib can improve metastasis-free survival in this population. About 700 adult male participants will be randomly assigned to receive either Saruparib or placebo along with ADT. There are two groups: Cohort A includes 400 participants with newly diagnosed high-risk or very high-risk prostate cancer treated with primary RT or with high-risk biochemical recurrence after radical prostatectomy receiving salvage RT. Cohort B includes 300 participants with very high-risk locally advanced prostate cancer receiving primary RT combined with ADT and abiraterone. Saruparib and placebo will be given orally, and standard ADT and abiraterone with prednisone/prednisolone will be administered as per the regimen. Participants will be followed for up to about 93 months to monitor metastasis-free survival and overall safety. Assessments include imaging scans like CT, MRI, bone scans, and PSMA-PET to confirm disease status. The study also monitors organ function, performance status, and treatment adherence. An independent committee will review safety and efficacy data throughout the trial to ensure participant well-being and study integrity.

Immunoglobulin A nephropathy (IgAN) is a kidney disease caused by the build-up of immune protein complexes in the kidneys, leading to inflammation and possible kidney damage. This Phase 3 study is evaluating how well mezagitamab, compared to a placebo, reduces protein levels in the urine (proteinuria) in adults with primary IgAN. It also aims to assess the safety and tolerability of mezagitamab and its ability to maintain kidney function over the long term. Participants will be randomly assigned to one of two groups in the main study: two-thirds will receive mezagitamab injections under the skin, and one-third will receive placebo injections that look identical but have no active medicine. Treatment will occur in two 1-year cycles, each including about six months of dosing and six months of observation with monthly check-ups. An open-label group will include a small number of participants with lower proteinuria or kidney filtering issues, including those who previously received mezagitamab in another study; these participants will receive mezagitamab similarly to the main group. During the study, participants will visit the clinic several times for assessments. Researchers will monitor changes in proteinuria from the start through week 36, along with safety and kidney function. They will also perform regular evaluations and check-ups throughout each treatment and observation period to track participants' health and response to treatment.

Researchers are evaluating how well nipocalimab works compared to a placebo in adults with moderate to severe systemic lupus erythematosus (SLE), a chronic disease where the immune system attacks healthy tissues causing swelling and redness in various organs. This is a Phase 3, randomized, double-blind, placebo-controlled, multicenter study focused on adults aged 18 to 75 who have active SLE symptoms and have been diagnosed for at least 24 weeks. Participants will receive either nipocalimab or a placebo alongside standard of care treatments, which include protocol-defined topical and systemic therapies. Nipocalimab and placebo are administered as drugs while maintaining background treatments. The study monitors participants over time, including a primary outcome measurement at Week 52 to assess the percentage of participants achieving a systemic lupus erythematosus responder index (SRI)-4 composite response. During the study, participants will be regularly assessed for disease activity, vital signs, and safety. Screening includes physical examinations, medical history review, vital signs, and electrocardiograms. Researchers will monitor disease activity scores and evaluate response to the treatment at Week 52. Safety is closely observed throughout the study, with particular attention to any adverse reactions or changes in health status. The total participation and follow-up extend at least through Week 52.

Researchers are evaluating the safety and effectiveness of a new oral medicine called vepugratinib compared with a placebo in adults with advanced or metastatic urothelial carcinoma, a type of bladder cancer that has a specific FGFR3 genetic alteration. This Phase 3 study aims to see if vepugratinib combined with two other drugs, enfortumab vedotin (EV) and pembrolizumab, can improve treatment outcomes for people who have not received prior systemic therapy for their cancer. Participants will receive either vepugratinib or placebo taken orally alongside enfortumab vedotin and pembrolizumab, both administered by intravenous infusion. The study is randomized, double-blind, and placebo-controlled to ensure reliable comparison between the vepugratinib and placebo groups. Treatment and monitoring will continue for up to approximately 6 years, allowing long-term assessment of safety and treatment effects. During the study, participants will be regularly evaluated for treatment-related side effects, response rates, and how long the cancer remains controlled without progression. Researchers will use established criteria to measure tumor response and will conduct thorough safety monitoring over the entire study period. Participation may last up to six years, during which participants will undergo laboratory tests, imaging, and clinical assessments to track their health and treatment response.