Yonago

This research aims to observe and track growth patterns and the clinical progression of Hypochondroplasia HCH in children. It focuses on collecting various growth measurements and other relevant data over time to better understand this condition. The study is sponsored by BioMarin Pharmaceutical and involves children diagnosed with Hypochondroplasia, up to 15 years old. Participants include children confirmed to have Hypochondroplasia through genetic testing. The study is observational, meaning there are no experimental treatments involved. Children will be monitored regularly to record changes in growth and body proportions, with data collection occurring every six months over the course of the study, which may last up to 15 years. Throughout the study, children will undergo assessments every six months to measure growth velocity, height, body mass index BMI, and body segment ratios. Quality of life and severity impressions from both patients and caregivers will be evaluated annually. Researchers will also track medical events, use of growth hormone treatments, and any limb lengthening surgeries. This long-term follow-up allows for comprehensive monitoring of growth and health outcomes in children with Hypochondroplasia.

Researchers are comparing two treatment combinations for adults with advanced nonsquamous non-small cell lung cancer NSCLC that have a specific KRAS p.G12C mutation and are negative for PD-L1 expression. The study aims to evaluate progression-free survival and overall survival between participants receiving sotorasib with platinum doublet chemotherapy and those receiving pembrolizumab with platinum doublet chemotherapy. This phase 3, randomized, open-label trial is led by Amgen and includes participants with stage IV or advanced stage IIIBC NSCLC. Participants will be randomly assigned to receive either sotorasib orally combined with carboplatin and pemetrexed, or pembrolizumab intravenously combined with the same chemotherapy drugs. These treatments are given as front-line therapy. The study includes a treatment period with these drug combinations and monitoring for outcomes such as response rates and quality of life over several years. During the study, participants will be regularly assessed through various measures including survival status, tumor response, and quality-of-life questionnaires focusing on lung cancer symptoms. Researchers will monitor safety by tracking adverse events, vital signs, and laboratory tests. Treatment concentrations of sotorasib will also be measured up to 64 days after starting. The total study duration includes follow-up for up to approximately 5.5 years to fully evaluate treatment effects and outcomes.

Researchers are evaluating whether combining ASP-1929 photoimmunotherapy PIT with pembrolizumab improves treatment outcomes for patients with locoregional recurrent squamous cell carcinoma of the head and neck HNSCC without distant metastases. This phase 3 randomized study aims to compare the effectiveness and safety of ASP-1929 PIT plus pembrolizumab against pembrolizumab alone or pembrolizumab combined with chemotherapy as first-line treatments. The main goal is to see if the combination improves overall survival for eligible patients. Participants will be randomly assigned in a 221 ratio to one of three treatment groups ASP-1929 PIT at a dose of 320 mgm plus pembrolizumab, ASP-1929 PIT at 640 mgm plus pembrolizumab, or a control group receiving standard care with either pembrolizumab alone or pembrolizumab plus chemotherapy according to the physicians choice. ASP-1929 PIT involves an intravenous infusion followed by illumination of the tumor with red light using a special laser system. Pembrolizumab is given as an intravenous infusion every 3 or 6 weeks. Chemotherapy options include carboplatin, cisplatin, 5-fluorouracil, paclitaxel, or docetaxel for up to 6 cycles. Treatment with ASP-1929 PIT and pembrolizumab may continue for up to 24 months. During the study, participants will undergo regular assessments including imaging evaluations to measure tumor response, safety monitoring for adverse events, and quality of life questionnaires. Researchers will track overall survival and other outcomes such as response rates and progression-free survival for up to about 48 months. Pharmacokinetic and immunogenicity tests will also be conducted in the experimental arms. Participants will have ongoing evaluations throughout the treatment period and follow-up to monitor effectiveness and safety.

Researchers are evaluating if adding LY3537982 olomorasib to standard anti-cancer drugs improves treatment for participants with untreated advanced non-small cell lung cancer NSCLC that has a specific KRAS G12C gene change. This Phase 3 treatment study includes participants with locally advanced or metastatic NSCLC and aims to compare this combination against standard care. The study is sponsored by Eli Lilly and Company and could last up to 3 years depending on individual response and disease progression. Participants receive LY3537982 orally combined with pembrolizumab given intravenously in 21-day cycles. Some groups also receive chemotherapy drugs pemetrexed and platinum cisplatin or carboplatin intravenously. There are different dose levels and combinations being tested, including placebo groups for comparison. Treatment continues until specific discontinuation criteria are met. Parts of the study are randomized and double-blinded, with some parts non-randomized for safety lead-in. During the study, participants have regular assessments including imaging scans to measure tumor response, blood tests, and questionnaires about symptoms and quality of life. Researchers monitor side effects and survival outcomes. The main measures include progression-free survival and treatment-emergent adverse events over about one year, with overall survival followed for up to three years. Participants are closely followed throughout treatment and after to evaluate the effects and safety of the study medications.

Researchers are studying repotrectinib TPX-0005 in adults and adolescents with advanced solid tumors that have specific gene rearrangements in ALK, ROS1, or NTRK1-3. The trial aims to find the safest and most effective dose in Phase 1, and then evaluate how well the drug works in Phase 2 across different patient groups with these gene changes. This includes patients with tumors that have spread and those with brain involvement, focusing on response rates and survival outcomes. The study involves oral doses of repotrectinib. Phase 1 includes dose escalation to determine dose-limiting toxicities, maximum tolerated dose, and recommended dose for Phase 2. A sub-study also examines drug interactions with midazolam. In Phase 2, participants are assigned to one of six groups based on their tumor type and prior treatments, including different lines of targeted therapy and chemotherapy. Treatments continue according to protocol with regular monitoring. Participants will have measurable tumors confirmed by imaging reviewed centrally. They undergo physical exams, lab tests, and safety assessments throughout the study. Researchers measure response rates, duration of response, progression-free survival, overall survival, and clinical benefit over several years. The study monitors drug levels in blood and evaluates safety closely. Participation may last years with follow-up to assess long-term outcomes and effects on brain metastases.

Researchers are evaluating the effects of efgartigimod on thyroid function in adults with Graves Disease, focusing on its safety, how it is processed in the body, and its impact on antibody levels and the immune system. This Phase 3 study involves a randomized, double-masked, placebo-controlled design to better understand treatment outcomes for participants whose disease is not well controlled with antithyroid drugs. Participants may receive efgartigimod PH20 SC or a placebo via subcutaneous injection using a prefilled syringe during the first two parts of the study, which include a double-blinded treatment period and a treatmentobservation period. In the third part, participants receive open-label efgartigimod treatment. The study spans between 63 and 135 weeks depending on each participants response to treatment. Throughout the study, participants undergo regular assessments of thyroid hormone levels, antibody levels, and immune response markers. Safety is monitored continuously, including tracking adverse events. The primary outcome measures focus on the percentage of participants achieving normal thyroid hormone levels without antithyroid drugs at 24 weeks. Additional evaluations include quality of life questionnaires and long-term monitoring of treatment effects and tolerability.

Researchers are evaluating treatments for people with advanced non-small cell lung cancer NSCLC that have specific genetic changes called HER2 mutations. This lung cancer has spread or cannot be controlled with current treatments. The study aims to compare how well a drug called sevabertinib works and how safe it is compared to standard treatment for this type of cancer. Participants will receive either sevabertinib tablets twice daily or standard treatment consisting of pembrolizumab combined with platinum-based chemotherapy given every 21 days by infusion. Those on standard treatment who experience disease progression may switch to sevabertinib. Treatment continues as long as participants benefit and do not have severe side effects or until they or their doctor decide to stop. During the study, participants will have imaging scans like CT, PET, MRI, and X-rays to monitor cancer spread. Doctors will also perform health checks including blood and urine tests, heart monitoring with ECG, pregnancy tests for women, and ask about symptoms or side effects. The main measure is progression-free survival assessed up to about 2 years, with additional outcomes related to survival, response rates, side effects, and quality of life followed up to 4 years.

This research aims to evaluate the long-term safety and tolerability of brivaracetam in children and adolescents with epilepsy, including those who participated in earlier studies or are newly enrolled in Japan with partial-onset seizures. The study also seeks to assess pharmacokinetic data in Japanese participants. The study is an open-label, single-arm, multicenter trial focusing on pediatric epilepsy treatment with brivaracetam. Participants will receive brivaracetam tablets or oral solution administered twice daily in two equal doses. Dosage varies based on weight up to 5 mgkgday for those weighing 11 to less than 20 kg, up to 4 mgkgday for those weighing 20 to less than 50 kg, and no more than 200 mgday. Directly enrolled participants in Japan will receive 1 to 4 mgkgday, not exceeding 200 mgday. The study includes long-term follow-up for up to 5 years. During the study, participants will be monitored for treatment-emergent adverse events, serious adverse events, and adverse events leading to discontinuation of the drug. Evaluations occur from Day 1 through safety visits over the 5-year period. Researchers will assess safety, tolerability, and pharmacokinetics. The total participation duration extends up to 5 years, with ongoing safety evaluations throughout this time.

Researchers are evaluating the use of ivonescimab combined with chemotherapy compared to pembrolizumab combined with chemotherapy as the first treatment for people with metastatic non-small cell lung cancer NSCLC. This Phase 3, randomized, double-blind, multiregional study involves around 1600 patients divided into two groups based on NSCLC histology squamous and non-squamous. The main goals are to assess overall survival and progression-free survival, with additional focus on treatment response and safety. Participants are randomly assigned to receive either ivonescimab or pembrolizumab along with platinum-doublet chemotherapy. Both treatments are given as intravenous injections. The two histology groups will be analyzed separately to understand how each treatment works within these subtypes of NSCLC. This design helps compare the effects of the two treatment combinations. During the study, participants will be monitored for survival and disease progression over several years. Safety assessments include tracking side effects from the start of treatment through 30 to 90 days after the last dose or start of other cancer therapies, with follow-up lasting up to two years. The study includes regular evaluations to measure tumor response and overall health, ensuring comprehensive monitoring throughout the participation period.

Researchers are conducting a combined Phase 2b and Phase 3 clinical trial to study CSL300 Clazakizumab in adults with end stage kidney disease ESKD who are undergoing maintenance dialysis. The study aims to find the right dose of CSL300 and then evaluate its effect on cardiovascular outcomes and safety in people with systemic inflammation and either atherosclerotic cardiovascular disease ASCVD or diabetes. This is a randomized, double-blind, placebo-controlled study involving multiple centers. Participants will receive intravenous IV administration of either CSL300 or a placebo. The Phase 2b part focuses on determining the appropriate dose of CSL300 compared to placebo over about 12 weeks, while the Phase 3 part examines CSL300s effect on cardiovascular events over approximately five years. The study includes different dosing groups in Phase 2b and a larger comparison of CSL300 versus placebo in Phase 3. During the study, participants will be monitored regularly with blood tests that measure inflammation markers such as high-sensitivity C-reactive protein hs-CRP, cardiovascular events, and safety outcomes. Researchers will track changes in various blood components and adverse events up to 32 weeks in Phase 2b and follow cardiovascular outcomes for up to five years in Phase 3. The total participation lasts through these periods with scheduled assessments to evaluate treatment effects and safety.