The purpose of the study is to see how well Concizumab works in preventing bleeding episodes compared to the previous treatment the children were receiving. The study will involve children who have not used Concizumab before. Participants will receive regular injections of Concizumab over a period of time, and the number of bleeding episodes they experience will be monitored and compared to their previous treatment.

Throughout the study, researchers will also keep track of any side effects or reactions to the medication, such as injection site reactions or the development of antibodies against Concizumab. The study aims to provide valuable information on the safety and effectiveness of Concizumab in managing bleeding episodes in young children with hemophilia.

The purpose of the study is to assess how safe the medicine is and whether it shows early signs that it can shrink tumors when used alone or with the hormone blocker. Participants start with low doses that are gradually increased while doctors watch for side effects, known as dose‑limiting toxicities, and determine the highest dose that can be given safely, called the maximum tolerated dose. After the safe dose is found, more patients receive that dose for several treatment cycles, with regular clinic visits, blood tests, and imaging scans to see if the tumors are responding. The study follows a phase 1/2 trial design, beginning with a safety‑focused part and then moving to a larger group to look at tumor response and how long the benefit lasts.

The purpose of the study is to see whether a higher radiation dose improves overall survival. Participants will be randomly assigned to receive either the standard radiation dose or a higher dose, then all will continue with the same durvalumab treatment. The study follows patients for several months, checking how long they live, how the cancer responds, any side effects, and how they feel in daily life.

The purpose of the study is to determine whether the new medicine works as well as the existing one. Participants will receive a series of these eye injections over several months and will have regular eye examinations. Vision will be checked using a test called BCVA, measured with an ETDRS chart, which is a standardized eye chart used to assess how well a person can read letters. Doctors will also look for signs of fluid inside the retina, called IRF and SRF, and will measure the size of the abnormal blood‑vessel area, known as CNV, using imaging methods such as FA and SD-OCT. Safety will be monitored throughout the study, including checking for any immune reactions called ADAs.

The purpose of the study is to evaluate how effective imlunestrant is compared to these standard treatments in preventing the return of breast cancer in patients who have already received two to five years of standard endocrine therapy. Endocrine therapy is a treatment that helps to stop or slow the growth of cancer by blocking the body’s natural hormones. Participants in the study will be randomly assigned to receive either imlunestrant or one of the standard treatments. The study will monitor participants over a period of time to see how well the treatments work in preventing cancer from coming back.

Throughout the study, participants will receive regular check-ups and assessments to monitor their health and the effectiveness of the treatment. The study aims to provide valuable information on whether imlunestrant can offer a better option for patients with this type of breast cancer, potentially improving outcomes and reducing the risk of cancer recurrence. The study will also look at the overall health and quality of life of participants while they are receiving treatment.

During the 24‑week period, participants are randomly assigned to one of the two groups and neither the participants nor the study staff know which treatment is given (double‑blind). Regular visits include blood tests that measure NT-proBNP, a protein that rises when the heart is under stress, and imaging tests such as an ultrasound of the heart (echocardiogram) that assess measurements like left ventricular end-diastolic volume index, left ventricular end-systolic volume index, E/e′ ratio, global longitudinal strain and left atrial volume index. Participants also complete the Kansas City cardiomyopathy questionnaire to report symptoms and quality of life, and a routine 12‑lead Electrocardiogram is performed to check the heart’s electrical activity. Throughout the study, safety is monitored by recording any side effects and checking routine laboratory values.

The purpose of the study is to determine whether nerandomilast can more effectively delay death or disease worsening compared with the placebo.

Participants will be randomly assigned to receive either the active medication or the placebo for about one year. During this time, they will attend regular clinic visits where doctors will check skin thickness, lung function (including a test called FVC that measures how much air can be forced out of the lungs), physical ability, and overall health. The study will track any serious health events, changes in symptoms, and overall survival throughout the treatment period.

The purpose of the study is to find out whether daily treatment with Elamipretide can improve heart and muscle function and be safe to use. Participants will be asked to receive the assigned injection each day for a period of about one and a half years, with regular clinic visits to monitor health, record any side effects, and perform simple walking and strength tests.

During the visits, researchers will measure how far a person can walk in six minutes, how quickly they can stand up and sit down several times, and the strength of certain leg muscles using a handheld device. These tests help show any changes in the ability to move around and perform daily activities, as well as overall disease severity as judged by both the participant and the doctor.

The purpose of the study is to determine whether the drug can reduce brain amyloid, a protein that accumulates in Alzheimer’s, as measured by a brain scan called PET. Participants will receive a series of injections over about two years and will attend regular visits that include simple blood draws, a spinal fluid sample (CSF), and brief cognitive tests to monitor safety and any changes.

The purpose of the trial is to find out whether early use of depemokimab can lower the number of worsening episodes and keep asthma under better control. Participants will be randomly assigned to receive either the study drug or the placebo, with injections administered in a clinic setting every six months. Over a period that may extend up to three years, they will attend regular visits where doctors will check symptoms, record any serious attacks, and perform simple breathing tests.

During the study, several easy‑to‑understand tools are used: the ACT questionnaire asks about daily symptoms, the AQLQ asks how asthma affects quality of life, and the ACQ-5 measures recent symptom severity. Lung capacity is measured with a test called FEV1, which shows how well the lungs move air in and out, providing an overview of overall lung function. Safety checks are also performed at each visit to monitor how participants tolerate the treatment.

The main aim of the study is to evaluate how often the tumors shrink, known as an objective response. Participants receive the study drug in treatment cycles that repeat every few weeks, with regular visits for physical exams, blood tests, and imaging scans to check the size of the tumor and overall health.

Tumor shrinkage is classified as a complete response when the cancer disappears, a partial response when the tumor size decreases by a significant amount, or stable disease when the tumor does not grow or shrink much. Throughout the trial, doctors monitor for adverse events, which are side effects or any unwanted medical problems, and record any that are serious or lead to stopping the treatment.

Participants will receive a series of injections of either the study drug or the placebo over several months, with regular clinic visits for safety checks and simple breathing tests. One key breathing test measures the amount of air expelled in the first second of a forced breath, known as Forced Expiratory Volume in 1 second, to see how lung function changes. Throughout the study, people will complete questionnaires that assess health status and symptoms, such as the SGRQ-C (a quality‑of‑life survey), the CAAT (a symptom score), and the E-RS:COPD diary (records daily breathing problems). Researchers will also monitor for any side effects, using terms like TEAEs (any undesirable effects), AESIs (specific safety concerns), and SAEs (serious problems). Blood samples will be taken to check the drug level and to look for antidrug antibodies, which are the body’s immune response to the medication.

The purpose of the study is to evaluate whether the combination of the new protein and the chemotherapy works well and is safe for these patients. Participants receive the study drug by infusion (a slow drip into a vein) together with chemotherapy cycles given every few weeks. The treatment period lasts several months, with regular visits to check for side effects and to see how the tumor responds.

After the treatment phase, children are followed for a period of time to monitor disease behavior and record any long‑term effects. Blood tests, imaging scans, and physical examinations are used to assess progress, and any problems are managed by the medical team. The information gathered will help determine if this approach could become a new standard option for children with this serious condition.

The purpose of the study is to evaluate safety and find the best dose of the new medicines when used alone or in combination. Participants receive an IV infusion, which means the medicine is delivered directly into a vein, and they swallow tablets on a regular schedule. Treatment cycles are repeated every few weeks, and patients return to the clinic for regular check‑ups, blood tests, and simple examinations to watch for any side effects.

The main goal of the trial is to find out whether LY4268989 can bring more participants into clinical remission—periods when symptoms are absent or very mild—than the placebo during the first 10 weeks and to see if the benefit continues through a longer, 52‑week maintenance phase for those who respond early. Participants will start by taking the study tablets daily for about ten weeks (the induction phase). If they show improvement, they may continue the same treatment for up to a year (the maintenance phase), with regular visits to check how they are doing.

During the study, doctors will use a scoring system called the Modified Mayo Score to decide whether a person has reached remission; this score looks at stool frequency, bleeding, endoscopic findings, and overall health. “Induction” refers to the initial treatment period aimed at quickly reducing inflammation, while “maintenance” means the ongoing treatment intended to keep the disease under control. Participants will have routine check‑ups, blood tests, and questionnaires to monitor safety and how well the medication works.

The purpose of the study is to evaluate whether Orelabrutinib can delay the worsening of disability compared with placebo. Participants will receive the assigned tablet each day for several years and will attend regular clinic visits where their ability to perform everyday tasks is checked and brain scans using MRI are performed to look for new lesions. The study will track how long it takes before a confirmed increase in disability occurs and will record any safety concerns throughout the trial.

The purpose of the study is to determine whether Admilparant can be used safely over an extended period without causing serious side effects. Participants will take the medication each day and will attend regular visits where healthcare staff will check vital signs such as blood pressure and heart rate, review simple blood tests, and perform a basic heart test known as an ECG, which records the heart’s electrical activity. Any side effects that arise will be recorded and evaluated.

The study will continue for many months to gather enough information about safety and tolerability. Throughout the study, participants will be asked to report any new symptoms or problems, and they will receive routine medical check‑ups to monitor their overall health while using the medication.

The purpose of the study is to assess the safety and tolerability of these treatment regimens. Participants will receive a single injection at the start and then continue receiving additional injections according to a fixed schedule over many months. Regular visits will be scheduled to check health status, and an examination of the intestine using a camera (often called an endoscopy) will be performed around week 48 to see how the lining has responded.

Throughout the trial, researchers will closely watch for any side effects that arise after treatment and will record any new health problems. The endoscopic examination will help determine whether the inner surface of the colon shows signs of healing, providing an indication of how well the medication may be working while ensuring participants remain safe.

The goal of the trial is to find out whether adding Venetoclax to the standard three‑drug mix can improve overall survival, which means living longer after the treatment starts.

In this phase 3 study, children are placed by chance into one of two groups: one group receives the three standard drugs, and the other group receives the same three drugs plus the oral Venetoclax. Treatment is given in cycles over several weeks, and the children are checked regularly to see how they are doing and to record any side effects. The study follows each participant for a period of time to see how long they live and how they feel.

The main goal of the study is to see whether a single dose of the medicine is safe and whether it can raise protein levels to a normal range, with some participants receiving a second dose later to assess longer‑term effects. Participants will receive the infusion, then attend regular visits over several months during which blood samples and simple health checks are performed.

Researchers will watch for any side effects, measure the amount of the protective protein in the blood, and test how well it works using a laboratory test called a elastase inhibition assay. They will also check lung function and liver health through routine exams and questionnaires to understand how the treatment impacts overall well‑being.

The main goal of the trial is to learn how safe the medicine is and how well patients can tolerate increasing doses. Participants will receive the drug in a series of treatment cycles that last about four weeks each, with the amount of medicine gradually increased in early groups to find the highest dose that can be given without unacceptable side effects.

During the study, patients will have regular check‑ups, blood tests, and scans to watch for any side effects and to see how the cancer responds. Any problems that arise will be recorded and managed by the medical team, and the study will continue until the predetermined number of treatment cycles is completed or if a participant chooses to stop early.

The purpose of the trial is to determine whether NXT007 works at least as well as Emicizumab in preventing bleeds. Participants receive regular injections for several months and attend scheduled visits where doctors check their health, collect blood samples, and ask about daily activities and quality of life. The main way the study measures success is by counting the average number of bleeding episodes that need treatment each year, called the annualized number of treated bleeds.

Throughout the study, safety is closely watched. Researchers look for any side effects such as reactions at the injection site, allergic responses, or signs of clotting problems. Participants also complete simple questionnaires about how their condition affects everyday life, helping to assess both the medical and personal impact of the treatments.

The purpose of the trial is to evaluate the safety and efficacy of the new combination therapy. Over a period of about four weeks, subjects will take the study medication once daily, while other participants receive a matching placebo. Safety will be monitored through reports of any side effects, regular blood tests, standard 12‑lead ECG recordings to check heart rhythm, and measurements of Sweat chloride levels, which reflect how the disease is affecting the body.

Effectiveness will be assessed by measuring lung function using the percent predicted forced expiratory volume in one second (ppFEV1) and by having participants complete a disease‑specific questionnaire that evaluates breathing symptoms (CFQ R). The study involves several clinic visits for these tests and for overall health checks.

The purpose of the study is to see whether adding GB-0895 can lower the number of serious asthma attacks over a one‑year period. Volunteers will receive the assigned injection at regular intervals for about 52 weeks and will attend routine visits where doctors will check lung function using simple breathing tests, ask about daily symptoms, and have participants fill out questionnaires that measure how asthma affects daily life and overall well‑being. Terms such as “subcutaneous” mean the medication is delivered under the skin, and “systemic corticosteroids” refer to steroid medicines taken by mouth or injection that help reduce inflammation during severe attacks.

After the earlier treatment has finished, participants are assigned to receive either pumitamig or durvalumab. The study is randomized, which means the treatment is chosen by chance, and open-label, which means the treatment is known. The study team then follows the cancer over time and watches for changes, such as whether it stays stable, shrinks, or grows, and also checks how safe each medicine is and how well it is tolerated.

The purpose of the study is to see whether oral semaglutide can help slow worsening of memory, thinking, and daily function in people with early Alzheimer’s disease, and to check its safety. The study is randomised, which means the treatment is assigned by chance, and double-blind, which means neither the participants nor the study team knows who receives semaglutide or placebo during the study. Treatment is taken by mouth over a long period, and the study follows changes over time.

Participants take the study tablets regularly and are seen at planned visits during the trial. These visits are used to monitor health, review how the person is doing, and record any changes in memory, daily activities, or side effects. The study compares how people do over time in the semaglutide and placebo groups.

Children in the study receive eye drops in a single-dose container and are followed over several months. During the study, eye health, vision, eye comfort, and possible side effects are checked at different visits and by phone calls. The study also looks at changes in the length of the eye, which can increase as myopia gets worse, as well as other eye findings such as redness, eye surface irritation, eye pressure, and the condition of the cornea, which is the clear front part of the eye.

The trial compares T10430 with placebo to learn more about safety and possible benefit. The treatment is given to both eyes, and the study follows participants for about one year.

The aim is to see if adding Panzyga can lower the chance of a major infection or death in this vulnerable group. Participants will receive the assigned infusion once every few weeks for several months, and they will be checked regularly by doctors for any signs of infection, side effects, or changes in health. The study is set up so that neither the participants nor the doctors know which infusion is being given, to keep the comparison fair.

Throughout the trial, any infection that occurs will be recorded, and an independent group of experts will decide whether it meets the definition of a major infection. Safety will also be monitored by looking at reported side effects and routine lab tests. The information gathered will help determine whether the treatment is safe and effective for preventing serious infections in people with weak immune systems due to their underlying disease and therapy.

The purpose of the study is to compare the effectiveness of a new medicine with that of standard chemotherapy. The investigational drug being tested is Sofetabart Mipitecan (LY4170156). For participants with platinum-resistant disease, the new drug may be given alone and compared with the doctor’s choice of chemotherapy drugs such as carboplatin, paclitaxel, gemcitabine, topotecan or doxorubicin, or with Mirvetuximab Soravtansine. For those with platinum-sensitive disease, the new drug is combined with bevacizumab and compared with a standard platinum‑based two‑drug chemotherapy regimen plus bevacizumab.

Participants receive the study medicines through an IV infusion every few weeks and attend regular clinic visits where doctors perform physical examinations and imaging scans to check how the cancer is responding. Treatment continues until the cancer grows, side effects become unacceptable, or the study period ends, which may be several months for each participant.

In the study, treatment is given over a long period of time, and people are followed while they receive one of the study medicines. The study looks at how often a blood clot in a vein happens after treatment starts. The medicines being studied are baricitinib, adalimumab, and etanercept.

Participants in the study will receive either the IKT-001 film-coated tablet or a placebo. The study is designed in two parts to observe how the medication affects different aspects of the condition over a period of time. During the study, changes in pulmonary vascular resistance, which is the resistance to blood flow through the lung vessels, and the six-minute walk distance, which is a test measuring how far a person can walk in six minutes to assess physical capacity, will be monitored.

Participants in the study will receive medications through an intravenous infusion, which is a method of delivering medicine directly into a vein. The study involves comparing different combinations of drugs to see how they affect progression-free survival, a term used to describe the length of time during and after treatment that a person lives with the disease without it getting worse. Other factors being observed include overall survival and the quality of life, which tracks how well a person can perform daily activities and their general well-being during the study.

The goal of the study is to see how pegtibatinase affects levels of total homocysteine (tHcy) and methionine (Met) in the blood. High levels of these substances are common in people with this disease. During the study, blood samples will be taken to monitor these levels and to ensure that the treatment is being tolerated well by the body.

During the study, participants will take these medications as part of a treatment plan. The process involves monitoring the disease to see how the body responds to the combined medicines. The study will follow the participants over time to see how long the treatment keeps the cancer under control and to ensure the medications are being used safely.

Participants in this study will be randomly assigned to receive either the experimental drug CSL624, which contains vamifeport trihydrochloride in a hard capsule form, or a placebo. The study is double-blind, meaning neither the participants nor the researchers know which treatment is being administered. During the study, the amount of iron in the liver will be monitored using MRI, a type of medical imaging that uses magnets to create detailed pictures of the inside of the body, to measure the liver iron concentration.

The purpose of the study is to compare the effectiveness and safety of the two treatments in reducing the need for red blood cell transfusions. Participants are randomly assigned to receive either elritercept or epoetin alfa and will be treated for up to 24 weeks. During this time, they will have regular clinic visits where blood samples are taken to check hemoglobin levels, which indicate how well the blood is carrying oxygen, and doctors will record whether transfusions are still needed.

The study involves a series of visits scheduled roughly every few weeks. At each visit, a healthcare professional will administer the injection, perform a quick blood test, and ask about any side effects. The overall goal is to see if participants can stay without transfusions for a sustained period while their hemoglobin improves.

The new medication, identified by the code name INCB123667, is given as a tablet taken by mouth each day. Participants may receive one of several standard chemotherapy options chosen by their doctor, including paclitaxel, topotecan, gemcitabine, or doxorubicin, all of which are administered through an intravenous (IV) infusion, meaning the drug is delivered directly into a vein.

After enrollment, participants are assigned to receive either the study pill or one of the IV chemotherapy drugs and are followed with regular clinic visits, imaging scans to check tumor size, blood tests, and quality‑of‑life questionnaires for several months. The trial will record how long patients live without the disease getting worse, known as progression‑free survival, and how long they live overall, referred to as overall survival, to evaluate the relative benefit of the treatments.

In the first part of the trial, small groups of participants will receive increasing amounts of the drug given by an intravenous infusion (a medicine delivered directly into a vein) to determine how the body tolerates it and to find the dose that seems most promising. Once a recommended dose is set, the second part continues with more participants receiving that dose, while doctors watch for side effects and check how the tumors respond. Tumor changes are measured using a standard method called RECIST v1.1, which compares scans taken before and after treatment to see if the cancer gets smaller, stays the same, or grows.

Throughout the study, participants will have regular medical visits, blood tests, and imaging scans to monitor safety and any signs that the tumor is responding. Researchers will record any adverse events, track how long any tumor shrinkage lasts, and note how long participants remain free from disease progression. The overall goal is to gather information that could lead to larger studies and eventually new treatment options for people with advanced solid tumors.

The purpose of the study is to determine how well patients respond to these treatments over a specific period. Participants may receive different doses of Mounjaro administered via subcutaneous injection, which is a method of delivering medication just under the skin. The study will monitor several factors, including the pathologic complete response, which refers to the total disappearance of cancer cells in a tissue sample, and changes in weight. The course of the study involves regular follow-up assessments to track the progress of the disease and the safety of the medications used.

For those with a high amount of the protein, AZD5335 will be compared to mirvetuximab soravtansine. For those with a low amount of the protein, AZD5335 will be compared to a treatment chosen by the doctor, which may include doxorubicin hydrochloride, liposomal, topotecan, or paclitaxel. These treatments are delivered through an intravenous infusion, which means the medicine is passed directly into a vein.

During the study, participants will receive their assigned treatment and will be monitored to see how long the cancer remains stable without growing. This period is referred to as progression-free survival. The study also looks at overall survival, which is the total length of time from the start of the study until death from any cause.