Participants are randomly assigned to receive either the combination therapy or a single oral drug, and treatment continues until disease progression or unacceptable side effects occur. Visits include regular health checks, blood tests, and imaging scans to monitor the tumor and overall health. The study follows patients for several months to record how long they live, how long the disease stays stable, and any side effects that arise.

After a qualifying attack, participants are randomly assigned to receive either the experimental infusion or the placebo, both given through a vein. They continue the usual steroid treatment and are followed for about three months, with visits to check vision, strength, walking speed, hand dexterity, and overall disability using simple tests and questionnaires. The study records whether additional rescue treatments are needed, how quickly symptoms improve, and any side effects, allowing researchers to see if the added drug provides a meaningful benefit.

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 aim of the trial is to determine whether the combination of sarilumab and a 52‑week prednisone taper works better than prednisone taper alone in achieving lasting symptom control. Participants are randomly assigned to receive one of two dose levels of the study drug (150 mg or 200 mg) every two weeks, while all continue the steroid taper for one year, and they are followed for the same period to see how they respond.

Throughout the year, researchers check whether participants reach “remission,” meaning they have no significant pain or stiffness, and they record any side effects or laboratory changes. They also track how long remission lasts, whether symptoms return (a “flare”), and how the condition affects physical and mental well‑being using standard questionnaires. Safety is monitored continuously, and any serious problems are reported promptly.

Participants will be randomly assigned to receive either the active medication or the placebo for about one year, with regular clinic visits for injections and health checks. Blood samples will be taken to monitor liver enzymes (ALT and AST), a blood test called the ELF score that estimates liver scarring, and other measures such as cholesterol, blood sugar control (HbA1c), and body weight. Imaging tests, including a special ultrasound that measures liver stiffness (VCTE‑LSM), an MRI‑based scan (MRE), and an MRI that quantifies liver fat (MRI‑PDFF), will be performed at the start and at the end of the study to see how the liver is responding. Any side effects or abnormal lab results will be recorded throughout the study period.

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 purpose of the study is to evaluate the safety of the recommended dose of this medication in the pediatric population. Children who need diagnostic or therapeutic percutaneous vascular procedures will be given the drug during the procedure and then observed for several days. Researchers will watch for any bleeding, breathing problems such as wheezing or shortness of breath, changes in kidney function, vital signs like heart rate and blood pressure, and any other side effects that may occur.

After the infusion, participants remain in the hospital for monitoring, and follow‑up checks may be scheduled to ensure recovery and to record any adverse events. The study collects information on how well the medication is tolerated, without using any comparison or placebo, and aims to provide data that could help improve care for children with this heart condition.

The investigational treatment is an injectable form of efimosfermin alfa, given under the skin (subcutaneous injection). Participants are randomly assigned to receive either the study drug or a placebo, and neither the participants nor the study staff know which one is given (double‑blind). The study lasts about one year, with regular clinic visits for injections and safety checks.

The main goal is to see whether the drug can improve liver health by reducing inflammation and decreasing scarring (fibrosis) compared with the placebo. Researchers will look at changes in the liver tissue, blood tests, and overall health over the treatment period.

The purpose of the study is to see whether giving a higher amount of this replacement therapy can reduce or eliminate the signs of synovitis over a year. Participants will receive regular infusions of the medicine, will have periodic checks of joint health using ultrasound imaging, and will be followed for about 12 months to record any bleeding events and any side effects.

Throughout the trial, safety will be monitored by recording any adverse events, and the amount of clotting factor in the blood will be measured to understand how long therapeutic levels are maintained. The study will collect information on how often bleeding occurs in joints and the overall number of bleeds, as well as whether the joint inflammation improves.

The purpose of the trial is to compare how well the new medicine works against the standard treatment in shrinking tumors and helping patients live longer. Participants will be randomly assigned to one of the two treatment arms and will receive the assigned therapy in repeated cycles every few weeks. Throughout the study, doctors will perform regular safety checks, blood tests, and imaging scans to see how the disease responds, and they will record any side effects that occur.

The purpose of the study is to find out whether the medication can slow the narrowing of the valve and improve the ability to exercise. Participants are randomly assigned to receive either the study drug or the placebo for about a year, with regular clinic visits. During the study, doctors will use an echocardiogram (an ultrasound picture of the heart) to see how big the valve opening is, and a peak VO2 test, measured during a CPET (a breathing and heart test done while exercising), to check how well the body uses oxygen. A CT scan (a detailed X‑ray picture) will also be performed to look at calcium buildup in the valve. If the valve becomes too narrow, doctors may discuss procedures such as TAVR (a catheter‑based valve replacement) or SAVR (surgical valve replacement).

Throughout the trial, participants will be monitored for any side effects, and safety checks will be done at each visit. The study team will collect information about health changes and any additional treatments needed, while keeping participants’ personal information confidential.

The purpose of the study is to evaluate whether the new medicine can lower the number of treated bleeds compared with standard therapy. Participants will be assigned to receive either the new medicine or the standard factor VIII for about six months, attending regular visits where any bleeding events are recorded, quality‑of‑life questionnaires are completed, and safety checks such as blood tests for antibodies or reactions at the injection site are performed. The trial will monitor how often injections are needed, the amount of medicine used, and any side effects that arise during the treatment period.

Participants in the study will take the medication in the form of a capsule, which is taken by mouth once a day. The study will monitor the response to the treatment over a period of time to see how well it works in managing the conditions. Some participants may receive a placebo, which is a substance with no active medication, to compare the effects of the treatment. The study aims to gather information on how the medication affects the body and its potential side effects.

By participating in this study, researchers hope to better understand the effectiveness of gandotinib in treating myeloproliferative neoplasms and to explore its potential as a treatment option for patients with these conditions. The study will help determine if this medication can improve the quality of life for those affected by these blood cancers.

The purpose of the trial is to determine whether MK-8748 is at least as effective as aflibercept in preserving or improving vision. Participants will receive a series of injections into the eye over the course of about one year, with regular visits for eye checks, vision testing, and imaging that may involve the fluorescein dye.

Vision is measured using best-corrected visual acuity, which is the sharpest vision possible with the best glasses or contacts, and the results are recorded as letters on an ETDRS chart, a standard eye‑test chart. Throughout the study, doctors will monitor participants for any side effects or safety concerns.

Participants in the study will be randomly assigned to receive either cabozantinib plus supportive care or just supportive care. The study will monitor the participants over a period to see how the treatment affects the progression of the disease. The trial will also assess the safety of cabozantinib and how the body processes the drug, which is known as pharmacokinetics. The study will involve regular check-ups and assessments to track the health and progress of the participants.

The trial aims to provide valuable information on whether cabozantinib can help improve the outcomes for young people with osteosarcoma. The study will last for several months, and participants will be closely monitored throughout the process to ensure their safety and to gather data on the effectiveness of the treatment. The results of this study could potentially lead to better treatment options for those affected by this challenging condition.

The purpose of the study is to see if the new combination can keep the cancer from getting worse for a longer time than the standard treatments. Participants are randomly assigned to receive either the new three‑drug regimen or the standard therapy, and they take the medicines in repeated cycles while visiting the clinic for regular check‑ups and imaging scans. The main result being measured is progression‑free survival (PFS), which means the time until the cancer grows or the patient dies, and it is evaluated using standard imaging rules called RECIST and analyzed with the Kaplan‑Meier statistical method. Safety and side‑effects are recorded and graded according to the CTCAE system.

The purpose of the study is to assess how effective and safe sonelokimab is for adults with moderate to severe hidradenitis suppurativa. Participants in the study will receive either sonelokimab or a placebo over a period of 16 weeks. During this time, researchers will monitor the participants to see if there is a 75% improvement in their condition, which is measured by a specific score called the Hidradenitis Suppurativa Clinical Response (HiSCR) score. The study will also track any side effects or adverse events that occur during the treatment period.

Throughout the study, participants will undergo regular check-ups, including physical examinations and laboratory tests, to ensure their safety and to gather data on the treatment’s effects. The trial aims to provide valuable information on whether sonelokimab can be a beneficial treatment option for those suffering from hidradenitis suppurativa, potentially improving their quality of life by reducing the severity of their symptoms.

Participants in the study will be randomly assigned to receive either Satralizumab or a placebo, which is a substance with no active medication. The study will last for a period of 48 weeks, during which participants will receive regular injections and attend scheduled visits to monitor their progress. The main goal is to see if there is a reduction in the bulging of the eyes, known as proptosis, after 24 weeks of treatment. The study will also look at other factors, such as changes in eye appearance and quality of life, as well as any side effects that may occur.

Throughout the study, participants will be closely monitored by healthcare professionals to ensure their safety and to gather information on how Satralizumab affects their condition. This research aims to provide valuable insights into the treatment of Thyroid Eye Disease and potentially improve the quality of life for those affected by this condition.

Participants in the study will be randomly assigned to receive either Upadacitinib or a placebo, which is a substance with no active medication. The study is designed to be double-blind, meaning neither the participants nor the researchers will know who is receiving the actual medication or the placebo. This helps ensure that the results are not influenced by expectations. The study will last for a period of time, during which participants will take the medication orally in the form of a modified-release tablet. The goal is to observe any changes in the severity of eczema symptoms and overall skin condition over the course of the study.

Throughout the study, participants will be monitored for any improvements in their eczema symptoms, such as reduced itching and inflammation, as well as any potential side effects. The study aims to provide valuable information on whether Upadacitinib can be an effective treatment option for those suffering from moderate to severe eczema. By participating in this study, researchers hope to gain insights that could lead to better management and treatment options for individuals with this challenging skin condition.

The purpose of the study is to evaluate how effective and safe this treatment is for patients with Uveitic Macular Edema. Participants in the study will receive either the actual medication or a placebo. The study will monitor changes in vision and eye health over time, with particular attention to improvements in visual clarity and reductions in retinal swelling. The study will also assess the safety of the treatment by tracking any side effects that may occur.

Throughout the study, participants will have regular check-ups to measure their vision and eye condition. The study will last for a period of up to 52 weeks, during which the effects of the treatment will be closely observed. The goal is to determine if the treatment can significantly improve vision and reduce swelling in the retina for those affected by this condition.

The purpose of the study is to see how well the combination treatment works compared to the single medication in preventing the return of melanoma after it has been surgically removed. Participants in the study will be randomly assigned to receive either the combination treatment or the single medication. Some participants may receive a placebo, which is a substance with no active medication. The study will last for up to 12 months, during which time participants will receive regular infusions and be monitored for any changes in their condition.

Throughout the study, researchers will keep track of how long participants remain free from melanoma returning, as well as other important health outcomes. These include how long participants live without the cancer spreading to other parts of the body, overall survival rates, and any side effects experienced. The study will also assess changes in participants’ quality of life and physical functioning. This information will help determine the effectiveness and safety of the combination treatment compared to the single medication.