The main purpose of the trial is to determine whether the experimental cell therapy can improve lung function compared with standard care. Participants are randomly assigned to receive either the new cell therapy or the usual medicines, and the study is open‑label, meaning both doctors and participants know which treatment is being given. After the initial treatment, participants attend regular clinic visits for several months during which safety checks and health assessments are performed.

Effectiveness is primarily measured by changes in Forced Vital Capacity, which is the amount of air a person can forcefully exhale after a deep breath. Secondary evaluations include the Modified Rodnan Skin Score, a simple scale that rates skin thickness, and the DLCO, a test that shows how well the lungs transfer oxygen into the blood. Additional observations track how lung function and skin thickness change over time and how long it takes for the disease to worsen.

The study is testing whether an experimental medicine named prasinezumab can help protect thinking skills in these individuals. The drug is given through an IV infusion, which means it is delivered directly into the bloodstream using a small tube inserted into a vein. Participants will receive either the medicine or a placebo, a simple salt solution that looks the same as the active treatment, so that neither the participants nor the doctors know which one is given. The main goal is to see if the medicine can slow or prevent loss of mental function compared with the placebo.

People who join the trial will come to the clinic for regular visits over about two years. At each visit they will have brief safety checks such as blood tests and heart monitoring, and they will complete simple questionnaires and short memory and thinking tests that are easy to understand. The schedule includes several infusion sessions spaced out over the study period, allowing researchers to track any changes in thinking abilities and overall health throughout the trial.

The purpose is to determine whether long‑term use of Admilparant is safe and does not lead to serious side effects.

Participants will take one tablet each day for several years while doctors perform regular check‑ups. At each visit routine blood work, a quick heart test called an ECG that records the heart’s electrical activity, and measurements of blood pressure and heart rate are done. The study records any side effects, changes in test results, or reasons to stop the medication, and follows each person from the start until the study ends.

The purpose of the study is to determine whether nemolizumab can safely reduce skin thickness and improve overall health in adults with Systemic sclerosis while identifying the most effective dose.

Adults who join the trial will receive a series of subcutaneous injections over a 52‑week main treatment period, after which they may continue receiving the medication for an additional 156 weeks if they choose to stay in the study. Throughout the study, participants will have regular clinic visits where doctors will assess skin thickness using a scoring system called the modified Rodnan Skin Score, measure lung capacity with a test known as forced vital capacity, and evaluate overall response using the Composite Response Index in Systemic Sclerosis. Safety will be monitored by checking vital signs, laboratory tests, and any side effects that arise.

The purpose of the trial is to evaluate the long‑term safety and tolerability of the study drug in this patient population. After an initial screening, participants will receive the assigned medication at regular intervals over an extended period while undergoing routine health checks, including measurements of vital signs, blood laboratory tests, and a heart test known as an electrocardiogram. Physical examinations will assess walking ability, standing up from the floor, climbing stairs, and upper‑limb function, and blood samples will be taken to monitor drug levels and any immune response. The study is open‑label, so all participants know they are receiving the investigational therapy, and it continues beyond the earlier phase of the research.

The purpose of the study is to assess the safety and tolerability of the different treatment regimens. After an initial screening visit, participants will begin a series of injection visits that continue for several months, with regular clinic appointments to check health status, record any side effects, and perform routine laboratory tests. The study follows participants for up to about four years to observe long‑term outcomes.

Safety monitoring includes tracking any new medical problems that arise during the trial. In addition, doctors will use a camera procedure called endoscopic examination to look inside the colon and evaluate improvement of the disease after about one year. This information, together with the safety data, helps determine whether the antibodies are well tolerated and potentially beneficial for people with ulcerative colitis.

The purpose of the trial is to determine whether the combination of these therapies can keep the cancer from returning for a longer time compared with placebo.

After surgery, participants are randomly assigned to receive either the active combination of medicines or the placebo, with injections given at regular intervals over several months. Throughout the study, participants attend follow‑up visits where doctors check for any side effects, monitor overall health, and perform routine scans or tests to see if the cancer comes back.

Participants receive the medication by an intravenous infusion, meaning it is given through a vein, with the amount of drug increased gradually in the early part of the study to find a safe level, followed by a larger group receiving that level to see early signs of effectiveness. Throughout the study, regular blood tests are done to measure PSA (a protein that can indicate prostate cancer activity), check organ function, and look for any side effects. Imaging scans are also performed at set intervals to see if tumors shrink or stop growing. The study follows each participant for several months, recording any adverse events, changes in laboratory results, and overall health status.

The primary aim of the trial is to determine whether the new combination improves the time patients live without their disease getting worse and, for a portion of the study, also extends overall survival compared with standard care.

Participants are randomly assigned to receive either the experimental medicines or the usual therapy, with treatment given in repeated cycles of intravenous infusions and oral tablets over several months. Throughout the study, doctors monitor tumor size using imaging scans evaluated by the criteria called RECIST 1.1, record how long the disease stays stable (progression free survival), note the length of life from start of treatment (overall survival), and assess how many patients experience tumor shrinkage (objective response rate). Safety is evaluated by tracking side effects, vital signs, laboratory tests and heart rhythm recordings.

This clinical trial is focused on studying Type 1 Diabetes in adults who are also overweight or have obesity. The treatment being tested is called tirzepatide (also known by its code name LY3298176), which is given as a solution for injection using a pre-filled pen. The purpose of the study is to see if tirzepatide can help control blood sugar levels better than a placebo.

Participants in the study will receive either tirzepatide or a placebo once a week. The study will last for about 40 weeks. During this time, the effects of tirzepatide on blood sugar levels will be closely monitored. The study aims to determine if tirzepatide is more effective than a placebo in managing blood sugar levels in people with Type 1 Diabetes.

This study is important for understanding how tirzepatide can help people with Type 1 Diabetes who are also dealing with being overweight or obese. The results could provide valuable insights into new treatment options for better managing blood sugar levels in these individuals.

Participants in the study will take lasmiditan as needed for their migraine attacks over a 12-month period. The study will monitor how well the medication is tolerated by the participants and will track any side effects that may occur. The study will also look at how many participants stop using the medication due to any adverse effects.

Throughout the study, participants will be closely observed to ensure their safety and to gather information on how lasmiditan works in treating migraines in children. This research aims to provide valuable insights into the long-term use of lasmiditan for managing migraines in pediatric patients.

Participants in the study will receive Nemtabrutinib in the form of a tablet taken by mouth. The study is divided into two parts. In the first part, the focus is on understanding the safety and how well the body can tolerate Nemtabrutinib. In the second part, the study will look at how well the medication works in treating the different types of blood cancers mentioned. Some participants may receive a placebo, which is a substance with no active medication, to compare the effects of Nemtabrutinib.

The study will take place over a period of time, with participants being monitored for any side effects and the effectiveness of the treatment. The goal is to gather information that could help improve treatment options for people with these types of blood cancers. Participants will be closely observed by medical professionals throughout the study to ensure their safety and to collect valuable data on the medication’s impact.

The main goal is to see whether long‑term use of the medication is safe and whether it continues to provide clinical benefit for people with this condition. Participants will receive the study drug every day for several years and will attend regular clinic visits where they will be asked about any side effects, have simple physical tests such as walking speed measured with the 10MWT, and undergo breathing assessments using the FVC test, which checks how well the lungs can move air in and out while sitting. Blood samples will also be taken to monitor levels of serum CK, an enzyme that rises when muscle damage occurs.

During the study, researchers will track changes in muscle strength, walking ability, and breathing function from the start of the trial to its end. Any new health problems or worsening of existing symptoms will be recorded, and the overall safety of the medication will be closely monitored throughout the study period.

Participants will receive the medicine according to the doctor’s plan and will be followed for several months. During the follow‑up they will have routine lab tests that check things such as the amount of protein in the urine, kidney function measured by eGFR, and blood levels of waste products. Doctors will also record any serious side effects, hospital visits, or need for treatments like dialysis, kidney transplant, or management of nephrotic syndrome. Information will be collected at regular intervals, such as every few months, to see how the disease and the patient’s health change over time.

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 purpose of the trial is to determine whether MK-1045 can achieve a higher rate of complete remission (no detectable cancer) and improve overall survival (how long participants live) compared with blinatumomab. Participants will receive a series of treatment cycles lasting several weeks, during which doctors will monitor for adverse events (side effects) and check for minimal residual disease negativity (absence of tiny amounts of cancer cells). Some participants may later undergo allogeneic hematopoietic stem cell transplantation, a procedure that replaces diseased blood‑forming cells with healthy ones from a donor, if deemed appropriate by their physicians.

The purpose of the study is to see which approach keeps the cancer from getting worse for a longer time, measured as progression‑free survival. Participants receive a series of IV infusions of the two drugs every few weeks, and those in the combination arm also take the lenvatinib capsule each day. Treatment continues for several months, with regular check‑ups that include scans and safety assessments to monitor how the disease is responding and to watch for any side effects.

Participants will receive the assigned tablet daily and the infusion every few weeks, with regular clinic visits for safety checks, blood tests and imaging scans such as CT scans to look for changes in tumor size. The study will continue until the disease gets worse, a participant stops treatment, or the trial ends. Researchers will record how long the cancer stays stable (progression‑free survival), overall survival, side effects, and quality‑of‑life questionnaires.

Participants are assigned at random to receive either ASP3082 plus chemotherapy or the placebo plus chemotherapy. The study drug is delivered by intravenous infusion, a process where the medication is slowly introduced into a vein. Treatment cycles are repeated every few weeks, and patients are monitored over several months for changes in tumor size, side effects, and overall health status before the study concludes.

The aim is to see whether the combination improves the lining of the intestine better than the injectable medication alone over a 12‑week period. Participants will receive the assigned medication(s) at regular intervals and will have routine visits where doctors may use a thin camera (a endoscopic exam) to look at the intestine and check for improvement. The study will last about three months, after which the results will be evaluated.

The purpose of the trial is to determine whether the experimental drug can lower disease activity compared with a placebo. Adults will be randomly assigned to receive either the study drug or the placebo, and neither the participants nor the study staff will know which treatment is given. Injections will be given at regular intervals over about six months, with clinic visits to assess muscle strength, skin involvement, and any side effects. Researchers will track changes using simple scores that measure overall improvement and skin disease activity, and they will also monitor whether participants can reduce their steroid dose safely.

The purpose of the study is to compare how well the two approaches work and how safe they are in older patients whose tumors have a high level of the protein PDL1 or an unknown level. Participants are randomly assigned to receive either the immunotherapy‑first plan or the chemotherapy‑first plan, and the assigned treatment is continued until the disease progresses or side effects become unacceptable, after which the other treatment is started. The study follows patients for at least a year to see how many are alive after 12 months and also records early deaths within four months.

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 purpose of the trial is to keep participants who are still benefiting from the drug under medical supervision while safety is monitored. After joining, participants will continue to receive opnurasib on a regular schedule for as long as it remains helpful and tolerable. Visits will include simple check‑ups to record any side effects, any changes in dosage, and overall health. The study does not involve any additional invasive procedures beyond the usual care for this condition.

The purpose of the study is to compare the effects of Odevixibat with a placebo in children with Biliary Atresia. Participants will receive either Odevixibat or a placebo once daily. The study will last for up to 104 weeks, during which time the health of the participants will be closely monitored. The main focus will be on the time it takes for participants to require a liver transplant or experience other serious health issues related to their liver condition.

Throughout the study, various health parameters will be assessed, including liver function tests and overall health status. The study aims to provide valuable information on whether Odevixibat can help improve the outcomes for children with Biliary Atresia who have had the Kasai procedure. Participants will be regularly checked for any side effects or changes in their health to ensure their safety during the trial.

The purpose of the trial is to identify a safe and effective dose of the new medication, to see how well it works against these cancers, and to understand how it interacts with other treatments. Early parts of the study increase the dose to find the highest amount patients can tolerate, followed by later parts that treat specific cancer types either alone or combined with the listed drugs. A small group of participants will also receive medicines that are processed by the enzymes CYP3A4 and CYP2C8 to check for possible drug‑interaction effects.

Participants will take the study tablet each day and receive the other medicines by intravenous infusion, meaning the drugs are given through a vein. Regular clinic visits will include physical checks, blood tests, and imaging scans that are evaluated using RECIST criteria, a system that measures how tumor size changes over time. The trial will continue for several months, with close monitoring for side effects, dose adjustments if needed, and assessments of tumor response.

The purpose of the study is to evaluate the efficacy and safety of the radiopharmaceutical alone or in combination with these hormone blockers. Participants receive a series of injections over several months, and those in the combination arms take one of the oral pills daily. Throughout the study, doctors monitor pain levels, overall well‑being, and any side effects, and they check blood tests for an enzyme called alkaline phosphatase that can show how the bones are responding.

The trial follows each person from the first dose until the disease progresses, they stop treatment, or the study ends. Researchers record how long patients live without the disease getting worse (progression‑free survival) and overall survival, as well as quality of life, to understand the potential benefit of the new therapy.

The purpose of the study is to evaluate how the treatment affects the objective response rate, which is the proportion of patients whose tumors shrink or disappear. After enrollment, participants are randomly assigned to one of the three treatment groups and receive the assigned therapy in repeated cycles over several months, with regular clinic visits for safety checks and blood draws. Tumor changes are measured using the standard imaging criteria known as RECIST, and any adverse events are closely monitored. Blood samples are also taken to assess the drug’s pharmacokinetics, meaning how the body absorbs and clears the medication.

Key outcomes include the percentage of patients achieving tumor shrinkage, the time until the disease shows progressive disease (when the cancer grows again), and the length of time patients remain alive, referred to as overall survival. Safety information and laboratory test results are collected throughout the study to understand the treatment’s risk profile.

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 assess the effectiveness of a predefined OCS tapering schedule in adult participants with Generalized Myasthenia Gravis treated with ravulizumab and oral steroids. Participants start with a regular dose of the oral steroid and then follow a step‑by‑step plan to gradually lower (taper) the dose over several weeks, while receiving regular check‑ups to monitor muscle strength and overall health. The study follows participants for a few months, including visits at the beginning, after the tapering period is finished, and during a follow‑up phase to see if the steroid dose can be reduced or stopped without worsening the disease or causing problems such as adrenal insufficiency, a condition where the body’s stress‑hormone production is low.

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 main goal of the trial is to find the dose of GIA632 that works best for increasing skin color in people with this type of vitiligo. Participants will receive a series of injections over several months, with regular check‑ins to monitor any changes in the appearance of the skin and to watch for possible side effects. The study also uses a simple scoring system called the facial Vitiligo Area Scoring Index to measure how much the facial skin has repigmented, helping researchers understand whether the treatment is effective and safe.

The purpose of the study is to find out whether the new medicine can improve breathing and reduce serious flare‑ups compared with the placebo. People who join will be randomly assigned to one of the two groups, and neither the participants nor the study staff will know which treatment each person receives (double‑blind). Over several months, participants will come to the clinic for routine visits where their lung function, symptoms and overall health will be checked.

During the study, participants will receive the injections at set intervals and will have simple breathing tests, such as measuring forced expiratory volume in one second (FEV1), which shows how much air can be exhaled quickly. They will also answer short questionnaires about how they feel, and will have basic safety checks like blood tests and a quick heart rhythm check (ECG). All of this information helps researchers see if the medicine works and is safe.