During the study, volunteers will attend regular visits over an 18‑month period, beginning with a baseline assessment and followed by follow‑up visits roughly every six months. At each visit, blood samples will be taken to measure levels of several substances that indicate inflammation, such as IL-1β, IL-6, and TNFα, as well as markers of blood vessel health, clotting, oxidative stress, and cellular aging. Simple explanations of these markers will be provided, for example, describing them as signals that the body uses to show whether inflammation or stress is occurring. Additional tests will track changes in weight, blood sugar control, and cholesterol levels. The overall design allows researchers to compare the changes in these measurements between the Metformin group and the placebo group to see if the drug influences the biological processes associated with aging and inflammation.

The purpose of the study is to find out how well different doses of DII235 work in lowering lipoprotein(a) levels compared to placebo, and to check if the treatment is safe and well-tolerated. The study will also look at whether DII235 can reduce lipoprotein(a) levels to specific target amounts over time. People taking part in this study will have either existing heart and blood vessel disease, which means they have had problems like heart attacks, procedures to open blocked arteries, narrowing of arteries in the heart, brain, or legs, or they have Type 2 diabetes mellitus, which is a condition where the body does not properly control blood sugar levels.

During the study, participants will receive injections under the skin over a period of 18 months. The study doctors will measure lipoprotein(a) levels in the blood at different time points to see how much they change from the beginning of the study. Measurements will be taken at various times including around day 60, day 180, day 240, and day 360. The study will also track any unwanted effects, check blood test results, and monitor vital signs like blood pressure and heart rate to make sure the treatment is safe. Participants will need to continue taking their regular medications for heart health and cholesterol as prescribed by their doctor throughout the study.

The purpose of this research is to determine if VS-01 can help treat overt hepatic encephalopathy when given once daily for up to 4 days. The study will compare two different treatment durations (3 hours and 4 hours) of VS-01 combined with standard care against standard care alone. The medication contains citric acid as its main component and is administered through intraperitoneal delivery, which means it is given directly into the abdominal cavity.

During the study, patients will receive either VS-01 with standard treatment or standard treatment alone. The treatment period lasts up to 4 days, during which doctors will monitor how quickly patients’ symptoms improve. The study will also look at how the body processes the medication and track any changes in blood and abdominal fluid composition to ensure the treatment is safe.

The purpose of the study is to determine whether SGLT-2 inhibitors can improve the nervous system’s control of heart function and slow the progression of cardiovascular autonomic neuropathy in people with type 2 diabetes. Participants in the study will receive SGLT-2 inhibitor treatment for 6 months. During this time, they will undergo tests to measure heart rate variability (changes in time between heartbeats) and other indicators of nerve function related to the heart. Some tests will include monitoring blood sugar levels using a flash glucose monitoring system worn for 2 weeks, and a mixed meal test to assess how the body processes food.

The study is designed for adults over 45 years old with type 2 diabetes whose blood sugar levels are not optimally controlled despite current treatments. Participants will need to be on stable medication regimens for at least 3 months before joining the study.

The study will follow children under 10 years of age over a period of several years to measure changes in their thinking abilities and intelligence. Researchers will use age-appropriate intelligence tests to track how children’s cognitive abilities develop while receiving sepiapterin treatment. During the study, children will continue their prescribed diet that controls protein and phenylalanine intake.

The research will also track quality of life measures and blood phenylalanine levels over time. This long-term follow-up will help determine if sepiapterin can provide lasting protection for brain development in children with PKU.

Participants in the study will receive morphine, and researchers will observe how it is absorbed and how it affects the body. The study will also involve the use of naloxone hydrochloride, an antidote for morphine, and lidocaine hydrochloride, a local anesthetic, to better understand their roles in managing pain and potential side effects. The study will take place over a short period, with participants receiving these medications through injections.

The goal is to develop a more personalized approach to treating pain, particularly for those with obesity and sleep apnea, by understanding how these conditions affect the way morphine works in the body. This research could lead to better pain management practices for surgical patients, especially those undergoing weight-loss surgery. The study is expected to provide valuable insights that could benefit future patients by tailoring pain relief methods to individual needs.

The purpose of the study is to compare the accumulation of the Ga-exendin tracer in the pancreas of women with and without a history of gestational diabetes. This will help researchers understand the differences in beta cell function between these two groups. The study will involve a series of imaging sessions where participants will receive the tracer and undergo PET/CT scans to assess how the tracer accumulates in their pancreas.

Participants will be monitored over a period to observe any changes in the tracer’s accumulation and to correlate these findings with the function of their beta cells. This research could provide valuable insights into the long-term effects of gestational diabetes on pancreatic function and help in developing better diagnostic and treatment strategies for managing diabetes in the future.

The main goal of the study is to assess how the body absorbs and processes a single dose of Rivaroxaban when Sotorasib is present in the body at a steady level. Participants will take these medications orally, in the form of film-coated tablets. The study will also look at the safety of taking these medications together, using a standard method to evaluate any side effects.

Participants will be involved in the study for a period of time, during which they will take the medications and have their health monitored. The study aims to provide valuable information on the interaction between these two drugs, which could help in understanding their combined effects in the body. This information is important for ensuring the safe use of these medications in the future.

The purpose of the study is to see if this imaging method can help classify the progression of diabetic kidney disease. Participants in the study will receive injections of these substances and undergo MRI scans to assess kidney function. The study will look at how well the kidneys are working by measuring things like metabolism and blood flow in the kidneys. The study will also check how reliable the imaging method is by repeating the scans to see if the results are consistent.

Throughout the study, participants will be monitored to ensure their safety and to gather information on how their kidneys are functioning. The study aims to provide new insights into diabetic kidney disease and potentially improve how this condition is diagnosed and monitored in the future.

Evolocumab is a medication known as a PCSK9 inhibitor, which helps lower cholesterol levels by increasing the liver’s ability to remove cholesterol from the blood. It is administered as a solution for injection under the skin. Atorvastatin is a type of drug called a statin, which is taken orally in tablet form. Statins work by reducing the amount of cholesterol produced by the liver.

The purpose of this study is to learn more about how these medications influence cholesterol and bile acid metabolism. Participants will receive either the medication or a placebo and will be monitored for changes in their cholesterol and bile acid levels over a period of time. This research aims to provide valuable insights into the effects of these drugs on the body’s metabolic processes.

The purpose of the study is to improve knowledge about how the body naturally produces a hormone called oxytocin in people with hypopituitarism. Participants will receive either the study medication or a placebo, and the study will follow a randomized, crossover design. This means that participants will receive both the medication and the placebo at different times during the study. The study will look at how the medication affects the pattern of oxytocin hormone secretion.

Throughout the study, researchers will also assess various aspects of participants’ well-being, such as mood, quality of life, and eating behavior, to see how these might be related to changes in oxytocin levels. The study is expected to continue until the end of 2024, providing valuable insights into the relationship between hypopituitarism and oxytocin production.

The purpose of the study is to explore how effective semaglutide is in treating young adults with early-onset obesity, especially those who have not had success with lifestyle changes alone. Participants will be divided into groups based on their previous response to lifestyle interventions. Some participants will receive semaglutide, while others will receive a placebo. The study aims to understand the differences between those who respond well to lifestyle changes and those who do not.

Throughout the study, researchers will monitor changes in body weight and body composition, which refers to the proportion of fat and non-fat mass in the body. They will also track how many participants achieve significant weight loss, defined as losing at least 5%, 10%, 15%, or 20% of their body weight. The study is expected to provide valuable insights into the treatment of obesity in young adults and help identify the underlying mechanisms that make some individuals more resistant to lifestyle changes.

The purpose of the study is to find out if a lower dose of Metformin is as effective as a higher dose in improving health outcomes for women with PCOS. Participants will take the medication in the form of tablets, which are to be taken orally. The study will last for a period of 48 weeks, during which participants will be monitored for changes in weight, hormone levels, and any side effects such as nausea or abdominal pain. The study will also assess the impact of Metformin on menstrual regularity and overall quality of life.

Throughout the study, various health indicators will be measured, including weight, hormone levels like testosterone, and metabolic markers such as fasting glucose and insulin levels. The study aims to provide valuable insights into the optimal use of Metformin for managing PCOS, potentially leading to improved treatment strategies for women affected by this condition.

Participants in the study will be randomly assigned to receive either Verapamil SR or a placebo, which looks like the medication but does not contain the active ingredient. The study will last for 12 months, during which participants will take the medication or placebo once daily by mouth. The main goal is to observe changes in the body’s ability to produce insulin in response to food intake over this period.

Throughout the study, participants will undergo regular check-ups and tests to monitor their health and the effects of the treatment. These tests will include measuring the levels of a substance called C-peptide, which indicates how well the body is producing insulin. The study will also track other health markers, such as blood sugar levels and insulin requirements, to gather comprehensive data on the potential benefits of Verapamil SR for people with Type 1 Diabetes.

The purpose of the study is to see if oxytocin can help reduce anxiety and improve the ability to recognize facial and body expressions in people with central diabetes insipidus. Participants will use the nasal spray twice daily for about 28 days. During this time, researchers will assess changes in anxiety levels and the ability to correctly identify emotions through specific tasks. The study aims to determine if these improvements are greater in those receiving oxytocin compared to those using the placebo.

Throughout the study, additional outcomes will be monitored, such as emotion recognition, empathy, and responses to stress. Researchers will also look at how the brain responds using MRI scans, as well as other psychological and metabolic outcomes. Safety will be a key focus to ensure the well-being of all participants. The trial is expected to provide valuable insights into the potential benefits of oxytocin for individuals with central diabetes insipidus.

The study is testing a medication called empagliflozin, which is usually used to treat diabetes, to see if it can help increase the number of neutrophils in patients with GSD Ib. The purpose of the study is to evaluate the safety and how well patients tolerate empagliflozin when used for treating neutropenia in GSD Ib. Some participants will receive empagliflozin, while others may receive a placebo. The study will monitor participants over a period to see if empagliflozin helps restore neutrophil numbers and function.

Participants in the study will take the medication in the form of film-coated tablets. The study will involve regular visits to monitor the safety of the treatment and to check for any side effects. The goal is to gather information on how safe empagliflozin is for patients with GSD Ib and whether it can effectively help manage neutropenia in these patients.

Participants in the study will receive either sotagliflozin or a placebo, which is a tablet that looks like the medication but does not contain the active drug. The study will last for about three months, during which the effects of the medication on kidney health will be monitored. This will be done using a special imaging technique called MRI (Magnetic Resonance Imaging) to measure how well the kidneys are being supplied with oxygen. The goal is to see if sotagliflozin can improve kidney oxygenation compared to the placebo.

The study aims to provide valuable insights into how long-term use of sotagliflozin might help manage kidney health in people with Type 1 Diabetes and chronic kidney disease. By understanding these effects, researchers hope to find better ways to support kidney function and slow down the progression of kidney disease in these patients.

The purpose of this research is to determine if short-term treatment with vildagliptin can prevent the development of diabetes in the first year after kidney transplantation. The medication is taken by mouth, with patients receiving up to 100 mg daily for a period of 8 weeks. During the study, patients will receive standard medications that help prevent rejection of the transplanted kidney, including tacrolimus, mycophenolic acid, and steroids.

Throughout the study, patients will have their blood sugar levels monitored regularly. Other aspects of health will also be checked, including kidney function, blood composition, and overall well-being. The study will track how many patients develop diabetes within one year after their transplant. Diabetes will be identified through blood sugar measurements or the need for diabetes medication.

The purpose of the study is to evaluate the safety and effectiveness of elafibranor in adults with PSC. Participants will take the medication daily for a period of 12 weeks during the initial phase of the study. After this period, there is an option for participants to continue in a long-term extension phase, where they can receive elafibranor for a longer duration to further assess its safety and tolerability. Throughout the study, participants will have regular check-ups to monitor their health and any changes in their condition.

This study aims to provide valuable information about the potential benefits and risks of using elafibranor for treating PSC. By participating, individuals with PSC can contribute to research that may lead to better treatment options for this challenging liver disease. The study is conducted under strict guidelines to ensure the safety and well-being of all participants.

The purpose of the study is to observe how Verapamil SR affects the preservation of beta-cell function over a long period. Participants in the study will take 360 mg of Verapamil SR orally once a day. The study will last for 24 months, during which participants will have their beta-cell function measured at the start and at the end of the study. This will be done using a test called the mixed-meal tolerance test (MMTT), which helps assess how well the body is producing insulin. Some participants may have previously been treated with either a placebo or Verapamil SR for 12 months in a prior study.

Throughout the study, changes in blood glucose control, insulin requirements, and any severe low blood sugar episodes will be monitored. Additionally, the study will track any adverse events or changes in vital signs, such as heart rate and blood pressure, to ensure the safety of the participants. The study aims to provide valuable insights into whether Verapamil SR can help maintain insulin production in people with Type 1 diabetes, potentially leading to better management of the condition.

Participants in the study will take PTC923 for a period of up to 24 months. During this time, researchers will monitor the safety of the treatment by checking for any side effects and conducting regular health assessments, including laboratory tests and physical exams. The study will also look at how the treatment affects the participants’ quality of life and their ability to tolerate dietary phenylalanine. Some participants will be asked to complete questionnaires about their quality of life, depending on their age and language.

The study aims to provide valuable information about the safety and effectiveness of PTC923 in managing Phenylketonuria. By understanding how this treatment works over a longer period, researchers hope to improve the care and dietary management of individuals with PKU. The trial is open-label, meaning that both the participants and the researchers know which treatment is being administered. This approach helps in closely monitoring the effects of the treatment throughout the study.

Participants in the study will be randomly assigned to receive either the combination of Zibotentan and Dapagliflozin or Dapagliflozin alone. Some participants may receive a placebo. The study will last for about 24 months, during which time participants will take the medication orally, in the form of a film-coated tablet. Throughout the study, participants will have regular check-ups to monitor their kidney function and overall health. The main focus will be on changes in kidney function over time, as well as other health indicators related to kidney disease.

This study aims to provide valuable information on whether the combination of Zibotentan and Dapagliflozin can offer better protection for the kidneys in people with Chronic Kidney Disease and High Proteinuria. By participating, individuals will contribute to research that could lead to improved treatments for this condition in the future.

The purpose of the study is to evaluate the safety of OP-1250 and to see how well it works compared to the standard treatments. Participants in the study will receive either OP-1250 or one of the standard treatments. OP-1250 is taken as a tablet by mouth, while some of the standard treatments are given as injections. The study will last for a period of up to 42 days, during which the effects of the treatments will be monitored.

Throughout the study, doctors will closely observe participants to ensure their safety and to gather information on how the treatments affect the cancer. This includes checking for any side effects and measuring how the cancer responds to the treatment. The information collected will help determine if OP-1250 could be a better option for treating this type of breast cancer in the future.

Participants in the study will be randomly assigned to receive either Sepiapterin or Sapropterin for a certain period, and then they will switch to the other treatment. This type of study is called a crossover study. The study will last for several weeks, during which participants will take the medication in the form of either soluble tablets or powder for oral use. The researchers will monitor the participants’ blood phenylalanine levels to see how well each treatment works. Some participants may also receive a placebo during the study.

The study aims to provide valuable information about the effectiveness of Sepiapterin compared to Sapropterin in managing PKU. By participating, individuals with PKU can contribute to research that may improve treatment options for this condition in the future. The study is open to both male and female participants who are at least two years old and have been diagnosed with PKU. Participants will need to continue their current diet and follow the study procedures as instructed by the research team.

The purpose of the study is to see if semaglutide can help people with HIV and obesity lose more weight than they would with just diet and exercise. Participants in the study will receive semaglutide as an injection using a pre-filled pen. The study will last for several weeks, during which participants will be monitored for changes in their body weight and other health markers. Some participants will receive a placebo instead of semaglutide to help researchers understand the true effects of the medication.

Throughout the study, researchers will also look at other health factors, such as changes in body fat, immune system function, and overall quality of life. The study aims to provide valuable information on how semaglutide can be used to support weight loss in people living with HIV and obesity, potentially leading to better health outcomes for this group.

The purpose of the study is to evaluate the safety and tolerability of mRNA-3745 in participants with Glycogen Storage Disease Type 1a. Participants will receive the treatment through an intravenous (IV) injection, which means it will be administered directly into a vein. The study will start with a single dose and may progress to multiple doses, depending on how participants respond. Throughout the study, participants will be closely monitored to see how their bodies react to the treatment and to check for any side effects.

This study is designed to be adaptive, meaning that the researchers may adjust the treatment plan based on the results they observe. Participants will be involved in the study for a period of time, during which they will have regular check-ups and tests to monitor their health and the effects of the treatment. The study aims to provide valuable information about the potential benefits and risks of using mRNA-3745 for treating Glycogen Storage Disease Type 1a.

The purpose of the study is to evaluate the safety and effectiveness of mRNA-3705 in people with MMA. The study is divided into two parts. In the first part, researchers will focus on understanding how safe and tolerable the treatment is for participants. In the second part, they will look at how well the treatment works by comparing the frequency of metabolic decompensation events (MDEs), which are episodes when the body cannot properly process certain substances, with the standard care currently available.

Participants in the study will receive the treatment and be monitored over time to see how their bodies respond. The study will also measure changes in the levels of certain substances in the blood, as well as any side effects that may occur. This research aims to provide valuable information that could lead to better treatment options for people living with Isolated Methylmalonic Acidemia.

Participants in the study will receive tacrolimus orally, which means they will take it by mouth. The study will last for a period of 14 days. During this time, researchers will monitor changes in the participants’ glycemic profile, which refers to the levels of sugar in the blood. The goal is to determine if there is a significant change in blood sugar levels from the start of the treatment to the end of the 14-day period.

This study is important for understanding how tacrolimus might affect blood sugar levels in patients who are on dialysis and preparing for a kidney transplant. The findings could help improve the management of these patients’ conditions and provide insights into the safe use of tacrolimus in this specific group of patients.

The purpose of the study is to evaluate the safety and feasibility of administering Diamyd through injections into a lymph node in the groin area. Participants in the study will receive either two or three injections, spaced one month apart. The study is open to individuals aged 8 to under 18 years who have a specific genetic marker known as the HLA DR3-DQ2 haplotype and have multiple antibodies associated with Type 1 diabetes, indicating they are at increased risk for developing the disease.

The study aims to determine if this method of administering Diamyd is a viable option for preventing or delaying the onset of Type 1 diabetes in those at risk. Participants will be monitored to assess the safety of the treatment and to gather information on how well the treatment can be administered in this way. The study is expected to continue until September 2026.

The purpose of the study is to evaluate whether the combination of these medications, or each one individually, is better than a placebo in improving liver health over a period of 48 weeks. Participants will be randomly assigned to receive either the combination therapy, one of the medications, or a placebo. The study is designed to be double-blind, meaning neither the participants nor the researchers will know who is receiving which treatment until the study is completed. This helps ensure that the results are not biased.

Throughout the study, participants will have regular check-ups and tests to monitor their liver condition and overall health. The main goal is to see if there is an improvement in liver disease activity and no worsening of liver fibrosis after 48 weeks. The study will also look at other factors, such as the resolution of liver inflammation and fat buildup. Participants will be closely monitored to ensure their safety and well-being during the trial.

The purpose of the study is to determine if the combination of Balcinrenone and Dapagliflozin is more effective in reducing the amount of albumin in the urine than Dapagliflozin alone. Participants in the study will be randomly assigned to receive either the combination treatment or Dapagliflozin by itself. The study will last for about 12 weeks, during which time participants will take the medication orally, meaning by mouth, in the form of tablets or capsules.

Throughout the study, participants will have regular check-ups to monitor their health and the effects of the treatment. The main focus will be on the change in the level of albumin in the urine from the start of the study to the end of the 12 weeks. This will help researchers understand the potential benefits and safety of the combination treatment for people with Chronic Kidney Disease and albuminuria.

The study will involve participants receiving three doses of Diamyd over a period of time, with the goal of assessing its impact on the body’s ability to produce insulin and maintain blood sugar levels. Participants will be monitored for changes in their insulin production and blood sugar control over the course of the study. The study will also look at the safety of the treatment by tracking any side effects or reactions that occur during the trial.

In addition to Diamyd, the study will also involve the use of Divisun 2000 IE tabletter, which contains colecalciferol, commonly known as Vitamin D3. This is a vitamin supplement taken orally in tablet form. The study will last for a period of up to 24 months, during which participants will undergo regular assessments to monitor their health and the effects of the treatment. The overall aim is to determine if Diamyd can help preserve the body’s natural insulin production in people with newly diagnosed Type 1 Diabetes.

The purpose of this study is to evaluate how safe mRNA-3705 is when used over a longer period. Participants will receive the treatment through an intravenous infusion, which means it is delivered directly into the bloodstream. The study will monitor participants for any side effects and changes in their condition over time. This includes looking at levels of certain substances in the blood that are related to MMA, as well as any changes in the frequency of hospital visits or the impact of the disease on daily activities like school or work.

In addition to mRNA-3705, the study may involve other medications such as dexamethasone, paracetamol, cetirizine dihydrochloride, ibuprofen, and famotidine, which are used to manage symptoms or support the treatment process. Some participants may receive a placebo, which is a substance with no active medication, to help compare the effects of the treatment. The study is expected to continue until January 2029, allowing researchers to gather comprehensive data on the long-term safety and effectiveness of mRNA-3705 in managing MMA.

Participants in the study will receive metreleptin for a period of 24 months. During this time, researchers will monitor the effects of the treatment on the participants’ health, focusing on changes in blood sugar levels and fat levels in the blood. The study will also look at how the body responds to the treatment over time, including any potential immune reactions. Some participants may receive a placebo, which is a substance with no active medication, to compare the effects of metreleptin.

The study aims to gather information on how metreleptin can help manage symptoms of partial lipodystrophy, such as high blood sugar and high levels of fats in the blood. By the end of the study, researchers hope to better understand the benefits and any risks associated with metreleptin treatment for this condition. Participants will be closely monitored throughout the study to ensure their safety and well-being.

The purpose of the study is to assess the safety and tolerability of AZD4831 in adults with NASH who do not have cirrhosis but do have liver fibrosis, which is a type of scarring. The study will also look at how the body processes the drug. Participants will be randomly assigned to receive either AZD4831 or a placebo, and neither the participants nor the researchers will know who is receiving which treatment. The study will last for about 12 weeks, during which time participants will have regular check-ups to monitor their health and the effects of the treatment.

Throughout the study, participants will have their liver function and other health markers checked to see how they change over time. The main focus will be on changes in liver enzymes, which are indicators of liver health. The study aims to provide valuable information on the potential benefits and safety of AZD4831 for people with NASH, which could lead to new treatment options for this condition in the future.

The purpose of the study is to assess the safety and effectiveness of daily injections of Metreleptin over a 12-month period. Participants will receive the treatment through a subcutaneous injection, which means the medication is injected under the skin. The study will monitor changes in health markers such as blood sugar levels and fat levels in the blood, which are often affected in people with partial lipodystrophy.

Throughout the study, participants will have regular check-ups to track their progress and any changes in their condition. The study aims to provide valuable information on how Metreleptin can help manage partial lipodystrophy and improve the quality of life for those affected by this condition.

The purpose of the study is to evaluate how well metreleptin works in managing the symptoms of generalized lipodystrophy and its associated conditions. Participants in the study will receive metreleptin injections over a period of 12 months. Throughout the study, researchers will monitor changes in the participants’ health, including their blood sugar levels and fat levels in the blood. The study aims to understand how metreleptin affects these health markers and whether it can help improve the quality of life for young children with this condition.

Participants will be closely observed by healthcare professionals to ensure their safety and to gather important information about how metreleptin works in their bodies. The study is open-label, meaning that both the participants and the researchers know that metreleptin is being administered. This trial is an important step in finding better treatments for children with generalized lipodystrophy and related health issues.

The purpose of the study is to understand how abiraterone affects the metabolism of oxycodone, which is a pain-relieving medication. Participants will receive a single dose of 15 mg of oxycodone in the form of a hard capsule. The study will compare the effects of oxycodone in men who are taking abiraterone with those who are not. This will help researchers learn more about how these medications interact in the body.

Throughout the study, participants will be monitored to see how their bodies process oxycodone. This involves measuring certain aspects of the medication in the body over time. The study aims to provide valuable information that could help improve the treatment of pain in patients with prostate cancer who are also taking abiraterone. Participants will be part of the study for a short period, and the research is expected to conclude by April 2024.

The study will use various substances naturally present in the body (called biomarkers) to determine which patients might respond better to which medication. These biomarkers include substances like adiponectin, irisin, and others that can indicate how well the body processes sugar. The study will also measure standard indicators of diabetes control such as HbA1c (a measure of average blood sugar levels over time).

Participants in the study will be taking either dapagliflozin tablets or semaglutide tablets while continuing their usual metformin treatment. Some patients may also receive insulin glargine, which is given by injection under the skin. The study will monitor how these different treatments affect blood sugar control and other body functions related to diabetes.