The purpose of the study is to find reliable markers, known as biomarkers, that can be used to measure the progression of Huntington’s disease from its early stages. Participants in the study will include both individuals who have symptoms of the disease and those who are in the pre-symptomatic stages, meaning they have the genetic mutation but do not yet show symptoms. The study will take place over a period of time, with participants receiving the injection and undergoing various tests and assessments to track changes in their condition.

Throughout the study, researchers will collect data at different intervals to analyze how the disease progresses in each participant. This information will help in understanding the effectiveness of the biomarkers in predicting disease progression. The ultimate goal is to improve the ability to monitor and treat Huntington’s disease more effectively in the future. Participants will be closely monitored, and their health and safety will be a top priority throughout the study.

Participants in the study will be randomly assigned to receive either metformin or a placebo. 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 approach helps ensure that the results are unbiased. The trial will assess changes in cognitive abilities using specific tests that are sensitive to the progression of Huntington’s disease, even in its early stages.

The study will take place over a period of time, during which participants will take the medication orally. The primary focus will be on cognitive tests that measure various aspects of brain function, such as the ability to process information and verbal fluency. These tests are part of a larger scale used to track the progression of Huntington’s disease. The goal is to determine if metformin can positively impact these cognitive measures and potentially slow down the progression of the disease.

The purpose of the study is to evaluate the safety and tolerability of this new treatment in adults with early-stage Huntington’s Disease. Participants in the study will receive the treatment through a procedure that delivers it directly into the brain. The study will monitor participants over time to observe any changes in their condition and to assess the treatment’s effects on the disease. Some participants may receive a placebo, which is a substance with no active treatment, to help compare the results.

Throughout the study, various health checks will be conducted, including monitoring vital signs, performing electrocardiograms (ECG), and conducting physical and neurological examinations. Blood and urine tests will also be done to ensure the safety of the participants. The study will also look at changes in brain structure using magnetic resonance imaging (MRI) and will measure the levels of the treatment in the body. The trial is expected to continue until the end of 2029, with the aim of gathering comprehensive data on the treatment’s safety and potential benefits for people with Huntington’s Disease.

The purpose of this study is to evaluate how safe and well-tolerated multiple doses of VO659 are when given to participants with these conditions. Participants will receive the treatment in increasing doses to observe how their bodies respond. The study will monitor various health parameters, including physical and neurological examinations, vital signs, and laboratory tests, to ensure the safety of the participants. The study will also look at how the body processes the medication, including how long it stays in the body and how it is eliminated.

Throughout the study, participants will undergo regular check-ups and tests to monitor their health and any potential side effects. The study aims to gather important information that could lead to new treatment options for people with Spinocerebellar Ataxia and Huntington’s Disease. This research is an important step in understanding how VO659 can be used to help manage these challenging conditions.

The purpose of this study is to evaluate the safety and tolerability of AB-1001 in adults with early manifest Huntington’s Disease and to determine the appropriate dose for future research. During the study, participants will receive several types of brain scans including MRI scans with gadoteric acid (a contrast agent), PET scans using fludeoxyglucose, and other medical assessments. Some participants may receive methylprednisolone, a corticosteroid medication, as part of their treatment.

The study will monitor participants for 52 weeks after receiving the treatment. Throughout this period, various tests will be performed to check brain structure and function, measure disease markers in blood and spinal fluid, and assess changes in disease symptoms and daily functioning. These assessments will help determine how well the treatment is working and identify any potential side effects.

The study will be conducted over a period of 12 months. During this time, participants will take the medication orally and will have regular check-ups to monitor their health and the effects of the treatment. These check-ups will include various assessments, such as blood tests and other evaluations, to track changes in the levels of the protein related to Huntington’s Disease. The study aims to gather information on how well PTC518 works and its safety profile.

By participating in this study, researchers hope to gain valuable insights into the potential benefits of PTC518 for individuals with Huntington’s Disease. The ultimate goal is to find a treatment that can help manage the symptoms and progression of this condition. The study is designed to ensure the safety and well-being of all participants throughout the research process.

The purpose of the study is to assess how safely and effectively tominersen, administered as a solution for injection, works in comparison with a placebo. The study is designed to understand if tominersen can help reduce certain signs related to Huntington’s disease that are measured by changes in biomarker levels and overall patient ability to perform daily activities. Participants will undergo evaluations using clinical tests and procedures such as MRI to track changes over time.

Tominersen belongs to a group known as antisense oligonucleotides, which are designed to target and reduce specific proteins in the body, like the mutant huntingtin protein in this case. Throughout the trial, researchers will measure various aspects, including safety indicators and biomarker levels, to determine if tominersen provides any benefits for individuals with early signs of Huntington’s disease.

Participants in the study will take both Thiamine and Biotin orally. Throughout the study, there will be regular check-ups with a neurologist, both in-person and remotely, to monitor the participants’ health and any side effects. Blood and other tests will be conducted to ensure the safety of the treatment. The study will also measure levels of Thiamine in the body and assess changes in the brain and motor functions over time.

The study will last until the end of 2025, with regular visits and assessments to track the progress and effects of the treatment. Participants will be monitored for any changes in their condition, including motor skills and overall quality of life. The goal is to gather information that could help improve treatment options for people with Huntington’s disease in the future.

Participants in the study will be randomly assigned to receive either the PTC518 tablet or a placebo, which looks like the treatment but does not contain the active substance. The study is designed to be double-blind, meaning neither the participants nor the researchers will know who is receiving the actual treatment or the placebo. This helps ensure that the results are not influenced by expectations about the treatment. The study will last for up to 24 months, during which participants will take the tablets and have regular check-ups to monitor their health and the effects of the treatment.

Throughout the study, various assessments will be conducted to monitor the safety of PTC518 and its impact on the disease. These assessments will include checking for any side effects, changes in vital signs, and physical examinations. Additionally, the study will measure changes in brain structure using a method called vMRI and evaluate specific protein levels in the blood and cerebrospinal fluid. The goal is to gather comprehensive data on how PTC518 affects the progression of Huntington’s Disease over time.

Participants in the study will receive MBF-015 in addition to their standard care. The study will monitor how the body processes the medication and its effects on the disease. This includes checking the levels of MBF-015 in the blood and cerebrospinal fluid (CSF), which is the fluid surrounding the brain and spinal cord. The study will also look at changes in certain biological markers that can indicate the progression of the disease, as well as any changes in the participants’ ability to perform daily activities and their overall mental and physical health.

The trial will involve regular visits to the study center for assessments, including blood tests, physical examinations, and possibly MRI scans, which are imaging tests that help visualize the brain. Participants will be closely monitored for any side effects or changes in their condition throughout the study. The goal is to gather information that could lead to better treatment options for people living with Huntington’s disease.