Dystonia is a movement disorder that causes muscles to contract and/or spasm. This may be painful and can affect the person's ability to complete daily tasks. Dystonia may affect one or multiple parts of the body. Botulinum toxins (BoNT) are the only approved drug in the United States to treat dystonia, and this is only for dystonia of the neck or the eye. There are currently no approved oral treatments for dystonia. Most current treatments only provide relief of symptoms.
The purpose of this study is to learn about the effects of the research drug (VIM0423), to find the best dose for treating dystonia, and to see how safe VIM0423 is for patients with dystonia.
This research study is studying VIM0423 as a possible treatment for dystonia. It is being developed to be a combination dose of: VMA-1001 given with VMA-1002.
• VMA-1001 and VMA-1002 will be taken in separate oral doses at the same time.
• VMA-1001 is an extended release (ER) modified version of trihexyphenidyl (THP).
• VMA-1002 is a formulation of bethanechol (BTC).
THP and BTC are medicines approved by the U.S. Food and Drug Administration (FDA); however, the Sponsor is investigating a different formulation of THP referred to as VMA-1001 and a different formulation of BTC referred to as VMA-1002. The purpose is to attempt to minimize some side effects of THP and is therefore considered an investigational drug in this study. An investigational use is one that is not approved by the FDA.
You may be in this study for up to 32 weeks from the time you consent until the last study visit.
You will be seen at the study site 6 times (Screening, Day 1, Day 30, Day 60, Day 95, and Day 125) and will complete 4 telephone calls (Day 6, Day 13, Day 20 and Day 105). You may be asked to come for extra visits at any time during the study if the study doctor decides that extra tests are needed for your safety.
Side effects associated with the study drug are dry mouth, dry eyes, blurred vision, dizziness, mild nausea and feeling nervous.
You do not need to take part in this study to receive treatment for your isolated dystonia. The study doctor will explain other options that are available to you. Your other choices may include treatment with other medicines for isolated dystonia, another investigational treatment, treatment that makes you feel more comfortable but will not have an effect on your isolated dystonia, or no treatment.
The purpose of this study is to test the safety of NXC-201 at different doses in participants with relapsed/refractory AL amyloidosis, and to confirm the best dose for further testing. In addition, the study will evaluate the effectiveness of NXC-201 in treating relapsed/refractory AL amyloidosis.
AL amyloidosis is a rare systemic disorder caused by an abnormality of plasma cells (a type of white blood cell that is part of the immune system) in the bone marrow. Misfolded proteins produced by these cells can build up in and around tissues, nerves and organs, gradually affecting their function. This can cause progressive and widespread organ damage.
NXC-201 is made using a person's own T Cells (immune system cells that protect the body from infections, cancer, and other possible harms). The T cells are collected then genetically modified (changes are made to the DNA or genes) outside of the body in a laboratory. A virus is used to introduce a gene that creates a protein (called a chimeric antigen receptor or CAR) on the surface of T cells. The virus then becomes inactive. The changes are designed to help the NXC-201 cells find and destroy plasma cells that have a protein on their surface called B-cell maturation antigen (BCMA). T-cell therapies like NXC-201 are called CAR T-cell therapies. After being reinjected, the CAR-T cells multiply and spread throughout the body.
NXC-201 is an investigational "treatment", which means it has not been approved by the US Food and Drug Administration (FDA) for the treatment of AL Amyloidosis or any other disease.
Calling the study drug a "treatment" in this consent form does not indicate that it will be effective in treating your AL Amyloidosis.
Before receiving NXC-201, participants will receive lymphodepleting chemotherapy (or lymphodepletion) with cyclophosphamide and fludarabine to briefly weaken (suppress) your immune system. The lymphodepletion will help prepare the body for receiving NXC-201. Cyclophosphamide and fludarabine are FDA-approved for use as lymphodepleting chemotherapy.
This study is sponsored by Nexcella, Inc., which is responsible for funding and organizing the study.
This is a randomized, double-blind, multicenter Phase IIIb study comparing remibrutinib tablets with dupilumab injections in adults with chronic spontaneous urticaria (CSU) that is not well controlled by second-generation H1-antihistamines. Participants will receive either remibrutinib or dupilumab for 12 weeks, alongside their usual antihistamine. The goal is to see which treatment works faster and better at reducing symptoms like hives and itching. If remibrutinib is not yet available commercially after the study, participants may continue taking it in an optional extension phase.
The investigational varicella vaccine (hereafter referred to as VNS vaccine) is a new
candidate varicella vaccine derived from the Oka strain. The main rationale for the
development of VNS vaccine is to provide an additional alternative varicella vaccine as an advantage from a public health perspective to prevent varicella disease
This research explores how ultrasound, a common medical imaging tool, can be used to look at the ligaments that support the base of the thumb. These ligaments are important because they help the thumb stay stable during everyday tasks like opening jars, writing, or buttoning clothes. When these ligaments become weak or damaged, it can cause pain and lead to joint problems like arthritis. Right now, it's not easy for doctors to check the condition of these ligaments without advanced scans or surgery. By using ultrasound, which is safe and non-invasive, we hope to find a quicker and easier way to examine ligament health.
Many people with Overactive Bladder (OAB) continue to have symptoms such as urinary urgency, frequency, and leakage even while taking medication. This research study is being done to see whether using the ZIDA sock, an FDA approved wearable device, can help reduce symptoms when added to a person's usual OAB medication. The ZIDA sock provides mild electrical stimulation to a nerve near the ankle and is worn like a regular sock. Participants will use the ZIDA sock at home once a week for 30 minutes over 12 weeks. Participants will be asked to complete bladder diaries to record urinary symptoms and questionnaires about their bladder symptoms, quality of life, and satisfaction with the device. Study staff will also ask about any side effects or discomfort related to device use. The information collected will help determine whether the ZIDA sock is a helpful add-on treatment for people with OAB who still have symptoms despite medication.
This study is for patients who have been diagnosed with bladder cancer and are eligible to receive chemotherapy and radiation therapy while keeping their bladder. The study is testing an investigational radiation schedule called stereotactic body radiation therapy (SBRT), which delivers higher doses of radiation over fewer treatment sessions. "Investigational" means it has not been approved by the United States Food and Drug Administration (FDA).
Participants will be randomly assigned (like flipping a coin) to one of two groups. One group will receive the usual chemotherapy and standard radiation therapy schedule, which involves lower radiation doses given five days a week for 4 to 5 weeks. The other group will receive the usual chemotherapy and the study radiation schedule (SBRT), which delivers higher doses of radiation in fewer treatments over a shorter period of time.
The primary purpose of this study is to find out whether the shorter radiation schedule is as effective as the usual radiation schedule at preventing bladder cancer from getting worse and avoiding bladder removal surgery.
Radiation therapy and chemotherapy will be given while the bladder remains in place. After treatment is complete, participants will be followed by the study doctor for up to 5 years to monitor for side effects and whether the cancer returns or progresses.
All participants can expect to complete questionnaires about symptoms and quality of life at several time points during and after treatment. These questionnaires are part of the research and will not be shared with treating physicians. There will be a total of 16 patients enrolled locally over 48 months.
This study focuses on adolescents who experience both substance use problems and post-traumatic stress disorder (PTSD), which often occur together and can seriously impact mental health, school performance, and relationships if left untreated. The goal is to evaluate a trauma-focused treatment called Concurrent Treatment of PTSD and Substance Use Disorders for Adolescents (COPE-A), which is designed to address both conditions at the same time. Earlier pilot studies showed that COPE-A is safe, feasible, and effective in reducing substance use and PTSD symptoms compared to a supportive counseling approach.
In this larger study, 120 adolescents from across the U.S. will receive treatment through telehealth. Researchers will compare COPE-A to person-centered therapy to see which leads to greater improvements in substance use and PTSD symptoms. The study will also examine effects on depression, HIV risk behaviors, relationships, and overall quality of life, while using brief daily surveys to track changes over time.
The purpose of this study is to better understand how the oral microbiome (bacteria in the mouth) may be associated with different neurologic conditions like Alzheimer's disease and Multiple Sclerosis. This will require one visit, typically less than an hour. We will collect some information about the subject's past medical history including dental history as well as collect saliva and perform a nasal swab. This will also include a measure of genetics. There will be an optional blood collection. Samples will also be stored for future research.
The Car study is being done to collect real-world evidence of how commercially available MicroVention intracranial aneurysm treatment devices function and look at safety outcomes over a year when used per standard of care. The treating physician will use the device they think is most appropriate for each patient. The study will also be used to develop a full database in an effort to inform future studies and further the understanding of the safety and performance of these devices across a wide range of patient populations and disease characteristics.