The study will test an investigational drug known as 4D-710. An "investigational drug" is one that has not yet been approved for use by a Health Authority such as the United States Food and Drug Administration (FDA). 4D-710 is being studied as a potential treatment for cystic fibrosis lung disease. This is the first study to test 4D-710 in humans. The purpose of this study is to see if 4D-710 is safe for use in adults with cystic fibrosis and to see if 4D-710 can help lung function.
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.
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 study is researching an experimental drug called dupilumab. The study is focused on children with active eosinophilic esophagitis (EoE; an inflammatory disease of the esophagus) which impacts feeding and nourishment. The aim of the study is to see how safe, tolerable, and effective the study drug is when given for 24 weeks to children with active EoE. Focsing on participants aged ≥6 months and weighing ≥5 kg and<15 kg.
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.
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.
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 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.
This study is seeking participants with BAG3-associated dilated cardiomyopathy (DCM). BAG3-DCM is a rare genetic disorder. Dilated cardiomyopathy is a condition that causes the heart to have a harder time pumping blood to the rest of the body which can lead to heart failure. Current treatment for BAG3-DCM is focused on improving heart function and preventing advanced heart failure with medicines, procedures and devices.
This study involves gene therapy. This will be the first time that a BAG3 gene containing study drug will be tested in human volunteers. The purpose of this research is to learn whether the investigational gene therapy RP-A701 is safe and effective for patients with BAG3-DCM. Gene therapy involves the addition of one or more genes to your cells to replace a missing gene or correct malfunctioning genes. Investigational means it is not currently approved by the Food and Drug Administration (FDA). RP-A701 will be given as a one time infusion into a vein in your arm.
Participation in this study will last about 2 years and include at least 18 visits including an inpatient hospitalization stay of at least 5 days. Study related procedures include review of your medical records, study drug infusion, immunosuppressant and antibiotic medications, echocardiogram (ultrasound test of your heart) exercise testing, electrocardiogram (recording of your heart's electrical activity), heart biopsy (collecting a piece of heart tissue), cardiac MRI, questionnaires, heart rhythm monitoring and ICD interrogations, and collection of blood, saliva, urine and stool collection. Study related risks related to gene therapy and those related to study procedures including risks of the heart catheterization, radiation, and biopsy, exercise testing, blood draw risks, genetic testing risks, the risk of loss of confidentiality and unknown risks.
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.