This study is for patients that have been diagnosed with metastatic or locally advanced unresectable solid tumors with tumor protein 53 (TP53) mutation/loss with or without brain metastasis. This study is testing an investigational drug called AO-252. "Investigational" means it has not been approved by the United States Food and Drug Administration (FDA). The primary purpose of this study is to determine the maximum tolerated dose (MTD), the recommended phase II does (RP2D), and the safety profile of AO-252. The drug is given to participants orally. Participants can expect to be on this study for approximately 24 months, followed by a 12-month follow-up period.

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.

Study B7981028 is a Phase 3 long-term, double-blind extension study aimed at evaluating the safety and efficacy of ritlecitinib in participants with severe alopecia areata (AA). This study includes individuals who have completed previous ritlecitinib studies, B7981031 or B7981027, and are eligible to enroll in the B7981028 study. The research seeks to gather more comprehensive data on the treatment's effects over an extended period.

This study is a double‑blind, placebo‑controlled research study to evaluate the safety and effectiveness of a skin patch treatment for peanut allergy in children ages 1 to 3. The patch delivers a very small amount of peanut protein through the skin and is designed to help the immune system become less sensitive to peanuts over time.

Participation in the study will last approximately 34 weeks. Participation is voluntary, and participants may withdraw at any time.

The purpose of this Phase III study is to evaluate the efficacy and safety of iptacopan compared to placebo (both administered in combination with standard of care) in adult participants aged at least 18 years to ≤ 60 years and adolescents (12-17 years in non-EU countries and 16-17 years in EU countries) with idiopathic IC-MPGN. The study aims to demonstrate a reduction in proteinuria and stabilization in estimated glomerular filtration rate (eGFR) in participants treated with iptacopan compared to placebo. Change in patient-reported fatigue will also be evaluated. Alternative complement pathway (AP) dysregulation is believed to underlie the clinical manifestations and progression of IC-MPGN. Serum complement (C3) and other complement pathway biomarkers will be assessed to demonstrate that iptacopan reduces AP activity and targets the underlying cause of disease.

This study is for male subjects that have been diagnosed with prostate cancer. Subjects are expected to remain in the study for a minimum of 12years or longer. There will be a total of 6 subjects locally enrolled. Subjects may experience the following risks: Bladder or prostate capsule perforation, Bladder neck contracture, Bleeding or blood in the urine, Bruising, Cancer progression, Electric shock/burn, Embolism, Incontinence or overactive bladder, and Infection.

Based on the research priorities identified by Cystic Fibrosis families and clinicians, the goal of the STOP PEDS randomized controlled trial is to evaluate the long- and short-term safety and efficacy of the antibiotic strategies in the management of outpatient Pulmonary Exacerbation in children with Cystic Fibrosis.

This phase 2, multicenter, randomized, placebo-controlled, double-blind, dose-ranging trial evaluates the efficacy and safety of zasocitinib in participants with nonsegmental vitiligo. The maximum trial duration for an individual participant is approximately 61 weeks (427 days), including a screening period of up to 35 days, a treatment period of up to 52 weeks, and a 4-week safety follow-up period. Participants will be randomly assigned to a blinded treatment with zasocitinib 15 mg QD, 30 mg QD, 75 mg QD, placebo/zasocitinib 30 mg, or placebo/zasocitinib 75 mg QD, via an IRT system.

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 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.