UC Davis Health Clinical Studies

Experimental Erdafitinib for Relapsed/Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorder

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This is a Phase 2 study of an experimental drug called JNJ-42756493 (erdafitinib). In a Phase 2 study, the goal is to find out what effects, good and/or bad, a drug has on your tumor or type of cancer. We are using JNJ-42756493 (erdafitinib) in this study because it has been shown to block the growth of cancer cells with changes (mutations) in the FGFR1/2/3/4 genes. These mutations help cancer cells grow in test tubes and in animals. You are eligible for this study because your tumor was found to have a mutation in one of these genes. JNJ-42756493 (erdafitinib) is considered a study drug in the treatment of your tumor or type of cancer, we do not know if it will work against the type of tumor you have. A Phase 1 study of JNJ-42756493 (erdafitinib) has been completed in adults with cancer. In the Phase 1 study, researchers determined the dose of JNJ-42756493 (erdafitinib) can be given without too many side effects. This study will be the first time that JNJ-42756494 (erdafitinib) is given to children. The dose used in this study will be based on the dose used in adults. If you have bad side effects, your dose may be decreased. The goals of this study are: The main goal is to test any good and bad effects of the study drug JNJ-42756493 (erdafitinib) on your tumor. A second goal of the study is to evaluate side effects that might be caused by JNJ-42756493 (erdafitinib). Another goal is to learn more about the pharmacology (how your body handles the drug) of JNJ-42756493 (erdafitinib)

Drug study, Phase 2
Any, age 1 to 21 years old

Safety and Dose Study of an Experimental Viral Vector Used for Gene Transfer in Hemophilia A Participants

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This study will test a study drug called experimental BAX 888. It is known as a gene transfer product for people with severe hemophilia A. This is a first-in-human clinical research study, which means BAX 888 has not been tested in human subjects before. The use of BAX 888 in this research study is investigational. This means that BAX 888 has not been approved by the FDA to treat hemophilia A. There is currently not enough information to know how well BAX 888 works to treat conditions like hemophilia A. This will be examined in this clinical research study. The main goals of this study are: • To investigate the safety of BAX 888 when it is given as a single infusion (a slow injection into a vein) to subjects with hemophilia A. • To investigate the optimum amount of BAX 888 necessary for the human body to produce enough Factor 8. • To look at how BAX 888 affects your immune system and the activity of clotting Factor 8 in your blood. • To answer scientific questions about gene transfer for hemophilia A.

Drug study, Phase [/1/,/ /2/]
Male, age 18 to 75 years old

A Study of Gene Edited Autologous Neoantigen Targeted TCR T Cells With or Without Anti-PD-1 in Patients With Solid Tumors

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The purpose of this study is to test the safety and effectiveness of an experimental cancer treatment called NeoTCR-P1. This is a research study for people who have been diagnosed with cancer. Specifically: - advanced melanoma, - bladder cancer, - colorectal cancer, - ovarian cancer, - breast cancer, or - prostate cancer The study is for patients whose cancer has gotten worse after standard therapy and no additional therapies are available. This research study will take some of your own white blood cells, modify them in a laboratory, and re-infuse them back into you. White blood cells are part of the body’s immune system. They help the body fight infection and other immune diseases. Types of white blood cells are granulocytes (neutrophils, eosinophils, and basophils), monocytes, and lymphocytes (T-cells and B-cells). In this study, your T-cells will be modified so that they can identify and possibly kill your cancer cells. Cancer cells have markers, called “neoantigens”, on their surface that are unique and specific to each person’s tumor. You can think of neoantigens as a tumor’s hidden fingerprint. Your T-cells will be modified to recognize your tumor’s specific neoantigen. The modification is a genetic change, or gene transfer, to your normal T-cells. This means that a section of DNA (genetic material) in your T-cells will be replaced. A specific DNA sequence will be put in its place to help your T-cells identify and locate your tumor cells. A receptor is a protein molecule on a cell’s surface. It receives chemical signals from outside the cell and translates them into a response inside the cell. This modification will add a new receptor to your T-cells so that they recognize cancer cells expressing neoantigens specific to your tumor. This type of genetic modification has not been tested in humans. These modified T-cells are called “NeoTCR-P1” T-cells. NeoTCR-P1 will be given back to you through your vein in one infusion. Unlike standard drugs that are broken down by your body, NeoTCR-P1 is a “living drug.” It is able to proliferate (expand the number and type of stimulated T-cells) extensively in participants. The actual number of cells that expand after infusion will vary from participant to participant. The use of NeoTCR-P1 is experimental and has not been approved by the Food and Drug Administration (FDA) for use outside this study. NeoTCR-P1 is being tested in humans for the first time under this study. Providing your blood for the collection of genetic material to be used in the production of the study drug is not optional in this study.

Biological study, Phase 1
Any, age 18 years or older

A Study Testing the Long-term Safety and Effectiveness of Experimental VX-445 Combination Therapy in Cystic Fibrosis

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This study is being done to learn more about the effectiveness and safety of an experimental combination of medicines. The combination of VX-445/tezacaftor (TEZ)/ivacaftor (IVA) will be tested in patients with cystic fibrosis. • The triple combination VX-445/TEZ/IVA is an experimental drug. This means the study drug is not approved by the FDA. It is still being tested for safety and effectiveness. • TEZ in combination with IVA (TEZ/IVA) is approved in certain countries for use in some people living with CF who are 12 years or older. • IVA is approved in a number of countries globally, for use in some people living with CF who are as young as 6 months, depending on the country. If you would like to know in which countries or for which people living with CF TEZ/IVA or IVA has been approved, please ask your Study Doctor.

Drug study, Phase 3
Any, age 12 years or older

A Study of Experimental VX-445 Combination Therapy for Cystic Fibrosis with Genetic Mutations (F508del and Gating or Residual)

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This study will test the effectiveness and safety of the experimental medicine VX-445. It will be combined with TEZ and IVA in subjects with cystic fibrosis (CF). • The triple combination VX-445/TEZ/IVA is an experimental drug. This means the Study Drug is not approved by the FDA and is still being tested for safety and effectiveness. • TEZ combined with IVA (TEZ/IVA) is approved in some countries for use in some people living with CF who are 12 years or older. • IVA, the third Study Drug used in this study, is approved in a number of countries globally. It is used for people living with CF who are as young as 6 months, depending on the country. • Please ask the Study Doctor if you would like to know in which countries or for which people living with CF TEZ/IVA or IVA has been approved.

Drug study, Phase 3
Any, age 12 years or older

A Study of Experimental Genetic Testing for the APOL1 Gene in Kidney Transplantation (APOLLO Study)

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The purpose of this study is to test kidney donors and kidney transplant recipients for apolipoprotein L1 gene (called APOL1). We will test for this gene and its variants (or forms of the gene). We hope to see how these may affect kidney donors and recipients. Genes control the traits that you inherit from your family such as your eye color or blood type. Variation or changes in genes can also put some people at risk for developing certain diseases. Previous research has shown that variation in the APOL1 gene can put people at higher risk for kidney disease. The purpose of this study is to test kidney donors and recipients for APOL1 gene variants. We hope to learn how this gene may impact people who receive a kidney transplant and those who donated a kidney.

Other Study
Any, age -1 years or older

Study of Using "Omics" (genes, metabolites, and proteins from blood and heart tissue) to Learn More About Heart Disease

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This study is being done to find ways to better understand and identify Heart Failure. We are interested in studying “Omics.” This is the study of all molecules, such as genes, metabolites, and proteins in blood, stool, urine and cells of the heart. We will use Omics studies and clinical information to create a comprehensive evaluation of Heart Failure. This will allow us to learn if there are better ways to determine how your heart is doing. Through "Omics" studies, we can identify thousands of genes, metabolites, and proteins. This will help us search for early signs of cardiovascular disease. What we learn can be used to guide treatments and lessen the problems associated with heart disease.

Other study
Any, age 21 years or older

A Study of the Experimental Medicine ANAVEX2-73 For Rett Syndrome

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This research is being done to answer important questions that might help change or improve treatment for Rett Syndrome. Rett syndrome (RTT) is a rare genetic progressive neurodevelopmental disorder. It is mostly linked to mutations in the MECP2 gene. This affects multiple brain functions including communication, gait, and hand use. Because the MECP2 is gene is on the chromosome, few males live past birth. Therefore, most affected individuals with RTT are females. RTT becomes evident between 1.5-3 years of life. Signs are early developmental regression, expressive language and fine motor (hand) function difficulties . Other clinical manifestations are: - repetitive hand movements - impairment in ambulation - severe intellectual disability - breathing abnormalities while awake - seizures - sleep problems - behavioral problems such as anxiety, disruptive behavior, and mood abnormalities. The experimental drug being tested in this study is called ANAVEX®2-73. Since it is still being studied (experimental), it is not yet approved by the U.S. Food and Drug Administration (FDA), and doctors cannot prescribe it. We want to get more information about how ANAVEX®2-73 might: - improve cognition and sleep, - decrease anxiety and other behaviors, - improve motor function and seizures, if applicable in people with RTT.

Drug study, Phase 2
Female, age 18 to 45 years old

A Study of Experimental Human Factor 8 Gene Therapy SB-525 For Severe Hemophilia A

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We invite you to take part in a research study because you have severe hemophilia A and you are a male, 18 years of age or older. Researchers want to find out more about an experimental drug called SB-525. An experimental drug is still being tested and is not approved for sale in the United States by the FDA. SB-525 belongs to a new class of treatment called gene therapy. The main purpose of this study is to see if receiving SB-525 is safe and tolerable. Also, the potential effect of SB-525 for improving blood clotting will be evaluated. SB-525 consists of substances called vectors (also referred to as carriers). Vectors carry a modified version of the gene for making Factor Eight clotting protein (FVIII). The drug is designed by Sangamo. Scientists think that SB-525 might help your body produce more FVIII. The vector used in this study is a recombinant adeno-associated virus (AAV) that has been modified. These are viruses can infect humans but do not cause any known disease. These viruses are commonly-used vectors for gene transfer and have been changed in the laboratory. They can no longer grow or reproduce in the human body because most of their DNA has been removed and replaced with a gene coding for factor eight. After a single infusion of SB-525 into a vein in your arm, the vector can go to the liver to introduce the gene for factor eight. The study team wants to find out if, after SB-525 infusion, your liver cells will produce FVIII protein and release it into your blood circulation.

Biological study, Phase [/1/,/ /2/]
Male, age 18 years or older
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