UC Davis Health Clinical Studies

A Study to Test Dystrophin (muscle protein) Levels in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

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Your child has been invited to take part in this research study because he has Duchenne Muscular Dystrophy (DMD). DMD is caused by a mutation (or error) in the gene for dystrophin. Dystrophin is a protein that keeps muscles healthy by keeping the structure of muscle cells. One type of mutation in the dystrophin gene is called nonsense mutation. This type of mutation is the cause of DMD in about 15% of boys with the disease. A nonsense mutation in the dystrophin gene causes a premature stop signal. This premature stop signal tells the body to stop making the dystrophin protein in muscle before the protein is complete. The result is a shortened dystrophin protein that does not work and weakens the muscles. The purpose of this research is to measure levels of dystrophin. It will be measured before and after nine months of treatment with an investigational drug called ataluren. This study is for children who: - are at least 2 years old and younger than eight years old. - can walk. - have Duchenne Muscular Dystrophy (DMD) caused by a nonsense mutation of the dystrophin gene.

Drug study, Phase 2
Male, age 2 to 7 years old

A Study of Experimental Combination Treatment with Chemotherapy, Nivolumab, and / or BMS-986205 for Bladder Cancer

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This study will test the effectiveness (how well the drug works), safety, and side effects of the experimental drug called BMS-986205. It will be tested in combination with another cancer medicine called nivolumab plus chemotherapy. This study is for patients who have bladder cancer that has grown into the muscle wall of the bladder. These drugs will be compared to just chemotherapy. BMS-986205 is a small molecule and nivolumab is a human monoclonal antibody. A small molecule such as BMS-986205 is a chemical that is designed to block the function of a protein (called an enzyme) within the body. BMS-986205 is an IDO1 inhibitor. Blocking the IDO pathway may help your immune system to fight abnormal cancer cells in the body. An antibody is a type of protein that your immune system (the body's defense system against harmful particles) uses. Antibodies find and destroy foreign particles (such as bacteria and viruses). Scientists can now make antibodies in the laboratory and produce them for the treatment of many different diseases. Nivolumab is an anti-PD-1 antibody. It works by attaching to and blocking a molecule called PD-1. PD-1 is a protein that is present on different types of cells in your immune system and controls parts of your immune system by shutting it down. Antibodies that block PD-1 can prevent PD-1 from shutting down the immune system. This allows immune cells to recognize and destroy cancer cells. Nivolumab is approved to treat certain cancer types.

Biological study, Phase 3
Any, age 18 years or older

A Study of the Long-Term Safety and Tolerability of Experimental ZYN002 in Children and Adolescents With Fragile X Syndrome (FXS)

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Fragile X Syndrome (FXS) is a genetic condition caused by a mutation in the Fragile X mental retardation 1 (FMR1) gene. This mutation is thought to cause changes to the “endocannabinoid system.” This is a certain pathway in the central nervous system. Disruption in this pathway can lead to FXS symptoms such as: - mild to severe intellectual or developmental disability - speech and language difficulties - behavioral, social and emotional difficulties. Cannabidiol (CBD) is part of the Cannabis/Marijuana plant. However, it does not have the same effects on the brain that recreational marijuana has. It may affect the endocannabinoid pathway. The Drug Product ZYN002 is a pharmaceutically manufactured CBD. It is being developed as a clear gel that can be applied to the skin (called transdermal delivery). This provides consistent, controlled levels of CBD in the blood when it is given once or twice a day. The purpose of this study is to evaluate the long-term safety, tolerability and effectiveness of the experimental drug ZYN002. It will be used in the treatment of symptoms of Fragile X Syndrome (FXS). There are currently no FDA approved medications shown to treat FXS. ZYN002 is an experimental treatment. This means that it is not approved by the FDA and must be tested to see if it is an effective treatment.

Drug study, Phase [/2/,/ /3/]
Any, age 3 to 18 years old

A Study of the Experimental Cytomegalovirus (CMV) Vaccine (HB-101) in Kidney Transplant Patients

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The purpose of this clinical research study is to see if an investigational anti-Cytomegalovirus (CMV) vaccine known as HB-101 is safe to use and helps patients. The main purpose of this study is find out:  How well HB-101 could protect from significant CMV infections.  The side effects of HB-101. CMV is a common infection that can be dangerous when your defense against commonly mild infections is low. For example, this can happen in patients who receive a kidney from a donor. Most people whose immune system is working well with CMV infection have no symptoms. If any, symptoms are mild illness such as fever, sore throat, fatigue, and swollen glands. However if your immune system is weak, CMV can affect your lung, gut, eye or other organs and can create severe damages. This study involves the use of an new investigational anti-CMV vaccine known as HB-101. HB-101 is a vaccine that is believed to prevent CMV infection after kidney transplant by forming antibodies and stimulating your immune cells against CMV. This vaccine doesn’t contain eggs or mercury. HB-101 contains a Genetically Modified Organism (GMO). The vaccine uses genes from two different viruses, but it can’t reproduce any infectious viruses in your body. The HB-101 vaccine is constructed from parts of a LCMV. The parts of the LCMV used is not alive and cannot reproduce or give you an infection. When HB-101 vaccine is introduced inside your body, it tricks your body in thinking it has CMV. It teaches your immune system to make antibodies to protect you against CMV infection. The HB-101 vaccine is not live and can’t spread in your body or outside your body in the environment.

Biological study, Phase 2
Any, age 18 to 99 years old

A Study of Experimental Pembrolizumab (MK-3475) in Combination With Chemotherapy & Endocrine Therapy for Breast Cancer

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The purpose of this study is to: o Test the safety of the study drug, pembrolizumab (MK-3475), with certain types of chemotherapy and endocrine therapy. o See how well the study drug, pembrolizumab, works when combined with chemotherapy and endocrine therapy. o To see if combining the study drug with chemotherapy will have a positive outcome on your surgery, compared to these treatments alone. o To see if combining the study drug with chemotherapy and endocrine therapy keeps your cancer from returning sooner. o To see if combining the study drug with chemotherapy and endocrine therapy helps you live longer. o See how your body handles the study drug. This is a study to test Pembrolizumab for your type of breast cancer. It has not been approved by the FDA to treat breast cancer, but it is approved to treat other types of cancer. The chemotherapies being used in this study (Paclitaxel, Doxorubicin, Epirubicin and Cyclophosphamide) are approved. They are widely used, as a treatment for your type of breast cancer. Endocrine therapy is also approved to treat your type of breast cancer. You will receive endocrine therapy as part of your treatment during the study. The use of these drugs together with pembrolizumab is experimental. “Experimental” means that the study drug is currently being tested. It is not approved by the U.S. FDA

Biological study, Phase 3
Any, age 18 years or older
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