UC Davis Health Clinical Studies

Thiamine Responsive Disorders (TRD) Among Infants in Lao PDR

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Thiamine is a vitamin that is essential for healthy development of young children. In Laos, some young children are at risk of thiamine deficiency. Thiamine deficiency causes beriberi. Some children may have milder forms of illnesses, and those are more difficult to diagnose. But if those children receive thiamine, it helps to them to get healthy. This research study will describe which illnesses are associated with thiamine deficiency. The goal is to help determine which children who need thiamine and to ensure that they receive thiamine as part of their medical treatment. Mothers are also invited to take part in this study as it will examine risk factors that can lead to thiamine deficiency in young children.

Drug study
Any, age -1 years or older

A Study of Experimental Combination Treatment for Relapsed, Refractory or Progressive Neuroblastoma

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With the hope of finding a new therapy for fighting the type of cancer that you have, you will receive the following drugs on this study: · Irinotecan, temozolomide, dinutuximab, and sargramostim (GM-CSF) or · Irinotecan, temozolomide, dinutuximab, and sargramostim (GM-CSF) with eflornithine (DFMO) The drugs irinotecan and temozolomide have been used to treat adults and children with different types of cancer. This drug combination has also been used in patients with relapsed/refractory cancers. This includes patients with relapsed/refractory NBL. This drug combination was well tolerated. This means it did not result in too many side effects for patients. Dinutuximab works differently than most standard chemotherapy drugs. Dinutuximab is a monoclonal antibody. Monoclonal antibodies are proteins made in the lab, designed to attach to specific targets on cancer cells. When dinutuximab attaches to NBL cells, the body’s immune system is stimulated to attack and kill the NBL cells. Dinutuximab is a kind of cancer therapy called immunotherapy. Unlike chemotherapy and radiation it targets cancer cells without destroying nearby healthy cells. Dinutuximab has been given with irinotecan and temozolomide in patients with relapsed/refractory NBL. This combination was well tolerated. GM-CSF is a substance that is similar to one made by the body. Under normal circumstances, the body makes small amounts of GM-CSF. This helps it produce normal infection-fighting white blood cells. It is now possible to make GM-CSF outside of the body and give humans much higher amounts than their own bodies make. In the lab and in animals, GM-CSF increases the anti-cancer effect of monoclonal antibodies like dinutuximab. Eflornithine (DFMO) is an investigational drug that is not yet approved by the FDA for use in cancer patients. DFMO has been approved by the FDA for treatment of an infection called trypanosomiasis. DFMO blocks the production of chemicals called polyamines that are important in the growth of cancer cells. This drug has been used in adults with cancer. It has also been tested both alone and in combination with chemotherapy in children with cancer. It has been tested in children with neuroblastoma. There is some evidence that polyamines may play an important role in childhood tumors. This is especially true in tumors that depend on the abnormal activity of MYC genes (like neuroblastoma). In the laboratory, DFMO and chemotherapy are able to reduce the growth of neuroblastoma tumors. DFMO may also help the body’s immune system kill NBL cells, and may help immunotherapies like dinutuximab work better. We want to find out if giving DFMO along with dinutuximab, GM-CSF, irinotecan, and temozolomide is tolerated. We also want to see how effective the drug combination is against relapsed or refractory NBL. The overall goal of this study is to find out what effects: - DFMO given with - irinotecan, - temozolomide, and - dinutuximab has on children and young adults with relapsed or refractory NBL. In addition to the treatment goal, we would like to answer some biology research questions that might benefit future patients. We will also ask you about any pain you are having during therapy. These studies are described later in this form.

Biological study, Phase 2
Any, age 1 years or older

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

A Study of Experimental Blinatumomab for Localized B Cell Lymphoblastic Lymphoma (B-LLy)

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You are being asked to take part in this research study because you have been diagnosed with Standard Risk-High B-cell Lymphoblastic Leukemia (SR-High B-ALL). Taking part in this study is voluntary. You may choose not to be in this study. If you decide not to be in this study, you will not be penalized and you will not lose any benefits to which you are entitled. You will still receive medical care. The overall goal of this study is to: • Compare the effects, good and/or bad, of adding blinatumomab to standard chemotherapy in subjects with SR-High B-ALL to find out which is better. The treatment involves cancer fighting medicine called chemotherapy. You have already been receiving chemotherapy for the past month. The treatment may also include the investigational medicine blinatumomab. The treatment on this study takes about 2-2 ¼ years. It is divided into 5-7 phases, depending on the treatment plan you receive. All people who receive cancer treatment are at risk of having side effects. In addition to killing tumor cells, cancer chemotherapy can damage normal tissue and produce side effects. In this study you will get 1 of 2 treatment plans. The 2 treatment plans are called Arms C and D. The 2 treatment plans are the same except for whether or not you will get the investigational medicine blinatumomab. The rest of the treatment that is given is standard therapy for people with SR-High B-ALL. On this study, Arm C is standard therapy and Arm D uses the investigational medicine blinatumomab in addition to standard therapy.

Drug study, Phase 3
Any, age -1 to 31 years old

A Study of Renal Anhydramnios Fetal Therapy (repeated infusions of replacement amniotic fluid into the womb)

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This research is being done to test repeated infusions of replacement amniotic fluid into the womb. We hope this can reliably rescue the lung function of fetuses that do not make urine, and as a result, have no amniotic fluid. This condition occurs when the fetus has a severe birth defect called congenital bilateral renal agenesis (CoBRA). It can also be caused by fetal renal failure (FRF). We hope to discover whether this fetal intervention can make early pregnancy renal anhydramnios, called EPRA, reliably survivable after birth. We are also doing this study to better understand what happens to EPRA fetuses in the womb that do not receive therapy. During EPRA, the fetus does not have well-developed or functional kidneys and does not make urine. Fetal urine makes up what we commonly call amniotic fluid. This fluid surrounds the fetus and performs many vital functions. The most important function of amniotic fluid is to allow development of the lungs. In normal fetal life, the fetus gets oxygen and releases carbon dioxide through the placenta. Because gas exchange occurs in the placenta, the lungs do not need to function in the womb. They do need to go through a complex developmental process to be ready to work after birth. This developmental process involves the formation of tiny sacs called alveoli in the lungs. They allow the baby to absorb oxygen from the air it breathes in and expel carbon dioxide from the air it breathes out once born. A critical part of the formation of these alveoli is the fluid that fills the lungs during fetal life. The fluid made by the fetus’s lungs causes the alveoli to stretch and grow. If there is no amniotic fluid in the womb, the lung fluid easily escapes from the lungs and cannot perform its vital function of making the alveoli grow. Normally, the amniotic fluid creates enough pressure in the womb to keep the lung fluid in the lungs. Since the amniotic fluid and the lung fluid connect when the fetus opens its mouth and windpipe, the pressure of the amniotic fluid pushes on the lung fluid. This pressure on the lung fluid must be present for the alveoli to develop correctly. Even if the baby’s lungs work well enough after birth for the baby to survive, the baby will still have no kidneys and need dialysis. A pediatric surgeon will need to place a dialysis catheter into the baby’s abdomen. This will allow fluid and toxins to be removed from the baby as a replacement for kidney function and urination. This process is commonly known as peritoneal dialysis. The baby may also need special assistance with feeding and breathing. All of this requires advanced neonatal care in a neonatal intensive care unit (NICU). It is likely that surviving babies will need to be in the NICU for several months before being able to go home. There are frequent challenges with peritoneal dialysis. This includes infection and dialysis catheter malfunction. Not all babies survive because of complications that can arise from peritoneal dialysis. Additionally, surviving babies will eventually need a kidney transplant to survive long-term. Current survival for infants with complete renal failure who survive to receive a kidney transplant is about 70-80%. Organs can come from either living or deceased donors. They require the baby to take medicine every day to prevent his or her body from rejecting the donated kidney. These babies also have problems with the urinary system that normally drains urine from the kidneys namely the bladder and the urethra. This will become an issue after kidney transplant because the urine will need a place to drain and be expelled. A urologic surgeon will need to be involved to help manage this. He or she may need to make a new bladder out of intestine or do other reconstructive surgery.

Procedure study
Female, age 18 to 60 years old

Study to Evaluate the Effectiveness and Safety of Experimental PANZYGA in Children With Chronic Immune Thrombocytopenia (ITP)

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PANZYGA is a solution for intravenous (IV) dosing (injection of the drug with a needle into a vein). It contains human antibodies also called immunoglobulin (Ig), which are protective blood proteins. PANZYGA is licensed in the US for use in adult and pediatric patients with Primary Immune Deficiency. It is also approved for adults with Immune Thrombocytopenia (ITP). For children, the study drug is an experimental drug. An experimental drug is one that has not been approved for use in this population but has been approved for this study. ITP is a blood disorder in which the immune system produces antibodies that attack and destroy the body's own blood platelets. These platelets play a major part in the blood clotting process. In a healthy person 150,000 to 400,000 platelets per microliter blood is normal. In an ITP patient this may fall below 5,000. The mechanism of action of immunoglobulins in ITP is not fully understood. However, intravenously administered immunoglobulins (IVIg) have become a first-line treatment option for selected patients with ITP. The dose to be given is also generally accepted. The purpose of this study is to investigate the effectiveness and safety of PANZYGA. It will be tested in pediatric patients with chronic ITP. We hope to obtain marketing authorization for the treatment of ITP in children.

Biological study, Phase 4
Any, age 1 to 18 years old

Extension Study to Test Effectiveness and Safety of Experimental PF-04965842 for Adolescents With Atopic Dermatitis

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You are being asked to take part in this research study because you have moderate to severe atopic dermatitis (AD). This is also known as atopic eczema. The purpose of this research study is to learn about the long-term safety and effectiveness of the study drug, PF-04965842. This study is for participants who previously participated in PF-04965842 AD Phase 3 trials. PF-04965842 is an experimental drug. This means it has not been approved for sale by the FDA. PF-04965842 is a tablet, which is to be taken by mouth and swallowed. PF-04965842 blocks an enzyme called Janus kinase. Janus kinase acts like a switch for the cells of the immune system (the body’s defense against infection and inflammation). By turning off this switch, the cells of the immune system are expected to produce fewer chemicals believed to cause AD. However, there is no guarantee that participating in this study will help your AD.

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

Study of Experimental JAK1 Inhibitor With Medicated Topical Therapy in Adolescents With Atopic Dermatitis

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You are being asked to take part in this research study because you have atopic dermatitis (AD). This is also known as atopic eczema, which may be of moderate to severe intensity as evaluated by the study doctor. The purpose of this research study is to test the efficacy and safety of two doses of the study drug (PF-04965842). They will be compared to a placebo when taken together with medicated and non-medicated therapy applied to the skin. Researchers will compare the results of taking the higher dose, lower dose, and placebo to see if there are any differences, when taken with the background medication. PF04965842 is an experimental drug. An experimental drug is one that is not approved by the FDA for use in this country. A placebo looks like the study drug but does not contain any active ingredients. Researchers use a placebo to see if the study drug works better or is safer than not taking anything. PF-04965842 is a blocker of an enzyme called Janus kinase (JAK). JAK acts like a switch for the cells of the immune system (the body’s defense against infection and inflammation). By turning off this switch, the cells of the immune system should produce fewer chemicals believed to cause AD. However, there is no guarantee that participating in this study will help your AD.

Drug study, Phase 3
Any, age 12 to 17 years old
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