UC Davis Health Clinical Studies

A Study of the Drug Activity, Safety, and Tolerability of Experimental Ceftazidime-avibactam for Infections in Newborns & Infants

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You are being asked to take part in this study because your infant has or is suspected to have a bacterial infection. The infection requires treatment with IV antibiotics. The purpose of this study is to learn about the effects of the study drug combination of ceftazidime (CAZ) and avibactam (AVI). We hope to confirm the best dose for treating bacterial infections in infants and neonates. Ceftazidime (CAZ) is an antibiotic which is approved for use in infants and neonates. However, avibactam (AVI) is not approved for use in infants and neonates. The study drug, CAZ-AVI, is an experimental drug because it is not approved for use in neonates and infants from birth to less than 3 months of age.

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

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

Experimental Iobenguane I-131 or Crizotinib and Standard Therapy For Younger Patients With Newly-Diagnosed High-Risk Neuroblastoma

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In this study, researchers want to find out if we can improve the treatment for subjects with high risk Neuroblastoma (NBL). We will study the addition of experimental drug I-MIBG or the experimental drug Crizotinib to standard therapy. We also want to find out if we can reduce the number of stem cell transplants from two to one. I-MIBG is an experimental anticancer drug. It has not been approved by the FDA for use in treating high-risk NBL. I-MIBG has been used to treat NBL that did not respond to therapy or NBL that has come back. I-MIBG has been shown to be well tolerated in children with cancer. I-MIBG can only be given at certain hospitals and you may need to travel to another hospital if you choose to participate in this study. Some patients in the study will receive only one stem cell transplant instead of two back-to-back stem cell transplants. Subjects who have only one stem cell transplant will get two chemotherapy drugs called busulfan and melphalan (BuMel). These are given instead of the drugs usually used for back-to-back stem cell transplants. BuMel is an accepted transplant chemotherapy regimen in Europe and other parts of the world. It is not part of current Children's Oncology Group (COG) recommended therapy. Crizotinib is an experimental anticancer drug. It has not been approved by the FDA for use in treating high-risk NBL. Crizotinib has been approved by the FDA for use in treating adults with certain types of lung cancer that has spread. Crizotinib has been well-tolerated in children and adults with cancer. Crizotinib will only be used in subjects whose tumor shows changes in a gene called ALK. Changes in the ALK gene are only found in 10-15% of children with high-risk NBL. The overall goals of this study are to: • Compare the effects, good and/or bad, of the experimental drug I-MIBG added to current COG recommended therapy. • Compare the effects, good and/or bad, of the experimental drug crizotinib added to current COG recommended therapy. Another goal of this study is to: • Evaluate the effects, good and/or bad, of using the experimental drug I-MIBG in the setting of single cell transplant with BuMel conditioning during Consolidation therapy. These effects, good and/or bad, will be studied but will not be directly compared to current COG recommended therapy.

Biological study, Phase 3
Any, age -1 to 30 years old

A Study of Experimental Accelerated vs Standard Chemotherapy for Germ Cell Tumors (formed from reproductive cells)

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Standard treatment for advanced germ cell tumors includes three chemotherapy drugs. This is called BEP (Bleomycin, Etoposide, and cisPlatin). BEP is given with a drug called pegfilgrastim or filgrastim. This makes white blood cells grow. These drugs are given over the course of 3 weeks. This study will compare the standard chemotherapy regimen with an accelerated chemotherapy regimen. It will use the same drugs to see if the accelerated chemotherapy regimen is beneficial but not more toxic than the standard chemotherapy regimen. The accelerated chemotherapy is experimental. This method works by giving the dose of chemotherapy drugs over a shorter length of time. Instead of the standard 3 week-cycle, patients would receive their treatment in a 2 week-cycle. The overall goal of this study is to determine whether accelerated BEP will be effective and well-tolerated for patients with advanced GCTs. We don’t yet know if accelerated treatment is helpful in advanced GCTs and we are hoping this study will answer that question.

Drug study, Phase 3
Any, age 11 to 45 years old

The PROSpect Study: A Pediatric Study to Test the Best Breathing Position for Children When They Are Sick

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This study is being done to learn the best way to help children breathe when they are sick. When children’s lungs are very sick they are on a machine to help them breathe. There are different types of breathing machines that can be used. The two types are usual or high frequency: 1) A usual breathing machine imitates a person’s normal breathing. 2) A high frequency breathing machine delivers fast and shallow breaths. There are also different positions a child can be in while on a breathing machine: 1) Lying on their back with a slight change in position every two hours. 2) Lying on their stomach for at least 16 hours each day with a slight change in position every 2 hours. When needed for medical care, your child will be turned on their back, and then returned to lying on his or her stomach. At UC Davis, we use both types of breathing machines and place children in both positions. The main purpose of this study is to find out if one approach is better than others in reducing the number of days a child spends on a breathing machine. The length of time your child spends on the breathing machine may be shorter or longer from taking part in this study.

Other study
Any, age -1 to 18 years old

A Study of an After-School Program to Teach Food Literacy in Food, Agriculture, Natural resources, and Human (FANH) sciences.

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UC Davis is doing a research study. We are asking you to allow your child to be a part of this research study because they are in the age group we would like to help and are a student at a local school. We would like to learn more about whether a new program helps children learn about nutrition, gardening, and being active. This project will focus on low-income communities. It will expand on existing after-school programs. Adolescents from these communities have higher high school dropout rates. Extracurricular activities, including after-school programs may help. They can improve graduation rates and increase college enrollment. This project plans to educate high school kids in food, agriculture, natural resources, and human (FANH) sciences. We will also motivate them to graduate high school, attend college, and major in FANH sciences. The project will empower adolescents to learn more and be excited about FANH sciences.

Behavioral study
Any, age 9 to 18 years old

Online Developmental Screening Study

Have you had a new baby? Learn about your baby's development!

Researchers at the UC Davis MIND Institute on the UC Davis Medical Center campus in Sacramento are conducting an online study tracking the development of infants from 6 to 36 months of age. The goal of the study is to help parents understand their child’s development and gain access to resources when needed. This study tests new online methods of tracking developmental milestones in babies. The UC Davis MIND Institute is a collaborative international research center, committed to the awareness, understanding, prevention and care of neurodevelopmental disorders.

Longitudinal screening study
Any, age 6 months

A Long-Term Safety and Tolerability Study of Experimental Givinostat in Duchenne Muscular Dystrophy

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This study will test the long-term safety and effectiveness of the experimental medicine Givinostat. It will be given to patients with Duchenne Muscular Dystrophy (DMD). This long-term study is for patients who have already participated in a study with Givinostat. It provides the opportunity for them to continue treatment. This study is for patients with Duchenne Muscular Dystrophy currently taking experimental Givinostat.

Drug study, Phase [2, 3]
Male, age 7 years or older

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 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
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