UC Davis Health Clinical Studies

A Study of Experimental Ataluren for Nonsense Mutation Dystrophinopathy (nmDBMD)

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This study is for boys with Duchenne/Becker muscular dystrophy (DMD/BMD). The condition studied in this study is caused by a nonsense mutation in the dystrophin gene. Participants must have been treated with ataluren in a past clinical trial or treatment plan. The main goal of this study is to learn whether experimental ataluren is safe for patients with DMD/BMD. Experimental means that ataluren is not approved by the FDA to be used except in a research study. The FDA has allowed the use of ataluren in this study. We hope to find out if it could one day be made more widely available for doctors to prescribe it for patients with DMD/BMD.

Drug study, Phase 3
Male, age -1 years or older

A Study of Different Presentations of Rapid-Onset Dystonia Parkinsonism and Other Movement Disorders

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You have been asked to participate because you may have symptoms of: - Rapid-Onset Dystonia-Parkinsonism, - Atypical Parkinsonism, - Dystonia, or other movement disorders, specifically AHC (Alternating hemiplegia of childhood) - Cerebral Palsy patients Dystonia is a movement disorder with involuntary muscle contractions. These force certain parts of the body into abnormal, sometimes painful, movements or postures. Dystonia can affect any part of the body including the arms and legs, trunk, neck, eyelids, face, or vocal cords. You may also be asked to take part if you have a family member with Rapid Onset Dystonia-Parkinsonism (RDP) or other movement disorder. The purpose of this study is to find more information about the symptoms of RDP. We want to learn how many persons have it or the gene for it, and about how the symptoms may change over time. You are free to stop your participation at any time. Currently families with RDP have been identified in the United States and Europe.

Other Study
Any, age 2 years or older

A Study of Dose Confirmation and Safety of Experimental Crizanlizumab for Pediatric Sickle Cell Disease

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The purpose of this study is to find out if a drug, previously approved for adults, is safe and effective for children with Sickle Cell Disease (SCD). SCD is a rare blood disorder that causes red blood cells (RBC) to have a “sickle” shape. They are destroyed more quickly than normal cells, so patients with SCD have fewer red blood cells. They also have painful episodes, called Vaso-occlusive Crises (VOC). These episodes are caused by cells in the blood and blood vessels sticking together and slowing down the flow of blood. The name of the experimental treatment is Crizanlizumab (SEG101). Part of determining whether this study treatment is safe and effective for children is to determine how much of the drug should be given (dosage). The participants in the study will be given various amounts based on age and other medical factors as determined by the study doctors. This drug is currently approved by the FDA for use in adults (16 years and older) and has been proven effective is reducing the complications of SCD. This drug is not currently approved for children. Because it is not approved for children, it is called an experimental drug for the purposes of the study. This is the first time that this study treatment is being given to children in a study. In a previous study for adults, the study treatment decreased the number of VOC episodes. The study treatment also resulted in longer periods between episodes of VOC in adults.

Drug study, Phase 2
Any, age Up to 17 years old

Genetic Testing to Determine Therapy For Pediatric Relapsed or Refractory Advanced Solid Tumors

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The main purpose of this study is to learn how well tumors that have specific genetic changes (mutations) respond to drugs that “target” those changes. This combination of a tumor with a mutation and a drug that aims at that mutation is called a “match.” There are two steps for participants in this study: 1. The first step is called screening. The purpose of screening is to perform tests on your tumor tissue to find out what mutations, if any are present in your tumor. To be eligible for screening, you must have a tumor sample already available for testing, or there must be a way to do a biopsy of your tumor that your doctor thinks is safe. The consent form you are reading now is for the screening step of the Pediatric MATCH study. 2. The second step is the investigational treatment part of the study. In this step we will see whether there is an investigational (experimental) drug available through the Pediatric MATCH study that targets the mutations (if any) that are found in your tumor. If so, your study doctor will tell you about the drug and explain the specifics of getting that drug. He or she will explain the potential side effects and benefits of the drug. In some cases, the side effects of these drugs in children might not be known. If you consent and are eligible, you will then receive the drug and we will look to see what its effects are on your tumor.

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

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
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