UC Davis Health Clinical Studies

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
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