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This first-in-human study will evaluate the safety, tolerability, pharmacokinetics, and antitumor activity of TORL-1-23 in patients with advanced cancer.

The primary purpose of this study is to record outcomes and patient characteristics in the Cancer Center’s and BMT databases for patients who are undergoing an allogeneic (donor) hematopoietic stem cell transplant. The data will be analyzed for transplant “milestones” such as time to blood count recovery (engraftment) and how patients are doing at 3 months and 6 months after the transplant. Participation in this study will not alter treatment or medical care. All information for this study will be collected from medical records.

This is a new type 1 diabetes onset study for ages 12-35 years old. We are looking at JAK inhibitor drugs to see if they can preserve beta cell function.

This study is for people who feel very hungry all the time, have trouble controlling eating (hyperphagia) and have Prader-Willi Syndrome (PWS). ARD-101 is being studied to see if it can help the body release certain gut hormones that may help reduce excessive hunger and food-seeking behaviors in people with PWS. The investigational treatment is a tablet taken by mouth and swallowed whole. The study will continue for up to 20 weeks (about 5 months).

People planning to undergo a bone marrow transplant and are at risk for developing problems with the lungs related to this planned therapy. The types and seriousness of lung problems that may develop after transplant can be very different and currently, we don’t exactly know what risk factors influence who develops them or how they may respond to therapy. Also, we do not know what the best test is to monitor lung function after transplant, especially in children and young adults. The purpose of this study is to help investigators learn more about lung problems after bone marrow transplant including what is the best method for diagnosing lung problems and following how well the lungs are working. In this study, clinical information, laboratory results and imaging findings will be collected from medical records to assist researchers in learning more about lung complications after bone marrow transplant.

We are doing this study to learn about how we can best educate families about things that affect the health of children including eating together and cooking. If you agree to be in this study, you, your child, and your family will be randomly (by chance) put into one of three groups (Group 1, Group 2 or Group 3). Each group will receive a different type of education on childhood health.

This is a global, multicenter, prospective, observational, longitudinal registry conducted to characterize the natural history and real-world clinical management of patients diagnosed with AHP. This protocol will not recommend the use of any specific treatments, visits, or procedures. No medication is provided as part of registry participation.

We are studying the relationship between cerebrovascular health and balance control associated with aging. Cerebrovascular function will be assessed with an ultrasound of cerebral blood flow and EKG done while participants exercise. We will also do an EEG with balance testing. Participants will complete two sessions, each 1.5 -2.5 hours and 2.5-3 hours in duration, 1 to 14 days apart. We will compare the results of younger, middle-aged and older adult participants.

We are doing this research to collect a wide range of samples, clinical information, and measurements that will be used for future research into ALS and related neurological diseases. Participants will be asked to complete 7 in-person study visits and monthly remote self-assessment activities. Access to a personal device (computer and/or smartphone or tablet) that is connected to the internet is needed to complete the monthly remote activities.

The purpose of this study is to determine the safety and effectiveness of allogeneic hematopoietic cell transplant in persons with an inherited metabolic disorder or osteopetrosis and if it is effective in reducing or slowing the symptoms associated with the genetic error. The study uses a chemotherapy conditioning regimen that prepares the body to accept the donor hematopoietic cells.

This study is intended to find the safest dose of a new combination of drugs (ALX148 and T-DXd) and to start to determine how effective it is at treating advanced or metastatic breast cancer. This study is an addition to the ongoing ISPY study program.

The objective of this study is to describe how activation of distinct pathways in and around the subthalamic nucleus (STN) and internal segment of the globus pallidus (GPi) correlate to changes in sleep outcomes in movement disorders patients after deep brain stimulation (DBS) surgery targeting these structures.

Through this research, the study staff hopes to better understand how DBS works and to define the optimal site in the brain for DBS treatment for Parkinson’s Disease. You will be asked to come for one study visit where you will perform some physical and mental tasks while on and temporarily off your medications and DBS treatment. Participation in this research study will take 7-8 hours.

This study is for people who have essential tremor and will be receiving the Boston Scientific Vercise Genus deep brain stimulation (DBS) system. We are studying the possibility that a kind of deep brain stimulation called "coordinated reset" stimulation (CR-DBS) will be more effective than current DBS treatment for essential tremor. We expect that participants will be in this research study for about 9 to 11 months.

This study will examine the brain activation associated with the transition between movements in Parkinson’s disease, and if these changes are related to the development of problems with moving (like freezing of gait) and thinking. We will look at brain activity associated with the vestibular (inner ear) and cortical (surface of the brain) systems. People with Parkinson’s disease (especially people with freezing of gait) sometimes have difficulty initiating changes in movement types, such as going from standing to walking, turning, or switching between slow and fast repetitive movements. The difficulty in movement transitions may be associated with an overactive vestibular system (inner ear) or cortical system. In each experiment in this study, we expect about 20-25 participants with Parkinson’s disease and freezing of gait, 20-25 participants with Parkinson’s without freezing of gait, and 20-25 control participants

We are conducting this research to try to find a better way of treating Parkinson's disease, specifically postural instability and gait disturbances (PIGD) by looking at certain brain characteristics using 3T magnetic resonance imaging (MRI.) We think we could discover how to make DBS more effective for such things as postural gait instability and other symptoms of PD. Participants will be asked to come to our research location, walk on a treadmill, and have a 3T MRI.

We hope to identify patterns in the brain that may be related to symptoms of PD, such as problems with walking. We are also testing different stimulation settings to see if they might be more effective than the settings that are currently used for treatment. This research may aid in our understanding of PD and how to treat it more effectively with DBS.

Anxiety and stress are significant problems for cancer survivors. The purpose of this study is to learn what effect a 14-day course of kava can have on anxiety and stress in cancer survivors, and about the side effects of kava for cancer survivors.

This phase II trial studies how well combination chemotherapy works in treating patients (≤ 30 years old) with newly diagnosed stage II-IV diffuse anaplastic Wilms tumors (DAWT) or favorable histology Wilms tumors (FHWT) that have come back (relapsed).This trial may help doctors find out what effects, good and/or bad, regimen UH-3 (vincristine, doxorubicin, cyclophosphamide, carboplatin, etoposide, and irinotecan) has on patients with newly diagnosed DAWT and standard risk relapsed FHWT (those treated with only 2 drugs for the initial WT)and regimen ICE/Cyclo/Topo (ifosfamide, carboplatin, etoposide, cyclophosphamide, and topotecan) has on patients with high and very high risk relapsed FHWT (those treated with 3 or more drugs for the initial WT).

Currently, there are no approved treatment options for pediatric subjects with proteinuric kidney conditions. The study will look at the safety, efficacy, and pharmacokinetic (PK) trial in children ≥1 to <18 years treated for up to 108 weeks with the drug sparsentan.

This study will look at an investigational study drug, called olorofim, to determine how safe the study drug is, how well it is tolerated and whether it is effective compared to AmBisome® (a standard of care treatment) to treat invasive fungal disease (IFD). We expect that you will be in this research study for up to 18 weeks or just over 4 months.

The purpose of this study is to learn about the safety and efficacy of PC945 when given in combination with the antifungal therapy that is normally given for this condition, also known as the standard of care (SoC). This study will also assess how PC945 is processed in the body (e.g., distributed, transformed, and removed) by measuring the levels of PC945 in your blood and lungs; this is called pharmacokinetics (PK).

This study is part of our ongoing program of research to understand how the brain and behavior are impacted by cannabis use. We are particularly interested in whether the use of medical cannabis products in adulthood has impacts on learning skills, other aspects of cognition such as memory, attention, and behavioral control, and whether the brain circuits that control these functions change within four months of treatment initiation. Individuals between the ages of 30 and 75 years will be studied at the start of treatment with the plan to retest participants after four months.

The purpose of this study is to learn more about an investigational drug called ifinatamab deruxtecan (I-DXd; DS-7300. It is being studied to see if it is safe, and if cancer improves while taking it. I-DXd is a type of drug called an antibody drug conjugate (ADC). ADCs are made to attach to tumor cells to deliver chemotherapy directly to tumor cells while sparing healthy cells.

Stem cell transplants (sometimes referred to as a bone marrow transplants) have been done for over 40 years but research continues to further refine the method to reduce side effects without affecting transplant success. The purpose of this study is to improve on transplant outcomes while reducing the potential side effects based on what has been learned from previous transplant studies using a reduced intensity preparative regimen. Information collected during this study (transplant outcomes and side effects) will be compared with the outcomes of the previous reduced intensity conditioning transplant study that enrolled more than 300 patients since 2002.

In this study, participants will complete two to three appointments within two weeks. During these appointments, they will complete interviews and questionnaires about their life experiences and mental health. An EEG test will also be conducted while they complete computerized tasks. We are recruiting two groups of participants for this study. One group will include individuals who experience hallucinations, delusions, paranoia, or a psychosis spectrum disorder (i.e., schizophrenia), and the other group will be individuals who do not have a diagnosis or family history of schizophrenia, bipolar disorder, or autism spectrum disorder.

The purpose of this research is to test how well the investigational study drug, quizartinib, works in patients with AML without a FLT3-ITD mutation who have not received any prior treatment for the disease. The study will test how it works when taken with standard chemotherapy and then taken alone to prevent the disease from coming back.

The purpose of this study to examine the effects of cigarette smoking and drinking alcohol on the formation of DNA damage in the mouth. The overall goal is to identify DNA damage that may be important to the development of head and neck cancers.

People with type 2 diabetes have a higher risk for heart disease than people without diabetes. We are doing this research to learn more about which diabetes medications are the best for lowering the risk of heart and kidney disease for people with type 2 diabetes. People who are in the study will be randomly (by chance) assigned to take one of two types of diabetes medications that have been approved by the FDA. Your usual care provider will continue to manage your diabetes care.

The I-SPY2 study uses 10 years of results to help your doctor understand more about your tumor and how to classify it better. This means your doctor will have more information and might be able to offer you a new treatment that could work better than the usual treatments. We need better treatments and better ways to identify which patients will benefit most from particular treatments.