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Comparing Treatments for Severe Aplastic Anemia in Young People
The purpose of this study is to find out whether immune suppressive therapy or bone marrow transplant is better for treating a disease called severe aplastic anemia in young patients who don't have a matched sibling donor.
Study Topics
Rare Disease Cancer Cancer: Blood Cancer: Children, Teens Children, Infants, Teen Health
Early Chronic Obstructive Pulmonary Disease (COPD) in Younger Patients
Chronic obstructive pulmonary disease (COPD) is a lung disease that causes blocked airflow from the lungs. The purpose of this study is to learn more about COPD in younger people that are at risk.
Study Topics
Studying the Drug SNDX-5613 for Patients with Acute Myeloid Leukemia
The study aims to learn more about the drug SNDX-5613. We want to find the best dose for patients with a blood cancer called acute myeloid leukemia. We also want to see how this drug works when used with two other drugs, daunorubicin and cytarabine.
Study Topics
Studying a Medicine for Babies with Alagille Syndrome
The purpose of this study is to learn more about a medicine called Odevixibat for babies with Alagille Syndrome. We want to see if this medicine can safely help reduce itching in babies with this health condition.
Study Topics
Genetic Disorders Rare Disease Children, Infants, Teen Health
Study of Non-Invasive Therapy for Female Patients with Interstitial Cystitis/Bladder Pain Syndrome
The purpose of this study is to see how well a non-invasive therapy works to treat interstitial cystitis/bladder pain syndrome. The therapy uses a device that sends magnetic waves into the body which may help the body better heal itself. We want to find out if the study therapy can reduce pelvic pain and urinary symptoms.
Study Topics
Studying Blood Particles to Help Treat Cancer
The purpose of this study is to learn more about tiny particles in the blood called extracellular vesicles. By studying this, we hope to find out more about different types of cancer and how to treat them better. We will look at the blood samples from patients with similar cancers to see if we can discover new information. This could help doctors choose the best treatments for cancer patients in the future. We are looking for volunteers with urothelial, prostate, or kidney cancer. The study team will provide more information.
Study Topics
Using Levocarnitine to Protect the Liver During Chemotherapy
The study aims to find out if the study drug, levocarnitine, can reduce severe liver damage caused by chemotherapy in patients with certain cancers.
Study Topics
Kidneys, Liver, Pancreas Cancer Cancer: Leukemia Cancer: Lymphoma
New Test to Diagnosis Mycosis Fungoides
Mycosis Fungoides (MF) is a disease that affects the skin. The purpose of this study is to develop a new test to help diagnose MF.
Study Topics
Study of Bempedoic Acid for Treating High Cholesterol in Children
Heterozygous familial hypercholesterolemia (HeFH) is an inherited genetic disorder. It causes dangerously high cholesterol levels. This study will see if bempedoic acid works at lowering cholesterol in kids with HeFH. Bempedoic has been tested in adults, but it has not been tested in children or young adults yet.
Study Topics
Studying a New Blood Test for Patients with Blood Cancer
The purpose of this study is to create a new blood test for patients with blood cancer. We want to see if this new blood test can help predict which patients will benefit from a type of immunotherapy called CAR T-cell therapy. We are looking for volunteers with blood cancer that are eligible to receive CAR T-cell therapy. The study team will provide more information.
Study Topics
Cancer Cancer: Blood Cancer: Leukemia Cancer: Lymphoma Cancer: Myeloma
Studying Repotrectinib for Treating Solid Tumors in Children and Young Adults
The purpose of this study is to learn more about a new cancer medication called repotrectinib for children and young adults. We want to see if this medication can safely treat patients with a solid tumor that has spread from the original tumor site to the area around it or to other parts of the body. The study team will provide more information.
Study Topics
Learning More About a New Study Drug for Treating Uveitic Macular Edema
The layer at the back of the eye is called the retina. Build?up of fluid in the retina is called uveitic macular edema. The purpose of this study is to find out if the study drug RO7200220 can help control inflammation and remove the fluid build-up in the retina to help your vision.
Study Topics
Learning More About a New Study Drug for Treating Uveitic Macular Edema
The purpose of this study is to test a new mix of drugs for people with a type of blood cancer called multiple myeloma. We want to see if adding a new drug, belantamab mafodotin, to the usual treatment of carfilzomib, lenalidomide, and dexamethasone works better. This study will help doctors find out if this new mix helps patients live longer and feel better.
Study Topics
A Study of the Drug ALPN-303 for Patients with Autoimmune Cytopenias
An autoimmune cytopenia is a condition when your immune system, which usually fights germs, accidentally attacks your own blood cells. This study wants to find out if the study drug ALPN-303 is safe and helpful for treating this condition.
Study Topics
Treating Multiple Myeloma with Study Drug ABBV-383
The purpose of this study is to see how the study drug, ABBV-383, affects patients with multiple myeloma that has returned or been difficult to treat. Some volunteers will get increasing doses of the study drug. We want to see if the increasing doses can reduce a side effect known as cytokine release syndrome.
Study Topics
Study of Quick Start Durvalumab for Treating Stage III Non-Small Cell Lung Cancer
Durvalumab is usually started 30 days or more after radiation treatment. The purpose of this study is to find out if it is possible to start Durvalumab within 14 days after radiation treatment. We want to see how the quick start affects you and your cancer.
Study Topics
Testing the Study Drug Tegavivint for Advanced Liver Cancer
The purpose of this study is to learn more about the study drug tegavivint. We want to see if this study drug is safe and useful to treat patients with liver cancer. Volunteers must be diagnosed with liver cancer that cannot be removed surgery and is worsening. Study team will provide more information.
Study Topics
Testing RINVOQ for the Treatment of Adults with Lupus
The purpose of this study is to find out how well the study drug RINVOQ works for treating adults with systemic lupus erythematosus. We also want to find out how safe the study drug is for patients. RINVOQ is an FDA approved medication for other conditions.
Study Topics
Using Tagraxofusp to Treat Cancer in Young People
The purpose of this study is to see if adding the study drug, Tagraxofusp, to the usual treatment helps people with refractory (difficult to treat) and/or relapsed (cancer that has returned after previous treatment) hematologic malignancy (cancer). We also want to learn more about any side effects of the study drug.
Study Topics
Cancer Cancer: Blood Cancer: Children, Teens Children, Infants, Teen Health