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Studying a New Medicine for Kids and Teens with Generalized Myasthenia Gravis
Generalized myasthenia gravis is a disease that makes muscles weak because the body's defense system attacks the spot where nerves and muscles connect. The purpose of this study is to learn more about a new medicine for kids and teens with this disease. Our researchers want to find out if the medicine is safe, how it works inside the body, and if it helps kids feel better and do everyday things more easily.
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Rare Disease Brain and Memory Children, Infants, Teen Health
Studying Causes of Heart Failure
This purpose of this study is to understand what causes heart failure. Some people with early-stage heart failure might not know they have it because they feel fine, while others may have many or few symptoms. We want to learn why some people get heart failure and how to better classify it for targeted treatments. We are recruiting volunteers with and without heart failure to learn more.
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Studying the Drug Metformin for Older Patients with Heart Failure
This study wants to see if the medicine Metformin XR can help older people with this heart problem. We also want to learn about the side effects and see if it can make their health better.
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New Treatment for ANCA Vasculitis in Kids and Teens
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Rare Disease Arthritis, Lupus, and Autoimmune Disorders Children, Infants, Teen Health
A Study of New Treatment for Damaged Knee Cartilage
This study is testing a treatment called CARTISTEM® for people with knee cartilage damage and osteoarthritis. Researchers want to learn how safe it is and how well it works compared to a standard knee surgery used to treat damaged cartilage. This study is for volunteers with knee osteoarthritis diagnosed by their doctor.
Study Topics
Arthritis, Lupus, and Autoimmune Disorders Bones, Joints, Muscles
Studying a New Medicine for Crohn's Disease
The purpose of this study is to learn more about a new medicine for people with Crohn's disease who have not gotten better with other treatments. Volunteers in the study will get either the new medicine or a look-alike with no medicine in it. Our researchers will check if the new medicine works and is safe.
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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.
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A Study of the Drug ORIC-944 for Patients with Prostate Cancer
The purpose of this study is to learn more about the study drug ORIC-944. We want to see if this study drug is safe and useful to treat patients with prostate cancer that has spread to other areas of the body.
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Studying a New Medicine for Children With JMML
The purpose of this study is to learn more about a medicine called trametinib. Researchers want to see if the medicine used with other cancer treatments, is safe and works well for children and young adults who were recently diagnosed with juvenile myelomonocytic leukemia (JMML). Researchers hope to find better treatment options for children with this rare blood cancer.
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Comparing Treatments for Patients with Newly Diagnosed AML
The purpose of this study is to see how well the study drug, CPX-351 works for treating acute myeloid leukemia. We want to find out if this approach works better than the usual approach. We also want to learn more about the possible side effects of this treatment.
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Treating Neuroblastoma in Young People with Naxitamab
The purpose of this study is to test the safety and effectiveness of adding a new drug called naxitamab to the usual treatment for a type of cancer called neuroblastoma.
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Cancer Cancer: Head, Neck Cancer: Thyroid, Other Glands Children, Infants, Teen Health
Studying Inobrodib for Multiple Myeloma
The purpose of this study is to learn more about a medicine called inobrodib. Our researchers will study this medicine along with two other medicines among people with multiple myeloma. The study will look at how safe this treatment is and how well it works when other treatments have stopped working.
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Studying Cystic Fibrosis Treatments for People without Modulators
The purpose of this study is to collect health information and samples from people who have cystic fibrosis (CF). By doing this, researchers can learn more about CF and find new ways to treat it. We also want to help people with CF understand what it's like to be part of a research study. We are looking for volunteers who have CF and do not take a certain type of medicine called CFTR modulators.
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Studying a Breast Cancer Medicine Given Under the Skin
The purpose of this study is to find out if the breast cancer medicine trastuzumab deruxtecan (T-DXd) is safe when given as a shot under the skin. The medicine is already used through an IV, but our researchers will study a quicker way to give it. We hope this new way will help people with advanced breast cancer.
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Studying Chemo Before Surgery for Abdominal Sarcoma
The purpose of this study is to learn more about giving chemotherapy before surgery for a type of cancer called retroperitoneal sarcoma. We want to see if this helps lower the chance of the cancer coming back. We will compare two groups: one that gets surgery only and one that gets chemotherapy first and then surgery. This will help doctors learn which treatment works better in the future.
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Studying a New Medicine for Muscle Weakness Diseases
The purpose of this study is to see if a new medicine called anifrolumab helps people with muscle weakness diseases called polymyositis and dermatomyositis. The medicine will be given as a shot under the skin, along with regular treatment. The study will check if this medicine helps people feel better and need less of their usual medicine.
Study Topics
Studying the Medicine Olutasidenib for AML After Venetoclax
The purpose of this study is to learn more about the medicine olutasidenib. Researchers want to see if this medicine can help adults with a type of blood cancer called acute myeloid leukemia (AML) that has come back or has not improved after treatment with venetoclax.
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Learning More about Sleep Problems Among Lung Cancer Survivors
The purpose of this study is to learn more about sleep problems among patients who have had lung cancer. We want to better understand how to find and help address sleep problems by asking questions about how sleep affects patients' health and feelings. The goal is to find better ways to help our patients sleep.
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Use of a Bone Marrow Transplant for Patients with Severe Aplastic Anemia
The purpose of this study is to help people with severe aplastic anemia, a condition where the body does not make enough blood cells. We want to see if a bone marrow transplant can be a safe and useful first treatment for patients with this condition, even if they have not tried other treatments before. This could improve their chances of recovery and lead to better health outcomes.
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Preventing Chickenpox Among Children
The purpose of this study is to learn more about a new chickenpox vaccine for young children. Chickenpox, also called varicella, is common in children and can cause fever, itchy rashes, and serious infections. Doctors have safely used chickenpox vaccines in children for many years, but there is currently only one option available. This study will help researchers see how well a new vaccine works compared to the one already in use.
Study Topics
Healthy Volunteers Screening and Prevention Children, Infants, Teen Health