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Learning More About Using ABBV-383 to Treat Amyloidosis
The purpose of this study is to find out how safe and effective the study drug, ABBV-383, is in treating people with with amyloidosis.
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Studying a New Treatment for Virus After Transplant
Epstein-Barr Virus-Associated Post-Transplant Lymphoproliferative Disease (EBV/PTLD) is a virus that can happen after receiving a solid organ transplant or allogenic cell transplant. The purpose of this study is to learn more about a new treatment called tabelecleucel. We want to see how well this treatment works for patients with this virus who did not get better with a treatment called rituximab or rituximab plus chemotherapy.
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Comparing Two Study Drugs for Treatment of Vasculitis
Eosinophilic Granulomatosis with Polyangiitis is a disease that makes small blood vessels swell up in people with asthma. We want to see how two different study drugs, depemokimab and mepolizumab, work to treat this disease. We also want to learn about the possible side effects of each study drug.
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New Treatment for ANCA Vasculitis in Kids and Teens
Active ANCA-associated Vasculitis is a disease where the body's immune system accidentally attacks its own small to medium-sized blood vessels. This makes the blood vessels swollen and damaged. The purpose of this study is to learn more about a treatment to help kids and teens with this disease. We want to see if the treatment called avacopan is safe and helpful.
Study Topics
Rare Disease Arthritis, Lupus, and Autoimmune Disorders Children, Infants, Teen Health
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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Learning More about Eye Movement among ALS Patients
The purpose of this study is to learn how ALS, a disease that affects nerve cells, changes the way people move their eyes. What the researchers learn from this study could help make better tools for people with ALS. We also hope to help doctors track the disease more easily and learn more about how ALS develops. The researchers plan to share what they find so it can help people living with ALS, their caregivers, and other scientists.
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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.
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Rare Disease Cancer Cancer: Blood Cancer: Children, Teens Children, Infants, Teen Health
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
Studying the Medicine Apitegromab for Babies with Muscle Weakness
Spinal muscular atrophy is a disease that makes muscles weak and hard to move. The purpose of this study is to test a new medicine called apitegromab. We want to see if it is safe and helpful for babies under 2 years old who still have trouble moving even though they already got another treatment called survival motor neuron therapy.
Study Topics
Genetic Disorders Movement Disorder Neurology Disorders Rare Disease Bones, Joints, Muscles Children, Infants, Teen Health
Finding Muscle Changes in Myotonic Dystrophy Patients
Myotonic dystrophy (DM) is a disease that causes muscles to weaken and waste away. The purpose of this study is to find the best ways to measure muscle weakness and changes in patients with DM using MRI scans. Understanding these changes will help plan future studies to track the disease and test new treatments. We are seeking healthy volunteers as well as volunteers who have been diagnosed with myotonic dystrophy.
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