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Comparing Two Medicines for Essential Thrombocythemia
The purpose of this study is to see if a new medicine called bomedemstat is safe and works well for people with a blood disease called essential thrombocythemia. We will compare bomedemstat to another medicine called hydroxyurea. The goal is to find out which medicine helps more with symptoms like tiredness and high platelet counts.
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Tracking the Long-Term Safety of the Medicine AGAMREE
Duchenne muscular dystrophy (DMD) is a disease that makes muscles get weaker over time. The purpose of this study is to track the long-term safety of a medicine called AGAMREE for this disease. People in the study will have yearly checkups and answer questions about their health. We are looking for volunteers who have DMD and are currently taking AGAMREE.
Study Topics
Genetic Disorders Men's Health Rare Disease Bones, Joints, Muscles Children, Infants, Teen Health
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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Collecting Data on Patients with Generalized Myasthenia Gravis
General Myasthenia Gravis is a rare disease that makes it hard for nerves and muscles to work together. This study collects information about people with this disease in a patient registry. The registry helps scientists learn new things that might help others in the future.
Study Topics
Neurology Disorders Rare Disease Arthritis, Lupus, and Autoimmune Disorders
Studying a New Treatment Combination for Mantle Cell Lymphoma
The purpose of this study is to learn more about a new medicine combination to help patients with a type of blood cancer called mantle cell lymphoma. We want to see if a new medicine called sonrotoclax combined with the medicine zanubrutinib works better than zanubrutinib alone. Volunteers in the study will be randomly assigned to get either the new medicine or a placebo along with zanubrutinib.
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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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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.
Study Topics
Rare Disease Brain and Memory Children, Infants, Teen Health
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.
Study Topics
Studying a New Treatment for Early-Stage ALS
Amyotrophic Lateral Sclerosis (ALS) is a disease that slowly makes it harder for your brain to talk to your muscles, causing weakness and trouble moving. The purpose of this study is to learn more about a new treatment for this disease called VHB937. We want to see if this treatment can safely slow down the disease among patients with early-stage ALS.
Study Topics
Movement Disorder Neurology Disorders Rare Disease Bones, Joints, Muscles
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
Using a Small Device to Track Muscle Changes in ALS
Amyotrophic Lateral Sclerosis (ALS) is a disease that slowly makes muscles weaker and harder to control, making it tough to walk, talk, or breathe. The purpose of this study is to see how a small device called the mScan can measure muscle changes in people with ALS. The device uses a tiny, painless electrical signal to check muscles. Our researchers hope this will help track ALS over time and see how well treatments work.
Study Topics
Movement Disorder Neurology Disorders Rare Disease Bones, Joints, Muscles
Studying the Medicine Bitopertin for Sun Sensitivity
Erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP) are rare disorders that are passed down in families and cause a chemical to build up in the blood. This makes being in sunlight hurt their skin and can also affect their liver. The purpose of this study is to find out if a new medicine called bitopertin can help people with EPP or XLP stay in the sun longer without pain.
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Studying a Rare Blood Condition Over Time
The purpose of this study is to learn more about a rare blood problem called plasminogen deficiency. Researchers will collect blood, urine, and health information to find out who gets symptoms and how to help them. The goal is to improve care for people with this condition.
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Studying the Drug MT-7117 for Patients with a Rare Blood Disorder
The purpose of this study is to learn more about the study drug MT-7117. We want to see if this study drug is useful for patients with a rare blood disorder called Erythropoietic Protoporphyria. We also want to see if this study drug is tolerable to take.
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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
Studying a New Medicine for Myelofibrosis
The purpose of this study is to learn more about a new medicine called Canakinumab. We want to see if it helps people with a rare bone marrow cancer called myelofibrosis. The medicine will be used along with another medicine that patients are already taking. The goal is to find out if this new medicine is safe and if it helps people feel better.
Study Topics