4D-710 in Adult Patients With Cystic Fibrosis
NCT05248230
Recruiting
This is a Phase 1/2 multicenter, open-label, single dose trial of 4D-710 investigational gene therapy in adults with cystic fibrosis.
Gender:
ALL
Ages:
18 years and above
Trial Updated:
08/22/2025
Locations: University of Alabama Child Health Research Unit, Birmingham, Alabama +18 locations
Conditions: Cystic Fibrosis Lung
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Cerebral Palsy and the Study of Brain Activity During Motor Tasks
NCT01829724
Recruiting
Background: - Two ways to study the brain while people are moving are near-infrared spectroscopy (NIRS) and electroencephalography (EEG). NIRS uses light to look at blood flow in the brain when it is active. EEG records electrical activity in the brain. Both have been used safely for many years, even in very young children. NIRS or EEG can be used while a person is moving to show which parts of the brain are the most active. Researchers want to use NIRS and EEG to study brain activity during mo... Read More
Gender:
ALL
Ages:
Between 5 years and 100 years
Trial Updated:
08/22/2025
Locations: National Institutes of Health Clinical Center, Bethesda, Maryland
Conditions: Cerebral Palsy, Children
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A Real-World Study to Gain Clinical Insights Into Roche Ophthalmology Products
NCT05476926
Recruiting
The VOYAGER study is a primary data collection, non-interventional, prospective, multinational, multicenter study. It is designed to collect real-world, long-term data to explore long-term effectiveness, safety, clinical insights, treatment patterns, and factors driving the treatment decisions among patients being treated with specified Roche ophthalmology products in approved retinal indications (Faricimab for neovascular age-related macular degeneration \[nAMD\], diabetic macular edema \[DME\]... Read More
Gender:
ALL
Ages:
18 years and above
Trial Updated:
08/22/2025
Locations: Retinal Research Institute, LLC, Phoenix, Arizona +308 locations
Conditions: Neovascular Age-related Macular Degeneration, Diabetic Macular Edema, Retinal Vein Occlusion, Branch Retinal Vein Occlusion, Central Retinal Vein Occlusion, Hemi-retinal Vein Occlusion
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Natural History Study of Progressive Multifocal Leukoencephalopathy (PML)
NCT01730131
Recruiting
Background: - Progressive multifocal leukoencephalopathy (PML) is a severe viral infection of the brain. It is caused by JC virus. Many people have this virus in their bodies all their life, but it is usually kept in check by their immune system. If the immune system does not work right because of a disease or medication, the virus becomes active and can damage cells in the brain. Not much is known about PML or how it affects the immune system. Researchers want to study people with PML to bette... Read More
Gender:
ALL
Ages:
Between 2 years and 120 years
Trial Updated:
08/22/2025
Locations: National Institutes of Health Clinical Center, Bethesda, Maryland
Conditions: Progressive Multifocal Leukoencephalopathy
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Evaluating the Addition of the Immunotherapy Drug Atezolizumab to Standard Chemotherapy Treatment for Advanced or Metastatic Neuroendocrine Carcinomas That Originate Outside the Lung
NCT05058651
Recruiting
This phase II/III trial compares the effect of immunotherapy with atezolizumab in combination with standard chemotherapy with a platinum drug (cisplatin or carboplatin) and etoposide versus standard therapy alone for the treatment of poorly differentiated extrapulmonary (originated outside the lung) neuroendocrine cancer that may have spread from where it first started to nearby tissue, lymph nodes, or distant parts of the body (advanced) or that has spread from where it first started (primary s... Read More
Gender:
ALL
Ages:
18 years and above
Trial Updated:
08/22/2025
Locations: Alaska Women's Cancer Care, Anchorage, Alaska +221 locations
Conditions: Advanced Extrapulmonary Neuroendocrine Carcinoma, Metastatic Extrapulmonary Neuroendocrine Carcinoma, Recurrent Extrapulmonary Neuroendocrine Carcinoma, Unresectable Extrapulmonary Neuroendocrine Carcinoma
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Development of Magnetic Resonance Spectroscopic Imaging Techniques for Imaging Metabolites in Human Brain and Muscle
NCT01266577
Recruiting
Background: - Magnetic resonance imaging (MRI) is a widely used scanning technique to obtain images of the human body and evaluate activity in the brain. A particular MRI method called magnetic resonance spectroscopy (MRS) can be used to study brain chemistry as well, which may help researchers who are studying new treatments for psychiatric illnesses. Researchers are interested in improving current MRI and MRS techniques, as well as developing new MRI and MRS techniques to view and measure bra... Read More
Gender:
ALL
Ages:
Between 18 years and 65 years
Trial Updated:
08/22/2025
Locations: National Institutes of Health Clinical Center, Bethesda, Maryland
Conditions: Brain Mapping, Healthy Volunteer
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A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
NCT06138639
Recruiting
This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single intravenous (IV) infusion of SGT-003 in participants with Duchenne muscular dystrophy. There will be 5 cohorts in this study. Cohort 1 will include participants 4 to \< 7 years of age. Cohort 2 will include participants 7 to \< 12 years of age. Cohort 3 will include participants 0 to \< 4 years of age. Cohort 4 will include participants 12 to \< 18 years of age. Cohort 5 will... Read More
Gender:
MALE
Ages:
Between 0 years and 17 years
Trial Updated:
08/22/2025
Locations: Arkansas Children's Hospital, Little Rock, Arkansas +9 locations
Conditions: Duchenne Muscular Dystrophy
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Apheresis of Patients With Immunodeficiency
NCT01212055
Recruiting
Background: - Gene therapy is being investigated as a possible treatment for individuals with immunodeficiency diseases or other conditions that make it difficult to fight off infection. Gene therapy avoids problems with donor identification and possible rejection of bone marrow transplant by using the patient s own modified blood cells to help treat the disease. Researchers are interested in collecting stem cells from the blood of individuals with immunodeficiency diseases in order to use the... Read More
Gender:
ALL
Ages:
Between 18 years and 40 years
Trial Updated:
08/22/2025
Locations: National Institutes of Health Clinical Center, Bethesda, Maryland
Conditions: LAD-1, DOCK8, GATA2 Deficancy
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Testing the Addition of Radiation Therapy to the Usual Immune Therapy Treatment (Atezolizumab) for Extensive Stage Small Cell Lung Cancer, The RAPTOR Trial
NCT04402788
Recruiting
This phase II/III trial compares the effect of adding radiation therapy to the usual maintenance therapy with atezolizumab versus atezolizumab alone in patients who have already received atezolizumab plus chemotherapy for the treatment of small cell lung cancer that has spread outside of the lung or to other parts of the body (extensive stage). Immunotherapy with monoclonal antibodies, such as atezolizumab, may help the body's immune system attack the cancer, and may interfere with the ability o... Read More
Gender:
ALL
Ages:
18 years and above
Trial Updated:
08/22/2025
Locations: University of Alabama at Birmingham Cancer Center, Birmingham, Alabama +410 locations
Conditions: Extensive Stage Lung Small Cell Carcinoma
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Belatacept in Heart Transplantation
NCT06478017
Recruiting
This is a phase 2, prospective, multi-center, open-label clinical trial. Sixty-six (66) primary heart transplant recipients will be randomized (1:2) to receive either standard-of-care, tacrolimus-based immunosuppression, or a belatacept-based regimen with gradual tacrolimus withdrawal over 9-months post-transplant. Both study arms will receive CellCept® (mycophenolate mofetil- MMF) or Myfortic® (mycophenolate sodium). Corticosteroids will be continued throughout the study in the belatacept arm.... Read More
Gender:
ALL
Ages:
Between 18 years and 71 years
Trial Updated:
08/22/2025
Locations: Cedars Sinai Heart Institute/ Cedars Sinai Medical (Site # 71146), Los Angeles, California +3 locations
Conditions: Heart Transplant
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A Study to Evaluate the Long-Term Safety and Efficacy of Povorcitinib in Participants With Moderate to Severe Hidradenitis Suppurativa
NCT06212999
Recruiting
The purpose of this study is to evaluate long-term safety and efficacy of povorcitinib in participants with moderate to severe hidradenitis suppurativa who completed the 54 weeks of study treatment within the originating parent Phase 3 studies (INCB 54707-301 \[NCT05620823\] or INCB 54707-302 \[NCT05620836\]).
Gender:
ALL
Ages:
18 years and above
Trial Updated:
08/22/2025
Locations: Investigative Site US303, Phoenix, Arizona +171 locations
Conditions: Hidradenitis Suppurativa (HS)
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Clinical and Scientific Assessment of Pain and Painful Disorders
NCT02707029
Recruiting
Background: Researchers want to better understand pain by studying people with and without different kinds of pain. To do this, researchers will expose people to pleasant and unpleasant sensations. They will ask them questions about their pain. Researchers also want to see if these people are eligible for other research studies at the National Center for Complementary and Integrative Health. Objectives: To study the experience of pain. Also to find people eligible to join other NIH studies.... Read More
Gender:
ALL
Ages:
Between 12 years and 120 years
Trial Updated:
08/22/2025
Locations: National Institutes of Health Clinical Center, Bethesda, Maryland
Conditions: Normal Physiology, Pain
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