Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C
NCT00344331
Recruiting
This study will evaluate clinical and laboratory tests that might be useful in determining if an investigational drug can slow the progression of Niemann-Pick Disease, Type C (NPC), a genetic disorder that results in progressive loss of nervous system function. The study will: 1) look for a clinical or biochemical marker that can be used as a measure of response to treatment, and 2) define the rate of progression of biochemical marker abnormalities in a group of NPC patients who will later be in... Read More
Gender:
ALL
Ages:
Between 1 day and 120 years
Trial Updated:
06/13/2025
Locations: National Institutes of Health Clinical Center, Bethesda, Maryland
Conditions: Niemann-Pick Disease, Type C
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Characterization of Patients With Uncommon Presentations and/or Uncommon Diseases Associated With the Cardiovascular System
NCT01143454
Recruiting
Background: - Researchers are interested in studying individuals who have known or suspected metabolic or genetic diseases that put them at a high risk for heart diseases or diseases of their blood vessels. To improve the results of the study, both affected and nonaffected individuals will be asked to provide blood and other samples and will undergo tests to evaluate heart and lung function. Nonaffected individuals will include relatives of affected individuals and healthy nonrelated volunteers... Read More
Gender:
ALL
Ages:
Between 1 year and 100 years
Trial Updated:
06/13/2025
Locations: Suburban Hospital, Bethesda, Maryland +1 locations
Conditions: Cardiomyopathy, Li-Fraumeni Syndrome, Parkinson's Disease, Atherosclerosis, Cardiovascular Capacity
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Natural History Study of Children and Adults With Medullary Thyroid Cancer
NCT01660984
Recruiting
Background: - Medullary thyroid cancer (MTC) is a rare cancer of the thyroid gland. In children and adults, it is often part of a condition called Multiple Endocrine Neoplasia 2 (MEN2). MEN2 is usually caused by a genetic mutation, and it can cause a number of problems in addition to MTC. These problems include adrenal gland tumors, hormone changes, and problems with the bones and other organs. Not much is known about how MTC develops over time, especially in people with MEN2. Researchers want... Read More
Gender:
ALL
Ages:
4 months and above
Trial Updated:
06/13/2025
Locations: National Institutes of Health Clinical Center, Bethesda, Maryland
Conditions: Medullary Thyroid Cancer
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Genome Transplant Dynamics
NCT02423070
Recruiting
Study Description: Heart and lung transplants can save lives, but long-term success is often limited by organ rejection that is hard to detect early. This study is testing a new, non-invasive blood test that looks for small pieces of DNA from the donor organ in the patient s blood. We believe higher levels of this donor DNA may signal early rejection before damage becomes permanent. Hypothesis: We believe that measuring donor-derived DNA in the blood can help detect early signs of rejection a... Read More
Gender:
ALL
Ages:
Between 14 years and 80 years
Trial Updated:
06/13/2025
Locations: MedStar Washington Hospital Center, Washington, District of Columbia +5 locations
Conditions: Thoracic Organ Transplantation
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Effects of Sucralose on Drug Absorption and Metabolism (The SweetMeds Study)
NCT03407079
Recruiting
Background: Artificial sweeteners like sucralose are found in many foods and drinks. Sucralose might affect hormones and cause health changes. Objective: To see if sucralose changes how medicines are absorbed and processed, how hormones are secreted, gut bacteria, and how fat cells are metabolized. Eligibility: People ages 18-60 who: * Are black or Hispanic * Weigh more than 110 pounds * Have a body mass index of 25-40 * Do not have a condition that requires drug treatment Design: Partic... Read More
Gender:
FEMALE
Ages:
Between 18 years and 60 years
Trial Updated:
06/13/2025
Locations: National Institutes of Health Clinical Center, Bethesda, Maryland
Conditions: Healthy Volunteers, Overweight
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Optical Coherence Tomography Angiography in Subjects With Retinal Vascular Disease
NCT04505618
Recruiting
This study will perform a prospective, longitudinal analysis of clinical and imaging findings from normal controls and subjects with retinal vascular disease to better define the diagnostic imaging criteria that signify change in disease stage. This includes disease progression in early stages of disease or disease regression with appropriate standard-of-care treatment.
Gender:
ALL
Ages:
Between 18 years and 99 years
Trial Updated:
06/13/2025
Locations: Wilmer Eye Institute, Baltimore, Maryland
Conditions: Diabetic Retinopathy, Retinal Vein Occlusion, Hypertension,Essential, Retinal Vascular Disorder
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NADPH Oxidase Correction in mRNA-transfected Granulocyte-enriched Cells in Chronic Granulomatous Disease (CGD)
NCT05189925
Recruiting
Background: CGD is caused by a gene mutation. For people with CGD, their cells cannot kill germs well, so they can get frequent or life-threatening infections. Researchers want to see if a new procedure can help a person s cells kill germs for a short time. It uses messenger RNA (mRNA) to deliver correct instructions for the gene mutation to the cells. Objective: To test a procedure in which mRNA is added to a person s blood cells. Eligibility: Males aged 18-75 with CGD with a mutation in t... Read More
Gender:
MALE
Ages:
Between 18 years and 75 years
Trial Updated:
06/13/2025
Locations: National Institutes of Health Clinical Center, Bethesda, Maryland
Conditions: Chronic Granulomatous Disease, Infection
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Comparing Combinations of Targeted Drugs for Advanced Non-Small Cell Lung Cancer That Has EGFR and MET Gene Changes (A Lung-MAP Treatment Trial)
NCT05642572
Recruiting
This phase II Lung-MAP treatment trial test the combination of targeted drugs (capmatinib, osimertinib, and/or ramucirumab) in treating patients with non-small cell lung cancer that may have spread from where it first started to nearby tissue, lymph nodes, or distant parts of the body (advanced) and that has EGFR and MET gene changes. Capmatinib and osimertinib are in a class of medications called kinase inhibitors. They work by blocking the action of the abnormal protein that signals cancer cel... Read More
Gender:
ALL
Ages:
18 years and above
Trial Updated:
06/13/2025
Locations: Cancer Center at Saint Joseph's, Phoenix, Arizona +72 locations
Conditions: Recurrent Lung Non-Small Cell Carcinoma, Stage IV Lung Cancer AJCC v8
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Alternate Doses and Dosing Schedules of Belantamab Mafodotin for Treatment of Triple-Class Refractory Multiple Myeloma
NCT05847569
Recruiting
This phase II trial tests alternate doses and dosing schedules of belantamab mafodotin in treating patients with triple-class multiple myeloma that has come back (after a period of improvement) (recurrent) and/or does not respond to treatment (or that has not responded to previous treatment) (refractory). Belantamab mafodotin is a monoclonal antibody, belantamab, linked to a chemotherapy drug, mafodotin. Belantamab is a form of targeted therapy because it attaches to specific molecules (receptor... Read More
Gender:
ALL
Ages:
18 years and above
Trial Updated:
06/13/2025
Locations: Mayo Clinic in Florida, Jacksonville, Florida
Conditions: Recurrent Multiple Myeloma, Refractory Multiple Myeloma
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Vitaccess Real MG Registry
NCT06064461
Recruiting
Vitaccess Real MG (VRMG) is a patient registry designed to capture longitudinal observational data on myasthenia gravis (MG), its treatment, and impact on symptoms, daily activities, and quality of life (QoL). The duration of the registry is 10 years from launch, and approximately 600 patients will be recruited in the US and Europe with no defined upper limit. The registry will link relevant patient- and healthcare professional (HCP)-reported data with clinical data from medical records. Patien... Read More
Gender:
ALL
Ages:
18 years and above
Trial Updated:
06/13/2025
Locations: HSHS St. Elizabeth's Hospital, O'Fallon, Illinois +6 locations
Conditions: Myasthenia Gravis
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A Follow-up Study to Test Long-term Treatment With Nerandomilast in People With Pulmonary Fibrosis Who Took Part in a Previous Study With Nerandomilast
NCT06238622
Recruiting
This study is open to people with idiopathic pulmonary fibrosis (IPF) or progressive pulmonary fibrosis (PPF). They can only take part if they have completed treatment in a previous study with a medicine called nerandomilast or BI 1015550. The goal of this study is to find out how well people with pulmonary fibrosis tolerate long- term treatment with nerandomilast. The study also tests whether nerandomilast improves lung function and prolongs the time until symptoms get worse, participants need... Read More
Gender:
ALL
Ages:
18 years and above
Trial Updated:
06/13/2025
Locations: University of Alabama at Birmingham, Birmingham, Alabama +371 locations
Conditions: Idiopathic Pulmonary Fibrosis, Progressive Pulmonary Fibrosis
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A Study of Psilocybin for PTSD
NCT06407635
Recruiting
The proposed open-label, controlled study at the Johns Hopkins Center for Psychedelic and Consciousness Research (CPCR) will test the following primary hypotheses in adult patients with chronic PTSD who are currently taking a serotonin reuptake inhibitor: psilocybin therapy will be feasible and safe for participants, significantly remediate PTSD symptoms, and enhance wellbeing and quality of life. In addition, the study will examine whether elements of evidence-based trauma-focused psychotherapy... Read More
Gender:
ALL
Ages:
Between 21 years and 75 years
Trial Updated:
06/13/2025
Locations: Johns Hopkins Center for Psychedelic and Consciousness Research, Baltimore, Maryland
Conditions: Post Traumatic Stress Disorder
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