Natural History and Structural Functional Relationships in Fabry Renal Disease Treatment Outcomes(Changes)in Fabry Renal Disease Study
NCT01581424
Recruiting
The investigators will perform a study with two major components. The first is a natural history study of untreated Fabry patients. This study component will detail kidney microscopic structural changes in Fabry patients before starting enzyme replacement therapy and will correlate these changes with kidney function, including glomerular filtration rate and urinary albumin excretion rate. The investigators will perform studies on samples obtained at baseline, or before enzyme replacement therapy... Read More
Gender:
ALL
Ages:
Between 1 year and 75 years
Trial Updated:
08/21/2025
Locations: University of Minnesota, Minneapolis, Minnesota +1 locations
Conditions: Fabry Disease
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The Physiological Responses and Adaptation of Brown Adipose Tissue to Chronic Treatment With Beta3-Adrenergic Receptor Agonists
NCT03049462
Recruiting
Background: Brown adipose tissue (BAT) is a type of fat in the body. It may prevent weight gain, improve insulin sensitivity, and reduce fatty liver. Researchers want to see if BAT helps the body burn energy. Objective: To learn more about how BAT works to burn energy. Eligibility: People ages 18-40 with a body mass index between 18 and 40 Design: Participants will be screened with: Medical history Physical exam Blood, urine, and heart tests Dietitian interview Participants will have... Read More
Gender:
ALL
Ages:
Between 18 years and 40 years
Trial Updated:
08/21/2025
Locations: National Institutes of Health Clinical Center, Bethesda, Maryland
Conditions: Polycystic Ovary Syndrome
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Resiniferatoxin to Treat Severe Pain Associated With Advanced Cancer
NCT00804154
Recruiting
This study will examine the safety of giving the experimental drug, resiniferatoxin (RTX), to treat severe pain in patients with advanced cancer. RTX is a chemical extracted from a cactus-like plant. It is similar to capsaicin, the active ingredient in hot pepper. RTX has relieved pain and reduced the need for pain medication in several animal experiments. It works by destroying nerves that transmit pain information. People at least 18 years of age with severe pain from advanced cancer at or be... Read More
Gender:
ALL
Ages:
Between 18 years and 99 years
Trial Updated:
08/21/2025
Locations: National Institutes of Health Clinical Center, Bethesda, Maryland
Conditions: Intractable Pain, Palliative Care
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Low-Dose Danazol for the Treatment of Telomere Related Diseases
NCT03312400
Recruiting
Background: DNA is a structure in the body. It contains data about how the body develops and works. Telomeres are found on the end of chromosomes in DNA. Some people with short telomeres or other gene changes can develop diseases of the bone marrow, lung, and liver. Researchers want to see if low doses of the hormone drug danazol can help. Objective: To study the safety and effect of low dose danazol. Eligibility: People ages 3 and older with a telomere disease who have either very short te... Read More
Gender:
ALL
Ages:
Between 3 years and 99 years
Trial Updated:
08/21/2025
Locations: National Institutes of Health Clinical Center, Bethesda, Maryland
Conditions: Telomere Disease
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Compassionate Use of Metreleptin in Previously Treated People With Generalized Lipodystrophy
NCT02262832
Recruiting
Background: - Generalized lipodystrophy can cause high blood fat levels and resistance to insulin. This can lead to health problems including diabetes. Researchers have found that the drug metreleptin improves health in people with this disease. Objective: - To test the safety and effectiveness of metreleptin. Eligibility: * People ages 6 months and older with generalized lipodystrophy who: * have received metreleptin through NIH studies AND * cannot get it through approved or compassionate... Read More
Gender:
ALL
Ages:
Between 6 months and 98 years
Trial Updated:
08/21/2025
Locations: National Institutes of Health Clinical Center, Bethesda, Maryland
Conditions: Lipodystrophy, Diabetes, Hyperlipidemia
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Natural History Study of Monoclonal B Cell Lymphocytosis (MBL), Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma (CLL/SLL), Lymphoplasmacytic Lymphoma (LPL)/Waldenstrom Macroglobulinemia (WM), and Splenic Marginal Zone Lymphoma (SMZL)
NCT00923507
Recruiting
Background The development of new technologies now allow scientists to investigate the molecular basis and clinical manifestations of monoclonal B cell lymphocytosis (MBL), chronic lymphocytic leukemia(CLL)/small lymphocytic lymphoma (SLL), lymphoplasmacytic lymphoma (LPL)/Waldenstrom macroglobulinemia (WM), and splenic marginal zone lymphoma (SMZL). Applying these methods in a natural history study can help identify processes involved in disease progression, and possibly lead to the discovery... Read More
Gender:
ALL
Ages:
Between 18 years and 110 years
Trial Updated:
08/21/2025
Locations: National Institutes of Health Clinical Center, Bethesda, Maryland
Conditions: Waldenstrom Macroglobulinemia, Lymphoplasmacytic Lymphoma, Monoclonal B-Cell Lymphocytosis, Small Lymphocytic Lymphoma, CLL (Chronic Lymphocytic Leukemia)
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A Clinical Study of MK-2214 in People With Early Alzheimer's Disease (MK-2214-004)
NCT07033494
Recruiting
Researchers want to know if the study treatment called MK-2214 works to slow certain changes in the brains of people with Alzheimer's disease (AD). AD is a type of dementia that can cause loss of memory, communication (such as speech), and decision-making skills. It can limit a person's ability to do daily tasks. MK-2214 is a study treatment designed to slow down AD. The goals of the study are to learn: * If MK-2214 slows the spread of tau in the brain compared to placebo. Tau is a protein tha... Read More
Gender:
ALL
Ages:
Between 50 years and 85 years
Trial Updated:
08/21/2025
Locations: JEM Research Institute ( Site 1046), Atlantis, Florida +2 locations
Conditions: Early Alzheimer's Disease
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A Basket Clinical Study to Assess Glycerol Tributyrate in Patients With Mitochondrial Encephalopathy, Lactic Acidosis, Stroke-like Episodes (MELAS) or Leber's Hereditary Optic Neuropathy-Plus (LHON-Plus)
NCT06792500
Recruiting
This is a parallel arm non-randomized dose-escalation, open-label basket exploratory phase 1 clinical trial where Mitochondrial encephalopathy, lactic acidosis, stroke-like episodes (MELAS) and Leber's hereditary optic neuropathy-Plus (LHON-Plus) participants will undergo simultaneous enrollment in two disease-based arms and receive daily oral doses of glycerol tributyrate to assess its safety and potential for efficacy using clinical, biochemical, and molecular evidence. This study will utiliz... Read More
Gender:
ALL
Ages:
Between 18 years and 65 years
Trial Updated:
08/21/2025
Locations: Children's National Hospital, Washington, District of Columbia
Conditions: MELAS Syndrome, Lebers Hereditory Optic Neuropathy With Extra Ocular Symptoms (LHON-Plus)
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A Study to Test Whether Vicadrostat (BI 690517) in Combination With Empagliflozin Helps People With Heart Failure and a Weak Pumping Function of the Left Side of the Heart
NCT06935370
Recruiting
This study is open to adults with chronic heart failure (HF) who have a reduced left ventricular ejection fraction (LVEF) of less than 40%. People can join the study if they have been diagnosed with chronic HF at least 3 months before the study. The purpose of this study is to find out whether a medicine called vicadrostat, in combination with another medicine called empagliflozin, helps people with chronic heart failure. In this study, participants are put into 2 groups randomly. Participants... Read More
Gender:
ALL
Ages:
18 years and above
Trial Updated:
08/21/2025
Locations: Alliance for Multispecialty Research, LLC, Mobile, Alabama +597 locations
Conditions: Heart Failure
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Natural History and Development of Spondyloarthritis
NCT01422694
Recruiting
Background: - Spondyloarthritis (SpA) is a group of bone and joint disorders that may cause back and joint pain and stiffness. In some cases, SpA can lead to abnormal bone growth affecting the joints and spine. Some patients have SpA without ever developing these growths, while others develop them after only a few years. Researchers are interested in studying people with SpA and their relatives to determine which people are more likely to develop more severe conditions. Objectives: - To ident... Read More
Gender:
ALL
Ages:
2 years and above
Trial Updated:
08/21/2025
Locations: National Institutes of Health Clinical Center, Bethesda, Maryland
Conditions: Arthritis, Spondylitis, Ankylosing
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Safety, Reactogenicity, and Immunogenicity Trial of STX-S SARS-CoV-2 Vaccine in Adults
NCT07095231
Recruiting
This phase 1 clinical trial will evaluate the safety, reactogenicity, and immunogenicity of STX-S in adults who have previously received a primary series and at least one booster with an authorized or licensed SARS-CoV-2 parenteral vaccine. The study is designed as a non-randomized, open-label, dose-escalation clinical trial evaluating three dose levels of STX-S. A sample size of 60 participants (20 participants per dose cohort) is anticipated. The primary objective is to evaluate the safety and... Read More
Gender:
ALL
Ages:
Between 18 years and 64 years
Trial Updated:
08/21/2025
Locations: University of Rochester Medical Center - Vaccine Research Unit, Rochester, New York +2 locations
Conditions: COVID-19
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Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID)
NCT01306019
Recruiting
This is a Phase I/II non-randomized clinical trial of ex vivo hematopoietic stem cell (HSC) gene transfer treatment for X-linked severe combined immunodeficiency (XSCID, also known as SCID-X1) using a self-inactivating lentiviral vector incorporating additional features to improve safety and performance. The study will treat 35 patients with XSCID who are between 2 and 50 years of age and who have clinically significant impairment of immunity. Patients will receive a total busulfan dose of appro... Read More
Gender:
MALE
Ages:
Between 2 years and 50 years
Trial Updated:
08/21/2025
Locations: National Institutes of Health Clinical Center, Bethesda, Maryland
Conditions: X-linked Severe Combined Immunodeficiency (XSCID)
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