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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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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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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Assessment of Remote Approaches for Identification of Autonomic Dysfunction Among Survivors of Leukemia and Lymphoma
NCT06747910
Recruiting
This study seeks to determine if diagnosing cardiac autonomic dysfunction (AD) can be done remotely with the same accuracy as in-person testing. If so, the identification of AD could happen sooner, facilitating remote studies of the condition and potentially reducing the risk of illness. Childhood cancer survivors, particularly survivors of acute lymphoblastic leukemia (ALL) and Hodgkins's lymphoma (HL), appear to be at increased risk for AD. Primary Objectives: * To determine the sensitivity... Read More
Gender:
ALL
Ages:
18 years and above
Trial Updated:
08/21/2025
Locations: St. Jude Children's Research Hospital, Memphis, Tennessee
Conditions: Childhood Cancer
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Baltimore Longitudinal Study of Aging
NCT00233272
Recruiting
Background: - The Baltimore Longitudinal Study of Aging (BLSA) is a clinical research program on human aging that began in 1958. Volunteers of different ages join the study when they are healthy, and have follow-up visits for life. Visits last for multiple days. Participants are evaluated for many physical elements as well as for brain function. Physical tests are given. Information on mood, personality, and social aspects of life is also collected. This program has contributed more than any ot... Read More
Gender:
ALL
Ages:
Between 20 years and 120 years
Trial Updated:
08/21/2025
Locations: National Institute of Aging, Clinical Research Unit, Baltimore, Maryland
Conditions: Aging
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Clinical and Genetic Studies of Li-Fraumeni Syndrome
NCT01443468
Recruiting
Background: - Li-Fraumeni syndrome (LFS) is a genetic condition that increases the risk for some types of cancer. LFS may lead to cancer of the bone or connective tissue, breast, and brain. It may also increase the risk for certain types of leukemia and other cancers. The only known cause of LFS is a change (called a mutation ) in a gene known as TP53. However, not all people with LFS have a TP53 mutation. Researchers want to study other possible genetic causes of LFS, and factors that may incr... Read More
Gender:
ALL
Ages:
All
Trial Updated:
08/21/2025
Locations: National Cancer Institute - Shady Grove, Bethesda, Maryland +1 locations
Conditions: Li-Fraumeni Syndrome, Neoplasms, Tp53 Mutations
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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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Stone and Laser Therapies Post-Market Study (SALT)
NCT06982235
Recruiting
The objective of the SALT Study is to obtain post-market safety and device performance data for Boston Scientific's Stone and BPH study devices used during a diagnostic and/or therapeutic urinary tract procedure (e.g., transurethral or percutaneous access routes) or benign prostatic hyperplasia (BPH) treatment.
Gender:
ALL
Ages:
All
Trial Updated:
08/21/2025
Locations: University of Miami Hospital, Miami, Florida
Conditions: Benign Prostate Hypertrophy(BPH), Renal Calculi, Ureteral Stones, Kidney Stones, Calculi, Urinary, Urinary Tract Procedure
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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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Avexitide for Treatment of Post-Bariatric Hypoglycemia
NCT06747468
Recruiting
AVX-001 (LUCIDITY) is a Phase 3 study to evaluate avexitide compared to placebo in participants with post bariatric hypoglycemia (PBH) related to Roux-en-Y gastric bypass (RYGB). The study will assess avexitide compared to placebo for safety and efficacy, measured by reduction of hypoglycemic events. The study includes a Screening period with a Run-in period (of up to 6- and 3-weeks, respectively); a randomized, double-blind, placebo-controlled study treatment period of 16 weeks; and a two-part... Read More
Gender:
ALL
Ages:
18 years and above
Trial Updated:
08/21/2025
Locations: Stanford Health Care - Endocrinology Clinic, Stanford, California +20 locations
Conditions: Post Bariatric Hypoglycemia
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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 Study to Compare the Efficacy and Safety of Golcadomide in Combination With Rituximab (Golca + R) vs Investigator's Choice in Participants With Relapsed/Refractory Follicular Lymphoma (GOLSEEK-4)
NCT06911502
Recruiting
The study is designed as a multicenter, randomized, open label Phase 3 study to compare the efficacy and safety of golcadomide in combination with rituximab vs investigator's choice in participants with relapsed/refractory follicular lymphoma who have received at least one line of prior systemic therapy.
Gender:
ALL
Ages:
18 years and above
Trial Updated:
08/21/2025
Locations: Local Institution - 0225, Birmingham, Alabama +153 locations
Conditions: Follicular Lymphoma
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