Diagnosis and Treatment of Patients With Inborn Errors of Metabolism
NCT00369421
Recruiting
Researchers intend on diagnosing and treating certain inborn errors of metabolism. By doing this researchers hope to expand their knowledge about these disorders and provide access to patients of interest for research, teaching, and clinical experience. Patients participating in this study will be examined and treated on an out patient basis, if practical. However, patients requiring specialized tests or treatments will be admitted to the NIH Clinical Center as necessary. Researchers will use o... Read More
Gender:
ALL
Ages:
Between 1 month and 115 years
Trial Updated:
08/22/2025
Locations: National Institutes of Health Clinical Center, Bethesda, Maryland
Conditions: Arterial Calcification Due to Deficiency of CD73
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A Phase Ⅲ Clinical Study of HLX22 in Combination With Trastuzumab and Chemotherapy for the Treatment of Gastroesophageal Junction and Gastric Cancer
NCT06532006
Recruiting
This is a double-blind, randomized, multiregion, comparative phase Ⅲ clinical study designed to evaluate the efficacy and safety of HLX22 in combination with trastuzumab and chemotherapy as first-line treatment in patients with HER2-positive locally advanced/metastatic adenocarcinoma of the gastric and/or gastroesophageal junction (G/GEJ).Eligible subjects will be randomized to the two groups based on a 1:1 ratio. Enrolled subjects shall be treated with the study drug until the loss of clinical... Read More
Gender:
ALL
Ages:
18 years and above
Trial Updated:
08/22/2025
Locations: Los Angeles Cancer Network, Anaheim, California +87 locations
Conditions: Gastroesophageal-junction Cancer, Monoclonal Antibody, Gastric Cancer, HER2-positive Gastric Cancer
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Dinutuximab With Chemotherapy, Surgery and Stem Cell Transplantation for the Treatment of Children With Newly Diagnosed High Risk Neuroblastoma
NCT06172296
Recruiting
This phase III trial tests how well the addition of dinutuximab to Induction chemotherapy along with standard of care surgical resection of the primary tumor, radiation, stem cell transplantation, and immunotherapy works for treating children with newly diagnosed high-risk neuroblastoma. Dinutuximab is a monoclonal antibody that binds to a molecule called GD2, which is found on the surface of neuroblastoma cells, but is not present on many healthy or normal cells in the body. When dinutuximab bi... Read More
Gender:
ALL
Ages:
30 years and below
Trial Updated:
08/22/2025
Locations: Children's Hospital of Alabama, Birmingham, Alabama +162 locations
Conditions: Neuroblastoma, Ganglioneuroblastoma, Nodular
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A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
NCT05861986
Recruiting
This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered as an early intervention in pediatric participants with spinal muscular atrophy (SMA) and 2 SMN2 copies who have previously received onasemnogene abeparvovec. Participants are children \< 2 years of age genetically diagnosed with SMA.
Gender:
ALL
Ages:
Between 3 months and 24 months
Trial Updated:
08/22/2025
Locations: University of Arkansas for Medical Sciences, Little Rock, Arkansas +15 locations
Conditions: Muscular Atrophy, Spinal
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Studies of Brain Function and Course of Illness in Pediatric Bipolar Disorder
NCT00025935
Recruiting
This study seeks to learn more about the symptoms of severe mood dysregulation in children and adolescents ages 7-17. Children and adolescents with severe mood dysregulation (SMD) display chronic anger, sadness, or irritability, as well as hyperarousal (such as insomnia, distractibility, hyperactivity) and extreme responses to frustration (such as frequent, severe temper tantrums). Researchers will describe the moods and behaviors of children with these symptoms and use specialized testing and b... Read More
Gender:
ALL
Ages:
Between 7 years and 60 years
Trial Updated:
08/22/2025
Locations: National Institutes of Health Clinical Center, Bethesda, Maryland
Conditions: Mood Disorder
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A Study to Evaluate the Long-Term Efficacy and Safety of Mirikizumab in Participants With Moderately to Severely Active Ulcerative Colitis (LUCENT 3)
NCT03519945
Recruiting
This study is designed to evaluate the long-term efficacy and safety of mirikizumab in participants with moderately to severely active ulcerative colitis (UC). The study will last up to 3 years. Participants who complete the 3-year study may continue to receive mirikizumab until it is (outside of this study) in their country or until they meet other discontinuation criteria.
Gender:
ALL
Ages:
Between 18 years and 80 years
Trial Updated:
08/22/2025
Locations: Digestive Health Specialists, Dothan, Alabama +361 locations
Conditions: Ulcerative Colitis
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A Randomized Controlled Trial to Test the Effects Oxytocin and Vibration Have on Heat Pain Threshold After UV-B Burn
NCT06651476
Recruiting
This study aims to answer the question: Does oxytocin increase the pain threshold on thermal heat pain in the presence of vibration on an area of skin exposed to a mild sunburn?
Gender:
ALL
Ages:
Between 18 years and 55 years
Trial Updated:
08/22/2025
Locations: Atrium Health Wake Forest Baptist Medical Center, Winston-Salem, North Carolina
Conditions: Sunburn
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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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Focal Therapy With Stereotactic Body Radiation Therapy (SBRT) for Patients With a Single Prostate Tumor
NCT05616650
Recruiting
Background: The current standard treatment of prostate cancer is either surgery or radiation. Typically, this includes either the removal or radiation of the whole prostate gland. Many people now seek out focal therapy options to decrease the side effects of treatment. Until now, several forms of physical destruction with heat (thermal ablation), cold (cryotherapy), sound waves (HIFU), laser (FLA), and electrical energy (IRE). A new type of radiation (SBRT) may be an effective way to cure men o... Read More
Gender:
MALE
Ages:
Between 18 years and 120 years
Trial Updated:
08/22/2025
Locations: National Institutes of Health Clinical Center, Bethesda, Maryland
Conditions: Prostatic Neoplasms, Prostate Cancer, Prostate Adenocarcinoma
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RNA-Lipid Particle (RNA-LP) Vaccines for Recurrent Adult Glioblastoma (GBM)
NCT06389591
Recruiting
This is a Phase I study to demonstrate the manufacturing feasibility and safety, and to determine the maximum tolerated dose (MTD) of RNA-LP vaccines in adult patients with recurrent glioblastoma.
Gender:
ALL
Ages:
Between 18 years and 80 years
Trial Updated:
08/22/2025
Locations: UF Health, Gainesville, Florida
Conditions: Recurrent Glioblastoma
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Collection of Bone Marrow Aspirate From Normal Volunteers
NCT03949647
Recruiting
Background: Bone marrow is the soft material found inside most large bones of the body. Bone marrow produces red blood cells, white blood cells, and platelets that are released into the blood stream. Inside the marrow, these cells start off as young, immature cells called progenitor cells. Researchers want to study these cells in healthy people. Objective: To understand how progenitor cells change with age. Eligibility: Healthy people ages 18 and older Design: Participants will be screene... Read More
Gender:
ALL
Ages:
Between 18 years and 120 years
Trial Updated:
08/22/2025
Locations: National Institute of Aging, Clinical Research Unit, Baltimore, Maryland
Conditions: Normal Aging
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Personalized Environment and Genes Study
NCT00341237
Recruiting
Despite the overwhelming focus on genetic and genomic causes of human disease over the past two decades, it has been estimated that genetics is currently known to explain only 20% and 40% of the etiology of common disease. Thus, it is becoming increasingly apparent that human disease is a consequence of both genetic susceptibility and environmental exposures. Importantly, while individuals cannot change their genetic composition, we do have the ability both personally and as a society, to influe... Read More
Gender:
ALL
Ages:
Between 18 years and 120 years
Trial Updated:
08/22/2025
Locations: NIEHS Clinical Research Unit (CRU), Research Triangle Park, North Carolina
Conditions: Diabetes, Heart Disease, Asthma
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