Cancer in Inherited Bone Marrow Failure Syndromes
NCT00027274
Recruiting
Background: A prospective cohort of Inherited Bone Marrow Failure Syndrome (IBMFS) will provide new information regarding cancer rates and types in these disorders. Pathogenic variant(s) in IBMFS genes are relevant to carcinogenesis in sporadic cancers. Patients with IBMFS who develop cancer differ in their genetic and/or environmental features from patients with IBMFS who do not develop cancer. These cancer-prone families are well suited for cancer screening and prevention trials targeting... Read More
Gender:
ALL
Ages:
Between 1 day and 100 years
Trial Updated:
08/22/2025
Locations: National Institutes of Health Clinical Center, Bethesda, Maryland +1 locations
Conditions: Diamond Blackfan Anemia, Dyskeratosis Congenita, Fanconi Anemia, Shwachman Diamond Syndrome, Inherited Bone Marrow Failure Syndrome, Aplastic Anemia
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Testing the Effects of Novel Therapeutics for Newly Diagnosed, Untreated Patients With High-Risk Acute Myeloid Leukemia (A MyeloMATCH Treatment Trial)
NCT05554406
Recruiting
This phase II MyeloMATCH treatment trial tests whether the standard approach of cytarabine and daunorubicin in comparison to the following experimental regimens works to shrink cancer in patients with high risk acute myeloid leukemia (AML): 1) daunorubicin and cytarabine liposome alone; 2) cytarabine and daunorubicin with venetoclax; 3) azacitidine and venetoclax; 4) daunorubicin and cytarabine liposome and venetoclax. "High-risk" refers to traits that have been known to make the AML harder to t... Read More
Gender:
ALL
Ages:
Between 18 years and 59 years
Trial Updated:
08/22/2025
Locations: University of Alabama at Birmingham Cancer Center, Birmingham, Alabama +182 locations
Conditions: Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic/Myeloproliferative Neoplasm, Acute Myeloid Leukemia Post Cytotoxic Therapy, Acute Myeloid Leukemia, Myelodysplasia-Related
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Send-In Sample Collection to Achieve Genetic and Immunologic Characterization of Primary Immunodeficiencies
NCT03610802
Recruiting
Background: The immune system helps the body fight infections. Primary immunodeficiency disorders (PIDs) are diseases that make it easier for people to get sick. Many PIDs are inherited. This means parents can pass them on to their children. Knowing what causes a person s PID is important to decide what treatment to give them. Objective: To test samples from people with a PID or people related to someone with a PID to find out what causes PIDs. Eligibility: People ages 99 or younger who hav... Read More
Gender:
ALL
Ages:
Between 1 day and 99 years
Trial Updated:
08/22/2025
Locations: National Institutes of Health Clinical Center, Bethesda, Maryland +2 locations
Conditions: Primary Immunodeficiency
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A Study of a Virtual Education-Based Transition Intervention to Improve Transition Readiness in Adolescent and Young Adults With Congenital Adrenal Hyperplasia
NCT05663320
Recruiting
Background: Children with congenital adrenal hyperplasia (CAH) can survive well into adulthood with proper treatment. But the change from pediatric to adult care can be challenging. Many people with CAH need extra support as they learn to take control of their own health care needs. Researchers have studied how people respond to different types of patient education. Now researchers want to find out if virtual education, via computer, is a good way to teach people how to manage CAH as they becom... Read More
Gender:
ALL
Ages:
Between 16 years and 22 years
Trial Updated:
08/22/2025
Locations: National Institutes of Health Clinical Center, Bethesda, Maryland
Conditions: Congenital Adrenal Hyperplasia
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Familial Mediterranean Fever and Related Disorders: Genetics and Disease Characteristics
NCT00001373
Recruiting
This study is designed to explore the genetics and pathophysiology of diseases presenting with intermittent fever, including familial Mediterranean fever, TRAPS, hyper-IgD syndrome, and related diseases. The following individuals may be eligible for this natural history study: 1) patients with known or suspected familial Mediterranean fever, TRAPS, hyper-IgD syndrome or related disorders; 2) relatives of these patients; 3) healthy, normal volunteers 7 years of age or older. Patients will under... Read More
Gender:
ALL
Ages:
Between 2 months and 115 years
Trial Updated:
08/22/2025
Locations: Childrens National Medical Center, Washington, District of Columbia +4 locations
Conditions: Familial Mediterranean Fever (FMF), Autoinflammation, Periodic Fever, Fever, Genetic Diseases, ROSAH, ALPK1
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Tumor-agnostic Precision Immuno-oncology and Somatic Targeting Rational for You (TAPISTRY) Platform Study
NCT04589845
Recruiting
TAPISTRY is a Phase II, global, multicenter, open-label, multi-cohort study designed to evaluate the safety and efficacy of targeted therapies or immunotherapy as single agents or in rational, specified combinations in participants with unresectable, locally advanced or metastatic solid tumors determined to harbor specific oncogenic genomic alterations or who are tumor mutational burden (TMB)-high as identified by a validated next-generation sequencing (NGS) assay. Participants with solid tumors... Read More
Gender:
ALL
Ages:
All
Trial Updated:
08/22/2025
Locations: Southern Cancer Center, Daphne, Alabama +162 locations
Conditions: Solid Tumors
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Observational Study of Cardiac Arrhythmias During Treatment With BTK Inhibitors or Venetoclax
NCT05724121
Recruiting
Background: Bruton s tyrosine kinase inhibitors (BTKi) are used to treat a form of leukemia. But taking BTKi can also increase a person s risk of developing an abnormal heart rhythm. This can cause sudden death. In this natural history study, researchers want to learn how BTKi affects the heart. Objective: To identify and monitor the effects of BTKi on the heart. Eligibility: People aged 18 and older currently receiving or planning to receive BTKi or venetoclax. Design: Participants who h... Read More
Gender:
ALL
Ages:
Between 18 years and 110 years
Trial Updated:
08/22/2025
Locations: National Institutes of Health Clinical Center, Bethesda, Maryland
Conditions: Chronic Lymphocytic Leukemia (CLL), Waldenstr(SqrRoot)(Delta)m s Macroglobulinemia, Mantle Cell Lymphoma, Sudden Cardiac Death, Cardiac Arrhythmias, Hematologic Malignancies
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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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Evaluating New Radiation Techniques for Cardiovascular Imaging
NCT01621594
Recruiting
Title: Evaluating New Radiation Techniques for Cardiovascular Imaging Background: - Imaging studies such as computed tomography (CT) scans involve the use of radiation to create the pictures. Heart and blood vessel CT scans can cause high radiation exposure. Different methods of creating CT pictures have been developed to reduce the radiation dose. Researchers want to see how effective these new methods are in producing accurate CT scans. Objectives: - To study new ways of taking pictures of... Read More
Gender:
ALL
Ages:
Between 18 years and 100 years
Trial Updated:
08/22/2025
Locations: National Institutes of Health Clinical Center, Bethesda, Maryland
Conditions: Coronary Disease
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Natural History of Stroke: Cause and Development
NCT00009243
Recruiting
The purpose of this study is to learn more about stroke and obtain information that may serve as the basis for future investigations. It will 1) establish a registry of patients with cerebrovascular disease (stroke); 2) characterize the natural history of acute stroke and transient ischemic attacks (TIA)-an interruption of blood flow to the brain that causes stroke symptoms for a short period of time); and 3) evaluate the data to generate ideas for future studies. Patients 18 years of age or ol... Read More
Gender:
ALL
Ages:
Between 18 years and 120 years
Trial Updated:
08/22/2025
Locations: Medstar Washington Hospital Center, Washington, District of Columbia +2 locations
Conditions: Brain Disease, Ischemic Attack, Transient, Cerebrovascular Accident, Cerebrovascular Disorder, Vascular Diseases
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Targeted Therapy Directed by Genetic Testing in Treating Patients With Locally Advanced or Advanced Solid Tumors, The ComboMATCH Screening Trial
NCT05564377
Recruiting
This ComboMATCH patient screening trial is the gateway to a coordinated set of clinical trials to study cancer treatment directed by genetic testing. Patients with solid tumors that have spread to nearby tissue or lymph nodes (locally advanced) or have spread to other places in the body (advanced) and have progressed on at least one line of standard systemic therapy or have no standard treatment that has been shown to prolong overall survival may be candidates for these trials. Genetic tests loo... Read More
Gender:
ALL
Ages:
All
Trial Updated:
08/22/2025
Locations: University of Alabama at Birmingham Cancer Center, Birmingham, Alabama +454 locations
Conditions: Advanced Malignant Solid Neoplasm, Locally Advanced Malignant Solid Neoplasm, Metastatic Malignant Solid Neoplasm, Unresectable Malignant Solid Neoplasm, Recurrent Endometrial Carcinoma, Recurrent Fallopian Tube Carcinoma, Recurrent Ovarian Carcinoma, Recurrent Primary Peritoneal Carcinoma, Anatomic Stage III Breast Cancer AJCC v8, Anatomic Stage IV Breast Cancer AJCC v8, Metastatic HER2-Negative Breast Carcinoma, Unresectable HER2-Negative Breast Carcinoma, Malignant Female Reproductive System Neoplasm, Recurrent Malignant Female Reproductive System Neoplasm, Recurrent Malignant Solid Neoplasm
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A Study to Evaluate the Efficacy, Safety, Pharmacokinetics (PK), and Pharmacodynamics (PD) of Satralizumab in Participants With Anti-N-methyl-D-aspartic Acid Receptor (NMDAR) or Anti-leucine-rich Glioma-inactivated 1 (LGI1) Encephalitis
NCT05503264
Recruiting
The purpose of this study is to assess the efficacy, safety, PK, and PD of satralizumab in participants with NMDAR and LGI1 encephalitis.
Gender:
ALL
Ages:
12 years and above
Trial Updated:
08/22/2025
Locations: University of Alabama at Birmingham, Birmingham, Alabama +82 locations
Conditions: NMDAR Autoimmune Encephalitis, LGI1 Autoimmune Encephalitis
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