Falls Church, Virginia Clinical Trials

Cancer in Inherited Bone Marrow Failure Syndromes

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 …

F For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR)

Inflammatory and Infectious Diseases of the Nervous System

Background Inflammation is how the body reacts to infection or injury. Infections or inflammation in the brain and nerves can be serious. There aren t always good tests to detect this. Researchers want to learn more about how diseases affect the brain and nerves to develop better tests and treatments. …

F For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR)

A Natural History Study Seeks to Understand the Clinical, Genomic, Pharmacological, Laboratory, and Dietary Determinates of Pyrimidine and Purine Metabolism Disorders

Background Pyrimidine and purine metabolism disorders (DPPMs) affect how the body metabolizes chemicals called pyrimidines and purines. DPPMs can cause dysfunctions throughout the body, especially in the brain, blood, kidneys, and immune system. People with DPPMs might have no symptoms, mild symptoms, or they may have severe, chronic symptoms, that …

P Peter Stacpole, MD

North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)

The North American Mitochondrial Disease Consortium (NAMDC) maintains a patient contact registry and tissue biorepository for patients with mitochondrial disorders.

F For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR)

Studies of Disorders With Increased Susceptibility to Fungal Infections

Background Researchers are interested in studying disorders that make individuals more susceptible to fungal infections, specifically infections with the Candida yeast. These disorders are often related to problems with the immune system and may have genetic factors, which suggests that researchers should study not only the individual with the disorder, …

F For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR)

Human Movement Database

This study will collect information on the different ways people control limb and body movements. This information will be used to develop a database on normal movements and adaptive movements of people who have diseases that affect the way they move. The database will serve as a tool to improve …

F For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR)

A Natural History Study of Metabolic Sizing in Health and Disease

Background Scientists have long used simple measures (such as height and weight) to estimate how much a person s body uses food (calories) as energy, as commonly called the metabolic rate. But metabolism varies among people with similar body sizes. Scientists now believe the old formulas for estimating metabolic rates …

A Prospective Registry Study to Assess Real-world Patient Characteristics, Treatment Patterns, and Longitudinal Outcomes in Patients Receiving Mavacamten and Other Treatments for Symptomatic Obstructive Hypertrophic Cardiomyopathy (Obstructive HCM)

The purpose of this study is to evaluate the safety of mavacamten in patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM) treated in the real-world setting. The registry study also provide a real-world understanding of the current obstructive HCM patient population, treatment patterns, and clinical relevant outcomes for patients with symptomatic …

F For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR)

Collection of Blood From Patients With Cancer, Other Tumors, or Tumor Predisposition Syndromes for Genetic Analysis

Background Some genes may be associated with a greater chance of side effects during cancer treatment. These genes may also make certain treatments less effective. Researchers want to collect blood or cheek swab samples from people having cancer treatment to study these genes. Objectives To obtain a blood or cheek …

Collection of Serum and Tissue Samples From Patients With Biopsy-Proved or Suspected Malignant Disease

Selected patients suspected of having or with prior biopsy proof of malignant disease will be seen in the Urologic Oncology Branch, NCI. Blood samples may be collected at the time of the initial visit and at periodic intervals during the course of the disease. These samples will be stored in …