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Evaluation of Provider vs. Patient Mediated Cascade Genetic Testing of First-Degree Relatives of Patients With Newly Diagnosed Colorectal Cancer
This clinical trial compares patient (proband)-mediated communication to provider-mediated communication for improving genetic testing in first-degree relative…
A Phase I/II Study of Nula-cel in Autologous CD34+ Hematopoietic Stem Cells to Convert HbS to HbA for Treating Severe Sickle Cell Disease
This study is a first-in-human, single-arm, open-label Phase I/II study of nula-cel in approximately 15 participants, diagnosed with severe Sickle Cell Disease…
Genetic Analysis of Immune Disorders
The purposes of this study are to 1) identify the genes responsible for certain immune disorders, 2) learn about the medical problems they cause, and 3) learn …
Evaluation of Progression in Multiple Sclerosis by Magnetic Resonance Imaging (MRI)
Studies performed under 89-N-0045 are designed to examine the natural history of multiple sclerosis (MS) using MRI and immunological measures. In addition to s…
An Open-label, Phase I Study to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Surovatamig Following Single-ascending Dose and Step-up Dose Administration to Adult Participants With Rheumatoid Arthritis or Systemic Lupus Erythematosus
This open-label, Phase I study will assess the safety and tolerability of surovatamig and characterise its PK and PD following subcutaneous administration to p…
Analysis of Cutaneous Phosphorylated Alpha-Synuclein to Identify Patients at Risk of Progressing From Essential Tremor to Parkinson's Disease
Background and Rationale Essential tremor (ET) affects over 6 million Americans and approximately 5% of adults over age 60. Patients with ET have a 10-20 times…
A Prospective, Observational Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy
This is a prospective observational study conducted to evaluate safety, tolerability, and functional outcomes of patients with DMD newly initiating oral givino…
Measuring the Effects of Netarsudil, Latanoprost, and Combination Therapy on Episcleral and Retinal Blood Flow in Ocular Hypertension and Glaucoma Suspects Using Erythrocyte Mediated Angiography In Vivo
The goal of this clinical trial is to compare participants treated with latanoprost to those treated with Rocklatan (netarsudil/latanoprost) to determine wheth…
A Randomized Phase 2 Trial of Fianlimab and Cemiplimab +/- Ipilimumab or Ipilimumab Plus Nivolumab in First-line Advanced Renal Cell Carcinoma (RCC)
This three-arm randomized phase 2 trial will enroll advanced clear cell RCC patients (all IMDC risk groups). Patients will be randomized 2:2:1 to either Arm A …
Safety and Effectiveness of Left Bundle Branch Area Pacing Versus Conventional Cardiac Resynchronization Therapy in Heart Failure
This study will compare two different methods to pace the heart to treat heart failure including: 1. The current standard method of implanting a pacing lead i…
Individualized Response Assessment to Hyperthermic Intraperitoneal Chemotherapy (HIPEC) for the Treatment of Peritoneal Carcinomatosis From Peritoneal Mesothelioma or Atypical Mesothelial Proliferation or From Ovarian, Colorectal, or Appendiceal Primaries
Background: Cytoreductive surgery (CRS) removes tumors in the abdomen. HIPEC is hyperthermic (heated) chemotherapy that washes the inside of the abdomen. CRS …
Studies of the Pathogenesis and Natural History of Systemic Lupus Erythematosus (SLE)
This protocol will evaluate patients with systemic lupus erythematosus (SLE) and their relatives to learn more about how the disease develops and changes over …