This prospective discovery study is designed to demonstrate the feasibility of using an unshielded, mobile MCG device in an Emergency Department (ED) setting. The main question it aims to answer is whether interpretable data can be reliably collected as part of an ED wo...

The purpose of this study is to evaluate the efficacy and safety of Luspatercept when administered at the maximum approved dose in low-risk Myelodysplastic Syndrome participants who require red blood cell transfusions.

This platform protocol is designed to be flexible so that it is suitable for a wide range of settings within health care systems, for remote settings, and in community settings where it can be integrated into COVID-19 programs and subsequent treatment plans. Thi...

The purpose of this research study is to evaluate the effectiveness and safety of Ritlecitinib in skin and blood in persons with Cutaneous T-Cell Lymphoma (CTCL). CTCL is a rare type of cancer that starts in the white blood cells and eventually can result in rashes ...

GEn1E-1124-002 is a two-part Phase 2 study to evaluate the safety and tolerability of GEn-1124 in subjects with ARDS. Treatment with IV infusion dosing within 48 hours of ARDS diagnosis. Subjects will be given a second dose approximately 8 hours after the first dose...

Aortix is a circulatory support device for chronic heart failure patients on medical management who have been hospitalized for acute decompensated heart failure (ADHF) and are resistant to diuretic therapy. Eligible ADHF patients with diuretic resistance (irrespective o...

The objective of this study is to understand the effects of HIV cure strategies on the virus and immune cells that reside within the gastrointestinal tract. Subjects receiving therapies with the potential for HIV cure will undergo a colonoscopy to obtain gastrointestina...

This Phase 3 multicenter study evaluates the maintenance of efficacy, safety and tolerability of ecopipam tablets in children, adolescents and adults in the treatment of Tourette's Disorder (TD). The study includes an open-label period followed by double-blind, pl...

Mal de Débarquement Syndrome (MdDS) is an under-recognized balance disorder, which is manifested by persistent false sensations of oscillatory self-motion (rocking/swaying) and/or pulling in a specific direction (gravitational pull). Patients with MdDS typically exp...

Neurofibromatosis type 1 (NF1) is the most common genetic tumor predisposition syndrome, affecting up to 1 in 2500 individuals. Cutaneous neurofibromas are benign with self-limited growth; however, tumor burden may be excessive, tumors do not regress, and they can b...

To generate preliminary safety and effectiveness data for brain-responsive neurostimulation of thalamocortical networks as an adjunctive therapy in reducing the frequency of generalized seizures in individuals 12 years of age or older with Lennox Gastaut Syndrome (L...

Phase 1b Study of R289 in Patients with Lower-risk Myelodysplastic Syndromes (LR MDS)

The Ryan White HIV/AIDS Program (RWHAP) for low-income people with HIV (PWH) is a key resource for reducing HIV health disparities and scaling up evidence-based interventions. As RWHAP serves >50% of US PWH, RWHAP outcomes are vital to achieving "getting-to-zero"/ E...

The purpose of this research study is to determine if treatment with Elagolix will improve body weight, waist circumference, muscle strength, cortisol secretion, blood glucose, cholesterol, and bone quality as well as mood and quality of life in a female patient wit...

The primary objective of the trial is to assess long-term safety and tolerability of apraglutide in subjects with SBS-IF.

FORWARD, the Fragile X Registry and Database, is the largest collection of clinical and demographic data of the Fragile X syndrome (FXS) population in the United States. FORWARD was created to improve the care and quality of life for those living with FXS. Information c...

The purpose of this study is to collect, store, and study blood samples from people with Fragile X Spectrum Disorders. The samples from this study will provide a research for researchers to conduct genetic and other studies. This information may be used to advance the m...

This is a randomized, double-blind, placebo-controlled, multiple-center study, to assess the efficacy and safety of Cannabidiol administered as ZYN002 for the treatment of children, adolescent, and young adult patients with Fragile X Syndrome (FXS). Eligible partici...

This is an open-label Phase 2 study evaluating the long term safety and tolerability of GFB-887 in patients with focal segmental glomerulosclerosis (FSGS), and treatment-resistant minimal change disease (TR-MCD)

The goal of this clinical study is to test how well the study drug, lenacapavir (LEN), works in preventing the risk of HIV.