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 ...

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...

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 with mi...

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 and adolescent patients with Fragile X Syndrome (FXS). Eligible participants will par...

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 primary objective of this study is to evaluate the efficacy of lenacapavir (LEN) in preventing the risk of human immunodeficiency virus (HIV) - 1 infection relative to the background HIV-1 incidence rate. The study will be conducted in 2 parts: a cross-secti...

This study is to investigate the safety, tolerability and efficacy of Sulindac (HLX-0201) and Gaboxadol (HLX-0206) in males with Fragile X Syndrome (FXS) with confirmed full FMR1 mutation treated over a 10 week period in an outpatient setting.

This is a randomized, double-blind, placebo-controlled, proof-of-concept (PoC), Phase 2 study to assess the safety, PK, and PD of SC administration of HM15912 in adult subjects with SBS-associated intestinal failure (SBS-IF).

The purpose of this study is to examine cardiopulmonary function in Chronic Fatigue Syndrome (CFS) patients and determine how it relates to the common symptom of Post-exertional malaise (PEM). Subjects will complete a maximal exercise test on 2 subsequent days. Tota...

Approximately 216 patients with acute pancreatitis and accompanying SIRS will be randomized at approximately 30 sites. Patients will be randomly assigned to either Auxora at one of three dose levels or one of three placebo volumes to maintain the double-blind. Study...

The primary objective of the trial is the confirmation of the efficacy of apraglutide to evaluate the efficacy of weekly subcutaneous apraglutide in reducing parenteral support dependency.

The primary objective of the trial is the confirmation of the efficacy of apraglutide to evaluate the efficacy of weekly subcutaneous apraglutide in reducing parenteral support dependency.

This is a Phase 2A, single dose, open-label study to evaluate the safety, tolerability, and efficacy of a low-dose, 40-minute infusion into the veins (intravenous infusion or "IV") of ketamine in children with ADNP syndrome (Activity-Dependent Neuroprotective Protein). ...

This is a phase 2a study evaluating the safety and tolerability of multiple ascending doses of GFB-887 in patients with diabetic nephropathy (DN), focal segmental glomerulosclerosis (FSGS), and treatment-resistant minimal change disease (TR-MCD).

The primary objective of this study is to evaluate the efficacy of magrolimab in combination with azacitidine compared to that of azacitidine plus placebo in previously untreated participants with intermediate/high/very high risk myelodysplastic syndrome (MDS) by Re...

Working with patients at risk for suicide is highly stressful for clinicians and often elicits powerful negative emotional responses that may adversely affect suicidal outcomes. A proposed explanation has been that negative emotional responses may result in less emp...