DevRobust Treatment for Mal de Débarquement Syndrome
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...
Age: 18 - 78 years
Gender: All
DPCP to Treat Cutaneous Neurofibromas Associated With NF1
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...
Age: 18 years - 66+
Gender: All
RNS System LGS Feasibility Study
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...
Age: 15 years - 66+
Gender: All
Study of R289 in Patients With Lower-risk Myelodysplastic Syndromes (LR MDS)
The study will be an open-label, Phase 1b study of R289 to determine tolerability and
preliminary efficacy in patients with LR MDS who are relapsed, refractory/resistant,
intolerant, or have inadequate response to prior therapies such as erythropoietin (EPO),
thro...
Age: 18 years - 66+
Gender: All
Testing a Novel Data-to-Suppression (D2S) Intervention Strategy in the Ryan White HIV/AIDS Program
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...
Age: Birth - 66+
Gender: All
An Oral GnRH Antagonist to Treat Mild Autonomous Cortisol Excess (MACE) Due to Adrenal Adenomas in Postmenopausal Women
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...
Age: Birth - 66+
Gender: Female
Fragile X Online Registry With Accessible Research Database (FORWARD)
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...
Age: 0 - 99 years
Gender: All
Fragile X Spectrum Disorders Biorepository
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...
Age: 0 - 99 years
Gender: All
Clinical Study of Cannabidiol in Children and Adolescents With Fragile X Syndrome
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...
Age: 3 - 17 years
Gender: All
An Open-Label, Long-term Study of GFB-887 in Patients With Glomerular Kidney Diseases
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)
Age: 18 - 75 years
Gender: All
Study to Assess the Effectiveness and Safety of Lenacapavir for Human Immunodeficiency Virus (HIV) Pre-Exposure Prophylaxis
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...
Age: 16 years - 66+
Gender: All
Outpatient Treatment With Anti-Coronavirus Immunoglobulin
The primary objective of the Outpatient Treatment with Anti-Coronavirus Immunoglobulin (OTAC)
(INSIGHT 012) trial is to compare the safety and efficacy of a single infusion of
anti-COVID-19 hyperimmune intravenous immunoglobulin (hIVIG) versus placebo among adults w...
Age: 18 years - 66+
Gender: All
STIMULUS MDS-US : Sabatolimab Added to HMA in Higher Risk MDS
Main objective of this study is to describe and evaluate safety and efficacy of MBG453
(sabatolimab) in combination with FDA approved HMAs of investigator's choice (IV Decitabine
or Azacitidine /SC Azacitidine /Oral Decitabine (cedazuridine combination (INQOVI))
Age: 18 - 99 years
Gender: All
Ocrelizumab for Preventing Clinical Multiple Sclerosis in Individuals With Radiologically Isolated Disease.
This is a multicenter, randomized, double-blind, placebo-controlled, Phase 4 study in which
eligible patients with RADIOLOGICALLY ISOLATED SYNDROME (RIS) (as defined by meeting 2017
McDonald criteria for DIS) will be randomized 1:1 to receive ocrelizumab treatment o...
Age: 18 - 40 years
Gender: All
Phase 2 Study to Assess the Safety, PK, and PD in SBS-IF Subjects
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).
Age: 18 years - 66+
Gender: All
Cardiovascular Analysis of PEM
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...
Age: 25 - 60 years
Gender: All
A Study of Auxora in Patients With Acute Pancreatitis and Accompanying SIRS
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...
Age: 18 years - 66+
Gender: All
Trial to Evaluate Efficacy and Safety of Apraglutide in SBS-IF
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.
Age: 18 years - 66+
Gender: All
Trial to Evaluate Efficacy and Safety of Apraglutide in SBS-IF
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.
Age: 18 years - 66+
Gender: All
Low-Dose Ketamine in Children With ADNP Syndrome
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). ...
Age: 5 - 12 years
Gender: All