Vaso-occulsive crisis is a complication of Sickle cell disease in which the red blood cells (RBC) change shape, causing congestion within the blood vessels that leads to pain and tissue damage.
The study medication FT-4202, an oral tablet, is believed to reduce the rate of sickle cell polymerization and improve RBC membrane function, thereby reduction sickling of RBCs and their hemolysis (breakdown of red cells) that causes vascular obstruction and anemia.
This study will consist of a 52-week, randomized (volunteers are selected by chance to receive study either study medication or placebo) , placebo controlled (a placebo is a look-alike pill that contains no active medication). There will be 17 study visits.
The study is followed by a 52-week open label extension study in which all participants will receive study medication. There will be 11 study visits.
The purpose of this research is to test out a new way of treating generalized anxiety disorder (GAD) with low intensity focused ultrasound pulsation (LIFUP). If the subject agree to participate in the research study, the participation will last approximately 10.5 hours over seven (7) days. The visits are as follows: 1- consent and MRI, 2- and 3- focused ultrasound will be delivered outside of the MRI environment, 4- MRI plus focused ultrasound, 5- assessments 1 week post focused ultrasound, and 6- one month follow-up assessments. Research studies are voluntary and include only people who agree to take part.
The Drug Product ZYN002 is a transdermal CBD gel. CBD is the primary non-euphoric cannabinoid contained in the Cannabis sativa L. plant. The CBD contained within ZYN002 is a pharmaceutically produced Active Pharmaceutical Ingredient (API) that is chemically identical to the CBD present in Cannabis. ZYN002 is currently being evaluated in clinical trials in children and adolescents with Fragile X Syndrome (FXS), autism spectrum disorder, 22q11.2 deletion syndrome, and developmental and epileptic encephalopathies. The safety and efficacy of ZYN002 in the treatment of behavioral symptoms in children and adolescents with FXS has been evaluated in three studies: Study ZYN2-CL-009, a completed open-label, multiple-center, multiple-dose study (n=20); Study ZYN2-CL-016, a completed randomized, double-blind, placebo-controlled, multiple-center study (n=212 randomized); and Study ZYN2-CL-017, an ongoing open-label extension and expanded access study to assess the long-term safety and tolerability of ZYN002 (n=240). The present protocol for ZYN2-CL-033 (RECONNECT) is designed to evaluate the efficacy and safety of ZYN002 for the treatment and behavioral symptoms in children and adolescents with genetic evidence of full mutation FXS. Qualified subjects that complete ZYN2-CL-033 will have the opportunity to roll over to the open label ZYN2-CL-017 study.
Smell loss has been reported in more than 50% of older adults. Currently there are no validated methods for classifying patients with smell loss in aging based on either disease site or process. This study aims to better understand how age affects the sense of smell. This study will involve those 50 and older and will take up to 5 years, with 5 follow up annual visits, or can be done remotely.
Islet transplantation is a clinical procedure to treat patients with chronic pancreatitis after removal of their pancreases. Islet survival is influenced by several factors, including but not limited to triggering an inflammatory response. The loss of islet cells during transplantation can cause surgical diabetes, in which the patient will need insulin injections to regulate their blood sugar. The goal of this study is to test whether co-transplantation of the patient's stem cells, called mesenchymal stromal cells (MSCs), along with their islet cells, will protect transplanted islet cells from death, therefore reducing the patient's chances of getting surgical diabetes. MSCs can modulate immune cells and are a promising resource for cell-based therapy.
Patients with cardiovascular disease (CVD) are at increased risk of depression following hospital discharge. Unfortunately, most depressed CVD patients do not receive appropriate care for their depression, often because they are unable to, or are fearful of traveling to providers for the regimen of 8-12 weekly visits of therapy such as Behavioral Activation (BA). This study will compare the effectiveness of BA for depression, delivered via Telehealth, to standard post-CVD hospital discharge best practices. It is predicted that patients who receive BA will have better mental health outcomes and will be less likely to be re-hospitalized compared to patients who receive standard post-discharge care.
This study is for patients that have been diagnosed with recurrent or new head and neck squamous cell carcinoma (HNSCC). Patients either have had a recurrence (the tumor has comeback) or a new head and neck cancer in a different area of the head and neck (second primary) which has been removed by surgery. The study drug used is called pembrolizumab. The purpose of this study is to see if adding pembrolizumab to radiation or if using pembrolizumab by itself after surgery compared to the usual approaches with chemotherapy and radiation will increase life expectancy. Participants can expect to be on this study for up to 12 months and then followed for 5 years.
This study is for patients who have been diagnosed with relapsed (came back) and/or refractory (not responding to treatment) large B-cell lymphoma (LBCL). The investigational product is Lisocabtagene Maraleucel and is administered by infusion. Participants will have had the blood collection (leukapheresis - a laboratory procedure where white blood cells are separated from a sample of blood) procedure, where the T cells (white blood cells) were collected and genetically modified in a laboratory in order to manufacture the lisocabtagene maraleucel T cells for disease treatment. The lisocabtagene maraleucel T cells that were produced do not meet all of the prespecified release criteria to be used as a routine prescription drug as required by the Food and Drug Administration (FDA). This is called a nonconforming lisocabtagene maraleucel. The purpose of this study is to allow participants to be treated with their nonconforming lisocabtagene maraleucel. Participants can expect to be on the study for up to 3 months following the infusion of nonconforming lisocabtagene maraleucel.
Underserved, racial and ethnic minority communities are experiencing higher rates of COVID-19 cases and associated mortality compared to whites due to long standing social and structural inequities that also drive disparities in chronic diseases such as stroke, cardiovascular disease, diabetes, and hypertension. Patients with underlying chronic diseases who are recovering from COVID-19 depend on the support of family and friends (informal caregivers/care partners) who are being exposed to the same pandemic and racial stressors, exposure that can affect the health and quality of life of both partners. The primary goal of this study is to test the efficacy of an adapted, telehealth-enhanced intervention that targets barriers impacting family illness management behaviors of Black/African American (AA) adult COVID-19 survivors and carepartner dyads for improved quality of life and COVID/chronic illness health related outcomes.