Pediatric traumatic injury is the leading cause of death and morbidity among US adolescents and are associated with mental health and health risk outcomes, including posttraumatic stress and depression, deficits in physical recovery, social functioning and quality of life, which if unaddressed, may contribute to increased use of health care services. In 2015 our team launched the Trauma Resilience and Recovery Program (TRRP) at Medical University of South Carolina, a scalable and sustainable, technology-enhanced, multidisciplinary stepped model of care – one of the few in the US - that provides early intervention and direct services to improve access to evidence-based mental health care after traumatic injury for children, adults and families. We have found this model of care to be feasible and acceptable to adolescent patients (ages 12-17) at each level of service. TRRP includes 3 major steps: (1) in-hospital education, brief risk reduction session, and tracking patients' emotional recovery via an automated text-messaging system, (2) a 30-day screen via telephone to identify patients who are good candidates for psychological treatment, and (3) providing referral to best-practice telehealth-based or in-person assessment and treatment. We have partnered with three accredited Level I and II pediatric trauma centers, Prisma-Health Upstate, Children's of Alabama, and Boston Children's Hospital, and propose a multi-site hybrid 1 effectiveness-implementation randomized controlled trial with 300 adolescent (ages 12-17) traumatic injury patients to assess the extent to which TRRP promotes improvement in quality of life and emotional recovery and gather preliminary data on the potential for TRRP to be implemented in other Level I trauma centers. This study will provide valuable data on the efficacy, preliminary effectiveness and potential for implementation of an innovative, cost-effective, sustainable technology-enhanced intervention designed to address the unique needs of adolescent injury patients and mitigate short- and long-term impact of injury on mental health, quality of life, and overall well-being.
This study is for patients who have advanced cancers, such as head and neck cancer, colorectal cancer, breast cancer, and others. This study is testing a new treatment for these types of cancer. The new test drug is called ficerafusp alfa. Pembrolizumab is an approved drug by the United States Food and Drug Administration (FDA) for the diseases described in this study and will be used as approved. The type of cancer a patient has will determine whether he/she will get ficerafusp alfa alone or ficerafusp alfa in combination with pembrolizumab. Participants will receive either ficerafusp alfa alone, ficerafusp alfa in combination with pembrolizumab or ficerafusp alfa, depending on your cancer until the cancer gets worse, they experience bad side effects, or until they withdraw consent, or until the Investigator considers it is in his/her best interest to discontinue the study drug.
This study is for newly diagnosed asymptomatic high-risk patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). The purpose of this study is to find out if starting treatment with the investigational products, venetoclaz and obinutuzumab (V-O) early (before symptoms occur) affect how long you live compared to the usual approach of starting treatment after showing symptoms. Participants can expect to receive treatment for up to 12 months, until the cancer gets worse or until the side effects are too great. After study completion, participants will continue to be followed for up to 10 years.
The purpose of this research study is to identify brain activation patterns in response to deep brain stimulation (DBS). To participate in this study, participants are required to have had a DBS implant or to be healthy controls without a history of a neurological disorder. Participants will undergo screening, a motor assessment session and a 30-minute Magnetic Resonance Imaging (MRI) session where their DBS device will be turned on and off in a cycled pattern. This study will be separated over 3 visits (Visit 1 for screening, Visit 2 for motor assessments and Visit 3 for the MRI scans). The total study duration will be approximately 5 hours.
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.
This study is for patients with gallbladder cancer. The purpose of this study is to compare the usual treatment (surgery plus chemotherapy after) to using chemotherapy both before and after surgery.
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.
This study will be looking at biopsies taken from mediastinal lymph nodes and a blood sample from patients who have known or suspected metastatic lung cancer. These two samples will be compared using next-gen sequencing to explore if either sampling method offers better results.
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.