This phase 2 study is enrolling patients who have acute myeloid leukemia (AML) with certain biomarkers. This study is being done to see the effectiveness of different combinations of drugs to treat AML. It will involve 3 groups of patients receiving different combinations of Gilteritinib, Azacitidine and Venetoclax. Gilteritinib is an investigational drug, Azacitidine and Venetoclax are FDA approved. The main purpose of the study is to see if the amount of leukemia in the patient's body can be lowered by adding the drug Gilteritinib to the Standard of Care of treating AML with Azacitidine and Venetoclax. The study will include approximately 147 patients. The patients will be randomized into the three groups (like flipping a coin), Group 1 will receive just Azacitidine + Venetoclax, Groups 2 and 3 will also receive Gilteritinib but Group 2 will receive it for more time within a treatment cycle. Patients will complete screening after participating in this treatment trial or SOC therapies. Patients will continue treatment until disease progression, unacceptable toxicity, study closure, death, or withdrawal of consent. The main risk is that the study drugs may not be as good as the usual approach for their cancer or condition at shrinking or stabilizing their cancer. Patients may have none, some, or all of the side effects listed or not listed in the protocol, and they may be mild, moderate, or severe. There is no direct benefit for them in participating in this study.
Currently, there are no FDA-approved medications for the treatment of irritability associated with Autism Spectrum Disorder (ASD). This study is designed to look at the usefulness and safety of lumateperone (CAPLYTA) for the treatment of irritability associated with ASD among pediatric participants between the ages of 5 to 17 years. The study will last approximately 26 weeks and the participants will receive the study drug, lumateperone.
This study aims to study the natural progression of P-SYN quantification by conducting skin biopsies in patients with Parkinson's Disease (PD) and REM sleep behavior disorder (RBD). Participation will be over an 18-month period and will include skin biopsies and clinical examinations every six months. Subjects will be male or female between the ages of 50-100 and have a diagnosis of PD or RBD.
The study is a 48-week clinical study to compare study medication NTX-001 used intraoperatively to promote nerve regeneration compared to the standard of care on surgery for acute nerve damage in the hand on subjects between the ages of 18 to 80 years old. Subjects who present with sever nerve damage of the hand and need surgery to repair the nerve damage within 48 hours of injury. After surgery, subjects will complete 12 total study visits: 6 in person and 6 additional telehealth visits. During the in-person clinic visits, subjects will be assessed by physical examinations, vital signs, safety laboratory assessment and complete questionnaires. Subjects with motor/mixed motor injury will also complete an EMG and NCV assessments with an Occupational Therapist at 2 of the in-person visits. During the telehealth visits, subjects will complete questionnaires.
This study is an 8-week cannabis reduction trial among adults (ages 18+; N=176) with CUD who are interested in reduction to assess effects of cannabis reduction on: 1) cannabis-related problems and consequences, 2) objective measures of sleep and cognitive performance, and 3) patient-reported improvements, including quality of life and CUD severity. All participants will receive CUD treatment and cannabis use will be comprehensively assessed via detailed self-report and urinary cannabinoids.All participants will receive cognitive behavioral treatment (CBT4CBT- a computerized cognitive behavioral therapy) enhanced with motivational interviewing, brief counseling, and financial incentives provided on a sliding scale for demonstrating reduction in urinary cannabinoids.
As growing research suggests noninvasive brain stimulation techniques have the potential to adjunct current treatments or treat Seizure-Type Functional Neurologic Disorder (FND-seiz), also known as Psychogenic Non-Epileptic Seizures (PNES), we aim to evaluate whether a form of accelerated intermittent theta burst transcranial magnetic stimulation (a-iTBS-rTMS), is a practical and well-tolerated treatment for people with this disorder. Transcranial Magnetic Stimulation or TMS uses magnetic pulses to stimulate a part of the brain involved in mood and thinking, the left dorsolateral prefrontal cortex, which has established benefits in disorders known to coincide in patients with FND-seiz, such as depression.
As an open-label, early feasibility study, enrolled participants will receive 6 to 10 treatment sessions each day over 3 to 5 days, with the goal of completing 30 total sessions. This approach was selected because similar protocols have already been shown to be safe and effective in other conditions, and the shortened treatment schedule in comparison to other protocols may make participation easier for people living with FND-seiz. The main goal of the study is to see how many participants can safely and comfortably complete at least 20 of the 30 TMS sessions.
The researchers will also evaluate changes in seizure frequency, quality of life, mood, post-traumatic stress symptoms, physical health, social functioning, and overall satisfaction with treatment. These outcomes will be measured before treatment and again four weeks afterward. The researchers also aim to explore whether people with overlapping conditions, such as depression or PTSD, respond differently to the treatment. Finally, given the overlap between epilepsy and FND-seiz, not all TMS providers are comfortable treating patients with FND-seiz when TMS is indicated for other conditions, thus the researchers aim to outline a protocol to ensure safety and increase TMS access for FND-seiz patients.
Patients with drug-resistant epilepsy often experience problems with mood, thinking, or behavior that cannot be explained by seizure activity alone. This study will examine how cognitive and mood-related brain regions communicate in patients undergoing routine intracranial electroencephalogram (iEEG) seizure assessment in the Epilepsy Monitoring Unit at the Medical University of South Carolina (MUSC). While the clinical electrodes are in place, we will apply brief single magnetic pulses (single-pulse transcranial magnetic stimulation, or spTMS) to the scalp in specific brain regions and record the brain's electrical response through the existing electrodes; no additional surgery is required. We will compare the responses to stimulation of an emotionally and cognitively relevant region (left dorsolateral prefrontal cortex) with a contrast site (primary motor cortex). We will also investigate whether momentary brain rhythms and seizure-related electrical activity affect responses propagation through the brain. The findings may help identify measurable brain signaling patterns ("biomarkers") to understand how cognitive-emotional brain networks work in people with epilepsy and inform future personalized non-invasive brain stimulation methods for treating neurological and psychiatric disorders.
Newborns who are born premature or infants who suffer brain injury are at risk for motor problems. The common motor skills of reaching and grasping that infants have to learn can be weaker on one side of the body, depending on the site of the brain injury. These skills are routinely practiced with an occupational therapist once or twice a week, to help the infant strengthen these skills. A high intensity therapy program of constraint induced movement therapy (CIMT) may be available for the infant, but it takes from 40-120 hours total treatment time for most infants to improve their motor skills.
Transcutaneous auricular vagus nerve stimulation (taVNS) stimulates a branch of a major nerve by the ear, called the vagus nerve, that may help improve your child's ability to learn motor skills. CIMT involves placing a soft mitt constraint on the stronger arm and hand while encouraging your child to use the weaker arm and hand during intensive therapy sessions. By using both CIMT and the nerve stimulation together, we hope your child's movement skills will improve more than with therapy alone.
The purpose of this study is to evaluate the safety and effectiveness of taVNS to improve motor skills when paired with the minimal amount of CIMT and whether a measure of the strength of the brain circuit to the arm and hand muscles can tell us how well a child may respond to this therapy.
This study is for people who have experienced a traumatic event in the past one year and drink alcohol. The research involves completing a five week behavioral treatment for stress and alcohol use. Participants will complete surveys during visits. Participants may also be asked to complete a interview about their experiences.
Using surveys researchers will assess caregiver choice, caregiver quality of life (CQOL), caregiver sleep, caregiver stress, and caregiver burden (CB). Some participants answering surveys (approximately 20), will be chosen by researchers to participate in an interview to gain a deeper understanding of the perspectives of individuals who assume caregiving responsibilities without a personal sense of choice.