All Clinical Trials

This screening and multi-sub-study Phase 1b/2 trial will establish a method for genomic screening followed by assigning and accruing simultaneously to a multi-study "Master Protocol (BAML-16-001-M1)." The specific subtype of acute myeloid leukemia will determine which sub-study, within this protocol, a participant will be assigned to evaluate investigational therapies or combinations with the ultimate goal of advancing new targeted therapies for approval. The study also includes a marker negative sub-study which will include all screened patients not eligible for any of the biomarker-driven sub-studies. Read Less

This is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of Cipaglucosidase Alfa/Miglustat treatment in enzyme replacement therapy (ERT)-experienced and ERT-naïve pediatric subjects with Pompe disease, aged 0 to \< 18 years Read Less

Brain metastases are a source of much morbidity and mortality in adults with primary solid malignant tumors. With improvements in systemic therapy that prolong survival but have limited central nervous system penetration, patients with brain metastases are at increasing risk of developing and experiencing long-term side effects from treatment of brain metastases. The overarching goal of this study is to better understand the determinants of RT-associated changes in white and gray matter function and associated neurocognitive decline. Read Less

This is a prospective, longitudinal, observational study of free-living activity trackers and patient reported outcomes to test the hypothesis that daily activity will have stronger prognostic value than 6MWD in patients with pulmonary hypertension after 12 weeks. Read Less

This phase II trial studies how well standard of care hypofractionated radiation therapy followed by durvalumab with or without tremelimumab works in treating patients with hepatocellular cancer (liver cancer) that has spread to other places in the body (advanced) and that is growing, spreading, or getting worse (progressing) after treatment with PD-1 inhibitor immunotherapy. In some patients, cancer cells and immune cells start to express signals that stop the body's immune system from killing the cancer. New drugs being developed, such as durvalumab and tremelimumab, are designed to target and block these signals and may help increase the immune response to prevent or slow down cancer growth. Hypofractionated radiation therapy delivers higher doses of radiation therapy over a shorter period of time and may help the immune system work even better. Giving durvalumab with or without tremelimumab after radiation therapy may work better than radiation therapy alone in treating patients with liver cancer. Read Less

The QUIT-Mobile study proposes to use mobile phone self-monitoring and feedback to enhance and sustain over 12-months the impacts of the Quit Using Drugs Intervention Trial (QUIT), an effective screening and brief intervention (SBI) previously successful in reducing risky drug use (i.e., moderate use) in low-income, diverse patients over a 3-month follow up. The investigators will conduct the QUIT-Mobile study for patients who receive care in clinics of federally qualified health centers (FQHC) in Southern California over 12-months follow up. The study is an Effectiveness-Implementation Hybrid Type 1 design consisting of a single-blind, 3-arm, RCT with adult, mostly Latino FQHC primary care patients with risky drug use (ASSIST score 4-26), randomized to 3 conditions (n=200/arm, n=600 total): 1) QUIT-Mobile; 2) standard QUIT; 3) Usual Care. Qualitative data on implementation facilitators and barriers will inform future scale-up and sustainability, in addition to cost data analyses. The aims are to examine effectiveness in reducing risky drug use and cost-effectiveness comparing the three arms over 3-, 6- and 12-months. Drug use measures include self-reports for past 30-days and urine drug screen validation for underreporting (acknowledging that people with moderate risk drug use have sporadic drug use patterns requiring longer self-report recalls for drug use that urine screens may not detect). The 3-arm study enables testing of the independent and synergistic effects of QUIT-Mobile compared to QUIT and both to Usual Care. The 12-month timeline reflects annual primary care visits when screening and brief intervention would be repeated routinely. The QUIT intervention contains 3 primary components: 1) patient screening with the WHO ASSIST, 2) brief clinician advice (\&lt;3 minutes) including opioid overdose prevention education, and 3) 2- and 6-week telephone drug-use health coaching sessions utilizing motivational interviewing and cognitive behavioral techniques, delivered by paraprofessional health coaches. QUIT-Mobile tests the addition of mobile phone self-monitoring, automated feedback, and coach monitoring dashboard to enhance and sustain QUIT\&#39;s drug use reductions using mobile-web app, text-messaging (SMS), or interactive voice response (IVR). Read Less

The purpose of this study is to monitor physiological and molecular changes during and following CAR-T cancer cell therapy, towards improved management of adverse events including Cytokine Release Syndrome and neurotoxicity. Our study aims are to improved early detection and precise management of adverse events for patients receiving Chimeric antigen receptor T- cell (CAR-T): 1. To assess the feasibility, including accuracy, usability, and usefulness of wearable sensors in CAR-T patients. 2. To generate comprehensive multiomic profile analysis following CAR-T therapy. 3. To perform integrated analysis of wearables sensor data, omics data, and symptom/clinical data. Read Less

Sickle Cell Disease is one of the most common genetic diseases in the United States, occurring in approximately 1 in 400 births. Approximately 100,000 individuals are diagnosed with SCD in the United States. Mortality for children with SCD has decreased substantially over the past 4 decades, with \>99% of those born in high resource settings, including the United States, France, and England, now surviving to 18 years of age. However, the life expectancy of adults with SCD is severely shortened. Dysfunction of the heart, lung, and kidney is directly associated with decreased life expectancy. With the variety of curative therapies that are now available for SCD, long-term health outcomes studies are time-sensitive. As of now, efforts to determine long-term health outcomes following curative therapies for SCD have been limited. Though curative therapies initially should provide a cure for symptoms of SCD, there is the risk of late health outcomes to consider. Defining health outcomes following curative therapy is essential to improve personalized decision-making when considering curative versus disease-modifying therapeutic options. The primary goal of this study is to determine whether curative therapies for individuals with SCD will result in improved or worsening heart, lung, and kidney damage when compared to individuals with SCD receiving standard therapy. The investigators will also explore whether certain genes are associated with a good or bad outcome after curative therapy for SCD. Read Less

Polysomnography (PSG) and drug-induced sleep endoscopy (DISE) are widely used diagnostic studies for assessing obstructive sleep apnea (OSA) severity and collapse patterns of the upper airway anatomy during sleep. Hypoglossal nerve stimulation (HNS) therapy for obstructive sleep apnea suffers from variable response at the level of the soft palate. The Investigators propose a study examining the physiologic effect of ansa cervicalis stimulation (ACS) alone and in combination with HNS during PSG and DISE. Read Less

The proposed project involves a high-quality randomized controlled (RCT) design that examines the effects of a remotely-delivered, racially-tailored exercise training program for immediate and sustained improvement in walking dysfunction, symptoms, and health-related quality of life (HRQOL) among African-Americans with MS residing in rural environments. The primary analysis will test the hypothesis that those who are randomly assigned into the intervention condition (i.e., exercise training) will demonstrate (a) improvements in outcomes from baseline that (b) are sustained over 4-months of follow-up compared with those in the control condition (i.e., stretching). Read Less

Dental Fear and Anxiety is an emotional experiences affecting many children and adolescents which can lead to behavioral problems in the dental setting. The purpose of the proposed study is to investigate the effect of virtual reality on anxiety, behavior, and pain in children undergoing restorative dental procedures. Children enrolled in the study will include patients ages 6-18 who are healthy and require two or more dental sealants (at least one sealant per side). The participants in the study will serve as their own control using a split-mouth cross-over randomized control clinical trial design and will be randomly assigned to receive virtual reality or traditional behavior guidance techniques first during dental sealant placement. The objectives of this study are to explore the associations between the use of virtual reality distraction during dental sealant placement with Frankl score, FLACC scale, and anxiety based on the change in heart rate, and pain based on self-reported FPS-R when compared with to the control group. The crossover design will be assessed by a linear mixed model with patient treated as a random effect. This model will include treatment, visit, treatment by visit interaction term as well as a treatment sequence variable. Prior to analysis, the primary outcome measures will be assessed for normality using a Shapiro-Wilk statistic as well as quantile-quantile (QQ) plots. Should the data deviate sharply from a normal distribution, normalizing transformations will be sought and applied to the data. Read Less

The goal of this clinical trial is to compare pregnenolone and placebo (a placebo is a look-alike substance that contains no active drug) in Operation Enduring Freedom/Operation Iraqi Freedom/Operation New Dawn (OEF/OIF/OND)-Era Veterans with a history of chronic Traumatic Brain Injury (TBI). The main questions it aims to answer are: * Does pregnenolone improve psychological health, overall physical function, cognition, symptoms of PTSD, and pain more than placebo over the 8-week study period, and what is the most effective dose of the drug that is safe and well-tolerated? * What are the biological effects of pregnenolone, and how do pregnenolone and other molecules change over the course of treatment? (and do pregnenolone and other molecules predict clinical improvement?) Participants who are eligible and consent to participate in the study will: * be randomized in a 1:1 ratio to take pregnenolone or placebo * be given pregnenolone or placebo to take each day at home * will participate in 6 visits over 11 weeks for tests, exams and procedures that are for study purposes (each visit will last 1.5 - 3 hours) * be evaluated at each visit to determine if there are any bad reactions to the study drug and if study participation is still appropriate * be financially compensated for their visit time and travel cost Read Less