Digital Therapeutic for Adolescent Depressive Symptoms in Cardiology and Gastroenterology
Depressive SymptomsMajor Depressive Disorder7 moreThe primary aim of this pilot study is to assess the feasibility, acceptability, and preliminary evidence of efficacy of a self-guided, cognitive behavioral therapy (CBT)-based mobile app intervention (SparkRx) for symptoms of depression among adolescents being treated in specialty medical care settings at Children's Hospital of Los Angeles (CHLA).
CFTR Modulators and Gastrointestinal Complications
Cystic FibrosisGastrointestinal System Disease5 moreTo elucidate the similarities and distinctions in non-pulmonary manifestations of cystic fibrosis (CF) including distal intestinal obstruction syndrome (DIOS) incidence and pancreatic enzyme replacement therapy (PERT) use between US and UK CF populations in a parallel study using data from the UK and US CF registries. To assess how CFTR modulators impacted upon recorded PERT use and incidence of DIOS.
Domperidone for Relief of Gastrointestinal Disorders
GastroparesisGERD5 moreThe purpose of this study is to make Domperidone available to patients with gastrointestinal disorders who have failed standard therapy and who might benefit from it.
Efficacy and Safety of Grazoprevir (+) Uprifosbuvir (+) Ruzasvir (MK-3682B) (MK-5172 + MK-3682 +...
HepatitisHepatitis C8 moreThis is a randomized, multicenter, 2-part, open-label trial of the combination regimen of grazoprevir (GZR [MK-5172]; 100mg), uprifosbuvir (UPR [MK-3682]; 450 mg) and ruzasvir (RZR [MK-8408]; 60 mg) with and without Ribavirin (RBV) in cirrhotic (C) or non-cirrhotic (NC) participants infected with hepatitis C virus (HCV) previously failing a direct-acting antiviral regimen (DAA). The combination regimen, referred to as MK-3682B, will be administered as two fixed-dose combination (FDC) tablets, given once-daily. The study will evaluate the efficacy of MK-3682B with or without RBV as assessed by the proportion of participants achieving Sustained Virologic Response 12 weeks (SVR12) after the end of all study therapy.
Sirolimus for Eosinophil-Associated Gastrointestinal Disorders
Eosinophilic GastroenteritisEosinophilic EsophagitisBackground: Eosinophil-associated gastrointestinal disorders (EGID) are a group of related disorders that affect the esophagus, stomach, and bowel. There are two major types of EGID, eosinophilic esophagitis and eosinophilic gastroenteritis. They are caused by the body's immune system being activated by food allergens, which then damages the gut wall. People with EGID have large numbers of eosinophils (a type of white blood cell) in their gut. EGID can cause difficulty swallowing, abdominal pain, or nausea. At present, there are no drugs specifically approved to treat EGID. Most adults who have EGID receive steroid therapy to manage the symptoms. However, long-term steroid use may cause other problems in the body. Researchers want to see if low-dose sirolimus can be used to treat EGID. Sirolimus is a drug used to prevent rejection of transplanted organs. It may be able to keep the body's immune cells from being activated by food allergens and decrease the eosinophils. Objectives: - To see if low dose sirolimus is safe and decreases blood or gut eosinophils in EGID. Eligibility: Individuals between 18 to 65 years of age who have EGID. Participants must also have an elevated blood eosinophil count and positive blood tests for IgE antibodies to foods. Design: Participants who are on medicine for EGID or related symptoms must be on a stable dose for 1 month before screening and stay on that dose throughout the study. Participants will be screened with a medical history and physical exam, and review their symptoms. They will provide blood and urine samples. They will also have heart and lung function tests. Some participants may have allergy skin tests. At the first study visit, participants will have 2 days of inpatient tests. They will repeat the tests from the screening visit. They will also have a full analysis of the esophagus, stomach, and small intestine. On the second day, they will start to take sirolimus as a liquid with orange juice or water. Participants will continue to take sirolimus at home. They will record their doses and any symptoms. They will also have a visit to provide blood samples about 2 weeks after the first study visit. At the second study visit (about a month after the first visit), participants will repeat the tests from the screening visit. The sirolimus dose may be adjusted as needed. Participants will take sirolimus for at least another 28 days. Depending on the dose of the drug and the blood test results, some participants may need to take it for up to 112 days. Those who take the drug for a longer period will have additional study visits with tests. There will be another study visit when participants stop taking the drug. The last visit will be a final follow-up visit.
A Home-Based Approach Study to Evaluate the Efficacy and Safety of Alectinib in Locally-Advanced...
NeoplasmsColorectal Neoplasms30 moreThis study will evaluate the efficacy and safety of alectinib in participants with Anaplastic Lymphoma Kinase (ALK)-positive locally advanced or metastatic solid tumors other than lung cancer.
Detection of Jaundice From Ocular Images Via Deep Learning
OphthalmologyArtificial Intelligence1 moreOur study presents a detection model predicting a diagnosis of jaundice (clinical jaundice and occult jaundice) trained on prospective cohort data from slit-lamp photos and smartphone photos, demonstrating the model's validity and assisting clinical workers in identifying patient underlying hepatobiliary diseases.
Impact of Improving GI Symptoms on Autism Symptoms and Oxidative Stress
Autistic DisorderConstipation1 moreThe clinical manifestation of autism spectrum disorder (ASD) is complex, with medical and mental health disruptions that occur with the three core behavioral criteria used for diagnosis (social behavior, communication, restricted interests/repetitive behavior). Co-occurring medical conditions, such as gastrointestinal dysfunction (GID), often are overlooked when designing research strategies to understand the mechanisms underlying the expression of ASD. This study was initially a collaboration between Children's Hospital Los Angeles (CHLA) and the Children's Hospital at Vanderbilt University. The current research project proposes to recruit subject at CHLA and the Center for Autism and Developmental Disorders (CAND), a Children's Hospital of Orange County and University of California Irvine (UCI) Health collaborative program. In Aim 1, the investigators will characterize GID in pediatric populations with ASD. Over a 12 month period, subjects will receive standard of care for their GID, typically functional constipation. The study population will be characterized with a standardized instrument for diagnosing functional GI disorders in children, the Questionnaire on Pediatric Gastrointestinal Symptoms, and with the clinical acumen of an experienced pediatric gastroenterologist. Nutritional information also will be collected to determine whether there are patterns of GIDs that correlate with dietary and nutritional status. The in-depth characterization and treatment of GIDs in children with ASD will provide a unique way of determining if ASD symptom and GID symptom improvement are related to each other. In Aim 2, the investigators will do in-depth assessment of each subjects functional status for social communication, emotional regulation, cognitive function, speech-language, sensory integration, and a biomarker of oxidative stress. The latter will be measured in urine samples over the course of one year. There are no direct interventions for autism symptoms in this study. Rather, subjects will receive standard of care for the GID diagnosis and secondary effects on ASD symptoms will be evaluated. Our power calculation shows that the investigators will be adequately powered with the proposed study design and recruitment targets. As part of the study, the investigators have developed a collaboration with investigators in the University of Southern California (USC) School of Engineering, in which the investigators will work with them to develop computational tools to assist in the characterization of videotaped Autism Diagnostic Observation Schedule (ADOS-2) assessments.
Cardiac Structure and Function in Patients With Cystic Fibrosis
Cystic FibrosisCystic Fibrosis-related Diabetes14 moreIn a prospective observational cohort study (n = 100), the investigators aim to assess the correlation between cardiac biomarkers, advanced echocardiography and cystic fibrosis genotype and severity and determine whether these are prognostic markers of heart disease in patients suffering from cystic fibrosis (CF).
Efficacy and Tolerability of ABT-869 Versus Sorafenib in Advanced Hepatocellular Carcinoma (HCC)...
Hepatocellular Carcinoma Non-resectableHepatocellular Carcinoma Recurrent13 moreThe primary objective of this study is to assess the overall survival (OS) of oral linifanib given as monotherapy once daily (QD) compared to sorafenib given twice daily (BID) per standard of care in subjects with advanced or metastatic HCC.