There are about 173942 clinical studies being (or have been) conducted in United States. The country of the clinical trial is determined by the location of where the clinical research is being studied. Most studies are often held in multiple locations & countries.
The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of multiple escalating oral doses of PF-06954522 in adult participants with inadequately controlled type 2 diabetes mellitus (T2DM) on metformin (Part A) and optionally in non-diabetic participants with obesity (Part B).
The study objective is to improve accuracy in the early detection of neurodevelopmental impairment, especially CP, by evaluating the timepoint (in weeks post term age) that the Prechtl GMA is most useful for prediction of neurodevelopmental impairment at two years of age in children with and without medical complexity. The study team plans to recruit 100 healthy, term-born infants and 250 infants at risk of developing CP for a total of 350 enrolled infants.
The "Genomic medicine Risk Assessment Care for Everyone" (GRACE)" intervention project will develop a scalable end-to-end solution for risk assessment and management that meets the needs of those populations living in low resource settings. The long-term goal is to increase access to and uptake of risk-informed evidence-based guidelines that will improve population health through better patient outcomes, higher quality of life, and decreased costs. The three primary aims are: Aim 1: Develop a scalable implementation framework that guides each unique clinical setting, including low resource settings, in deploying GRACE effectively for the needs of their patients and providers. Aim 2: Facilitate the potential for genomic medicine to promote population health by broadening access to and uptake of genomic risk assessment by the general population through a pragmatic implementation-effectiveness trial of GRACE. Aim 3: Reduce health disparities related to genomic medicine by allowing individual adaption of GRACE to suit their level of resources, education, and access within a pragmatic implementation-effectiveness trial. Three sets of participants will be engaged: patients (n=750), providers (n=25), and family members of "probands" (i.e., patients that have a genetic change that increases risk, n~500). Patient participants will be asked to complete a baseline survey, enter their family health history information into MeTree (a family health history web-based platform) and complete a survey about their experience using the platform. Subsequent study procedures will depend on: 1) the results of their MeTree risk evaluation, 2) their acceptance/declination of genetic testing (for those categorized as needing testing by MeTree), and 3) the results of the test (for those accepting testing). Provider participants will be providers who are the primary care physicians treating one or more patients enrolled in the patient participant group. Providers will be notified on a patient by patient basis once the patient participant under their care has complete the risk assessment process and the risk report is available from MeTree. At study completion, provider participants will be asked to complete a survey about their demographics, practice, and experiences with the study. Blood relatives of the probands who are identified by the proband as open to engaging with the study will be contacted and offered genetic counseling and genetic testing.
Moesin deficiency was initially described in 7 male participants aged 4 to 69 years and is characterized by lymphopenia of the 3 lineages and moderate neutropenia. Genetically, 6 out of 7 participants had the same missense mutation in the moesin gene located on the X chromosome. The 7th patient has a mutation leading to the premature introduction of a STOP codon into the protein.Clinically the 7 participants with X-linked moesin-associated immunodeficiency all presented with recurrent bacterial infections of the respiratory, gastrointestinal or urinary tracts, and some had severe varicella.Therapeutically, in the absence of a molecular diagnosis and due to his SCID-like phenotype, one patient was treated with geno-identical hematopoietic stem cell transplantation . The remaining are untreated or treated with immunoglobulin substitution and/or prophylactic antibiotics. Since this study, the moesin gene has been integrated into DNA chips used for the molecular diagnosis of immune deficiencies in several countries. Physicians in Canada, the United States, Japan, South Africa and Europe have contacted us with a total of 16 known participants to date. Because of their very low severe, uncontrolled CMV infection and the absence of treatment recommendations, two 2 American participants were treated with allogeneic transplantation with severe post-transplant complications (1), and one of the participants died as a result of the transplant. Management of XMAID participants therefore varies widely from country to country, depending on age at diagnosis and clinical picture. It ranges from no treatment treatment (associated with recurrent infections and skin manifestations), IgIv substitution and/or antibiotic prophylaxis antibiotic prophylaxis, with low toxicity and apparent efficacy, and allogeneic transplantation, with all the risks risks involved (graft-related toxicity, graft versus host, disease, rejection, risk of infection). The Investigators therefore feel it is important to review the diagnosis, clinical presentation and management of X-MAID participants. The study the investigator propose will enable to understand the presentation of X-MAID participants, establish guidelines and provide the best treatment for each patient according to his or her clinical picture
D8820C00001 is an exploratory, non-interventional, unblinded, observational study evaluating the acceptability, feasibility and performance of methods to collect, transport and test biospecimens in participants ≥ 18 years of age with an active CDI. Participants will also be monitored for recurring episodes of diarrhea and will need to complete validated PROs and study evaluation questionnaires
The goal of this observational study is to evaluate the feasibility of using wearable sensor and digital technologies to measure motor and speech function in adults with autoimmune Myasthenia Gravis (MG). The main question[s] it aims to answer are: - To measure the correlation of sensor-based measures of motor function with existing outcome measures including the MG-ADL, MGQOL15r, QMG, MGComposite, and Neuro-QOL Fatigue scales. - To develop and validate tablet-based digital assessments of speech and facial expression and to compare with existing outcome measures. Participants will wear a pendant sensor for 7 days and then participate in tablet-based and in-person myasthenia-specific physical examinations. This will be performed in concert with routine care in the Massachusetts General Hospital MG clinic.
E-cigarettes have emerged as an effective strategy for replacing cigarettes among smokers from the general population, but there is a dearth of studies investigating their utility in replacing cigarettes among smokers with opioid use disorder (OUD). This study aims to evaluate the feasibility and acceptability of implementing a cigarette harm reduction intervention involving e-cigarettes in office-based buprenorphine clinics.
This is an open-label, multicenter, Phase 1/2, first-in-human (FIH), dose-escalation and cohort-expansion study of LAT010 to evaluate the safety, tolerability, immunogenicity, PK, PD, and antitumor activity in patients with advanced solid tumors. The study consists of 2 parts: Phase 1 dose-escalation and Phase 2 cohort expansion.
Racial and ethnic based stressors, such as microaggressions, are pervasive, distressing, and result in lasting negative repercussions for minoritized students at predominantly white institutions (PWIs). These racial and ethnic based stressors are experienced in addition to the universally experienced stressors of higher education. Negative repercussions of microaggressions include increased drop out or transfer rates, distress, fatigue resulting in decreased academic performance, and depression and posttraumatic stress symptoms. Expressive writing (EW) may be a scalable intervention for addressing the negative repercussions resulting from microaggressions experienced by minoritized students at PWIs. Previous research suggests that EW for stressful life events results in benefits such as reduced depression and posttraumatic stress symptoms, improved coping strategies, and reduced activity restriction. Despite such benefits, EW was not designed to specifically address microaggressions in a minoritized student population. Informed by the ADAPT-ITT model, our research group conducted a pilot study with similar procedures. This pilot study demonstrated the acceptability of an adapted version of the EW intervention titled Writing Wrongs (WW), as well as recommended future modifications for WW. In the current study we aim to conduct a randomized-controlled trial to establish the efficacy of WW in alleviating clinical symptoms. We hypothesize that WW will improve symptoms of racial and discriminatory trauma and symptoms of depression, anxiety, and posttraumatic stress over time and compared to an assessment-only condition. We will conduct exploratory analyses to examine short-term changes in affect within and across sessions and across conditions. We will recruit minoritized students enrolled at a PWI. Participants will complete a pre-intervention assessment prior to being randomized into the two conditions. Participants in the intervention condition will engage in three sessions of WW and complete measures of clinical symptoms across multiple time points (i.e., pre-intervention, immediately after the final writing session, one week after the final session). Participants in the assessment-only condition will be administered the same measures at the same timepoints and given access to the WW after completing the study. If found to be efficacious, WW has the potential to be widely disseminated to minoritized college students who experience microaggressions.
This study aims to compare symptom provocation, physiologic response, and rate of perceived (RPE) between the Buffalo Concussion Treadmill Test (BCTT) and a Modified Dynamic Exertion (mEXiT) test after sport related concussion in adolescents aged 14-17. Participants will be enrolled at their initial concussion clinical visit within 14 days of their injury, and randomly assigned to either the BCTT or mEXiT group. Participants will complete a single visit.