Kornimate/medical-research-clean huggingface dataset:
metformin in children with fragile x syndrome

Conditions: Fragile X Syndrome; Metformin

Summary: this study is a controlled trial of metformin in children with fragile x syndrome(fxs). the age of fxs children range from 2 to 16 years old. participants will be randomized in a double-blind design to either drug or placebo for 6-month period. the primary objectives are to assess metformin in treatment of behavior problems, cognitive and language with fragile x syndrome.

this is a single-center study at the children's hospital of fudan university for fxs patients aged 2 to 16 years inclusive. it is a randomized, double-blind, placebo-controlled trial of metformin. studies showed that there were pathogenically over activates of mtor and mapk/erk pathways in fxs. metformin, a guanidine derivative ,has been shown to reduce mtorc1 pathway activity in an ampk-dependent manner and has also been shown to reduce mapk pathway activity. metformin treatments for fxs have been reported in animal experiments, some open label trials of metformin in fxs patients had been reported. therefore, metformin has potential to rescue symptoms in children with fxs. in this study, researchers hope to investigate the improvement effect of metformin on fxs symptoms such as behavior problems, cognition, language. the intervention period is 6 months, follow-up visit at 1 year. the researchers will also assess the side effects of the study medication throughout the trial.

Eligibility criteria:
inclusion criteria: * genetic testing confirms the diagnosis of fxs * participate in the study with the informed consent of the guardian * bmi\>the 3rd percentile * not taking more than 2 therapeutic drugs * able to receive regular follow-up visits exclusion criteria: * malnutrition * primary heart disease * severe infection or acute clinical illness * gastrointestinal, renal, or hepatic disease * previous history of lactic acidosis * previous use of metformin intolerant * use of angiotensin converting enzyme inhibitors, use of anticoagulants, vitamin b12 deficiency, alcohol consumption * unstable systemic diseases other than fxs * changes in clinical medication
effect of early hydrocortisone on risk of gastrointestinal perforations in extremely preterm infants

Conditions: Intestinal Perforation

Summary: a large, randomised control trial, the premiloc trial, has established that giving low dose hydrocortisone prophylactically in the first ten days of life reduces the risk of bronchopulmonary dysplasia in babies born before 32 weeks' gestation. however, the premiloc trial was underpowered to investigate rarer side effects, such as gastrointestinal perforation. this study aims to establish whether the odds of gastrointestinal perforation increase when extremely preterm infants are given prophylactic hydrocortisone in the first ten days of life. this retrospective cohort study will use routinely collected data from the u.k. national neonatal research database. the investigators will examine the records of all infants born before 28 weeks' gestation and cared for in english and welsh neonatal units between 2016 and 2023. infants will be considered exposed if they received hydrocortisone for at least eight consecutive days, beginning on postnatal day 1 or 2. the primary outcome will be gastrointestinal perforation, as recorded in the infant's neonatal unit record. this outcome will be validated with the original care teams for a sample of babies. data will be analysed using a propensity score matched approach to reduce the impact of confounding.


Eligibility criteria:
inclusion criteria: infants who: * were admitted to a neonatal unit between the 1st january 2016 and 31st march 2023 and * received any of their care in a nhs neonatal unit in england and wales (part of uk neonatal collaborative and therefore contributing data to the nnrd) and * were born before 28 weeks' gestation. exclusion criteria: * they have missing data for principal background variables (gestational age at birth, birth weight, year of birth and date of death for those that died). * their recorded birthweight absolute value z score exceeds 4 or is missing. * they died on postnatal day 1 or 2 .

Keywords: preterm infant hydrocortisone
norwegian study on district treatment of st-elevation myocardial infarction

Conditions: Acute Myocardial Infarction

Summary: in the district areas of norway patients with acute myocardial infarction with st-elevation, are treated with thrombolysis. an increasing part of them receives thrombolysis before arrival to the local hospital.usually these patients have been sent to an invasive center if thrombolysis fails or the patient gets ischemic symptoms during the stay. this study will compare this strategy against immediate transportation to an invasive center after the patient has received thrombolysis.


Eligibility criteria:
inclusion criteria: * duration of painless than 6 hours. * st-segment elevation of at least 0.1 mv in two or more extremity leads or at least 0.2 mv in two or more precordial leads. * the patient is getting thrombolysis. * time to reach an invasive center is more than 1 hour. * age 18-75 years. exclusion criteria: * known serious renal failure (creatinin \> 250 mmol/l) * pregnancy * cardiogenic chock * life threatening arrythmias * other serious diseases with life expectancy less than 1 year. * inability to perform an informed consent

Keywords: acute myocardial infarction fibrinolysis health effects /quality of life
improving the assessment of hypersomnolence

Conditions: Disorders of Excessive Somnolence

Summary: the overall purpose of this study is to employ a pragmatic, multidimensional assessment of hypersomnolence that "piggybacks" on routine clinical care in patients with suspected disorders of central nervous system (cns) hypersomnia, to determine whether these additional objective and subjective assessments are useful in the diagnosis and management of these patients.


Eligibility criteria:
inclusion criteria: * age 18-89 * referred for polysomnography/multiple sleep latency test at wisconsin sleep, the sleep medicine clinic and laboratory of the university of wisconsin-madison exclusion criteria: * unstable medical condition that, in the opinion of the investigators, would make it unsafe for the subject to participate
role of ct perfusion in predicting poor outcome after subarachnoid hemorrhage

Conditions: Subarachnoid Hemorrhage; Cerebral Vasospasm; Delayed Cerebral Ischemia

Summary: prospective evaluation of patients with subarachnoid hemorrhage (sah) will be done by computed tomography angiography (cta) and perfusion imaging (ctp) for any correlation between degree of vasospasm and perfusion deficit as well as evaluating the ability of ctp to predict delayed cerebral ischemia.

cerebral vasospasm is a serious complication of subarachnoid haemorrhage . in the first 2 weeks of sah, angiographic vasospasm is seen up to 40-70% of patients and causes ischemic deficits in 15-36% of patients. the best clinical indicator of significantly reduced brain perfusion (cerebral blood flow (cbf)\<20 ml per 100 g/min) is the presence of new neurologic deficits. however, clinical symptoms may be vague and mimic other conditions in patients with sah. ct perfusion can be used in the evaluation of patients with possible vasospasm after subarachnoid hemorrhage (sah). it can thus be used to assess cerebral ischemia and infarction as a result of vasospasm after sah. the presence of cerebral vasospasm identified with transcranial doppler, digital subtraction angiography, or ct angiography (cta) is frequently used to confirm dci. presence of vasospasm, however, does not prove the presence of ischemia and absence of vasospasm does not rule out. better diagnostic tests in the acute stage of deterioration, possibly caused by dci, are therefore needed. in patients with sah, ctp has recently been shown to be promising for detection of early ischemia.

Eligibility criteria:
inclusion criteria: * all patients presented with subarachnoid haemorrhage exclusion criteria: * patients with abnormal renal functions with creatinine ≥ 2 mg/dl/ * patients with hypersensitivity to contrast media. * contraindication to radiation as pregnancy.

Keywords: ct perfusion subarachnoid hemorrhage vasospasm delayed cerebral ischemia
hanyang university medical center arthritis network

Conditions: Arthritis, Rheumatoid

Summary: rheumatoid arthritis is a relatively common autoimmune disease that affects about 1% of the population. its impact on health-related quality of life of the affected patients and the national medical cost are enormous. however, a systemic prospective cohort study of rheumatoid arthritis has not yet been established in korea, so data on characteristics and prognosis of korean rheumatoid arthritis and their response to therapy and adverse events are limited. therefore establishment of large prospective cohort of korean rheumatoid arthritis is imperative. with inauguration of a steadfast prospective cohort of korean rheumatoid arthritis patients in the hospital for rheumatic diseases at hanyang university, where patients gather from all over korea forming the largest and most diverse patient pool in korea, we will be able to define the characteristics and prognosis of korean rheumatoid arthritis patients and gather information on treatment response and adverse events. this will eventually lead to improvement of health-related quality of life in korean rheumatoid arthritis patients and provide opportunity for reliable comparison study with research institutes from other countries. the data gathered through the cohort will provide valuable foundational information for the genetic studies, pharmacological economic analyses, and standard treatment guidelines of korean rheumatoid arthritis patients.

this study is a longitudinal prospective cohort of korean rheumatoid arthritis patients comprising of all korean adult rheumatoid arthritis patients satisfying the 1987 revised american college of rheumatology (acr) criteria and voluntarily agreeing to participate in the study with following objectives; primary objectives: 1. gather information on treatment response and adverse events of various pharmacological therapies for treatment of korean rheumatoid arthritis patients 2. identify the incidence and mortality rates of concurrent diseases including the cardiovascular \& gastrointestinal diseases and assess the contributing factors involved secondary objectives: enrich understanding of disease characteristics, treatment response and adverse events in korean rheumatoid arthritis to promote health and increase the health-related quality of life in korean rheumatoid arthritis patients

Eligibility criteria:
inclusion criteria: * korean adult rheumatoid arthritis patients satisfying the 1987 revised american college of rheumatology (acr) criteria

Keywords: arthritis, rheumatoid cohort studies
descartes-11 in patients with relapsed/refractory multiple myeloma

Conditions: Multiple Myeloma

Summary: this phase i study will test the safety and anti-myeloma activity of ascending doses of descartes-11 (autologous cd8+ t-cells expressing an anti-bcma chimeric antigen receptor) in eligible patients with active multiple myeloma.


Eligibility criteria:
inclusion criteria: * active multiple myeloma that is refractory after at least 2 prior lines of therapy; * measurable disease; * adequate vital organ function; and * no active infection.

Keywords: car-t cart car t cell car t-cell multiple myeloma bcma b cell maturation antigen b-cell maturation antigen descartes-11
prognostic biomarkers in co poisoning

Conditions: Carbon Monoxide Poisoning; Hyperbaric Oxygenation; Biomarkers

Summary: mitochondrial and oxidative stress participate in the pathogenic mechanisms of carbon monoxide (co)-induced toxicity. thus, serum indicators of mitochondrial and oxidative stress could be useful for predicting neurocognitive prognosis of post-co poisoning. this prospective observational study of consecutive patients requiring hyperbaric oxygen therapy (hbo2) for acute co poisoning measured serum biomarkers of mitochondrial (growth differentiation factor 15 \[gdf15\]; fibroblast growth factor 21 \[fgf21\]) and oxidative (8-oxo-2'-deoxyguanosine \[8-ohdg\] and malondialdehyde \[mda\]) stresses at arrival at the emergency department (0 h), and at 24 h and 7 days after hbo2 completion. we evaluated neurocognitive outcomes using the global deterioration scale (gds; favorable \[1-3 points\] or poor \[4-7 points\] outcomes).

mitochondrial and oxidative stress participate in the pathogenic mechanisms of carbon monoxide (co)-induced toxicity. thus, serum indicators of mitochondrial and oxidative stress could be useful for predicting neurocognitive prognosis of post-co poisoning. this prospective observational study of consecutive patients requiring hyperbaric oxygen therapy (hbo2) for acute co poisoning measured serum biomarkers of mitochondrial (growth differentiation factor 15 \[gdf15\]; fibroblast growth factor 21 \[fgf21\]) and oxidative (8-oxo-2'-deoxyguanosine \[8-ohdg\] and malondialdehyde \[mda\]) stresses at arrival at the emergency department (0 h), and at 24 h and 7 days after hbo2 completion. we evaluated neurocognitive outcomes using the global deterioration scale (gds; favorable \[1-3 points\] or poor \[4-7 points\] outcomes). levels of serum mitochondrial (gdf15 and fgf21) and oxidative stress (8-ohdg and mda) biomarkers pre- and post-hbo2 were compared, to assess whether hbo2 reduced stress. in addition, we evaluated whether the blood test results correlated with the patient's neurocognitive prognosis at 1 month.

Eligibility criteria:
inclusion criteria: * co poisoned patients exclusion criteria: 1. age \< 19 years 2. a history of metabolic diseases 3. co-ingestion of drugs and alcohol, which may affect the biomarkers 4. discharge from the ed or transfer to another hospital 5. refusal to undergo hbo2 6. refusal to enroll in this study 7. lack of follow-up for assessing neurocognitive outcomes.
safety, tolerability, and efficacy study of ubx1325 in patients with neovascular age-related macular degeneration (envision)

Conditions: Neovascular Age-related Macular Degeneration

Summary: this study is intended to assess safety, tolerability and biological activity of a repeat ivt injection of ubx1325 in patients with wet amd.

this is a phase 2 study. during screening, every patient will receive a run-in injection of aflibercept. on day 1, patients will be randomized to either the ubx1325 arm or the aflibercept arm. patients randomized to the ubx1325 arm will receive ubx1325 on day 1, weeks 4, 24, and 28; and sham procedure on weeks 8, 16, 24, 32, and 40. patients randomized to the active-control aflibercept arm will receive aflibercept per label every 8 weeks starting from day 1 until week 40. in addition, these patients will receive ubx1325 on week 24 and week 32; and a sham procedure at week 4 and week 28.

Eligibility criteria:
inclusion criteria: * patients aged ≥50 years. * active cnv associated with age-related macular degeneration as evidenced on fa and sd-oct with presence of intraretinal or subretinal fluid at screening and day 1 * bcva in the study eye (most affected) of 70 to 20 etdrs letters (equivalent to 20/40 to 20/400 on the snellen chart) at screening. * patients who have the capacity to give informed consent and who are willing and able to comply with all study-related procedures and assessments. exclusion criteria: * concurrent disease in the study eye or structural damage, other than wet amd, that could compromise bcva, prevent bcva improvement, require medical or surgical intervention during the study period, confound interpretation of the results, or interfere with assessment of toxicity or cfp in the study eye. * any ocular/intraocular/periocular infection or inflammation in either eye in the past 12 weeks prior to screening * subretinal hemorrhage with bleeding area of 4 or greater disc area in the study eye * history of vitreous hemorrhage in the study eye within 2 months prior to screening * any condition, including laboratory findings and findings in the medical history or in the pre-study assessments, that, in the opinion of the investigator, constitutes a risk or contraindication for participation in the study or that could interfere with the study objectives, conduct, or evaluation or prevent the patient from fully participating in all aspects of the study * significant media opacities, including cataract, which might interfere with va, assessment of toxicity, or fundus imaging

Keywords: wet amd
testing the utilization of a mobile health app for patients undergoing cystectomy surgery for bladder cancer

Conditions: Invasive Bladder Cancer

Summary: this is a pilot study that involves human subjects undergoing cystectomy surgery for bladder cancer, testing the utilization of a mobile health app that promotes postoperative patient engagement, along with provider monitoring and care. the aim of this pilot study is to assess feasibility and acceptability of an internet-based mobile health tool following cystectomy discharge. objectives will be met by assessing weekly adherence, determining acceptability of mobile health questions post-surgery by patients and providers, and by obtaining participant feedback regarding the tool. complications, re-admissions, and resource utilization will also be monitored throughout the study. the hypothesis is that an mobile health intervention that tracks pros, provides educational content, and allows real-time feedback via internet-enabled devices has the potential to improve the quality of care delivery and overall patient experience following surgery by decreasing resource utilization and improving symptom control and communication.

radical cystectomy will be used as a model to examine the utilization of an electronic, internet-based mobile health intervention that can prospectively collect pro data while providing timely feedback to patients and clinicians. this mobile health intervention will utilize a well-established platform (twistle) that can be accessed through multiple technologies, including laptops, smartphones, and other computer-based systems. the objective is to evaluate the feasibility of pre-specified patient reported outcomes and educational content in an existing mobile health communications platform which tracks patient symptoms and provides real-time feedback to patients and providers following cystectomy discharge. patient reported outcome questions and responses will be inputted using validated questions with branching logic to drive feedback based upon responses. the intent of this study is to evaluate feasibility and acceptability of this mobile health tool following cystectomy discharge. usual care will continue in coordination with this intervention.

Eligibility criteria:
minimum age: 18 years maximum age: 99 years sex: all gender based: no criteria: inclusion criteria: * male or female patients with invasive bladder cancer scheduled for radical cystectomy at unc within the next 30 days * english-speaking * ability to access the internet (either themselves or with assistance from their caregiver) * ability to read * ability to provide informed consent. exclusion criteria: * inability to read * inability to provide informed consent * severe psychiatric illness * cystectomy surgery without cancer diagnosis * non-english speaking
testing interventions to reduce alcohol consumption among outpatients in a dental setting

Conditions: Alcohol Addiction; Mouth Diseases

Summary: the investigators wish to investigate the feasibility of using a web based drinking app (www.drinksmeter.com) to reduce alcohol consumption among patients attending an outpatient clinic in a dental setting.

any intervention on alcohol use is relevant to patients attending an oral medicine clinic, as alcohol can contribute to/or worsen mouth disorders. for example, research confirms that white and red patches of the lining of the mouth in regular tobacco and alcohol users can change to mouth cancer. many people do not know that drinking alcohol would increase their mouth cancer risk. any new or follow-up patients attending our clinic and reporting drinking over the recommended guidelines (as given by european code against cancer) will be eligible to participate. the study will start on the first visit to the clinic and/or at their follow-up visit. for each volunteer the study will last 3 months. participants will be allocated to two groups, those receiving routine brief counselling on the day of their visit and those to whom a web-based electronic tool to self-assess is introduced. they will be motivated to use it in the following weeks to reduce consumption . the study will evaluate alcohol consumption levels using tested questionnaires at base-line and exit. the investigators will assess the feasibility of the use of an electronic tool in our study population, whether it provided better outcomes compared to standard care.

Eligibility criteria:
inclusion criteria: * patient attending the department of oral medicine at king's college hospital and consuming alcohol over recommended guidance (european code against cancer) exclusion criteria: * patient which cannot understand english * complete lack of it literacy * patient in contact with a specialized alcohol service

Keywords: drinksmeter alcohol counselling
effect of 2-week continuous glucose monitoring on glycemic management in patients new-to-insulin on hospital discharge

Conditions: Diabetes

Summary: the proposed study will be a randomized, prospective, non-blinded study of 120 participants with type 1 or type 2 diabetes that are new-to-insulin on hospital discharge. on hospital discharge, participants will be assigned to either the intervention of wearing a continuous glucose monitor (cgm) for 2 weeks or blood glucose monitoring (bgm) for 2 weeks. they will have a 2-week follow up visit, during which insulin doses will be adjusted as needed, and a 3-month follow-up visit, at which point hba1c will be measured.


Eligibility criteria:
inclusion criteria: 1. male or female aged 18-100 years 2. known history of type 1 or type 2 diabetes 3. admitted to nyu langone hospital - long island between september 1st, 2022 and august 31st, 2023 4. new initiation of insulin therapy, including basal insulin regimen, basal-bolus insulin regimen, or mixed insulin regimen at the time of hospital discharge exclusion criteria: 1. prior to admission use of home insulin therapy 2. current use of systemic corticosteroids 3. active pregnancy; as pregnancy requires different blood glucose targets, subjects known to be pregnant will be excluded from this study. subjects will not be tested for pregnancy outside of testing performed in routine medical care; pregnancy will be determined by patient self-reporting. females of childbearing potential will not be instructed to avoid pregnancy, however if they became pregnant during the study (detected by self-reporting), they will be withdrawn from the study.

Keywords: continuous glucose monitoring (cgm)