Kornimate/medical-research-clean huggingface dataset:
a study to evaluate the safety, pharmacokinetics, and pharmacodynamics of ivacaftor in subjects with cystic fibrosis who are less than 24 months of age and have an ivacaftor-responsive cftr mutation

Conditions: Cystic Fibrosis

Summary: the purpose of this study was to evaluate the safety of ivacaftor treatment, and pk of ivacaftor and metabolites in participants with cystic fibrosis (cf) who are \<24 months of age at treatment initiation and have an ivacaftor-responsive cf transmembrane conductance regulator (cftr) gene mutation.


Eligibility criteria:
inclusion criteria: * confirmed diagnosis of cf by sweat chloride value or cf mutation criteria. * have 1 of the following 10 cftr mutations on at least 1 allele: g551d, g178r, s549n, s549r, g551s, g1244e, s1251n, s1255p, g1349d or r117h (eligible in regions where ivacaftor is approved for use). part a/b group may also have other ivacaftor-responsive mutations. * hematology, serum chemistry, and vital signs results at screening with no clinically significant abnormalities that would interfere with the study assessments, as judged by the investigator. exclusion criteria: * history of any illness or condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the participant * colonization with organisms associated with a more rapid decline in pulmonary status at screening (only for parts a and b) * history of abnormal liver function or abnormal liver function at screening * history of solid organ or hematological transplantation * use of any moderate or strong inducers or inhibitors of cytochrome p450 (cyp) 3a within 2 weeks before day 1 * participation in a clinical study involving administration of either an investigational or a marketed drug within 30 days or 5 terminal half-lives before screening * hemoglobin (hgb) \<9.5 g/dl at screening * chronic kidney disease of stage 3 or above * presence of a non-congenital or progressive lens opacity or cataract at screening other protocol defined inclusion/exclusion criteria may apply.
active thoracic compromised distal landing in tevar

Conditions: Thoracic Aortic Aneurysm; Aortic Diseases

Summary: thoracic endovascular aortic repair (tevar) has become the mainstay of aortic intervention for descending thoracic aortic pathology. its durability hinges on achieving adequate seal in the endograft landing zones (lzs), with the distal lz largely understudied. only recently are suboptimal distal lzs receiving attention for their role in major complications including stent graft migration, type 1b endoleaks, and distal stent graft-induced new entry tears, all of which can contribute to further aortic degeneration. the thoracic distal lz. when a short distal lz was reported, the distal endoleak rate ranged from 3.5% to 33%. this led to reintervention in 19%, along with coverage of the celiac trunk in more than half of short lz cases. the objective of this study was to evaluate the outcomes of tevar patients with compromised distal landing zone (cdlzs) treated with distal active fixation stent-grafts (dafs), distal scallop stent-grafts, distal aptus heli-fx endoanchors, and standard stent-grafts.


Eligibility criteria:
inclusion criteria: * patients with descending thoracic aortic aneurysms (dtaas) with a maximal aortic diameter \> 55mm or rapid growth (\>10mm / year). * patients presenting with at least 2 cognizable features related to an adverse aortic morphology will be considered for inclusion. the following features will be evaluated for the inclusion: distal lz length ≤25-mm, distal lz diameter ≥38-mm, lz conical morphology, distal descending thoracic aorta angulation ≤116° or tortuosity index ≥1.4 exclusion criteria: * emergency setting, including hemodynamic instability at time of enrollment * patients with aortic dissection pathology
non pharmacological management of behavioural symptoms in nursing home

Conditions: ALZHEIMER Disease

Summary: background: behavioural and psychological symptoms of dementia (bpsd) are often reported in institutions for the elderly. objective: to evaluate the effectiveness of a staff education intervention to manage bpsd in older people with a diagnosis of dementia.

background: behavioural and psychological symptoms of dementia (bpsd) are often reported in institutions for the elderly. objective: to evaluate the effectiveness of a staff education intervention to manage bpsd in older people with a diagnosis of dementia. methods: the trial was conducted in 16 nursing homes; 306 patients with a diagnosis of dementia and presenting bpsd were selected. nursing homes were randomly allocated to an intervention group or a control group. an eight-week staff education and training programme was conducted in the nursing homes in the intervention group. the main outcome measures were the cohen-mansfield agitation inventory (cmai) and an observation scale score. assessments were done at baseline (w0), at the end of the 'intervention' period (w8) and 12 weeks after (w20).

Eligibility criteria:
inclusion criteria: * alzheimer disease exclusion criteria: * not alzheimer disease
a study of dengue vaccine in healthy children, teenagers and adults in india

Conditions: Healthy Volunteers

Summary: the main aims of the study are to learn about side effects and a participant's immune response to takeda's dengue vaccine when given twice within 3 months. participants will receive 2 doses of their randomized treatment (vaccine or placebo). children, teenagers and adults will receive one dose of either the vaccine or placebo on day 1 and the second dose of either the vaccine or placebo 3 months later. up to 4 blood samples will be taken throughout the study. during the study, participants will visit their study clinic 6 times.

the vaccine being tested in this study is called tdv (live, attenuated). tdv is being tested to prevent dengue. this study will assess the safety and immunogenicity of tdv in healthy participants. the study will enroll approximately 480 patients. participants will be randomly assigned (by chance, like flipping a coin) to receive either tdv or placebo- which will remain undisclosed to the participant, and investigator during the study: * cohort 1, ≥18 to ≤60 age group: tdv * cohort 1, ≥18 to ≤60 age group: placebo * cohort 2, ≥4 to \<18 age group: tdv * cohort 2, ≥4 to \<18 age group: placebo this multi-center trial will be conducted in india. the overall duration of the study is approximately 9 months.

Eligibility criteria:
key inclusion criteria: 1. participants who can comply with trial procedures and are available for the duration of follow-up. key exclusion criteria: at screening and at vaccination: 1. a body mass index (bmi) ≥35 kg/m\^2. 2. intent to participate in another clinical trial at any time during the conduct of this trial. 3. plans to receive any of the following: 1. a licensed vaccine within 14 days (for inactivated vaccines) or 28 days (for live vaccines) prior to tdv or placebo administration. 2. a coronavirus vaccine within 14 days prior to tdv or placebo administration. 3. a vaccine authorized for emergency use within 28 days of tdv or placebo administration. 4. known substance or alcohol abuse within the past 2 years that may interfere with his/her ability to comply with requirements for trial participation. 5. receipt of previous vaccination against dengue virus. 6. previous participation in any clinical trial of a dengue candidate vaccine. at vaccination: 1. participants with febrile illness or moderate or severe acute illness, or infection, at the time of random assignment. 2. participants medicated with antipyretic and/or analgesic medication(s) within 24 hours prior to tdv or placebo administration. note: other protocol defined inclusion/exclusion criteria may apply.

Keywords: drug therapy
effect of an integrated care pathway on asthma care in hospital

Conditions: Asthma

Summary: integrated care pathways (icp) coalesce medical and nursing work practices, unifying and directing care in line with current best practice and guidelines. we wish to examine the introduction of an icp for children with wheeze/asthma admitted to our hospital. we will determine whether more rigid, but guided care results in faster recovery, quicker discharge with better education and fewer prescribing errors, but also determine whether staff feel alienated by the rigidity of practice, and parents perceive any benefit to the changes introduced.this will be a cluster randomised trial (random weeks) of standard or icp care in 180 patients admitted to sick kids with wheeze/asthma. parents will be asked to complete a questionnaire on admission and contacted at 10-14 days post discharge and asked to recall education provided at discharge; no other patient or parent intervention will be required.it is expected to take 40 weeks to recruit 180 patients.

children admitted to hospital with wheeze/asthma are provided with care by a large number of clinicians with varying levels of skills and experiences. providing a uniform, safe level of care, according to evidence based guidelines is challenging given the levels of experience within each hospital. integrated care pathways are a multidisciplinary document incorporating medical, nursing, pharmacy and observational charts within a single chronological document. the study is to assess whether such documents can speed recovery and discharge, with care adhering more closely to evidence based guidelines for treatment and education. in addition we wish to assess patient safety (through prescribing errors) and parental and staff attitudes to the icp introduction. children arriving at the emergency department will be provided with care by either intergrated care pathway or standard care in 7 day clusters, randomised in 8 week blocks. comparison: we will compare the time from arrival at hospital to discharge from the ward as our primary outcome. other comparisons to be made for: speed of recovery of heart rate and respiratory rate time to require no supplemental oxygen time to achieve 4 hourly spacing of bronchodilator number and degree of prescribing errors provision of education to parents provision of care adhering to asthma guidelines (accurate discharge dose of prednisolone from emergency dept, use of multidose salbutamol rather than nebuliser) number of clinical contacts required with patients staff opinion of icp before and after introduction parental opinion of care in different groups parental recall of advice/education provided to them during admission possible hawthorne effect during period of the study

Eligibility criteria:
inclusion criteria: * children aged 2-16 years (inclusive) arriving at the emergency department with acute wheeze/asthma exclusion criteria: * children requiring high dependency care * children with significant neurological or cardiac disease

Keywords: asthma children integrated care pathway evidence based medicine prescribing errors
pulmonary substudy: a substudy of strategic timing of antiretroviral treatment (start)

Conditions: Chronic Obstructive Pulmonary Disease; HIV

Summary: the purpose of this study is to find out if starting anti-retroviral therapy (art) above 500 cluster-of-differentiation-4 (cd4)+ cells/milliliter (ml) ('early art group') slows the rate of decrease in lung function over time compared to waiting to start art until the cd4+ drops below 350 cells/ml ('deferred art group'). lung function normally declines with age, and both human immunodeficiency virus (hiv) infection and art have been shown to case a decline in lung function as well. decline in lung function can be an early indicator of chronic obstructive pulmonary disease (copd), a significant cause of sickness and death in people with hiv. in this study, lung function will be measured at baseline and every year thereafter by using a spirometer.


Eligibility criteria:
inclusion criteria: * simultaneous co-enrollment in the start study * signed informed consent to the pulmonary substudy * age \>= 25 years exclusion criteria: * an episode of respiratory illness with 2 or more symptoms of cough, wheezing, breathlessness, or increase in sputum production within the 6 weeks before baseline spirometry. * use of asthma medications (bronchodilator, inhaled corticosteroid, leukotriene inhibitor, or theophylline) for 2 or more consecutive weeks within the 6 months before baseline spirometry. * relative contraindications to spirometry, such as chest or abdominal or eye surgery within the 3 months before baseline spirometry, known retinal detachment at the time of baseline spirometry. * known allergy to albuterol/salbutamol * relative contraindications to albuterol/salbutamol, such as resting heart rate of \>110 beats per minute, or a known serious or recurrent or uncontrolled cardiac condition (such as unstable coronary artery disease, decompensated heart failure, or recurrent tachyarrhythmias).

Keywords: hiv copd fev1 respiratory function
rct study on the therapeutic effect of escitalopram loaded with xingpijieyu formula on resting state eeg energy abnormalities in severe liver depression and spleen deficiency type mdd

Conditions: Major Depression

Summary: to compare the clinical efficacy of xingpijieyu formula or its placebo combined with escitalopram (esc) in the treatment of moderate and severe mdd, and to explore a new method of integrated traditional chinese and western medicine for the treatment of moderate and severe mdd with severe liver depression and spleen deficiency, as well as its neurological mechanism for regulating the relative power (rp) of resting-state eeg spectra.

mdd patients, especially severe mdd patients have heavy burden of disease, high risk of suicide, difficulty in single drug treatment, and poor compliance with combined western medicine treatment. it is urgent to explore new methods of integrated traditional chinese and western medicine in the treatment of moderate and severe mdd to enhance efficacy and reduce toxicity. a double-blind, randomized, controlled trial (rct) was conducted to compare the efficacy of xingpijieyu formula or its placebo combined with escitalopram in the treatment of mdd with severe liver depression and spleen deficiency. we aim to explore the clinical efficacy of xingpijieyu formula combined with escitalopram and its possible multi-target neural mechanism.

Eligibility criteria:
inclusion criteria: 1. outpatient/inpatient, age 18-45 years old, right-handed; 2. western medical diagnosis meets the diagnostic criteria of mdd in dsm-5, without psychiatric features; 3. in accordance with the diagnostic criteria of depression and liver-stagnation and spleen-deficiency in the "guidelines for diagnosis and treatment of internal diseases in traditional chinese medicine" issued by the china association of chinese medicine in 2008; 4. 17-item hamilton depression scale score ≥18; 5. educational experience ≥5 years; 6. the current episode has not used antidepressants, antipsychotics, mood stabilizers, or other medications that can act on the central nervous system for at least 4 weeks; 7. have not received physical therapy, such as mect, rtms, etc., in the past 6 months; 8. the total duration of the disease ≤10 years. exclusion criteria: 1. any current or previous mental disorder other than mdd according to dsm-5; 2. diagnosis of syndromes other than depression and liver-stagnation and spleen-deficiency in the "guidelines for diagnosis and treatment of internal diseases in traditional chinese medicine" issued by the china association of chinese medicine in 2008; 3. suicidal ideation or suicidal behaviour (hamd-17 item 3 \> 2); 4. drinking alcohol in the week prior to the enrollment; 5. patients with neurological disorders, somatic disorders, organic brain diseases, and tumour; 6. sleep disorders other than insomnia disorders currently diagnosed according to dsm-5, such as obstructive sleep apnea syndrome, periodic limb movement disorder, restless legs syndrome, etc.

Keywords: depression xingpijieyu formula escitalopram relative energy electroencephalogram
study of the thyroid function and echostructural morphology in patients affected with rasopathies (ecoras2023)

Conditions: RASopathy

Summary: the study aims to evaluate the prevalence of thyroid disease, particularly with autoimmune pathogenesis (isolated hyperthyrotropinemia, hyperthyroidism, hypothyroidism, thyroid nodules) and/or morphostructural abnormalities of the thyroid gland in patients with rasopathy genetically confirmed by ngs technique (analysis of the genes: braf, cbl, hras, kras, lztr1, map2k1, map2k2, mras, nras, ppp1cb, ptpn11, raf1, rit1, rras2, shoc2, sos1, sos2) and to compare the data obtained in our sample with those of the general population. the secondary aim of the study is to evaluate the association between vitamin d deficiency and/or other abnormalities of bone metabolism and thyroid disease and/or morphostructural anomalies of the thyroid gland in patients with rasopathy.

all patients will undergo blood sampling aimed at studying thyroid function and autoimmunity (tsh, ft3, ft4, anti-tpo, anti-tg, anti-tsh receptor antibodies) and biomarkers of bone metabolism (calcium, phosphorus, alp isoenzyme bone, pth, osteocalcin, vitamin d) and a systematic color doppler ultrasound examination of the thyroid gland in order to evaluate dimensions, echostructure and echogenicity of the thyroid parenchyma, presence of nodular lesions, vascularization and laterocervical lymphadenopathy.

Eligibility criteria:
inclusion criteria: * genetically confirmed rasopathy * age 3-25 years exclusion criteria: * previous radiotherapy treatments, known exposure to ionizing radiation * iodine deficiency * use of iodine-based compounds or drugs that interfere with thyroid function * congenital hypothyroidism/hyperthyroidism * severe obesity with metabolic complications
european registry of dialysis treatment of pediatric acute kidney injury (aki)

Conditions: Acute Kidney Injury

Summary: acute kidney injury (aki) is a frequent clinical condition in hospitalized, in particular, in critically ill children. moreover, aki is an independent predictor of mortality. an incidence of aki in pediatric intensive care units (picu) between 10 and 62% has been reported in recent clinical trials adopting prifle or akin criteria, with the highest risk present in cardiac surgery patients. despite significant developments in the management of aki, the overall mortality rate of patients with aki has not improved significantly. currently, there is no consensus concerning the optimum dialysis modalities to adopt in pediatric aki. no studies have prospectively compared the efficacy of different types of rrt for pediatric aki. while pd remains the most commonly used modality in children worldwide, over the last decade crrt has become the preferred treatment modality for critically ill children with aki in north america. the investigators have recently conducted a survey among 34 european pediatric nephrology centers in the escape network to obtain current information on dialysis management practices in children. approximately 900 children with aki requiring dialysis are managed at these 34 centers per year. this number supports the creation of a prospective european aki registry.

the main scope of the registry is to report the epidemiology and outcome of children with aki treated with dialysis in over 30 pediatric nephrology centers in europe. secondary aims are to verify the association of a specific dialysis modality with the outcome and the association of primary disease, co-morbidities, nephrotoxic agents, fluid overload, anuria, basic hemodynamic parameters (bp, hr), basic nutritional intakes (protein and calorie supply) with the outcome. data capture will be exclusively web-based via electronic case report forms. every participating site will be provided with a unique code and password that identify the corresponding site.

Eligibility criteria:
inclusion criteria: * children with aki at hospital admission or who developed aki during hospitalization treated with dialysis (pd, hd, crrt) * age: 0-18 yrs exclusion criteria: * children with known preexisting ckd (i.e.: aki on ckd)

Keywords: paediatric kidney
treatment of multiple system atrophy using intravenous immunoglobulins

Conditions: Multiple System Atrophy

Summary: multiple system atrophy (msa) is a progressive sporadic neurodegenerative disorder leading to widespread loss of brain cells that results in parkinsonian, cerebellar and autonomic dysfunction. the cause of the msa remains unclear. available treatment is symptomatic only and does not alter the course of disease. although the cause of msa remains unclear, there is evidence of presence of common neuroinflammatory mechanisms in the msa brains including activation of microglia and production of toxic cytokines. this research protocol is based on hypothesis that the msa progression can be altered by blocking the neuroinflammatory activity. this protocol includes administration of intravenous immunoglobulin (ivig). ivig contains antibodies derived from human plasma which can block the inflammatory responses in the brain that can lead to loss of brain cells.


Eligibility criteria:
inclusion criteria: 1. male or female older than 17 years. 2. clinical diagnosis of probable multiple system atrophy 3. provide written informed consent to participate in the study 4. understand that they may withdraw their consent at any time exclusion criteria: 1. women who are pregnant or lactating 2. in the investigator's opinion, have any other significant systemic, hepatic, cardiac or renal illness. 3. in the investigator's opinion, the subjects are significantly dehydrated, as determined by clinical evaluation including measurement of skin turgor, blood urea nitrogen and creatinine values.

Keywords: multiple system atrophy, msa, intravenous immunoglobulin, ivig
comparison of prasugrel and ticagrelor in the treatment of acute myocardial infarction

Conditions: Myocardial Infarction; Angioplasty, Balloon, Coronary; Platelet Aggregation Inhibitors

Summary: this study evaluates the efficacy of prasugrel and ticagrelor in the treatment of acute myocardial infarction.

study objectives: 1. compare the efficacy and safety of prasugrel and ticagrelor in acute myocardial infarction treated with emergent pci. 2. assess the safety of switching to clopidogrel after remission of the acute phase of mi in patients for whom economic barriers do not allow to continue treatment with prasugrel or ticagrelor. all randomized patients with acute myocardial infarction have been treated with standard therapeutic procedures in accordance with the guidelines of european society of cardiology (esc). participation of patients in the study is not connected to any deviations from the esc guidelines recommendations.

Eligibility criteria:
inclusion criteria: 1. acute myocardial infarction (\> 1mm st elevation in at least 2 related leads or st depression \> 2mm in 3 leads or new bbb) with an indication to emergent (within 120 minut from admission to the pci center) coronary angiography and pci, 2. signed informed consent. exclusion criteria: 1. history of stroke, 2. serious bleeding within last 6 months, 3. indication to an oral anticoagulation (e.g. atrial fibrillation, artificial valve, thromboembolism etc...) 4. use of ≥ 300 mg of clopidogrel or another antiplatelet agent (except of aspirin and lower dose of clopidogrel) before randomization, 5. low body weight (\ 75 years of age), 6. moderate or severe liver dysfunction, 7. ongoing therapy with a strong cyp3a4 inhibitor (e.g. ketoconazole, clarythromycine, nefazodone, ritonavir, atazanavit), 8. hypersensitivity to prasugrel or ticagrelor.

Keywords: acute myocardial infarction primary coronary intervention antiplatelet therapy