Kornimate/medical-research-clean huggingface dataset:
muscle response to enzyme replacement therapy in pompe disease

Conditions: Pompe Disease

Summary: this study is to study the response of muscle cells from pompe disease after enzyme replacement therapy


Eligibility criteria:
inclusion criteria: * patients with pompe disease, prepare or on enzyme replacement therapy * signed informed consent exclusion criteria: -

Keywords: pompe disease muscle enzyme replacement therapy
treatment with quetiapine for youth with substance use disorders and severe mood dysregulation

Conditions: Substance Use Disorders; Bipolar Disorder; Mood Disorders

Summary: this study proposes to use quetiapine as an adjunct treatment to treatment as usual to improve both substance use disorder (sud) and mood symptoms in youth with sud and severe mood dysregulation (smd). this is a randomized, double blind placebo controlled parallel design study. youth with symptoms of mood dysregulation and active substance use that meets criteria for a sud will be randomized to adjunct treatment with quetiapine or placebo. the investigators hypothesize that treatment with quetiapine will lead to a reduction in substance use, improvement in mood, and lead to greater engagement in outpatient treatment.


Eligibility criteria:
inclusion criteria: * male or female age 15 to 24 * meet dsm-5 criteria for a substance use disorder * substance use ≥ 14 days of past 28 days (i.e. use ≥ 50% of days in the past 28 days) * if subject in restricted setting/care (e.g. inpatient detox unit or residential treatment) for ≤ 2 weeks, then use ≥ 50% of days while outside of restricted setting (e.g. 7 days of substance use out of 14 days in unrestricted setting) * subjects need to have been in an unrestricted setting for at least 2 weeks prior to screening * meets dsm-5 criteria for bipolar disorder or disruptive mood dysregulation disorder or dsm iv criteria for mood disorder not otherwise specified * symptoms of smd: youth self report (ysr) or adult self report (asr) aaa ≥180 * stable to be treated in outpatient level of care exclusion criteria: * current methamphetamine use disorder * current unstable opioid use disorder (i.e. \< 3 months on medication assisted treatment for an opioid use disorder) * pregnant or breastfeeding * placement in a restricted setting (e.g. detox or residential treatment) for ≥ 2 weeks out of past 28 days prior to screening visit, or placement in a restricted setting at anytime during study participation * unwilling or unable to use effective birth control * unwilling or unable to sign release of information for the treatment program providing behavioral treatment * for participants \>17 years-unable or unwilling to identify emergency contact

Keywords: youth adolescent young adult substance use disorder bipolar disorder quetiapine mood dysregulation
paclitaxel or s1 plus cisplatin in concurrent chemoradiotherapy for squamous cell carcinoma of esophagus

Conditions: Esophageal Squamous Cell Carcinoma

Summary: the purpose of this study is to determine which regimen is better for esophageal squamous carcinoma in concurrent chemoradiation(ccrt),paclitaxel or s1 plus cisplatin.

radiotherapy:patients will be conducted ct simulation, and three-dimensional conformal radiation therapy (3dcrt) was performed. 1.8-2.0 gy/fraction, 5 fractions a week, with a total dose of 60-66gy will be delivered for all patients by 6-mv-x-ray of linear accelerator. chemotherapy: patients will be concurrently administered with irradiation every 3 weeks with pt regimen (cisplatin of 20 mg/m2/d, d1-3; ptx(paclitaxel)of 135mg/m2/d, d1) for 4 cycles or ps regimen (cisplatin of 20 mg/m2/d, d1-3; s1(tegafur gimeracil oteracil potassium capsule)of 50mg/m2/d, d1-14) for 4 cycles. for the first 2-cycles of chemotherapy they will be concurrently given with irradiation and the remained 2 cycles, after irradiation.

Eligibility criteria:
inclusion criteria: 1. pathologically proven squamous cell carcinoma of esophagus . 2. locally advanced esophageal cancer , with no operation indication 3. m1a disease defined ( ajcc 1998) , including celiac lymph node for lower thoracic esophageal cancer and cervical lymph node involvement for upper thoracic esophageal cancer 4. subjects haven't been given neither radiotherapy nor chemotherapy before 5. age of 18-70 6. ps ≦2 7. hemogram : wbc≧ 4000/mm3 or anc ≧ 2000 /mm3 and platelet ≧ 100,000/mm3 . biochemistry : got/gpt ≦ 3.5 times , cr ≦ 1.5 mg/dl and bilirubin ≦ 2.0 mg/dl 8. with no difficulty in eating 9. expected lifetime ≧3 months exclusion criteria: 1. invasion to surrounding organ ( t4 disease ) . 2. distant metastasis , except m1a disease . 3. complete obstruction of the esophagus, or patients who have the potential to develop perforation 4. women in status of pregnancy 5. patients who have complications exist as following: * uncontrolled angina and heart failure, have a history of hospitalization in 3 months * a history of myocardial infarction in the past 6 months * there is a need for antibiotic treatment of acute bacterial or fungal infection * chronic obstructive pulmonary disease, or other lung disease requiring hospitalization * drug addiction, alcoholism and aids disease or long-term virus carriers * uncontrollable seizures, or loss of insight because of mental illness
localized muscle vibration post stroke

Conditions: Stroke

Summary: background and objectives: lower extremity functional impairments are common consequences of stroke. thus, continuous examination of effective treatment interventions for lower extremity functions after stroke is a necessity. localized muscle vibration (lmv) is one of the treatment interventions that incorporate sensory stimulation to improve motor cortical excitability. this study aimed to investigate the influences of 10-minutes lower extremity lmv application along with conventional physical therapy (cpt) on activities of daily living (adl) and motor recovery on the hemiparetic lower extremity post-stroke. methods: a sample of 37 patients with stroke (8 females) was randomly allocated to either conventional physical therapy (cpt) control group (n=18) or conventional physical therapy and localized muscle vibration (cpt+lmv) experimental group (n=19). all patients received 3 sessions per week of cpt for 8 weeks. the cpt+lmv experimental group received 10-minute lmv at the end of each cpt session. the outcome measures used were the barthel index (bi), berg balance scale (bbs), and goniometry for range of motion (rom) assessment.

all patients in both experimental and control groups received cpt protocol consisted of 30 minutes of manual resistance exercise (3 sets/10 repetitions each for knee extensors and flexors) and 30 minutes of treadmill training, short breaks (1-5 minutes) were allowed, depending on the patient tolerance. the training was conducted for 3 days per week for 8 weeks. the patients in cpt+lmv experimental group received localized muscle vibration at the end of each cpt session. the lmv was applied for 10 minutes perpendicularly along quadriceps muscle fibers. the frequency set at 30 hz, amplitude of 2 mm.

Eligibility criteria:
inclusion criteria: confirmed diagnosis of stroke exclusion criteria: if they had any serious orthopedic injury/disease, cardiopulmonary problems or suffered from a neurological disease (other than stroke).
use of ffr-ct in stable intermediate chest pain patients with severe coronary calcium score

Conditions: Observational

Summary: a prospective, blinded multicenter study for evaluation of chest pain patients with severe coronary calcium (agatston score \> 399). the objective is to evaluate if an initial non-invasive strategy with coronary computed tomography angiography (ccta) including functional flow reserve derived from ccta (ffr-ct) is as effective as invasive coronary angiography (ica) including functional flow reserve (ffr) for the detection and exclusion of obstructive coronary artery disease (cad). study hypothesis: initial non-invasive anatomic and functional testing is non-inferior to an invasive anatomic and functional testing strategy.

prospective, blinded multicenter study evaluating the diagnostic performance of coronary computed tomography angiography (ccta) including functional flow reserve derived from ccta (ffr-ct) for the detection and exclusion of significant obstructive coronary artery disease (cad). the reference standard vil be invasive coronary angiography (ica) including functional flow reserve (ffr). patients referred for elective ccta because of suspected stable cad are considered. if the initial routine non-enhanced ct scan shows an agatston score \> 399, the patient is eligible for study inclusion. four sites in the region of southern denmark will participate (odense university hospital, svendborg hospital, vejle hospital and esbjerg hospital). a total of 278 patients will be included.

Eligibility criteria:
inclusion criteria: * age ≥ 18 years * an estimated likelihood for the presence of significant cad between 15% and 85% * clinical stable patients with symptoms of suspected cad referred for ctta * initial routine non-enhanced ct scan shows an agatston score \>399 * written informed consent * accept to undergo elective invasive coronary angiography (ica) within 90 days exclusion criteria: * known prior myocardial infarction * prior percutaneous coronary intervention (pci) * prior coronary artery bypass surgery (cabg) * pacemaker or internal defibrillator lead implantation * prosthetic heart valve * atrial fibrillation * renal insufficiency (\<40 ml/min) * known anaphylactic reaction to iodinated contrast * pregnancy

Keywords: angina chest pain diagnostic tests
pharmacogenomic testing in major depressive disorder

Conditions: Major Depressive Disorder

Summary: this is a two-arm double-blind prospective randomized controlled trial (rct) to evaluate clinical impact of pharmacogenomic testing on the treatment of major depressive disorder. participants will be randomly assigned to two groups: pharmacogenomic-guided therapy group (guided group) and treatment as usual group (tau group). the primary hypothesis is the pharmacogenomic-guided treatment group will demonstrate significantly higher percent improvement in depression score compared to treatment-as-usual group.

to a large extent, variability in antidepressant efficacy can be explained by genetic variations that affect medication-metabolizing enzymes, drug transporters, and medication targets. recent reviews demonstrated significant potential of pharmacogenomic testing in improving treatment of major depressive disorder. one of the major barriers towards successful implementation of pharmacogenomic testing for patients with major depressive disorder is lack of systematic evaluation of impact of this approach in routine clinical care settings. the major goal of this study is to systematically evaluate impact of comprehensive pharmacogenomic testing on the treatment of major depressive disorder in ambulatory setting.

Eligibility criteria:
inclusion criteria: 1. clinical diagnosis of major depressive disorder (mdd) 2. prescription of index antidepressant medications 3. minimum score of 14 on the 17-item hamilton rating scale for depression (hamd-17) exclusion criteria: 1. diagnosis of bipolar disorder (any type), schizophrenia, or schizoaffective disorders 2. active diagnosis of substance abuse or dependence 3. current suicidal ideation 4. previous suicidal attempts 5. a person has already had pharmacogenetic testing done.

Keywords: pharmacogenomic testing depression
saliva cortisol measurement as a screening test for suspicious cushings syndrome in children.

Conditions: Cushings Syndrome

Summary: cortisol overproduction ( cushings syndrome) is a condition where the patient have a special fat distribution, with accumulation of fat around the abdomen and in the neck, with thinner limbs and weak musculature. the condition can be difficult to separate from adipositas in general. screening for hypercortisolism in adults has been easier the last years, because we can measure free cortisol in saliva samples at late night. in children we still dont have reference ranges for saliva cortisol in children, so the screening is still troublesome, based on collection of 24-hour urine.the meaning of this study is to establish reference ranges for saliva cortisol, and evaluate in a cohort with adipose children if screening with saliva cortisol is sensitive and specific enough to be the new screening test for hypercortisolism in children too.

the investigators have collected saliva samples morning and late evening for cortisol measurements in a total of 330 children ( 660 samples) in the age from 4 to 16 years ols. this samples are going to be used to define a reference range for saliva cortisol in children. the investigators are then collecting 24 hour urine and saliva samples ( morning and late evening) from children at the obesity clinic haukeland university hospital for verification of the reference range , and to estimate if the test is good enough to identify the patients that have cushings syndrome. since the diagnose is very seldom in children, we will get help from a 3. line referral center in london to include patients with true cushings syndrome.

Eligibility criteria:
inclusion criteria:children aged 4-16 years. healthy population from selected kindergarten and schools, and patients refered to the obesity clinic haukeland university hospital, for examination and optionally treatment. - exclusion criteria:use of any kind of steroids ( oral, liniments, inhalations etc) -
norwegian carotid plaque study

Conditions: Ischemic Stroke; Carotid Artery Disease; Embolic Stroke

Summary: background: in 30-40% the cause of ischemic stroke remains undetermined. most likely, this category hides an additional number of strokes caused by artery-to-artery embolisms due to unidentified atherosclerotic disease, or caused by cardioembolism. both types are associated with a high risk of recurrent ischemic events and multiple cerebral infarctions. large-artery atherosclerosis of the brain-supplying arteries is the assumed underlying cause in 10 to 15% of ischemic stroke, mostly deriving from the extracranial carotid artery. carotid intima-media thickness (cimt) measured by 2-dimensional (2d) b-mode ultrasonography and estimation of the overall atherosclerotic plaque burden aids future risk prediction. arterial wall changes, artery caliber variations, degree of stenosis, local hemodynamic alterations and certain plaque characteristics are important for the evaluation of plaque vulnerability and vascular risk stratification. transcranial doppler monitoring (tcdm) is a non-invasive bedside examination eligible for detection of microemboli in the human cerebral circulation. hypothesis: atherosclerotic stenosis and plaque characteristics can be more accurately assessed by the combination of routine 2d ultrasound, contrast enhanced ultrasound (ceus), and 3-dimensional (3d) ultrasound. tcdm, ceus and 3d visualization of the carotid plaque improve the differentiation of stroke etiology and quantification of plaque vulnerability, and aid the prediction of future risk for cerebrovascular events in the individual patient. aims: assessment of prevalence and frequency of microemboli signals (mes) in unselected patients with cerebral ischemia, the influence of antithrombotic drugs on mes, and the relationship between mes and recurrent stroke or transient ischemic attack (tia). categorization of atherosclerotic carotid artery disease by use of routine and advanced neurosonographic techniques combined with anamnestic and clinical data. development of a visualization solution tailored for 3d visualization of carotid arteries and semi-automatic plaque segmentation.

* standardized questionnaire * extracranial and transcranial duplexsonography * extracranial contrast-enhanced ultrasound * extracranial 3d-ultrasound * transcranial doppler monitoring * clinical work-up incl. radiological and cardiological procedures, scoring etc

Eligibility criteria:
inclusion criteria: * symptomatic carotid artery disease * asymptomatic carotid artery disease
a real-world assessment of the demographic, clinical characteristics and outcomes of a brazilian cohort of previously untreated extensive stage-small cell lung cancer receiving durvalumab combined with platinum-etoposide in (es-sclc) in brazil

Conditions: Extensive-stage Small-cell Lung Cancer

Summary: the central objective of this study is to characterize the demographic of an es-sclc brazilian cohort treated with durvalumab. secondarily, to assess the outcomes of durvalumab-based regimens in 1l treatment of es-sclc brazilian patients from the private health care setting.


Eligibility criteria:
inclusion criteria: * have a documented diagnosis of es-sclc (newly diagnosed patients, as well as limited stage relapsed patients, are eligible); * have been treated with a durvalumab-based regimen for 1l es-sclc in the past (retrospective) or is currently being treated with a durvalumab-based regimen for 1l es-sclc (prospective); * male or female adult patients ≥ 18 years of age (as per local durvalumab approved label); * provision of consent in a signed informed consent form (icf) (allowing for data to be captured from existing medical records). if the patient´s data is entirely retrospective (i.e., the patient already presented progression, death, or completed 18 months of durvalumab-based treatment), the icf may be waived, as decided by the local ethics committee. exclusion criteria: * since the study is observational, there are no exclusion criteria. if the patient meets all the inclusion criteria, he/she will be eligible for the study.
tisagenlecleucel vs blinatumomab or inotuzumab for patients with relapsed/refractory b-cell precursor acute lymphoblastic leukemia

Conditions: Acute Lymphoblastic Leukemia

Summary: this trial aims to compare the benefits and risks of tisagenlecleucel to blinatumomab or inotuzumab in adult patients with relapsed or refractory all. this trial investigates tisagenlecleucel as an additional treatment option for this patient population with high unmet medical need.


Eligibility criteria:
inclusion criteria: 1. signed informed consent. 2. age ≥ 18 years. 3. subject with cd19-expressing b-all. 4. adequate organ function. 5. patients considered in any of the following settings are eligible: 1. untreated first or second relapse 2. refractory to primary induction therapy 3. refractory to first salvage therapy or 4. relapse after allogenic stem cell transplant. exclusion criteria: 1. patients presenting with untreated first relapse of all more than 24 months after initial diagnosis 2. presence of extra-medullary disease. 3. history or presence of clinically relevant cns pathology, or uncontrolled cns leukemia. 4. history of veno-occlusive disease (vod). 5. active neurological autoimmune or inflammatory disorders. 6. active acute graft-versus-host disease (gvhd), grade 2-4. other protocol-defined inclusion/exclusion may apply.

Keywords: acute lymphoblastic leukemia tisagenlecleucel intouzumab blinatumomab car-t ctl019
a clinical trial of tqa3038 injection in healthy adult subjects

Conditions: Chronic Hepatitis b

Summary: this is a phase 1 study in which healthy adult subjects will receive tqa3038 or placebo and will be assessed for safety, tolerability, pharmacokinetics. in the single ascending dose (sad) part, healthy adult subjects will receive one dose of tqa3038 or placebo, administered subcutaneously (sc).


Eligibility criteria:
inclusion criteria: * male or female age 18～60 years. * body mass index (bmi) 19 - 26 kg/m\^2. exclusion criteria: * any clinically significant chronic or acute medical condition that makes the volunteer unsuitable for participation； * history or evidence of drug or alcohol abuse； * history of intolerance to sc injection；
a phase 2 study comparing tas-102 versus topotecan or amrubicin to treat small cell lung cancer following platinum-based chemotherapy

Conditions: Small Cell Lung Cancer (SCLC)

Summary: the purpose of this trial is to compare the effects of tas-102 with either amrubicin or topotecan (drugs used in small cell lung cancer) on lung cancer to find out the effects on survival, how much time may pass without disease progression, and the safety of tas-102.

this is a multicenter, open-label, two-arm, randomized phase 2 study of tas-102 versus investigator's choice of therapy in patients requiring second-line chemotherapy for sclc refractory or sensitive to first-line platinum-based chemotherapy. investigator's choice of therapy is defined as second-line chemotherapy with iv topotecan (europe/japan) or iv amrubicin (japan). patients will be stratified by response to first-line platinum-based chemotherapy (sensitive vs refractory). sensitive patients are defined as patients who did not progress within 90 days after the last dose, and refractory patients are defined as patients who never responded or who responded but had radiologic progression \< 90 days after the last dose.

Eligibility criteria:
inclusion criteria: 1. has provided written informed consent 2. is ≥18 years of age for patients enrolled in europe; or ≥20 years of age for patients enrolled in japan 3. has definitive histologically or cytologically confirmed sclc (limited or extensive disease) 4. has progressed or had recurrence within 30 days prior to randomization 5. has at least one measurable lesion, as defined by recist criteria version 1.1 6. ecog performance status of 0, 1, or 2 7. is able to take medications orally 8. has adequate organ function (bone marrow, kidney and liver) 9. women of childbearing potential must have a negative pregnancy test and must agree to adequate birth control if conception is possible. males must agree to adequate birth control. exclusion criteria: 1. has cerebral metastases (unless metastases have been treated and controlled, metastases have been stable for at least 2-months post-intervention, and patient is not receiving corticosteroid treatment) 2. certain serious illnesses or medical condition(s) 3. has had certain other recent treatment e.g. major surgery, anticancer therapy, extended field radiation, received investigational agent, within the specified time frames prior to study drug administration 4. has received tas-102 5. has unresolved toxicity of greater than or equal to ctcae grade 2 attributed to any prior therapies 6. is a pregnant or lactating female

Keywords: second line small cell lung cancer, (sclc), refractory or sensitive to first line platinum based chemotherapy
electronic triggers: microhematuria trigger

Conditions: Hematuria

Summary: the research team will evaluate the effectiveness of an inbasket results message that prompts providers to place appropriate follow up orders.

the research team will evaluate the effectiveness of an inbasket results message that prompts providers to place appropriate follow up orders. the message will notify users that a patient has clinically significant microscopic hematuria and is at high risk for underlying malignancy and will include the official recommendations (imaging with a ct urogram, urology evaluation with cystoscopy).

Eligibility criteria:
inclusion criteria: * 35 years of age or older * 3+ rbc on urinalysis * urinalysis sent during an outpatient encounter * any of the following: 60 years of age or older; rbc \> 25; current or prior tobacco use; gross hematuria exclusion criteria: * under 35 years of age * cystoscopy within 1 year prior to urinalysis * nitrite positive and leukocyte esterase positive * nitrite positive and \>100 wbc * leukocyte esterase positive and \>100 wbc * know gu cancer diagnosis * urinalysis ordered by urology provider