Kornimate/medical-research-clean huggingface dataset:
low dose treosulfan based conditioning regimen in hsct for nijmegen breakage syndrome

Conditions: Nijmegen Breakage Syndrome

Summary: the aim of the current study is to evaluate the safety and efficacy of low dose treosulfan based conditioning regimen in hsct in nijmegen breakage syndrome

nijmegen breakage syndrome (nbs) is a dna repair disorder. the only curative option for combine immunodeficiency in nbs is allogeneic hematopoietic stem cell transplantation (hsct). standard myeloablative conditioning regimens in dna repair disorders lead to increased morbidity and mortality after hsct. low doses of alkylators are used to reduce toxicity rates, which, however, increase the risks of mixed chimerism and graft failure. the data of treosulfan usage in nbs are sparse. to evaluate the safety and efficacy of low dose treosulfan based conditioning regimen in nbs, treosulfan 21g/m2 in combination with fludarabine 150mg/mg, cyclophosphamide 40mg/kg, thymoglobulin (genzyme) 5mg/kg and rituximab 100mg/m2 will be used from day -6 to -1 day, followed by stem cell infusion. the primary endpoint is event-free survival, where graft failure, death, and malignancies are considered as events.

Eligibility criteria:
inclusion criteria: 1. patients aged ≥ 3 months and \< 21 years 2. patients diagnosed with nbs eligible for an allogeneic hsct 3. signed written informed consent signed by a parent or legal guardian
wnt, notch and hedgehog activity in chemo-naive tumors collected during staging of esophageal cancer patients

Conditions: Esophageal Cancer

Summary: cancer results when undifferentiated cells grow in an uncontrolled manner, crowding out normal cells, causing morbidity and ultimately mortality. the cancer stem cell theory suggests that most tumors undergo a process of differentiation through which a relatively rare cancer stem or progenitor cell (csc) gives rise to more differentiated populations of cells (including transiently amplifying cells) comprising the bulk of the tumor. as a result of this cellular diversity, one or more cells within the tumor are likely to be resistant to therapy. among cells resistant to a given therapy, only cscs can repopulate the tumor. a key feature of this resistant subset of cscs is that they repopulate a tumor resistant to the original intervention. the cellular programs driving the uncontrolled proliferation of many solid tumors result from aberrant activity of wnt, shh, and/or notch signaling pathways in esc. thus, therapies that down-regulate the activity of these fundamental pathways in cscs will be effective in the treatment of cancer. the investigator's research program focuses on the elucidation of signaling mechanisms, control of cellular processes and discovery of small molecules that selectively target wnt, shh, and notch signaling pathways that are fundamental to cscs. our preliminary results identified a novel notch associated protein nack that functions as a transcriptional co-activator of notch. moreover, nack is expressed in human solid tumors and is required for cell survival and tumor growth in notch -dependent tumor cells. the investigator's aim is to further interrogate the link between notch and nack. specific aims: * identify and isolate the cancer stem cell populations from primary chemo naive esophageal tumor samples. * interrogate the status of the notch,( the link between notch and nack), wnt and hedghog pathways in the chemo naive esophageal tumor as well as in specific cell populations, such as the csc. * determine the degree of cross-talk between these pathways and which of these pathways is essential for the self renewal properties and tumorigenic properties of the esc population. * identify critical targets for therapeutic intervention in csc populations.


Eligibility criteria:
inclusion criteria: * patients diagnosed with an esophageal cancer and scheduled for a standard of care endoscopic ultrasound. * patient must sign a consent to be a participant in this protocol. exclusion criteria: * patients not diagnosed with an esophageal cancer. * patients that have undergone a previous staging endoscopic ultrasound.

Keywords: esophageal cancer cancer stem cell csc wnt hegdehog notch signaling pathway chemo-naive
a shared decision making intervention for diabetes prevention in women with a history of gestational diabetes mellitus

Conditions: Gestational Diabetes Mellitus; Pre-diabetes; Diabetes Mellitus, Type 2

Summary: our goal is to test whether shared decision making for diabetes prevention can help women with a history of gestational diabetes mellitus (gdm) who are at high risk of developing type 2 diabetes (t2dm) increase weight loss and adoption of evidence based strategies to lower their risk of incident diabetes.

women with a history of gestational diabetes mellitus (gdm) are at high risk of developing type 2 diabetes (t2dm) but evidence from the diabetes prevention program (dpp) indicates that lifestyle change and metformin use in this population are clinically equivalent, each reducing the incidence of t2dm by approximately 50%. shared decision making (sdm) is an attractive approach in this situation where several options are available. we can use a decision aid to make the decision explicit, describe the available options with equipoise, elicit patient preferences, and help patients make an informed decision that is right for them. there are no existing studies evaluating sdm for diabetes prevention among women with a history of gdm. therefore, this study aims to test the effectiveness of an rct evaluating sdm for diabetes prevention on weight loss among overweight/obese women with a history of gdm and hemoglobin a1c between 5.7-6.4%, as well as uptake of lifestyle change and/or metformin use and other patient-reported outcomes (e.g., physical activity, eating patterns, patient activation, health-related quality of life). the study will recruit 310 patients from two large health care systems (n=155 from ucla health and n=155 from intermountain healthcare) who will be randomized to either usual care or in-person shared decision making for diabetes prevention.

Eligibility criteria:
inclusion criteria: * bmi 25 kg/m2 or above (23 or above if asian american woman) * history of gestational diabetes mellitus * history of prediabetes in prior 36 months defined by either 1) most recent hba1c 5.7-6.4% or 2) most recent fpg 100-125 mg/dl or 3) prior diabetes diagnostic codes + abnormal labs * patient at ucla or intermountain healthcare systems with provider visit or labs in prior 12 months exclusion criteria: * delivery within prior 12 months * history of diabetes (i.e., prior hba1c 6.5% or above, or 2 or more fasting plasma glucose 125 or above, or prior diagnostic code) * use of an anti-glycemic medication in prior 12 months * last available glomerular filtration rate (egfr) \<45 ml/min * bmi \>60 kg/m2 * bariatric surgery with prior 12 months * active eating disorder * currently pregnant or planning to get pregnant in the next 12-24 months * completed a prediabetes sdm consult in past * currently enrolled in a cdc recognized diabetes prevention program (dpp) * inability or concerns about doing \>150 minutes of physical activity per week * history of metformin intolerance * non-english primary language

Keywords: diabetes prevention shared decision making
vessel wall mr imaging to explore sex-differences of intracranial arterial wall changes after suspected stroke

Conditions: Intracranial Atherosclerosis; Acute Stroke; Transient Ischemic Attack

Summary: despite advances in stroke care, women continue to face worse outcomes after stroke than men. this disparity in outcomes may be related to biologic sex-differences that manifest in the development and progression of atherosclerosis. decades of cyclic changes in the hormonal milieu lead to different metabolic profiles in women. these changes may also explain sex-differences in risk factor profiles of atherogenesis and plaque composition. the investigators' objective is to conduct a cross-sectional mr imaging study of suspected stroke patients to compare the burden and composition of intracranial atherosclerosis and risk factors between men and women. results from this study are expected to show that sex and sex-specific risk factors should be considered at the outset of stroke evaluation for risk-stratification. in the era of precision medicine, the investigators propose the role of sex should be a starting point in the clinical evaluation of stroke.


Eligibility criteria:
inclusion criteria: * older than 18 years * suspected stroke patients * no contraindications for an mri exam or gadolinium intravenous contrast * no need for an emergent interventional treatment (e.g., thrombectomy) exclusion criteria: * younger than 18 years * pregnant

Keywords: intracranial atherosclerotic disease acute stroke transient ischemic attack vessel wall imaging
clinical pharmacological study of al-43546 ophthalmic product in subjects with shortened tear film break up time

Conditions: Dry Eye Disease

Summary: the purpose of the study is to observe lacrimal fluid condition (tear film break-up time) chronologically after a single dose of al-43546 ophthalmic products（0.15%, 0.25% and vehicle）and 0.1% sodium hyaluronate ophthalmic solution and compare lacrimal fluid retention time between them.


Eligibility criteria:
inclusion criteria: * subjects with nibut of either eye is 8 seconds or less and other eye is 10 seconds or less at screening test and nibut of either eye before instillation in period i is 10 seconds or less. exclusion criteria: * diagnosis of severe sjogren's syndrome (history of the use of punctal plug one time or more, etc.) in either eye. * subjects who will use ophthalmic solutions (excluding the specified artificial tear) or ophthalmic ointment in either eye during the study period.
trial aiming at assessing the effect of humidification on the comfort of the patient receiving an oxygen therapy

Conditions: Oxygen Inhalation Therapy

Summary: in france, the most used humidifying system during oxygen therapy is a non heated device with weak efficiency and non-proven clinical benefits. this study aims to assess the lack of benefits of non-heated humidification compared to no humidification, as regards to the comfort of the patient receiving oxygen therapy.


Eligibility criteria:
inclusion criteria: * admission in intensive care unit * oxygen therapy started for less than two hours in icu exclusion criteria: * age under 18 * pregnancy * participation in other trials with the same endpoints * absence of registration in french health care system * patient protected by law * tracheotomised or intubated patient * patient with non invasive ventilation * oxygen therapy started for more than two hours in icu * moribund

Keywords: oxygen mask oxygen cannula humidification oxygen therapy intensive care unit
trial of pm02734 administered in combination with erlotinib in patients with advanced malignant solid tumors

Conditions: Advanced Malignant Solid Tumors

Summary: phase i multicenter, open-label, dose-escalating clinical and pharmacokinetic trial of pm02734 administered in combination with erlotinib to determine the safety and tolerability and to identify the dose limiting toxicity (dlt) and the recommended dose (rd) of the combination of pm02734 and erlotinib, in patients with advanced malignant solid tumors.

phase i multicenter, open-label, dose-escalating clinical and pharmacokinetic trial of pm02734 administered in combination with erlotinib to determine the safety and tolerability and to identify the dose limiting toxicity (dlt) and the recommended dose (rd) of the combination of pm02734 and erlotinib, determine the preliminary pharmacokinetics (pk) of the combination, evaluate the preliminary pk/pharmacodynamic correlation, evaluate the preliminary antitumor activity, perform a preliminary pharmacogenomic (pgx) study to explore molecular predictors of response to erbb receptor antagonists and pm02734 in patient with advanced malignant solid tumors.

Eligibility criteria:
inclusion criteria: * voluntary written informed consent form. * histologically or cytologically confirmed advanced malignant solid tumors. * measurable or non-measurable disease following (recist) * age ≥ 18 years. * life expectancy ≥ 3 months. * performance status ecog ≤ 2. * recovery from any drug-related adverse events (aes) derived from previous treatments. * appropriate bone marrow, liver and renal function. * left ventricular ejection fraction (lvef) within normal limits for the institution. * women of childbearing potential must have a negative serum pregnancy test before study entry. both men and women must agree to use a medically acceptable method of contraception throughout the treatment period and for three months after discontinuation of treatment. exclusion criteria: * prior therapy with pm02734. * pregnant or lactating women. * less than four weeks from radiation therapy. * evidence of progressive central nervous system (cns) metastases. or any symptomatic brain or leptomeningeal metastases. * other relevant diseases or adverse clinical conditions. * any other major illness that, in the investigator's judgment. * limitation of the patient's ability to comply with the treatment or to follow-up at a participating protocol. * ingestion of potent cytochrome cyp3a4 inhibitors. * treatment with any investigational product in the 30-day period prior to the first infusion. * known hypersensitivity to any component of pm02734 or erlotinib.

Keywords: pm2734 tumors pharmamar erlotinib tarceva
cardiac rehabilitation of stroke survivors (srp-cross)

Conditions: Stroke; Cerebrovascular Accident

Summary: this study examines the effectiveness of the cardiac rehabilitation program for stroke patients. the study will examine if patients with stroke, who receive cardiac rehabilitation in addition to their standard of care treatments, demonstrate improved recovery of function. it will also examine if these patients have reduced hospital readmission, reduced rate of recurrent stroke, and mortality.


Eligibility criteria:
inclusion criteria: * 18 years or older * stroke diagnosis (ischemic or hemorrhagic) / radiologic evidence of acute stroke * medically cleared by a cardiologist for participation in the cardiac rehabilitation program with no contraindications to cardiac rehabilitation per american association of cardiovascular and pulmonary rehabilitation (aacvpr) guidelines * ability to transfer on/off the recumbent bike with or without an assistive device safely, with or without assistance from another person * ability to follow simple commands and communicate pain or distress * admission to an inpatient rehabilitation facility post-stroke * signed informed consent form exclusion criteria: * presence of subarachnoid hemorrhage, intracranial aneurysm, intracranial hemangioma, or arteriovenous malformation * medical disorders that preclude participation in the study as determined by the principal investigator. * inability to have baseline assessment within 60 days post-stroke diagnosis * patient considered unable to comply with study requirements * known terminal illness with life expectancy less than 1 year * compliant diagnosis eligible for traditional cardiac rehabilitation covered by insurance * unable to understand/speak english

Keywords: cardiac rehabilitation stroke stroke rehabilitation stroke recovery exercise cardiovascular disease cardiovascular function cognitive function physical function neurorehabilitation transition of care post stroke
ultrasound evaluation of gastric emptying in diabetic patients

Conditions: Diabetes

Summary: this is a prospective cohort study. the main objective is to compare the frequency of a "full stomach" in the diabetic population compared to healthy controls 2 h after clear liquid and 6h after a light meal, and evaluate the mean gastric emptying time of clear liquid and light meal in diabetic patients.


Eligibility criteria:
inclusion criteria: * diabetic and non-diabetic patients scheduled for elective surgery aged 18 to 85years american society of anesthesia physical status classification i to iii body mass index \< 40 kg/ cm2. exclusion criteria: * pregnancy (currently or within the past 3 months) previous surgery of the upper gastrointestinal tract achalasia
observation of clinical routine care for heart failure patients implanted with biotronik crt devices

Conditions: Heart Failure

Summary: the registry is primarily designed to assess outcome, efficacy and residual safety aspects of crt based on long-term data from an unselected, real-life clinical set-up. moreover, the observation of the patient status should help to find possible predictors for hf events and to identify areas of improvement for crt and for crt device settings.


Eligibility criteria:
inclusion criteria: * planned de novo implantation of or upgrade to a crt system for treatment according to the intended use * patient is able to understand the nature of the registry and has provided written informed consent * patient is willing and able to use the cardiomessenger and accepts the home monitoring concept * remote monitoring using the home monitoring® platform is planned for the patient exclusion criteria: * standard contraindication for crt * already or previously implanted with crt system * • age \< 18 years * participation in another interventional clinical investigation other than the registry-based trials of bio\|stream.hf * pregnant or breastfeeding

Keywords: cardiac resynchronization therapy crt clinical routine
development of a tailored life-sustaining treatment decision support intervention for stroke surrogate decision makers

Conditions: Stroke

Summary: the trial is testing an investigator-developed decision support tool for surrogate decision makers for stroke patients that are unable to make medical decisions for themselves. a historical usual care control group will be enrolled during tool development. the tool will then be tested in surrogates of hospitalized stroke patients.

decision support tools, specifically recommended in the 2010 affordable care act, have been shown to improve the quality of decisions and reduce burden on the decision maker in multiple clinical settings, yet almost none have focused on life-sustaining treatments in acute critical illness such as stroke. this study proposes a comprehensive frame shift in how the health care team and surrogates collaborate on decisions regarding life-sustaining treatments for acute illness, by developing a tailored web-based stroke decision support intervention. this web-based tool will be developed for use by the surrogate decision maker during the acute stroke hospitalization and will be designed to facilitate high quality patient-centered decisions and minimize adverse effects on the surrogate. the two project phases are: 1) decision support intervention development; and 2) pilot testing in surrogates of hospitalized stroke patients. a novel ordinal prognostic model will be developed and incorporated into the tool. the project expects 25 surrogate-patient pairs in the control group, and 25 surrogate-patient pairs in the intervention group, which equals a total of 100 subjects.

Eligibility criteria:
surrogates: inclusion criteria: * self-identify as the surrogate decision maker for eligible patient * able to read and communicate in english without an interpreter * limited to one surrogate per patient exclusion criteria: * no prior relationship with patient * dementia or other cognitive or health condition that would impair their ability to participate patients: inclusion criteria: * ischemic stroke or spontaneous intracerebral hemorrhage * impaired decisional capacity (per treating team) * enrolled on or before full hospital day 5 * minimum illness severity (either): * national institutes of health stroke scale ≥ 10 * glasgow coma scale ≤12 exclusion criteria: * no surrogate available for study procedures * already on comfort measures only * physician refuses to allow approach for consent * pregnancy

Keywords: surrogate decision maker
tpe in septic patients and influence on organ failure

Conditions: Multiple Organ Failure

Summary: therapeutic plasma exchange (tpe) should used for patients with septic shock in a controlled, prospective study focusing on the organ functions of the patients.

therapeutic plasma exchange (tpe) should used for patients with septic shock in a controlled, prospective study. investigators will study three groups: 1. untreated control group; 2. tpe group, plasma separation carried out by filtration; 3. tpe group, plasma separation carried out by centrifugation. all treated patients should treated two (minimal) to five times by tpe, depending on the need of norephinephrine for the therapy. for each session 40 ml/kg body weight plasma should be exchange. fluid resuscitation will be done with fresh frozen plasma. the study will focusing on the organ functions of the patients, especially liver-, kidney- and nervous function and also displayed in the sofa score on study days 3 and 7.

Eligibility criteria:
inclusion criteria: * septic shock, beginning of shock \< 24 hours exclusion criteria: * participation in an another clinical trial within the last 30 days * participation in this study at an earlier date * simultaneous participation in another clinical trial * pregnancy * unpredictable bleeding (over 2 erythrocyte concentrates daily) * polyneuropathy (known before the beginning of sepsis)

Keywords: septic shock fresh frozen plasma