Kornimate/medical-research-clean huggingface dataset:
a pilot therapeutic trial using hydroxyurea in type i spinal muscular atrophy patients

Conditions: Muscular Atrophy, Spinal

Summary: the objectives of this trial are: to establish a safety profile for use of hydroxyurea in children with type i spinal muscular atrophy; to identify reliable outcome measures for hu treatment in type i sma; and to detect the clinical efficacy of hu treatment in children with type i sma.

sma is a neuromuscular disorder characterized by degeneration of spinal cord motor neurons and muscular atrophy. sma is classified into three clinical subtypes according to the severity and age of onset (types i, ii and iii). type i sma (also called severe, infantile or acute sma, or werdnig-hoffman disease) is the most severe phenotype. the onset of symptoms is within the first 6 months of life, and weakness of intercostal muscles and lack of airway protection lead to respiratory insufficiency and aspiration pneumonia, often resulting in early infant death. in our laboratory, our preliminary results indicate that hu treatment significantly increases both smn mrna expression and intact smn protein levels in vitro. these data confirm previous observations that in vitro treatments of sma lymphocytes with hydroxyurea resulted in augmentation of the smn2 gene expression in a dose and time related manner. based on these exciting pre-clinical data, coupled with the well-documented side-effect profile of hu in children, we are conducting a pilot clinical trial using hu in children with type i sma. this clinical trial study is intended to establish the safety profile in children with type i sma; to identify reliable outcome measures; and to detect the possible clinical efficacy of hu treatment in children with type i sma.

Eligibility criteria:
inclusion criteria:1. laboratory confirmation of a homozygous deletion or mutation of the smn1 gene 2. clinical diagnosis of type i sma (never achieved independent sitting) 3. onset of disease before the age of 6 months 4. enrollment in study within 6 months of diagnosis exclusion criteria:1. known hematological disorders, such as chronic anemia (defined as platelet count less than 100,000/mm\^3) in two contiguous measures in two weeks 2. severe systemic disorders such as congenital heart disease, other major birth defects involving internal organs, or severe birth asphyxia 3. participation in sma clinical trials for other experimental drugs 4. requiring continuous respiratory support before the initiation of hu treatment
a study of the effect of high altitudes on physiological and metabolic markers in adults living at qinghai-tibet plateau for work purpose

Conditions: Residence or Prolonged Visit at High Altitude as the Cause of Mountain Sickness

Summary: this ambispective cohort study aims to evaluate the effect of high altitudes environment on human's physiological and metabolic markers, specific markers of main human systems, and incidence rate and severity of chronic mountain sickness (cms).

qinghai-tibet plateau is a high altitudes area with average 3,000-5,000 meters height in china. as the partial pressure of oxygen in inspired air falls with increasing terrestrial elevation above sea level, many physiological variables in human body could be affected by this hypobaric hypoxic environment. up to date, there are no large scale clinical trials assessing these changes in chinese population. the study here would like to provide the newest medical information on the changes of physiological variables and correlation between specific risk factors in chinese adults living at high altitudes for work purpose.

Eligibility criteria:
inclusion criteria: * be provided verbal or written informed consent; * men and women ≥ 30 and ≤ 65 years of age; * adults living at qinghai-tibet plateau for work purpose, and the altitude of residence is more than 2,500 meters; * providing electronical or paper medical examination reports which conducted in 2016. exclusion criteria: * pregnant or breast-feeding women; * be excluded by principal investigator for subject's compliance or safety reasons.
multiple rising dose study of mk-6194 in participants with atopic dermatitis (mk-6194-008)

Conditions: Dermatitis, Atopic

Summary: the primary objective of this study is to characterize the safety and tolerability of mk-6194 following multiple doses among participants with moderate to severe atopic dermatitis who are unresponsive to other therapies.


Eligibility criteria:
the main inclusion and exclusion criteria include but are not limited to the following: inclusion criteria: * clinical diagnosis of atopic dermatitis for at least 6 months prior to the screening visit. * atopic dermatitis is of at least moderate severity. * history of inadequate response to a stable (≥1 month) regimen of medium to high potency topical corticosteroids or calcineurin inhibitors as treatment for atopic dermatitis within 6 months before the screening visit. * body mass index (bmi) ≥18 and ≤38 kg/m2 at the screening visit. exclusion criteria: * concurrent significant skin disease other than atopic dermatitis (such as psoriasis) or a concurrent clinically significant disease. * significant organ dysfunction that is unstable or inadequately treated within 6 months prior to screening. * history of cancer (malignancy), with the exceptions: of adequately treated nonmelanomatous skin carcinoma or carcinoma in situ of the cervix or; other malignancies that have been successfully treated with appropriate follow up. * history of myocardial infarction, congestive heart failure, uncontrolled arrhythmias, cardiac revascularization, stroke, uncontrolled hypertension, or uncontrolled diabetes within 6 months of screening. * history of organ or tissue allograft. * history of symptomatic herpes zoster within 16 weeks of randomization, or any history of disseminated herpes simplex, disseminated herpes zoster, ophthalmic zoster, or central nervous system (cns) zoster. * major surgery within 3 months prior to the screening visit or has a major surgery planned during the study. * received a live or attenuated virus vaccine within 4 weeks prior to the screening visit or intends to receive live or attenuated virus vaccination during the course of the study and for 12 weeks after the last dose of study drug. * currently receiving any chronic systemic (oral or intravenous) anti-infective therapy for chronic infection (such as pneumocystis, cytomegalovirus, herpes zoster, or atypical mycobacteria).
a phase 1/2 study of bpi-361175 in subjects with advanced solid tumors

Conditions: Non-small Cell Lung Cancer

Summary: this is a phase i/ii, open-label study to evaluate the safety, tolerability, pharmacokinetics and anti-tumor activity of bpi-361175 tablets in patients with advanced solid tumors including advanced non-small cell lung cancer (nsclc).this is a three-stage study, consisting of phase ia dose escalation, phase ib dose expansion and pivotal phase ii study. the pivotal phase ii study will be designed based on data generated from the phase i studies.


Eligibility criteria:
inclusion criteria: * male or female ≥ 18 years old; * patients with histologically or cytologically confirmed diagnosis of inoperable locally advanced or recurrent/metastatic non-small cell lung cancer (nsclc) with egfr sensitive mutations. patients must have progressed from or be intolerant to or be unfit for standard treatment, or the standard treatment does not exist; * for dose expansion and phase ii, patients must be willing to provide tumor tissues (archived tumor tissue samples within 2 years or fresh tumor tissues) and/or blood samples for central lab testing; * measurable or evaluable disease; * adequate bone marrow, liver, and renal function. exclusion criteria: * unstable, symptomatic primary cns tumors/metastasis or leptomeningeal metastases which are not suitable for enrollment, as judged by investigators; * pregnancy or lactation; * other protocol specified criteria.
different level of ecswt in post mastectomy lymphedema

Conditions: Lymphedema of Upper Limb

Summary: to investigate the effectiveness of different dosages of ecsw in the treatment of post mastectomy lymphedema volume and quality of life (qol)

the treatment program included two sessions per week for eight weeks. this study was approved by the ethical committee faculty of physical therapy cairo university. all patients signed a consent form involves their agreement to participate in this study.

Eligibility criteria:
inclusion criteria: - criteria of selecting the patients from hospital included the following; 1. their ages ranged from 30 to 50 years. 2. all patients were examined carefully by physician before the study procedure. 3. all patients were free from any other pathological conditions or histories of other health abnormalities except arm lymphedema. 4. they had undergone radical or modified radical mastectomy with axillary lymph node dissection with or without radiotherapy intervention. 5. only ambulant subjects without any aides will be selected. 6. the degree of lymphedema in all subjects was grade 2 to 3 according to the classification of foldi.(foldi et al., 2006), (appendix ii ). 7. all patients will be medically stable. 8. each patient will sign a consent form which in that insures her eligibility in the study. 9. all patients were treated by the same doctor and physiotherapist. exclusion criteria: * the subjects had been excluded from the study if they met one of the following criteria; 1. the patients had recurrent malignancy, active infection, and clinical evidence of obstructive venous diseases. 2. patients contraindicated to eswt due to bilateral, acute, and chronic inflammation as well as due to metastasis and poor skin condition were excluded. 3. the patients had neurological or orthopedic problems, and diabetes. 4. patients with primary lymphedema. 5. cardiopulmonary disease which decrease the patient activites.

Keywords: lymphedema, shock wave, breast cancer
perfluorohexylcotane (nov03) for the treatment of signs and symptoms of dry eye disease associated with meibomian gland dysfunction (gobi study)

Conditions: Dry Eye Disease (DED)

Summary: this trial will evaluate the efficacy, safety, and tolerability of nov03 ophthalmic solution in comparison to a saline control for the treatment of the signs and symptoms of dry eye disease (ded) associated with meibomian gland dysfunction (mgd).


Eligibility criteria:
inclusion criteria: * signed icf (informed consent form) * subject-reported history of drye eye disease (ded) in both eyes * ability and willingness to follow instructions, including participation in all study assessments and visits exclusion criteria: * women who are pregnant, nursing or planning pregnancy * unwillingness to submit a blood pregnancy test at screening and the last visit (or early termination visit) if of childbearing potential, or unwillingness to use acceptable means of birth control * clinically significant slit-lamp findings or abnormal lid anatomy at screening and visit 1 * ocular/peri-ocular malignancy * history of herpetic keratitis * active ocular allergies or ocular allergies that are expected to be active during the study * ongoing ocular or systemic infection * wear contact lenses within 1 month prior to screening or anticipated use of contact lenses during the study * intra-ocular surgery or ocular laser surgery within the previous 6 months, or have planned ocular and/or lid surgeries over the study period * presence of uncontrolled systemic diseases * presence of known allergy and/or sensitivity to the study drug or saline components * use of any topical steroids treatments, topical cyclosporine, lifitegrast, serum tears or topical anti-glaucoma medication within 2 months prior to screening
a study to investigate the effect of sb-705498 on chronic cough

Conditions: Rhinitis

Summary: this study is designed to loook at the affect of oral sb-705498 on cough following an inhaled capsaicin challenge


Eligibility criteria:
inclusion criteria: * male or female between 30 -75 (part a) and 18-75 (part b) years of age inclusive. * non-child bearing women or women of child bearing potential if they agree to use contraception as indicated by the protocol * non-smoker for at least 6 months with a pack history \<5 pack years (pack years = (no. of cigarettes smoked/day/20) x no. of years smoked). * body weight \> 50 kg and body mass index (bmi) within the range 19 - 30.0 kg/m2 (inclusive). * capable of giving written informed consent. * agree to use contraception listed as acceptable * normal 12-lead ecg at screening. * chronic cough (part b only) * good general health, apart from chronic cough (part b only), as determined by a responsible physician. exclusion criteria: * a history of gastrointestinal, hepatic, renal or multiple cardiovascular risk factors. * positive pre-study drug/alcohol screen. * positive pre-study hepatitis b surface antigen or positive hepatitis c antibody result within 3 months of screening. * a positive test for human immunodeficiency virus (hiv) antibody (if determined by the local standard operating procedures (sops)). * history of regular alcohol consumption within 6 months of the study. * exposure to more than four new chemical entities within 12 months prior to the start of the study. * participation in a clinical trial with a new molecule entity or any other clinical trial within 30 days of the start of the study. * use of prescription or non-prescription drugs, as well as of vitamins, herbal and dietary supplements (including st john's wort) within 7 days prior to study. * known history of lung cancer * current treatment with oral corticosteriods or other immunosupressive agents * fev1 less than 80% of predicted value at screening * any subject who does not reach c5 following 250um oral capsaicin * history of drug or other allergy that, in the opinion of the investigator or gsk medical monitor, contraindicates their participation. * donation of blood or blood products in excess of 500ml within a 56 day period prior the start study. * pregnant females as determined by positive serum or urine human chorionic gonadotropin (hcg) test at screening or prior to dosing. * lactating females. * unwillingness or inability to follow the procedures outlined in the protocol. * cotinine levels indicative of smoking or history or regular use of tobacco- or nicotine-containing products within 6 months prior to screening. * consumption of red wine, seville oranges, grapefruit or grapefruit juice from 7 days prior to dosing.
feasibility and preliminary effects of fasting-mimicking diet in asian americans with prediabetes

Conditions: Prediabetes; Prediabetic State

Summary: this is a pilot study to determine the feasibility of studying the fasting-mimicking diet (fmd), a dietary approach that involves the consumption of a specifically formulated, calorie-restricted nutrition regimen with a customized macronutrient composition, ratio, and quantity over a 5- day period, on a larger scale in asian americans with prediabetes and to examine the preliminary effects of the diet in study participants. the main questions the study aims to answer are: 1. what are the recruitment, adherence, and attrition rates of eligible participants into the study? 2. does one fmd cycle result in changes in fasting blood glucose levels and physical measurements in study participants? participants will be asked to undergo one cycle of fmd (for 5 days), fill out surveys, and come in for a pre-fmd and post-fmd study visit, during which physical measurements and fasting blood glucose and ketone levels will be measured.


Eligibility criteria:
inclusion criteria: * identify as asian american * diagnosed with prediabetes according to at least one of the following: * hba1c 5.7%-6.4% * fasting blood glucose 100 to 125 mg/dl (5.6 to 6.9 mmol/l ) * oral glucose tolerance test (ogtt) 140-199 mg/dl at two hours exclusion criteria: * individuals with a personal history of diabetes mellitus (icd-10 codes e08-e13), cardiovascular disease, mental illness, drug dependency, pregnancy, and special dietary restrictions * individuals taking insulin or insulin-like drugs and individuals taking hypoglycemic agents other than metformin * individuals who are pregnant or breastfeeding * individuals with anaphylaxis food allergies * individuals who are allergic to tree nuts (macadamia, cashew, almond, pecan), soy, oats, sesame, or celery/celeriac * individuals who have completed the fmd in the past year
administration of arginine supplementation in preterm infants

Conditions: Necrotizing Enterocolitis

Summary: calprotectin is a cytosolic component of neutrophils .fecal calprotectin(fc) is a useful marker for exacerbation of inflammatory bowel disease in children .fc may be a useful marker for necrotizing enterocolitis (nec). nec is one of the most common ,deadliest and enigmatic intestinal problems encountered mostly in premature infants. the precise pathophysiology of nec is unclear ,but major factors thought to play an important role include an immature intestine ,an inflammatory response to intestinal microbes,enteral feedings and intestinal ischemia-reperfusion injury.diagnosis of nec is not easy clinically and up to now there is not a simple laboratory test to differentiate nec at an early stage from other conditions in the neonate. arginine is the substrate for no production in the gut and its deficiency may cause vasoconstriction and gut injury and thus predispose to nec. in previous studies arginine supplementation was found to reduce the incidence of nec in premature infants but more studies are needed for the use of arginine supplementation for the prevention of nec. the investigators aim is to measure the fecal calprotectin in very low birth weight (vlbw) infants during the first month of life as an inflammatory marker of the bowel and evaluate whether premature infants receiving arginine supplements had lower calprotectin values compared to the premature infants that did not . the investigators hypothesize that arginine supplementation in preterm infants reduces the inflammation of the gut which will be shown by the lower fecal calprotectin values of the premature infants receiving arginine supplementation.


Eligibility criteria:
inclusion criteria: * preterm neonates born at alexandra hospital during the study period * \< 34 weeks gestational age * \< 1500gr birth weight exclusion criteria: * major congenital abnormalities * inborn errors of metabolism * parents not consent

Keywords: necrotizing enterocolitis arginine supplementation calprotectin
mat pilates method in the treatment of women with fibromyalgia

Conditions: Fibromyalgia, Primary

Summary: introduction: physical exercises have been recommended to improve overall well-being in patients with fibromyalgia, with the main goal of repairing the effects of lack of physical conditioning, and improving symptoms especially pain and fatigue. very well estimated and widely known are the studies that support the use of the pilates method as effective in improving the symptoms of the disease. objective: to evaluate the effectiveness of the soil method in improving pain in women with fibromyalgia in the city of santa cruz, rn. methodology: this is a randomized controlled trial with blind evaluator, where 60 patients with fibromyalgia diagnosis are divided into two groups. the intervention group, perform an exercise program based on the pilates method in soil and another, considered control group, participate in a program of aerobic exercises in the pool. both groups conduct supervised exercise programs 2 times a week for a period of 12 weeks. the evaluation instruments used in an eva (visual pain scale); fiq questionnaire - fibromyalgia impact questionnaire; functional ability by the "timed up and go" test and 6-minute walk test; a quality of sleep by the pittsburgh sleep quality index (psqi-br) and an ess-br (epworth sleepiness scale); finally, a general quality of life for the sf-36. statistical analysis: data are analyzed by t-student, mann-whitney test, repeated-measures anova and intention-to-treat analysis.


Eligibility criteria:
inclusion criteria: * female patients with fibromyalgia diagnosed according to the classification criteria of the american college of rheumatology, * aged 18 to 60 years, * with vas between 3 and 8, * who signed the free and informed consent term. exclusion criteria: * patients with uncontrolled hypertension; * decompensated cardiorespiratory disease; * history of syncopes or arrhythmias induced by physical exercise; * diabetes unbalanced; * psychiatric disorders; * history of regular physical exercise (at least 2 times a week) in the last 6 months * any other condition that makes it impossible for a patient to perform physical exercises.

Keywords: fibromyalgia, pilates, exercises
the role of dysmyelination in cognitive impairment of psychotic spectrum disorders

Conditions: Schizophrenia; Bipolar Disorder

Summary: this is a single center study that uses both between-group comparisons and correlational analyses to establish biomarkers of dysmyelination and cognitive impairment in psychotic spectrum disorders using imaging and neuropsychological assays.the study will provide non-invasive biomarkers of cognitive dysfunction in psychotic spectrum disorder.


Eligibility criteria:
inclusion criteria: patients: * current dsm-5-defined diagnosis of a schizophrenia or bipolar disorder. a best estimate diagnostic approach will be utilized in which information from the diagnostic interview for genetic studies (digs) is supplemented by information from family informants, psychiatrists, and medical records to generate a diagnosis as needed * no alcohol or substance abuse during the last 6 month * no current substance-induced psychotic disorder or a psychotic disorder due to a general medical condition determined by dsm-5 criteria * ages 18 to 30 years old; * any race * competent and willing to sign informed consent * within 5 years from the disease onset. siblings: * have the same biological parents as their psd sibling * any race * no current or past history of psychotropic medication usage * no alcohol or substance abuse during the last 6 months * competent and willing to sign informed consent; * ages 18 to 30 years old. healthy controls: * matched for age to psd patients * no current or past history of psychotropic medication usage * no prodromal symptoms and no family history of psd * no alcohol or substance abuse during the last 6 months * competent and willing to sign informed consent. * all attempts will be made to recruit controls with similar parental ses as patients. however, given that psd are both a neurodevelopmental and familial disorder, exact matching for educational level or iq may neither be possible nor desirable. have the same biological parents as their psd sibling * any race * no current or past history of psychotropic medication usage * no alcohol or substance abuse during the last 6 months * competent and willing to sign informed consent; * ages 18 to 30 years old. exclusion criteria: * a serious neurological or endocrine disorder or any medical condition or treatment known to affect the brain, 2) organic brain disorder, mental retardation, or significant medical illness; * significant risk of suicidal or homicidal behavior; * must not have met dsm-5 criteria for current alcohol or drug dependence in the last 6 months; * contraindications to mri scanning (i.e., metal implants, pacemakers, pregnancy, etc.); * documented loss of consciousness (loc) for longer than 30 minutes or loc with any neurological sequelae.

Keywords: psychotic spectrum disorders