Kornimate/medical-research-clean huggingface dataset:
hypertrophic myopathy in children

Conditions: Congenital Disorders

Summary: the purpose of this study is to identify the patients seen in our practice who are seen with hypertrophic myopathy diagnosis in order to better understand the presenting characteristics of their disease, the diagnostic testing to determine the diagnosis, the methods used to follow the disease progression and management practices used in caring for these patients. the objectives of this data review will be an analysis to determine if there is a methodology that will foster improved diagnostic speed and accuracy, and determine the best management practices based on outcomes in these patients.

the charts of children followed at sibley heart center of children's healthcare of atlanta will be identified using the mysis system and screening for the appropriate icd-9 codes for the types of hypertrophic myopathy. these codes include: 425.1, 425.4, 425.7, 425.8, and 425.9. screening will also be done for known anomalies that are associated with hypertrophic myopathy as seen in some syndromes and metabolic disorders. data collection: age at presentation (dob) history - prenatal, family, current surgical procedures physical exam results ekg interpretation cxr results echocardiography results mri results holter monitors results exercise testing results radionuclear study results spiral ct study results genetics testing results medication usage enzyme replacement therapy usage diagnostic laboratory results statistics: a generalized linear model will be performed. significance will be defined at p≤ 0.05. interim monitoring of accumulated data will be performed. one interim analysis will be performed after 3 months of accrual with additional analyses being performed at the end of data collection. demographic data will be summarized for all subjects. for each patient summary statistics will include the mean, standard deviation, frequency distribution, minimum, maximum and range.

Eligibility criteria:
inclusion criteria: * seen at sibley heart center, children's healthcare of atlanta * diagnosed with hypertrophic myopathy exclusion criteria: * those who do not meet inclusion criteria

Keywords: pediatric cardiac hypertrophic myopathy
efficacy and safety of amn107 in patients with gastrointestinal stromal tumors (gist) who have failed both imatinib and sunitinib

Conditions: Gastrointestinal Stromal Tumors

Summary: this study will evaluate the efficacy and safety of nilotinib in gastrointestinal stromal tumors patients who failed imatinib and sunitinib therapy.


Eligibility criteria:
inclusion criteria: * documented disease progression during imatinib and sunitinib therapy or intolerance to imatinib and/or sunitinib * at least one measurable site of disease on ct/mri scan * ps≤2 * normal organ, electrolyte, and bone marrow function exclusion criteria: * previous treatment with nilotinib or any other drug in this class or other targeted therapy * treatment with any cytotoxic and/or investigational drug ≤ 4 weeks prior to study entry * impaired cardiac function * use of coumarin derivatives (i.e. warfarin) * women who are pregnant or lactating other protocol-defined inclusion/exclusion criteria may apply.

Keywords: nilotinib amn107 sunitinib sunitinib malate imatinib imatinib mesylate tyrosine
predictors and outcomes of meropenem treatment failure among patients with septic shock in intensive care unit

Conditions: Predictors and Outcomes of Meropenem Treatment Failure

Summary: lately, the inappropriate used of meropenem in critically ill patient were increased. therefore, increased development of drug resistance bacteria towards meropenem. as known that carbapenem-resistance enterobacteriaceae (cre) is part of complication when meropenem is widely used in the intensive care unit. cre are very difficult to treat within gram negative bacteria as it encodes carbapenemase enzyme which breaks down carbapenem anti-microbial such as meropenem. the widespread carbapenemase production in the enterobacteriaceae was unknown until the early 2000 until first reported in 2001. despite that, most doctors and physician favourite, and still prescribe meropenem as the antibiotic of choice for the critically ill patients empirically. this is because of its broad spectrum of coverage for bacteria. thus, a number of meropenem treatment failure were increased as resistance increase.this study will evaluate the appropriate use of meropenem and determine the predictors of meropenem treatment failure as well as the patient outcomes.as a result, it can be a guidance prior prescribing the meropenem base on patient clinical condition and parameters while balancing the risk and benefits of its used.


Eligibility criteria:
inclusion criteria: 1. septic shock patient admitted to icu 2. age equal or more than 18 years old (no upper limit of age) 3. receive meropenem for at least 3 days exclusion criteria: 1. meropenem as prophylaxis 2. patient who had culture carbapenem resistant enterobacteriaceae organism (cre) 3. patient who admitted with proven diagnosis of cardiogenic shock
optimization principles in hemiparetic gait

Conditions: Stroke

Summary: this project seeks to identify the how walking impairments in stroke survivors contribute to mobility deficits through the use of behavioral observations and computational models. the chosen approach integrates biomechanical analyses, physiological assessments and machine learning algorithms to explain how asymmetries during walking influence balance and the effort required to walk. ultimately, the results of this work may lead to more personalized rehabilitation strategies to improve walking capacity and efficiency, and ultimately reduce fall risk in stroke survivors.


Eligibility criteria:
inclusion criteria for control participants: * no musculoskeletal injury or conditions that limit walking ability * no history of neurological disorders or severe head trauma * absence of cognitive impairment as demonstrated by a mini-mental score greater than 24 inclusion criteria for post-stroke participants * presence of unilateral brain lesion from a single stroke * weakness confined to one side * ability to walk on a treadmill for five minutes continuously without a cane or walker * absence of cognitive impairment as demonstrated by a mini-mental score greater than 24

Keywords: walking gait stability energetic cost
compassionate use study of methylnaltrexone

Conditions: Opioid-induced Constipation

Summary: this is an open label compassionate use study of subcutaneously administered methylnaltrexone (mntx) in participants with advance medical illness and opioid-induced constipation.


Eligibility criteria:
inclusion criteria: * advanced medical illness (that is; terminal illness, such as incurable cancer or end stage aqcuired immunodeficiency syndrome \[aids\]) with a life expectancy of one to six months. * on an opioid regimen for the control of pain/discomfort for at least seven days. * has opioid-induced constipation. * stable vital signs and systolic blood pressure greater than or equal to (\>=) 85 millimeters of mercury (mmhg), and diastolic blood pressure \>=45 mmhg (supine or sitting). * females of childbearing potential must have a negative pregnancy test (serum or urine). * on a laxative regimen (for example, stool softener and senna or equivalent) for at least 3 days prior to treatment. participants who have discontinued laxatives due to intolerability or lack of efficacy are also eligible. exclusion criteria: * participants with known hypersensitivity to methylnaltrexone, naltrexone or naloxone. * participants who received any investigational new drug (experimental) except for methylnaltrexone in the previous 30 days. * participants who are constipated with a disease process suggestive of gastrointestinal obstruction, impaction or diagnosed with a current abdominal pathologic process which may represent a non-opioid cause of bowel dysfunction. * participants who are constipated and have active, clinically significant diverticulitis. * participants with a surgically acute abdomen. * participants being treated with opioids for diarrhea, dyspnea, cough, pulmonary edema, or congestive heart failure. * individuals with a known drug addiction. * females who are pregnant or nursing.
sleeve gastrectomy and roux-en-y gastric bypass in the treatment of type 2 diabetes mellitus.

Conditions: Obesity; Type 2 Diabetes; Bariatric Surgery

Summary: obesity is a growing epidemic throughout the world and is followed by increasing incidence of type 2 diabetes that accounts for 90-95% of all cases of diabetes. weight loss is a major objective, although difficult to achieve with medical treatments. many recent studies demonstrated that bariatric surgery has the potency to achieve marked and sustained weight loss, and is also associated with a significant improvement in control of type 2 diabetes. the principal aim of this study is to compare two types of bariatric procedures, the roux-en-y gastric bypass (rygbp) and sleeve gastrectomy (sg). the study hypothesis is that these procedures have equal efficacy with regard to resolution of type 2 diabetes.


Eligibility criteria:
inclusion criteria: 1. type 2 diabetes mellitus 2. bmi between 35 and 50kg/m2. 3. males and females 4. age between 18 and 60 years. exclusion criteria: 1. severe ongoing psychiatric disorder, alcoholism and substance abuse. 2. redo operations after previous bariatric procedures. 3. type 1 diabetes or other non-type 2 forms of diabetes 4. end stage renal disease, retinopathy, neuropathy
study to investigate dosage, efficacy, and safety of perampanel given as monotherapy in patients with epilepsy

Conditions: Epilepsy; Partial-Onset Seizures; Primary Generalized Tonic-Clonic Seizures

Summary: the primary objective of this study is to assess the retention rate of perampanel when given as secondary monotherapy in routine clinical care.

this study is a retrospective study of participants who received perampanel as primary or secondary monotherapy at approximately 40 centers in europe, asia, and australia. primary monotherapy is defined as the administration of perampanel in the absence of any concomitant antiepileptic drugs (aeds). secondary (conversion) monotherapy is defined as the conversion of perampanel from adjunctive therapy to monotherapy by withdrawing concomitant aeds.

Eligibility criteria:
inclusion criteria: * diagnosis of epilepsy * received perampanel as primary or secondary monotherapy at any time between 1 jan 2013 and 15 oct 2015 * provided written informed consent by the participant or the participant's legally authorized representative signed for the use of medical records per local requirements exclusion criteria: * not applicable

Keywords: perampanel e2007 phase 4 fycompa
assessment of stress and anxiety for patients undergoing mri using, holy quran, music, and natural sounds

Conditions: Anxiety; Stress; MRI Contrast Media Adverse Reaction

Summary: the aim of the current study is to analyze the effect of listening to quran or music or natural sounds on anxiety and stress level among patients undergoing mri.

after informed consents will be obtained from patients undergoing magnetic resonance image (mri). a randomized controlled parallel design will be used to assess the effectiveness of listening to quran, music, and natural sound to reduce anxiety and stress level among patients undergoing mri in palestine. a control group will receive the traditional care during mri without listening to any sound, while the intervention groups will listen to either quran, music, or natural sounds during the mri scan. notice that each patient will choose the preferred method to be in one of the four mentioned groups. this study will include participants with predetermined specific eligibility criteria.

Eligibility criteria:
inclusion criteria: * who agreed to participate in this study. * both sex. * age of 18 years and above. * fully conscious and oriented. exclusion criteria: * referrals from the intensive care unit. * life-threatening situations. * general ill health, * serious psychological disorders. * invasive mr imaging procedures (such as biopsies). * being under the age of 18, and * exams that were particularly scheduled for other investigations.
job-seekers training for patients with drug dependence - 1

Conditions: Substance-Related Disorders

Summary: the purpose of this study is to examine the effectiveness of a 12-hour basic job-training program designed to give drug abuse treatment patients the skills they need to find and secure a job and set vocational goals and methods for locating employment. the intervention to be evaluated is modeled after the job seekers' workshop, which was manualized and has been tested in trials.


Eligibility criteria:
* dsm-iv drug/alcohol abuse/dependence * enrolled in drug treatment for at least 30 days * unemployed or underemployed * expressed interest in employment * demonstrate english comprehension

Keywords: behavioral intervention
international multicenter registry for mechanical recanalization procedures in acute stroke

Conditions: Acute Stroke

Summary: the purpose of the endostroke registry is to gather information on predictors of good or poor clinical outcome following mechanical recanalization therapies for acute ischemic stroke.

mechanical recanalization procedures are increasingly used in large vessel stroke, i.e. in proximal middle cerebral artery (mca) or basilar artery (ba) occlusion. this trend is due to the limited efficacy of the only approved acute stroke treatment, systemic thrombolysis, in large vessel occlusion but also due to new technical innovations triggering the development of numerous devices for thrombus extraction. within large single-arm trials, some of these devices demonstrated their potential with respect to acceptable complication- and substantial recanalization rates. nevertheless, clinical outcome of these stroke patients is frequently poor despite an elaborate, aggressive, but also complex and costly treatment regimen. the factors determining good or poor clinical outcome in mechanical recanalization remain to be determined. the goal of the endostroke-registry is the systemic evaluation of technical and clinical features that might be important for the further development and evaluation of endovascular treatment strategies for acute stroke. special emphasis is laid on the assessment of clinical outcome (90 day mrs). concerning technical and periprocedural aspects, a focus is put on time issues i.e. intra-hospital time delays, duration of angiographic procedures until vessel patency is achieved.it is planned to enroll approximately 500 patients in this registry. in first line, prospective patients will be included in the register (start date january 2011) but retrospective inclusion of patients being treated within the three years before the start of the register is allowed as long a consecutive patient registration is guaranteed (to exclude selection bias). data analysis will include univariate and multivariate approaches with respect to clinical outcome measures.

Eligibility criteria:
inclusion criteria: * patients being treated with mechanical recanalization devices due to an acute proximal arterial vessel occlusion of brain supplying arteries exclusion criteria: * patients being treated with mechanical recanalization devices for venous vessel occlusion. * patients being treated with mechanical recanalization devices due to an acute vessel occlusion which occured as a complication of an angiographic procedure scheduled for other reasons (i.e. as a complication of a diagnostic angiography or coiling procedure for cerebral aneurysms)

Keywords: stroke, acute mechanical recanalization interventional neuroradiology clinical registry
a safety, tolerability, and efficacy study of vx-880 in participants with type 1 diabetes

Conditions: Diabetes Mellitus, Type 1; Impaired Hypoglycemic Awareness; Severe Hypoglycemia

Summary: this study will evaluate the safety, tolerability and efficacy of vx-880 infusion in participants with type 1 diabetes (t1d) and impaired awareness of hypoglycemia (iah) and severe hypoglycemia.


Eligibility criteria:
key inclusion criteria: * clinical history of t1d with \> 5 years of duration of insulin dependence * at least two episodes of documented severe hypoglycemia in the 12 months prior to enrollment * stable diabetic treatment * consistent use of continuous glucose monitor (cgm) for at least 3 months before screening and willingness to use cgm for the duration of the study key exclusion criteria: -prior islet cell transplant, organ transplant, or cell therapy other protocol defined inclusion/exclusion criteria may apply
sensory modulation dysfunction and posttraumatic stress disorder

Conditions: Posttraumatic Stress Disorder; Sensory Processing Disorder

Summary: to explore the role of sensory modulation dysfunction (smd) (i.e., a neurodevelopmental state altering the sensory perception, severely interfering with daily function) as a risk factor for posttraumatic stress disorder (ptsd), its co-occurring pain, and impeded cognitive functions, following exposure to combat trauma.

background and aim: posttraumatic stress disorder (ptsd) is interwoven with chronic pain, and the latter co-occurs with sensory modulation dysfunction (smd). moreover, smd was found as a risk factor for chronic pain and hampered executive functioning. currently, the sensory domain is neglected in the ptsd research realm, though findings indicate its link to ptsd. thus, this study proposes to bridge a current gap in ptsd knowledge base, and specifically, our general aim is to uncover the role of smd in predicting combat trauma-related symptomatology, altered executive function, and clinical pain. methods: this is a single-blind randomized control study, comprising 4 assessments: pre (t1); post (t2); 10 (t3); and 40 (t4) days follow-up, following experimental manipulation (see figure 1). participants with and without smd will be randomly allocated to experimental and control groups that will watch traumatic movie scenes simulating combat and non-traumatic movie scenes, respectively. the assessor will be blinded to group allocation. population: one-hundred healthy individuals aged 18 to 28 years, with and without smd. participants with smd will be identified via a standardized questionnaire during screening. the sample size was calculated based on power analyses via g\*power 3 software derived from p-value of .05 and statistical power of .80. tools: both objective and subjective outcome measures will be applied consisting of self-report questionnaires, psychophysical-, physiological-, emotional reactions, and executive function performance-based- testing will be applied. the experimental manipulation will comprise of the trauma film paradigm. expected results: findings may advance the understanding of ptsd development and thus not only serve in attenuating the risk for ptsd among combatants but also may contribute to developing preventative measures for ptsd among combatants.

Eligibility criteria:
inclusion criteria: * intact or corrected vision * proficiency in hebrew * exclusion criteria: * neurological disorders * psychiatric disorders * neurodevelopmental disorders * substance use disorder * chronic pain * regular intake of medications. * cultural and societal backgrounds that might bias participant reaction to study protocol (i.e., nationality)

Keywords: sensory processing executive function trauma peritrauma
tsh receptor antibody heterogeneity in children and adolescents with graves' disease

Conditions: Graves' Disease

Summary: graves' disease, the most common form of hyperthyroidism in children, is caused by thyrotropin (tsh) receptor antibodies (trabs) that mimic the action of tsh. the disease leads to significant morbidity in children both due to the prolonged course of antithyroid medication often required for sustained immunological remission and the high risk of relapse when medication is withdrawn. the ability to predict which patients are most likely to fail medical management would greatly improve the choice of therapy. in the past, large goiter size, age at diagnosis, increased biochemical severity, and decreased body mass index have all been associated with a poorer prognosis, but these clinical indicators lack sensitivity and specificity. preliminary data suggest that the new trab assays are both sensitive and specific for the measurement of trabs in children with graves' disease. in addition, variation in these antibodies over time is not the same in all patients. the goal of this proposal will be to prospectively follow children with newly diagnosed graves' disease and use microarray technology to determine if there are genes whose expression differ in patients who respond to medical therapy versus those who will need more definitive therapy earlier in their disease.

in the present grant proposal, we plan to utilize two new assays (binding and bioassay) in order to identify additional predictors of graves' disease and apply them to a well characterized group of patients with graves' disease followed prospectively. more specifically, we plan to further investigate the antibodies by measuring lambda: kappa light chain antibody ratios in pediatric patients. we will assess epitope heterogeneity by using novel chimeric proteins in which specific portions of the tsh receptor have been replaced with the closely related lh receptor. we will utilize microarray technology to determine if there are differences in gene expression profiles in responders versus non responders. it is hoped that these methods will lead to an improved ability to follow disease progression and to monitor efficacy of therapy.

Eligibility criteria:
inclusion criteria: * age 2-21 years * suppressed thyroid stimulating hormone (tsh) * elevated triiodothyronine (t3), thyroxine (t4) exclusion criteria: * pregnancy * toxic nodule * currently receiving steroids or thyroid hormone replacement * bacterial, viral, radiation, or autoimmune thyroiditis

Keywords: autoimmune thyroid disease children and adolescents tsh receptor antibodies