Kornimate/medical-research-clean huggingface dataset:
macrophagic activation and immunophenotypic profile of patients with gaucher's disease

Conditions: Patients With Gaucher's Disease

Summary: the aim of this study is to evaluate macrophaging activity and immunologic profile of patients with gaucher 's disease. for this, one blood sampling will be performed.


Eligibility criteria:
inclusion criteria: * adults (age equal or above 18. * patients with gaucher 's disease treated in the "grand ouest" competence center * patients affiliated to social security * informed consent signed exclusion criteria: - children (age\<18)
real-world outcomes on tecfidera (bg00012, dimethyl fumarate) post-tysabri (bg00002, natalizumab)

Conditions: Relapsing-Remitting Multiple Sclerosis

Summary: the primary objective of the study is to evaluate relapse activity, as measured by the proportion of participants relapsed at 12 months, in participants with relapsing-remitting multiple sclerosis (rrms) who transition from tysabri (bg00002) to tecfidera (bg00012) in the real-world setting. the secondary objective is to further evaluate relapse activity, defined as annualized relapse rate (arr), hospitalization and intravenous corticosteroid use, during the first year of tecfidera treatment following transition from tysabri treatment.

the study period will consist of a single time point retrospective medical chart abstraction with no required study visits or procedures. data collection for this study is expected to last up to approximately five months.

Eligibility criteria:
key inclusion criteria: * diagnosis of rrms per mcdonald criteria * received at least 12 months of continuous treatment with tysabri monotherapy prior to initiation of tecfidera. note: continuous treatment with tysabri is defined as treatment uninterrupted by other disease-modifying treatment. * initiated treatment with tecfidera at least 12 months prior to enrollment into the study * patient has sufficient available medical records for data abstraction to meet the objectives of the study key exclusion criteria: * diagnosed with a progressive form of multiple sclerosis (ms) (progressive-relapsing, primary progressive, secondary progressive) * received treatment with any of the following after discontinuation of tysabri and before initiation of treatment with tecfidera (i.e., during washout period): interferon-beta, glatiramer acetate, fingolimod, teriflunomide, rituximab, alemtuzumab, ocrelizumab or any investigational compound for the treatment of rrms * received bg00012, or other formulations of dimethyl fumarate, or fumaderm® or compounded fumarates at any time prior to initiation of treatment with tecfidera * history of progressive multifocal leukoencephalopathy (pml) while on tysabri or within 6 months of discontinuing treatment with tysabri note: other protocol-defined inclusion/exclusion criteria may apply
chronic kidney disease and heart failure with preserved ejection fraction: the role of mitochondrial dysfunction

Conditions: Renal Insufficiency, Chronic; Heart Failure With Preserved Ejection Fraction

Summary: the purpose of this study is to investigate the role of mitochondrial derived oxidative stress on exercise capacity and arterial hemodynamics in hfpef patients with and without chronic kidney disease.

heart failure is a public health epidemic affecting 6.5 million americans. heart failure with preserved ejection fraction (hfpef) accounts for a large burden of heart failure with the incidence and cost associated with the disease projected to double in the next 20 years. the pathophysiology of hfpef has not yet been fully elucidated and no proven therapies for improving outcomes in hfpef currently exist, posing major diagnostic and therapeutic challenges. the addition of chronic kidney disease (ckd) presents a complicated cardio renal syndrome that manifests a distinctly different phenotype and exacerbates the diagnostic and therapeutic challenges of hfpef. this study aims to address the urgent need to establish treatment targets and therapies by investigating potential underlying biological contributors to hfpef and its symptoms. mitochondrial dysfunction is consistently reported in ckd and heart failure. mitochondrial dysfunction has been implicated in cardiac, skeletal muscle and vascular dysfunction and is therefore an attractive target for a 'whole systems' therapeutic approach that would encompass exercise intolerance and abnormal blood vessel hemodynamics. a known contributor to and subsequent cyclical result of mitochondrial dysfunction is an abnormally heightened production of mitochondria derived oxidative stress. this study will address the role of mitochondria derived oxidative stress in mitochondrial dysfunction, exercise intolerance and large blood vessel hemodynamics hfpef patients with and without ckd.

Eligibility criteria:
inclusion criteria: 1. above the age of 18 years 2. a clinical diagnosis of stable stage c heart failure with nyha class ii-iii symptoms 3. a left ventricular ejection fraction \>50% exclusion criteria: 1. current cancer 2. current pregnancy 3. current antioxidant supplement use and unwilling to have a 7-day antioxidant washout period before the beginning the trial and to continue antioxidant disuse throughout the trial. 4. current antiretroviral medication use 5. absolute contraindications to exercise testing according to the american college of sports medicine guidelines 6. fluid overload 7. unable to provide informed consent
objective evaluation of the scratch collapse test with dynamometer, a prospective multicenter trial.

Conditions: Carpal Tunnel Syndrome; Ulnar Nerve Compression

Summary: carpal tunnel syndrome and compression of the ulnar nerve at the elbow are common pathologies, which are treated surgically. diagnosis is usually based on an electromyogram (emg), as well as symptomatology, etiology of typical symptoms and an evocative clinical examination. provocative tests performed in consultation include the tinel and phalen sign for the carpal tunnel, and the tinel and prolonged flexion sign for the ulnar nerve at the elbow. the scratch collapse test (sct) has recently emerged as a new provocation test to help diagnose nerve compression in the upper limb. this non-invasive, pain-free test looks for a reduction in the force of external rotation of the shoulder by applying resistance (the doctor's arm), before and then after a sensory stimulus by "scratching" the area of compression. nevertheless, this test remains controversial and not based on objective measurements. our aim is therefore to assess shoulder external rotation force, and thus tbs, objectively with a dynamometer, before and after stimulation in cases of median nerve compression syndrome at the carpal tunnel and ulnar nerve compression syndrome at the elbow, when these are clinically and electromyographically proven. this test has already been studied in the literature, but the results in terms of sensitivity and specificity are highly disparate. one study has already published negative results on the subject, with the limitation that the trial was monocentric. through this multicenter study, principal investigator wish to highlight the very probable subjectivity of the sct when it is performed. as the resistance is applied by the physician's arm, the investigator cannot determine the force applied against the patient, unlike with a measurement object. the principal investigator expects this study to refute the notion that external shoulder rotation force decreases after trigger zone stimulation in cases of proven nerve compression syndrome. the results of this study will thus make it possible to discontinue the use of this technique if it does not help in the diagnosis of compression. the literature shows a lack of prospective, objective studies involving a large number of patients.


Eligibility criteria:
inclusion criteria: * patient affiliated or entitled to a social security plan * patient who has received informed information about the study * patient of legal age * patient with ulnar nerve or carpal tunnel compression confirmed by emg during preoperative consultation exclusion criteria: * recurrence of nerve compression * any history that may affect arm sensitivity * infra-electromyographic nerve compression syndrome (pure ulnar nerve instability)
expanded access for atb200/at2221 for the treatment of iopd

Conditions: Pompe Disease Infantile-Onset

Summary: this is an expanded access program (eap) for eligible participants designed to provide access to atb200/at2221.

this program is being offered on a patient by patient basis.

Eligibility criteria:
inclusion criteria: 1. patient is male or female and 0 to \< 18 years old. 2. patient has a diagnosis of pompe disease, based on documentation of gaa genotyping demonstrating variants associated with iopd. 3. patient does not currently qualify for or is unable to enroll (eg, due to location, etc) in any commercial drug-sponsored ongoing clinical trial. 4. patient is currently being treated with an approved ert and is declining (eg, worsening in motor function, respiratory function/parameters, cardiac function, etc) based on treating physician's clinical judgement. 5. the patient's legal guardian or representative has given informed consent (and assent when age appropriate for regional requirements) to treatment prior to administering atb200/at2221 in a manner consistent with all national requirements. 6. if of reproductive potential and sexually active, female subjects agree to use a highly effective method of contraception throughout the duration of the treatment and for up to 90 days after their last dose of atb200/at2221. exclusion criteria: 1. patient has a hypersensitivity to any of the excipients in atb200, alglucosidase alfa, or at2221 2. patient has a medical or any other extenuating condition or circumstance that may, in the opinion of the investigator and/or amicus, pose an undue safety risk to the patient. 3. concomitant use of miglitol (eg, glyset), non-at2221 form of miglustat (eg, zavesca), acarbose (eg, precose or glucobay), voglibose (eg, volix, vocarb,or volibo)

Keywords: expanded access compassionate use
a prospective, non-interventional cohort study of subsolid pulmonary nodules

Conditions: Solitary Lung Nodule; Multiple Pulmonary Nodules

Summary: some studies have shown that the model for judging and predicting the growth of sub-solid pulmonary nodules through big data and deep learning can detect nodule growth earlier. since most of the training data come from large foreign samples, most of the validated data are ct data from a single center or a few centers, and their generalization ability needs to be further verified. in order to better study subsolid pulmonary nodules in the lungs in china, we plan to conduct a prospective, multicenter, non-interventional observational cohort study.

through the follow-up of pulmonary nodules, artificial intelligence based on ct was used to study the natural evolution process of subsolid pulmonary nodules, as well as the development law and prognosis of pulmonary subsolid nodules under treatment or no treatment according to clinical guidelines.

Eligibility criteria:
inclusion criteria: * age≥ 18 years old, gender is not limited * confirmed subsolid pulmonary nodule by ct, initial pulmonary nodule ≥ 5 mm (including subsolid components) and ≤ 30 mm, do not shrink or disappear after 3 months follow-up. * ecog 0～2 * able to provide ct in dicom format * informed consent obtained exclusion criteria: * long-term use of immunosuppressants and hormone drugs * history of previous malignancy (if there is no recurrence or carcinoma in situ for more than 5 years after receiving curative therapy, it can be enrolled) * inability to cooperate with regular follow-up * poor general condition, with the life expectancy less than 6 months

Keywords: lung nodule prospective observational study
multispectral fluorescence as a tool to separate healthy and disease related lymphatic anatomies in prostate cancer.

Conditions: Prostate Cancer

Summary: multispectral imaging of the lymphatic draining pattern of the tumor and the abdominall wall/lower limb to evaluate technical feasibility to differentiate these patterns and in the future reduce the amount of complications that result from damage to lymphatic structures.

by preventing unnecessary resection of healthy lymphatic tissue during extended lymph node dissections, the investigators will be able to reduce the degree and amount of complications that result from damage to the lymphatic structures. the investigators aim to evaluate the technical feasibility of imaging two different lymphatic drainage profiles, namely that of healthy tissue (i.e. the lower limbs/abdominal wall) and that of the primary tumour (i.e. prostate). to realize the differentiation, real-time multispectral fluorescence imaging of two spectrally different tracers (the lymphangiographic tracer fluorescein (injected in the lower limbs and abdominal wall) and sentinel node (sn) specific tracer indocyanine green (icg)-99mtc-nanocolloid (injected in the tumour, followed by a control lymphoscintigraphy and spect/ct)) will allow for multispectral (or multicolor) fluorescence. complementary to the routine surgical procedure, e.g. radical prostatectomy with extended pelvic lymph node dissection (eplnd), the lymphatic drainage pattern of both the tumour and of healthy tissue, i.e. lower limbs and abdominal wall, will be determined in the surgical field/surgical specimens.

Eligibility criteria:
inclusion criteria group a: * male, aged ≥ 18 years. * who performance status 0,1, or 2. * written informed consent. * histopathologically confirmed adenocarcinoma of the prostate * increased risk of nodal metastases according to the briganti 2012 nomogram (\> 7%) * scheduled for surgical (laparoscopic) prostatectomy including eplnd- * suitable for rp and eplnd, as per institutional guidelines exclusion criteria group a: * prior abdominal or inguinal surgery (e.g. appendectomy) * history of allergy to iodine, food or medicinal induced urticaria, asthma, eczema, or allergic rhinitis * hyperthyroid or thyroidal adenoma * kidney insufficiency * history of oversensitivity to fluorescite composites * patients using beta-blockers inclusion criteria group b: * male, aged ≥ 18 years. * who performance status 0,1, or 2. * written informed consent. * histopathologically confirmed adenocarcinoma of the prostate * increased risk of nodal metastases according to the briganti 2012 nomogram (\> 15%) * scheduled for surgical (laparoscopic) prostatectomy including eplnd- * suitable for rp and eplnd, as per institutional guidelines exclusion criteria group b: * prior abdominal or inguinal surgery (e.g. appendectomy) * history of allergy to iodine, food or medicinal induced urticaria, asthma, eczema, or allergic rhinitis * hyperthyroid or thyroidal adenoma * kidney insufficiency
covid-19 positive outpatient thrombosis prevention in adults aged 40-80

Conditions: COVID-19

Summary: a multi-center adaptive randomized placebo-controlled platform trial evaluating the efficacy and safety of anti-thrombotic strategies in covid-19 adults not requiring hospitalization at time of diagnosis

the covid-19 outpatient thrombosis prevention trial is a multi-center adaptive randomized double-blind placebo-controlled platform trial to compare the effectiveness of anti-coagulation with anti-platelet agents and with placebo to prevent thrombotic events in patients diagnosed with covid-19 who have evidence of increased inflammation based on elevated d-dimer and hscrp levels, yet are not admitted to hospital as covid-19 related symptoms are currently stable. participants will all be adults between 40 and 79 years who will be enrolled from approximately 100 facilities, such as emergency rooms and other settings where a physician is present to evaluate the patient for inclusion and exclusion criteria.

Eligibility criteria:
inclusion: * covid-19+ in past 14 days * platelets \> 100,000 * egfr \> 30ml/min exclusion: * hospitalized * contradiction/ other indication for anti-coagulation * pregnancy * active cancer
pupillary response to incremental tetanic stimulations under propofol-remifentanil tci

Conditions: Pain

Summary: in patients under standardized steady-state general anesthesia, 6 tetanic stimulations of incremental intensities were applied to the patients. pupillary dilation in response to these stimulations was recorded.

the aim of the study was to investigate a possible correlation between the intensity of a nociceptive stimulus and the magnitude of pupillary reflex dilation in patients anesthetized with propofol and remifentanil target-controlled infusion. anesthetic protocol: 10 minutes of steady-state general anesthesia (before extubation during a scheduled gynecological surgery) with remifentanil target concentration of 1 ng/ml, and propofol target concentration adjusted to maintain bispectral index between 45 and 55. study period: 6 tetanic stimulations of incremental intensities were applied to the patients: 10, 20, 30, 40, 50, 60 milliamps pupillary dilation in response to these stimulations was recorded. then, propofol and remifentanil infusions were discontinued, and patients were extubated.

Eligibility criteria:
inclusion criteria: * scheduled gynecological surgery requiring general anesthesia and orotracheal intubation * written informed consent exclusion criteria: * ophthalmic disease * neurologic disease * preoperative chronic pain or pain medication * medication interfering with autonomous nervous system (beta blockers, alpha agonists, metoclopramide, droperidol, atropine, catecholamines)

Keywords: general anesthesia
pk and safety study of pf-04171327 in healthy japanese and western subjects in fasting and fed conditions

Conditions: Healthy

Summary: this phase 1 study is the first clinical trial in japanese subjects. this study is designed to evaluate the single- and multiple-dose pharmacokinetics, safety and tolerability of pf-04171327 oral tablet in healthy adult japanese and western subjects in fasting and fed conditions.


Eligibility criteria:
inclusion criteria: * healthy male and/or female subjects of non childbearing potential between the ages of 18 and 55 years. * body mass index (bmi) of 17.5 to 30.5 kg/m2; and a total body weight \>45 kg (99 lbs). * japanese subjects who have four japanese grandparents born in japan. exclusion criteria: * confirmed fasting glucose more than 100 mg/dl at screening and day 0 in both cohort a and b. * corticosteroid use of more than 5 mg prednisone equivalent per day for more than 6 weeks. * evidence or history of clinically significant hematological (including anemia), renal, endocrine, pulmonary, gastrointestinal, cardiovascular, hepatic, psychiatric, neurologic, or allergic disease (including drug allergies, but excluding untreated, asymptomatic, seasonal allergies at the time of dosing).

Keywords: phase 1 pharmacokinetics food effect healthy japanese rheumatoid arthritis
health literacy 3d kidney model patient education trial

Conditions: High Blood Pressure; Kidney Disease, Chronic

Summary: high blood pressure has a different rate effect on many minoritized individuals. african americans, as a result of high blood pressure, are six times more likely to go through kidney failure compared to white counterparts. hispanic communities are also disproportionately affected by high blood pressure and, unfortunately, generally have lower knowledge about the effects on the kidneys as well. unfortunately, many minoritized and underserved communities are affected by the weight of the social determinants of health, i.e., housing, education, food, health care, etc., that contribute to large disparities in health outcomes. these factors lead to poor care management, less ability to manage care effectively, and disengagement from care. health education is vital to recognizing and communicating with minoritized patients with hypertension-induced chronic renal disease. as the patient's knowledge of the condition grows, this can significantly influence the trust between clinicians and patients. effective and patient-centered education can inspire patients to be proactive in their prevention strategies with clinicians. this study's overall goal is to assess the utility of a novel patient education tool (3d-printed healthy kidney and diseased kidney models) in optimizing patient education for underserved populations who have hypertension with a potential lead to hypertension-induced chronic renal disease.

the overall aims of the study are to: aim 1: examine the feasibility of creating a high-quality life-size 3d reconstruction of a healthy kidney model and diseased kidney model through collaboration with nih 3d print exchange and the university of kentucky college of engineering. aim 2: assess the acceptability and preliminary efficacy of 3d print model-supported patient education in improving the patients' medical understanding, motivation for self-care, intention to adhere to the clinician's instructions, and better trust between clinician and patient. aim 3: continuously implement of 3d-printed models for patients in new or current care education so that it can be sustainable after the research project is concluded. to achieve aim 2, up to 100 patients with hypertension-induced chronic renal disease will be enrolled and randomized to receive either standard clinician-led patient education (tau) or patient education employing the 3d kidney model. a brief pre-post structured survey will be implemented to measure changes in patients' understanding of their condition, as well as acceptability of the 3d model for those assigned to the experimental education condition.

Eligibility criteria:
inclusion criteria: * ages 18 and older * seeking medical care in study location * current diagnosis of hypertension and renal disease exclusion criteria: -
pursuit: real world use of the eclipse system

Conditions: Fecal Incontinence

Summary: a prospective, open label post-market registry to collect patient reported outcomes and fitting metrics (e.g. sizes used) in subjects using the eclipse system in a commercial setting.

up to 150 subjects enrolled, in up to 25 sites. population of all adult female patients with fecal incontinence (fi) who present at, or are identified at, participating sites are eligible. this includes patients who are newly prescribed eclipse, and those already using eclipse who return for an annual renewal visit during the enrollment period. includes fitting of the patient for the eclipse system with collection of relevant health history data. patients are provided a temporary device for a trial usage period. multiple device sizes may be attempted to find the correct fit for the patient, at which point the patient is provided with the long term use eclipse insert and enters the treatment period. during the fitting and treatment period, patient experience will be assessed by surveys, st. mark's (vaizey) scores, and the validated global pgi-i index (patient global impression of improvement). surveys will be perform at the fitting follow up and at 12 months of device usage, and optionally at 3, 6, and 9 months of device usage.

Eligibility criteria:
inclusion criteria: 1. adult female 2. diagnosis of fecal incontinence 3. clinician recommendation of the eclipse system 4. subject provides informed consent and hipaa authorization no exclusion criteria.