Kornimate/medical-research-clean huggingface dataset:
a first-in-human study of epi-321 in facioscapulohumeral muscular dystrophy

Conditions: Facioscapulohumeral Muscular Dystrophy

Summary: the goal of this clinical trial is to learn how safe and tolerable epi-321 is and whether there may be early signs it is working in male or female adult (18 to 75 years) participants with facioscapulohumeral muscular dystrophy (fshd) type 1 condition. the main questions it aims to answer are: how safe is epi-321 and how well can people handle it over time? how does epi-321 interact with its target and does it show early signs of working? participants will receive a single dose of epi-321 through a vein while being closely watched in a hospital and visit the clinic regularly for tests and checkups for about 5 years after getting epi-321.

epi-321 is an investigational drug product comprising a recombinant adeno-associated viral vector, serotype rh74 (aavrh74), for the delivery of genetic material encoding an epigenetic editor designed to address the root case of fshd. aavrh74 has been shown to transduce human skeletal muscle efficiently in the clinical experience. epi-321's transgene product, a non-cutting, nuclease-dead mini, clustered regularly interspaced short palindromic repeat (crispr)-associated protein (dcasonyx) with fuse epigenetic modulators, is designed to selectively bind the d4z4 repeat region via the accompanying guide rna, methylate cpg groups within the region near the dux4 gene on chromosome 4q35, and thus repress the expression of toxic dux4 protein, ameliorating the downstream pathology that drives fshd. as it is under a muscle-specific promoter, the dcasonyx-fused protein is expected to be preferentially and actively expressed in muscle tissue following a single intravenous (iv) dose. epi-321-02 clinical trial is an open label dose ascending study of epi-321 for safety and tolerability to determine the best dose for a future trial of drug activity. two dose levels will be evaluated. in addition, this study will collect secondary outcome data on muscle function, imaging characteristics, and other markers of disease activity at the baseline and throughout the study to assess their utility as measures of drug activity in a future clinical trial.

Eligibility criteria:
inclusion criteria: * able and willing to provide informed consent * male or female 18 to 75 years of age * clinical diagnosis of fshd with genetic type 1 * fshd ricci clinical severity score 2 to 4 (on 5-point scale) * has adequate liver function * has adequate kidney function exclusion criteria: * has an anti-aavrh74 total binding antibody titer \> 1:400 * requires a walker or wheelchair for ambulation * pregnant and/or breastfeeding at baseline or is planning to become pregnant during the first 12 months following epi-321 administration * has fshd type 2 * has a concurrent or past medical conditions could jeopardize the safety of the participant

Keywords: facioscapulohumeral muscular dystrophy epi-321 muscular dystrophy
randomized, double-blind, multicenter, placebo-controlled study of pregabalin for pain associated with diabetic peripheral neuropathy

Conditions: Diabetic Neuropathy, Painful

Summary: to evaluate the efficacy and safety of pregabalin at 300 mg/day and 600 mg/day (bid) in patients with painful diabetic peripheral neuropathy.


Eligibility criteria:
inclusion criteria: * visual analogue scale (vas) of pain is higher than 40 mm. * diagnosis of type 1 or 2 diabetes mellitus for at least 1 year exclusion criteria: * malignancy within the past 2 years. * neurologic disorders unrelated to diabetic neuropathy that may confuse the assessment of neuropathy pain
childhood asthma and environment research study - chaser study

Conditions: Asthma

Summary: "childhood asthma and environment research study - chaser study" is an observational longitudinal study in outpatient clinic of pediatric allergology \& pulmonology (pap) of respiratory disease research center (rdrc) within the institute of biomedicine and molecular immunology (ibim) of the national research council (cnr) of palermo (rdrc-ibim cnr), italy. the first phase - transversal study will identify determinants of pediatric asthma control the second phase - longitudinal study, in patients with persistent asthma, will be focused on: * predictors for asthma exacerbations and loss of control * real life adherence to asthma therapy guidelines * phenotypic characterization through the collection of saliva samples * assessment of alterations and inflammatory changes in the nasal mucosa through nasal brushing * evaluation of the presence of 8-isoprostane, nicotine, cotinine and bisphenol-a through urine analysis. chaser project is expected to improve the knowledge of pediatric asthma in patients with different level of control, identifying predictors for asthma exacerbations and loss of control.


Eligibility criteria:
inclusion criteria: * age 5-16 years * asthma * not asthma exclusion criteria: * chest deformity * active smoking
study of orbec® with prednisone therapy in the treatment of patients with graft versus host disease (gvhd)

Conditions: Acute Gastrointestinal Graft vs Host Disease

Summary: use of an oral topically-active glucocorticoid with limited side effects will control the gastrointestinal inflammatory process of gvhd and minimize glucocorticoid exposure.


Eligibility criteria:
inclusion criteria: * receipt of allogeneic hematopoietic cell transplant * diagnosis of gi graft vs. host disease (gvhd) * no gi infection * must be able to swallow tablets * must be able to read and understand informed consent * adequate birth control methods for the duration of the study exclusion criteria: * significant skin gvhd * liver gvhd * persistent vomiting * hiv positive * pregnancy/lactation

Keywords: bdp orbec gvhd beclomethasone dipropionate bone marrow transplant hematopoietic cell transplant hct stem cell transplant marrow transplant sct beclomethasone 17,21-dipropionate
s1119 peruvian h. pylori water contamination study

Conditions: Helicobacter Pylori Infection

Summary: the objective of this study is to elucidate the role of drinking water derived from the public source in lima, peru and transmission of h. pylori infection using genotypic techniques.

the objective of this study is to detect the presence of helicobacter pylori in the drinking water derived from the public network by molecular and genomic techniques in districts of lima (brena, chorrillos, comas, el agustino, la victoria, lince, los olivos, miraflores, puente piedra, rimac, san isidro, san juan de lurigancho, san martin de porres, villa el salvador, and villa maria del triunfo) with high and low incidence of gastric carcinoma. the secondary objective of this study is to establish correlations between the findings in water and those of the stomach of patients with symptoms of dyspepsia who live in those districts.

Eligibility criteria:
inclusion criteria: * adults between the ages of 21-70 years old who have lived for more than 10 years in the lima districts listed in protocol, * who have a biopsy proven diagnosis of infection by h. pylori. * participants must be in good general condition, mentally competent and able to provide informed consent; * must not show evidence of dehydration or jaundice; * must be willing to complete the patient questionnaires. * participants must not have known allergies to any of the antibiotics used in the study. exclusion criteria: * pregnancy

Keywords: helicobacter pylori water contamination lima, peru
validation study of posturology platforms for evaluating postural control of hemiparetic and neuro-muscular patients

Conditions: Neuromuscular Diseases; Hemiparesis; Charcot-Marie-Tooth Disease

Summary: the instrumental evaluation of standing postural control by posturographic analysis supplements the clinical evaluation and, as such, is recommended by the french national authority for health (has 2007). the quantitative data obtained after a standardized postural examination appear relevant for the longitudinal follow-up of neuromuscular patients and hemiparetic patients. neuro muscular (nm) diseases are progressive, these instrumental evaluations can highlight a deterioration or stabilization of postural control possibly not observable with the scores on clinical scales. the k-force plates, recently developed appear to be an interesting alternative to the stabilometry platforms currently used. static and dynamic conditions in upright position are tested. moreover, posturologic, kinematics data and clinical scores are correlated to increase understanding of strategies during postural control tasks in patients with stroke and in patients with nm disorders.

to date, no clinical validation study of these k-force plates force platforms has been proposed. the investigator hypothesize that the data obtained by the plates are comparable to the measurements made by the reference tool, the amti platforms during a static and dynamic posturographic examination in mnm, hemiparetic and healthy subjects. the strategies of the patients (stroke and nm) during the dynamic task are also explored with kinematic analysis in correlation with data from force platforms and clinical scores.

Eligibility criteria:
"eligible couples must present all the following criteria : * signing of the informed consent form * men and women aged from 18 * single stroke hemiparetic patient, or patient with neuro-muscular disease, or healthy subject free of neurological disorder and other disease * patient able to stand alone at least 30 seconds without technical assistance (multiple repetitions) to be included, patient should not present any of the following criteria : * bilateral brain injury, cerebellar syndrome * other disease or defect that may interfere with the study such as visual, vestibular or other related uncontrolled medical condition * major cognitive impairment that does not allow evaluation * pregnant women, breastfeeding * non affiliated to a social security scheme"

Keywords: neuromuscular diseases charcot-marie-tooth disease hemiparesis
study to compare two formulations of cp-675,206 monoclonal antibody

Conditions: Melanoma

Summary: this is an open-label, 2-arm study in which patients are randomized to receive either the formulation that is being used in clinical trials or the formulation that will be used when if the drug becomes commercially available. the purpose of this study is to compare the pharmacokinetics of the two formulations.


Eligibility criteria:
inclusion criteria: * stage iii or stage iv melanoma * no evidence of disease following resection of melanoma lesions * recovered from all prior surgical or adjuvant treatment-related toxicities exclusion criteria: * history of chronic inflammatory or autoimmune disease * history of inflammatory bowel disease
comparison of human insulin to insulin inhalation solution in children with type 1 diabetes

Conditions: Diabetes; Diabetes Mellitus, Type 1

Summary: this trial is conducted in europe. the aim of this trial is to compare the pharmacokinetic profile of two different methods of insulin administration in children with type 1 diabetes.


Eligibility criteria:
inclusion criteria: * type 1 diabetes * treatment for at least 12 months * hba1c lesser or equal to 11.0% * body weight between 25 and 80 kg * capable to use the device exclusion criteria: * any diseases other than diabetes requiring prescriptive medication * known or suspected allergy to trial product or related products * active proliferative retinopathy as judged by the investigator * recurrent severe hypoglycaemia as judged by the investigator * the receipt of any investigational drug within 4 weeks prior to this trial
the effects of sedative on the fluid responsiveness in critically ill patients

Conditions: Shock; Sepsis

Summary: hypotension and bradycardia are often observed following induction of dexmedetomidine or propofol sedation.cardiac preload decrease by sedative agents was often considered as one of main causes for this hypotension.the investigators hypothesized that hypotension after induction of sedation is caused by decrease of preload by sedative agents,and passive leg raising (plr)test could predict this event.dexmedetomidine or propofol infusion in patients with circulatory failure decrease cardiac preload and enhance preload-dependency and fluid responsiveness.

in this clinical trial,the investigators hypothesized dexmedetomidine or propofol infusion in patients with circulatory failure decrease cardiac preload and enhanced preload-dependency and fluid responsiveness,and plr induced pulse pressure variation (plr-δpp) could predict the hypotension during dexmedetomidine induction in critically ill patients.before dexmedetomidine or propofol infusion, the investigators will conduct passive leg raising test. at first, the patient's trunk was elevated 45 degrees for the first set of measurements. then, the lower limbs were raised to a 45° angle while the patient's trunk was lowered to a supine position to measure peak bp (usually within 30-90 s). hemodynamic profiles planned to be measured are sbp, dbp, cvp and hr.sedation was induced with a dexmedetomidine 0.5μg/kg over 10 min loading, followed by a maintenance infusion of 0.2-0.7 μg/kg/h for one hour. the dose of propofol was titrated targeting the richmond agitation sedation scale (rass) ranged from -2 to -1 (bispectral index: 60-75).hypotension was defined as a sbp less than 80 mmhg or dbp was less than 50 mmhg, or greater than 30% decrease from baseline for longer than 60 s. bradycardia was defined a hr 50 /min or greater than 30% change from baseline for longer than 60 s. receiver operator characteristic curve analysis was performed to evaluate sensitivity and specificity of the value plr-δpp to detect patients at high risk of hypotension.

Eligibility criteria:
inclusion criteria: * patients with at least one of the clinical manifestations of acute circulatory failure; patients undergoing dexmedetomidine or propofol infusion exclusion criteria: * deep venous thrombosis or elastic compression stocking * an increase in the intra-abdominal pressure confirmed by clinical examination * serious central nervous system pathology (head trauma, severe dementia, acute stroke, uncontrolled seizures) * severe liver disease(child-pugh class c) * unstable angina or acute myocardial infarction * left ventricular ejection fraction less than 30% * heart rate(hr) less than 50/min, heart block with second- or third degree * systolic blood pressure (sbp) less than 90 mmhg despite 2 vasopressors infusion continuously before the begin of dexmedetomidine infusion
breast density measurements in digital mammography and breast tomosynthesis systems of different pixel size

Conditions: Mammographic Breast Density

Summary: breast cancer (bc) is the most frequently diagnosed cancer in women worldwide. the cause of developing bc is currently unknown. however, there are several risk factors, such as volumetric breast density (vbd), which have been proved to have a relationship with bc. the assessment of breast density is typically performed subjectively using the 4-classes density classification described by american college of radiology. however, in the last years, automated breast density software tools have been developed (e.g. volpara, quantra). in this work, vbd measurements from volparatm (v. 1.5.4.0) will be compared between full-field digital mammography (ffdm) and digital breast tomosynthesis (dbt) of the same pacient ('combo mode'). images will be acquired in a hologic selenia dimensions, the most common ffdm/dtb system, where image resolution is different for ffdm (70 um) and dbt (140 um). this study (1) evaluates the consistency between volpara's breast density measurements and spatial distribution for the two imaging techniques (ffdm and dbt) in a hologic selenia dimensions, and (2) compares vdb measurements with other similar tool (quantra) and radiologists breast density manual classification.

monocentric, prospective and observational study which will analyse a series of 300 cases (ffdm + dbt) from consecutive women. image acquisitions will take place within the área de radiología mamaria y ginecológica located at parc taulí hospital universitari, sabadell. the purposes of this study will be explained to each potential candidate and if agreed to participate, a consent form will be signed. inclusion criteria: * subjects over 18 years old. * acceptance of participation through a signed consent form. * asymptomatic subjects exclusion criteria: * subjects refusing to participate or sign the consent form. * subjects with prior history of breast surgery or biopsy with markers. * subjects with breast prostheses.

Eligibility criteria:
inclusion criteria: * acceptance of participation through a signed consent form. * asymptomatic subjects exclusion criteria: * subjects refusing to participate or sign the consent form. * subjects with prior history of breast surgery or biopsy with markers. * subjects with breast prostheses. * subjects with biopsy marker clips

Keywords: breast cancer breast density volpara quantra bi-rads digital mammography digital breast tomosynthesis
sagittal balance through ages

Conditions: Spinal Deformity

Summary: the parameters of the sagittal balance of the cervical, thoracic and lumbar spine vary according to the types of spino-pelvic organization (types 1-4 according to roussouly). these parameters have been described as a function of age in previous scientific work without taking into account the pelvic anatomy (pelvic incidence and spino-pelvic organization). this work, carried out within the framework of a round table of the french society of spinal surgery (sfcr), would make it possible to provide a more detailed repository of existing radiographic parameters.


Eligibility criteria:
inclusion criteria: * minor or major subject aged 5 to 100 * subject having carried out a telecolonne or eos type x-ray for spinal balance (low back pain, sciatica, cervico-brachial neuralgia, inequality of length of the lower limbs) in spinal surgery consultation * subject not having expressed its opposition, after information, to the re-use of its data for the purposes of this research. exclusion criteria: * subject with surgical history, fractures, osteoporotic packing, vertebral deformation (scoliosis, kyphosis, spondylolisthesis grades 2-5), vertebral osteolysis (spondylodiscitis, tumor or metastasis). * subject who expressed his opposition to participate in the study * subject under guardianship or curatorship * subject under judicial protection

Keywords: spinal deformity low back pain sciatica cervico brachial neuralgia
erector spinae versus intercostal nerve blocks with liposomal bupivacaine for analgesia in thoracic surgery

Conditions: Lung Cancers

Summary: this clinical trial compares efficacy in postoperative pain management in thoracic surgery between erector spinae block versus liposomal bupivacaine injections.


Eligibility criteria:
inclusion criteria: * video assisted pulmonary resection, pleurodesis or decortication exclusion criteria: * allergy to local anesthetics * patient undergoing bilateral surgery * surgery is emergent as deemed by the principal investigator
the course of low back pain

Conditions: Low Back Pain; Type of Leg Pain (Radicular, Non-radicular); Lifestyle (Sedentary Behavior and Physical Activity); Start Back Screening Tool

Summary: this study aims to research the possible associations of education level, sedentary lifestyle, and type of low back pain (radicular vs non-radicular) with the course of low back pain and recovery rate.


Eligibility criteria:
inclusion criteria: * low back pain * age 18 years or older exclusion criteria: * pregnancy
inflammasome activation via circulating metabolites

Conditions: Human Immunodeficiency Virus

Summary: the clinical challenges confronting patients with hiv has shifted over the past 10 years from acquired immunodeficiency syndrome to chronic diseases including atherosclerosis, neurocognitive disorders, and osteoporosis. chronic low grade inflammation and monocyte activation have been consistently associated with comorbidities in hiv patients. indeed, recent studies indicate that inflammatory mediators including il-6, il-1, scd14 and s cd163 produced by monocytes, but not t-cell activation, predict non-aids-related events in virologically suppressed hiv-infected persons treated with combined antiretroviral therapy (cart), highlighting the important role of monocyte activation in the occurrence of comorbidities in cart-treated hiv infected patients. yet, the underlying molecular pathways of persistent monocyte activation in cart treated hiv-infected patients remains incompletely characterized. our preliminary results: 1/ establish a link between the activation of the inflammasome, the increased of pyrimidine-derived metabolites and the cardiovascular risk in a cohort of elderly patients; 2/ show that treated hiv-patients are characterized by increased soluble il-1b or il-18 in their blood suggesting that the inflammasome pathway is activated. objectives: in this study we will characterize the molecular pathways underlying persistent monocyte activation in treated hiv patients, through the implication of the activation of the inflammasome machinery: 1. characterization of nod like receptor (nlr) expression in monocytes for il-1b and il-18 secretion (inflammasome activation); 2. characterization of circulating metabolites that active the inflammasome machinery; 3. evaluation of the link between the activation of the inflammasome, the increased of circulating metabolites and the non-aids related comorbidities.


Eligibility criteria:
hiv patients : inclusion criteria: * aged 18 years and above * hiv-1 infected patients * enrolled in the ciadis substudy of the anrs co3 aquitaine cohort * patients receiving antiretroviral therapy * hiv-1 rna load below the detection limit of 40 copies/ml * with a written and signed informed consent exclusion criteria: * hiv-2 or hiv-1/hiv-2 co-infection control patients : inclusion criteria: * aged 18 years and above exclusion criteria: * hiv-1, hiv-2 or hiv-1/hiv-2 infection

Keywords: hiv inflammasome