Kornimate/medical-research-clean huggingface dataset:
investigating muscle failure in diabetic myopathy

Conditions: Diabetes Mellitus; Obesity; Chronic Hyperglycaemia; Weight Loss; Diabetic Myopathy; Sarcopenia

Summary: the goal of this observational study is to assess if diabetes and obesity are independently related to functional and structural muscle deficits, and how muscular deficits relate to metabolic properties of diabetes and obesity. all studies will include clinical muscle strength and contractile examinations, functional tests, and mr imaging and spectroscopy techniques. the main questions this project aims to answer are: 1. is chronic hyperglycemia in type 1 and 2 diabetes associated with functional and structural deficits of skeletal muscles unrelated to the presence of neuropathy? 2. is obesity associated with functional and structural impairments of skeletal muscles unrelated to the presence of type 2 diabetes ? 3. does weight loss improve muscle metabolic flexibility and economy and modify skeletal muscle function and structure in obese subjects with and without type 2 diabetes? the project will include three studies, intended to answer the hypotheses listed above: study 1: evaluation of functional and structural muscular deficits of diabetic myopathy in relation to prolonged hyperglycemia prior to and 6 months following glycemic improvement in patients with type 1 and 2 diabetes study 2: functional and structural muscular deficits in severely obese subjects with and without type 2 diabetes prior to assisted weight loss. study 3: changes in functional and structural muscle properties following assisted weight loss in severely obese subjects with and without type 2 diabetes - a 1-year follow-up study.


Eligibility criteria:
inclusion criteria: study 1: * age: 18-60 years * bmi: 18.5 - 39, minimum weight = 50kg * chronic hyperglycemia: hba1c of ≥ 70 mmol/mol with a duration of ≥ 2 months prior to enrollment * physical activity: less than 3 x 60 min of structured physical activity per week study 2 \& 3: * age: 25-60 years * bmi: ≥ 35 * physical activity: less than 3 x 60 min of structured physical activity per week healthy control participants: * age: 18-60 years * bmi: 18.5 - 30, minimum weight = 50kg * physical activity: less than 3 x 60 min of structured physical activity per week exclusion criteria: study 1, 2, 3 and healthy controls: * diabetic neuropathy with expected motor deficits * uncontrolled cardiovascular or pulmonary disease, peripheral vascular disease, osteoarthropathy of the lower extremity, or any neurological og rheumatological disease which may affect muscle function, as well as any other disease that may effect ones ability to perform physical activity. * any magnetic resonance contraindications * any condition that by the principal investigator is expected to affect the participants ability to execute the study elements specifically for healthy control participants: * the presence of diabetes or pre-diabetes (hba1c ≥42 mmol/mol)
secondary findings from high-throughput sequencing: how to announce them with respect to the patient's needs

Conditions: Development Disorders

Summary: high-throughput whole-genome sequencing (wgs) is bringing new opportunities in the diagnosis of rare diseases. it will more frequently lead to a primary diagnosis (aim of the genetic consultation), but it may also lead to the discovery of mutations not related to the patient's disease. these findings are called "incidental findings" (if) and may give rise to preventive or curative interventions in a personalised medicine approach. the question of proposing to patients access to all or part of these findings is a matter of debate in france and elsewhere. this question has given rise to new challenges and new needs that professionals must respond to by implementing appropriate management and new skills. it raises specific ethical issues, which require precise understanding of the expectations and experiences of patients. patients' diagnostic trajectories must also meet criteria for efficacy and financial and organisational sustainability for the healthcare establishments and, for the healthcare system. our project aims to assess the expectations of patients/parents with regard to this opportunity, and to determine how information should be provided to patients and how they should be accompanied to ensure efficient and appropriate management.


Eligibility criteria:
inclusion criteria: * adults or parents of of deceased foetuses/children (alive or deceased) or adults living under guardianship or deceased, with development disorders(dd) who will undergo wes for the first time for diagnostic purposes * consent to take part in the study * desire to screen for at least one group of if * able to speak fluent french. exclusion criteria: * patients withough national health insurance cover * absence of consent from the patient or his/her legal representative
the effect of ventilated vest on thermoregulation during exercise in hot environment

Conditions: Body Temperature Changes

Summary: the purpose of this preliminary experiment is to measure the effect of a novel ventilation system on the physiological strain during exercise in high heat load while wearing ballistic protective vest. ten young and healthy participants will participate in the study. they will all go through a six days heat acclimation period in a heat chamber according to a valid protocol. afterwards they will be randomly exposed to 4 conditions during four separate consecutive days.

ten young and healthy civilian participants will participate in the study. they will all go through a six days heat acclimation period in a heat chamber according to a valid protocol. after heat acclimation the participant will be randomly exposed to 4 conditions during four separate consecutive days: 1) walking on a treadmill for two hours in hot-wet conditions (30oc, 70% rh) while wearing military uniforms and a vest (control group); 2) walking on a treadmill for two hours in hot-dry conditions (40oc, 30% rh) while wearing military uniforms and a vest (control group); 3) walking on a treadmill for two hours in hot-wet conditions (30oc, 70% rh) while wearing military uniforms and a vest with a ventilator (experimental group); 4) walking on a treadmill for two hours in hot-dry conditions (40oc, 30% rh) while wearing military uniforms and a vest with a ventilator (experimental group). during the heat exposures, rectal temperature, skin temperature and heart rate will be continuously monitored. subjective comfort measures, heat strain indices, sweat rate and sweat evaporation will be also measured/calculated. the differences in the above measures, during exposure to exercise in the heat between ventilator and no ventilator will be then analyzed.

Eligibility criteria:
inclusion criteria: * age 21-28 * healthy * after medical checkup * after signing concent form exclusion criteria: * heart disease * respiratory disease * baseline bp above 140/90 mmhg * sleep disorders * diabetes * anhydrosis * skin disease * acute illness in last 3 days prior to the examination
intrathecal enzyme replacement therapy for spinal cord compression in mucopolysaccharidosis (mps) i

Conditions: Mucopolysaccharidosis I; Lysosomal Storage Diseases; Spinal Cord Compression

Summary: the investigators are studying the use of enzyme replacement therapy into the spinal fluid for treatment of spinal cord compression in the hurler-scheie and scheie forms of mucopolysaccharidosis i (mps i). funding source -- fda oopd

enzyme replacement therapy (ert) has been developed for mucopolysaccharidosis i (mps i), a lysosomal storage disorder. ert helps many physical ailments due to the disease, but does not treat the central nervous system, due to inability to cross the blood brain barrier. our purpose is to test delivery of ert to the spinal fluid via intrathecal injection in patients with mps i. in this pilot study, we will use recombinant human α-l-iduronidase administered intrathecally once per month for four months to individuals with the hurler-scheie and scheie forms of mps i and spinal cord compression. if successful, intrathecal delivery could represent a practical, straightforward method of treating central nervous system disease due to lysosomal storage.

Eligibility criteria:
inclusion criteria: * hurler-scheie,scheie form of mps i, of hurler 2 years after hematopoietic stem cell transplantation * spinal cord compression * age greater than 8 years * able to provide legal informed consent * aware of clinical treatment option of observation without treatment or surgical decompression * negative urine pregnancy test at screening (non-sterile females of child-bearing potential only) * currently using two acceptable methods of birth control (non-sterile females of child-bearing potential who are sexually active only) * willing and able to comply with study procedures exclusion criteria: * severe (hurler) form of mps i * desires surgical or medical treatment of spinal cord compression * spinal cord compression that warrants immediate surgical intervention * pregnancy or lactation * hematopoietic stem cell transplantation within 2 years of study enrollment * receipt of an investigational drug within 30 days of enrollment * infusion reactions to laronidase that required medical intervention, prophylaxis, or altered enzyme administration * significant anti-iduronidase antibody titer * recent initiation of intravenous laronidase (within past 6 months) * presence of cervical subluxation or similar external pathology as the major cause of cord compression symptoms for which surgical intervention should be immediately undertaken

Keywords: mucopolysaccharidosis hurler-scheie scheie laronidase spinal cord compression central nervous system enzyme replacement therapy intrathecal la biomed
a study of tnm001 in chinese healthy preterm and term infants

Conditions: Respiratory Syncytial Virus Infections

Summary: the purpose of this clinical trial is to evaluate the safety, tolerability and pharmacokinetics (pk) profile of tnm001 injection in healthy preterm and term infants. the main questions it aims to answer are: * the safety and tolerability of tnm001 injection * the pharmacokinetic (pk) profile of tnm001

this phase ib/iia study is designed to assess the safety, tolerability, and pharmacokinetics (pk) profile tnm001 in healthy preterm and term infants. this study will also compare the incidence of respiratory syncytial virus（rsv） infection between different doses of tnm001 and placebo, which will be used to select the dose to be studied in the later phase of clinical trials of tnm001. approximately 30 subjects will be randomized and will be dosed once on day 1 and followed up until day 151. around 6 investigational study centres participate in the study.

Eligibility criteria:
key inclusion criteria: 1. healthy preterm infants and term infants within 1 year old of age 2. infants who are in the first rsv infection season at the time of randomization key exclusion criteria: 1. any fever or acute illness within 7 days prior to dosing 2. lrti prior to randomization 3. received any anti-rsv monoclonal antibody or rsv vaccine 4. any other circumstances that, in the opinion of the investigator, may interfere with the assessment of the study drug or the interpretation of the study results 5. the subject is a child of the investigator or his/her subordinate study personnel or relatives or sponsor staff
pharmaco-economic study of ultiva in intensive care unit(icu)subjects

Conditions: Sedation

Summary: the study will evaluate the pharmaco-economic consequences of the use of a remifentanil based regimen compared with a conventional sedative based regimen in terms of duration of mechanical ventilation, length of stay in icu, difference in extubation time and use of concomitant sedative agents.


Eligibility criteria:
inclusion criteria: * icu patients with an expected duration of mechanical ventilation for 2 to 3 days and requiring analgesia and sedation. exclusion criteria: * icu patients resuscitated in the previous 24 hours, neurotrauma or expecting major surgery, not likely to survive of with limit care status.

Keywords: icu mechanical ventilation analgesia sedation
the scandinavian aed and mobile bystander activation trial

Conditions: Out-Of-Hospital Cardiac Arrest; Cardiac Arrest, Sudden

Summary: sudden cardiac death is a major health problem in the western world. in europe alone nearly 300 000 patients are affected annually. the majority of victims suffering from an out-of-hospital cardiac arrest (ohca have an initial cardiac rhythm that can be treated by means of defibrillation. in most emergency medical systems (ems) time to defibrillation is too long and survival averages 10 %.however, when laymen operated automated external defibrillators (aeds) are used within the first minutes, 7 out of 10 may survive. the aim of "the scandinavian aed and mobile bystander activation" (samba) trial is to evaluate if a mobile phone positioning system and a smartphone application will increase the proportions of patients with an attached automated external defibrillator (aed) before arrival of the emergence medical system (ems). mobile phone technology and a smartphone application will be used identify and recruit nearby cpr-trained lay people and automated external defibrillators (aeds) to patients suffering out-of-hospital cardiac arrest (ohca). the system is currently running in stockholm sweden and in the gothenburg region.

early defibrillation in out-of-hospital cardiac arrest is associated with high survival rates. if the operator at the dispatch center suspects an out-of-hospital cardiac arrest he or she will activate the mobile positioning system (mps) wich is integrated in the operators computer environment. after activation, the system will locate all cpr-trained volunteers nearby the suspected ohca. by computer based 1:1 randomization lay volunteers will be alerted and dispatched in 50 % of all suspected ohcas in where the system is triggered by the dispatchers. in the intervention group, the activation of the system is supposed to result in lay volunteers to be alerted and directed to fetch the nearest aed and commence early defibrillation. dependent on geographical and logistical circumstances, at a minimum one lay volunteer or more will be alerted to provide bystander cpr only. in the control group the mps will be activated and lay responders will be dispatched to suspected ohca to perform cpr only. in both groups the ordinary ems services are dispatched in a regular fashion.

Eligibility criteria:
inclusion criteria: * all ems-treated ohcas within the stockholm county, copenhagen and vgr area and in whom the mobile positioning system is activated at the emergency dispatch center exclusion criteria: * crew witnessed ohcas * patients \<8 years of age * ohcas due to trauma, intoxication, or suicide * ohcas not treated by the ems due to ethical reasons or obvious signs of death. * no ohca cases
the adherence to oral anticoagulant in chinese patients with nvaf

Conditions: Nonvalvular Atrial Fibrillation

Summary: investigate the compliance of oral anticoagulants in chinese nvaf patients and find out the causes, so as to provide basis for formulating effective measures to improve the compliance of oral anticoagulants.

1. the general information about these patients was recorded such as gender, age, smoking history, drinking history, heart failure, hypertension, diabetes, coronary heart disease, stroke history, tia history, peripheral vascular disease, etc. cha2ds2-vasc score and has-bled score were recorded. 2. medication information and follow-up information was collected.for the patients included in the study, the medication records of oral anticoagulants (outpatient, emergency, hospitalization), adverse reactions, anticoagulant use history, drug withdrawal or dressing change, disease changes, etc. within three years were recorded by referring to the patient's medical records.

Eligibility criteria:
inclusion criteria: nvaf; cha2ds2-vasc≥2（femal≥3）; after starting the medication, the duration of continuous medication was more than 3 months exclusion criteria: the patients had the records of anticoagulants in other hospitals; patients lost to follow-up; patients who do not need long-term anticoagulants after successful radiofrequency ablation or left atrial appendage occlusion. the follow-up time was 3 years or the following conditions occurred: the patient's condition changed and no longer used oral anticoagulants or died, the follow-up was stopped.

Keywords: adherence new oral anticoagulants
the role of serum leptin levels in multiple sclerosis disease activity while on interferon beta 1a (rebif) treatment

Conditions: Multiple Sclerosis

Summary: to determine a correlation between serum leptin levels, nitric oxide preceding a relapse and change in leptin and nitric oxide levels during exacerbation in relapsing remitting multiple sclerosis patients.

this is an observational pilot study in patients with definitive relapsing-remitting multiple sclerosis to determine whether there is a correlation in serum leptin levels and serum nitric oxide levels during a clinically observed increase in disease activity. each subject will followed for 12 months while taking interferon beta-1a subcutaneous injections 3 times a week.

Eligibility criteria:
inclusion criteria: * confirmed diagnosis of multiple sclerosis per update of mcdonald criteria * initiating treatment with interferon beta 1a subcutaneous injection with minimal dosing of 22mcg. * 18-60 years of age. * women who are not pregnant or breast feeding, and who do not intend to become pregnant. * stable dose of concomitant medications for 30 days prior to baseline. efforts should be made to maintain steady doses of concomitant medications for the duration of the study. exclusion criteria: * treatment on other interferon preparations, glatiramer acetate, in last 30 days or 6 months on natalizumab, mitoxantrone, cyclosporine or other immunomodulators as determined by the pi. * treatment with any other investigational treatments within the past year or treatment with any investigational treatments for multiple sclerosis within the last year. * any progressive form of ms. * known hypersensitivity to interferon beta 1a. * inability to administer subcutaneous injections * inability to undergo laboratory evaluation. * seropositivity for hiv (by medical history) * diabetes mellitus tyle i or ii (by medical history) * hepatitis b or c (by medical history) * uncontrolled psychiatric disorder. * any unstable illness that the investigator's opinion precludes participation in this study. * inability to maintain compliance with study protocol. * implanted devices or metal which would contraindicate mri. * clinically significant abnormalities in cbc or cmp * mmse \<25

Keywords: ms serum leptin levels during relapse multiple sclerosis relapse
a study to evaluate the safety, pharmacokinetics, and antitumor activity of ak114

Conditions: Advanced or Metastatic Solid Tumors; Malignancy; Metastasis

Summary: a phase 1 study to evaluate the safety, tolerability, pk, immunogenicity, pharmacodynamics, and preliminary antitumor activity of ak114.

this is a first-in-human (fih), phase 1a, multicenter, open-label, single-arm dose-escalation study of ak114 to evaluate the safety, tolerability, pk, pharmacodynamics, antitumor activity and immunogenicity in adult subjects with advanced or metastatic solid tumors. the study is comprised of dose escalation phase. approximately 30 subjects will be treated in this study.

Eligibility criteria:
inclusion criteria: 1. written and signed informed consent 2. age ≥ 18 3. subjects must have histologically or cytologically confirmed advanced or metastatic solid tumor 4. subject must have at least one measurable lesion according to recist v1.1 5. eastern cooperative oncology group (ecog) performance status score ≤ 1 6. at the time of day 1 of the study, subjects with central nervous system (cns) metastases must have been treated 7. available archived tumor tissue sample (block or a minimum of 10 unstained slides of formalin-fixed paraffin-embedded tissues) to allow for correlative biomarker studies 8. subjects may opt to provide two fresh biopsy samples (pretreatment and on treatment), where clinically appropriate 9. adequate organ function 10. use acceptable method of contraception from screening, and must agree to continue for 120 days after the final dose of investigational product exclusion criteria: 1. history of severe hypersensitivity reactions to other monoclonal antibodies 2. history or concurrent gastrointestinal perforation, surgery and wound healing complications, hemorrhage events 3. patients with clinically significant cardiovascular disease 4. subjects with a condition requiring systemic treatment with either corticosteroids (\> 10 mg daily prednisone equivalents) or other immunosuppressive medications within 14 days of investigational product administration 5. active or prior documented autoimmune disease within the past 2 years 6. history of primary immunodeficiency 7. history of organ transplant or hematopoietic stem cell that requires use of immunosuppressive medications 8. known allergy or reaction to any component of the investigational product formulation. 9. history of interstitial lung disease or noninfectious pneumonitis except for those induced by radiation therapies. 10. prior treatment with canakinumab.
covid-19 respiratory distress and antithrombotic drugs in subject's habits

Conditions: Sars-CoV2

Summary: current management of vid-19 consists of oxygen support without specific treatment. hospitalization may suddenly require delayed resuscitation. this evolution corresponds to an atypical acute respiratory distress syndrome (ards), the mechanism of which is poorly understood and difficult to predict. the general pathophysiology of ards, the vascular tropism of sars-cov2, the description of coagulopathy and numerous pulmonary artery thromboses in resuscitation and the encouraging investigation of antithrombotics in general ards


Eligibility criteria:
inclusion criteria: * major subject (≥18 years) * subject hospitalised in hus or ghrmsa for covid-19 * the diagnosis covid-19 confirmed by smear or evoked by ct scan or chest x-ray without a differential diagnosis retained in the medical record * subjects who, after being informed, do not wish to have their data reused for the purposes of this research exclusion criteria: * subject who expressed opposition to participating in the study * subject under guardianship or trusteeship * subject under safeguard of justice * patient hospitalized in a geriatric service or in a ward not labeled covid (surgery, oncology,...) or in a short-term hospitalization unit without transfer to another service. * serious patient on admission to the emergency room and recused from resuscitation.

Keywords: sars-cov2 covid-19 antithrombotic treatments ards hypoxemia thrombosis pulmonary arteries endothelial