Kornimate/medical-research-clean huggingface dataset:
safety, tolerability and pk/pd evaluation of intravenous administration of mrt5201 in patients with otc deficiency

Conditions: Ornithine Transcarbamylase Deficiency

Summary: this phase 1/2, first-in-human study will evaluate the safety and tolerability of single escalating doses of mrt5201 administered intravenously to subjects with otc deficiency (otcd). this study will also evaluate the effect of a single dose of mrt5201 on metabolic markers of otcd and ureagenesis; and determine an acceptable dosing interval of mrt5201.


Eligibility criteria:
inclusion criteria: * have a documented diagnosis of otcd. * documented history of ≥1 symptomatic hyperammonemia event with ammonia ≥100 µmol/l * subject's otcd is stable as evidenced by meeting the following criteria: * ammonia level \<175 µmol/l during the screening period and at baseline (day -1) * no clinical symptoms of hyperammonemia during the screening period and at baseline (day -1) * if using nitrogen scavenger therapy, must be on a stable regimen for ≥28 days prior to signing informed consent * subject has maintained a stable protein restricted diet (which may or may not include medical foods) and/or amino acid supplementation with no changes in calorie or protein goals and no changes in medical food and/or amino acid supplementation for ≥ 28 days prior to signing informed consent. exclusion criteria: * any laboratory abnormality that may put the subject at increased risk by participating in this study. * have any significant concurrent or past medical condition that would represent an unacceptable risk to the subject or might jeopardize the collection of high-quality data from the study. these include but are not limited to: * history of liver transplant, including hepatocyte therapy/transplant * history of liver disease * positive viral serology test results for hiv type 1 or 2 antibodies, hepatitis b surface antigen (hbsag) or hepatitis c virus (hcv) antibody * type i or type ii diabetes that is poorly controlled, in the opinion of the investigator * poorly controlled hypertension (defined as systolic blood pressure \[bp\] \> 150 mm hg or diastolic bp \> 90 mm hg) * use of anticoagulants or platelet inhibitors, including but not limited to heparin and non-steroidal anti-inflammatory drugs (nsaids). acetaminophen is permitted * participation in previous clinical studies evaluating investigational otcd therapies directed at expressing functional otc protein (eg, otc gene therapy studies, other mrna replacement therapy) that has led to the presence of anti-otc antibodies.

Keywords: otc urea cycle disorder ucd
esprit: european/australasian stroke prevention in reversible ischaemia trial

Conditions: Brain Ischemia; Transient Ischemic Attack; Arteriosclerosis

Summary: the objective of esprit was to compare the efficacy and safety of mild anticoagulation or a combination treatment of aspirin and dipyridamole with the efficacy and safety of treatment with aspirin alone after cerebral ischemia of arterial origin.

low-dose aspirin (asa) (at least 30 mg/day) prevents only 13% of subsequent vascular events after minor cerebral ischemia of arterial origin. anticoagulation (ac) has been proven highly effective in preventing vascular events after myocardial infarction and after cerebral ischemia in patients with atrial fibrillation. a previous study on the effects of ac after cerebral ischemia of arterial origin (spirit) showed that high intensity ac (inr 3.0 to 4.5) is not safe, but that mild ac (inr 2.0 to 3.0) was. the 2nd european stroke prevention trial (esps-2) reported a 22% relative risk reduction of the combination of asa and dipyridamole (dip) above that of asa only; its results, however, are subject to debate. study design: esprit was an open randomised controlled trial allocating patients who experienced a transient ischemic attack (tia) or a non-disabling ischemic stroke to either: a. oral ac (inr 2.0 to 3.0); b. the combination of dip (400 mg daily) plus asa (30-325 mg/day); or c. asa only (same dose). the mean follow-up was three years. primary outcome was the composite of vascular death, stroke, myocardial infarction or major bleeding. outcome assessment is blind.

Eligibility criteria:
inclusion criteria: * patients presenting in the participating hospitals with a tia or non-disabling stroke of atherosclerotic origin * randomisation within 6 months after the tia or minor stroke * modified rankin scale of 3 or less exclusion criteria: * (contra)indication to, or intolerance to, anticoagulants, dipyridamole, or aspirin * disease expected to cause death within weeks or months * source of embolism in the heart * moderate or severe ischemic damage to the white matter of the brain (leukoaraiosis) * anemia, polycythemia, thrombocytosis, or thrombocytopenia * planned carotid endarterectomy * intracranial bleeding or cerebral tumour * tia or stroke caused by vasculitis, migraine, or dissection * severe hypertension * liver failure * pregnancy * chronic alcohol abuse

Keywords: secondary prevention tia / minor stroke atherosclerotic origin tia (transient ischemic attack) prevention & control
massage therapy and port-a-catheter insertion

Conditions: Cancer; Anxiety; Pain; Surgery

Summary: the purpose of this study is to assess the feasibility and efficacy of massage therapy for reducing pre-operative anxiety and post-operative pain among predominantly low income minority cancer patients undergoing surgical placement of a port-a-catheter.

despite major advances in the understanding of cancer and its treatment, patients continue to suffer greatly. massage is now included in the national comprehensive cancer network guidelines for the treatment of refractory cancer pain (1), and many cancer patients are turning to massage and other complementary therapies to help alleviate both their psychological and physical symptoms. however, complementary therapies, such as massage, are often unaffordable or unavailable to predominantly low-income cancer patients at safety net hospitals like boston medical center. the vast majority of cancer patients receiving chemotherapy undergo implantation of a permanent central venous access device, often referred to as a port implantation or implanted port. although the implanted port carries multiple benefits for ease of treatment, after the procedure patients often complain of headaches, muscle stiffness and neck and shoulder pain that lasts for several days. pain medication is the only therapy commonly offered for this and is often inadequate (2). furthermore, since this is often the first surgical procedure for cancer patients at the beginning of their treatment, they often have significant levels of pre-procedure anxiety (3,4). safe, efficacious, and cost-effective interventions that can reduce the anxiety and pain related to port implantation are needed. this pilot study will look at how feasible and effective massage therapy is in reducing pre-operative anxiety and post-operative pain among bmc patients already undergoing surgical placement of an implanted port.

Eligibility criteria:
inclusion criteria: * patients must be adults within one month of diagnosis with any form of cancer. * patients must be scheduled to undergo, but have not yet received, port implantation. * patients must have the ability to understand and sign a written informed consent. exclusion criteria: * patients who are unable or unwilling to provide consent.

Keywords: discomfort port-a-catheters complementary and alternative medicine massage
mscs for treatment of azoospermic patients

Conditions: Male Infertility

Summary: in this study, we performed autologous bmdmsc transplantation to the testis of azoospermic patients in a trial to enhance and activate the spermatogonial stem cells of the testis and aiming to produce motile sperm.

bone marrow aspirated and stem cells were isolated, cultured and characterized using flow cytometer. stem cells prepared according to gmp regulations then injected into rete testis using special syringe. patients follow up will carried out for a year , three month interval. fsh, testosterone, testicular size as well as sexual potency will assessed every three months.

Eligibility criteria:
inclusion criteria: * all non obstructive azoospermic patients. * azoospermic patients due to administration of chemo/radiotherapy. exclusion criteria: * patients with testicular cancer. * patients with complete deletion of chromosome y azf (a,b,c) regions.

Keywords: azoospermia, spermatogonial arrest, maturation arrest.
syncope patient evaluation in the emergency department

Conditions: (Near) Syncope

Summary: patients with the symptom complex syncope or near-syncope are quite often in the emergency department. most patients with syncope have a positive prognosis. however some types of syncope are associated with an increased morbidity and mortality. it is a challenge to detect these patients reliably at an early stage. the current guidelines contain exact definitions concerning the characteristics of patients with syncope and recommendations on how to diagnose and treat these patients following standardized pathways. due to the fact that near-syncope is poorly defined and therefore seldom evaluated in clinical trials, data concerning near-syncope are rare. our aim was to characterize patients presenting with syncope or near-syncope to the emergency department regarding their risk profile, comorbidities and prognosis. first we evaluated if there is a difference between the two groups syncope and near-syncope concerning type and frequency of adverse events. furthermore we analyzed the routinely measured cardiac biomarkers nt-probnp and hs ctnt in patients with syncope or near-syncope. then we determined both their prognostic accuracy in predicting adverse events and their diagnostic accuracy in finding the underlying etiology. finally we analyzed a special patient collective, patients aged ≥ 65 years. it is assumed that elderly patients are suffering from comorbidities and age-related physiological and cognitive disabilities. we therefore hypothesize that elderly patients, in contrast to patients aged \< 60, display an increased risk of adverse events and that they have a poorer prognosis.


Eligibility criteria:
inclusion criteria: * (near) syncope exclusion criteria: * age \< 18 years * progressive impairment of consciousness * use of mind-altering drugs * non syncopal loss of consciousness: hyperventilation, hypoglycaemia, metabolic disorders, psychogenic pseudosyncope, tia or stroke, seizure, transient loss of consciousness caused by craniocerebral injury

Keywords: syncope near syncope prognosis emergency department
measurement of pro-angiogenic markers in patients with hepatic metastases undergoing sirt

Conditions: Liver Cancer

Summary: this study is being done to investigate the influence of giving radiation to the liver on tumors involving the liver. investigator is specifically looking at this effect on the blood vessels within the tumor. this effect will be measured by studying substances in the blood that the tumors produce and that cause blood vessels to grow. the effects seen on these substances may help design other treatments to improve the results of the radiation used to treat these tumors.

subjects are invited to participate in this study that have undergone a procedure as their standard of care. this procedure is known as selective internal radiation therapy (sirt), a procedure designed for the treatment of cancer in the liver. this includes cancer that has started in the liver as well as cancer that has spread to the liver. the purpose of the study is to collect blood samples to assess for "biological markers," or substances within the blood that may promote cancer growth by causing new blood vessels to form. this study will also use a new method of analyzing medical imaging (ct scan, pet scan) to try and better understand how cancer in the liver forms new blood vessels. sirt is standard therapy and not part of this study. this study involves blood draws only.

Eligibility criteria:
inclusion criteria: * patients between the ages of 18-85 who have been referred for sirt as standard of care treatment for hepatocellular carcinoma or colorectal metastases to the liver and qualify for treatment with sirt * life expectancy of at least 3 months exclusion criteria: - patients are excluded from participation in this study if they are not undergoing the sirt procedure

Keywords: pro-angiogenic markers sirt hepatic metastases
evaluate the effect of switching from daily injections of 20mg glatiramer acetate (ga) to 40mg ga three times a week in subjects with relapsing-remitting multiple sclerosis

Conditions: Multiple Sclerosis

Summary: the primary aim of this study is to observe any changes in mri in ms patients who have switched from 20mg injections/day to 3 40mg injections/week of glatiramer acetate.

the primary aim of this study is to observe the effect of switching from daily injections of 20mg glatiramer acetate (ga) (20mg/daily) to ga 40mg three times a week (40mg x 3/weekly) on thalamus pathology, as measured by changes in diffusion-tensor imaging (dti) in patients with relapsing-remitting multiple sclerosis (rrms). we hypothesize that ga 40mg x 3/weekly will exert similar, if not better effect on prevention of thalamic pathology, as compared to ga 20mg/daily. the secondary objective of this study is to investigate the effect of switching from ga 20mg/daily to ga 40mg x 3/weekly on evolution of microstructural changes in normal appearing white matter (nawm) and normal appearing gray matter (nagm), as measured by dti.

Eligibility criteria:
inclusion criteria: * ms patients diagnosed with ms according to the mcdonald criteria * ms patients having a relapsing disease course * being on ga monotherapy (20mg/daily sc) for at least 12 months prior to the standard of care mri at the time of switch to ga 40mg x 3/weekly * having standard of care 3t mri scan while on ga 20mg/daily treatment for at least 12-18 months prior to the start day of the of the ga 40mg x 3/weekly and at the time of switch to ga 40mg x 3/weekly age over 18 * pass mri health screening (in case of egfr \<59, the contrast will not be applied) * none of the exclusion criteria exclusion criteria: * patients who had a relapse within 30 days prior to mri scan date * patients who received steroid treatment within 30 days prior to the mri scan date * women who are pregnant, lactating or of childbearing age who do not consent to approved contraceptive use during the study * ms patients who used other imunomodulatory or immunosuppressant treatment other than ga during the 12 months prior to start of ga 40mg 3/weekly (e.g., ifn-β, mitoxantrone, cyclophosphamide, cladribine, fludarabine, cyclosporine, total body, azathioprine, methotrexate, ivig, cellcept, natalizumab, etc.)
vaccine study of mva-muc1-il2 in patients with prostate cancer

Conditions: Prostatic Neoplasms

Summary: this study involves the use of an experimental product, tg4010. the purpose of the study is to determine if tg4010 can stimulate the body's immune system to help it fight the cancer.

the experimental product, tg4010, is a modified vaccinia virus (already used in humans) into which two (2) genes have been placed. one gene is for a protein (muc1) found in cancer cells. the second gene is for human interleukin 2 (il2) which the body's immune system makes to help it fight cancer. the tg4010 is given as an injection under the skin (subcutaneous) once a week for six weeks followed by a schedule of every three weeks or every three weeks for the first twelve (12) weeks. if the therapy is working, it can be continued for up to 9 months until no further improvement or until the patient gets worse for as long as it is tolerated.

Eligibility criteria:
inclusion criteria: * rising psa after surgery or radiation for prostate cancer exclusion criteria: * metastasis or local disease * prior hormone treatment

Keywords: adenocarcinoma of the prostate
caregiver training: evidence of its effectiveness for cognitive and functional improvement in older adults

Conditions: Functional Ability; Cognitive Decline; Older Adults; Healthy Aging

Summary: due to demographic changes that have resulted in an aging population, the role of caregiver of an older adult has become very important in recent years. while numerous programs have been designed to lighten the caregiver's physical and emotional burden, fewer programs train caregivers to improve skills and level of independence in the person they care for. the objectives of this research study were to assess the benefits of a caregiver training program on the cognitive and functional status of older adults, as well as to compare the effects of this program according to type of caregiver (professional caregiver vs. family caregiver). methods: the sample was composed of 160 older adults: a) 100 received care from caregivers who had taken the training program (treatment group), of which 60 were professional caregivers and 40 were family caregivers; and b) 60 received care from caregivers who had not taken the program (control group). in order to evaluate program effects on cognitive and functional status, we used both direct measures (mmse, cape and euroqol) and caregiver reports (barthel and rmpbc).


Eligibility criteria:
inclusion criteria: * older adults: age 65 or older, receiving care, no diagnosis of dementia, and their informed consent. exclusion criteria: -

Keywords: older adults caregiver training program cognitive stimulation professional caregivers family caregivers
epidemiological and clinical characteristics of critically ill patients with covid-19

Conditions: COVID-19; Critical Illness

Summary: objective of this observational multicentric retrospective-prospective study is to describe the number and the characteristics of patients with reverse transcription polymerase chain reaction (rt-pcr) for sars-cov2 positivity admitted to intensive care units during the first 6 months of epidemic.


Eligibility criteria:
inclusion criteria: * adulthood * informed consent obtained exclusion criteria: * no informed consent obtained
neuropathology in adults intensive care unit patients with covid 19

Conditions: COVID 19

Summary: this is a single center observational autopsy study, conducted during the first wave of the coronavirus disease (covid-19) pandemic in france. the main objective is to evaluate brain damages in patients who died from covid-19 to inform the cause of death. investigations include macroscopic and histology examinations, and virology analyses.

setting: academic center designed as a referral center for patients with covid-19 design: single center observational autopsy study participants: intensive care unit (icu) adults who died from confirmed covid-19 during the first wave of the pandemic in france. oversight: as per legal health authority requirement only patients with suspected or confirmed severe acute respiratory syndrome (sars)- related to coronavirus 2 infection were admitted to the icu. the department of pathology and forensic medicine is allowed by legal authority to perform autopsy of non-survivors from covid-19 to inform the cause of death as per ministerial decree of july 12 2017 and march 28, 2020. in this context, of autopsy for medical purpose and public interest there is no requirement for institutional review board submission. however, consent from patients legal representative or closest relatives is required for autopsy.

Eligibility criteria:
inclusion criteria: * adults * admitted to the icu * confirmed covid-19 * died during icu stay exclusion criteria: * refusal from patient's legal representative or closest relative

Keywords: autopsy coronavirus brain inflammation
the efficacy and safety of zr2 versus r-chop-like regimen for elderly patients with newly diagnosed diffuse large b cell lymphoma.

Conditions: Diffuse Large B Cell Lymphoma

Summary: this is a prospective, single-center, open-label clinical study designed to evaluate the efficacy and safety of the zanubrutinib, lenalidomide and rituximab (zr2) versus rituximab combined with chop or cdop (r-chop or r-cdop) in elderly patients with diffuse large b cell lymphoma treated for the first time.

in this study, elderly dlbcl patients will be treated with zr2 regimen for the first-line treatment. investigators will compare the complete response rate, survival and incidence of adverse reactions between the rchop/rcdop chemotherapy and the zr2 regimen. in addition, immune function tests will be performed before treatment and every 2 courses after treatment, including peripheral blood lymphocyte-monocyte ratio, cytokines, immunoglobulins, t and b cells and their quantitative analysis. patients with zr2 regimen will undergo gene second-generation sequencing before treatment to compare the gene mutation differences between complete response (cr) and ≤ partial response (pr) in the efficacy of zr2 regimen, in order to find biomarkers with better efficacy in zr2 treatment. moreover, investigators intend to conduct pharmacokinetics/pharmacodynamics (pk/pd) correlation analysis of zr2 regimen and pharmacoeconomic evaluation of the two regimens.

Eligibility criteria:
inclusion criteria: * pathologically confirmed dlbcl * without treatment * ≥ 65 years old * measurable lesions on ct or pet-ct before treatment * life expectancy of at least 3 months * voluntary participation with the consent of the patient * heart, kidney, liver and other organ function evaluation were basically normal before treatment exclusion criteria: * patients who previously received chemotherapy * uncontrolled cardiovascular diseases, cerebrovascular diseases, thrombotic diseases, autoimmune diseases and serious infectious diseases * laboratory indicators before enrollment (unless caused by lymphoma): * neutrophils \< 1.5 × 10\^9/l * platelets \< 80 × 10\^9/l * alanine aminotransferase or aspartate aminotransferase \> 2 × uln * alkaline phosphatase or bilirubin \> 1.5 × uln * creatinine \> 1.5 × uln * patients who cannot comply with the agreement due to mental diseases or other unknown reasons such as pregnancy and lactation * hiv infection * if hbsag is positive, hbvdna should be tested, and patients with positive dna cannot be enrolled; if hbsag is negative and hbcab is positive (regardless of hbsab status), hbvdna should be tested, and patients with positive dna cannot be enrolled * other uncontrolled medical conditions that may interfere with the study

Keywords: diffuse large b cell lymphoma zanubrutinib elderly patients
toripalimab in combination with lenvatinib as neoadjuvant therapy in resectable hepatocellular carcinoma

Conditions: Hepatocellular Carcinoma

Summary: this study will evaluate efficacy and safety of toripalimab injection (js001) with or without lenvatinib as a neoadjuvant therapy in patients with resectable hepatocellular carcinoma (hcc)


Eligibility criteria:
confirmed by histopathological or cytological examination; the criteria for resectability is met; has at least one evaluable lesion according to the recist 1.1 standard and has not received local treatment; exclusion criteria: patients who previously received anti-programmed death receptor-1 (pd-1) antibody, anti-programmed death ligand-1 (pd-l1) antibody, anti-programmed death ligand-2 (pd-l2) antibody or anti-cytotoxic t lymphocyte-associated antigen-4 (ctla-4) antibodies, including those who have participated in a js001 clinical study; patients with a history of gastroesophageal varices or active cardia ulcers associated with a high risk of bleeding; patients who have upper gastrointestinal hemorrhage within 1 year; patients known to have fibrous layer hcc, sarcomatoid hcc or mixed cholangiocarcinoma and hcc; other protocol defined inclusion/exclusion criteria could apply