Kornimate/medical-research-clean huggingface dataset:
real world evidence study of danish fabry patients

Conditions: Fabry Disease

Summary: fabry is a rare x-linked metabolic lysosomal disorder caused by deficiency in the enzyme α-galactosidase a (alpha-gal a) by mutations in the gla gene, encoding the alpha-gal a enzyme, which catalyses glycosphingolipids, namely globotriaosylceramide (gb3). reduced or absent alpha-gal a activity leads to accumulation of gb3 in various organs as well as cellular dysfunction and inflammation causing phsyical symptoms and eventual organ failure. treatment has been available since 2001 for fabry patients - first enzyme replacement therapy and since 2016, an oral chaperone therapy, migalastat. although the initial trials of migalastat had some both short and extended outcome treatment comparisons, the overall evidence of clinical efficacy is based on too small numbers considering the heterogeneity of the fabry patient population as well as the very slow progression of the disease. though the body of real-world evidence is growing, there is a need for more publications of real-world long-term data on clinical outcomes with a focus on treatment with migalastat. research question: is the incidence and prevalence of fabry associated clinical events (faces) (cardiac, renal, and cerebrovascular) associated with sex, genotype, phenotype at time of diagnosis, biomarkers, and fabry specific therapy? objectives: * to investigate time to first fabry associated clinical events (face) (cardiac, renal, and cerebrovascular) with particular focus on migalastat clinical outcomes and treatment outcomes preceding migalastat therapy. * to investigate the incidence and prevalence of faces with respect to fabry specific treatment, migalastat, ert or no treatment. * to describe faces in accordance with different geno- and phenotypic groups. * to investigate the incidence and time to a first fatal or non-fatal cardiac, renal, and cerebrovascular clinical event, separated by each category. primary outcomes - time to first face (cardiac, renal, and cerebrovascular) with particular focus on migalastat on clinical outcomes and treatment outcomes preceding migalastat therapy. secondary outcomes * to investigate the incidence and prevalence of faces with respect to fabry specific treatment, migalastat, ert or no treatment. * to describe faces in accordance with different geno- and phenotypic groups to investigate the incidence and time to a first fatal or non-fatal cardiac, renal and cerebrovascular clinical event, separated by each category. exploratory outcomes - to describe disease progression with focus on organ involvement. the study design is a retrospective clinical and paraclinical follow-up of the danish national fabry cohort in the period 01.01.2001-31.12.2022. patient followed a structured yearly monitoring program as part of routine clincal care.


Eligibility criteria:
inclusion criteria: * genetically-verified fabry disease * age above or equal to 18

Keywords: cardiovascular diseases renal disease cerebrovascular disease
assessment of bmi-1 on protein and molecular levels in oral dysplasia and squamous cell carcinoma: a diagnostic study

Conditions: Oral Squamous Cell Carcinoma

Summary: the aim of the current study is to assess the validation of bmi-1 detection at both protein and molecular levels in oral epithelial dysplasia and oral squamous cell carcinoma as a biomarker for early cancer detection versus biopsy embedded in paraffin blocks.

head and neck squamous cell carcinoma (hnscc) including oral squamous cell carcinoma (oscc) has been reported as the sixth most common cause of cancer mortality in the world and the fifth most commonly occurring cancer. thus a compelling need for investigation of the underlying molecular events associated with oscc tumorigenesis has emerged for better understanding of such lesion. moreover, identification of biomarkers for early detection and prediction of prognosis became of extreme importance, as it was reported that early diagnosis has been vital for effective treatment of oscc and improved the survival rate of oscc patients. oscc may originate from malignant transformation of the normal oral mucosa, as well as from oral potentially malignant lesions (opmls) with different degrees of oral epithelial dysplasia (oed). the approach of a step-wise transition from opmls to oscc was well-established, but it could be difficult to predict if and when an opml would undergo full transformation and resulted in a tumor. thus, using specific molecular biomarkers able to identify oed lesions with higher potential for malignant transformation would be very beneficial. unfortunately, up to date there has been no tools available to monitor oed lesions or hnscc patients for early stages of local recurrences or distant metastases . among the recently introduced biomarkers, b-lymphoma moloney murine leukemia virus insertion region-1 (bmi1), a member of the polycomb group (pcg) genes, was considered to be pivotal in regulating stemness-related genes involved in maintaining the self-renewal ability of stem cells by promoting chromatin modifications. bmi1 was also known to be deregulated in various human types of cancer. previous studies have revealed the capability of bmi1 to be used as a prognostic marker in gastric, esophageal, nasopharyngeal cancer, prostate, breast, cervical and ovarian cancer, however, the role of bmi1 in maintaining self-renewal and tumorigenicity in hnscc or hnscc-derived cancer stem cells (cscs) remained to be clarified.

Eligibility criteria:
inclusion criteria: 1. in vitro studies. 2. samples used are oral dysplasia and squamous cell carcinoma. 3. diagnostic accuracy of bmi-1 marker on oral dysplasia and scc. 4. english language published articles only. exclusion criteria: 1. in vivo studies. 2. studies using any techniques other than immunohistochemistry or pcr. 3. samples using any carcinoma rather than squamous cell carcinoma. 4. samples using benign tumors. 5. samples using sarcomas.
acoart vi / vertebral artery ostium stenosis in china

Conditions: Ischemic Stroke; Transient Ischemic Attack

Summary: the purpose of this study is to determine whether deb is not inferior to common bare metal stent using under in long-term vessel patency and inhibiting restenosis in vertebral artery ostium stenosis

vertebral artery origin stenting is an established alternative to open surgical bypass for the treatment of vertebral artery ostium stenosis . debs are designed to promote arterial patency by reducing neointimal proliferation.

Eligibility criteria:
inclusion criteria: * aged between 18 and 80 years old * symptomatic vao stenosis refractory to amm (aggressive medical management) * etiology of vaos was atherosclerosis * the diameter of the normal segment of the artery beyond the stenosis between 3mm and 5mm * target lesion has stenosis ≥ 70% evidenced by angiography * score on the modified rankin scale ≤ 3 * nihss≤ 6 * patients have signed informed consent exclusion criteria: * in-stent restenosis in vertebral artery * severe calcified lesion or residual stenosis ≥30% after predilatation or flow-limiting dissection * tortuous or variable vessels * distal serial stenosis or distal vascular dysplasia of the stenosis segment * non-atherosclerotic arterial stenosis * non-vertebral artery stenosis caused tia or stroke * intracranial stent implantation within 12 months * intracranial hemorrhage occurred within 3 months * obvious thrombosis in brain vessel, or have received thrombolytic therapy 24 hours before procedure * active bleeding or coagulation disorders * serious liver/kidney damage, not suitable for routine surgical treatment * myocardial infarction or extensive cerebral infarction occurred within 2 weeks * uncontrolled high blood pressure * complicated intracranial tumor, cerebral arteriovenous malformation, or intracranial aneurysm * potential sources of cardiogenic thrombosis, such as mitral stenosis, atrial septal defect, aorta or mitral valve replacement, left atrial myxoma, etc * life expectancy shorter than 1 years * patients whit cognitive impairment or mental disorders * known hypersensitivity to aspirin, heparin, clopidogrel,paclitaxel, contrast medium, etc * pregnant and lactating women * patients who have participated in other clinical trials during the same period that lead to researchers who believe that patients may not be able to follow the trial program

Keywords: drug eluting balloon catheter stenting
comparison of silk suture and tissue adhesive for wound closure after impacted tooth

Conditions: Tooth, Impacted; Tooth Diseases

Summary: this study was administered to 21 female and 9 male patients. patients with impacted wisdom teeth on both sides of the mandible were selected. after extraction of the impacted teeth, silk sutures were used on one side and cyanoacrylate tissue adhesive on the other side for wound closure. the effects of these two materials on patient comfort were investigated.

the aim of this study was to compare the effectiveness of tissue adhesive (periacryl 90) and silk suture in bilateral mandibular impacted wisdom tooth surgery. in the study of 30 patients, 21 females and 9 males, 60 fully impacted lower wisdom teeth were extracted bilaterally and in the same position. one of the bilaretal teeth of the patients was randomly selected and tissue adhesive (periacryl 90) was applied for wound closure as the experimental group, while the other tooth was selected as the control group and the wound was closed with silk suture. the selected side was determined by a closed envelope procedure. after the first impacted tooth extraction, the patient waited until the wound healed and the symptoms disappeared completely, and then the other tooth was extracted. both extractions were evaluated on days 3 and 7 for wound healing, edema and trismus. wound healing was evaluated as good, acceptable and poor. vas (visual analog scale) was used for pain assessment

Eligibility criteria:
inclusion criteria: * volunteers with bilateral fully impacted mandibular wisdom teeth exclusion criteria: * patients were excluded from the study if they did not understand the clinical procedures of the study, * had allergies or intolerance to any of the substances used in the study, * patients on anticoagulant or psychiatric treatment, * pregnant or breastfeeding, * patients using oral contraceptiveswere, * diabetic, * patients with periodontal disease or active infection, * poor oral hygiene

Keywords: impacted wisdom teeth tissue adhesive silk suture
slv 308 and pramipexole for treatment of patients with early parkinson disease

Conditions: Early Stage Parkinson Disease

Summary: this is a multicenter, randomized, double blind, parallel group study of 6 months' treatment with slv308 as monotherapy in patients with early stage pd. an open label safety extension to this study is planned as a separate protocol for patients who are willing and eligible to participate.


Eligibility criteria:
inclusion criteria: * diagnosis of idiopathic parkinson's disease, modified hoehn \& yahr up to stage iii, updrs motor score (part iii) must have a total of at least 10 at baseline. exclusion criteria: * diagnosis is unclear or a suspicion of other parkinsonian syndromes, * patients who have undergone surgery for the treatment of pd, * current presence of dyskinesias, * motor fluctuations or loss of postural reflexes, * a history of non-response to an adequate course of l-dopa or a dopamine agonist, * patients for whom previously treatment with dopamine agonists needed to terminate because of induction of psychosis (i.e. hallucinations) and /or sleep attacks.

Keywords: parkinson disease
phase i study of s64315 administred intravenously in patients with acute myeloid leukaemia or myelodysplastic syndrome

Conditions: Acute Myeloid Leukaemia (AML); Myelodysplastic Syndrome (MDS)

Summary: the cl1-64315-001 study is a phase i, international, multicentre, open-label, non-randomised, non-comparative study. this study is designed in two parts: one part for dose escalation, one part for dose expansion.


Eligibility criteria:
inclusion criteria: * male or female aged ≥ 18 years; * patients with cytologically confirmed and documented de novo, secondary or therapy-related aml, excluding acute promyelocytic leukaemia (apl, french-american british m3 classification): * with relapsed or refractory disease without established alternative therapy or * secondary to mds treated at least by hypomethylating agent or * \> 65 years not previously treated for aml and who are not candidates for intensive chemotherapy nor candidates for established alternative chemotherapy or patients with cytologically confirmed and documented mds), in relapse or refractory after previous treatment line including at least one hypomethylating agent and have ≥10% bone marrow blasts; * eastern cooperative oncology group (ecog) performance status 0 to 2 * circulating white blood cells \< 10\^9 /l (with or without use of hydroxycarbamide). * adequate renal function defined as: • serum creatinine ≤ 1.5 x uln (upper normal limit) or calculated creatinine clearance (determined by mdrd) \> 50 ml/min/1.73m2. * ldh \< 2 x uln * adequate hepatic function defined as: * ast and alt ≤ 1.5 x uln * total bilirubin level ≤ 1.5 x uln, except for patients with known gilbert's syndrome (confirmed by the ugt1a1 polymorphism analysis), who are excluded if total bilirubin\>3.0 x uln or direct bilirubin \> 1.5 x uln * serum ck/cpk ≤2.5 x uln. exclusion criteria: * unlikely to cooperate in the study. * participant already enrolled in the study who has received at least one s64315 infusion. * pregnancy, breastfeeding or possibility of becoming pregnant during the study. * participation in another interventional study requiring investigational treatment intake within 2 weeks or at least 5 half-lives (whichever is longer) prior to first dose of s64315 (participation in non-interventional registries or epidemiological studies is allowed). * presence of ≥ ctcae grade 2 toxicity (except alopecia of any grade) due to prior cancer therapy, according to the national cancer institute common terminology criteria for adverse events (nci-ctcae, version 4.03) * unresolved ≥ ctcae grade 2 diarrhoea or medical conditions associated with chronic diarrhoea (such as irritable bowel syndrome, inflammatory bowel disease) * known carriers of hiv antibodies * known history of significant liver disease * uncontrolled hepatitis b or c infection * known active or chronic pancreatitis * history of myocardial infarction (mi), angina pectoris, coronary artery bypass graft (cabg) within 6 months prior to starting study treatment.
prolonged exposure to doxorubicin in patients with glioblastoma multiforme and diffuse intrinsic pontine glioma

Conditions: Glioblastoma (GBM); DIPG; Brainstem Glioma, Pediatric; Diffuse Spinal Glioma; Bilateral Thalamic Glioma; Gliomatosis Cerebri; Anaplastic Astrocytoma; Midline Diffuse Glioma

Summary: the standard therapy of glioblastoma (gbm) consists of gross total resection followed by focal irradiation to the tumor bed with concomitant and adjuvant temozolomide (tmz). the association of valproic acid and tmz during radiotherapy improves survival of gbm. preclinical studies suggested that doxorubicin had a strong antineoplastic activity against human gliomas. moreover, some studies showed that the continuous infusion of anthracyclines in patients with solid tumor ensured a better safety profile compared with bolus administration. based on these findings, the purpose of this study is to evaluate safety and efficacy of prolonged administration of doxorubicin in combination with radiotherapy, temozolomide and valproic acid in pediatric and adult patients with newly diagnosed gbm and diffuse intrinsic pontine glioma (dipg).


Eligibility criteria:
inclusion criteria: * males and females patients, aged \>3 years and \< 30 years; * newly diagnosed of gbm, dipg, diffuse brainstem glioma, diffuse spinal glioma, bilateral thalamic glioma, gliomatosis cerebri, anaplastic astrocytoma; * patients undergone either surgery or biopsy only; * no prior chemotherapy and/or radiotherapy; * life expectancy ≥ 4 weeks; * karnofsky/lansky ≥ 40 %; * written informed consent obtained from the patient/parents or legal representative; * adequate hematological function (leucocyte ≥ 2.0 x 10\^9/l -hemoglobin ≥ 10 g/dl - platelet ≥ 50 x 10\^9 /l); * adequate liver function (total bilirubin ≤ 2.5 x uln - alt/ast ≤ 5.0 x uln); * adequate renal function (serum creatinine ≤ 1.5 x uln); * adherence to trial treatment and compliance with the protocol exclusion criteria: * any disease or condition that contraindicates the use of the study drug (es. serious mental retardation, brain palsy, congenital syndrome, cardiomyopathy) * prior anti-cancer therapy * pregnancy or breastfeeding * non adequate contraception

Keywords: glioblastoma multiforme high grade glioma diffuse intrinsic pontine glioma dipg doxorubicin temozolomide malignant glioma brainstem glioma diffuse spinal glioma bilateral thalamic glioma anaplastic astrocytoma gliomatosis cerebri midline diffuse glioma
exelon patch and combination with memantine comparative trial

Conditions: Alzheimer's Disease

Summary: the primary objective is to compare the tolerability between rivastigmine patch monotherapy and combination therapy with memantine in patients with alzheimer's disease (ad). the secondary objective is to compare the efficacy and safety between rivastigmine patch monotherapy and combination therapy with memantine in patients with ad. the study hypothesis is that the tolerability of the combination therapy with memantine is not inferior to that of rivastigmine patch monotherapy in ad patients.

recently, the rivastigmine patch demonstrated efficacy comparable to the highest doses of rivastigmine capsules, with markedly improved tolerability profile. we hypothesized that combination of memantine and rivastigmine patch will be safe and well tolerated and result in more clinical benefit in patients with ad in comparison with rivastigmine patch monotherapy, for the mechanisms of the drugs are different.

Eligibility criteria:
inclusion criteria: * dementia by dsm-iv and probable ad by nincds-adrda * age of 50 to 90 years * mini-mental state examination (mmse) score of 10 to 20 * brain mri or ct scan consistent with a diagnosis of probable ad * the caregiver must meet the patient at least once a week and be sufficiently familiar with the patient to provide accurate data. * ambulatory or ambulatory-aided (is, walker or cane) ability * written informed consent will be obtained from the patient (if possible) and from the patient's legally acceptable representative. even if unable to provide written informed consent, the patient must assent verbally to participating in the study. exclusion criteria: * patients with evidence of severe or unstable physical illness, i.e., acute and severe asthmatic conditions, severe or unstable cardiovascular disease, active peptic ulcer disease, severe hepatic or renal disease, or any medical condition which would prohibit them from completing the study * any psychiatric or primary neurodegenerative disorder other than ad * any patients with hearing or visual problem that can disturb the efficient evaluation of the patients. * any patients with a history of drug addiction or alcohol addiction for the past 10 years * patients with bradycardia (bpm less than 50) or sick sinus syndrome or conduction defects (sino-atrial block, second ot third degree a-v blocks * clinically significant laboratory abnormalities to affect cognitive function (i.e.abnormal thyroid function test, abnormal low level of vitamin b12 or folate, or syphilis, etc) * history of allergy to topical products containing any of the constitution of the patches * current diagnosis of an active skin lesion * involved in other clinical trials or treated by experimental drug within 4 weeks * patients with hypersensitivity to cholinesterase inhibitors
restenosis intrastent: bioresorbable vascular scaffolds treatment with scoring balloon pre-dilatation (ribs vi scoring)

Conditions: Coronary In-stent Restenosis

Summary: treatment of patients with in-stent restenosis (isr) remains a challenge. this study will assess the efficacy of bioresorbable vascular scaffolds (bvs) (abbott vascular) in the treatment of patients suffering from isr after scoring balloon pre-dilatation.

treatment of patients with isr remains a challenge. currently both drug-eluting stents (des) and drug-coated balloons (dcb) are considered as the strategies of choice in this setting. however, data on the value of bvs in patients with isr is scarce. bvs are very effective to inhibit neointimal proliferation and they avoid the need of implanting a new permanent metal layer. accordingly, currently, there is a major interest to elucidate the potential value of bvs in patients with isr. the use of scoring balloon pre-dilatation has not been studied previously in this setting. this prospective spanish multicenter study will assess the clinical and angiographic outcome of patients with isr treated with bvs with scoring balloon pre-dilatation. bvs will be implanted in selected patients (fulfilling inclusion and exclusion criteria) presenting with either bms-isr or des-isr. care will be paid to ensure device optimization. scoring balloon pre-dilatation is mandatory. angiographic follow-up will be obtained at 6-9 months. a centralized angiographic corelab will be used to provide qca measurements. clinical follow-up will be also obtained at 1 year and then yearly. clinical events will be adjudicated by an independent clinical event committee.

Eligibility criteria:
in-stent restenosis with ischemia. signed informed consent irb approval inclusion criteria: patient * age \> 20 and \< 85 years of age * acceptance of late angiographic evaluation * angina or objective evidence of ischemia lesion * isr (\>50% diameter stenosis on visual assessment) * previous stent location known exclusion criteria: patient * inclusion in other clinical research protocol * allergy to antiplatelet agents * women in childbearing age * severe associated systemic diseases (including renal or liver failure) or diseases affecting life expectancy * recent myocardial infarction * time from index stent implantation \< 1 month * anticipated difficulties for late angiographic evaluation lesion * stent thrombosis or large thrombus within the stent * angiographic failure during initial stent implantation or persistence or large dissection. * severe tortuosity or calcification or major difficulties during previous stent implantation * vessel diameter \< 2.25 mm (visual assessment) * stenosis outside stent stent edge (edge isr are eligible) * very diffuse isr (\>30 mm in length)

Keywords: in-stent restenosis bioresorbable vascular scaffold stent drug-eluting stent coronary angiography scoring balloon