Kornimate/medical-research-clean huggingface dataset:
dose-ranging study of prx-102 in adult fabry disease patients

Conditions: Fabry Disease

Summary: this is the first human treatment with prx-102, an enzyme being developed as a long-term enzyme replacement therapy in patients with a confirmed diagnosis of fabry disease (alpha galactosidase deficiency). the safety, tolerability, and exploratory efficacy will be evaluated in this study of increasing doses. patients will be treated with infusions every two weeks for 12 months.

under the pb-102-f01 study protocol, patients will be enrolled into one of three prx-102 dosing groups (0.2 mg/kg, 1.0 mg/kg, 2.0 mg/kg) and receive prx-102 as an intravenous infusion every 2 weeks for 12 weeks (3 months). patients who finish the pb-102-f01 study will be enrolled in the pb-102-f02 extension study and receive the same dose they had received in the pb-102-f01 study for an additional 38 weeks (9 months).

Eligibility criteria:
inclusion criteria: * symptomatic adult fabry patients (≥18 yrs) * males: plasma and/or leucocyte alpha galactosidase activity (by activity assay) less than lower limit of normal (lln in plasma=3.2 nmol/hr/ml, lln in leucocytes=32 nmol/hr/mg/protein) * females: historical genetic test results consistent with fabry mutations * globotriaosylceramide (gb3) concentration in urine \> 1.5 times upper normal limit * patients who have never received enzyme replacement therapy (ert) in the past, or patients who have not received ert in the past 6 months and have a negative anti alpha galactosidase antibody test * egfr ≥ 60 ml/min/1.73m2 * the patient signs informed consent * female patients and male patients whose co-partners are of child-bearing potential agree to use a medically acceptable method of contraception, not including the rhythm method exclusion criteria: * participation in any trial of an investigational drug within 30 days prior to study screening * chronic kidney disease stages 3-5 (ckd 3-5) (appendix 7) * history of dialysis or renal transplantation * angiotensin converting enzyme (ace) inhibitor or angiotensin receptor blocker (arb) therapy initiated or dose changed in the 4 weeks prior to screening * severe myocardial fibrosis by mri (≥2 late-enhancement \[le\] positive left ventricular segments) (weidemann et al. 2009) * history of clinical stroke * pregnant or nursing * presence of hiv and/or hbsag and/or hepatitis c infections * known allergies to ert * known allergy to gadolinium based contrast agents * presence of any medical, emotional, behavioral or psychological condition that, in the judgment of the investigator and/or medical director, would interfere with the patient's compliance with the requirements of the study

Keywords: fabry disease alpha galactosidase deficiency metabolic storage disease
efficacy and safety of bi 655066/abbv-066 (risankizumab) in patients with severe persistent asthma

Conditions: Asthma

Summary: the objectives of this trial are primarily to evaluate the efficacy and safety of bi 655066/abbv-066 (risankizumab) as compared to placebo over a 24-week treatment period in severe asthma patients. the primary endpoint is time to first asthma worsening during the planned 24 week treatment period for active vs. placebo treated patients on top of standard of care therapy. upon demonstration of a meaningful clinical response, another important objective is the identification of biomarkers that can be used to target patients who will likely respond to treatment with bi 655066/abbv-066 (risankizumab).


Eligibility criteria:
inclusion criteria: 1. pre-bronchodilator clinic measured forced expiratory volume (fev1) of =40% and =85% of predicted normal. 2. one year history of asthma diagnosed by a physician, and have fev1 reversibility of =12% and an absolute change of at least 200 ml after administration of 400 µg salbutamol. 3. must be on at least medium dose inhaled corticosteroids and at least one other asthma controller medication for at least one year. 4. must have documented history of two or more severe asthma exacerbations in the last 12 months. exclusion criteria: 1. patients with a significant disease other than asthma. 2. patients who are not able to produce sputum or sputum samples of sufficient quality. 3. patients who had clinically relevant history of intubation for asthma exacerbation in the past year. 4. patients diagnosed with any concurrent respiratory disease. 5. recent history (within 6 months) of myocardial infarction or hospitalized for cardiac failure in the past year. 6. patients who have undergone thoracotomy with pulmonary resection. 7. patients who have undergone bronchial thermoplasty or radiotherapy procedure in the past year or have planned procedures during the study. 8. patients taking oral corticosteroids with a total daily dose of more than 20 mg prednisone (or equivalent) in the past 6 weeks. 9. pregnant or nursing women. 10. women of childbearing potential that, if sexually active, is unwilling to use a highly effective method of birth control. 11. clinically relevant acute infections or chronic infections. 12. have received any live bacterial or live viral vaccination in the last12 weeks. 13. have received bacille calmette-guerin (bcg) vaccination in the last 12 months. 14. have received treatment with ustekinumab (stelara®). 15. have received treatment with any other biologics in the last 3 months or within 6 times the half-life of the compound.
cytokine-guided robotic cystectomy

Conditions: Cancer

Summary: there is a recognised complication of surgery known as a 'paralytic ileus', where bowel function is reduced after an operation, causing an obstruction and resulting in nausea and vomiting. this complication is more common in patients that have robotic surgery due to the positioning required and the gas pressures required for keyhole/robotic surgery. while some of the factors involved in a paralytic ileus are known, the full mechanism and the chemicals involved are not yet fully understood. this study is looking at the level of specific chemicals called 'cytokines', and the changes in the level of these cytokines in the blood before and after robotic surgery, specifically during bladder removal (cystectomy). cytokine levels will be compared against post-operative recovery and whether a paralytic ileus is developed.

cytokines are signalling proteins that are release by immune cells in response to stress on the body. in this study the aim is to evaluate the concordance between cytokine rise (specifically monitoring the following cytokines: interferon (ifn)-gamma, tumour necrosis factor (tnf)-alpha, interleukin (il) -1beta, il-2, il-4, il-6, il-12 and il-17) and postoperative ileus (a condition where bowel function slows causing build up of faecal matter) following robotic radical cystectomy and the pneumoperitoneum (gas insufflation of the abdomen) pressures that were required intra-operatively. this study includes patients undergoing radical cystectomy. the study will measure pre-anaesthetic, post-anaesthetic, immediately post-operatively and two further post-operative serum cytokines levels and compare them with the pneumoperitoneal pressures required intra-operatively and the outcome of whether a clinical diagnosis of paralytic ileus was made. serum cytokine levels will be taken on five occasions: 1. st sample (baseline)-before induction of anaesthetic 2. nd sample-immediately after induction of anaesthetic 3. rd sample-immediately post-operative 4. th sample - 2 hours post-operative 5. th sample - 1 day post-operative no alterations will be made to the care of the patient, this is purely an observational study.

Eligibility criteria:
inclusion criteria: * patients aged between 40 and 75 years of age * patients undergoing a robotic radical cystectomy * patient consenting to enter the study * patients consenting for blood samples for cytokine analysis exclusion criteria: * patients \ 75 years of age * patients unwilling or unable to consent
optimal dose of a cognitive control training for depression vulnerability

Conditions: Major Depression in Remission

Summary: this study aims to examine the dose-response relationship of an online adaptive cognitive control training on depressive symptomatology and rumination. participants will be randomized over six groups, each receiving a different dose (0, 1, 5, 10, 15 or 20 sessions) of a cognitive control training in remitted depressed patients. an adaptive paced auditory serial addition task will be used as cognitive control training.

depression is often associated with cognitive impairments and recent studies have found that for some people, these cognitive problems persist after remission of depression. these cognitive impairments could be a risk factor for recurrence of depressive episodes. cognitive control training aims to address these cognitive impairments and decrease the risk of recurrence of depression. one promising operationalization of cognitive control training is the adaptive paced auditory serial addition task, but currently, it's unclear how many sessions one should complete in order to obtain cognitive- and emotional transfer effects. by comparing multiple groups with a different number of sessions (0, 1, 5, 10, 15 and 20), with measures at post (one month after baseline) and two follow-up periods (at 3 months and 6 months after baseline), this study examines the effects of an online cognitive control training on depressive vulnerability factors.

Eligibility criteria:
inclusion criteria: * history of ≥ 1 depressive episode(s) * currently in remission (≥ 3 months) * access to a computer with an internet connection exclusion criteria: * ongoing depressive episode * psychotic disorder (current and/or previous) * neurological impairments (current and/or previous) * excessive substance abuse (current and/or previous) * use of antidepressant medication is allowed if kept at a constant level

Keywords: depression remission relapse prevention cognitive control training internet intervention
diagnostics of clinical hyperandrogenism

Conditions: Anovulation; PCOS

Summary: the optimal cut-off value for diagnosing hirsutism in the modified ferriman-gallwey (mfg) point scale in the polish population is not known. the correlation of mfg with the severity of androgenetic alopecia in the ludwig scale (ls) is not known. the aim of the study is to determine the cut-off point for the diagnosis of hirsutism and to assess the correlation of the mfg scale score with the ls of androgenetic alopecia severity.

the aim of the study is to: * determine the cut-off point on the mfg scale, indicating hirsutism and to determine the correlation of the severity of androgenetic alopecia on the ludwig scale with the mfg value in the population of women diagnosed and treated at the university hospital in krakow due to anovulation. * to determine the severity of hirsutism and the severity of androgenetic alopecia in individual pcos phenotypes. inclusion criteria are as follows: i) oligoovulation, i.e. menstrual cycles every \ 35 days, ii) age 18-45 years, iii) diagnosis of pcos. exclusion criteria: none. to achieve the study objectives, the following will be performed: * visual assessment of the severity of hirsutism on the modified ferriman-gallwey scale * visual assessment of the severity of androgenetic alopecia on the ludwig scale.

Eligibility criteria:
inclusion criteria: * oligoovulation, i.e. menstrual cycles every \ 35 days * age 18-45 years * diagnosis of pcos. exclusion criteria: * none

Keywords: hirsutism normogonadotropic anovulation pcos androgenetic alopecia
texas childhood obesity research demonstration (tx cord) project -primary prevention study

Conditions: Pediatric Obesity

Summary: a systems approach emphasizes the linkage between individual behavior change strategies and social and physical environmental changes, which act synergistically to facilitate (or inhibit) healthy eating and active living. we hypothesize that among low income, ethnically diverse overweight and obese children, aged 2-12 years, a systems approach to child obesity will reduce bmi compared to primary prevention alone.


Eligibility criteria:
inclusion criteria: * children attending selected elementary schools or head start centers in the catchment areas. * ages 2 to 12 exclusion criteria: * children who are special needs

Keywords: pediatric obesity diet physical activity schools early care and education
study of fyu-981 in hyperuricemia (effect on two hyperuricemic types)

Conditions: Hyperuricemia

Summary: to investigate the pharmacodynamics, pharmacokinetics and safety of 7-day-repeated doses of fyu-981 administered orally to male hyperuricemic patients with uric acid-overproduction or uric acid-underexcretion type. in addition, to investigate the additive effects of the combination of fyu-981 and topiroxostat administered orally to male hyperuricemic patients with uric acid-overproduction type.


Eligibility criteria:
inclusion criteria: * japanese adult subjects * serum urate level: \>= 7.0mg/dl in patients * disease type: uric acid-overproduction type or uric acid-underexcretion type exclusion criteria: * gouty arthritis within a year before start of study treatment * mixed type in the classification of hyperuricemia
comparing the diagnostic role of pet/mri and of pet/ct among patients with gastric cancer

Conditions: Gastric Cancer Stage

Summary: the results of pet/mri and pet/ct in each recruited gastric cancer patients will be compared.

a prospective comparison of pet/mri and pet/ct for gastric cancer will be performed,to evaluate the value of pet/mri in predicting the preoperative staging and resectability of gastric cancer and investigate the role of pet/mri in gastric cancer diagnosis.the evaluation parameters are the diagnostic accuracy of gastric cancer staging( including the overall accuracy, sensitivity, and specificity) and the receiver operating characteristic (roc).

Eligibility criteria:
inclusion criteria: 1. histological diagnosis of gastric cancer 2. no previous treatment history for gastric cancer 3. without complications of obstruction,bleeding and so on 4. without severe diabetes,the levels of blood glucose \<6.2 mmol/l . 5. no cardiac pacemarker,never stimulator or other metal substitute materials in vivo. exclusion criteria: 1. with other neoplastic disease. 2. suffering acute inflammatory disease 3. history of chemo-radiotherapy 4. suffering prostate hyperplasia,glaucoma

Keywords: gastric cancer simultaneous pet/mri pet/ct
caisson transcatheter mitral valve replacement (tmvr)

Conditions: Mitral Regurgitation; Mitral Valve Disease; Mitral Valve Failure; Mitral Disease; Valve Heart Disease; Valve Disease, Heart

Summary: the purpose of this study is to assess the safety and performance of transcatheter mitral valve replacement (tmvr) system for the treatment of severe, symptomatic mitral regurgitation (mr).

the treatment guidelines for valvular heart disease indicate that surgical correction of primary mitral valve regurgitation (mr) is a class i recommendation. recent evidence indicates that valve replacement is at least as effective as repair in both primary and secondary mr patients. however, many patients are not referred for surgery as they are considered to be too high of a risk to undergo on-pump, open-heart procedures. percutaneous aortic valve replacement has made treatment of stenosed aortic valves available to high-risk surgical patients who would have otherwise been medically managed. percutaneous mitral valve (mv) replacement offers similar advantages. the feasibility of percutaneous mv replacement has been demonstrated in early feasibility studies. to meet this medical need, caisson interventional has developed a percutaneous delivery system for a bioprosthetic mitral valve. as with patients with aortic valve (av) deficiencies, this device can be used to provide needed therapy to patients who might not otherwise receive treatment beyond medical therapy. this study will provide information on the safety and performance of this system.

Eligibility criteria:
inclusion criteria: * has severe mitral regurgitation * new york heart association (nyha) class ii, iii, iva or heart failure * high risk for cardiovascular surgery exclusion criteria: * excessive calcification or thickening of mitral valve annulus * severe mitral stenosis, fused commissures, valvular vegetation or mass * left ventricular end diastolic dimension \> 7cm * left ventricular outflow tract obstruction * severe right ventricular dysfunction * stroke within 90 days; transient ischemic attack or myocardial infarction within 30 days of the index procedure

Keywords: percutaneous mitral valve replacement trans-septal mitral valve replacement transcatheter mitral valve replacement tmvr primary mr secondary mr structural heart disease heart failure degenerative mitral valve disease functional mitral valve disease
serotonin-norepinephrine reuptake inhibitor in prophylaxis of depression following fragility fractures

Conditions: Depression in Old Age; Fragility Fracture

Summary: the goal of this pilot study is to learn about the feasibility about prescribing anti-depressants at discharge in patients aged 50 years and older with a lower extremity fragility fracture. the main questions it aims to answer are: * what are the obstacles to enrolling patients and prescribing anti-depressants among older adults? * is it possible to start prescribing snri medication upon discharge? * what is the prevalence of depressive symptoms amongst patients with different types of injuries and weightbearing status? * what is a transition of care plan for patients who have geriatric depression and require further care? participants will: * undergo screening using the geriatric depression scale * start on duloxetine 30mg daily at time of discharge * report medication compliance and complete re-screening monthly * complete patient reported outcome measures and 3 months, 6 months, and 1 year * receive a referral to behavioral health, primary care, or psychiatrist for evaluation if they screen positive at any timepoint

as with many mental health diagnoses, there continues to be a stigma against receiving treatment and potentially even more so receiving prophylaxis16. identifying these hurdles at the patient level will allow us to better educate patients and physicians about the benefits of this intervention. secondly, this pilot study will help us determine if it is possible to start prescribing snri medication upon discharge. it will allow us to navigate the procurement of medications through medicare, prescribing practices at discharge and address patient concerns regarding starting a new medication.

Eligibility criteria:
inclusion criteria: * geriatric (greater than or equal to 65 years old) * lower extremity fragility fractures managed operatively exclusion criteria: * polytrauma * pathological fractures * patients on hospice * patients with previously diagnosed psychiatric disorders * patients with previously diagnosed dementia * patients already taking selective serotonin reuptake inhibitors (ssri)s and serotonin- norepinephrine reuptake inhibitors (snri)s * patients already taking mood stabilizing medication * unable to provide informed consent (no use of a legal authorized representative) * patients with pre-existing life limiting diagnoses (cancer, etc.)

Keywords: geriatrics serotonin-norepinephrine
a computer-based cognitive remediation program for adults with intellectual disability

Conditions: Intellectual Disability

Summary: adults with intellectual disabilities have great difficulty in adapting to social situations and relationships. cognitive impairment associated with intellectual disability are important factors to understand their difficulties in processing social information. in the field of recognition of facial emotions in particular, basic cognitive processes such as visuospatial and attentional functions, are heavily involved. cognitive remediation is a management tool widely used by practitioners to help patients who experience cognitive difficulties. currently, no program can meet specific and validated the problems are adults with intellectual disabilities manner in their daily functioning

réhabilitus is a remediation program focused on attentional functions and visuospatial, to limit the presence of behavioral disorders among adults with intellectual disabilities. the main objective is to validate the effectiveness of réhabilitus program on behavioral disorders in adults with intellectual disabilities without autism spectrum disorder associated.

Eligibility criteria:
inclusion criteria: * adults aged 18 to 45 inclusive; * behavioral disorders corresponding to a total score on the abc scale \> 15 * presence of a family caregiver (parent, friend) or professional (reeducator, professional of the medico-social sector) stakeholder of the project; * diagnosis of mild to moderate intellectual disability (assessed by wais-iv battery and vabs-ii less than 3 years ago; * french or secondary mother tongue; * psychoactive treatment unchanged during the month prior to inclusion; * adult or legal representative who has given written and informed consent to participate in the study. by default, the adult's oral agreement will be collected (as well as the written consent of the legal representative); * affiliation to the social security scheme or beneficiary of such a scheme. exclusion criteria: * neurological disorders of vascular, infectious or neurodegenerative origin; * taking medications for general medical purposes with a neurological or psychiatric impact (eg corticosteroids); * simultaneous participation in any other cognitive remediation program targeting attentional, visuospatial and social cognition; * refusal of participation of the person and/or his/her legal representative; * not family or professional caregiver; * presence of autistic spectrum disorders (evaluated by ados and adi if necessary according to the assessment of the investigator)
femtosecond laser-assisted anterior lamellar keratoplasty

Conditions: Keratoconus; Corneal Opacity

Summary: the purpose of the study is evaluating safety and clinical efficiency of full femtosecond laser-assisted anterior lamellar keratoplasty (fs-alk) for curing patients with keratoconus and corneal opacities

keratoplasty is the most widely spread tissue transplantation procedure. although penetrating keratoplasty (pkp) is still commonly used for curing corneal dystrophy and opacities of different genesis, lamellar techniques provide significant advantages in terms of safety and predictability. al in all 13 fs-alk procedures were performed for 11 eyes with advanced keratoconus and 2 eyes with superficial corneal scattering before and after surgery uncorrected visual acuity (ucva), best spectacle-corrected visual acuity (bscva), postoperative astigmatism, endothelial cell loss, central cornea thickness, residual recipient's tissue thickness, corneal hysteresis (ch) and corneal resistance factor (cfr) were evaluated . at 1-year follow-up confoscan investigation of donor-recipient interface was performed. all fs-alk procedures were performed with intralase fs 60khz femtosecond laser in a following way. at first a 80% thickness corneal graft was prepared. then maximum thickness recipients corneal dissection was performed according to oct (optical coherence tomography) data and superficial tissue was removed. transplant was fixed in a resulted bed by continuous suture.

Eligibility criteria:
inclusion criteria: * advanced keratoconus or stromal corneal scattering * normal endothelial cell density exclusion criteria: * acute keratoconus in the anamnesis * low endothelial cell density * age under 18 years

Keywords: femtosecond laser anterior lamellar keratoplasty atomic-force microscopy