Kornimate/medical-research-clean huggingface dataset:
needs assessment of knowledge, beliefs, and attitudes of patients with hemophilia b about gene therapy

Conditions: Gene Therapy; Hemophilia B

Summary: gene therapy is a paradigm-shifting treatment for hemophilia b patients, particularly in resource-limited countries where factor availability remains low. transparent and culturally sensitive communication around gene therapy is vital to the success of a high-quality consenting process. current literature on knowledge, beliefs and attitudes about gene therapy in resource-limited countries is inadequate. in addition, few educational resources to explain basic gene therapy concepts exist in languages other than english. this study aims to address these gaps in knowledge and aid for the development of educational resources to assist the informed consent processes for gene therapy in resource-limited countries. primary objective: to assess baseline knowledge, beliefs, and attitudes about gene therapy held by hemophilia b patients globally secondary objectives: 1. to explore healthcare workers' (i.e., physicians, nurses, social workers, educators/academic coordinators) perspectives regarding the education needs of hemophilia b patients globally 2. to explore healthcare workers beliefs and attitudes about gene therapy 3. to identify preferences of patients with hemophilia b and their healthcare workers on how/by what method or pathway educational content should be provided.

this prospective cohort study will use qualitative methods, specifically semi structured interviews to evaluate knowledge, beliefs, and attitude about gene therapy in patients 12 years and older with a diagnosis of moderate and severe hemophilia b (≤2% fix activity only), caregivers/parents and healthcare workers who care for them at st. jude and other global countries. participants will be interviewed virtually using an online video-conferencing platform, phone or in person. interviews will be performed by experienced qualitative interviewers in the official language of each participating site. interviews will last approximately 45-60 minutes and will be performed only once. all interviews will be audio recorded and obtained information will be analyzed using semantic content analysis to identify common themes.

Eligibility criteria:
inclusion criteria: * patients ≥12 years of age * diagnosis of moderate (fix ≥1% and ≤2%) or severe (\<1%) hemophilia b * parents or caregivers to patients with hemophilia 12-17 years of age inclusion criteria - healthcare worker: - doctors, nurses, social workers, pharmacists and educators who participate in the care of hemophilia b patients exclusion criteria: * diagnosis of hemophilia a * diagnosis of other non-hemophilia b bleeding disorders exclusion criteria - healthcare worker: * health care workers who do not participate in the care of hemophilia b patients * healthcare worker who is conducting the interviews
fluoroscopy radiation reduction during sacral neuromodulation lead placement

Conditions: Radiation Exposure; Overactive Bladder; Urge Incontinence; Urgency-frequency Syndrome

Summary: fluoroscopy is performed when placing a lead during a sacral neuromodulation procedure. during lead placement, subjects will receive either conventional or experimental fluoroscopic settings. the radiation exposure will be compared between the two groups.

patients scheduled for lead implantation during sacral neuromodulation in the operating room and consent will be included in this study. placement of the lead in the operating room with fluoroscopy is the standard of care in this procedure. fluoroscopy is performed with a c-arm and there are settings that change the radiation exposure. patients scheduled to undergo lead placement will be randomized to: a) the investigational (reduced radiation with fluoroscopic settings) or b. the control (conventional fluoroscopy). the surgeon can change fluoroscopy radiation exposure by changing c-arm settings from the reduced radiation to conventional fluoroscopic options. during the surgery, the surgeon may change the settings from reduced radiation (intervention) to conventional fluoroscopy (control) to safely place lead. the surgeon will use their judgment to optimally place lead and maintain safety during placement. any deviation from randomization will be recorded. the intervention arm is defined: reduced radiation fluoroscopy technique is performed by the c-arm set at 1 pulses-per-second and reduction of current. the control arm is defined: the standard of care in the conventional fluoroscopy, the c-arm is set at 30 pulses-per-second and the current set at the default.

Eligibility criteria:
inclusion criteria: * patients undergoing lead implantation for sacral neuromodulation that have overactive bladder as defined by urinary urgency, frequency, nocturia with or without urgency incontinence. exclusion criteria: * neurogenic bladder, bmi \>40, or peripheral neuropathy.

Keywords: sacral neuromodulation fluoroscopy
implementation of a personalized medicine (pharmacogenomics) service in a community pharmacy

Conditions: Stroke; Transient Ischemic Attack; Myocardial Infarction

Summary: the purpose of this study is to determine the feasibility of pharmacogenomics testing in a community pharmacy using clopidogrel as an example. the investigators hypothesize that this testing is feasible in this setting.

to determine if the study is feasible, we will examine the change in patient perception of pharmacogenomics testing (before and after the study), the percentage of patients interested in this service, the response rate of providers to pharmacist recommendations, the pharmacist time requirement, and reimbursement rate for pharmacist services.

Eligibility criteria:
inclusion criteria: * prescribed clopidogrel (plavix) by their prescriber * aged 18 or older * currently on clopidogrel therapy for one of the following indicated uses: post-acs, recent pci with stenting, history of tia or stroke exclusion criteria: * are unable to complete study materials (surveys) with or without assistance, including non-english speaking patients * are taking clopidogrel for a reason other than stated in inclusion criteria * are pregnant or nursing * have an allergy to aspirin

Keywords: pharmacogenomics feasibility community pharmacy personalized medicine clopidogrel plavix
a study evaluating the safety, pharmacokinetics, and clinical effects of intravenously administered pt-112 injection in subjects with advanced solid tumors and subsequent dose expansion cohorts

Conditions: Advanced Solid Tumors; CRPC; mCRPC; Metastatic Castrate-resistant Prostate Cancer; PT-112; Prostatic Neoplasms; Genital Neoplasms, Male; Urogenital Neoplasms; Neoplasms by Site

Summary: this is a phase 1/2, open-label, multi-center, non-randomized, dose-escalation study to be conducted in two parts: the dose escalation phase and the dose expansion phase. the dose escalation phase will determine the maximum tolerated dose (mtd) and recommended phase 2 dose(s) (rp2d) of pt-112 injection and evaluate its safety and tolerability, and pk (pharmacokinetics). the dose escalation phase is complete and no longer enrolling. the dose expansion phase has two cohorts: one cohort for the study of pt-112 in patients with thymoma and thymic carcinoma (cohort a), and one cohort for the study of pt-112 in metastatic castrate-resistant prostate cancer (mcrpc) (cohort d).

this is a phase 1/2, open-label, multi-center, non-randomized, dose-escalation study to be conducted in two parts: the dose escalation phase, and the dose expansion phase the dose escalation phase and the dose expansion thymoma cohort are complete and no longer enrolling. the dose expansion phase of the study of pt-112 in metastatic castrate-resistant prostate cancer (mcrpc) is open and enrolling.

Eligibility criteria:
key inclusion criteria: * male \>/= 18 years of age * histologically or cytologically confirmed adenocarcinoma of the prostate. * document current evidence of metastatic castration-resistant prostate cancer (mcrpc), where metastatic status is defined as having documented metastatic lesion(s) on either bone scan or ct/mri scan. * patients who have received at least three prior intended life-prolonging therapies for metastatic disease. * eastern collaborative oncology group (ecog) performance status of 0-1. * progressive disease, either measurable on physical examination or imaging by response evaluation criteria in solid tumors (recist v1.1) or pcwg3 or by informative tumor marker(s). * adequate organ function based on laboratory values. * if there is a known history of brain metastases, either treated or untreated, the disease must be stable. key exclusion criteria: * any cytotoxic chemotherapy within 21 days prior to initiation of study drug. * any immunomodulatory drug therapy, anti-neoplastic hormonal therapy, immunosuppressive therapy, corticosteroids, or growth factor treatment within 14 days prior to initiation of study drug. * bone marrow reserve which is not adequate for participation in this trial. * radiotherapy within 14 days prior to baseline. * fraction of radiotherapy to \>25 % of active bone marrow. * major surgery within 28 days prior to initiation of study drug.

Keywords: pt-112
courageous parents, courageous children

Conditions: Anxiety Disorder

Summary: anxiety disorders usually start in childhood and adolescence and are associated with social and occupational difficulties in adulthood. children who have a parent with an anxiety disorder and who find new situations distressing and avoid them are at an increased risk for developing an anxiety disorder. research suggests that anti-anxiety parenting can help children grow up courageous and calm. it is, however, difficult to parent in an anti-anxiety way when the parent has an anxiety disorder himself or herself. this research study will test the efficacy of a new program designed to prevent the onset or persistence of anxiety disorders in children at risk for anxiety disorders. the investigators will first help parents learn skills to cope with their own anxiety and then coach them to share these skills with their children and parent in an anti-anxiety way. the goal is to intervene early enough in the children's lives so that they can be free of anxiety disorders and lead happy, healthy and productive lives in adulthood.


Eligibility criteria:
parents: inclusion criteria: parents will be included if they have a current diagnosis of at least one anxiety disorder, one or more children between the ages two and eight years in their own care, capacity to provide informed consent, and ability to speak english well enough to benefit from the intervention. exclusion criteria: exclusion criteria for parents are receipt of an adequate course (6 or more sessions) of structured psychological therapy in the past 12 months, or plans to move out of nova scotia within the next three years. we will include parents with co-morbid mood disorders. however, those whose mood disorder is the primary complaint will be referred for treatment for their mood before being re-assessed. we will exclude parents with disorders that would interfere with the standard treatment protocol delivery; these include lifetime schizophrenia, autism, intellectual disability and current drug and alcohol use disorder. offspring: inclusion criteria: children are eligible if they are aged two to eight and if they have a score of inhibited temperament (negative emotionality) one standard deviation or more above the mean of an age-matched normative sample on the laboratory assessment of temperament. exclusion criteria: those with lifetime schizophrenia, autism, general intellectual disability, and current drug and alcohol addiction will be excluded, as these conditions would impact on the standard delivery of the parenting intervention. we will exclude children currently receiving treatment for anxiety.
a study to evaluate the effects on the single-dose drug levels of mavacamten in healthy participants

Conditions: Healthy Participants

Summary: the purpose of this study is to evaluate the effects on the single-dose drug levels of mavacamten in healthy participants.


Eligibility criteria:
inclusion criteria: * body mass index between 18 and 32 kg/m\^2, inclusive, at the screening visit * healthy, as determined by physical examination, vital signs, electrocardiograms (ecgs), and clinical laboratory assessments within the normal range at the screening visit and/or on day -1 exclusion criteria: * current or recent (within 3 months of study intervention administration) gastrointestinal disease * history of any significant drug allergy (such as anaphylaxis or hepatotoxicity) other protocol-defined inclusion/exclusion criteria apply

Keywords: mavacamten healthy participants bioequivalence hypertrophic cardiomyopathy
improving walking in older adults with knee osteoarthritis

Conditions: Knee Osteoarthritis

Summary: knee osteoarthritis (oa) accounts for a significant proportion of mobility limitations and is one of the most disabling problems facing the growing population of older adults. the long-term objective of this research is to reduce disablement of older adults with symptomatic knee oa. the principle of specificity of training indicates that exercises that closely approximate the goal functional activity are most effective in improving physical performance during that activity. based on this principle, the specific aim of this pilot study is to design a patient-specific gait training intervention using analysis of compensatory joint moments and energy expenditure. successful completion will inform rehabilitation for maintaining or improving mobility as well as explore the mechanism of effect.


Eligibility criteria:
inclusion criteria: * symptomatic knee osteoarthritis (knee osteoarthritis diagnosed by x-ray and frequent knee symptoms) * age 60 or older exclusion criteria: * acute or terminal illness * unstable cardiovascular condition or other medical conditions that may impair ability to participate such as pulmonary disease requiring the use of supplemental oxygen, neurological disease that affects gait, or lower limb musculoskeletal surgery in the past 6 months. * bilateral knee replacement

Keywords: osteoarthritis gait training knee pain rehabilitation physical therapy
liberia insecticide treated durable wall linings study: protocol for a cluster randomised trial (dl)

Conditions: Malaria, Falciparum; Anemia

Summary: a cluster-randomized control trial will study the effect of insecticide-treated wall lining on malaria transmission in bomi county, liberia. half of the villages enrolled in the study will receive insecticide-treated wall lining covering their walls and ceilings in addition to long-lasting insecticidal nets, while the other half will be protected by existing long-lasting insecticidal nets.

long-lasting insecticidal nets (llins) are the established vector control product for malaria control. in bomi county liberia, llins have been distributed but malaria prevalence in children under 5 remains high, with prevalence in study villages found to be 42% in april 2014. the major malaria vector species are resistant to pyrethroid insecticides approved for use in llins, and new control measures with non-pyrethroid active ingredients may protect the population.

Eligibility criteria:
inclusion criteria: * child resides in village * child presents with parent or guardian exclusion criteria: * child or guardian has history of ebola exposure * child has 3 symptoms of ebola
indole-3-carbinol effects on estrogen metabolism

Conditions: Obesity

Summary: this study will test a dietary supplement, indole-3-carbinol, for improving the estrogen profile in women across a range of body mass index.


Eligibility criteria:
inclusion criteria: * woman aged from 30 - 50 years * regular menstrual cycle exclusion criteria: * taking hormone replacement or hormonal contraception * thyroid disorder * uncontrolled asthma * bmi under 18.5 * allergy to cruciferous vegetables

Keywords: dietary supplement indole 3 carbinol estrogen
development of a new neuroimaging method aimed to differentiate mnesic abilities of alzheimer and depressed patients

Conditions: Alzheimer Disease; Depression

Summary: the purpose of this study is to use a new method to differentiate mnesic abilities of early alzheimer patients and patients with a depressive disorder. this method is based on the recording of cerebral activity while patients listen to familiar, unfamiliar and newly-learned music.


Eligibility criteria:
inclusion criteria: * being right-handed * recorded consent * for alzheimer patients: mini mental status examination (mmse) score \>= 18 * for patients with a depressive disorder : major depressive disorder according to diagnostic and statistical manual of mental disorders (dsm iv) and montgomery-asberg depression rating scale (madrs) score \>= 25 * for healthy controls : no cognitive deficit, madrs score \< 25, no depressive disorder according to dsm iv exclusion criteria: * auditory deficit * contraindication to a high-density electroencephalography exam * refusal to participate in the study

Keywords: electroencephalography (eeg)
evaluation of a desipramine ceiling in cocaine abuse - 1

Conditions: Cocaine-Related Disorders; Substance-Related Disorders

Summary: the purpose of this study is to confirm whether a blood level ""ceiling"" exists on desipramine effect in cocaine abuse.


Eligibility criteria:
please contact site for information.

Keywords: cocaine dependence
understanding changes in heart function after exercise in people with stroke

Conditions: Stroke

Summary: little is known about how exercise can affect heart function in stroke survivors. this study will look at changes in cardiovascular fitness and function after 6 months of exercise. volunteers will be tested before and after the program and again 2 months later to measure the effects on stroke risk factors, fitness level and flexibility of the arteries. blood tests will measure cholesterol levels and other signs of inflammation. this study will help us better understand how exercise after stroke can improve heart function and heart health, and ultimately help in lowering future stroke risk.

purpose to determine the effects of aerobic exercise after stroke on cardiorespiratory fitness, cardiovascular function, stroke risk factors, walking and health-related quality of life. this study will also examine the retention of these effects after the exercise program ends. methods a randomized controlled study design will be used. sixty participants will be randomized to participate in one of 2 treatment arms (6 months duration, 3 days / week, 1 hour / session): 1. balance and flexibility (non-aerobic) program will be comprised of individualized upper and lower body stretching, or balance activities, yoga- or tai chi-style classes. 2. aerobic program will be comprised of a structured, progressive aerobic training program. participants will be evaluated at 3 time points - baseline (0 months), post-intervention (6 months) and follow up (8 months) - for stroke risk factors, cardiorespiratory fitness, arterial stiffness, echocardiography measures, inflammatory biomarkers, ambulatory capacity and health-related quality of life.

Eligibility criteria:
inclusion criteria: * age 50-80 years old * has experienced a stroke * at least 12 months post-stroke * able to stand independently * able to walk at least 5 metres (assisted devices and guarded supervision is permitted) exclusion criteria: * stroke of non-cardiogenic origin (aneurysm, tumour) * actively engaged in other stroke rehabilitation services * class c or d american heart association risk criteria (uncontrolled arrhythmias) * resting blood pressure \> 160/100 mmhg * significant musculoskeletal problems (rheumatoid arthritis) or other neurological conditions (e.g. parkinson's) * has a pacemaker * pain or other co-morbidities (e.g. unclipped aneurysms, uncontrolled seizures etc.) which would preclude participation * behavioural issues that would limit participation in exercise

Keywords: stroke exercise rehabilitation risk factors
renal resistive index as a predictor of acute kidney injury and evaluation of fluid administration in sepsis

Conditions: Renal Resistive Index; Acute Kidney Injury; Sepsis

Summary: we aim from this study to investigate the role of renal resistance index (rri) in evaluation of acute kidney injury development and fluid administration in sepsis patients considering the change in rri values over 7 days from admission as a predictor of aki development

acute kidney injury (aki) is one of the most common problems in critically ill patients in the clinic. aki can be caused by various factors, such as hypovolemia, shock, major surgery, trauma, and heart failure, of which sepsis is the most common

Eligibility criteria:
inclusion criteria: * age over 21 years * meet sepsis 3 criteria (potential source of infection , host response and organ dysfunction) but not in septic shock. exclusion criteria: * patients during pregnancy. * patients with hepatorenal syndrome. * poor abdominal echogenicity eg.(morbid obesity ,increase intra abdominal pressure ) * severe acute or chronic renal insufficiency . * dialysis dependency. * renal transplantation. * known renal artery stenosis. * mono-kidney, kidney tumor, anatomic kidney abnormalities.
growth hormone and intrahepatic lipid content in patients with nonalcoholic fatty liver disease

Conditions: Nonalcoholic Fatty Liver Disease (NAFLD)

Summary: nonalcoholic fatty liver disease (nafld), fatty infiltration of the liver in the absence of alcohol use, is an increasingly recognized complication of obesity, with prevalence estimates of about 30% of individuals in the united states. a subset of these will develop progressive disease in the form of nonalcoholic steatohepatitis (nash), which can progress to cirrhosis and liver failure. nafld is expected to be the most common indication for liver transplantation by the year 2020. we hypothesize that growth hormone (gh) replacement will decrease intrahepatic lipid accumulation as quantified by 1h magnetic resonance spectroscopy (1h-mrs).


Eligibility criteria:
inclusion criteria: 1. ages 18 - 65 yr 2. nafld defined as demonstration of hepatic steatosis by imaging or biopsy in absence of significant alcohol consumption and other causes of hepatic steatosis. if liver imaging or biopsy has not been performed clinically, liver ultrasound will be performed as part of the screening visit. exclusion criteria: 1. serum creatinine \> 2 times the upper limit of normal 2. history of cancer, except for non-melanoma skin cancers 3. active carpel tunnel syndrome 4. diabetes mellitus, defined as a hemoglobin a1c \>6.5 or use of any medications prescribed to treat hyperglycemia. the exception is that the use of metformin is acceptable in patients whose hba1c has been =\<6.0 on two visits and whose weight has remained stable for six months. 5. contraindications to magnetic resonance imaging (mri). 6. pregnancy or desire to become pregnant. participants of reproductive age must agree to use contraception. 7. breastfeeding 8. aspartate and aminotransferase levels \>10x upper limit of normal