Kornimate/medical-research-clean huggingface dataset:
n2012-01: phase 1 study of difluoromethylornithine (dfmo) and celecoxib with cyclophosphamide/topotecan

Conditions: Neuroblastoma

Summary: this study will combine an oral drug called dfmo with celecoxib (also oral) and two iv chemotherapy medicines called cyclophosphamide and topotecan. * to find the highest dose of dfmo that can be given with celecoxib, cyclophosphamide and topotecan without causing severe side effects. * to find out the side effects seen by giving dfmo at different dose levels with celecoxib, cyclophosphamide and topotecan. * to measure the levels of dfmo in the blood at different dose levels. * to determine if your tumor gets smaller after treatment with dfmo, celecoxib, cyclophosphamide and topotecan. * to determine if specific gene changes in you or your tumor makes you more prone to side effects or affects your tumor's response to the combination of dfmo, celecoxib, cyclophosphamide and topotecan. * to determine if the amount of normal chemicals in your body called polyamines go down in response to dfmo, celecoxib, cyclophosphamide and topotecan, and whether you are more likely to have a good response to the treatment if they do.


Eligibility criteria:
inclusion criteria: * patients must be \> 2 years and \< 30 years of age when registered on study. * patients must have recurrent/progressive high-risk neuroblastoma, refractory high-risk neuroblastoma that had less than a partial response to standard treatment or persistent high-risk neuroblastoma that had at least a partial response to standard treatment. * all patients must have at least one site of evaluable disease. * patients must have adequate heart, kidney, liver and bone marrow function. * patients who have bone marrow disease must still have adequate bone marrow function to enter the study. * patients with other ongoing serious medical issues must be approved by the study chair prior to registration. exclusion criteria: * females of childbearing potential that do not have a negative pregnancy test. * patients that are pregnant, breast feeding, or unwilling to use effective contraception during the study * patients status post allogeneic stem cell transplant. * patients who, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study. * patients with disease of any major organ system that would compromise their ability to withstand therapy. * patients who are on hemodialysis. * patients with an active or uncontrolled infection. patients on prolonged antifungal therapy are still eligible if they are culture and biopsy negative in suspected radiographic lesions and meet other organ function criteria. * patients with active bleeding of the gi tract or patients who have symptoms associated with stomach irritation (known as gastritis). * patients who have had a seizure within 12 months prior to enrollment and patients receiving anti-convulsant therapy for a seizure disorder. * patients with known aspirin-hypersensitivity triad (asthma, allergic rhinitis, asa hypersensitivity). * patients with known hypersensitivity to celecoxib or other nsaids, aspirin or sulfonamides.
dexamethasone for reduction of post thoracotomy pain syndrome

Conditions: Chronic Pain; Dexamethasone; Thoracic Surgery; Cytokines

Summary: post thoracotomy pain syndrome still affects 50% of patients after a thoracic surgery. pathogenesis is unclear but there is growing evidence that neuro inflammation may play a significant role. dexamethasone is a very potent anti-inflammatory drug. the hypothesis of this study is that dexamethasone, given pre operatively, would help reduce the incidence of post thoracotomy pain syndrome 3 months after surgery, by reducing the inflammatory response, in patients undergoing elective thoracic surgery that includes thoracic epidural analgesia.


Eligibility criteria:
inclusion criteria: * elective thoracic surgery using a thoracotomy incision and thoracic epidural analgesia * asa i-iii * age 18-75 yo exclusion criteria: * contra-indication to thoracic epidural analgesia * patient refusal * patients already taking cortisone or dexamethasone * patients on chronic opioid medication
study to evaluate the safety and tolerability of rasagiline in advanced parkinson's disease patients

Conditions: Parkinson's Disease

Summary: this is a study to look at the effectiveness, tolerability, and safety of one dose of rasagiline in advanced parkinson's disease (pd) patients who have been treated with levodopa/carbidopa therapy.


Eligibility criteria:
inclusion criteria: * patients must have completed the week 26 visit of tvp 1012/133 (visit 06) in accordance with the protocol. * women must be postmenopausal, surgically sterile, or using adequate birth control methods. women of childbearing potential must have a negative pregnancy test at baseline/month 0. * patients must be willing and able to give informed consent. exclusion criteria: * serious or severe, test drug-related (probable or definite) adverse reaction in study tvp 1012/133. * premature discontinuation from study tvp 1012/133 for any reason. * a clinically significant or unstable medical or surgical condition which would preclude safe and complete study participation. such conditions may include cardiovascular, pulmonary, hepatic, renal, or metabolic diseases; or malignancies, as determined by medical history, physical exam, skin evaluation, laboratory tests, chest x-ray, or electrocardiogram (ecg).
energy balance and weight gain with ivacaftor treatment

Conditions: Cystic Fibrosis

Summary: ivacaftor is a novel, fda approved new therapy that addresses cystic fibrosis transmembrane conductance regulator (cftr) dysfunctions in subjects with cystic fibrosis (cf) and "gating mutations". the primary aim is to determine the mechanism(s) for weight gain in participants whom ivacaftor treatment was initiated based on clinical indications by cf care team. this longitudinal study will assess in detail energy expenditure, weight gain, body composition, and lung function in 24 subjects ≥6 years old with cf with a gating mutation before treatment and after three months treatment with ivacaftor. all subjects will be seen at the children's hospital of philadelphia's clinical translational research center.

cystic fibrosis (cf) is a genetic disease caused by mutations in the gene encoding the cf transmembrane conductance regulator (cftr), a chloride channel. most cf mutations either reduce the number of cftr channels at the cell surface (synthesis or processing mutations) or impair channel function (gating or conductance mutations). ivacaftor (kalydeco, vx-770) is a novel, fda approved new therapy that addresses cftr dysfunctions in subjects with cf and "gating mutations", specifically; it potentiates cftr channel function. for mutations like g551d that permit cftr expression at the cell membrane but compromise its activity, ivacaftor increases the probability that the channel is open and active. in previous randomized, double-blind, placebo controlled trials, ivacaftor treatment resulted in clinically significantly improvements in pulmonary function, weight and body mass index (bmi), and significant decreases in sweat chloride reflective of increased cftr activity. the improvements in lung function and weight occurred over the first 8 weeks of treatment, plateaued and were sustained over the 48 weeks of the trial. the mechanism for the rapid and sustained weight gain is not known. several mechanisms are considered in this proposal which may result in improved energy balance and energy utilization, and weight gain. these include decreased resting energy expenditure, increased energy and fat absorption from the gut, improved pancreatic enzyme and ph secretion, and increased energy intake. improvements in weight and bmi status are expected to result from this improvement in energy balance and utilization, with potential beneficial effects on muscle mass and function and quality of life.

Eligibility criteria:
inclusion criteria: * cystic fibrosis with one or two cftr gating mutations * age: 6 years and older * a clinical decision has been made for the subject to start ivacaftor treatment * in usual state of good health * family and subject commitment to the 3-month study protocol with two, 3-4 day visits to chop exclusion criteria: * fev1 \< 40% predicted * use of any inhibitors or inducers of cytochrome p450 (cyp) 3a * pregnancy or breast feeding * other illness affecting growth or nutritional status * subjects receiving total parenteral nutrition
office-based validation of the jvphome

Conditions: Congestive Heart Failure

Summary: the jvphome is a novel medical device that utilizes machine vision in order to facilitate assessment of jugular venous pressure (jvp) height relative to the sternal angle with the ultimate goal of enabling remote jvp monitoring. the current study aims to evaluate the ability of an enhanced version of the jvphome to enable remote identification of jvp height relative to ultrasound among congestive heart failure (chf) patients when the device is applied by the study team in the clinic.

chf is a common and devastating health problem estimated to affect upwards of 26 million individuals worldwide. following an initial hospitalization for heart failure, approximately 25% of patients are readmitted within the first 30 days of discharge. repeat heart failure hospitalizations are a major burden on both patients and healthcare systems. to date, there is an absence of non-invasive solutions proven to reduce the risk of re-hospitalization for chf. the key feature of the physical examination for assessing the volume status of chf patients is the jvp, which manifests as a biphasic pulsation along the neck. the height of the jvp relative to the sternal angle is a reflection of right atrial pressure, which increases in response to higher pressures transmitted from the left side of the heart. pathologic elevation of left sided heart pressures can result in fluid congestion in the lungs, which can cause patients to develop shortness of breath and reduced blood oxygen levels. although a crucial marker of volume status, accurate identification of the jvp can be challenging for physicians due to its pulsations often being subtle. the jvphome is a novel medical device designed to facilitate visualization of jvp height relative to the sternal angle through the use of machine vision. the current study aims to evaluate an enhanced version of the jvphome in a clinic setting when the device is applied to chf patients by clinicians. following its application, the jvphome will be allowed to record a video of the jvp and images will subsequently be transmitted to a secure cloud and undergo analysis with machine vision software. visualization of the jvp will be facilitated through simultaneous electrocardiographic and pulse oximetry recordings by the jvphome. following its application, data is transferred to a secure cloud server for subsequent processing and clinical review. jvp height of study participants will be concurrently assessed through the use of ultrasound (gold standard). clinicians that are blinded to ultrasound determined jvp height values will assess jvp height generated by the jvphome. analyses will then assess the accuracy of the jvphome relative to ultrasound and expert clinical assessment.

Eligibility criteria:
inclusion criteria: * age \>/= 18 years * congestive heart failure diagnosis receiving ongoing treatment * able to provide informed consent exclusion criteria: * conditions which preclude visualization of the jvp, including neck deformity, scarring, and facial hair

Keywords: remote monitoring, machine vision
research of biomarkers in duchenne muscular dystrophy patients

Conditions: Duchenne Muscular Dystrophy (DMD)

Summary: the purpose of this study is to identify potential biomarkers for the diagnosis, disease progression assessment and response to treatment in patients with duchenne muscular dystrophy.


Eligibility criteria:
inclusion criteria: * for patients: * diagnosis of dmd confirmed by genetic testing * age over 3 years * weight over 15 kg * informed consent signed * for controls: * age over 3 years * male gender * weight over 15 kg * subjects with national health insurance coverage * informed consent signed * nonacute or chronic muscular, allergic, infectious, endocrine or inflammatory disorder in the 3 weeks preceding inclusion exclusion criteria: * for patients: * concomitant chronic or acute muscular, endocrine, infectious, allergic or inflammatory disorder in the three weeks preceding the blood test * intake of medicines other than angiotensin-converting enzyme inhibitors, beta blockers, dietary supplements, vitamins, alendronate and methylphenidate. steroids (and medicines prescribed with them such as calcium supplements and proton pump inhibitors) will be discussed * mental retardation or autism * vaccination or treatment with immunoglobulins within the three months preceding inclusion * for controls: * concomitant chronic or acute muscular, neurological (including mental retardation and autism), infectious or inflammatory disorder in the three weeks preceding the blood test * vaccination or treatment with immunoglobulins within the three months preceding inclusion

Keywords: duchenne muscular dystrophy biomarkers transcriptomics mironics proteomics
a gene transfer therapy study to evaluate the safety of delandistrogene moxeparvovec (srp-9001) in participants with duchenne muscular dystrophy (dmd)

Conditions: Duchenne Muscular Dystrophy

Summary: this study was an open-label single-dose gene transfer therapy study evaluating the safety of delandistrogene moxeparvovec intravenous (iv) administration in boys with dmd. this study was originally designed to consist of 12 patients across 2 cohorts. cohort a would have included participants ages 3 months to 3 years, and cohort b included participants ages 4 to 7 years old. no participants were enrolled in cohort a.


Eligibility criteria:
inclusion criteria: * cohort a participants: 3 months to 3 years of age, inclusive * cohort b participants: 4 to 7 years of age, inclusive * definitive diagnosis of dmd based on documented clinical findings and prior genetic testing. * ability to cooperate with motor assessment testing. * cohort a participants: no previous treatment with corticosteroids. * cohort b participants: stable dose equivalent of oral corticosteroids for at least 12 weeks prior to screening and the dose is expected to remain constant (except for potential modifications to accommodate changes in weight) throughout the first year of the study. * cohorts a \& b: a frameshift mutation contained between exons 18 and 58 (inclusive). exclusion criteria: * exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression within protocol specified time limits. * abnormality in protocol-specified diagnostic evaluations or laboratory tests. * presence of any other clinically significant illness, medical condition, or requirement for chronic drug treatment that in the opinion of the investigator creates unnecessary risk for gene transfer. other inclusion or exclusion criteria could apply.

Keywords: duchenne gene therapy dmd dystrophin pediatric gene-delivery gene transfer therapy
pentacam findings after implantation of intrastromal corneal rings in keratoconus.

Conditions: Keratoconus

Summary: keratoconus is a progressive corneal ectatic disease characterized by paraxial stromal thinning and weakening, resulting in irregular astigmatism, corneal protrusion, and distortion of the anterior corneal surface. it is usually bilateral, although asymmetrical in most cases. intrastromal corneal rings are polymethylmethacrylate devices successfully used for the management of keratoconus, pellucid marginal degeneration, post-lasik ectasia and myopia. intrastromal corneal rings implantation is safe and reversible procedure that does not affect the central corneal area, and hence, avoids interference with visual axis. the goal of intrastromal corneal rings implantation is to improve visual acuity by regulariztion of the anterior corneal surface.


Eligibility criteria:
inclusion criteria: * keratoconus grade i, ii and iii according to the mean k amsler-krumeich classification. * contact lens intolerance. * thinnest location is more than 400 um. * maximum keratometry (kmax) between 48 and 64 d. * clear cornea with no vogt's striae. exclusion criteria: * previous corneal surgery. * grade iv with corneal scarring. * concurrent corneal disease in addition to kc. * autoimmune or systemic connective tissue diseases.
a dose escalation study of sta-9090 and docetaxel in patients with solid tumors

Conditions: Solid Tumor Malignancies

Summary: this is an open-label, phase 1, dose-escalation study to determine the recommended doses of sta-9090 (ganetespib) and docetaxel for the treatment of subjects with solid tumor malignancies. the safety and tolerability of the treatment will also be evaluated.


Eligibility criteria:
inclusion criteria: * must have histologically confirmed metastatic or unresectable malignancy with evidence of progression * if subject has been treated with docetaxel, must have evidence of persistent or progressive disease * measurable disease per recist * cns metastases are permitted if treated and radiographically and clinically stable for 4 weeks prior to first dose * ecog status less than or equal to 2 * life expectancy greater than 3 months * adequate hematological, hepatic and renal function as defined by protocol * willingness and ability to comply with study requirements * female subjects of childbearing age must have a negative pregnancy test at study entry * female subjects of child bearing age and males must agree to use adequate contraception as defined in the protocol exclusion criteria: * prior chemotherapy or investigational agents within 3 weeks or 5 times the agent's half life, whichever is shorter prior to first dose * radiotherapy within 2 weeks of first dose * surgery, radiotherapy or ablative procedure to the only area of measurable disease * major surgery within 4 weeks of first dose * poor venous access that would require an indwelling catheter for study drug administration * history of severe allergic or hypersensitivity reactions to sta-9090 or docetaxel or their diluents or excipients * baseline qtc \>470 msec or previous history of qt prolongation while taking other medications * peripheral neuropathy \> grade 1 * ventricular ejection fraction less than or equal to 55% at baseline * treatment with chronic immunosuppressants. however subjects may receive steroids for stable cns metastases * women who are pregnant or lactating * uncontrolled intercurrent illness

Keywords: solid tumor malignancies tumor cancer sta-9090 ganetespib hsp90 inhibitor
assessing the astigmatism reducing effect with toric iols in eyeswith low astigmatism

Conditions: Cataract; Astigmatism

Summary: comparison of the astigmatism reducing effect of a toric iol with a non toric iol in eyes with low corneal astigmatism.

with increasing demands of patients concerning refractive outcome after cataract surgery, toric intraocular lenses (iols) that correct corneal astigmatism have been introduced more widely to cataract surgery. originally, toric iols were used mainly for patients with high degrees of astigmatism. since a couple of years, toric iols are available from numerous manufacturers to correct moderate amounts of astigmatism which are much more prevalent with about 8% having a corneal astigmatism of 2.0d or more in the cataract population. 5using toric iols for these eyes results in less spectacle dependence of patients due to the astigmatic correction. other astigmatism reducing techniques, such as peripheral corneal relaxing incisions and opposite clear corneal incisions were shown to be less predictable. although the use of toric iols became the gold standard to correct corneal astigmatism during cataract surgery , there is still uncertainty, if low astigmatism should be corrected. visser et al. showed that moderate astigmatism of than 1.5d should be corrected for monofocal iols and hayashi showed that remaining astigmatism of 1.0d already decreases visual quality in eyes with multifocal iols. although correction of low corneal astigmatism appears to be beneficial, some hurdles have to be taken into account. in eyes with low corneal astigmatism the accuracy of measuring the astigmatism meridian is relatively low. furthermore, different corneal measurement techniques are not always comparable and it is difficult to know, which device shows the correct amount of astigmatism. additionally, the correction of the spherical equivalent is of high importance, as a refractive surprise will also attenuate the astigmatism reducing effect of the toric iol. aim of this study is to assess, if toric iols are useful in patients with low amounts of corneal astigmatism and to quantify the sources of error in toric iol power calculation.

Eligibility criteria:
inclusion criteria: * cataract * age 21 and older * regular corneal astigmatism 0.5d up to 1.5 d and difference between eyes not more than 0.75d * written informed consent prior to surgery exclusion criteria: * relevant other ophthalmic diseases such as: pseudoexfoliation, glaucoma, traumatic cataract corneal scars, and other co-morbidity that could affect capsule bag stability ( e.g. marfan syndrome) * irregular corneal astigmatism on corneal topography * in case of pregnancy (pregnancy test will be taken preoperatively in women of reproductive age)

Keywords: cataract low astigmatism
jab-23e73 in adult participants with advanced solid tumors with kras alteration

Conditions: Advanced Solid Tumors

Summary: this study is to evaluate the safety and tolerability of jab-23e73 in adult participants with advanced solid tumors

study jab-23e73-1002 is a multicenter, open-label phase 1/2a study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary anticancer activity of jab-23e73 as a single agent in adult patients with advanced solid tumors with kras alteration. this study consists of a phase 1a dose-escalation, followed by phase 1b dose-expansion and phase 2a indication expansion. after completing dose-escalation, the mtd or preliminary rp2d of jab-23e73 will be determined. the rp2d will be determined according to the safety, efficacy and pk data from phase 1b. the indication expansion phase will further explore the efficacy and safety in advanced kras-alternated tumors which consist of 3 cohorts: cohort c1, the crc cohort; cohort c2, the pdac cohort; and cohort c3, other solid tumor cohort.

Eligibility criteria:
inclusion criteria: * written informed consent. * participant must be ≥18 years of age at the time of signing the informed consent form (icf). * histologically or cytologically confirmed locally advanced or metastatic solid tumors that are not suitable for curative interventions. * patients must have kras alterations. * participants are required to provide an archived tumor sample. * patients with a life expectancy ≥3 months. * ecog performance status score of 0 or 1. * patients must have at least one measurable lesion as defined by recist v1.1. exclusion criteria: * inability to swallow oral medications, or presence of gastrointestinal dysfunction or gastrointestinal disorders that may significantly alter the absorption of the study drug. * patients who have previously been treated with kras g12c inhibitors, kras g12d inhibitors, or pan/multi-kras inhibitors. * known serious allergy to jab-23e73 or excipient. * patients with primary central nervous system tumors. * uncontrolled pleural effusion, pericardial effusion, or ascites requiring recurrent drainage procedures or medical intervention. * qt interval\>470 msec. * lvef ≤50% assessed by echo or muga.