Kornimate/medical-research-clean huggingface dataset:
safety study of tarceva in children with refractory and relapsed malignant brain tumors and newly diagnosed brain stem glioma

Conditions: Malignant Brain Tumor; Brain Stem Glioma,

Summary: the purpose of this study is to establish the recommended dose/maximum tolerated dose (mtd) of tarceva in children as single agent and in combination with radiation therapy

prognosis in relapsing malignant brain tumors is poor. those in brain stem gliomas is dismal; median survival of these children does not exceed 9 months. radiation therapy may result in early and transient amelioration of symptoms, but have not contributed to increase or prolong survival. moreover, chemotherapy has not increased this outcome to date.prados et al. reported encouraging results from a phase i study of tarcevatm/osi-774 alone or with temozolomide (tmz) in patients with malignant gliomas. of 25 evaluated patients, 6 experienced pr: 4 gbm (glioblastoma multiforme) and 1 grade 3 astrocytoma treated with tarcevatm alone, 1 gbm treated with tarcevatm/tmz; 2 had minor responses, and 3 stable diseases. these results in malignant glioma and the lack of efficacy in brain stem glioma with current treatment suggests the evaluation of this new therapeutic agent in children with relapsed brain tumors and upfront at diagnosis in brain stem glioma in combination with radiation therapy.

Eligibility criteria:
inclusion criteria: * histologically or cytologically confirmed malignant brain tumor * disease must be considered refractory to first line or relapsing after conventional therapy and for which no effective conventional treatment exists.· * newly diagnosed, histologically proven brain stem glioma, except pilocytic astrocytomas. * age: 1 to ≤ 21 years of age at study entry * life expectancy: at least 8 weeks * ecog performance status ≤ 1 or lansky-play scale\>= 70%, and including children with motor paresis due to disease * measurable or evaluable disease * no other serious concomitant illness * no organ toxicity \> grade 2 nci-ctc ae v3.0, except alopecia and neurological symptoms due to disease exclusion criteria: * patients with spontaneous intratumoral hemorrhage will not be included in the study, in exception of small post-biopsy hemorrhage due to biopsy procedure * pregnant and breast feeding woman * uncontrolled intercurrent illness or active infection * chemotherapy within 4 weeks prior to study medication (within 6 weeks, if the regimen contained a nitrosourea) * radiation therapy within 6 weeks prior to study medication * any clinical or non-clinical evidence of pulmonary dysfunction or pre-existing lung disease * severe cardiac pathology; history of myocardial infarction within the year prior to inclusion * any significant ophthalmologic abnormality, especially severe dry eye syndrome, keratoconjunctivitis sicca, sjögren syndrome, severe exposure keratitis or any other disorder likely to increase the risk of corneal epithelial lesions * treatment with coumarin (warfarin)

Keywords: tarceva refractory and relapsed malignant brain tumors newly diagnosed brain stem glioma
screening for barrett's esophagus in otolaryngology patients

Conditions: Extraesophageal Symptoms; Cough; Throat Clearing; Hoarseness; Difficulty Swallowing

Summary: the purpose of this project is to develop an accurate method to identify patients that suffer from acid reflux, but may not present with classic reflux symptoms (such as heart burn). additionally, it is the purpose of this project to utilize the unsedated small-caliber endoscopy (use) to assess the prevalence of barrett's esophagus in a population of patients with laryngopharyngeal reflux (lpr) symptoms and to define normal patterns of lpr. laryngopharyngeal reflux (lpr) is when a small amount of stomach contents and acid are pushed up through the esophagus and may affect the vocal cords and upper respiratory tract. it is suspected that patients with lpr symptoms may have a prevalence of barrett's esophagus similar to that found in a population with typical reflux symptoms. we propose to systematically test this hypothesis using the use. this project is designed to improve the methods of identifying patients with a form of acid reflux that is often undetected, and thus untreated. if this project is successful then more patients with barrett's esophagus will be identified; furthermore, screening and treatment for this pre-cancerous condition will be improved.


Eligibility criteria:
inclusion criteria: - exclusion criteria: * prior anti-reflux surgery, laryngeal surgery, trauma to larynx esophageal diverticulum pregnancy anticoagulation therapy esophageal varices ent malignancy history of recurrent epistaxis

Keywords: laryngopharyngeal reflux extraesophageal reflux symptoms
evaluation of galectin-3 in serum and saliva of pemphigus patients

Conditions: Galectin-3 and Pemphigus

Summary: assessment of the expression of galectin-3 in serum and saliva of pemphigus patients and compare it with age and sex matched controls. we want to detect whether a correlation exists between galectin-3 level in serum, as well as saliva and disease activity score by autoimmune bullous skin disorder intensity score (absis).

pemphigus is a potentially life- threatening immune mediated bullous disease that affects the skin and mucous membranes with a significant impact on quality of life (1). it is due to production of autoantibodies against desmogleins (adhesion molecules of the epidermis) leading to disruption of junctions between keratinocytes and subsequently acantholysis (2). pemphigus diseases are classified based on the clinical, histopathological features, as well as on the specific antigens against which the autoantibodies are produced. the main forms are pemphigus vulgaris (pv) and pemphigus folia¬ceus (pf), but other non-classical forms of pemphigus have also been described as paraneoplastic pemphigus, pemphigus herpetiformis, and iga pemphigus (3). pv is the main clinical form of pemphigus, accounting for approximately 70% of cases; it is also considered the most severe form of the disease (4). igg is the main immunoglobulin seen along the dermal-epidermal junction in pemphigus patients, in addition most patients generate ige autoantibody response. soluble cd23 and galectin-3 are the two main elements of the ige (5). galectin-3(gal-3) is a beta galactoside-binding lectins that is essential in the cell-to-cell or matrix adhesion, as well as plays an important role in cell growth, differentiation, macrophage activation, antimicrobial activity, angiogenesis, and apoptosis. it broadly exists in the nucleus and cytoplasm of various cell types or may be found extracellularly on the cell surface (6) .gal-3 is readily secreted to the cell surface and into biological fluids as serum, urine, tears and saliva from injured and inflammatory cells. so, it can be used as a sensitive diagnostic or prognostic biomarker for various pathological conditions (7). gal-3 has been used as a novel biomarker in the early detection of myocardial dysfunction and heart failure. many cardiac biomarkers which reflect cardiac inflammation and fibrosis may also contribute to the progression of kidney disease (8, 9) a previous study reported a significant decrease in galectin-3 cytoplasmic and nuclear expression around blisters compared with adjacent unaffected skin in pv, which may play a role in extension of acantholysis (5). to the best of our knowledge, no previous studies assessed the role of gal-3 in serum or saliva of pemphigus patients.

Eligibility criteria:
inclusion criteria: patients with active stage of pemphigus. exclusion criteria: 1. patients who were on topical ttt, systemic corticosteroids or other immunosuppressive drugs in the last one month. 2. patients with disease attack duration more than 1 week. 3. patients with any other concomitant dermatological or chronic systemic diseases. -
high dose inspiratory muscle training in lopd

Conditions: Late-Onset Pompe Disease; Lysosomal Disease

Summary: study objectives: 1) assess the safety and feasibility of high-dose inspiratory muscle training (imt) delivered remotely in late-onset pompe disease (lopd) and 2) determine its effects on respiratory and patient-reported outcomes.

this study aims to develop treatments that enhance respiratory strength and function to provide meaningful clinical improvements for people with lopd. identification of a cost-effective adjunctive intervention to address respiratory weakness remains critical to reduce disease burden, ease activity limitations and participation restrictions, and improve health-related quality of life. the proposed study will provide a high-dose inspiratory muscle training (imt) stimulus to enhance treatment efficacy and efficiency. our hypothesis is that high-dose imt is necessary to produce meaningful changes in respiratory muscle strength and other outcomes in participants with lopd.

Eligibility criteria:
inclusion criteria: * age ≥ 18 years * confirmed diagnosis of lopd * mip \>50% of predicted for sex and age * stable on current pompe disease treatment regimen \>6 months * able to follow directions for study participation * access to computer and smartphone/tablet with reliable internet connection for video visits and sensor-based respiratory technologies exclusion criteria: * presence of medical comorbidities that prevent meaningful study participation (e.g., copd gold iii-iv, significant mental illness, dementia) * use of continuous invasive or non-invasive ventilation while awake * prior history of gene therapy for lopd * inability to give legally effective consent * inability to read and understand english

Keywords: lopd
ezn-2279 in patients with ada-scid

Conditions: ADA-SCID; Adenosine Deaminase Deficiency; Severe Combined Immunodeficiency

Summary: the purpose of this study is to evaluate the safety, efficacy, and pharmacokinetics of ezn-2279 in patients with ada-deficient combined immunodeficiency currently being treated with adagen.


Eligibility criteria:
inclusion criteria: 1. diagnosis of ada-deficient combined immunodeficiency 2. stable clinical status while receiving therapy with adagen®. patients previously receiving gene therapy or undergoing hematopoietic stem cell transplantation who still require adagen® treatment are eligible. the dose of adagen® must be stable for at least 6 months prior to study entry. 3. have both of the following during the adagen® lead-in phase of the study prior to ezn-2279 transition: 1. trough plasma ada activity \>15 μmol/h/ml while receiving adagen® and 2. total erythrocyte daxp ≤0.02 μmol/ml from a trough blood sample 4. patients or parent/guardian must be capable of understanding the protocol requirements and risks and providing written informed assent/consent exclusion criteria: 1. autoimmunity requiring immunosuppressive treatment 2. patients with detectable neutralizing anti-adagen® antibodies at screening evaluation 3. severe thrombocytopenia (platelet count \<50 x 10\^9/l) 4. current participation in other therapeutic protocols for ada-deficient combined immunodeficiency 5. current or prior participation in another clinical study with an investigational agent and/or use of an investigational drug in the 30 days before study entry 6. known planned participation in a gene-therapy study for the planned duration of this study 7. any condition that, in the opinion of the pi, makes the patient unsuitable for the study 8. inability or unwillingness to administer adagen® or ezn-2279 on a one-time-per-week regimen 9. inability to comply with the study protocol 10. female patients who are pregnant or lactating 11. female patients who are breast-feeding 12. female subjects of childbearing potential who are not using an fda approved birth control method
phase 1 combination study of mm-151 with mm-121, mm-141, or trametinib

Conditions: Colorectal Cancer; Non-small Cell Lung Cancer; Squamous Cell Carcinoma of the Head and Neck

Summary: this is a phase 1 open-label, dose-escalation trial using "3+3" design, evaluating mm-151 co-administration with mm-121, mm-141, and trametinib at varying dose levels.

this is a two-part phase 1, non-randomized, open-label study of mm-151 plus mm-121, mm-141, or trametinib in patients with advanced, heregulin-positive lung, head and neck, and colorectal cancers. in part 1 of the study, cohorts of 3 or more patients will be treated at escalating doses of mm-151 in combination with mm-121, mm-141, and trametinib until a maximum tolerated combination dose for each combination is identified. in part 2 of the study, patients will be treated with the combination dose identified in part 1 of the study.

Eligibility criteria:
inclusion criteria: * patients must be \>18 years of age * patients must be able to provide informed consent * willing to abstain from sexual intercourse or to use an effective form of contraception during the study and for 90 days following the last dose of any study therapy). this applies to women of childbearing potential as well as fertile men and their partners * patients must be recovered from the effects of any prior surgery, radiotherapy or other antineoplastic therapy, up to ctcae grade 1 * patients must have either heregulin-positive cancer, cancer with ras mutation, igf-1 positive cancer, or ras wild type cancer. exclusion criteria: * patients who are pregnant or lactating * patients who have an active infection or with an unexplained fever \> 38.5°c during screening visits or on the first scheduled day of dosing (at the discretion of the investigator, patients with tumor fever may be enrolled.) * patients with untreated (primary) or symptomatic cns (primary or metastatic) malignancies; patients with cns metastases who have undergone surgery or radiotherapy or who have been on a stable dose of corticosteroids for at least 2 weeks and whose disease is stable prior to the first scheduled day of dosing will be eligible for the trial. * patients who have received other recent antitumor therapy including any standard chemotherapy or radiation within 14 days (and having passed the time of any actual or anticipated toxicities) prior to the first scheduled dose of the study treatment.

Keywords: heregulin-positive egfr erbb3 phase i oncology cancer igf-1 mekinhibitor igf-1r inhibitor kras nras
highcycle study: effect of acetazolamide on acute mountain sickness in women compared to men

Conditions: Acute Mountain Sickness

Summary: millions of people travel to high altitude for work or leisure activities and are exposed to reduced inspiratory oxygen partial pressure and hypoxemia that may lead to altitude illness, among which the most common form is acute mountain sickness (ams). the main ams symptoms are headache, malaise, weakness, and fatigue. prospective studies have shown that 20-60% of newcomers at 2500-4000m develop ams requiring them to take medications, while, at very high altitudes, ams may progress to high altitude cerebral oedema. whether women are more susceptible to ams remains insufficiently understood since no prospective study controlled for sex hormones, use of hormone contraception or assessed menstrual cycle phase (mcp) at altitude. therefore, women remain underrepresented and poorly characterized in high altitude studies. in addition, the efficacy and safety of 250 mg/day acetazolamide, the standard recommendation for ams prevention, has never been compared between sexes, although, women have presumably higher acetazolamide plasma concentration due to lower blood volume. given the known dose-dependent preventive but also side effects of acetazolamide and equal proportion of women and men among mountain travellers, there is an urgent need to conclusively quantify the efficacy and safety of pre-ventive acetazolamide therapy against ams in women compared to men.


Eligibility criteria:
inclusion criteria: * healthy, non-smoking men and women, age 18-44 years, without any disease and need of regular medication (including oral contraceptives). * bmi \>18 kg/m2 and \<30 kg/m2 * born, raised and currently living at altitudes \<1000 m * written informed consent * premenopausal women with an eumenorrheic cycle exclusion criteria: * other types of contraceptvies (contraceptives (hormonal intrauterine device, vaginal ring, subcutaneous injections or implants, among others) * pregnancy or nursing * anaemic (haemoglobin concentration \<10g/dl) * any altitude trip \<4 weks before the study * allergy to acetazolamide and other sulfonamides

Keywords: women hypoxia prevention acetazolamide altitude
ecg and echo characteristics of bbap

Conditions: Pacing Therapy; Cardiac Pacing

Summary: this is a prospective, single-site, non-randomized, interventional study. the purpose is to collect electrocardiographic (ecg) and echocardiographic (echo) parameters and computed tomography (ct) images (optional) during bachmann's bundle area pacing (bbap) and conventional pacing in the right atrial appendage (raa) to compare the differences between atrial pacing modalities and to further investigate the clinical outcomes of bbap.

bachmann's bundle (bb) has emerged as an alternative pacing location to conventional right atrial appendage (raa) pacing and is expected to avoid the interatrial conduction delay (iacd) and atrial dyssynchrony induced by raa pacing. a recent study reported in patients with atrial pacing indications and baseline iacd that decreased atrial arrhythmia burden, recurrence, and de novo incidence were found in bb pacing, compared with right atrial septal pacing and raa pacing. this study aimed to collect electrocardiographic (ecg) and echocardiographic (echo) parameters and ct images during bb area pacing (bbap) and conventional pacing in the right atrial appendage (raa) during regular pacemaker implantation procedures. the purpose is to compare the differences between atrial pacing modalities and to further investigate the clinical outcomes of bbap.

Eligibility criteria:
inclusion criteria: * patients aged \>= 18 years old * patients who are willing to provide informed consent * patients who are indicated to atrial pacing, dual-chamber pacing, or cardiac resynchronization therapy * patients who consent to bachmann's bundle area pacing exclusion criteria: * patients who are indicated for cardiac implantable electronic device replacement or upgrade * patients with defibrillation indications * patients who are pregnant or have a plan for pregnancy during the study * patients who have medical conditions that would limit study participation * patients who were already enrolled in other trials which would impact his/her participation

Keywords: bachmann&#39;s bundle pacing bradycardia physiological pacing
stem cell fistula plug in perianal crohn's disease

Conditions: Perianal Crohn's Disease

Summary: the investigators propose to study the safety of autologous mesenchymal stromal cell transfer using a biomatrix (the gore® bio-a®; fistula plug) in a phase i study using a single dose of 20 million cells. twenty adult patients (age 18 years or older) with refractory, complicated perianal fistulizing crohn's disease will be enrolled. subjects will undergo standard adjuvant therapy including drainage of infection and placement of a draining seton with continuation of pre-existing anti-crohn's therapy. six weeks post placement of the draining seton, the seton will be replaced with the msc loaded gore® bio-a® fistula plug as per current clinical practice. the subjects will be subsequently followed for fistula response and closure for 24 months. this is an autologous product derived from the patient and used only for the same patient.


Eligibility criteria:
inclusion criteria * males and females 18-65 years of age. * residents of the united states. * crohn's disease with single or multiple draining complex perianal fistulae (definition as below) for at least three months despite standard therapy (definition below). * concurrent therapies with corticosteroids, 5-asa drugs, thiopurines, mtx, antibiotics, and anti-tnf therapy are permitted. * all patients should have undergone a colonoscopy in last 12 months to rule out malignant or premalignant condition * have no contraindications to mr evaluations: e.g. pacemaker or magnetically active metal fragments, claustrophobia * ability to comply with protocol * competent and able to provide written informed consent * must have failed standard medical therapy including anti-tnf agents exclusion criteria * inability to give informed consent. * clinically significant medical conditions within the six months before administration of mscs: e.g. myocardial infarction, active angina, congestive heart failure or other conditions that would, in the opinion of the investigators, compromise the safety of the patient. * specific exclusions: evidence of hepatitis b, c, or hiv * history of cancer including melanoma (with the exception of localized skin cancers) * investigational drug within thirty (30) days of baseline * a resident outside the united states * pregnant or breast feeding. * history of clinically significant auto-immunity (other than crohn's disease) or any previous example of fat-directed autoimmunity * previous allergic reaction to a perianal fistula plug. * if liposuction is not technically feasible * allergic to local anesthetics * pregnant patients or trying to become pregnant. * non-enterocutaneous tracts (ie recto-vaginal, entero-vesicular)
ultraviolet light exposure and immunosuppression in cutaneous melanoma

Conditions: Melanoma

Summary: this project seeks to understand differences in the serum vitamin d levels and immune status in cutaneous malignant melanoma patients with different uv exposure histories in new mexico.

it is well established that ultraviolet radiation (uv) exposure is related to the development of melanoma. there is also evidence that immune reactions are altered after uv exposure in the skin (locally) and perhaps throughout the body (systemically). additionally, while the role of vitamin d and melanoma development has not been fully established, uv-b exposure is essential for vitamin d production in the skin. increased sun exposure is also related to the presence of solar elastosis, which might protect (1) or improve survival from melanoma. thus, melanoma represents a unique model for studying uv exposure, the immune system, and vitamin d. malignant melanoma is an antigenic cancer; therefore, the role of uv exposure-induced immunosuppression and vitamin d production in the recognition, destruction and growth inhibition of cancerous melanocytes is worth further study. to underscore the importance of this project, the scientific advisory committee of the melanoma research foundation and the steering committee of the society of melanoma research have indicated a need to collect more human data on the host immune response mechanisms in melanoma and also to focus on the skin as a whole microenvironment, moving away from only in vitro experiments.

Eligibility criteria:
inclusion criteria: * resident of new mexico * age between the 1 and 95 * newly diagnosed with cutaneous invasive melanoma; any stage permitted (icdo c44.0-9) between september 1, 2008 and december 31, 2011. * ssm (superficial spreading melanoma) and nm (nodular melanoma) histological subtypes are allowed * access to a telephone or be able to participate in a personal interview at the clinic * physically and mentally competent to complete a 1-hour telephone or personal interview exclusion criteria: * ocular melanoma, lmm (lentigo maligna melanoma) or alm (acral lentigo melanoma), and mucosal melanoma cases. * no access to a telephone or is not able to meet with the interviewer.

Keywords: melanoma skin cancer vitamin d vitamin d receptor sunlight cytokine