Kornimate/medical-research-clean huggingface dataset:
an open-label extension study of edasalonexent in boys with duchenne muscular dystrophy

Conditions: Duchenne Muscular Dystrophy

Summary: the galaxydmd study is a global phase 3, open-label, treatment extension study to evaluate the safety, tolerability, and durability of effect in long-term dosing of edasalonexent in pediatric patients with a genetically confirmed diagnosis of dmd. patients who completed cat-1004-201 or cat-1004-301 or siblings of these boys from 4-12 years of age (up to 13th birthday) will be enrolled. edasalonexent is an orally administered small molecule that inhibits nf-kb, which is a key link between loss of dystrophin and disease pathology and plays a fundamental role in the initiation and progression of skeletal and cardiac muscle disease in dmd.

the study includes a 104-week open-label treatment period with edasalonexent. patients who completed cat-1004-201 or cat-1004-301 and eligible siblings of these boys will be enrolled in this trial.

Eligibility criteria:
for patients who completed cat-1004-201 or cat-1004-301: inclusion criteria: * written consent/assent by patient and/or legal guardian as per regional and/or institutional review board (irb)/independent ethics committee (iec) requirements * completion of either cat-1004-201 or cat-1004-301 exclusion criteria: * in the investigator's opinion, unwilling or unable for any reason to complete all study assessments and laboratory tests and comply with scheduled visits, administration of drug, and all other study procedures for siblings of patients who completed cat-1004-201 or cat-1004-301: inclusion criteria: * written consent/assent by patient and/or legal guardian as per regional and/or institutional review board (irb)/independent ethics committee (iec) requirements * a sibling of a patient who completed either cat-1004-201 or cat-1004-301 * diagnosis of dmd based on a clinical phenotype with increased serum creatine kinase (ck) and documentation of mutation(s) in the dystrophin gene known to be associated with a dmd phenotype * followed by a doctor or medical professional who coordinates duchenne care on a regular basis and willingness to disclose patient's study participation with medical professionals exclusion criteria: * use of oral corticosteroids at screening; use of inhaled, intranasal, and topical corticosteroids is permitted * use of another investigational drug, idebenone, or dystrophin-focused therapy within 4 weeks. exception: patients who are currently on or plan to initiate treatment with approved oligonucleotide exon-skipping therapies, and expected to continue treatment throughout the study, will be eligible * use of the following within 4 weeks prior to day 1: immunosuppressive therapy, anticoagulants, cyclosporine, dihydroergotamine, ergotamine, fentanyl, alfentanil, pimozide, quinidine, sirolimus or tacrolimus * use of human growth hormone within 3 months prior to day 1 * other prior or ongoing significant medical conditions

Keywords: muscular dystrophies musculoskeletal diseases neuromuscular diseases dmd dystrophin dystrophy duchenne
light therapy in disorders of consciousness: behavioral, neuroimaging and (neuro)physiological assessments.

Conditions: Disorder of Consciousness

Summary: in this randomized, double-blind, placebo controlled project, the investigators would like to assess the effect of a specific light, as compared to placebo light, on wakefulness (circadian rhythms, homeostasy, sleep-wake cycle), awareness (perceptual and self consciousness), cognition (attention, memory) and underlying brain activity (electrophysiology and neuroimaging)

the investigators will use behavioral (crs-r, cap, actimeter, brainstem reflexes), physiological (body core temperature, saliva, urine, heart rate, blood sample), neuroimaging and neurophysiological tools (pet, fmri, tms-eeg, resting-state and auditory paradigm eeg) before, during and after treatment.

Eligibility criteria:
inclusion criteria: * disorder of consciousness * 6 weeks post-injury exclusion criteria: * dysautonomia * acute illnesses (infections with fever) * medication that is known to affect circadian rhythmicity (melatonin) * uncorrected sensorial deficits or documented history of significant neurological, neurosurgical, developpemental or psychiatric disorders * previously known cerebral lesions prior to the brain's insult that lead to disorder of consciousness * any contraindication to mri, eeg, tms-eeg, or pet (e.g., electronic implanted devices, active epilepsy, external ventricular drain) * not medically stable
national acute promyelocytic leukemia (apl) observational study napoleon-registry of the german aml intergroup

Conditions: Newly-diagnosed APL (de Novo or Therapy-related); Relapsed APL

Summary: the registry aims to document epidemiologic data, treatment and long-term outcome as well as quality of life of patients with apl. additionally, a biobanking project for further translational studies is integrated. prospective population-based non-interventional and non-randomized multicenter registry.

* collection of epidemiological data for apl: age distribution, prognostic factors, distribution of subgroups, incidence * documentation of efficacy and safety of the first line and salvage therapy in apl including * documentation of minimal residual disease (mrd) * correlation of clinical outcomes with chosen therapy * collection and evaluation of quality of life * validation of published prognostic factors / new potential prognostic factors * acquisition of bone marrow, peripheral blood and buccal swab samples for biobanking and translational studies under the umbrella of the specific study-group biobanking concepts

Eligibility criteria:
inclusion criteria: * newly-diagnosed apl (either de novo or therapy-related), within 12 months of diagnosis * or relapsed apl, within 12 months of diagnosis of relapse 1. confirmed by the presence of the translocation t(15; 17) 2. and / or confirmed by the detection of the fusion transcript of pml/rara exclusion criteria: * none

Keywords: apl acute promyelocytic leukemia aml m3
decreasing narcotics in advanced pelvic surgery

Conditions: Narcotic Use; Pain; Constipation; Nausea

Summary: in recent years, there has been an emphasis on the creation of "enhanced-recovery", "fast-track" or "multi-modal" pathways to improve perioperative care (1-4). the goal of these programs is to reduce the length of hospital stay, decrease narcotic usage while improving pain control, accelerate post-operative recovery, and expedite return to baseline functional status. pathways often are developed by a team of surgeons, nurses, pain specialists, anesthesiologists and other support staff. postoperative components often involve multi-modal analgesia, early return to activity and early return to a regular diet. the goal of this study is to evaluate the efficacy of a multi-modal pain regimen in advanced pelvic surgery with a primary goal of decreasing narcotic usage.


Eligibility criteria:
inclusion criteria: * women \>/= 18 years old * undergoing pelvic organ prolapse or incontinence surgery with the urogynecology department exclusion criteria: * males * \<18 years old * women unwilling or unable to consent * same-day-discharge surgery * history of chronic pain for which they use medications * current or active history of narcotic abuse * sleep apnea * liver or kidney dysfunction * sulfa allergy

Keywords: narcotic use pain constipation nausea
investigating systemic and local vascular responses to apelin in the context of renin-angiotensin upregulation

Conditions: Heart Disease; Heart Failure; Vasodilatation

Summary: the apelin-apj system is a relatively new discovery. it has generated interest in part due to it's apparent ability to counteract the renin-angiotensin system, which is frequently overactive in many cardiovascular disease. two of the main actions of apelin are to increase the pumping ability of the heart and cause blood vessels to relax. the investigators wish to assess if these actions are altered in the setting of normal renin-angiotensin activation and increased renin-angiotensin activity.


Eligibility criteria:
inclusion criteria: * \>18yr exclusion criteria: * lack of informed consent * age \< 18 years, * current involvement in other research studies, * systolic blood pressure \>190 mmhg or \<100 mmhg * malignant arrhythmias * renal or hepatic failure * haemodynamically significant aortic stenosis * severe or significant co morbidity * women of childbearing potential. * any regular medication * previous history of any cardiovascular disease

Keywords: renin angiotensin system apelin cardiac output vasodilatation
a retrospective study to investigate the current situation of biopsy testing in swedish inoperable non small cell lung cancer (nsclc) patients

Conditions: Non Small Cell Lung Cancer

Summary: lung cancer is one of the most deadly types of cancer and the leading cause of death in cancer in sweden. five year survival is 10% in men and 15% in women. approximately 3300 individuals in sweden are diagnosed each year and the incidence of adenocarcinoma is increasing. lung cancer patients are to a great extent currently being diagnosed by exfoliative cytology. however, new drugs leading to more personalized treatments will demand more specific classification of tumour types. today egfr mutation status is becoming an important factor when deciding treatment strategy for patients with non-small cell lung cancer. sufficient tumour material must be available if egfr mutation status is to be tested. core needle biopsy is one way to obtain the quantity of material needed when testing mutation status. the portion of patients having core needle biopsies is believed to vary greatly between hospitals in sweden, a difference from 20% to 70 % have been assumed, but is not yet confirmed in studies. this study will investigate the current situation and procedures when patients are diagnosed with lung cancer. the results can be used to describe any possible adverse events connected to the procedure and possibly contribute to development of a better decision tool to be used when deciding if a core needle biopsy is to be performed or not. more and more therapeutical targets having similar problems are likely to be developed in the future. an investigation of current quality and procedures when diagnosing lung cancer by biopsies will facilitate future diagnosing of lung cancer and ensure that personalized treatments can be offered to patients.


Eligibility criteria:
inclusion criteria: * diagnosis code c34 (icd-10). * inoperable lung cancer. * diagnosis of lung cancer made between 2010-05-31 and 2009-06-01. exclusion criteria: * diagnosis code c34.9b. * diagnosis code c34.9h.

Keywords: biopsy mapping non small cell lung cancer nsclc lung cancer diagnosis retrospective inoperable non small cell lung cancer
effectiveness, safety and tolerability study of rp-g28 for symptoms associated with lactose intolerance

Conditions: Lactose Intolerance

Summary: this is a phase 2 study designed to assess the ability of rp-g28 to improve lactose digestion and tolerance.

lactose intolerance is a common gastrointestinal (gi) disorder that develops in lactose maldigesters when consuming too much lactose or when lactose is added to a previously low-lactose diet. lactose intolerance is characterized by one or more of the cardinal symptoms that follow the ingestion of lactose-containing foods. these symptoms include; abdominal pain/cramps, bloating, flatulence \[gas\] and diarrhea. as such, most lactose intolerant individuals avoid the ingestion of milk and dairy products, while others substitute non-lactose containing products in their diet. based on the health implications from insufficient calcium intake over a lifetime, including increased risk of osteoporosis and hypertension, there is need in the medical community for a tolerable and convenient treatment that allows for all levels of milk and dairy product consumption in people suffering from mild to severe lactose intolerance. this study will evaluate a treatment that provides a simplified dosing regimen as well as the potential for extended relief from symptoms following a limited therapy regimen.

Eligibility criteria:
inclusion criteria: * male and female subjects. female subjects must be non-pregnant and non-lactating. * 18 to 64 years of age inclusive at screening * current or recent history of intolerance to milk and other dairy products * acceptable baseline lactose intolerance symptom scores * acceptable result on baseline hydrogen breath test * subjects must agree to refrain from all other treatments and products used for lactose intolerance during the study exclusion criteria: * disorders known to be associated with abnormal gi motility * history of surgery that alters the normal function of the gastrointestinal tract * past or present; organ transplant, chronic pancreatitis, pancreatic insufficiency, symptomatic biliary disease, celiac disease, inflammatory bowel disease (ibd), ulcerative colitis, crohn's disease, irritable bowel syndrome (ibs) * active gastric or duodenal ulcers or history of severe ulcers * diabetes mellitus (type 1 or type 2) * congestive heart failure * history of human immunodeficiency virus (hiv), hepatitis b or c * use of concurrent therapy(ies) for symptoms of lactose intolerance * uncontrolled bp defined as sitting systolic blood pressure (sbp) ≥160 mmhg or diastolic blood pressure (dbp) ≥95 mmhg at visit 1 * history of ethanol abuse in the past 12 months * history of drug abuse within 12 months * history or presence of malignancy within the past 5 years (except basal cell or squamous cell carcinoma removed from a sun-exposed area) * use of any investigational drug or participation in any investigational study within 30 days prior to screening

Keywords: lactose maldigestion dairy intolerance intolerance to milk intolerance to dairy milk intolerance gi disorder lactose metabolism gi symptoms after dairy ingestion
uptake and biodistribution of 18f-fluorocholine in histiocytic disorders by pet imaging and biopsy measurement

Conditions: Erdheim-Chester Disease; Langerhans Cell Histiocytosis; Histiocytic Disorders

Summary: the main purpose of this study is to discover how 18f-cholinefch distributes in the body, and see if it can distinguish active histiocytes (tumor) from inflammatory (non- tumor) cells. this tracer has the potential to give the investigators' team more information when trying to identify the tumor cells that are most important to collect with biopsy. in addition, the study will measure levels of 18f-cholinefch in the biopsy tissue.


Eligibility criteria:
inclusion criteria: * age ≥ 18 years old * suspected but pathologically unconfirmed diagnosis of erdheim-chester disease or langerhans cell histiocytosis or other histiocytic disorder on the basis of clinical diagnosis, radiologic features, or findings from prior biopsies or * confirmed diagnosis of erdheim-chester disease or langerhans cell histiocytosis or other histiocytic disorder and requiring additional diagnostic imaging and biopsy to determine mutational status in order to determine therapeutic options. exclusion criteria: * inability to undergo or cooperate with pet/ct scan (e.g., claustrophobia) * pregnant or nursing female

Keywords: 18f-fluorocholine pet imaging biopsy 15-268
comparison of loop-tip wire vs tradictional technique in the cannulation of the common bile duct

Conditions: Post-ERCP Pancreatitis

Summary: selective cannulation of common bile duct (cbd) by insertion of a guide-wire seems to be associated with fewer complications and post-ercp (endoscopic retrograde cholangiopancreatography) pancreatitis (pep) rate than the conventional biliary tree access with cannulotome (ct-25 cook medical) with contrast injection even if results are conflicting. the aim of our study is to test a new guide-wire (loop-tip wire), with a loop in the tip, for the prevention of pep and biliary tree access, in pep high-risk patients.


Eligibility criteria:
inclusion criteria: * high-risk patients for post-ercp pancreatitis exclusion criteria: * age \< 18 years * allargy to the contrast medium * previous biliary or gastric surgery * neoplastic patients * previous biliary or pancreatic sphincterotomy

Keywords: loop-tip ercp cannulation of cbd
vivaer: a correlation between symptom scores and objective findings

Conditions: Nasal Obstruction; Medically Unexplained Symptoms; Airway Obstruction; Airway Remodeling

Summary: this is a prospective, single-center study. this study seeks to determine the objective improvement in nasal airflow in patients after undergoing the vivaer procedure, which involves the use of a stylus to deliver controlled and targeted low energy radiofrequency heating (heating by applying high-frequency radio waves) to the nasal sidewall to gently reshape the tissues. patients who experience refractory, or medically unexplained, nasal obstruction often have symptoms that are not alleviated or resolved by standard non-surgical treatment options. vivaer, a recently developed, fda-approved procedure, is one of the standard surgical treatments for nasal obstruction. unlike most of the other established surgical treatments for nasal obstruction, however, vivaer is a minimally-invasive procedure, and it is an outpatient intervention that can be performed under local anesthetic. eligible patients who are enrolled in the study will undergo the vivaer procedure, and will return to the clinic for three in-office follow-up visits at 4, 12, and 24 weeks after the procedure.


Eligibility criteria:
inclusion criteria: * adults 18 years and older seeking treatment for nasal obstruction and willing to undergo an office-based procedure. * nasal obstruction, defined as ≥60 by the nose scale. * the nasal valve is the primary or significant contributor to the subject's nasal obstruction as determined by the investigator, based on clinical presentation, physical examination, or nasal endoscopy. * subjects has symptomatic improvement with use of external or internal nasal dilators, q-tip or curette test (manual intranasal lateralization), or the cottle maneuver (manual lateral retraction of the cheek). * subject experienced minimal symptomatic improvement after the four-week fluticasone steroid nasal spray regimen. exclusion criteria: * prior surgery to the nasal valve, rhinoplasty, septoplasty, inferior turbinate reduction or other surgical nasal procedures within the past 12 months. * severe and/or chronic sinusitis, recurrent sinusitis, or allergies leading to nasal obstruction and currently requiring oral corticosteroid therapy. * severe case of any of the following; septal deviation, turbinate hypertrophy, polyps, or ptotic nasal tip believed to be the primary contributor to the subject's nasal obstruction symptoms and warranting surgical intervention. * known or suspected allergies or contraindications to the anesthetic agents and/or antibiotic medications to be used during the study procedure session. * known or suspected pregnancy, or lactation. * other medical conditions that the investigator believed would predispose subject to poor wound healing or increased surgical risk.

Keywords: vivaer symptom improvement
novel stent placement technique for intestinal stenosis assisted by an ultrafine endoscope

Conditions: Colorectal Carcinoma

Summary: colorectal cancer is the third most common cancer worldwide and results in 8-25% acute malignant bowel obstruction. since dohmoto et al. first applied and reported the self-expanding metal stents (sems) in the treatment of colorectal stenosis, they have been widely used not only as an alternative treatment as a bridge to surgery (bts), but also as a mean of palliative option for stenosing lumen in clinical practice. numerous publications have reported that the stent placement technique showed 75% to 100% technical success rates and 84% to 100% clinical success rates. however, it is sometimes difficult to place due to the distorted anatomy or acute angulations in patients and other conditions with poor endoscopic visualization with the normal colonoscope. the aims of this study were to present our results with two novel sems implantation techniques.


Eligibility criteria:
inclusion criteria: * age ≥ 18 years old * patients with colorectal cancer stenosis * patients with intestinal stent implantation in digital subtraction angiography room exclusion criteria: * patients undergoing conventional intestinal stent implantation
plaque modification and impact on microcirculatory territory after drug-coated balloon percutaneous coronary intervention (plami).

Conditions: Coronary Artery Disease; Drug Coated-balloon; Intravascular Ultrasound; Plaque Modification; Percutaneous Coronary Intervention; Microvascular Coronary Artery Disease

Summary: coronary artery disease (cad) is one of the most common causes of mortality worldwide. despite drug eluting stents (des) are the most common treatment strategy, drug-coated balloons (dcb) represent an appealing alternative to des as they eliminate the risk of stent thrombosis and do not leave any type of metallic structure in the vessel wall. however, the evidence of the vessel wall healing processes, plaque remodeling, plaque composition and impact on coronary microcirculation after pci with dcb have not yet been characterized. the purpose of this study is to assess the changes in percentage atheroma volume evaluated by intravascular ultrasound (ivus) in patients undergoing dcb-pci.

the study will be an investigator-initiated, single-arm, open-label, pilot study in patients undergoing pci with dcb for the novo lesion. because of the exploratory nature of this study, no formal sample size calculation is required. on the basis of previous pilot studies with similar designs, a sample of 30 lesions is planned to be evaluated. after being informed about the study and the potential risks, all patients meeting all the inclusion criteria and none of exclusion criteria will give written informed consent. patients will go dcb-pci. ivus will be evaluated prior to pci-dcb, immediately after and at 3-month follow-up. angiography-derived coronary physiology will be assessed after the procedure using angio plus software (pulse medical imaging technology, shanghai, china). the angiography images will be used to obtain the imrangio values, prior, post to dcb-pci and 3 month follow-up.

Eligibility criteria:
inclusion criteria: * patient with coronary artery disease undergoing percutaneous coronary intervention with dcb. exclusion criteria: * aged \< 18 years. * cardiogenic shock. * st-segment elevation myocardial infarction. * use of mechanical circulatory support. * chronic total occlusions, bifurcation lesions, left main coronary artery disease, severe calcified lesions, graft interventions and in-stent restenosis. * inability to provide informed consent. * unable to understand and follow study-related instructions or unable to comply with study protocol. * currently participating in another trial. * pregnant women.