Kornimate/medical-research-clean huggingface dataset:
safety and clinical outcomes study: svf deployment for orthopedic, neurologic, urologic, and cardio-pulmonary conditions

Conditions: Neurodegenerative Diseases; Osteoarthritis; Erectile Dysfunction; Autoimmune Diseases; Cardiomyopathies; Emphysema

Summary: to evaluate for any adverse effects that may be related to the administration and reception of autologous adipose derived stromal vascular fraction (svf). secondarily, the study monitors the results of subjective and objective findings as it applies to the non-blinded deployment of autologous svf for various inflammatory and/or degenerative conditions including select orthopedic, neurologic, urologic and cardio-pulmonary conditions. svf deployments include intra-venous, intra-articular, and soft tissue injections.

svf stromal vascular fraction is obtained by lipoharvesting, procurement, and lipo-transfer as a same day operative procedure

Eligibility criteria:
inclusion criteria * patient must be age 16 or older * patient must have a degenerative disease or inflammatory disease that meets criteria for treatment under the irb which includes: arthritis, auto-immune disease,copd, cardiomyopathy, peyronies disease,interstitial cystitis, erectile dysfunction, and neurodegenerative disease such as parkinsons, als, neuropathy. * patient must be healthy enough to tolerate a local anesthetic exclusion criteria: * patient must not have active cancer * patient must not have active infection

Keywords: erectile dysfunction peyronies interstitial cystitis cardiomyopathy emphysema copd arthritis multiple sclerosis parkinson's
retrospective chart review study of patients with pik3ca-related overgrowth spectrum who have received alpelisib

Conditions: PIK3CA-Related Overgrowth Spectrum (PROS)

Summary: the study was a site-based retrospective non-interventional medical chart review of pediatric and adult male and female patients with pik3ca-related overgrowth spectrum (pros) who initiated alpelisib at least 24 weeks before the cut-off date at a map site. the study cut-off date was 09-mar-2020. patient-level data were abstracted from medical charts of all eligible patients at all participating sites. study completion date refers to the last date data was extracted. information from patients treated with alpelisib was used to describe the efficacy and safety of alpelisib in pros patients.

the index date (baseline) is defined as the date of alpelisib initiation. the study period is the period from the index date up to the most recent data available at the time of the cut-off date. the maximum follow-up was 187 weeks.

Eligibility criteria:
inclusion criteria: * patient (adult or pediatric) is ≥ 2 years of age * patient has a physician confirmed/documented diagnosis of pros * patient has a documented evidence of a mutation in the pik3ca gene * patient's condition was assessed by the treating physician as severe or life threatening and treatment was deemed necessary * patient has been treated with at least one dose of alpelisib, initiated on or before 23-sep-2019 (i.e. at least 24 weeks before the cut-off date of the 09-mar-2020) * patient has medical chart history available during enrollment in the novartis map * patient (or parent/guardian in case of pediatric patient) consented to participate in the study (as required by local ethics regulations) inclusion criteria for map enrollment (assessed at the time of alpelisib initiation)

Keywords: alpelisib pik3ca byl719 pros retrospective chart review study epik-p1
the role of epigenetic modifications in autism spectrum disorder

Conditions: Autism Spectrum Disorder

Summary: autism spectrum disorder is a neurodevelopmental disorder characterized by impaired social communication and repetitive or stereotyped behaviors. according to the world health organization , the prevalence of autism spectrum disorder is one person in 160.

genetic and non-genetic factors would contribute to the development of autism. however, the molecular mechanisms of asd are not clear and successful treatments are still under research. autism spectrum disorder can occur due to exposure to environmental pollutants which lead to epigenetic changes like dna methylation, acetylation and post-translational modifications. however, the role of epigenetic changes in autism spectrum disorder is still debated. epigenetic mechanisms represent a link through which environmental factors interact with the genetic factors resulting in modification of autism spectrum disorder risk through changes in gene expression. dna methylation and histone deacetylation are two major epigenetic mechanisms that regulate the gene expression at successive stages of brain development. brain derived neurotrophic factor is responsible for brain development. altered bdnf levels and expression may be closely associated with autism spectrum disorder. . glial fibrillary acidic protein is the hallmark intermediate filament protein in astrocytes, the main type of glial cells in the central nervous system. interestingly, glial fibrillary acidic protein is a marker of astroglial activation and the recent data indicated that glial fibrillary acidic protein could be implicated in the pathophysiology of autism. however, the underlying mechanisms for the role of brain derived neurotrophic factor and glial fibrillary acidic protein in autism spectrum disorder are poorly understood.

Eligibility criteria:
inclusion criteria: 1. - all enrolled children with autism spectrum disorder will be: 1. exhibit symptoms within the typical triad of autistic traits: communication impairment, social deficits, and ritualistic interests. 2. drug-naïve. 2. children with autism spectrum disorder and controls will be 2-6 years old. exclusion criteria: the control subjects will also clinically examined by the psychiatrist to exclude any sub-clinical autistic features. children with autism spectrum disorder and controls will excluded from the study if 1. they receive treatment for any reason. 2. -endocrinological disease, mental retardation, communication disorder, psychotic disorder, attention deficit hyperactivity disorder and learning disorders seen in the children or their family members.
efficacy of rostafuroxin in the treatment of essential hypertension

Conditions: Essential Hypertension

Summary: the purpose of this study is to verify the efficacy of rostafuroxin in the treatment of essential hypertension and to determine the best effective dose to be administered in the general hypertensive population and in a subset of this population in which genetic patterns could be involved in the etiology of essential hypertension.

elevated arterial pressure is probably the most important public health problem. about 30% of the world adult population are affected by hypertension in industrialised countries. development of a pharmacogenomic approach to the therapy of primary hypertension give new opportunities for the treatment of hypertension. this approach consists in the identification of the genetic-molecular mechanisms responsible for hypertension in a given subset of patients, and in the development of drugs able to interfere with such mechanisms, thus leading to very selective therapeutic interventions with enhanced efficacy and reduced side effects.

Eligibility criteria:
inclusion criteria: * patients with grade 1 or 2 of essential hypertension * less than 3 risk factors (age \> 55 if male, smoking, dyslipidemia, family history of cv disease occurring before 55 years in men and 65 in women * naive patients or currently on monotherapy or one combination tablet * sbp between 140 and 169 mmhg exclusion criteria: * atrial fibrillation or left or right vbbb * left ventricular hypertrophy * significant renal or hepatic disease * obesity \> 30kg/m2 * diabetes mellitus

Keywords: hypertension pharmacogenomic adducin ouabain rostafuroxin
bioequivalence of orfadin suspension compared to orfadin capsules, and the effect of food on the bioavailability of the suspension

Conditions: Healthy Volunteers

Summary: the study is primarily being performed in order to demonstrate bioequivalence between the orfadin (nitisinone) suspension and the marketed capsule formulation. the study will also contain a comparison of the bioavailability of the suspension given with food and on an empty stomach.

this is an open, randomized 3-way crossover study in 12 healthy volunteers. subjects will receive single oral 30-mg doses of nitisinone as a suspension (fasting and with food) and as hard gelatin capsules (fasting only). there will be a 2-week washout period between the doses. there will be a screening visit within 3 weeks prior to the first dose. during each treatment period, subjects will be admitted to the clinic from the afternoon on the day before drug administration (i.e., on day -1) and remain at the clinic until the 48-hour post-dose blood sample has been collected in the morning of day 3. they will then return for ambulatory visit in the morning of day 4 (72-hour sample). a follow-up visit will take place 7-14 days after the last dose.

Eligibility criteria:
inclusion criteria: * healthy male volunteers * age: 18-55 years, inclusive * bmi: 18,5-30,0 kg/m2, inclusive exclusion criteria: * heavy smokers * consumption of more than 4 cups of coffee per day. * history of drug and/or alcohol abuse. * positive drug screen or alcohol breath test. * positive screens for hepatitis b surface antigen, hepatitis c antibodies and human immunodeficiency virus (hiv) 1-2 antibodies. * enrollment in another concurrent clinical study * intake of an investigational medicinal product within three months prior to inclusion in this study. * donation of more than 50 ml of blood within 60 days prior to drug administration * donation of more than 1,5 liters of blood in the 10 months prior to first drug administration.

Keywords: orfadin nitisinone bioequivalence bioavailability
to evaluate effectiveness and safety of containing raltegravir in naive hiv non b or b infected patients

Conditions: Evaluated; Non B Subtype; Naive Patients

Summary: pilot open label multicentric study

the population vih1 not b is not unimportant in our region of the seine st denis, a tendency which seems to be in increase (47 % in 2006, 67 % in 2007). most of the studies evaluating the clinical, immunologic and virologic response to the arv according to the viral subcategories are corresponding and show comparable results for patients infected by hiv 1 of subcategory b or non b. in spite of these reassuring results, it is necessary to evaluate the efficiency of a new arv all the more a new class. it seems also necessary to observe attentively the profiles of resistance which will be selected at the carrier patient's of virus of subcategories not - b in failure of treatment. it will allow to determine if, because of the important polymorphisms of the viruses not - b, the evolution towards the resistance will be made differently.

Eligibility criteria:
inclusion criteria: * in order to be eligible to take part in this study, patients should meet all of the following criteria: patient aged at least 18 years; treatment naive patient infected with hiv-1; patients indicated for treatment containing raltegravir patient has at least 2 activates molecules in combination therapy according genotype realized in the visit of selection. patient has not a history or current evidence of opportunist infection within the 4 weeks before the selection patient who has received oral and written (information sheet) information about the study and who has agreed for the computer processing of his/her personal data. patients with chronic hepatitis, including chronic hepatitis b and/or c may enter the study as. exclusion criteria: * patients meeting one or both of the following criteria may not take part in the study * patient is reproductive potential without requiring the use of contraception * patient is pregnant or breast-feeding * patient using alcohol and\\or drug and\\or the other substance that might interfere with the patient participation * patient infected by hiv2 * patient has severe hepatic insufficiency. (liver enzymes \> 5n) * patient has the following laboratory values during selection * platelets \< 40.000 cell / mm3 * haemoglobin \< 8 g / dl during the selection * neutrophils \< 500 / mm3 * patient has associated treatments which can have interactions with raltegravir (cf rcp isentress ®) * patient should be considered by the investigator able to conform to the imperatives of the study procedures

Keywords: raltegravir, non b subtype, naive patients
rethink chronic-a mobile therapeutic game

Conditions: REThink Game; Treatment-as-usual

Summary: the general objective of the study is to test the efficacy of an online therapeutic game (rethink game) for the prevention of emotional disorders in children and adolescents aged between 8-16 years with a chronic physical disease.

the objective of the study is to test an online therapeutic game which, being an evidence-based prevention tool for children and adolescents, might contribute to preventing emotional disorders in children and adolescents aged between 8-16 years old diagnosed with a chronic physical disease. rethink is a scientifically validated therapeutic game for children and adolescents to improve their emotional skills. the enrollment will take place both online and in pediatric medical institutions. the research study will include one parent of the eligible child/adolescent. the children and adolescents will be randomized into two equal groups: the intervention group which benefits of the entire rethink game and the treatment-as-usual group which benefits of a free trial version of the app. three assessments will be performed to evaluate the variables of interest: before intervention (baseline), after intervention (post-intervention), and a follow-up six months after the post-intervention assessment was performed.

Eligibility criteria:
inclusion criteria: * are aged 8 to 16 years old * are diagnosed with a chronic physical disease * have consent from the parent or legal tutor to participate in the study * are fluent in romanian * have access to a mobile phone, tablet or laptop exclusion criteria: * have intellectual disabilities or physical limitations which make it impossible for the them to play the game * have traumatic brain injuries * have a diagnosed mental disorder * demised during the study period

Keywords: chronic physical disease youth emotional disorders therapeutic game prevention
a study observing the long-term, effectiveness and safety of odevixibat (bylvay) in patients with alagille syndrome (algs) who are receiving ongoing treatment

Conditions: Alagille Syndrome

Summary: this study will collect information from patients with alagille syndrome (algs) as they use odevixibat (bylvay) in their daily lives. odevixibat is a medicine that helps patients with algs, a rare disease that harms their liver and causes itching. the main aim of this study is to observe the long-term, everyday effectiveness and safety of the drug odevixibat in patients with algs who are receiving ongoing treatment.


Eligibility criteria:
inclusion criteria: * diagnosed with algs. * on (or starting) active odevixibat treatment. * signed informed consent and assent, as appropriate. consent/assent from the participant or legal representative should be obtained, as appropriate, before any study data collection is conducted. participants who turn 18 years of age (or legal age per country) while participating in the study will be required to provide consent for themselves. exclusion criteria: * currently participating in a clinical trial with odevixibat. * currently participating in any interventional clinical trial for algs. * have any contraindication to odevixibat as per the locally approved label. * had liver transplant before enrolment
selenium replacement and serum selenium level in severe sepsis and septic shock patients

Conditions: Sepsis; Shock, Septic

Summary: this study will be performed to determine whether selenium replacement reduces 28-day mortality of severe sepsis and septic shock patients, and to investigate whether selenium replacement contributes differently to the mortality reduction of the patients according to their initial serum selenium level.

this study is a single center, randomized, double-blind, placebo controlled trial. after the diagnosis of severe sepsis and septic shock, enrolled patients will be randomized. then, selenium or placebo will be intravenously administered to them for 7 days. hemodynamic and laboratory data will be recorded for 7 days and additional serum samples will be obtained at 0, 24, 72, and 168 hours post-treatment and stored. mortality will be observed for 28 days. an interim analysis will be conducted by the independent data safety monitoring board. serum selenium levels will be measured from the stored serum samples after the study completion.

Eligibility criteria:
inclusion criteria: * age 18 or older * clinical diagnosis of severe sepsis or septic shock exclusion criteria: * pregnancy or breast feeding * age younger than 18 * advanced directive state to withhold treatment * known allergy to selenium * cpr or death within 24 hours after the diagnosis of severe sepsis or septic shock * advanced malignancy without further treatment plan

Keywords: sepsis shock, septic selenium survival
leuprolide in treating adults with hypogonadotropism

Conditions: Hypogonadism

Summary: rationale: hypogonadotropism is an abnormal condition caused by decreased production of gonadotropins, a group of hormones that stimulate the parts of the reproductive system that produce and release eggs from the ovaries or sperm from the testicles. leuprolide may stimulate the production of gonadotropins and be effective in increasing testosterone in men and inducing ovulation in women. purpose: clinical trial to study the effectiveness of leuprolide in treating adults who have hypogonadotropism.

protocol outline: male patients receive leuprolide subcutaneously every 5 days for up to one year and are followed at weeks 4, 6, 8, and 12, then every 2 months. female patients receive leuprolide subcutaneously on days 1, 6, and possibly 11. patients are followed 2 months after the last injection. completion date provided represents the completion date of the grant per oopd records

Eligibility criteria:
protocol entry criteria: --disease characteristics-- diagnosis of hypogonadotropism men: small testes, abnormal semen analysis, and subnormal plasma testosterone levels without an elevation in gonadotropin levels women: amenorrhea and subnormal plasma estradiol levels, or lack of cornification of the vaginal mucosa without an elevation in gonadotropin levels or hypothalamic amenorrhea in normally estrogenized women with amenorrhea or oligomenorrhea and normal plasma free testosterone and gonadotropin levels patients with hyperprolactinemia eligible only if hypogonadotropism persists after correction of hyperprolactinemia by dopamine agonist therapy --prior/concurrent therapy-- at least 2 months since sex hormone treatment --patient characteristics-- other: * not pregnant * no chronic systemic, metabolic, or endocrine disease

Keywords: endocrine disorders hypogonadism rare disease
study of safety, tolerability and efficacy of mk0493 in obese patients (0493-008)(completed)

Conditions: Obesity

Summary: to assess the effect of mk0493 on body weight, blood pressure, mood, and appetite.


Eligibility criteria:
inclusion criteria: * patient is between 21 and 65 years * patient is able to read and understand and complete study questionnaires * patient is not pregnant or breast-feeding and does not plan to become pregnant for the duration of the study and post-study follow-up period exclusion criteria: * patient has a history of significant psychiatric disorder (such as major depression, bipolar disorder, bulimia, or anorexia nervosa * patient is hiv positive as determined by medical history * patient has undergone surgical treatment for obesity * patient plans to consume more than two 8 ounce glasses of grapefruit juice per day during the study * patient participated in another clinical study involving an investigational drug) within 3 months prior to visit 1 * patient is currently a heavy consumer of alcohol (\>2 drinks per day or \>14 drinks per week \[1 drink is defined as 2 oz hard alcohol, 5 oz wine, 12 oz beer\], or uses (including recreational use) any illicit drugs, or has a history of drug or alcohol abuse
a study to learn about the safety and effects of rimegepant to prevent migraine in chinese subjects.

Conditions: Migraine

Summary: the purpose of this study is to learn about the effects of rimegepant to help prevent migraine. this study is seeking for participants who: * are male and female of 18 years of age or older. * have at least 1 year history of migraine . * did not take any medication for migraine before the start of this study. the study will go on for around 30 weeks, including 4 phases and 11 visits. participants who are selected for the study will be randomly assigned to treatment groups. after which, the participants will enter a 12-week double-blind treatment (dbt) phase. after finishing the dbt phase, some selected participants may enter a 12-week open-label extension (ole) phase. participants will come back to the study site at the end of week 24 for the end of treatment (eot) visit. there will be a follow-up week 2 visit around 14 days after the eot visit. participants will be asked to take 1 tablet of study medicine every other calendar day. this need to be followed regardless of whether they have a migraine on that day or not. during the ole phase only, if a participant has a migraine on a non-scheduled dosing day, they may take 1 tablet of rimegepant orally disintegrating tablet (odt) as acute treatment for their migraine, if needed, with a maximum of 1 tablet of rimegepant per calendar day. the study team will look at how each participant is doing with the study treatment during the regular visits at the study clinic.


Eligibility criteria:
inclusion criteria: 1.target population: participant has at least 1 year history of migraine (with or without aura) consistent with a diagnosis according to the international classification of headache disorders, 3rd edition, including the following: 1. age of onset of migraines prior to 50 years of age 2. migraine attacks, on average, lasting 4 to 72 hours if untreated 3. per participant report, 4 to18 migraine attacks of moderate or severe intensity per month within the last 3 months prior to the screening visit (month is defined as 4 weeks for the purpose of this protocol) 4. 6 or more migraine days during observation phase 5. not more than 18 headache days during the observation phase 6. ability to distinguish migraine attacks from tension/cluster headaches. 7. participants with contraindications for use of triptans may be included provided they meet all other study entry criteria. exclusion criteria: 1. participant has a history of basilar migraine or hemiplegic migraine. 2. participants are excluded if they have had no therapeutic response with \> 2 of the 9 medication categories of preventive treatment of migraine after an adequate therapeutic trial in the past 3 years per investigator's judgement.

Keywords: rimegepent preventive treatment migraine.