Kornimate/medical-research-clean huggingface dataset:
a study with ro4917523 in patients with fragile x syndrome

Conditions: Fragile X Syndrome

Summary: this randomized, double-blind multiple ascending dose study will evaluate the safety and tolerability, pharmacokinetics and efficacy of ro4917523 in patients with fragile x syndrome. the patients will be randomized to receive either active drug or placebo. the anticipated time on study treatment is 6 weeks. the target sample size is \<100 patients.


Eligibility criteria:
inclusion criteria: * adult patients, 18 to 50 years of age * fragile x syndrome * iq less than 75 * reliable caregiver exclusion criteria: * current psychosis or presumption of psychosis * history of suicidal behavior or considered a high suicidal risk * severe self-injurious behavior * any unstable medical condition other than fragile x syndrome (e.g. hypertension, diabetes) * current seizure disorder
outcomes of secondary endovascular aortic repair after initial frozen elephant trunk procedure

Conditions: Thoraco-abdominal Aneurysm; Aortic Dissection

Summary: objective complex aortic pathology has been revolutionized with the use of hybrid prostheses such as the thoraflex® hybrid frozen elephant trunk (fet). the aim of this study was to evaluate the midterm results of secondary extension of the fet by thoracic endovascular aortic repair (tevar). few data are present in the literature regarding the outcomes of this secondary treatment. the investigators perform a prospective study between 2015 and 2022 in a tertiary aortic center on all consecutive patients having undergone tevar after fet implantation. the tevar endograft covered most of the 10 cm fet module with 2 to 4 mm oversizing. all patients were monitored by computerized angiography (cta) at 6-month and yearly thereafter. the aim of this study was the feasibility of theses secondary connexion and the mid term outcomes of the endovascular treatment.


Eligibility criteria:
inclusion criteria: patient with aortic cross replacement by fet and * type 1 and type 2 thoracoabdominal aneurysm (taaa) with a diameter at \> 6 cm or * an aortic dissection, type a (taad) or type b (tbad) with either organ malperfusion due to compression of the true channel or aneurysmal evolution of the dissected aorta, or * a defect in the expansion of the fet module exclusion criteria: * no aortic cross replacement by fet
motivational interviewing for medication adherence in asthma

Conditions: Asthma

Summary: the high burden of asthma appears to be related to poor asthma control, which is associated with more frequent asthma symptoms, greater bronchodilator use and functional impairment, and worse pulmonary function. despite the availability of effective treatments, more than 58% of asthmatics are poorly controlled. daily adherence to inhaled corticosteroid (ics) regimens is considered by experts to be one of the most important behavioral factors linked to achieving optimal asthma control. however, there is a paucity of research on interventions specifically designed to improve ics adherence among adult asthmatics. the vast majority of intervention studies to date used atheoretical interventions to target behavior change, relying mainly upon educational approaches which have been criticised for "failing to translate knowledge into action." this may be due to the fact that most education-based approaches do not specifically address or help patients overcome ambivalence about behavior change, which is necessary for ensuring daily adherence. motivational interviewing (mi) is a client-centred intervention that focuses on enhancing intrinsic motivation to change a particular behavior, and exploring and resolving ambivalence about behavior change. brief mi sessions (e.g., 1-5 x 15-30 minute sessions) have been shown to improve a variety of health behaviors (e.g., reduce alcohol consumption, improve dietary habits, increase exercise behaviour, and improve medication adherence) and health outcomes (reduce blood pressure, body mass index, and cholesterol levels). however, no studies to date have assessed the efficacy of using mi to improve ics adherence in asthmatics. this study aims to assess the efficacy of using mi to improve daily medication (ics) adherence in a sample of poorly controlled, non-adherent asthmatics. it is hypothesized that patients randomized to the mi condition will exhibit significantly improved ics adherence at 6 and 12-months post-intervention, independent of baseline levels and covariates, relative to patients randomized to the usual care control condition.


Eligibility criteria:
inclusion criteria: * patients 18 years and older * primary diagnosis of moderate-severe persistent asthma (as per gina) * prescribed inhaled corticosteroid medication (min dose of 250 µg fluticasone equivalent per day) for at least 12 consecutive months * uncontrolled asthma (≤ 19 on the asthma control test) * covered by a drug insurance plan (e.g., ramq) * non-adherent to ics medication (based on having filled less than 30% of their prescriptions over the last year) * able to speak english or french. exclusion criteria: * any other medical condition that confers greater illness morbidity than asthma (e.g., active cancer) * severe psychopathology (e.g., schizophrenia) * apparent cognitive or language deficit * are or plan to become pregnant or move outside of quebec over the course of the study.

Keywords: adherence asthma control asthma quality of life motivational interviewing
quercetin chemoprevention for squamous cell carcinoma in patients with fanconi anemia

Conditions: Fanconi Anemia; Squamous Cell Carcinoma

Summary: fanconi anemia (fa) is an autosomal recessive disease characterized by progressive bone marrow failure, variable congenital abnormalities and a predisposition to malignancy, particularly acute myeloid leukemia (aml) and squamous cell carcinoma (scc). improved transplant outcomes are modifying the natural history of fanconi anemia. improved transplant survival, no radiation exposure, and almost no gvhd increases the importance of addressing later scc even further. the investigators hypothesize that quercetin will prevent or delay the development of scc and associated complications, there by ameliorating or delaying the need for potentially lethal treatment with chemotherapy and/or radiation therapy for the same. funding source - fda office of orphan products development (oopd)

fanconi anemia (fa) is an autosomal recessive disease characterized by progressive bone marrow failure, variable congenital abnormalities and a predisposition to malignancy, particularly acute myeloid leukemia (aml) and squamous cell carcinoma (scc). currently, the only curative treatment option for the hematological complications of fa include hematopoietic cell transplantation (hct). the investigators hypothesize that quercetin will prevent or delay the development of scc and associated complications, there by ameliorating or delaying the need for potentially lethal treatment with chemotherapy and/or radiation therapy for the same. this study is an open-label, single arm study. this study will enroll approximately 45 post-hct patients with fa, and approximately 10 patients with fa without history of hct. in both groups, patients with or without existing pre-malignant lesions or history of scc will be allowed to participate, if they wish so and at the discretion of the pi. all patients will be treated with oral quercetin. the investigators will determine the efficacy of quercetin in reducing buccal micronuclei (a surrogate marker of dna damage and susceptibility to squamous cell carcinoma due to genomic instability) in post-hct patients with fanconi anemia (fa).

Eligibility criteria:
inclusion criteria: * diagnosis of fa * able to take enteral medication * patients ≥2 years exclusion criteria: * renal failure requiring dialysis * total bilirubin \>3 mg/dl and/or sgpt \>200 at time of enrollment * patients receiving digoxin therapy, who are unable to discontinue either treatment due to medical reasons * patients who are pregnant or breastfeeding or are at risk of pregnancy or fathering a baby and are unable to use acceptable methods of birth control during the length of the study * patients who have received quercetin supplementation or other antioxidants within the last 30 days * patients receiving radiation therapy, chemotherapy or immunotherapy for treatment of scc.

Keywords: hematopoietic cell transplantation
effects of home-based resistance exercise on body composition, muscle strength and glycemic control in people with type 2 diabetes.

Conditions: Diabetes

Summary: this study investigates the effects of home-based resistance exercise training, compared to a control group, on body composition, muscle strength and glycaemic control in people with type 2 diabetes. half participants will be in the home-based exercise group and half in the control group.


Eligibility criteria:
inclusion criteria: * physician diagnosed type 2 diabetes * no changes in anti-diabetic medication in the last 3 months exclusion criteria: * not currently or in the past year participating in any vigorous aerobic activity (\>1h per week) or any resistance exercise. * bmi of 45 or higher * blood pressure of 160/100mmhg or higher * currently receiving insulin therapy
effect of sarcopenia on hcc after lenvatinib and anti-pd-1 treatment

Conditions: Liver Cancer; Sarcopenia

Summary: sarcopenia is associated with the prognosis of hcc and cholangiocarcinoma. but there has been rare study focusing on the effect of sarcopenia on the prognosis of hcc treated with lenvatinib and anti-pd1.

by tracking the short-term and long-term results of hcc patients treated with lenvatinib and anti-pd1,the difference of short-term results between patients with sarcopenia and patients without sarcopenia was analyzed, and the correlation between sarcopenia and short-term and long-term results of patients after systemic treatment was explored, so as to improve people's awareness of sarcopenia and pay attention to its prevention and treatment.all implementation details are based on the newest ewgsop definition. the investigators consecutively admitted patients the questionnaire and evaluated the assessment of muscle strength (grip strength test and chair stand test), muscle quantity (l3 plane total skeletal muscle area) and physical performance (gait test) following the f-a-c-s approach, to confirm patients with sarcopenia accurately. and by tracking the short-term and long-term outcomes, the differences were analyzed and the relationship between sarcopenia and prognosis of systemic treatment was explored.

Eligibility criteria:
inclusion criteria: patients with clinical diagnosis of liver cancer * no cancer other than liver cancer has been diagnosed * age ≥18 years exclusion criteria: * patients who could not complete the standard tests and questionnaires * patients received other therapies * patients who had other causes of muscle weakness (injury, fracture, stroke, etc.) * patients who missing ct data or ct scan did not reach the level of the third lumbar vertebra (l3)

Keywords: liver cancer sarcopenia lenvatinib immune therapy prognosis
the characteristics, treatment patterns and prognosis of chinese male breast cancer: a real-world study

Conditions: Breast Cancer

Summary: the goal of this observational study is to investigate the features in chinese male breast cancer (mbc) patients. the main question it aims to answer is: * the characteristics, treatment patterns and prognosis of chinese mbc patients. the clinicopathological characteristics, treatment information and follow-up data of participants will be extracted from a chinese national database (chinese society of clinical oncology breast cancer database) and other involved chinese centers. researchers will investigate the features, survival associated factors and other relevant factors of this population.

the goal of this observational study is to investigate the features in chinese male breast cancer (mbc) patients. the main question it aims to answer is: * the characteristics, treatment patterns and prognosis of chinese mbc patients. the clinicopathological characteristics, treatment information and follow-up data of participants will be extracted from a chinese national database (chinese society of clinical oncology breast cancer database) and other involved chinese centers. researchers will investigate the features, survival associated factors and other relevant factors of this population. the collected variables included: survival time, survival status, year of diagnosis, age, stage, molecular subtype statuses, histology, grade, and surgery, radiotherapy and systemic therapies utilization.

Eligibility criteria:
inclusion criteria: * biological sex is male; * microscopically diagnosed as primary breast cancer. exclusion criteria: * presence of skin-origin malignancy on the breast.
sulphate-bicarbonate-calcium water, body weight and gut microbiota

Conditions: Overweight; Obesity

Summary: overweight and obese patients will be recruited and randomly assigned to two groups of intervention. to the first group \[sulphate-bicarbonate-calcium water and low-calorie diet (sw-d)\] will be administered "acqua santa di chianciano"®, associated to a personalized low calorie diet, while the second group \[tap water and low-calorie diet (tw-d)\] will follow the personalized low calorie diet but will be asked to drink the same quantity of tap water, over a 4 week period. stool samples will be collected and analyzed for changes in gut microbiota composition. patients' body weight will be recorded at the beginning and at the end of the study.

overweight (bmi\>25) and obesity in adults is a global public health concern because weight excess increases the relative risk of disease and mortality 1-4. a range of diseases, notably cardiovascular disease, diabetes and a number of cancers, are related to excess weight 5. traditional low-calorie diets are frequently ineffective 6. although a number of pharmacological approaches for treatment of obesity have been investigated, only few are safe and most of them have adverse effects 7,8. thus, further studies are necessary in order to find natural antiobesity remedies. gut microbiota composition is thought to influence body weight 9. as recently demonstrated in our previous study 10, "acqua santa di chianciano"® helps to maintain the body weight and the values of serum lipids stable in subjects under a relatively high-calorie diet. possible mechanisms may be a) changing the gut microbiota composition and/or b) increasing the concentration and/or the qualitative pattern of serum bile acids with a subsequent increase of the energy expenditure 11. this study wants to assess the effectiveness in losing weight of "acqua santa di chianciano"® in addition to a low-calorie diet and its effects on gut microbiota composition.

Eligibility criteria:
inclusion criteria: * women with bmi between 29 and 35 kg/m2 * age between 18 and 65 years exclusion criteria: * therapy with antibiotics, bile salts, cholestyramine, laxatives, pre- or probiotics during the last 3 months before enrollment * helycobacter pylori positivity * previous cholecystectomy * gallbladder disease * cholestasis * consumption of more than 20 g of alcohol/day * inflammatory bowel diseases * previous gastrointestinal surgery modifying the anatomy * pregnancy or lactating state * prescribed hypocaloric diet in the three previous months

Keywords: weight loss sulphate-bicarbonate-calcium water gut microbiota body composition bile salts
evaluation of new biomarkers predictive of efficacy betablockers in pecoma and vascular pediatric tumors

Conditions: PEComa; Hemangioma

Summary: pecoma is rare tumor affecting particularly patients with tuberous sclerosis. biological similarities were seen between pecoma and infantile hemangioma. propranolol is highly efficient to treat infantile hemangioma and we believe that this drug can also be useful for the treatment of pecoma. purpose : to understand the mechanism of action of propranolol in pecomas and related pediatric vascular lesions and to select possible novel targets of betablockers in vascular tumors related to pecomas by using yap oncogene activation as a molecular marker


Eligibility criteria:
inclusion criteria: * patients who will have biopsy or surgery for pecoma or vascular pediatric tumor (rich, nich, hemangioma or pyogenic granuloma) * a free, informed and written consent will be established exclusion criteria: * none

Keywords: propranolol betablocker angiogenesis
virtual reality vs standard-of-care for comfort during intravenous catheterization

Conditions: Intravenous Catheterization

Summary: children often need an intravenous catheter placement for delivery of fluids and medications, a procedure associated with pain and anxiety. in the emergency department topical anesthetics are frequently used. virtual reality (vr) is an immersive experience using sight, sound, and position sense. using vr may enhance distraction during the painful procedure and may reduce attention to pain. this study will randomize children (6 - 16 years old) to receive virtual reality or standard of care in addition to topical anaesthetics during iv placement procedure. investigators will measure pain, anxiety and satisfaction, amount of analgesics used and the level of success in placing the iv and compare between the two groups.


Eligibility criteria:
inclusion criteria: * children age 6 - \<17 * the managing physician determines a need for an intravenous catheterization (iv) procedure * parents will sign a consent form and children will sign an assent form exclusion criteria: * children with conditions that may prohibit participation or evaluation of the procedure (such as developmental delay, autism, others) * triage category 1 (resuscitation) * facial features or injury prohibiting wearing the vr goggles

Keywords: virtual reality pain anxiety
comparison of four different treatment regimens in treating women with stage i breast cancer

Conditions: Breast Cancer

Summary: rationale: radiation therapy uses high-energy x-rays to damage tumor cells. estrogen can stimulate the growth of breast cancer cells. hormone therapy using tamoxifen may fight breast cancer by blocking the uptake of estrogen by the tumor cells. combining radiation therapy and tamoxifen with surgery may kill more tumor cells. it is not yet known which treatment regimen is most effective for stage i breast cancer. purpose: randomized phase iii trial to compare the effectiveness of four different treatment regimens in treating women who have stage i breast cancer.

objectives: i. compare the effectiveness of wide local excision alone versus wide local excision followed by radiotherapy with or without tamoxifen versus wide local excision plus tamoxifen in women with stage i breast cancer. ii. assess local occurrence in the treated breast, regional recurrence, distant recurrence, death from breast cancer, and occurrence of cancer in the opposite breast in these patients when treated with one of these regimens. outline: this is a randomized study. patients are randomized to one of four treatment arms. all patients undergo wide local excision of the tumor and axillary node dissection. arm i: patients undergo surgery only. arm ii: patients undergo surgery followed by 25-35 radiotherapy treatments. arm iii: patients undergo surgery plus oral tamoxifen daily for 5 years. arm iv: patients undergo surgery followed by 25-35 radiotherapy treatments and oral tamoxifen daily for five years. patients are followed every 3 months for 2 years, and then every 6 months thereafter. projected accrual: a total of 1200 patients (300 per treatment arm) will be accrued for this study.

Eligibility criteria:
disease characteristics: histologically confirmed unilateral stage i invasive breast cancer no more than 1 tumor and no greater than 2 cm grade 1 tumor of any histological type or no special type or mixed type allowed no lymph node involvement no in situ carcinoma only or ductal carcinoma in situ with microinvasion no vascular invasion no paget's disease of the nipple well differentiated special type of primary mammary carcinoma including: tubular tubular/cribriform cribriform papillary mucoid no lobular, medullary, or other rarer types of mammary carcinomas including: adenoid cystic carcinoid secretory spindle cell apocrine no distant metastases hormone receptor status: not specified patient characteristics: age: under 70 sex: female menopausal status: not specified performance status: not specified life expectancy: not specified hematopoietic: not specified hepatic: not specified renal: not specified other: not pregnant or nursing no other systemic disease that may preclude study no other prior malignancy except adequately treated basal cell carcinoma of the skin or carcinoma in situ of the cervix prior concurrent therapy: no other concurrent adjuvant systemic therapy

Keywords: stage i breast cancer
cooled rf lesion mri characteristics

Conditions: Arthritis;Lumbosacral; Osteo Arthritis Knee; Sacroiliitis; Hip Osteoarthritis; Arthritis, Degenerative

Summary: this prospective, single-center, pilot study will assist in gaining an understanding of the actual crfa lesions in an in vivo situation in areas where crfa is utilized as a standard of care treatment option for the relief of chronic pain (cervical facet joints, thoracic facet joints, lumbar facet joints, sacroiliac (si) region, hip and knee).

this study will be a prospective, single-center, pilot study. adult subjects over the age 21 diagnosed with chronic joint pain (≥ 3 months), scheduled to receive radiofrequency denervation and meet the selection criteria are eligible to participate in this study. the specific targeted areas of interest in this study will include cervical facet joints, thoracic facet joints, lumbar facet joints, sacroiliac (si) region, hip and knee. the treating physician will follow standard of care treatment for all enrolled subjects. as illustrated in figure 1, the study consists of a screening visit, a treatment visit and a follow up visit. all subjects receiving crfa will receive an mri 2 - 7 days after the crfa procedure. subjects receiving crfa of the sacroiliac joint will also receive an mri within 30 days prior to the crfa procedure. the treating physician may also request an mri be performed prior to the procedure for subjects receiving crfa of other targeted areas. the point of enrollment for each subject is the time that they sign the informed consent form. mri data will be reviewed by the radiologist on a per patient basis to confirm lesion characteristics. it is anticipated that 3-5 subjects per level will be needed to fully quantify lesion characteristics, however; enrollment will remain flexible for each targeted area based on real time review of mri data.

Eligibility criteria:
inclusion criteria: 1. age ≥ 21 years 2. able to understand the informed consent form and provide written informed consent and able to complete outcome measures. 3. must be clinically appropriate candidate to receive crfa for treatment of chronic pain. 4. willing and able to receive an mri exclusion criteria: 1. unable to receive an mri (i.e. due to pacemaker or iron-based metal implant). 2. unable to receive an mri (i.e. iron-based metal implant). 3. extremely thin patients and those with minimal subcutaneous tissue thickness that would not accommodate a radiofrequency lesion of up to 14mm in diameter to limit the risk of skin burns. 4. active joint infection or systemic or localized infection at the needle entry site (subject may be considered for inclusion once infection is resolved.) 5. subject currently implanted with a pacemaker or defibrillator 6. in the event a pre-treatment mri is obtained; trauma or injury occurring to the targeted area between the baseline mri and crfa treatment 7. subject unwillingness or unable to comply with protocol requirements

Keywords: cooled radiofrequency ablation mri