Kornimate/medical-research-clean huggingface dataset:
a multiple ascending dose study of 9mw3011 in patients with non-transfusion-dependent β-thalassemia

Conditions: Beta-Thalassemia

Summary: this is a phase ib, randomized, double-blind, placebo-controlled, multiple ascending dose study . the objectives of the study are to evaluate the safety , tolerability, pharmacokinetics(pk), pharmacodynamics(pd), and immunogenicity of 9mw3011 in patients with non-transfusion-dependent β- thalassemia .

a total of 40 subjects diagnosed with non-transfusion-dependent β-thalassemia will be enrolled in this study and assigned into four dosage cohorts. in each cohort, subjects will be randomized in a 4:1 ratio to receive 9mw3011 or placebo via intravenous infusion.

Eligibility criteria:
key inclusion criteria: 1. male and female subjects aged 18 to 65 years (inclusive) 2. subject must have a documented genetic diagnosis of β-thalassemia or hemoglobin e/ β-thalassemia 3. subjects must meet the criteria for non-transfusion-dependent thalassemia 4. subjects must have a baseline hemoglobin level between 70-100 g/l(inclusive), based on 2 consecutive measurements taken at least 1 week apart within 4 weeks before randomization 5. subjects must have evidence of iron overload during screening 6. subject must have performance status: eastern cooperative oncology group (ecog) performance score of 0 to 1 7. subjects must fully understand the study procedures and methods, voluntarily participate in the trial, and sign an informed consent form key exclusion criteria: 1. subjects diagnosed with alpha-thalassemia 2. subjects diagnosed with hbs/beta-thalassemia or transfusion-dependent beta-thalassemia 3. subjects exhibit severe iron overload at the time of screening 4. in addition to thalassemia, subjects have any other forms of anemia and hematological disorders that the investigator assesses may compromise safety or influence study outcomes 5. combined with any significant systemic diseases or psychiatric disorders 6. subjects have new york heart association (nyha) class iii-iv heart failure and other cardiovascular diseases within 6 months prior to screening or currently present 7. during the screening or baseline period, subjects exhibiting a qtcf interval of ≥450ms for males and ≥470ms for females on a 12-lead electrocardiogram (ecg), or presenting an abnormal 12-lead ecg with clinical significance 8. uncontrolled hypertension before screening 9. a history of malignant neoplasm occurring within the last five years 10. severe infection requiring hospitalization or intravenous antimicrobial therapy, or uncontrolled systemic bacterial, fungal, or viral active infection 11. subject have received concomitant treatment that was not permitted by the protocol 12. subjects whose hematological parameters did not meet the inclusion criteria during screening 13. subjects with a history of substance abuse, as well as those who yield positive results on substance abuse screening 14. subjects who are unable to undergo mri scans 15. pregnant or lactating women 16. subjects presenting any other factors deemed unsuitable for participation assessed by the investigator
the th9/il-9 and early enteral nutrition in sepsis

Conditions: Sepsis; Intestinal Mucosal Barrier; Th9 Cells; Enteral Nutrition

Summary: the investigators aim to evaluate the roles of th9/il-9 in the mechanisms of early enteral nutrition (een) maintaining intestinal mucosal barrier in sepsis. the results of this study would lay the foundation for revealing the mechanisms of een improving immune imbalance of sepsis and provide a new idea to the early treatment of sepsis. in addition, the investigators also aim to evaluate the effects of different proportions of target total enteral nutrition on the prognosis of sepsis.


Eligibility criteria:
inclusion criteria: 1. clinical diagnosis of sepsis 2. within 3 days of sepsis onset before icu admission 3. no artificial nutrition (enteral or parenteral nutrition) were provided before icu admission exclusion criteria: 1. ileus 2. digestive tract hemorrhage 3. inflammatory bowel disease 4. abdominal hypertension (iap \>25mmhg) 5. cancer or chronic organ dysfunction (e.g., hepatic or renal dysfunction) 6. malnutrition or immunodeficiency 7. long-term use of hormones
the efficiency of technology-based motivation education on obesity university students' weight management.

Conditions: Overweight and Obesity

Summary: the aim of this study is to determine the effects of technology-based motivation education prepared according to the health belief model on obesity-related beliefs and attitudes, nutrition-exercise behaviors, self-efficacy and weight management in obese university students.

it is stated that the university period is risky in terms of acquiring negative health behaviors that may cause overweight and obesity. in addition, it is emphasized that the transition from overweight to obesity usually occurs between the ages of 18-29. there are non-formal education programs on obesity management for adolescence and adulthood. it is noteworthy that young people in the university period are not adequately represented in the current education programs, and at the same time, education programs specific to young people are limited. the aim of this study is to determine the effects of technology-based motivation education prepared according to the health belief model on obesity-related beliefs and attitudes, nutrition-exercise behaviors, self-efficacy and weight management in obese university students. this quasi-experimental pretest-posttest randomized controlled study will be carried out at sivas cumhuriyet university suşehri health college and suşehri timur karabal vocational school between february 1, 2022 and september 15, 2022. the sample of the study will consist of 84 students (experimental group n= 42 and control group n=42). a web-based technology based motivation program including motivational interviewing will be applied to the students in the experimental group. the control group will only receive web-based education on obesity. data will be collected using personal information form, obesity health belief model scale, nutrition-exercise behavior scale, self-efficacy-efficacy scale and website usability scale. physical measurements; a digital height-weight meter will be used for bmi measurement, and a tape measure will be used for waist and hip circumference. whether the data is normal or not will be determined by kolmogorov-smirnov, shapiro wilk tests. in order to determine whether there is a difference in the research, necessary parametric and nonparametric tests will be used depending on whether the assumptions are met or any or all of the assumptions are not met.

Eligibility criteria:
inclusion criteria: * 18-25 age range * body mass index (bmi) ≥ 25.0, * having internet access with a computer, tablet or mobile device. exclusion criteria: * under 18 years old or over 25 years old, * bmi \< 25.0, * continuously using regular medication, * presence of a pre-existing health problem (having a surgical operation in the last three months, having diseases with metabolic syndrome, being diagnosed with cancer, being diagnosed with an eating disorder, being diagnosed with a psychological illness, and being physically disabled), * participating in any weight management program in the last three months, * pregnancy.

Keywords: obesity motivational interviewing health belief model nursing
methylphenidate in adhd with trichotillomania

Conditions: ADHD; Trichotillomania

Summary: this study will evaluate the safety and effectiveness of methylphenidate in treating attention deficit hyperactivity disorder (adhd) in children with both adhd and trichotillomania. trichotillomania is an impulse control disorder. there is growing evidences of the involvement of dopaminergic neurotransmission in the pathophysiology of trichotillomania. reported increase in the prevalence of adhd among patients with impulse control disorders, such as pathological gambling as well as trichotillomania, may result from the overlapping pathophisiological background. it is hypothesized that in cases of adhd comorbid with trichotillomania methtylphenidate treatment will exhibit beneficial effects in both the adhd and the hair pulling.

thirty children and adolescents aged 6-18 years, diagnosed with adhd and trichotillomania, will receive mph monotherapy treatment for a period of 12 weeks, targeting both adhd and trichotillomania symptoms as rated by the adhd- rating scale (adhd-rs) and by the massachusetts general hospital hair pulling scale and clinical global impression-severity (cgi) scale. the rating scales will be assessed at baseline and at the endpoint (after 12 weeks).the side effects will be monitored via weekly spontaneous self reports by each participant. all results will be expressed as mean ±sd. student's paired t-test and anova test will be used as appropriate.

Eligibility criteria:
inclusion criteria: * dsm-iv diagnosis of trichotillomania * dsm-iv diagnosis of adhd * has not taken stimulants or alpha-adrenergic medications for more than 2 weeks prior to entering the study. exclusion criteria: * history of moderate or severe adverse event, related to mph * history of any psychotic disorder * current drug abuse, acute psychotic or affective disorder

Keywords: methylphenidate trichotillomania attention deficit
efficacy of everolimus-containing chemotherapy in her2- mbc patients with pi3k/akt/mtor mutations study

Conditions: Breast Cancer; PI3K/AKT/mTOR Pathway Mutation; Everolimus

Summary: the study aimed to investigate the efficacy of the everolimus-containing chemotherapy in metastatic breast cancer patients with pi3k/akt/mtor mutations.14 cases of her2- metastatic breast cancer patients with pi3k/akt/mtor mutations treated with everolimus-containing chemotherapy were retrospectively analyzed.the genetic profile in pi3k/akt/mtor pathway was studied.

the pi3k/akt/mtor pathway is frequently altered in breast cancer. everolimus is a selective inhibitor of mammalian target of rapamycin (mtor).the benefit of everolimus in metastatic breast cancer patients with pi3k/akt/mtor mutations remains unclear, especially in tnbc patients. the study aimed to investigate the efficacy of the everolimus-containing chemotherapy in metastatic breast cancer patients with pi3k/akt/mtor mutations. 14 cases of her2- metastatic breast cancer participants with pi3k/akt/mtor mutations treated with everolimus-containing chemotherapy were retrospectively analyzed.the genetic profile in pi3k/akt/mtor pathway was studied.

Eligibility criteria:
inclusion criteria: 1. patients with her2- metastatic breast cancer 2. patients with pi3k/akt/mtor mutation exclusion criteria: 1. patients treated with endocrinotherapy and everolimus. 2. the efficacy evaluation could not be available.
quadrant versus superficial parotidectomy

Conditions: Pleomorphic Adenoma of the Parotid Gland

Summary: the most common benign tumor of the parotid gland is the so called pleomorphic adenoma. although benign, this tumor may recur after surgical removal due to tumor cells left behind during the surgical operation. thus, pleomorphic adenomas have been treated with wide resection similar to malignant tumors. this extensive surgery often leads to injury to the motor nerves responsible for facial expression and eye protection. the investigators propose less extensive surgery which should be thorough enough to prevent tumor recurrence while keeping the facial nerve out of risk.

pleomorphic adenoma is notorious of its propensity to local recurrence due to the microscopic extracapsular extension of the tumor. superficial parotidectomy (sp) is the standard treatment. extracapsular dissection (ed) has been proposed to minimize the risk of facial nerve palsy. however, the oncologic safety of ed is controversial in the literature. we propose quadrant parotidectomy (qp) which is less radical than sp and less conservative than ed. qp entails dissection overlying one primary division of the facial nerve leaving the other division undisturbed while the resection safety margin is still generous. surgery for pleomorphic adenoma is a compromise between radicality and facial nerve morbidity. risk of local recurrence is imprecisely described in the literature and is universally regarded as infrequent and remote. many studies did not report recurrent cases. thus, facial nerve injury is the main concern of parotid surgeons and is considered the primary end point of this study.

Eligibility criteria:
inclusion criteria: * adult patients with pleomorphic adenoma of the parotid gland with cytology- proven diagnosis exclusion criteria: * collagen diseases * previous parotid surgery * previous facial nerve palsy * neuromuscular diseases affecting the face * patient refusal
autologous bone marrow stem cells transplantation in patients with temporal lobe epilepsy

Conditions: Epilepsy

Summary: this research investigates the use of autologous bone marrow stem cells for the treatment of medically refractory temporal lobe epilepsy patients.

the aim of this study is to evaluate autologous bone marrow stem cells transplantation as a safe and potentially beneficial treatment for patients with temporal lobe refractory epilepsy. we are mainly evaluating seizure frequency, hippocampal volume an cognitive performance

Eligibility criteria:
inclusion criteria: * temporal lobe medically refractory epilepsy patients with hippocampal atrophy exclusion criteria: * under 18 or over 65 years old patients * previous neurological surgery * other neurodegenerative diseases

Keywords: seizure frequency hippocampal volume adverse effects
metabolic and structural characterization of hub's vulnerability in neurological diseases assessed by ultra high field structural and functional mri

Conditions: Diffuse Diseases

Summary: the investigators hypothesize that hub alteration occurs both in diffuse diseases (ms, ad) as well as in more 'network specific' diseases (parkinson, als, epilepsy). this could impact on functional dysfunction not directly related to each disease, but that could induce common syndrome such as cognitive impairment observed in parkinson, partial epilepsy or als. the objective here is to test this hypothesis and provides better understandings on pathophysiological processes affecting those highly connected regions in 'diffuse' and 'focal' neurological diseases. the ultimate goal is to identify new clinical targets for trans-nosological approaches (dbs, cognitive rehabilitation ...). practically, the investigators will explore 200 patients classified in 5 cohorts of 40 patients suffering for ms, ad, parkinson, als, epilepsy, using the last advanced methods to assess structural and functional brain connectivity implemented on the human 7t mr scanner equipping the cemerem (chu timone, marseille, only 50 similar mr scanners worldwide). in addition to high resolution diffusion mri and rs-fmri, metabolic and ionic (sodium) mapping will complement the mr protocol to characterize the pathophysiological processes of hub injury. sixty healthy controls will also be explored wih the same protocol for normal database. the proposal aims at characterizing and comparing from a morphological-functional point of view, the hub regions of patients suffering from these five diseases, to demonstrate the pertinence to preserve hub integrity as a major therapeutic target whatever the disease.


Eligibility criteria:
inclusion criteria: * person female or male, more than 18-year-old, * person presenting unchecked general disease such as severe cancer, autoimmune disease, hepatic insufficiency, severe or untreated, shady arterial high blood pressure of the conduction or the disorder of the rhythm * person presenting chronic psychiatric disease, insane syndrome. * person presenting contraindication to the realization of an examination by mri (metallic claustrophobia, foreign body, pacemakers), * person benefiting from a social security cover, * person having read, understood and signed an informed consent after information exclusion criteria: * claustrophobia, * metallic foreign bodies, * pacemakers, * severe renal insufficiency
jointly managing jia online: an internet-based psycho-educational game for children with jia and their parents

Conditions: Juvenile Idiopathic Arthritis

Summary: arthritis in children is a long-term illness and it can make a child's life very difficult. in children 8-11 years old, the child and their family work together to deal with the problems that arthritis can cause. learning to cope with and manage the problems that come with arthritis can stop it from getting worse. it is important to create programs that teach children and families how to cope with and manage arthritis. this study will develop and test an online game that helps children learn how to better manage their arthritis. the goal of these studies is to test: (1) how easy to use and acceptable the online game is; and (2) if children who play the game feel less pain, have fewer limitations, and a better quality of life compared to children who do not play the game.


Eligibility criteria:
inclusion criteria: 1. age ≥ 8 and ≤ 11 years of age 2. diagnosed with jia (minimum 3 months) using international league of associations for rheumatology classification criteria96 3. active disease 4. child and primary parent/caregiver are able to speak and read english or french 5. participants are willing and able to complete online measures exclusion criteria: 1. cognitive impairments (as assessed by reviewing medical chart and consultation with the patient's rheumatologist) 2. major co-morbid illnesses (e.g., medical \[inflammatory bowel disease, cancer, diabetes\] or psychiatric \[depression, anxiety\]) which may impact their ability to understand and use the game or complete outcome assessments (as determined by their rheumatologist). 3. children currently participating in other cognitive behavioural therapy (cbt) interventions 4. usability testing participants
efficacy study of lucentis in the treatment of retinoblastoma

Conditions: Retinoblastoma

Summary: this study will evaluate the clinical efficacy of intravitreal injections of ranibizumab (lucentis) together with chemotherapy in the treatment of retinoblastoma as compared to chemotherapy alone.

this study will be a phase ii open label interventional case series. patients with retinoblastoma will be randomized to receive chemotherapy with or without intravitreal ranibizumab at a dose of 0.5mg/0.05 ml. patients will receive ranibizumab via a pars plana injection on a monthly basis for a total duration of therapy of 6 months. patients will be followed for 24 months .

Eligibility criteria:
inclusion criteria: * ability to provide written informed consent and comply with study assessments for the full duration of the study. * definite characteristic signs of retinoblastoma,group d base on intraocular international retinoblastoma classify, iirc. exclusion criteria: * history of surgical intervention for retinoblastoma in the study eye. * any previous disease in the study eye. * previous participation in any studies of investigational drugs within 1 month preceding day 0 (excluding vitamins and minerals). * previous participation in a clinical trial (for either eye) involving anti angiogenic drugs (pegaptanib, ranibizumab, anecortave acetate, protein kinase c inhibitors, etc.)

Keywords: efficacy
dose-range finding study for mk0893 (0893-008)

Conditions: Diabetes Mellitus, Type 2

Summary: a study to compare mk0893 to metformin or placebo for patients with type 2 diabetes (diabetes mellitus).


Eligibility criteria:
inclusion criteria: * patients who have type 2 diabetes mellitus, with suboptimal glucose control, while either not on aha (antihyperglycemic agent) therapy or on monotherapy or on low-dose combination therapy exclusion criteria: * patients have a history of type 1 diabetes mellitus * patients taking insulin or thiazolidinedione (tzd, a peroxisome proliferator-activated receptor \[ppar\]-gamma agonist) * patients who have a contraindication to metformin
clinical study of spinal muscular atrophy

Conditions: Spinal Muscular Atrophy

Summary: the investigators propose to prepare for clinical trials where sma patients are asked to join the research effort. the visits will include questions, physical exam, blood drawing, and sometimes x-rays and a skin biopsy. the investigators will use modern computer methods to process the information during which the investigators will plan a clinical trial. once the clinical trial begins, the investigators will offer sma patients participation if they meet the criteria for that trial. identifying an effective sma treatment is very important because there is currently none. clinical trials are the only way to decide whether a new treatment works in sma patients or not.

spinal muscular atrophy (sma) is one of the most devastating neurological diseases of childhood. affected infants and children suffer from progressive muscle weakness caused by degeneration of lower motor neurons in the spinal cord and brainstem. clinically, four phenotypes are distinguished within the continuous spectrum of disease severity based on the age of onset and the highest motor milestone ever achieved. sma is caused by homozygous deletion of the survival motor neuron-1 (smn1) gene. a related gene, smn2, produces low levels of full-length smn protein due to inefficient splicing. there is an inverse correlation between smn copy number and disease severity, presumably mediated by levels of full length smn protein. therefore, increasing the amount of full-length smn protein is a promising treatment strategy. several drugs targeting splicing efficiency have resulted in increased smn protein in preclinical assays and are now awaiting clinical testing. with the future objective to conduct clinical trials in sma, the proposed project has 3 specific aims: 1) to establish a web-based database that will serve to enroll the patient population and that will facilitate timely recruitment for future clinical trials; (2) to plan for clinical trials by a) developing reliable outcome measures, and (b) establishing the infrastructure needed to carry out efficient clinical trials, (c) convening meetings of preclinical and clinical researchers involved in sma drug development to select candidate drugs, and (3) to characterize the patient population from a clinical and molecular point of view.

Eligibility criteria:
inclusion criteria * clinical diagnosis of spinal muscular atrophy * genetic diagnosis of smn gene deletion * parents or if applicable subjects must give informed consent * must be capable of complying with the study procedures * female subjects of child-bearing potential must agree to undergo pregnancy test prior to radiological studies * diagnosis of sma before age 19 years exclusion criteria: * unstable medical condition precluding participation * significant respiratory compromise that would interfere with safe travel to site of evaluation. (the clinical site pi decides when air travel is not recommended and when the patient's location is not within a reasonably safe driving distance (upper limit 150-250 miles)

Keywords: pediatric neuromuscular disease