Kornimate/medical-research-clean huggingface dataset:
phase ii study of glucocerebrosidase in patients with gaucher disease

Conditions: Gaucher's Disease

Summary: objectives: i. evaluate the efficacy and toxicity of glucocerebrosidase enzyme therapy in patients with gaucher disease.

protocol outline: patients are treated with intravenous glucocerebrosidase every 2 weeks. the dose is based on clinical severity of disease and response to therapy.

Eligibility criteria:
protocol entry criteria: --disease characteristics-- gaucher disease with glucocerebrosidase deficiency confirmed by enzymatic or molecular assay at least 3 organ systems affected, based on the following criteria: anemia thrombocytopenia organomegaly bone deterioration on radiograph pulmonary compromise symptoms compromise daily activities or risk longevity no neurologic disease

Keywords: gaucher's disease inborn errors of metabolism rare disease sphingolipidoses
development of pre-pregnancy intervention to reduce the risk of diabetes and prediabetes

Conditions: Diabetes; Diabetes Mellitus, Type 2

Summary: this study is conducted in asia. the aim of this study is to assess the efficacy of a pre-pregnancy life style intervention to reduce the risk of diabetes and prediabetes.


Eligibility criteria:
inclusion criteria: * informed consent obtained before any trial-related activities. trial-related activities are any procedures that are carried out as part of the trial, including activities to determine suitability for the trial * nulliparity * not pregnant at the time of signing informed consent * own a smartphone with either android system version 4.1 and above or ios system 7.0 and above with internet access exclusion criteria: * female subject undergoing treatment for type 1 or 2 diabetes mellitus * subjects not residing in the district of seremban
efficacy,safety and quality of life after tookad® soluble vtp for localized prostate cancer

Conditions: Prostate Cancer

Summary: the aim of this study is to confirm the efficacy,the safety and the quality of life after tookad® soluble vtp in localized prostate cancer patients.

the study is designed as a multicenter, single-arm, open label, 12 months follow-up clinical trial. men with localized prostate cancer will be treated with tookad® soluble vtp under general anesthesia. patients will be followed-up for 12 months with patient questionnaires on qol, erectile and urinary functions, clinical evaluation with study visits at 1, 3, 6 and 12 months , prostate specific antigen(psa) testing at 3, 6 and 12 months. in addition, they will have biopsy at 6 months (± 2) and 12 months.

Eligibility criteria:
inclusion criteria: * histologically proven localized prostate carcinoma diagnosed using prostate biopsy showing: * gleason 3+3 prostate * gleason 3+4 prostate adenocarcinoma is acceptable provided it is not present in more than 2 cores and no more than 50% cancer in any core. * clinical stage up to ct2a - n0/nx - m0/mx. * serum prostate-specific antigen (psa) ˂ 20ng/ml * prostate volume ≥ 25 cc and ≤ 70 cc. * male subjects aged 18 years or older. * signed informed consent form by the patient. exclusion criteria: * unwillingness to accept the treatment. * any prior or current treatment for prostate cancer, including surgery, radiation therapy (external or brachytherapy)or chemotherapy. * any surgical intervention for benign prostatic hypertrophy. * any condition or history of illness or surgery that may pose an additional risk to men undergoing the vtp procedure. * life expectancy less than 10 years. * participation in another clinical study involving an investigational product within 1 month before study entry. * subject unable to understand the patient's informed consent document, to give consent voluntarily or to complete the study tasks, especially unable to understand and fulfill the health-related qol questionnaire. * subject in custody and or in residence in a nursing home or rehabilitation facility. * any condition or history of active rectal inflammatory bowel disease or other factors which may increase the risk of fistula formation; * any hormonal manipulation (excluding 5-alpha-reductase inhibitors) that alters androgen production or use of androgen supplements within the previous 6 months; * any history of urethral stricture disease; * any history of acute urinary retention within 6 months of study entry

Keywords: prostatic disease genital neoplasm,male urogenital neoplasm genital disease,male male urogenital disease neoplasms neoplasms by site prostatic neoplasm carcinoma
the participant agreement for contact tracing (pact) study: enhancing partner notification services.

Conditions: Chlamydia Infection; Contact Tracing

Summary: patients diagnosed with chlamydial infections (a sexually transmitted disease) are asked to notify their sex partners and tell them to seek medical evaluation. this project tests an enhancement to the materials provided to patients to help convince their partners to seek evaluation against the standard of care, which is a brief notification instruction. the desired outcomes are greater levels of notification by participants of their partners and lower levels of reinfection among participants.

patients diagnosed with chlamydial infections (a sexually transmitted disease) are asked to notify their sex partners and tell them to seek medical evaluation: patient referral. rates of actual referral by patients are unknown, but estimates derived from several evaluations suggest somewhere between 30 - 55% of partners are notified and tested (for chlamydia). current prevalence and yearly rates of infection suggest this level of partner notification and treatment is insufficient to control the disease. as a program, this project has disseminated notification activities to community health centers that see chlamydial infections, all under the aegis of the awardee (boston medical center). diagnosis and partner notification can be recorded remotely , but centrally accessed through a secure database. to enhance patient referral effectiveness, this project tests brief instructions to refer (standard of care) against a "kit" containing a specific notification of exposure to chlamydia, an accurate health message about the nature and prognosis of the infection (treated and untreated), options for seeking free or low-cost evaluation and treatment, and a satisfaction survey. a second, crossed condition is pure patient referral against a contract (72 hours to notify, after which study staff will refer cases to public health professionals for notification). principal outcomes measured are levels of notification by participants of their partners and levels of reinfection among participants. we also measure psychosocial mediating effects, as well as potential unintended consequences of patient referral: incident partner violence against prevalent (baseline rates) violence, depressed affect

Eligibility criteria:
inclusion criteria: * sexually active and * 15 years old or older and at least one of: * individuals with genitourinary symptoms requiring empiric treatment for chlamydia, or * people who self-report that they have had sex with someone who has been diagnosed with an std within the past 30 days or * asymptomatic female patients with cervicitis diagnosed via a routine pelvic examination. exclusion criteria: * subsequent lab confirmation of no chlamydial infection or * sex partner of previously enrolled person or * fear of violence from partner during notification.

Keywords: sexually transmitted disease structural intervention behavioral intervention
role of caregiver in the clinical pathway of patients with breast and prostate cancer

Conditions: Cancer

Summary: the following randomized longitudinal study will evaluate the long term impact of caregivers' involvement in cancer care pathway, including a psychological support intervention on the dyads to be delivered after the detection of a suspected cancer and before the visit with the oncologist for discussing therapeutic options.

the following randomized longitudinal study will evaluate the long term impact of caregivers' involvement in cancer care pathway, including a psychological support intervention on the dyads to be delivered after the detection of a suspected cancer and before the visit with the oncologist for discussing therapeutic options. moreover, a battery of self-report questionnaires will be administered, and sociodemographic and clinical data will be collected. the time window in which to deliver the support intervention is after the detection of a suspected cancer and before the visit with the oncologist, at the time when the patient undergoes diagnostic exams (to understand the type of tumor he/she is affected by), and the most suitable therapeutic protocol have to be discussed with the oncologist.

Eligibility criteria:
inclusion criteria: patient: * patient aged ≥18 years at the time of recruitment. * patients who have just received a cancer diagnosis and have yet to discuss with their oncologist the treatment to undergo after diagnosis of breast or prostate cancer. * early-stage cancer (i or ii). caregiver: * age ≥ 18 at the time of recruitment. * taking care of the patient. exclusion criteria: patients: * presence of early mental disorders (before age 40) or severe neurological disorder. * patients with advanced stage cancer for which the path is already defined (palliative care patients). caregivers: ● presence of early mental disorders (before age 40) or severe neurological disorder.
non-interventional study on the tolerability and efficacy of ivig

Conditions: Primary and Secondary Immunodeficiency and Other Conditions Requiring Regular Administration of Octagam 5% or 10% IVIG

Summary: non-interventional study on the tolerability and efficacy of octagam® 10%

a non-interventional study on the tolerability and efficacy of octagam 5% or 10% or panzyga. patients with any indication (replacement and immonomodulation) as prescribed by the treating physician. marketed octagam 5% or 10% or panzyga will be used according to the investigator prescription; intravenous (iv) administration.the primary objective is to detect and evaluate adverse drug reactions (adrs) occurring during or after the administration of octagam or panzyga in any indication, age group or treatment regimen, where a causal relationship to the administration of octagam or panzyga is suspected.

Eligibility criteria:
inclusion criteria: * any patient who needs to be treated with a product of this class because of his/her medical condition and whom the treating physician decides to prescribe octagam or panzyga regularly, may be included. exclusion criteria: * none
the surgical treatment of total brachial plexus avulsion injury-a retrospective study of 73 patients

Conditions: Brachial Plexus Injury

Summary: brachial plexus avulsion injury (bpai) caused by traction injury, especially total root avulsion, represents a severe handicap for the patient. despite recent progress in diagnosis and microsurgical repair, the prognosis in such cases remains unfavorable. we need to find an relatively optimal surgical treatment.

brachial plexus avulsion injury (bpai) caused by traction injury, especially total root avulsion, represents a severe handicap for the patient. despite recent progress in diagnosis and microsurgical repair, the prognosis in such cases remains unfavorable. nerve transfer is the most successful method for repairing cases of brachial plexus root avulsion. phrenic nerve transfer, accessory nerve transfer, intercostal nerve transfer and contralateral c7 transfer are all well-established technique in the treatment of certain severe brachial plexus lesions in adults. as reported in previous articles, shoulder function, elbow flexion, elbow extension, and wrist and finger function have been restored successfully by spinal accessory, phrenic, intercostal, and contralateral c7 nerve transfers . however, the results of just one function reconstruction were evaluated in those articles. few studies were found that focused on the whole function reconstructions of the affected limb. in different medical organizations even in our department, many different surgical strategies were used in treating total bpai patients and the results differed significantly. here, we investigated the results of different commonly used nerve transfer in order to determine a relatively optimal surgical strategy for treatment of total bpai patients.

Eligibility criteria:
inclusion criteria: * global root avulsion brachial plexus injury, a minimum postoperative interval of 3 years, all operations performed by the same medical team, nerve transfer was the only reconstruction method. exclusion criteria: * diabetes, volkmann contracture, fracture on the affected limb, rib fracture, brain trauma.

Keywords: brachial plexus; avulsion injury
predictors of in-hospital and short-term clinical outcomes in stemi patients undergoing primary pci

Conditions: STEMI

Summary: the study aims to assess the culprit-syntax score and no-reflow piano scores as possible predictors of in-hospital and short-term outcomes among patients with stemi undergoing primary pci

st-elevation myocardial infarction (stemi) is the leading cause of death in cardiovascular patients. percutaneous coronary intervention (pci) is recommended as the preferred treatment for patients with stemi. however, patients with (stemi) undergoing primary (pci) remain at high risk of adverse events, including death, reinfarction, stent thrombosis, and repeat revascularization. this underlines the need to identify predictors of impaired clinical outcomes in order to recognize higher-risk patients in the contemporary practice of primary pci. stemi patients show variable mortality risks, in order to assess individual risk, a number of mathematical models (scoring algorithms) have been developed as timi, grace 2.0, and alpha scores similarly, angiographic scores were developed to assess the outcomes in stemi patients, syntax score, has shown predictive value of in-hospital mortality among stemi patients. recently, the culprit-syntax score appears to be as successful as the syntax score in predicting stent thrombosis in patients with acs and more successful in predicting 30-day deaths and major complications. the no-flow phenomenon (nrp), one of the most common complications of primary pci, significantly reduces the benefit of this procedure. the occurrence of nrp following ppci leads to an unfavorable short-term prognosis by increasing the risk of complications and in some cases, of death. a new score system (the piano score) based on six clinical variables was constructed and validated, was variables to predict angiographic no-reflow in stemi patients undergoing primary pci, and very recently showed potential prognostic value in patients with ami undergoing primary pci.

Eligibility criteria:
inclusion criteria: patients presented with 1st episode of stemi and undergoing primary pci. for 1 year duration. - exclusion criteria: 1. patient with stemi in which thrombolytic therapy was used. 2. patient with a history of previous cabg surgery. 3. patient with a history of previous pci. 4. patient with a history of previous stemi. 5. patients with myocardial infarction with non-obstructive coronary arteries (minoca) 6. patient with spontaneous coronary artery dissection (scad).
effects of glp-1ras on reproductive outcomes in pcos and obesity

Conditions: Polycystic Ovary Syndrome; Obesity; Infertility, Female

Summary: the goal of this clinical trial is to examine whether benalutide combined with metformin and healthy lifestyle education in women with pcos and obesity prior to fertility treatment improves live birth rate and other reproductive, maternal and perinatal outcomes compared to metformin plus healthy lifestyle education.

benalutide, a short-acting recombinant human glp-1ras with nearly 100% homology to human glp-1, has been approved by the chinese national medical products administration for treating weight loss in individuals with obesity. up to now, there is a paucity of research on the potential impact of glp-1ras on reproductive, maternal and perinatal outcomes in women with pcos and obesity undergoing fertility treatment. given its effectiveness as a weight loss medication, benalutide theoretically holds promise for enhancing reproductive outcomes in women with pcos and obesity prior to fertility treatment by facilitating weight reduction.

Eligibility criteria:
inclusion criteria: women with a wish to conceive; women with pcos (rotterdam criteria); 28 kg/m2≤bmi\<35kg/m2; exclusion criteria: women with medical morbidity; endocrine disorders; abnormalities in liver and kidney function; other medications such as glucocorticoids, anti-androgen drugs ;(spironolactone, cyproterone acetate, flutamide, etc.) within the last 3 months; acute infections, severe cardiovascular disease, malignant tumors; ongoing weight loss therapy (\>5% weight loss in the last 3 months) or history of gastrointestinal surgery; uterine anomalies or untreated tubal hydrosalpinx; receiving a donor oocyte or donor sperm cycle; chromosome abnormality or recurrent miscarriage; planning preimplantation genetic testing (pgt); any other contraindications to fertility treatment; allergic to or contraindicated for glp-1 class medications.
study assessing the effect of 3-week treatment with one of three oral doses of quinagolide

Conditions: Ovarian Hyperstimulation Syndrome

Summary: women included in this study are undergoing controlled ovarian hyperstimulation for assisted reproductive technologies (art) and present with signs of moderate risk of developing ohss.

no data to be entered.

Eligibility criteria:
inclusion criteria 1. signed informed consent form, prior to screening evaluations 2. in good physical and mental health 3. pre-menopausal females between the ages of 21-37 years (both inclusive) at the time of randomisation 4. infertility for at least 1 year before randomisation, except for proven bilateral tubal infertility exclusion criteria 1. any clinically significant systemic disease 2. endocrine or metabolic abnormalities (pituitary, adrenal, pancreas, liver or kidney) 3. history of recurrent miscarriage 4. undiagnosed vaginal bleeding
ot intervention via telehealth for children with asd and adhd

Conditions: Autism Spectrum Disorder; Attention Deficit Hyperactivity Disorder

Summary: this study aims to measure the impact of a telehealth-based model of occupational therapy (ot) treatment on clinical outcomes for children with autism spectrum disorder (asd) and/or attention deficit hyperactivity disorder (adhd). half of the participants will receive clinic-based, in-person services and half of the participants will receive video-based, telehealth services.

children with autism spectrum disorder (asd) and/or attention deficit hyperactivity disorder (adhd) often receive occupational therapy (ot), which traditionally takes place in a clinical setting. this setting may present barriers and distractors that make it hard for children to achieve their ot goals. telehealth-based sessions provide an alternative, allowing the therapist to be virtually present within the home.once enrolled, children will randomly be assigned to be in one of two groups: (1) standard, in which children will receive clinic-based services as per standard of care or (2) telehealth, in which children will receive in-home services on a video-based platform on a timeline identical to what they would receive if they were in the standard group. children in both groups will participate for the duration of the episode of care, estimated to last 10-12 weeks. assessments will be completed at initial evaluation, discharge, and three months following discharge.

Eligibility criteria:
inclusion criteria: * has a autism spectrum disorder and/or attention deficit hyperactivity disorder * has an active occupational therapy referral * has occupational therapy goals that are directly related to performance of activities of daily living that might reasonably be affected by the environment (as determined by an evaluating therapist) exclusion criteria: * does not have autism spectrum disorder or attention deficit hyperactivity disorder * does not have an active occupational therapy referral * does not have occupational therapy goals that are directly related to performance of activities of daily living that might reasonably be affected by the environment (as determined by an evaluating therapist)
evidence based patient information and smartphone accelerometry to enhance physical activity in ms

Conditions: Multiple Sclerosis, Chronic Progressive

Summary: due to their ubiquitary distribution, smartphones might serve as an easy way/ possibility to use feedback mechanism in an app-based intervention program to increase physical activity in multiple sclerosis (ms) patients. internet based cognitive-behavioral interventions have been explored as effective in the last years. in addition, smartphone-based mobility assessment and intervention might be a promising approach in other ms types and for real-life mobility assessment in observational and interventional trials. the aim is to investigate the impact of a smartphone based information and feedback program on physical activity in a 3 months, randomised waiting-group controlled trial of 40 progressive ms patients.


Eligibility criteria:
inclusion criteria: * progressive ms exclusion criteria: * other major health disorder * edss above 6

Keywords: physical activity mobility
afatinib (gilotrif®) in patients suffering from tumors harboring neuregulin 1 (nrg1) gene alterations (specifically nrg1 gene fusion-positive advanced solid tumors)

Conditions: Solid Tumors

Summary: gene fusions are defined as two separate genes joined together (gene 1, gene 2), generating a novel fusion gene. nrg1 fusions are rare and complex with regard to the fusion/fusion partner. the specific nrg1 gene fusion will be identified by a specialized molecular testing lab.

afatinib is provided on a single-patient protocol basis to patients suffering from tumors harboring nrg1 fusions, a disease for which no satisfactory authorised alternative therapy exists. in the context of this mechanism it is planned to collect and analyze limited clinical data in order to investigate the safety and efficacy of afatinib in patients with advanced and/or metastatic solid tumours harboring nrg1 gene fusions.

Eligibility criteria:
inclusion criteria to participate in this program: * confirmed diagnosis of an advanced, unresectable and/or metastatic tumor harboring nrg1 gene fusions characterized as follows: * gene 1: nrg1 (prerequisite: conservation of the epidermal growth factor (egf) domain - this will be identified by the lab performing the molecular testing) * gene 2: all fusion partners are allowed (prerequisite: the region must be a coding region - this will be identified by the lab performing the molecular testing) * patient must have measurable or evaluable lesions (according to recist 1.1). * at least 18 years of age at the time of consent. exclusion criteria: * treatment with a systemic anti-cancer therapy or investigational drug within 14 days or 5 half-lives (whichever is shorter) of the first treatment with the study medication. * tumors carrying additional gene mutations other than nrg1 fusion where fda-approved targeted therapy is available * prior treatment with a therapy targeting erythroblastic leukemia viral oncogene homologue receptors (erbb) * any patient considered ineligible by the treating physician.

Keywords: nrg1 fusion