Kornimate/medical-research-clean huggingface dataset:
retrospective multicentre study on the impact of neonatal screening for congenital adrenal hyperplasia in italy

Conditions: Congenital Adrenal Hyperplasia

Summary: oservational, retrospective, multicenter study focused on neonatal screening for congenital adreanl hyperplasia.

the study refers to neonates who underwent neonatal screening for congenital adreanl hyperplasia between january 2006 and december 2019 at participating centres, but only patients diagnosed with congenital adreanl hyperplasia are enrolled in the study. the primary aims are: to assess the incidence of classic congenital adrenal hyperplasia in neonates who underwent neonatal screening in the reporting period; to calculate the sensibility of neonatal screening for classic congenital adrenal hyperplasia; to calculate the positive predictive rate. the secondary aims are: to describe the biochemical, hormonal, and clinical features of neonates at diagnosis of congenital adrenal hyperplasia; to assess the time required for diagnosis; to calculate the false positive rate; to calculate the diagnostic impact of neonatal screening for congenital adrenal hyperplasia.

Eligibility criteria:
inclusion criteria: * neonatal who underwent neonatal screening for congenital adrenal hyperplasia between january 2006 and december 2019 at participating centres; * patients diagnosed with congenital adrenal hyperplasia; * obtaining informed consent. exclusion criteria: • none.

Keywords: congenital adrenal hyperplasia
real-world validation of a prognostic prediction model for patients with acute exacerbations of chronic liver disease

Conditions: Chronic Liver Disease and Cirrhosis

Summary: our study is a multi-site, prospective, observational study including patients with acute exacerbations of cld. approximately 1500 patients from 24 hospitals in diverse regions in china will be enrolled and followed for 3 months. data about demographic data, laboratory tests, and clinical manifestations, collected through a mobile application, will inform real-time prognostic scores. the aim of this study is to validate the model's real-world applicability in non-specialist hospital settings.

the study is a multi-site, prospective, observational study conducted in 24 hospitals representing different regions across china. the choice of 24 hospitals was based on their representation of diverse geographical regions across china. these hospitals were selected to ensure a comprehensive validation of the screening model's efficacy in varied healthcare settings. data will be collected through a mobile application, providing real-time prognostic scores for patients. the study is divided into three parts: patient recruitment, in-hospital follow-up, and post-discharge follow-up . follow-up will last for 3 months. endpoints will be considered for all-cause death, survival and liver transplantation. the study is planned to enroll 1500 patients from january 2024 to january 2026.

Eligibility criteria:
inclusion criteria: * chronic liver disease with or without cirrhosis, including chronic viral hepatitis, alcoholic liver disease, non-alcoholic fatty liver disease, autoimmune liver disease, metabolic liver disease, chronic drug- induced liver disease and cryptogenic cirrhosis. the duration of underlying non-cirrhotic chronic liver disease should be longer than 6 months. * acute exacerbations of chronic liver disease, including acute decompensation (bacterial infection, hepatic encephalopathy, variceal bleeding, ascites and jaundice) in 1 month and liver injury (alt or ast \>3 uln , or tb\>2 uln) in 1 week. * patients are informed of the study purpose and willing to sign the informed consent. exclusion criteria: * \ 75 years old * with malignancy of liver or other organs * with other serious comorbidities (such as circulatory failure, respiratory failure, chronic kidney disease with end-stage renal failure, stroke within 6 months before admission, severe systemic autoimmune diseases or severe metabolic disease ) * pregnant * receiving immunosuppressive agents for non-hepatic diseases

Keywords: acute-on-chronic liver failure prognostic prediction model real-world validation
quinoa biscuit & cvd risk trial

Conditions: Cardiovascular Diseases; Dyslipidemias; Lipid Profile

Summary: cardiovascular disease (cvd) is one of the most common chronic diseases in older populations, which has been increasing in line with rising overweight and obesity levels in recent years. dietary intake is a major modifiable risk factor for cvd, and one such recommendation is to increase the intake of essential (omega-3) polyunsaturated fats in our diets, for example by consuming more oily fish. we know, however, from large population level dietary surveys, that many individuals within the united kingdom (uk) population are not consuming enough oily fish. therefore, alternative dietary sources of omega-3 polyunsaturated fats are required to help meet consumer needs. quinoa is a traditional andean seed crop consumed in a similar fashion to staple cereal grains in europe, and the popularity of quinoa has been growing worldwide because of its nutritional content and perceived healthiness. quinoa contains a small amount of fat, but the ratio of omega-6 and omega-3 essential fats is more favourable in quinoa than in other plant oils. an opportunity therefore exists to incorporate quinoa flour into more frequently consumed food products (e.g. biscuits) as an alternative means of increasing consumers omega-3 intake. the purpose of this study is to investigate the effect of consuming quinoa-enriched biscuits, compared to control, on markers of cvd risk over 4-weeks in older adults.


Eligibility criteria:
inclusion criteria: * free-living, apparently healthy adults * aged 50-75 years at recruitment * low fish consumers (\<2 servings/wk) * non-smokers * not regularly consuming plant stanols exclusion criteria: * non-free-living adults * aged \ 75 years at recruitment * fish consumers (2 servings/wk or more) * current smokers * pregnant/lactating females * coeliac disease, wheat intolerance or any other food allergy or intolerance that would prevent consumption of the biscuits * currently taking any fish oil-containing supplement * diagnosed with a chronic medical condition (such as diabetes; cvd autoimmune/ inflammatory disorders; cancer) * prescribed cholesterol or blood pressure lowering medications * daily consumption of plant stanols

Keywords: quinoa functional food cardiovascular disease cholesterol lipid profile randomised control trial
efficacy/safety study to confirm iberogast's efficacy in patients with irritable bowel syndrome diagnosed on rome iii criteria compared to placebo

Conditions: Irritable Bowel Syndrome

Summary: the study shall prove whether treatment of irritable bowel syndrome with iberogast is superior to placebo regarding the main criterium "pain"


Eligibility criteria:
inclusion criteria: * patients of either sex aged \>18 years * patients meeting the rome iii irritable bowel syndrome (ibs) diagnostic criteria. ibs is defined as recurrent abdominal pain or discomfort at least 3 days/month in last 3 months (onset at least 6 months ago) associated with two or more of the following: * improvement with defecation * onset associated with a change in frequency of stool * onset associated with a change in form (appearance) of stool * history of pain intensity with an average of worst abdominal pain in past 24 hours score of \> 30 on a daily measured vas (visual analogue scale) during screening phase exclusion criteria: * intake of stw5 within the last 5 years * concomitant treatment during the study (including screening phase) with any medication that could influence the gastrointestinal function * regular intake of nonsteroidal antiphlogistic drugs incl. cox-2-inhibitors (exception: acetylsalicylic acid for cardiovascular prevention up to 100 mg daily) * patients with known hypersensitivity to any component of the trial drugs * history of eating disorders * patients with a history of diseases with abdominal symptoms that can resemble ibs * presence of any other acute or chronic gastrointestinal disorder * history of abdominal surgery (cholecystectomy and appendectomy can be tolerated when performed at least one year previously) * known intolerance to azo dyes e 110 and e 151

Keywords: iberogast efficacy safety
whole-body mra at 3t - a comparison between two different scan protocols

Conditions: Atherosclerosis; Intermittent Claudication

Summary: to compare to different approaches in whole-body magnetic resonance angiography of patients suffering peripheral arterial disease. our hypothesis is that use of a new protocol improves the diagnostic quality of the wb-mra procedure.

patients suffering peripheral arterial disease must undergo imaging procedures before endovascular or surgical procedures. wb-mra is an imaging procedure that can visualise the arterial system. wb-mra can be performed in different manners. on option is to start the scan in the head region and continue down the body to the feet (standard protocol). another option is to use a hybrid protocol that first examines the head and lower leg followed by examination of the pelvic and upper leg region. a comparison between the standard and hybrid protocols has not been performed in a 3t high magnetic field system, therefore we will perform such a study.

Eligibility criteria:
inclusion criteria: * symptomatic lower extremity ischemia (claudication, ischemic wounds) * referred to digital subtraction angiography (dsa) exclusion criteria: * renal insufficiency (gfr \< 30 ml/min) * contra-indications for mri-examination (claustrophobia, metal-implants, pacemaker) * dementia * pregnancy/lactation * allergy to gadolinium based mri contrast agents * acute disease

Keywords: magnetic resonance whole body angiography hybrid protocol mri
phase 1/2 study of hs-10375 in patients with advanced or metastatic non-small-cell lung cancer(nsclc)

Conditions: Advanced or Metastatic NSCLC

Summary: hs-10375 is an oral, highly selective, small molecular inhibitor of egfr c797s. this study will evaluate the safety, tolerability, pharmacokinetics and clinical activity of hs-10375 in chinese advanced or metastatic nsclc.


Eligibility criteria:
inclusion criteria: 1. men or women greater than or equal to 18 years. 2. locally advanced or metastatic nsclc patients confirmed by histology or cytology, for which standard treatment is invalid, unavailable or intolerable . 3. pathological, nsclc tissue samples can be used to test egfr c797s mutation by central laboratory for phase 1b and phase ii subjects. 4. at least one measurable lesion in accordance with recist 1.1. 5. eastern cooperative oncology group (ecog) performance status: 0\~1. 6. estimated life expectancy \>12 weeks. 7. reproductive-age women agree to use adequate contraception and cannot breastfeed while participating in this study and for a period of 6 months after the last dose. likewise, men also consent to use adequate contraceptive method within the same time limit. 8. females must have the evidence of non-childbearing potential. 9. signed and dated informed consent form. exclusion criteria: 1. treatment with any of the following: * previous or current treatment with egfr c797s inhibitors. * any cytotoxic chemotherapy, anticancer chinese medicine and targeted small molecule inhibitors within 14 days of the first dose of hs-10375. * any investigational agents and large molecule antibodies within 28 days of the first dose of hs-10375. * local radiotherapy for palliation within 2 weeks of the first dose of hs-10375, or patients received more than 30% of the bone marrow irradiation, or large-scale radiotherapy within 4 weeks of the first dose of hs-10375. * major surgery (including craniotomy, thoracotomy, or laparotomy, etc.) within 4 weeks of the first dose of hs-10375. 2. inadequate bone marrow reserve or serious organ dysfunction. 3. uncontrolled pleural, ascites or pericardial effusion. 4. known and untreated, or active central nervous system metastases. 5. active autoimmune diseases or active infectious disease. 6. refractory nausea, vomiting, or chronic gastrointestinal diseases, or inability to swallow oral medications. 7. history of hypersensitivity to any active or inactive ingredient of hs-10375 or to drugs with a similar chemical structure or drugs belonging to the same category of hs-10375. 8. the subject who is unlikely to comply with study procedures, restrictions, or requirements judged by the investigator. 9. the subject whose safety cannot be ensured or study assessments would be interfered judged by the investigator. 10. pregnant women, breastfeeding women or woman who has a child-bearing plan during the study. 11. history of neuropathy or mental disorders, including epilepsy and dementia.

Keywords: advanced or metastatic nsclc egfr c797s mutation hs-10375
a study of irinotecan hydrochloride liposome in combination with 5-fu/lv in advanced solid tumors

Conditions: Advanced Solid Tumors

Summary: this is a dose-escalation and expansion, open label, single centre, phase ib study. in this study, the tolerability, safety, pharmacokinetics and efficacy of irinotecan hydrochloride liposome injection in combination with 5-fu/lv were studied in patients with advanced solid tumors. meanwhile, to determine the dose-limiting toxicity (dlt) and maximum tolerated dose (mtd) of irinotecan hydrochloride liposome.


Eligibility criteria:
inclusion criteria: 1. histologically or cytologically confirmed solid tumors documented as advanced or metastatic disease; 2. subjects must be considered relapsed or refractory to standard therapies, have been intolerant to standard therapies, or have refused standard therapy; 3. ecog: 0-1; 4. adequate organ and bone marrow function; 5. sign an informed consent. exclusion criteria: 1. patients with brain malignant tumor or active cns metastasis; 2. ugt1a1\*28 homozygous mutants; 3. clinically significant gi disorders; 4. significant cardiovascular disease; 5. active infection or uncontrolled fever; 6. pregnant or breast feeding patients; 7. allergic to a drug ingredient or component; 8. the investigators determined that other conditions were inappropriate for participation in this clinical trial.
the pro-parenting study: helping parents reduce behavior problems in preschool children with developmental delay

Conditions: Development Delay; Behavior Problem

Summary: the pro-parenting study seeks to determine the added benefit of targeting both parenting stress and parent management strategies to more effectively reduce behavior problems among children with developmental delay (dd). findings from this study will improve the scientific understanding of evidence-based interventions for behavior problems among children with dd and the mechanisms underlying therapeutic change.

behavior problems are a common and concerning challenge among children with developmental delay (dd). approximately 50% of children with dd have a comorbid mental disorder or serious behavior problems- a prevalence three times as high as that found in typically developing youths. behavioral parent training (bpt) is the gold-standard intervention for treating child behavior problems in typically developing children and in children with dd. however, high levels of parental stress are associated with reduced or no response to bpt for children with dd. consequently, parental stress may attenuate the efficacy of the gold-standard, empirically supported treatment for behavior problems among children with dd. as such, parental stress is a critical point of intervention for improving both parent and child outcomes in families of children with dd. the purpose of this study is to quantify the therapeutic benefit of adding a parent stress-reduction intervention prior to delivering bpt in order to more effectively reduce child behavior problems, and to investigate the mechanisms through which intervention outcomes occur.

Eligibility criteria:
inclusion criteria: * parent has a child ages 3 to 5 years with an agency-identified dd in one or more functional areas who is receiving early intervention or early childhood/ preschool special education through an individualized family service plan (ifsp) or individualized education plan (iep); * parent reports elevated child behavior problems, as indicated by a t-score of 60 or above on the total problems scale of the child behavior checklist; * parent reports elevated parenting stress, as indexed by a total score above the recommended cutoff at the 85th percentile on the parenting stress index-4. exclusion criteria: * parent screens positive for active psychosis, substance abuse, or suicidality; * parent is currently receiving any form of psychological or behavioral treatment at the time of referral; or * the child has sensory impairments or nonambulatory conditions that would necessitate the need for significant modifications to the lab and home visit protocols.

Keywords: intervention behavioral parent training mindfulness-based stress reduction
sequential treatment of cd20-positive posttransplant lymphoproliferative disorder (ptld)

Conditions: Post-transplantation Lymphoproliferative Disorder

Summary: post-transplantation lymphoproliferative disorder (ptld) develops in one to ten per cent of transplant recipients and can be ebv-associated. to improve long-term efficacy after rituximab monotherapy and to avoid the toxicity of chop seen in first-line treatment, the investigators initiated an international multicentre phase ii trial to test whether the subsequent application of rituximab and four courses of three-weekly chop would improve the outcome of patients with ptld: ptld-1, sequential treatment (st).


Eligibility criteria:
inclusion criteria: * ptld with or without ebv association, confirmed after biopsy or resection * measurable disease of \> 2 cm in diameter and/or bone marrow involvement * patients having undergone heart, lung, liver, kidney, pancreas, small intestine transplantation or other or a combination of the organ transplantations mentioned * karnofsky scale \>50% or ecog ≤ 3 * reduction of immunosuppression with or without antiviral therapy * a complete surgical extirpation of tumor was not performed * a radiation therapy was not performed * effective contraception for women in childbearing age * patient's written informed consent and written consent for data collection * patients are \> 18 years (or ≥ 15 years with parental agreement ) exclusion criteria: * life expectancy less than 6 weeks * karnofsky-scale \<50% or ecog =3 * treatment with rituximab before * known allergic reactions against foreign proteins * concomitant diseases, which exclude the administration of therapy as outlined by the study protocol * non-compensated heart failure * dilatative cardiomyopathy * myocardial infarction during the last 6 months * severe non-compensated hypertension * severe non-compensated diabetes mellitus * renal insufficiency (creatinine more than 3-fold of the upper normal value), not related to lymphoma. * hepatic insufficiency with transaminase values greater than 3-fold of the normal values and/or bilirubin levels \>3.0 mg/dl, not related to lymphoma * clinical signs of cerebral dysfunction * women during the lactation period, pregnant or of childbearing potential not using a reliable contraceptive method * involvement of the central nervous system by the disease * severe psychiatric disease * known to be hiv positive * missing written informed consent of the patient

Keywords: 1st-line therapy single agent rituximab chop ptld
tlk286 in combination with paraplatin (carboplatin) in recurrent ovarian cancer

Conditions: Ovarian Neoplasms

Summary: this is a dose-ranging, open label, phase 1-2a study of tlk286 in combination with paraplatin (carboplatin) in patients with recurrent ovarian cancer.


Eligibility criteria:
inclusion criteria * histologically or cytologically confirmed diagnosis of epithelial cancer of the ovary, fallopian tube, or primary peritoneal cancer * recurrent ovarian cancer or persistent disease following primary treatment * at least one, but no more than four, prior chemotherapy regimens (all platinum-containing counted as one) * at least 18 years of age exclusion criteria * pregnant or lactating women * history of prior malignancy except for adequately treated carcinoma in situ of the uterine cervix, basal cell or squamous cell skin cancer, or other cancer for which patient has been disease-free for at least 2 years * known leptomeningeal metastases or carcinomatous meningitis * having received whole pelvis radiation therapy
multiprofessional advance care planning and shared decision making for end of life care

Conditions: Palliative Patients; Surrogate Decision Maker

Summary: multiprofessional advance care planning and shared decision making for end of live care for terminal patients and their relatives. the aim of this study is to develop, implement and test a complex intervention for improving patients' preparation for and participation in end of life decisions. in cooperation with local, national and international partners, the investigators will focus on strategies to enhance advance care planning and shared decision making on end of life issues, and documentation and transferability of end of life decisions across health care settings in coordinated approach involving patients, their families and care givers (in and out of hospital).


Eligibility criteria:
inclusion criteria: -patients in which the treating physician on the ward would not be surprised if the patient died during the next year (surprise question of weissman et al 2011) * male and female patients above 18 years of age * signed informed consent after being informed * patients able to appoint a surrogate decision maker (sm) and/or responsible physician to be contacted after discharge exclusion criteria: -patients not capable of speaking german * patients having no responsible physician to be contacted after discharge and no relative/future surrogate decision maker (sm) * inhouse patients being discharged within the next 2 days or ambulatory patients not regularly coming to the wards (at least every two month) * patients assessed by their physician to be in obvious denial of their situation (illness/prognosis)
lrrk2 g2385r leucocyte and urine biomarker study

Conditions: Parkinson Disease

Summary: given the emerging evidence on lrrk2-related biomarkers, this project will focus on identifying leucocyte and urine biomarkers that are altered by the lrrk2 g2385r variant, and investigating the effects of lrrk2 kinase inhibitor (mli-2) on these biomarkers, using immunoblotting and mass spectrometry.


Eligibility criteria:
inclusion criteria: * diagnosis of parkinson's disease assigned by a movement disorder specialist according to clinical diagnostic criteria * age above 18 year-old * provision of a written informed consent exclusion criteria: - physical or mental incapacity to complete study protocol