Kornimate/medical-research-clean huggingface dataset:
study of danicopan as add-on treatment to ravulizumab or eculizumab in pediatric participants with pnh who have clinically significant extravascular hemolysis

Conditions: Paroxysmal Nocturnal Hemoglobinuria; PNH; Extravascular Hemolysis

Summary: the primary objective of this study is to evaluate efficacy of danicopan as add-on treatment to ravulizumab or eculizumab as assessed by hemoglobin (hgb) change from baseline at week 12 in pediatric participants with paroxysmal nocturnal hemoglobinuria (pnh) and clinically significant extravascular hemolysis (cs-evh).


Eligibility criteria:
inclusion criteria: * confirmed diagnosis of pnh. * cs-evh defined by: anemia: hgb ≤ 10.5 g/dl, and absolute reticulocyte count ≥ 100 × 109/l * treated with ravulizumab or eculizumab for at least 12 weeks immediately preceding day 1, the dose received should be stable during this period, and there should be no anticipated changes in dosage or interval during the first 12 weeks of this study. * all participants must be vaccinated against meningococcal infection from serogroups a, c, w, and y and serogroup b within 3 years prior to, or at least 14 days prior to day 1 * vaccinated against haemophilus influenzae type b (hib) and streptococcus pneumoniae exclusion criteria: * platelet count \< 30000/μl or there is a need for platelet transfusions. * anc \< 500/μl. * clinically significant laboratory abnormalities related to liver function, including: * alt \> 2 × uln or alt \> 3 × uln for participants with documented liver iron overload defined by serum ferritin values ≥ 500 ng/ml. * direct bilirubin \> 2 × uln, unless, in the investigator's opinion, is due to hemolysis or gilbert's syndrome based on medical history. * current evidence of biliary cholestasis. * known aplastic anemia or other bone marrow failure that requires hsct or other therapies, including anti-thymocyte globulin and immunosuppressants unless the dosage of immunosuppressant has been stable for at least 12 weeks before day 1 and is expected to remain stable through week 12. * history of a major organ transplant (eg, heart, lung, kidney, liver) or hsct. * known or suspected complement deficiency. * active bacterial or viral infection, a body temperature \> 38°c on 2 consecutive daily measures, evidence of other infection, or history of any febrile illness within 14 days prior to first study intervention administration.

Keywords: paroxysmal nocturnal hemoglobinuria pnh extravascular hemolysis ravulizumab danicopan
impact of covid19 on asthma

Conditions: Asthma; Covid19

Summary: the covid19 pandemic is having an immeasurable impact on the economy and on morbidity and mortality. knowledge and scientific evidence about this disease is advancing rapidly, but it is not yet known whether asthmatic patients suffering from covid19 have an exacerbation of asthma, or whether this viral infection has an impact on control and lung function in the short to medium term. the aim of this study is to define the changes that occur in these two parameters in asthmatic patients suffering from covid19. to this end, asthmatic patients who have suffered a sars-cov-2 infection and who have required an emergency consultation or hospitalisation will be collected in a pneumology consultation and matched by age, sex and severity of asthma with a group that has not suffered the same. both will be followed for one year, and lung function will be tested at six months and exacerbations and changes in act during the following year. these data are intended to improve the available knowledge on the impact of idvc19 on asthma patients with a view to making appropriate recommendations, prevention and treatment adjustments in line with the results obtained.


Eligibility criteria:
inclusion criteria: * 12 -80 years * asthma diagnosis according to guidelines * follow up on asthma clinics in one of the participant hospitals exclusion criteria: * other severe respiratory condition independent of asthma

Keywords: asthma asthma control covid19
the relationship between cognitive status and exercise capacity, fatigue perception in coronary artery patients

Conditions: Coronary Artery Disease

Summary: the study aimed to examine the relationship between cognitive status, exercise capacity, and fatigue perception in coronary artery disease patients.

exercise training for patients with coronary artery disease is an essential element of multicomponent cardiac rehabilitation. for the successful implementation of exercise-based cardiac rehabilitation programs, adequate cognitive function (e.g. executive function, selective attention, memory) is an important prerequisite. in patients with atherosclerotic coronary artery disease, the presence of coronary microvascular dysfunction as determined by low coronary flow reserve is associated with abnormal cerebral flow hemodynamics and mild but significant impairment in cognitive function. in the literature, there is no study examining the relationship between cognitive level and fatigue perception.

Eligibility criteria:
inclusion criteria: * volunteering to participate in the research * being diagnosed with coronary artery disease * to cooperate with the tests to be performed * signing a consent form exclusion criteria: * presence of neurological disease that may affect cognitive status * presence of clinically unstable cardiac disease * left ventricular ejection fraction \<30% * being diagnosed with end-stage renal failure * diagnosed with active myocarditis or pericarditis * being diagnosed with rhythm disorder * having amputation, peripheral arterial disease, musculoskeletal and/or neurological diseases that will prevent participation in functional capacity tests * not volunteering to participate in the study

Keywords: exercise test fatigue cognitive function
valuation of quality of life in children with asthma

Conditions: Asthma

Summary: the aim of the study is valuation of quality of life (ql) in children with asthma and analysis of the factors that can have influence on it. the clinical problems included into the aim are to evaluate: * the correlation between the ql and asthma control * the utility of the polish version of paqlq(s) by juniper, especially the understanding of each question and it's answers the results of this study can be very helpful in the future in developing a questionnaires to use in polish primary care, according to which the investigators would be able to see whether the asthma in our patients is being well controlled.

quality of life in people with asthma is strictly relevant with the correct treatment of the disease, that is why the correct screening of patient's quality of life, especially in children, is so important. exploring the quality of life (ql), we use different types of questionnaires to obtain a quantitative result, which we can interpret using a compatible key for it. pediatric asthma quality of life questionnaire with standardized activities (paqlq(s)) by juniper is a reliable measure of the influence of asthma on ql in children. the polish version of the questionnaire is a strict translation of the english one, however it is not completely validated, which in practice can evince in difficulties in understanding the meaning of the applied questions, probably because of the difference in the habits of both populations. the aim of the study is valuation of quality of life (ql) in children with asthma and analysis of the factors that can have influence on it. the clinical problems included into the aim are to evaluate: * the correlation between the ql and asthma control * the utility of the polish version of paqlq(s) by juniper, especially the understanding of each question and it's answers the results of this study can be very helpful in the future in developing a questionnaires to use in polish primary care, according to which we would be able to see whether the asthma in our patients is being well controlled.

Eligibility criteria:
inclusion criteria: * clinical diagnosis of asthma * asthma treatment at least 6 months before enrollment * must be able to do spirometry exclusion criteria: * children during the introductory period of the specific immunotherapy * other protracted diseases

Keywords: asthma control quality of life children polish version of paqlq(s) by juniper
study of gemcitabine chemoradiation and tnp-470 patients locally advanced, nonmetastatic adenocarcinoma of pancreas

Conditions: Pancreatic Neoplasms

Summary: the goal is to assess survival and patterns of failure in patients treated with gemzar-based chemoradiation and tnp-470 for locally advanced adenocarcinoma of the pancreas.


Eligibility criteria:
patients must have cytologic or histologic proof of adenocarcinoma of the pancreas. all patients must be staged with a physical exam and cxr. patients must have locally advanced disease defined as tumor extension to the celiac axis or sma, or occlusion of the smpv confluence (on contrast-enhanced ct). there must be no radiographic evidence of metastatic disease. patients must have had no prior chemotherapy or irradiation. patients must have a baseline karnofsky performance status of at least 60. patients must have adequate bone marrow reserve platelet count \>100,000/ml, hemoglobin\>9 g/dl, absolute granulocyte count must be \>1,500 cells/mm3,serum creatinine must be \<1.6 mg/dl. serum bilirubin must be less than 5 mg/dl prior to therapy with endoscopic or percutaneous drainage if necessary. patients must have estimated life expectancy of at least 12 weeks. patients must be at least 18 years of age. there will be no upper age restriction. female patients must have had childbearing potential terminated by surgery, radiation, or menopause, or attenuated by use of an approved contraceptive method (intrauterine device \[iud\], birth control pills, barrier device, or abstinence. patients cannot have hepatic or peritoneal metastases detected by ultrasound (us), ct scan, or laparotomy prior to chemoradiation. before receiving external beam irradiation, patients should be able to maintain adequate oral nutrition (90% to 100% of estimated need of calories and protein) and be free of significant nausea and vomiting. patients should have bilateral renal function, as determined on excretory urogram (ivp) or abdominal ct, or \>2/3 of one functioning kidney must be able to be shielded from the radiation beam. patients must have no fever or evidence of infection or other coexisting medical condition that would preclude protocol therapy. patients must sign an informed consent form

Keywords: metastatic pancreas cancer
evaluate the efficacy and safety of the prophylactic use of peg-rhg-csf in children with hematological malignancies

Conditions: Lymphoma; Leukemia

Summary: the incidence of infectious complications in hematological malignancies is higher than that in children with solid tumors, which may be related to the type and dose intensity of chemotherapy regimens used in hematological tumors. the treatment of childhood cancer has changed in the past few decades: intensive treatment and good supportive treatment can improve the 5-year survival rate of children. the aim of this study was to evaluate the efficacy and safety of prophylactic use of pegylated recombinant human granulocyte colony-stimulating factor (peg-rhg-csf) after chemotherapy in children with hematological malignancies.


Eligibility criteria:
inclusion criteria: 1. under the age of 18, no gender limit; 2. children with hematological malignancies, leukemia or lymphoma, diagnosed by bone marrow pathology or cytology; 3. the prophylactic use of peg-rhg-csf or rhg-csf after chemotherapy is intended to prevent neutropenia, and the chemotherapy regimen must meet the interval between two chemotherapy sessions at least 12 days; 4. the effect of chemotherapy in leukemia patients was complete remission, while that in lymphoma patients was complete remission or partial remission; 5. the expected survival time is more than 8 months; 6. liver and kidney function: liver function: total bilirubin (tbil), alanine aminotransferase (alt) and aspartate aminotransferase (ast) ≤ 3 times the upper limit of normal value, or≤5 times the upper limit of normal value when there is liver metastasis; renal function test: serum creatinine (cr) ≤ 1.5 times the upper limit of normal value; 7. eastern cooperative oncology group(ecog) performance status(ps) \<2; 8. the subjects had good mental consciousness, and the subject's legal guardian must sign an informed consent form; 9. researchers believe that the subject can benefit; exclusion criteria: 1. severe internal organ dysfunction; 2. those who used other test drugs of the same kind or accepted other clinical trials within 4 weeks before enrollment; 3. allergy to peg-rhg-csf, rhg-csf and other preparations or proteins expressed by escherichia coli; 4. researchers determine unsuited to participate in this trial.
effect of endoscopic papillary large balloon dilation on recurrent rate of patients with recurrentstones in bile duct

Conditions: Endoscopic Papillary Large Balloon Dilation

Summary: the recurrent rate of cbds in patients with recurrent cbds is high. it was reported that up to 60% of patients had stone recurrence within two years after stone retrieval by ercp. although eplbd is useful for for extraction of large cbds with less operation time and mechanical lithotripsy.it is not known whether eplbd could prevent the recurrence in patients with recurrent cbds.although harada et al found that eplbd might reduce the short-term recurrence of cbd stones in patients with previous es. it is a retrospective study with a small sample size (n=94). here a prospective, randomized controlled study including two tertiary centers was designed. the aim of this study was to investigate whether eplbd could reduce the recurrence rate in patients with recurrent cbds.


Eligibility criteria:
inclusion criteria: 1. age 18-80 2. patients with recurrent cbds after ercp exclusion criteria: 1. benign or malignant cbd stricture 2. recurrent stone within 3 months after ercp 3. previous endoscopic papillary large balloon dilation (eplbd) 4. prior surgery of bismuth ii and roux-en-y 5. septic shock 6. coagulopathy (inr\>1.3), platelet\<50000 or using anti-coagulation drugs 7. with expected life span less than 24 months 8. pregnant women 9. refusal or unable to give written informed consent
virtual agents-based digital interventions to improve health

Conditions: Mental Disorder; Mental Stress; Insomnia; Depression; Anxiety

Summary: kanopee is a free smartphone application providing screening, follow-up tools and autonomous digital interventions to lower psycho-social stress and its repercussions on sleep and behaviors in the general population. additionally, kanopee permits an adaptation of the intervention to the subject (i.e., an adapted waiting period, frequency, and content), enabling to perform innovative trial conditions. the objective of this long-term study is to evaluate the efficacy of kanopee on users among the french general population exposed to psycho-social stress.

kanopee was designed by sleep specialists and psychiatrists to propose autonomous interventions to improve sleep, physical activity and substance abuse ; aiming at lowering psycho-social stress's repercussions. the app provides weekly interactions with a virtual companion. at each interaction, users answer standardized questionnaires (e.g., insomnia severity index, cigarette dependence scale) enabling to characterize users' health status (i.e;, the severity of the complaints) and evolution over time, and therefore provide adapted interventions based on the literature (e.g., digital cognitive behavioral therapies, diaries, sleep hygiene recommendations, motivational interviewing). the app is freely available to the general population in france, enabling very large sample size, and the possibility to perform non-randomized trials depending on the selected interventions by the users and the subject's group.

Eligibility criteria:
inclusion criteria: * being aged 18 years old and over (the app is available for younger users but their data will not be analyzed) * having downloaded the app * having a smartphone android or iphone * being located in france * having accepted the electronic informed consent exclusion criteria: * having clicked on the "emergency button" (in the app, there is a warning message : this app is not for emergency situations. if you need an urgent psychological support, click here") * having downloaded the app but without answering any questionnaire

Keywords: e-health embodied conversational agent ecological momentary assessment ecological momentary intervention
novel pleural fluid, biopsy and serum biomarkers for the investigation of pleural effusions

Conditions: Respiratory Disease; Pleural Effusion; Lung Cancer

Summary: the purpose of this study is to prospectively asses established biomarkers in the diagnosis and prognosis of patients and will include assessment of a number of biomarkers, genomics and proteomics.

a prospective observation study examining the clinical utility of novel pleural fluid, biopsy and serum biomarkers for the investigation of pleural effusions (invest). the invest study will collect blood, pleural fluid and tissue samples from donors, allowing the opportunity to apply several novel blood and pleural fluid tests with interpretation of results in the context of clinical data and diagnostic suspicion. through the prospective application of multiple tests, we aim to validate the sensitivity and specificity of existing biomarkers and identify novel markers that aid in the diagnostic pathway of pleural diseases. because samples and data will be stored for future use, many other different tests, methods and techniques may be used in the future to provide information that we cannot foresee.

Eligibility criteria:
inclusion criteria: * undiagnosed pleural effusion or pleural thickening requiring investigation by one of the following: * pleural aspiration * pleural biopsies from medical thoracoscopy or * ultrasound guided biopsies as part of the clinical plan * suspected lung cancer undergoing bronchoscopy and biopsies. exclusion criteria: * inability to give written informed consent * inability to obtain pleural fluid, blood, endobronchial or pleural biopsies as applicable

Keywords: respiratory pleural effusion
a bioavailability study of sebacoyl dinalbuphine ester im injection in healthy volunteers

Conditions: Pain; Analgesia Disorder

Summary: this study is to evaluate the relative bioavailability of nalbuphine after single im injection of sebacoyl dinalbuphine ester (sde) injection and bain®, nalbuphine hcl im injection in healthy volunteers.


Eligibility criteria:
inclusion criteria: 1. subjects must be male or female adults in good health on the basis of medical history, physical examination, electrocardiogram, chest x-ray, and routine laboratory evaluations. 2. vital signs (after 3 minutes resting in a upright position) which are within the following ranges: ear body temperature between 35.0-37.5°c. systolic blood pressure, 90-140 mm hg. diastolic blood pressure, 50-90 mm hg. pulse rate, 50-90 bpm. fasting blood glucose, \< 110 mg/dl. 3. within -20% to +20% of the ideal body weight. for male subjects, body weight must be above 50 kg and for female subjects, body weight must be above 45 kg. 4. able to sign informed consent prior to study. 5. able to communicate well with the investigator and comply with the requirements of the study. exclusion criteria: 1. use of any alcohol-containing products, prescription medications or over-the-counter, non-prescription preparations (including herbal preparations) within 2 weeks prior to study entry unless deemed acceptable by the investigator (except up to 5 doses of ≤ 1000 mg of acetaminophen or ≤ 400 mg ibuprofen within this 2 week period). 2. alcohol or caffeine ingested within 48 hours prior to dosing. 3. significant illness within 2 weeks prior to dosing. 4. participation in any clinical investigation within 2 months prior to dosing or longer as required by local regulation. 5. donation or loss of more than 500 ml of blood within 3 months prior to dosing. donation or loss of more than 250 ml of blood within 2 months prior to dosing. 6. presence of cardiovascular disease. 7. presence of gastrointestinal disease. 8. presence of asthma or lung disease. 9. presence of liver disease or liver injury as indicated by an abnormal liver function profile such as ast, alt, gamma-gt, alkaline phosphatase, or total bilirubin. (value of ast or alt above 3 times of the upper limit of the normal range; other items clinically significant abnormality judged by investigator). 10. presence of impaired renal function as indicated by abnormal creatinine or bun values or abnormal urinary constituents. (value of creatinine or bun beyond the range from -20% of the lower limit of the normal range to +20% of the upper limit of the normal range; other items clinically significant abnormality judged by investigator) 11. presence of neurological disease. 12. presence of psychiatric disease. 13. subject is positive for hiv immunology test. 14. a known hypersensitivity to nalbuphine or its analogs. 15. history of drug or alcohol abuse within 12 months prior to dosing. 16. permanent confinement to an institution. 17. pregnant or lactating women. 18. individuals are judged by the investigator or pharmacokineticist to be undesirable as subjects for other reasons

Keywords: nalbuphine sde dinalbuphine sdn pain lt1001
application of patient blood management in the oncology patient affected by breast cancer

Conditions: Breast Cancer

Summary: evaluation of the percentage of transfusions in patients with breast cancer undergoing surgery after preventive treatment with haematinics.

preoperative anemia treatment is fundamental in patient blood management (pbm) programs, a multidisciplinary and multimodal strategy that improves clinical outcomes based on the patient's blood resource, promoting strategies to optimize hematopoiesis in candidates for elective surgery, in order to significantly reduce the use of blood products, addressing all modifiable transfusion risk factors before it is even necessary to consider the use of transfusion therapy itself. treatment with intravenous iron reduces the transfusion risk and consequently the adverse events related to the transfusion itself. nothing is specified in this regard for cancer patients; very often these patients come to surgery presenting an anaemic state that often requires correction with red blood cell transfusion. the aim of this clinical study is to prevent the number of perioperative transfusions in patients with breast cancer who have undergone or not undergone neoadjuvant chemotherapy and with hb values lower than or equal to 11 g/dl, who are candidates for destructive and/or reconstructive surgery.

Eligibility criteria:
inclusion criteria: * age \> 18 years; * patients diagnosed with histologically confirmed breast cancer and whether or not they have undergone neoadjuvant chemotherapy; * patients who are candidates for destructive and reconstructive breast surgery or not; * hb values less than or equal to 11 g/dl and/or transferrin saturation less than or equal to 20%; * written informed consent. exclusion criteria: * patients with known allergies to ferric carboxymaltose or its excipients; * ferritin values greater than 500 ng/ml; * patients unable to sign consent and comply with procedures.