Kornimate/medical-research-clean huggingface dataset:
somatic mosaicism in twins discordant for childhood cancer

Conditions: Pediatric Central Nervous System Tumor; Central Nervous System Tumor; Discordant Twin; Somatic Mutation

Summary: somatic mosaicism in cancer associated genes is one potential explanation for discordance in childhood cancer that has not been fully explored to date. this pilot study will focus on twins with central nervous system (cns) tumors who are identified through the children's oncology group's project: everychild (pec) registry or volunteer.

primary objectives: i. to conduct a descriptive study of same sex twins (mz and dz) discordant for childhood cns tumors identified through the children's oncology group project:everychild registry. ii. to compare the prevalence of somatic mosaicism in classic cancer-associated genes in monozygotic (mz) twins discordant for childhood brain tumors. in our pilot study, we will sequence blood and saliva dna samples from 25 mz twin pairs using a panel of 94 known cancer associated genes. secondary objectives: i. to compare the profile of mutations detected in dna extracted from pre- and post-treatment blood samples from a subset of cns tumor patients. the purpose of this aim is to ensure that differences in somatic mosaicism detected in twin pairs are not caused by cancer therapy. outline: twins with cns tumors will undergo blood and saliva dna samples with the goal is to learn more about genetic differences that may lead to cns tumor development.

Eligibility criteria:
inclusion criteria: * newly diagnosed patient with cns tumor or have recurrent disease and enrolled on apec14b1. * patient must have same sex twin. note: (history of) treatment on a cog therapeutic trial is not required. * patients must be diagnosed at \< 19 years of age at the time of diagnosis. * a family is eligible to participate if the twin with the cns tumor is deceased but has a blood sample banked through apec14b1. * all patients and/or their parents or legal guardians must provide informed consent. assent will be obtained for participants between the ages of 8-17 years. * all institutional, fda, and nci requirements for human studies must be met.
a study of the oral egfr inhibitor plb1004 in non-small cell lung cancer

Conditions: Non-Small Cell Lung Cancer

Summary: this is a phase i, multicenter, open-label, dose escalation and dose expansion study to assess the safety and pharmacokinetic profile of plb1004, a mono-anilino-pyrimidine compound, given alone in nsclc patients with egfr exon 20 insertion mutations，uncommon mutations, classical mutations (ex19del and l858r), and drug-resistant mutation (t790m). patients will be enrolled and dosed according to the most current protocol. this study is made of two parts. part 1 includes a dose escalation into 7 cohorts and patient allocation to these cohorts will be via slot allocation. each cohort has a minimum of 3 and a maximum of 6 patients for a total of 21 - 42 patients. the patient population of the dose escalation phase will include patients with advanced nsclc harboring egfr classical mutations or ex20ins mutations, or uncommon mutations. part 2 includes an expansion phase and the expansion phase will explore one or more dose levels of plb1004 in nsclc patients with egfr ex20ins mutations, classical mutations, or uncommon mutations.


Eligibility criteria:
inclusion criteria: 1. ability to understand and willingness to sign a written informed consent document; 2. male or female adult patients 18 years of age or older; 3. patients should have recovered from toxicities related to prior anti-tumor therapy; 4. patients should have recovered from the effects of major surgery; 5. have a documented egfr mutation by a local test in tissue or plasma; 6. at least 12 weeks life expectancy; 7. must have at least one measurable lesion per recist v 1.1; 8. sexually active males and females of childbearing potential must agree to take effective contraceptive measures. exclusion criteria: 1. received radiotherapy within 14 days before enrollment; 2. have significant or uncontrolled systemic disease; 3. have significant or uncontrolled cardiovascular disease; 4. have had other diagnosed malignant diseases that required treatment within the past 3 years besides nsclc; 5. currently have or had a history of interstitial lung disease, drug-induced interstitial lung disease, or radiation pneumonia that requires steroid therapy; 6. have known hypersensitivity to the similar drugs and excipients of plb1004; 7. pregnant or lactating women; 8. have used other experimental drugs within 2 weeks prior to the first dose of plb1004; 9. have any condition or illness that could affect the compliance with the protocol.
study of the safety of orencia in japanese children and adolescents with active juvenile arthritis of unknown origin

Conditions: Undifferentiated Arthritis

Summary: observational study of abatacept in the treatment of jia in japan.


Eligibility criteria:
inclusion criteria: * all jia participants who initiate treatment with orencia in accordance with the prescribing information exclusion criteria: * participants receiving orencia for an off-label indication other protocol defined inclusion/exclusion criteria could apply
urine output response to fluid and diuretic therapy in cardiac icu patients monitored with fize kuo®

Conditions: AKI - Acute Kidney Injury; Cardiac Surgery; Fluid Therapy; Hemodynamic Instability; Postoperative Complications

Summary: this prospective observational study evaluates the real-time urine output response to fluid therapy and diuretics in cardiothoracic intensive care unit (cticu) patients monitored with the fize kuo® device. the study aims to assess how continuous urine output monitoring can inform fluid management and improve patient outcomes post-cardiac surgery.

the study involves adult patients undergoing coronary artery bypass graft (cabg) or valve replacement/repair surgeries. utilizing the fize kuo® device, continuous urine output data will be collected to analyze responses to hemodynamic interventions, including fluid boluses and diuretics. secondary objectives include assessing urine output differences in patients with and without postoperative acute kidney injury (aki), responses to vasopressors, and capturing real-time urine output data from fize kuo® for observational analysis.

Eligibility criteria:
inclusion criteria: 1. adults aged 18 years and older scheduled for cabg or valve surgery and expected postoperative admission to cticu 2. ability to provide informed consent exclusion criteria: 1. pregnancy 2. preoperative dialysis (chronic or acute) 3. enrollment in other interventional trials assessing urine output or aki

Keywords: fize kuo urine output fluid management diuretics cardiothoracic surgery
abraxane combined with xeloda as neo-adjuvant chemotherapy in early breast cancer

Conditions: Breast Cancer

Summary: some studies have demonstrated the efficacy of abraxane in treating breast cancer in both adjuvant and metastatic settings. few data is available on abraxane in neoadjuvant treatment for breast cancer. the aim of this project is to evaluate tailored primary systemic therapy with weekly abraxane (125mg/m2) combined with q3week xeloda (1250mg/m2) in early operable (stage of t2-4n0-3m0) breast cancer. this study will be an open label non randomized, single arm, single center study.

the hypothesis is that tailored neoadjuvant chemotherapy with 4 cycles of px(weekly abraxane 125mg/m2, q3week xeloda 1250mg/m2, 21day/cycle) is feasible and achieves high response rates. it is proposed that 50 patients will be enrolled in this study. the target population is women with early breast cancer (stage of t2-4n0-3m0) who are eligible for primary systemic therapy. the primary objective of the trial is to determine the overall response rate. secondary endpoints will include response rates in axillary lymph nodes, safety and tolerability and the rate of breast conservation.

Eligibility criteria:
inclusion criteria: 1. provision of informed consent 2. women and men at least 18 years of age or older. 3. pathological confirmation of breast cancer 4. tumor stage(tnm):t2-4n0-3m0 5. no evidence of distant metastasis 6. adequate bone marrow, hepatic, and renal function 7. measurable disease as per recist criteria 8. karnofsky≥70 9. laboratory criteria: plt≥100\*109/l wbc≥4000/mm3 hgb≥10g/dl alt and ast\<2\*uln exclusion criteria: 1. presence of metastatic disease. 2. inflammatory breast cancer. 3. bilateral breast cancer. 4. previous chemotherapy or hormonal therapy for current breast neoplasm. 5. other malignant tumors (concurrent or previous). 6. pregnant woman. 7. hypersensitive to any drug in abraxane or xeloda regimen or any ingredient of abraxane or xeloda. 8. any severe systemic disease contraindicating chemotherapy

Keywords: neoadjuvant chemotherapy abraxane xeloda
mothif ii : therapeutic management and use of clotting factors in hemophilia a & b in france ii

Conditions: Hemophilia

Summary: mothif ii is a non-interventional, multicenter, retrospective, observational data collection in seven french haemophilia treatment centers of the berhlingo network. in the context of the arrival of new extended half-life products, the mothif ii study aims to describe the changes in therapeutic management of patients with hemophilia a \& b, following the provision of fviii and fix extended half-life factors in france; it will also permit to carry out a budget impact analysis to quantify the economic significance of this new era.

treatment regimens for haemophiliacs patients treated with clotting factors will be analysed globally for the patients of the two periods, by subgroup: * type of hemophilia: a or b * severity: severe, moderate, mild * history of inhibitors and then for each subgroup: * by regimen: 1) prophylaxis 2) on demand 3) iti * by type of clotting factors: fviii or fix or by-passing agents, plasma-derived or recombinant, standard or extended half-life clotting factors, inn will also be included in the analyse: * demographic data: age (years) * clinical data: weight (kgs) if available * treatment data: regimen, consumptions (number of ui of clotting factors consumed on the period) and costs of clotting factors (€).

Eligibility criteria:
inclusion criteria: * all patients hemophiliac a or b, with or without inhibitors, included in the nhemo (7 centres) and the behrlingo database, treated with factor 8 or factor 9 products or by-passing agents * all hemophilia a or b patients of any severity without inhibitors, included in the nhemo database and the behrlingo database, without any treatment by clotting factors * all hemophilia a or b patients having given consent to be included in the study (2 times 12 months), during their usual follow-up (consultation...). for the other patients, a derogation from the obligation to inform the patients will be made to the cnil. exclusion criteria: * patients under guardianship

Keywords: hemophilia a hemophilia b research database clotting factors regimen of treatment impact budget analysis
study of pathways curriculum for use with wpd

Conditions: Depression

Summary: study to assess the efficacy of a modified pathways curriculum in treating depressive symptoms in women with physical disabilities.


Eligibility criteria:
inclusion criteria: * women with physical disability requiring accommodation for major mobility limitations * ces-d score of 16 or higher - exclusion criteria: * active suicidality * diagnosis of schizophrenia, schizoaffective d/o, psychosis nos * currently receiving other psychotherapy -
safety and efficacy of tsha-102 in adolescent and adult females with rett syndrome (reveal adult study)

Conditions: Rett Syndrome

Summary: the reveal adult study is a multi-center, phase 1/2 open-label, dose-escalation study of tsha-102, an investigational gene therapy, in adult females with rett syndrome. the safety, tolerability, and preliminary efficacy of two dose levels will be evaluated. the study duration is estimated to be up to 63 months.


Eligibility criteria:
inclusion criteria: * participant has a clinical diagnosis of classical/typical rett syndrome with a documented pathogenic mutation of the methyl-cpg-binding protein 2 (mecp2) gene that results in loss of function. * participants must be willing to receive blood or blood products for the treatment of an ae if medically needed. exclusion criteria: * participant has another neurodevelopmental disorder independent of the mecp2 loss-of-function mutation, or any other genetic syndrome with a progressive course. * participant has a history of brain injury that causes neurological problems. * participant had grossly abnormal psychomotor development in the first 6 months of life. * participant has a diagnosis of atypical rett syndrome. * participant has a mecp2 mutation that does not cause rett syndrome. * participant requires invasive ventilatory support. * participant has contraindications for it administration of tsha-102 or lumbar puncture procedure, or other medical conditions, or contraindications to any medications required for it administration. * participant has uncontrolled seizures or a history of status epilepticus within the 3 months prior to enrollment.

Keywords: rett syndrome neurodevelopmental disorder rett mecp2
cervical spine clearance in obtunded trauma patients

Conditions: Coma; Wounds and Injuries

Summary: the optimal method of clearing the cervical spine in obtunded trauma patients is unclear. computed tomography (ct) identifies most injuries but may miss ligamentous injuries. magnetic resonance (mr) imaging has been widely used to exclude ligamentous injuries. we postulate that with the new generation of ct scanners, mr imaging is not needed to rule out significant injuries. our protocol for clearing the cervical spine in obtunded trauma patients depends on ct alone. we are prospectively following these patients and performing a clinical examination when they are awake to show this is a safe approach.

all obtps with gross movement of all four extremities admitted to metrohealth medical center (mhmc), the regional level i trauma center in cleveland oh, who underwent a ct cs at mhmc between october 2006 and september 2008 will be included. prospective data will be collected on these patients including age, gender, mechanism of injury, glasgow coma scale (gcs) on admission, results of ct cs, gcs at the time of collar clearance, day of collar clearance, collar complications, date of reexamination, and results of reexamination. we will define an obtunded patient as a patient who was unable to reliably describe the presence or absence of cs symptoms in the opinion of the examining physician. patients needed to have gross movement of all four extremities to be included in the study; if they did not, they were excluded. the presence or lack of sensory examination was not evaluated for the purposes of this study. we will define cervical collar complications as skin breakdown identified either by the skin care team during weekly rounds in the intensive care unit or by the treating physician at any time.

Eligibility criteria:
inclusion criteria: * subjects will include all patients (including children) after blunt trauma in whom cervical spine injury has not been ruled out admitted to metrohealth medical center from 10/1/2004-9/30/2006. exclusion criteria: * we are excluding patients with an obvious neurologic deficit attributable to the cervical spine. * we are excluding patients with abnormalities of the cervical spine that are identified on either plain films or ct. * we are also excluding those patients in whom the cervical spine can be cleared clinically due to normal mental status.

Keywords: obtunded blunt trauma patients
caffeic acid combining high-dose dexamethasone in management of itp

Conditions: Immune Thrombocytopenia

Summary: the project was undertaking by qilu hospital of shandong university in china. in order to report the efficacy and safety of caffeic acid tablets combining with high-dose dexamethasone for the treatment of adults with newly-diagnosed primary immune thrombocytopenia (itp).

the investigators are undertaking a multicenter, randomized, double blinded placebo-controlled trial of 240 primary itp adult patients from 8 medical centers in china. one part of the participants are randomly selected to receive caffeic acid tablets (given orally at a dose of 900mg per day for 3 consecutive months), combining with dexamethasone (given orally at a dose of 40 mg per day for 4 days, two cycles with an interval of 10 days); the others are selected to receive high-dose of dexamethasone treatment plus placebo. platelet count, bleeding and other symptoms were evaluated before and after treatment. adverse events are also recorded throughout the study. in order to report the efficacy and safety of caffeic acid tablets combining with high-dose dexamethasone therapy for the treatment of adults with itp.

Eligibility criteria:
inclusion criteria: * 1.meet the diagnostic criteria for immune thrombocytopenia; 2.untreated hospitalized patients, may be male or female, between the ages of 18 \~ 80 years; 3.to show a platelet count \<30 \* 10\^9/l, and with bleeding manifestations; 4.willing and able to sign written informed consent exclusion criteria: * 1.received chemotherapy or anticoagulants or other drugs affecting the platelet counts within 3 months before the screening visit 2.received second-line itp-specific treatments (eg, cyclophosphamide, 6-mercaptopurine, vincristine, vinblastine, etc) within 3 months before the screening visit 3.received high-dose steroids or ivig in the 3 weeks prior to the start of the study 4.current hiv infection or hepatitis b virus or hepatitis c virus infections 5.severe medical condition (lung, hepatic or renal disorder) other than chronic itp. unstable or uncontrolled disease or condition related to or impacting cardiac function (e.g., unstable angina, congestive heart failure, uncontrolled hypertension or cardiac arrhythmia) 6.female patients who are nursing or pregnant, who may be pregnant, or who contemplate pregnancy during the study period 7.have a known diagnosis of other autoimmune diseases, established in the medical history and laboratory findings with positive results for the determination of antinuclear antibodies, anti-cardiolipin antibodies, lupus anticoagulant or direct coombs test 8.patients who are deemed unsuitable for the study by the investigator

Keywords: itp caffeic acid dexamethasone
the characteristics and role of mucosal microbiome after treatment of clostridioides difficile infection

Conditions: Clostridioides Difficile Infection

Summary: this research is being done to study the ability of c. difficile to colonize the colonic mucosa of individuals with no prior history of c. difficile infection.

the ability of c. difficile to colonize the mucosa of the colon will be studied in healthy individuals with no history of c. difficile infection who are undergoing colonoscopy for colon cancer/polyp screening purposes.

Eligibility criteria:
inclusion criteria: * adults aged 18 and over * no history of c. difficile infection * undergoing clinically indicated colonoscopy for colon cancer screening or colon polyp surveillance purposes in the absence of active gi symptoms. exclusion criteria: * known active pregnancy * other known active gastrointestinal infectious process * inflammatory gastrointestinal conditions (microscopic colitis, inflammatory bowel disease, celiac disease) * vulnerable adults * any other disease(s), condition(s) or habit(s) that would interfere with completion of study, or in the judgment of the investigator would potentially interfere with compliance to this study or would adversely affect study outcomes
pulsed radiofrquency targeting mid cervical medial branches versus gon for cervicogenic headache

Conditions: Cervicogenic Headache

Summary: to detect the clinical efficacy of pulsed radiofrequency treatment targeting the mid cervical medial branches versus greater occipital nerve for cervicogenic headache

cervicogenic headache (cha) is one of the secondary headache disorders. the prevalence of cha was estimated to be \~4.1%, and most of the patients affected by cha report un-resolved, recurrent throbbing pain. it has been treated with many treatment modalities, such as the administration of medi-cines, physiotherapy, transcutaneous electrical nerve stimulation, and interventional pro-cedures. however, these treatments do not result in long-term relief for many patients and need to be repeated pulsed radiofrequency treatment (prf) is one of the modalities used to treat cha . cha has been known to originate from the convergence of the 3 upper cervical and tri-geminal afferents, and therefore, many physicians have performed prf targeting the upper cervical structures (occipital nerve, c2 dorsal root ganglion). however, this results in only short-term pain relief in the posterior head, and it can lead to some complications, such as vascular and nerve injuries. because of these limitations, we will attempt prf targeting the mid-cervical medial branches

Eligibility criteria:
inclusion criteria: 1. medically stable outpatients with confirmed diagnosis of cervicogenic headache according to international headache society 2. are reliable and willing to make themselves available for the duration of the study and are willing to follow up. 3. men or women older than 18years of age . 4. clear written informed consent from each participant in the trial. exclusion criteria: 1)pregnants, breastfeeding, or willing to be pregnant during the study. 2)presence of clinically significant medical or psychiatric condition that may increase the risk associated with the study 3)participation in any other type of medical research that may interfere with the interpretation of the study. 4)patients with haemocoagulation disorders, local infection or those who refused to consent
a study of the effectiveness of venetoclax tablets in adult acute myeloid leukemia participants ineligible for standard induction therapy in russian federation

Conditions: Acute Myeloid Leukemia (AML)

Summary: acute myeloid leukemia (aml) is a cancer of the blood and bone marrow and is the most common acute leukemia in adults. this study will evaluate how well venetoclax works to treat aml in adult participants who are ineligible for standard induction therapy in russian federation. venetoclax is a drug approved to treat acute myeloid leukemia. all study participants will receive venetoclax as prescribed by their study doctor in accordance with approved local label. adult participants with a diagnosis of aml who are ineligible for standard induction therapy will be enrolled. around 50 participants will be enrolled in the study in approximately 15 sites in russian federation. participants will receive venetoclax tablets to be taken by mouth daily according to the approved local label. the duration of the study is approximately 18 months. there is expected to be no additional burden for participants in this trial. all study visits will occur during routine clinical practice (approximately every 3 months) and participants will be followed for 18 months.


Eligibility criteria:
inclusion criteria: * newly diagnosed acute myeloid leukemia (aml) (de novo or secondary) and eligible to receive venetoclax as per russian ministry of health label * ineligible for standard induction therapy with cytarabine and anthracycline * eastern cooperative oncology group (ecog) score \>2 * white blood cell counts \<25 x 109 per liter * started venetoclax within 4 weeks of enrolling in study exclusion criteria: * acute promyelocytic leukemia * contraindications to venetoclax as listed on the approved local label in russian federation * neuroleukemia - active central nervous system (cns) involvement * participation in a clinical trial with an investigational drug for aml within 30 days prior to venetoclax treatment initiation

Keywords: acute myeloid leukemia (aml) venetoclax venclexta