Kornimate/medical-research-clean huggingface dataset:
diagnosis of mucopolysaccharidosis disorders in patients presenting with bilateral hip disease

Conditions: Mucopolysaccharidosis IV A; Mucopolysaccharidosis VI

Summary: background/objective: quantitative urine screening for mucopolysaccharides (mps) has been the primary method for detecting mucopolysaccharidoses in children. this method may not be sufficiently sensitive and may miss some patients with arylsulfatase b (arsb) deficiency. investigators propose to identify patients retrospectively and prospectively who carry a diagnosis of spondyloepiphyseal dysplasia, multiple epiphyseal dysplasia, bilateral proximal femoral epiphyseal dysplasia, or bilateral legg-calve-perthes. for these patients, investigators will perform enzyme testing on a blood sample which will identify mps vi or iva. patients who have an earlier diagnosis of mps are likely to have better health outcomes with medical management. therefore, it is important to determine effective diagnostic methods. investigators believe that bilateral hip involvement should alert the clinician to the possibility of mps vi and further examination. the purpose of this study is to test the hypothesis that the correct diagnoses of two mps storage disorders are delayed in patients with bilateral proximal femoral epiphyseal dysplasia and normal quantitative urine mps studies.


Eligibility criteria:
inclusion criteria: * less than or equal to 21 years of age * diagnosis of spondyloepiphyseal dysplasia or multiple epiphyseal dysplasia or bilateral legg-calve-perthes disease or bilateral proximal femoral epiphyseal dysplasia. exclusion criteria: * definitive etiology for above-mentioned diagnosis (i.e. other mps disease, known chondrodysplasia, meyer's dysplasia)

Keywords: spondyloepiphyseal dysplasia multiple epiphyseal dysplasia bilateral legg calve perthes bilateral proximal femoral epiphyseal dysplasia galns
efficacy of coenzyme q10 in pediatrics with type 1 diabetes mellitus

Conditions: Diabetes Mellitus, Type 1

Summary: the purpose of this study is to determine wether coenzyme q10 is effective in reducing markers of endothelial dysfunction in pediatric patients with type 1 diabetes mellitus and aslo to investigate its effect on glycemic control and lipid profile of those patients and its effect on patient's quality of life.


Eligibility criteria:
inclusion criteria: 1. age: patients from 8 to 18 years old. 2. outpatient with at least 1 year history of type 1 diabetes mellitus. 3. insulin requirement of more than or equal 0.5 u/kg/day. 4. approval to participate and give informed consent. exclusion criteria: 1. presence of systemic disorders such as celiac disease, hypothyroidism or hyperthyroidism. 2. preexisting cardiovascular disease or hypertension. 3. chronic kidney disease or chronic liver disease. 4. significant mental illness. 5. intake of other antioxidants such as ascorbic acid and α-tocopherol and omega3 supplement within the last 3 months. 6. intake of coenzyme q10 within the last 3 months.

Keywords: type 1 diabetes mellitus coenzyme q10 endothelial dysfunction pediatric patients
sympathetic activity in post-injury outcomes: impact on sleep and cardiovascular health investigation

Conditions: Hypertension; Cardiovascular Diseases; Sleep Disorder; Arrythmia; Sympathetic Nervous System; Injury Traumatic

Summary: the goal of this observational study is to learn about the long-term health of united states military service members who were injured during combat. the main questions it aims to examine are: * how does the severity of a combat injury impact 1) cardiovascular risk, 2) the sympathetic nervous system and arrhythmias, 3) blood pressure, and 4) sleep disorders? * are self-reported mental health symptoms related to sympathetic nervous system hyperactivity, sleep disorders, and cardiovascular risk in combat-injured service members? this study will recruit from a sample of participants in another research study called the wounded warrior recovery project (wwrp) who 1) agreed to be contacted about future research studies and 2) have a record of a combat injury within the injury severity score ranges required for this study. participants will: * provide demographic information and a medical history review * visit a local laboratory for biometrics measurements and to provide blood and urine samples * wear an ambulatory electrocardiogram monitor for 24 hours per day for seven consecutive days * wear a home sleep test monitoring device for one night * wear a blood pressure monitor for 24 consecutive hours on the day after the home sleep test at the end of the study, participants will be asked to mail back the home sleep test and blood pressure monitors. prepaid package materials will be provided.


Eligibility criteria:
inclusion criteria: * enrolled in the wounded warrior recovery project (wwrp) and previously agreed to be contacted regarding future research * able to complete the informed consent process, be mailed study devices, and complete laboratory draws within the united states * age greater than or equal to 18 years exclusion criteria: * no injury severity score or 3 \< injury severity score \< 15 * unable to complete the informed consent process, be mailed study devices, and complete laboratory draws within the united states * not enrolled in the wwrp or enrolled in the wwrp but did not agree to be contacted about future research
enhancing graft vs leukemia via delayed ex-vivo co-stimulated dli after non-myeloablative stem cell transplantation

Conditions: Acute Myelogenous Leukemia; Acute Lymphoblastic Leukemia; Myelodysplastic Syndrome

Summary: this is a new platform in non-myeloablative allogeneic stem cell transplantation to improve survival by harnessing the immunologic potential of donor t-cells to induce and maintain long-term remissions in patients with hematologic malignancies without undue toxicity. this study involves is the first study in humans directed at optimizing the graft vs leukemia effect by infusing activated t-cells from healthy donors prophylactically, months after recovery from the initial transplant. investigators are studying whether the activation of donor cells prior to infusion will enhance the patient's ability to "seek and destroy" residual malignant cells while also helping the immune system to fight infection without increasing the immune reaction against the host.


Eligibility criteria:
patient-related parameters: * patients must have a healthy histocompatible donor (a, b and dr match); either sibling or unrelated volunteer identified through the nmdp * age between 18 and 70 years old * life expectancy greater than 3 months. * ecog performance status 0-1. * patients must have acceptable organ function: * total bilirubin \<2.0 * ast and alt \< 3 x normal, unless increases are thought to be either from non-hepatic causes (i.e hemolysis) or related to underlying disease (such as liver involvement with leukemia); * creatinine \ 40 ml/min (calculated or collected); * cardiac: an ejection fraction \>40% on muga or echocardiogram; * pulmonary: corrected dlco \>50% exclusion criteria: subjects: * patients who have had chemotherapy or radiotherapy within 4 weeks prior to entering the study. patients must have recovered from adverse events due to agents administered more than 4 weeks earlier. * patients with uncontrolled or untreated central nervous system involvement * patients with uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements. * hiv positive patients are excluded * pregnant women * patients who are breastfeeding donors: * sibling donors will be evaluated according to the standard practice of the university of pennsylvania bone marrow and stem cell transplant program * unrelated donor evaluations and consent will be performed by an nmdp donor center according to standard guidelines and procedures.

Keywords: aml all mds allogeneic transplant acute myelogenous leukemia acute lymphoblastic leukemia myelodysplastic syndrome adoptive cell therapy non-myeloablative
study of et140202 t cells in adults with advanced hepatocellular carcinoma

Conditions: Hepatocellular Carcinoma; Liver Cancer; Liver Neoplasm; Metastatic Liver Cancer

Summary: this is a open-label, dose escalation, multi-center, phase i / phase ii study to assess the safety of an autologous t-cell product (et140202) in adult subjects with advanced alpha-fetoprotein (afp) positive/human leukocyte antigen (hla) a-2 positive hepatocellular carcinoma (hcc).

the purpose of this study is to investigate a genetically modified autologous t-cell therapy for advanced hepatocellular carcinoma (hcc). et140202 t cells are autologous t cells genetically modified to carry a tcr-mimic (tcrm) construct capable of mediating cell killing by targeting tumor specific intracellular antigens and addressing solid tumor therapy challenges.

Eligibility criteria:
inclusion criteria: * signed written informed consent obtained prior to study procedures * histologically confirmed hcc with serum afp \>200ng/ml at time of screening and following most current line of therapy or radiographic diagnosis of hcc with serum afp \>400ng/ml at time of screening and following most current line of therapy.. * metastatic or locally advanced, unresectable hcc * must have failed or not tolerated, at least one line of systemic therapy for advanced hcc * molecular human leukocyte antigen ("hla") class i allele typing confirms participant carries at least one hla-a2 allele * life expectancy of at least 4 months * karnofsky performance scale greater than or equal to 70 * at least 1 measurable lesion on imaging by recist * child-pugh a or b7 * absolute neutrophil count greater than or equal to 1,500/mm\^3 * platelet count greater than or equal to 30,000/mm\^3 exclusion criteria: * clinically significant cardiac disease * clinically significant pre-existing illness or active infection * clinically significant central nervous system (cns) or neural dysfunction * active autoimmune disease requiring therapy * active malignancy other than hcc unless expected survival is greater than or equal to three years without any treatment (exception: hormone/androgen-depravation therapy) and without any organ involvement * history of organ transplant * compromised circulation in portal vein, hepatic vein, or vena cava due to obstruction * advanced hcc involving greater than one-third of the liver

Keywords: hepatocellular carcinoma hcc advanced hcc late-stage hcc liver cancer liver neoplasm metastatic liver cancer metastatic hcc t-cell therapy immunotherapy
symptomatic study investigating degarelix in patients suffering from prostate cancer

Conditions: Prostate Cancer

Summary: the purpose of this trial was to see how well a new trial drug (degarelix) worked on lower urinary tract symptoms (also known as luts) in prostate cancer patients as compared to how a standard drug hormonal treatment worked on the same symptoms. the advancement/worsening of prostate cancer may be associated with luts and the symptoms may impact the ability to urinate normally and thereby the quality of life for these patients. patients were randomly selected (like flipping a coin) to receive either degarelix or standard hormone therapy (combination of goserelin and bicalutamide) for a 3 month treatment period. during this period the relief of urinary symptoms was evaluated via a questionnaire filled in by patients and addressing the severity and frequency of their symptoms.


Eligibility criteria:
inclusion criteria: * patient has given written informed consent before any trial-related activity is performed * has a confirmed prostate cancer in which this type of treatment is needed. exclusion criteria: * previous treatment for prostate cancer * previous trans-urethral resection of the prostate * current use of 5-alpha reductase inhibitor or α-adrenoceptor antagonist. * patients in need of external beam radiotherapy to be started at the same time as hormone therapy * certain risk factors for abnormal heart rhythms/qt prolongation (corrected qt interval over 450 msec., torsades de pointes or use of certain medications with potential risk) * history of severe untreated asthma, anaphylactic reactions, or severe urticaria and/or angioedema. * hypersensitivity towards any component of the investigational product * other previous cancers within the last five years with the exception of prostate cancer and some types of skin cancer. * clinical disorders other than prostate cancer including but not limited to renal, haematological, gastrointestinal, endocrine, cardiac, neurological, psychiatric disease, alcohol or drug abuse or other conditionals as judged by the investigator.
effects of intensive systolic blood pressure lowering treatment on out-of-office blood pressure - an ancillary study to effects of intensive systolic blood pressure lowering treatment in reducing risk of vascular events (esprit) study

Conditions: Blood Pressure

Summary: this study aims to evaluate the effect of intensive treatment (a target clinic based systolic blood pressure(bp) \<120mmhg) on 24-hour ambulatory blood pressure and home blood pressure, as well as the effect on white-coat uncontrolled hypertension and masked uncontrolled hypertension, compared with standard treatment (a target clinic based systolic bp \<140mmhg).

this study aims to evaluate the effect of intensive treatment (a target clinic based systolic bp \<120mmhg) on 24-hour ambulatory blood pressure and home blood pressure, as well as the effect on white-coat uncontrolled hypertension and masked uncontrolled hypertension, compared with standard treatment (a target clinic based systolic bp \<140mmhg). the population of this study is from an ongoing multi-centre open label clinical trial which evaluated the effect of intensive systolic hypertension treatment on major cardiac events (esprit study). esprit study enrolled patients with high risk of cardiovascular disease, who were aged≥50 years old with a systolic bp≥130 mmhg, and assigned them randomly to intense hypertension group (a target clinic based systolic bp \<120mmhg) or standard hypertension group (a target clinic based systolic bp \<140mmhg), with a 3-year follow-up, evaluating the effect of intense hypertension treatment on major cardiac events. from those eligible ones for the trial, the investigators plan to select 710 to participate in this study. during the 2-3 years of follow-up of the main trial, the participants will be monitored using 24-hour ambulatory blood pressure monitoring, meanwhile one-week home blood pressure monitoring will also be conducted. the two monitoring approaches will be performed twice; once every half year, based on which this study will evaluate the effect of intensive hypertension treatment on 24-hour ambulatory blood pressure and home blood pressure, as well as the effect on white-coat uncontrolled hypertension and masked uncontrolled hypertension.

Eligibility criteria:
inclusion criteria: 1. eligible for the follow up of the main study; 2. content to participate in the sub-study. exclusion criteria: 1. frequently working during night-time; 2. the non - dominant arm could not wear 24-hour ambulatory blood pressure monitoring due to radiation therapy or physical impairment; 3. keep engaged in out-door activities of high physical intensity, e.g. farming or porter, etc.; 4. life expectancy is less than 1 year; such as diagnosed with heart failure, cancer, or end-stage renal disease; 5. the difference of clinical systolic pressure between upper arms was more than 10 mmhg; 6. unable to use smart-phone to upload family blood pressure monitoring data.

Keywords: intense pressure lowering treatment out-of-office blood pressure
the role of indocyanine green to identify sentinel lymph node in uterine cancer

Conditions: Endometrial Neoplasm

Summary: endometrial cancer is the most common gynecologic malignancy in developed countries and the second one in developing countries following cervical cancer. the primary treatment for endometrial cancer involves total hysterectomy and bilateral salpingo-oophorectomy, with adjuvant radiotherapy and/or chemotherapy reserved for patients with advanced disease or who have risk factors for relapse. the tumor is confined to the uterus in 85% of endometrial cancers. hence, it's controversial to perform systematic lymphadenectomy to all patients. the primary purpose of the present study is the to investigate the feasibility of sentinel lymph node determination in endometrial cancer patients operated via conventional laparoscopy. to determine sentinel lymph nodes cervical indocyanine green will be injected prior to the surgery. during the surgery using fluorescent imaging systems sentinel lymph nodes will be removed and examined by frozen section. afterwards, systematic pelvic lymphadenectomy will be performed. in case of type ii endometrial cancer, grade 3, metastatic sentinel lymph nodes or macroscopically enlarged paraaortic lymph nodes, paraaortic lymphadenectomy will also be performed. the secondary purpose of the study is to compare the conventional laparoscopy in sentinel lymph node mapping of endometrial cancer patients.


Eligibility criteria:
inclusion criteria: * pathologic diagnosis of endometrial cancer * clinical stage i or ii exclusion criteria: * contraindications for operation (hematological or coagulation disorders) * advanced stage * pregnancy or puerperium * fertility preserving surgery
timing of levodopa treatment in parkinson's disease

Conditions: Parkinson's Disease

Summary: the elldopa study is a controlled clinical trial in patients with newly diagnosed pd to determine the optimal timing and dosing with levodopa (sinemet or its generic equivalents).

the time to begin levodopa therapy has been controversial for many years, and yet every patient with pd, along with his/her treating doctor, needs to make this decision. one school of thought is that levodopa may lead to developing motor fluctuations and involuntary movements, and therefore its introduction should be delayed. the opposing school of thought argues that it is the worsening severity of the disease over time that makes the patient susceptible to these problems, and argues that the best response to levodopa is in the early stages of the illness when an improved quality of life can be optimized with levodopa. another debated issue is whether levodopa offers protection or is harmful to the remaining dopamine neurons. the latest studies in tissue culture show that when glia (the brain's supportive cells) tissue is present in addition to the nerve cells, the glia tissue becomes protective against any levodopa toxicity. because glia tissue is present in brain, the argument has been made that levodopa should not be toxic in living brain tissue. a few studies have been carried out in animal models of pd. two of these animal studies suggest levodopa is toxic to neurons, and two show that levodopa is not toxic and may actually have a protective effect. so there is no convincing or consistent evidence that levodopa damages dopamine neurons in humans or animal models of pd. with this uncertainty as to what levodopa may be doing to the remaining dopamine cells in patients with pd, there is a strong need to make the determination in patients as to whether levodopa protects or worsens the progression of pd. primary end point: progression of pd as determined by change in a pd rating scale, the updrs, between baseline examination and examination at week 42. these two examinations are conducted by a primary rater who sees the subjects only twice: at baseline and at week 42, so as not to be influenced by any effects the subject may have experienced during the 40 week exposure to investigational medication.

Eligibility criteria:
inclusion criteria: * early, mild pd, not requiring medications * age 30 or older * duration from time of diagnosis of pd: less than 2 years * hoehn \& yahr stage 1 or 2 * exposure to levodopa or dopamine agonist of 14 days or less

Keywords: parkinson's elldopa l-dopa levodopa sinemet tremor
the role of brain activation and cerebral blood flow in mental fatigue.

Conditions: Mental Fatigue; Healthy

Summary: to assess the changes in brain activation and cerebral blood flow due to mental fatigue in a placebo-controlled study. more specifically we want to assess whether brain activation \[measured with blood oxygen level dependent (bold) functional resonance imaging (fmri)\] is decreased during a flanker task in a mentally fatigued state and whether this coincides with a decline in cognitive performance. in addition we also want to evaluate whether cerebral blood flow \[measured with arterial spin labeled (asl) fmri\] in a resting state is reduced when mentally fatigued.


Eligibility criteria:
inclusion criteria: * healthy (no neurological/cardiorespiratory/psychological disorders) * no medication * non-smoker * between 18 and 35 years old exclusion criteria: * injuries * illness * pacemaker, neurostimulator, medication pump or metal implants * use of medication or any kind of drugs * use of alcohol, caffeine and heavy efforts 24 hours before each trial * not eating the same meal the night before and the morning of each trial
a study of ro5045337 [rg7112] in patients with advanced solid tumors

Conditions: Neoplasms

Summary: this study will determine the maximum tolerated dose and the optimal associated 4 weekly dosing schedule of ro5045337, administered as monotherapy in patients with advanced solid tumors. a first cohort of patients will receive the starting dose of 20mg/m2/day, once daily for 10 days in each 28 day cycle. subsequent cohorts of patients will receive dose escalations, and possible changes in dosing schedule, based on tolerability and pharmacokinetic knowledge gained from prior treatment cohorts. the anticipated time on study treatment is until disease progression or intolerable toxicity.


Eligibility criteria:
inclusion criteria: * adult patients, \>=18 years of age; * solid tumor malignancies; * failed prior therapies, or no standard therapy available; * ecog performance status of 0-2. exclusion criteria: * patients receiving any other agent or therapy to treat their malignancy; * pre-existing gastrointestinal disorders which may interfere with absorption of drugs; * clinically significant cardiovascular disease.