Kornimate/medical-research-clean huggingface dataset:
effect of sacubitril/valsartan on reduced right ventricular ejection fraction in patients with ctd

Conditions: Myocardial Injury; Connective Tissue Diseases

Summary: heart failure, one of the leading causes of connective tissue disease (ctd) mortality, has attracted increasing attention. currently, no known study had focused on the effect of sacubitril/valsartan on right ventricular dysfunction and in the systemic disease induced heart disease. we aimed to observe the effect of sacubitril/valsartan on primary endpoints (6 minutes walking test and myocardial fibrosis) in ctd patients with right ventricular ejection fraction reduction (rv-hfref).

patients with ctd frequently exhibit multi-organ pathophysiological and functional damage. heart failure, one of the leading causes of ctd mortality, has attracted increasing attention. mostly, patients with ctd present with nonspecific cardiac symptoms, normal ecg, and preserved left ventricular ejection fraction (lvef) and therefore do not receive an early cardiac diagnosis and treatment. pulmonary arterial hypertension (pah), right ventricular (rv) dilatation and hypertrophy might become the first and the most frequent cardiac findings. sacubitril/valsartan, an angiotensin receptor-neprilysin inhibitors, is a "superstar" which inhibits both neprilysin and renin-angiotensin aldosterone system that closely related to the heart failure mechanism. it has been strongly recommended as class i drug in treating the patient with chronic hfref from 2017 acc/aha/hfsa heart failure guideline for the ability of dramatically reduce the cardiovascular mortality rate. cardiovascular magnetic resonance (cmr) is able to depict myocardial characteristics from structure to tissue properties using cine and late gadolinium enhancement (lge) sequences. newly developed imaging studies to date include t1 mapping and t1-derived extracellular volume estimation. all the previous studies in ctd have been restricted to patients with advanced cardiac involvement. together with clinical assessment and multi-imaging tests, the aim of the present study is going to observe the effect of sacubitril/valsartan on primary endpoints (6 minutes walking test and myocardial fibrosis) in ctd patients with rv-hfref.

Eligibility criteria:
inclusion criteria: * age between 18-75 years old. * confirmed ctd（including systemic lupus erythematosus, myositis, polymyositis, systemic sclerosis, sarcoid, sjögren's syndrome or mixed connective tissue disease） * sledai ≤ 6 in patients with sle or esr ≤ 30 in patients with ssc * already have opt for ctd at least 3 month * rvef ≤ 45% * providing written informed consent exclusion criteria: * documented coronary artery disease or prior angiography for coronary artery disease (\>50% stenosis). * patients with known congenital heart disease or other systemic diseases that might induce rvref. * patients with standard metallic contraindications to cmr or an estimated glomerular filtration rate \< 30 ml/min/1.73 m2.

Keywords: connective tissue diseases sacubitril/valsartan cardiovascular magnetic resonance extracellular volume exercise tolerance
effect of a hydrolyzed rice protein-based formula on growth, tolerance, and health-related quality of life in infants with cow's milk protein allergy.

Conditions: Cow Milk Protein Allergy

Summary: the main purpose of this study is to demonstrate that the growth of infants fed with the test formula is non-inferior those fed with the control formula. the study will also evaluate the gastrointestinal tolerance, quatliy of life and acceptability of the new rice protein-based formula in infants with cow's milk protein allergy (cmpa).


Eligibility criteria:
inclusion criteria: 1. written informed consent has been obtained from at least one parent (or legally acceptable representative \[lar\]), if applicable) 2. infant gestational age ≥ 37 completed weeks 3. infant birth weight of ≥ 2.5 kg and ≤ 4.5 kg 4. singleton birth 5. infant age ≤ 8 months 6. mother has previously decided to fully formula-feed, and infant is no longer breastfeeding or receiving breast milk 7. suspected cmpa as per standard clinical practice and in conjunction with at least 2 symptoms present from the list shown below: 1. inconsolable crying, regurgitation, liquid stools or constipation, skin atopic lesion, cow's milk provoked temporary urticaria/angioedema or vomiting, bloody streaks in stool, or respiratory symptoms 2. for diagnosis based on either a positive ige blood test, skin prick test or food challenge, only at least 1 symptom from above list needs to be present 8. infant is receiving a strict cow's milk elimination diet and will continue receiving this diet until the completion of the study 9. infant's parent(s)/lar is of legal age of majority, must have parental authority, must understand the informed consent form and other study documents, and is willing and able to fulfill the requirements of the study protocol exclusion criteria: 1. history of intolerance to ehf formula 2. any chronic medical diseases (except atopic eczema), chromosomal or major congenital anomalies (based on medical history and/or commonly performed diagnostic criteria) 3. major gastrointestinal disease / abnormalities (other than cmpa) 4. known or suspected lactose intolerance or malabsorption 5. known or suspected soy allergy 6. glucose-galactose malabsorption 7. rice protein allergy or intolerance (e.g., rice protein-induced enterocolitis syndrome). 8. immunodeficiency 9. persistent wheeze or chronic respiratory disease 10. severe uncontrolled eczema 11. history of severe anaphylactic reaction (e.g., requiring ≥ 2 doses of epinephrine) to cow's milk or breast milk at any time prior to enrollment 12. weight-for-age value \ 2 standard deviations from the who child growth standards median at enrollment 13. height-for-age \ 2 standard deviations from the who child growth standards median at enrollment 14. infant's parent has other medical or psychiatric condition that, in the judgement of the investigator, would make the infant inappropriate for entry into the study 15. currently participating or having participated in another interventional clinical study within 4 weeks prior to enrollment
progressive resistance training in patients with class iii obesity

Conditions: Obesity

Summary: to test the strength and physical performance outcomes of a 12-week progressive resistance training (prt) exercise program in a small cohort of patients with class iii obesity who are preparing for bariatric weight loss surgery at beth israel deaconess. the study will also test patient compliance to the 12 week prt program. whereas prt exercise is currently recommended as a part of preoperative clinical care for bariatric surgery patients, little research has been done to measure the acceptance and effectiveness of a standardized prt exercise protocol for improving health and perioperative care. preliminary data from this study could be used to justify larger studies and further investigation.


Eligibility criteria:
inclusion criteria: * men and women 18-65 years old * a bmi of ≥40 kg/m2 * considering weight loss surgery at bidmc * participating in multidisciplinary preoperative program: * been determined by a mental health professional (i.e., psychologist or social worker) to be well-informed, motivated, and not symptomatic for psychopathology that would put the person at risk for injury or failure with wls * expressed strong desire for significant weight loss * experienced documented failure of long-term weight loss using nonsurgical methods * been informed of and accepts risks of surgery * cleared by their primary care physician to be in stable health and capable of moderate level exercise * willing to comply with expected attendance and participation at all intervention sessions and testing appointments * has reliable transportation to the intervention location exclusion criteria: * orthopedic limitations that would preclude exercise testing and training

Keywords: bariatric surgery class iii obesity resistance training strength fitness
frontline combination car-t cell therapy for multiple myeloma or plasmacytoma

Conditions: Multiple Myeloma; Plasmacytoma

Summary: the aim of this clinical trial is to assess the feasibility, safety, and efficacy of car-t cell therapy targeting multiple cancer cell antigens in high-risk multiple myeloma or plasmacytoma as part of a frontline treatment regimen for patients. another goal of the study is to learn more about the persistence and function of these car-t cells in the body.

multiple myeloma (mm) is the second most common malignant hematological cancer in the world, which begins with the malignant proliferation of plasma cells in bone marrow. it has been a difficult disease to treat, and most patients will eventually relapse, especially for those with high-risk genotypes. at present, the therapeutic drugs for mm include glucocorticoids, cytotoxic drugs, immunosuppressants, protease inhibitors, monoclonal antibodies and cell therapies. among those, immunotherapy has been proven to be a revolutionary treatment with great potential of curing this disease. the frequently targeted mm antigens include cd38, cd138, cd19 and bcma, and recently, gprc5d. bcma, the b cell maturation antigen, also known as cd269 or tnfrsf17, is a member of tumor necrosis factor receptor superfamily, which is highly expressed on the surface of plasma cells and partially expressed on plasma cell-like dendritic cells. it has been an ideal target for mm immunotherapy. gprc5d, the g-protein-coupled receptor c57 subtype d and a seven-transmembrane protein, is highly expressed on the surface of plasma cells but not in other healthy cells, and thus it has become a potential target for the treatment of mm. the expression of gprc5d is unrelated to bcma, so the combination therapy targeting these antigens may bring a complementary and synergistic therapeutic outcome in patients. this trial is aimed to test the safety and efficacy of combining these different car-t cells targeting bcma and gprc5d, and in combination with well-established therapeutics as a frontline treatment for the high-risk mm or plasmacytoma patients. another goal of this study is to investigate the persistence and function of these car-t cells in the body.

Eligibility criteria:
inclusion criteria: * male and female subjects with multiple myeloma or plasmacytoma * strictly complete remission (scr) is a treatment goal * expected survival \> 12 weeks * after prior auto-sct is eligible regardless of other prior therapies * adequate venous access for apheresis, and no other contraindications for leukapheresis * voluntary informed consent is given and commitment to continued follow-up exclusion criteria: * pregnant or lactating women * uncontrolled active infection * active hiv, hepatitis b or hepatitis c infection * concurrent use of systemic steroids. recent or current use of inhaled steroids is not exclusionary. * any medical conditions that may preclude participation

Keywords: multiple myeloma chimeric antigen receptor bcma gprc5d
molecular genetic basis of invasive breast cancer risk associated with lobular carcinoma in situ

Conditions: Breast Cancer; Lobular Carcinoma; Invasive Breast Cancer

Summary: this study is being done in order to better understand the biology of an abnormal lesion found in breast tissue called "lobular carcinoma in situ" (lcis). we are interested in studying lcis. the lcis is not a cancer itself, but is a marker for an increased risk of cancer. we would like to look for lcis in breast tissue removed during surgery from patients with cancer or at high risk for cancer. if lcis is found, we will search for genes that are expressed (turned on or off) differently than in normal breast tissue. the identification of such genes would help us better understand the biology of lcis, and its possible relationship to breast cancer.

lcis) is a monoclonal pathologic entity which is subject to characterization at the molecular genetic level, and that these molecular genetic alterations may be used to predict the subsequent development of invasive breast cancer. prophylactic mastectomy specimens from women with multifocal lcis, and invasive breast cancer specimens which display coexisting lcis, will be examined for x-chromosome inactivation patterns and loss of heterozygosity to assess for monoclonality. if clonality is present, we will assess for microsatellite instability, and a microarray-based comparative genomic hybridization (cgh) technique will be used to identify genetic alterations present in lcis. lastly, lcis biopsy specimens from untreated patients who, after follow-up did or did not develop invasive breast cancer, will be evaluated to determine whether the nature or extent of any identified genetic alterations can be correlated with the subsequent development of invasive breast cancer. we hypothesize that a fraction of lcis lesions will reflect a monoclonal origin, that those lesions of monoclonal origin will display evidence of specific molecular genetic alterations, and that these specific alterations will correlate with the likelihood of the subsequent development of invasive breast carcinoma.

Eligibility criteria:
inclusion criteria: * multifocal lobular carcinoma in situ treated with prophylactic mastectomy or lumpectomy * invasive breast cancer (lobular or ductal) with coexisting lobular carcinoma in situ treated with mastectomy or lumpectomy * biopsy proven, untreated lobular carcinoma in situ * invasive lobular cancer with or without coexisting lobular carcinoma in situ treated with mastectomy or lumpectomy exclusion criteria: * no paraffin blocks available * no residual lobular carcinoma in situ in paraffin blocks

Keywords: biopsy breast 01-135
implementation of an evidence-based intervention to improve head impact safety in youth football

Conditions: Head Injury; Sport Injury

Summary: the objective of this study is to evaluate the feasibility of implementing an evidence-based intervention program, communities aligned to reduce concussion and head impact exposure (coach) on a larger scale. coaches of 12u (12 years old and under), and 13u (13 years old and under) teams within six youth football organizations will pilot test the intervention. aim 1 will conduct focus groups with coaches, parents, and organizational leaders to assess organizational needs, capacity, and readiness to adopt the intervention program. aim 2 will evaluate the effectiveness of the intervention program at reducing hie and injuries and evaluate implementation success. aim 2 results (intervention outcomes) are reported herein.

coaches of teams at the 12u and 13u levels within the six youth football organizations will be invited to pilot test the intervention program. intervention outcomes will be compared to historic controls (k25hd101686 - nct04908930). video taken at all practices will be used to monitor team activities of 12u and 13u teams across all organizations participating in the intervention; incidence of injuries will be monitored across all teams. athletes (n=30) enrolled across two of six participating teams will be recruited to participate in parallel biomechanical and video data collection to evaluate the frequency and severity of head impacts experienced throughout the season. biomechanical data will be collected using instrumented mouth guards and verified with video. preliminary effectiveness will be measured by the percentage of coaches who choose to adopt and maintain the intervention program. head impact exposure and injury rates will be compared across the intervention and control teams.

Eligibility criteria:
inclusion criteria: * all athletes participating on the prospective teams will be eligible for the study, including those with braces longer than 6 months exclusion criteria: * athletes will be excluded from participation if they have had braces less than 6 months, or have dental appliances that may impede the fit of the mouthpiece device (e.g., herbst appliance).

Keywords: sport safety youth football head impact exposure
a study assessing b-vec compared to matching placebo for the treatment and prevention of corneal abrasions in dystrophic epidermolysis bullosa

Conditions: Dystrophic Epidermolysis Bullosa; DEB - Dystrophic Epidermolysis Bullosa; Recessive Dystrophic Epidermolysis Bullosa; Dominant Dystrophic Epidermolysis Bullosa

Summary: b-vec-eye-01 is a phase 3 double-blind, intra-patient crossover study, to evaluate the safety and efficacy of ophthalmic beremagene geperpavec (b-vec) versus matched placebo in pediatric and adult subjects with recurrent corneal abrasions due to dystrophic epidermolysis bullosa (deb).

subjects participating in the sponsor's natural history study (protocol b-vec-eye-nhs) for at least 12 weeks and that meet all other eligibility criteria receive weekly ophthalmic administration of investigational product (ip), b-vec or placebo, for the first 12-week intervention period. during the second intervention period subjects will receive the alternative ip for an additional 12 weeks. ip will be administered in the home setting by a healthcare provider. the investigator (or designee) will contact subjects (or their parent/legal guardian) to assess for any adverse events or changes in medications, treatments, or procedures. subjects (or their parent/legal guardian) will complete weekly symptom diaries and monthly questionnaires to document corneal abrasion symptoms and frequency and gauge overall subject (or parent/legal guardian) impressions of disease severity and disease/symptom changes during the study.

Eligibility criteria:
inclusion criteria: 1. the subject and/or their parent/legal guardian must provide informed consent/assent and must be able to and willing to follow study procedures and instructions. 2. age 6 months or older at time of informed consent/assent. 3. confirmed diagnosis of deb with a mutation in the col7a1 gene. 4. meets minimum corneal abrasion symptom frequency in the b-vec-eye-nhs study. exclusion criteria: 1. initiation of any new treatment regimen or change in treatment for ocular disease during the run-in period except for preservative free topical antibiotics or artificial tears/lubricants associated with standard of care treatment of corneal abrasions. 2. treatment with an investigational agent or off-label ophthalmic use of an approved product during the run-in period or planned use during the study (exceptions may be approved by the medical monitor on a case-by-case basis). 3. any condition that, in the opinion of the investigator, would impact the completion of all study-related assessments, interfere with the administration of study drug, and/or poses an additional risk to the subject. 4. women who are pregnant or nursing. 5. subject who is unwilling to comply with contraception requirements per protocol. 6. subject is known to be noncompliant or is unlikely to comply with the requirements of the study protocol in the opinion of the investigator.

Keywords: dystrophic epidermolysis bullosa deb corneal abrasions
an observational study using multimodal sensors to measure cognitive health in adults and distinguish mild cognitive impairment from normal aging

Conditions: Mild Cognitive Impairment; Normal Aging

Summary: the primary objectives are to develop and validate a classifier using multimodal passive sensor data and metrics derived from normal iphone and apple watch usage to distinguish individuals with normal cognition from those with mild cognitive impairment (mci) and to develop and validate a cognitive wellness score that tracks fluctuations in cognitive performance over time using multimodal passive sensor data and metrics derived from normal iphone and apple watch usage.


Eligibility criteria:
key inclusion criteria: * fluent in spoken and written english * minimum 8 years of education or 8th grade equivalent. * willing to authorize the use of confidential health information in accordance with applicable privacy regulations. * willing to wear an apple watch for daily use for the duration of the study. * willing to use their personal iphone for routine daily use for the duration of the study. * technical requirements: iphone models: iphone 8 or newer ios versions: current publicly available ios version * access to one of the following personal computing devices. microsoft windows based personal computers (laptops and desktops) mac personal computers (laptops and desktops) apple ipad tablets * access to wifi or hardwired internet access in participant's place of residence. * active email address for enrollment account creation and for use in relaying study information. * active mobile telephone number for use in relaying study information when appropriate. * primary address within the united states (50 states and washington, dc; not a post office (po) box). key exclusion criteria: * inability to comply with study requirements. * any self-reported medical history of dementia (such as alzheimer's disease, frontotemporal dementia, dementia with lewy body, vascular dementia, rapidly progressive dementia, or parkinson's disease dementia). * self-reported dependence on others to perform core activities of daily living, including feeding, dressing, bathing, toileting, or grooming. note: other protocol defined inclusion/exclusion criteria may apply.
registry for patients with achondroplasia / hypochondroplasia (ompr-ach/hy)

Conditions: Achondroplasia; Hypochondroplasia

Summary: this registry is a observational, single-center study designed to collect clinical data on patients with achondroplasia and hypochondroplasia.


Eligibility criteria:
inclusion criteria: * confirmed diagnosis of achondroplasia/hypochondroplasia * patients (and/or parents legal guardian when required) able to provide informed consent exclusion criteria: * absence of diagnosis of achondroplasia/hypochondroplasia * patients (and/or parents legal guardian when required) not able to provide informed consent
a retrospective analysis of raltegravir use in minority hiv infected women in houston, texas

Conditions: HIV Infection

Summary: raltegravir is the first marketed strand-transfer inhibitor of hiv-1 that was fda approved in 2007. it is currently one of the preferred treatment regimens for hiv by the department of health and human services. it has become a widely used antiretroviral therapy option for hiv infected patients. it provides good tolerability and a favorable lipid profile for patients when compared to some other antiretroviral treatment options. little data is reported about efficacy in a minority patient population. moreover, data in an indigent minority population in the united states has not been aggregated before. therefore this study will investigate the efficacy of raltegravir in minority women residing in houston, tx who are hiv infected.


Eligibility criteria:
inclusion criteria: 1. hiv-infected women who picked up raltegravir in the year 2013 at thomas street health center. 2. minority women -black/african american, hispanic/latino exclusion criteria: * n/a

Keywords: hiv infection raltegravir use women