Kornimate/medical-research-clean huggingface dataset:
edit-301 for autologous hematopoietic stem cell transplant (hsct) in participants with transfusion-dependent beta thalassemia (tdt)

Conditions: Transfusion Dependent Beta Thalassemia; Hemoglobinopathies; Thalassemia Major; Thalassemia Intermedia

Summary: the purpose of this study is to evaluate the safety, tolerability, and efficacy of treatment with edit-301 in adult participants with transfusion dependent beta thalassemia

this is a phase 1/2 single-arm, open-label, multicenter study evaluating the safety, tolerability, and efficacy of a single unit dose of edit-301 for autologous hematopoietic stem cell transplant in adult participants with tdt, age 18 to 35 years, inclusive

Eligibility criteria:
key inclusion criteria: diagnosis of transfusion dependent b-thalassemia as defined by: * documented homozygous β-thalassemia or compound heterozygous β-thalassemia including β-thalassemia/hemoglobin e (hbe) based on historical data in medical records, and * history of at least 100 ml/kg/year or 10 u/year of packed red blood cell (rbc) transfusions in the 2 years prior to signing informed consent * clinically stable and eligible to undergo autologous hsct * karnofsky performance status ≥ 70 key exclusion criteria: * available 10/10 human leukocyte antigen (hla)-matched related donor * prior hsct or contraindications to autologous hsct * participants with associated a history of α-thalassemia and \> 1 alpha chain deletion, or alpha multiplications as documented in medical records * participants with a history of other inherited hemoglobinopathy or thalassemic mutation (hb s, c, d or other) as documented in medical records * prior receipt of gene therapy * inadequate bone marrow function, as defined by white blood cell count of \< 3 x 10\^9/l or a platelet count \< 100 x 10\^9/l (without hypersplenism), per investigator judgement * inadequate organ function * advanced liver disease * any prior or current malignancy, or immunodeficiency disorder, * immediate family member with a known or suspected familial cancer syndrome * clinically significant and active bacterial, viral, fungal, or parasitic infection

Keywords: beta-thalassemia hemoglobinopathies crispr-cas 12a autologous cd34+
pain neuroscience education for acute and sub-acute low back pain

Conditions: Low Back Pain; Pain, Chronic; Pain, Back

Summary: to determine if pain neuroscience education (pne) would result in positive clinical changes in patients presenting with acute or sub-acute low back pain (lbp).

background: pain neuroscience education (pne) has shown efficacy in treating chronic pain. clinicians may believe pne is not suitable for acute and sub-acute pain. subgroupings of low back pain (lbp) imply some patients with lbp may respond favorably to pne. objective: to determine if pne would result in positive clinical changes in patients presenting with acute or sub-acute lbp. methods: eighty consecutive patients with lbp \< 3 months were enrolled in the study. patients completed a demographics questionnaire, leg and lbp rating (numeric pain rating scale - nprs), disability (oswestry disability index), fear-avoidance (fear-avoidance beliefs questionnaire), pain catastrophization (pain catastrophization scale), central sensitization (central sensitization inventory), pain knowledge (revised neurophysiology of pain questionnaire), risk assessment (keele start back screening tool), active trunk flexion and straight leg raise (slr). patients received a 15-minute verbal, one-on-one pne session, followed by repeat measurement of lbp and leg pain (nprs), trunk flexion and slr.

Eligibility criteria:
inclusion criteria: * a complaint of lbp with or without leg pain less than 3 months, and * a willingness to participate exclusion criteria: * a) were under age 18 (minor); * b) had undergone lumbar surgery; * c) could not read or understand the english language; * d) presented with any cognitive deficits rendering them unsuitable for pne (i.e., stroke, traumatic brain injury, etc.), * e) declined to participate or * f) presented with a medical etiology (red flag) associated with their lbp.
movement strategies during balance tasks in children with and without developmental coordination disorder

Conditions: Development Coordination Disorder; Typical Developed Children

Summary: the main objective of the study is to gain insights in how children learn a balance task and whether there is a difference between children with and without developmental coordination disorder. furthermore, the investigators are interested in the brain activity of these children while learning this new balance task.


Eligibility criteria:
inclusion criteria: for children with dcd * children with the clinical diagnosis of developmental coordination disorder (dcd) based on criteria of the dsm-5 (apa, 2013), diagnosed by a pediatrician. * children who have no formal diagnosis are evaluated by the research team using the diagnostic criteria as outlined in the inclusion criteria, these children will be included as "suspected of having dcd" * balance problems need to be present and objectified with the balance evaluation systems test for children, second edition (kids-bestest-2). (total score below 80%) for children without dcd * total score at or below the 25th percentile on the movement assessment battery for children, second edition * normal postural control, objectified by the kids-bestest-2 (score at or above 80%) * match with children with dcd based on their sex, age and potential comorbidities. exclusion criteria: presence of: * intellectual problems * visual problems * vestibular problems * neurological conditions * not able to follow instructions due to behavioral problems * refuse to participate

Keywords: postural control movement strategies brain activity postural balance [mesh] pediatrics [mesh] motor skills disorders [mesh]
asthma symptom perception study

Conditions: Asthma

Summary: asthma affects 8% of the united states population ages \>60 years and causes considerable harm: older adults are 4 times more likely to die from asthma and have twice the risk of hospitalization. the burden of asthma is notably greater among minoritized older adults. research suggests that perception of expiratory airflow limitation may be a major determinant of asthma outcomes in older adults, and that older adults are substantially less aware of airway obstruction than younger adults. these observations suggest that perception of airflow limitation is a potential target for improving outcomes of older patients with asthma. the research team completed a pilot randomized controlled trial (rct) of an intervention that trains older adults with asthma to better perceive expiratory airway obstruction through feedback via peak expiratory flow (pef) prediction and couples this feedback with motivational interviewing (mi) to promote change in asthma self-management behaviors. compared to an attention control, the intervention improved pef, perception of airflow limitation and asthma control. in this project, the research team will conduct a fully powered rct to test the intervention's efficacy among 300 adults ages ≥60 years with uncontrolled asthma who are on controller medications (daily maintenance or as needed) recruited from underserved inner-city medical practices in new york city. patients will be randomized to the intervention or a time and attention matched educational control. the intervention and control will be delivered in 3 sessions over 6 weeks. the study will test the impact of the intervention on perception of expiratory airflow limitation in older adults with asthma, examine the efficacy of the intervention for improvements in lung function (pef), self-reported asthma control (asthma control questionnaire \[acq\] scores), quality of life (asthma quality of life questionnaire \[aqlq\] scores), and emergency department and hospital use, and test the intervention's impact on mean daily ics dose used (daily maintenance and as needed). data will be collected at baseline, 1-month, 6-months (primary analyses of effectiveness) and 12-months post-intervention. in secondary analyses, the research team will test the sustainability of treatment effects with vs. without the booster treatment session (active booster vs. attention control booster) delivered immediately after the 6-month assessment on outcomes at 12-months.


Eligibility criteria:
inclusion criteria: * age \> 60 years * english or spanish speaking * self-report or physician diagnosis of asthma \>1 year ago * uncontrolled asthma exclusion criteria: * dementia * chronic obstructive pulmonary disease (copd), or other chronic respiratory illnesses, * congestive heart failure (chf, new york heart association \[nyha\] stages 4-5) * cigarette smoking \>15 packs-years
elliptical physical activity and sexual dysfunction in males with irritable bowel syndrome

Conditions: Irritable Bowel Syndrome; Erectile Dysfunction

Summary: psychological disturbances resulting from irritable bowel syndrome causes sexual dysfunction in men such as weak erection of penis. the different exercise forms are important in improving symptoms of this syndrome and exercise also may improve sexual dysfunction in men

forty irritable bowel syndrome men with sexual dysfunction ( weak erection of penis or erectile dysfunction) will divided into group i or group ii . the group will contain twenty subjects. group i will perform ellpitical physical activity (40 minutes on elliptical trainer three times weekly. the duration of physical activity will be 12 weeks. group ii will not perform any physical activity.

Eligibility criteria:
inclusion criteria: * men with irritable bowel syndrome * men with erectile dysfunction * married men exclusion criteria: * obese men * chest/renal disease men * cardiac/metabolic disease men * systemic disease men
comparison of the effects of controlled schroth exercise and home exercise programs

Conditions: Scoliosis Idiopathic; Scoliosis; Adolescence

Summary: the aim of this study is to compare the effects of schroth 3d exercise method on home symmetry, trunk topography, scapula symmetry, pelvic symmetry, health related quality of life and cosmetic deformity perception in adolescents with idiopathic scoliosis

cobb angle measurement, which is accepted as the gold standard in patients with scoliosis, is a radiological measurement. however, in continuous follow-up, the search for other methods to obtain objective data that will provide a short term evaluation on the patient in determining the efficacy of conservative treatment continues. in our study, individuals who meet the criteria will be evaluated at the beginning and end of the study. sociodemographic features and scoliosis characteristics (cobb angle, degree of bone maturation according to risser, apex, vertebra rotation angle) will be recorded. posterior trunk asymmetry index (potsi) will be used to determine body asymmetry, and topographic measurements will be performed with artec eva 3d scanner and motion analysis will be performed with biomechanical motion analysis sistem (model is bts smart dx100) . individuals will evaluate their cosmetic deformities with the walter reed visual evaluation scale. scoliosis research society-22 will be used to measure quality of life.

Eligibility criteria:
inclusion criteria: * patients with scoliosis diagnosed and followed up by the attending physician * 10-16 age group, * patients who voluntarily agreed to participate in the study * living in antalya/turkey exclusion criteria: * mental retardation, congenital scoliosis or spinal deformity, spinal surgery, neuromuscular disease, rheumatologic disease, tumor, cardiovascular or pulmonary disease, vertigo attack or balance problem, neurological disease * i̇ndividual who does not sign the consent form

Keywords: schroth exercise, conservative treatment, scoliosis
feasibility of dance in people with copd

Conditions: COPD

Summary: despite the benefits associated with individuals with chronic obstructive pulmonary disease (copd) engaging in pulmonary rehabilitation (pr) programs, the benefits diminish by 6 months. a new innovative approach is needed to increase motivation to exercise and to minimize diminution of effect. dance is a fun and interactive activity, which has shown benefits in other populations, such as parkinson's disease and stroke. the aim of this study was to investigate the feasibility of dance in individuals with copd following pr.


Eligibility criteria:
inclusion criteria: * clinical diagnosis of copd confirmed by spirometry (fev1/fvc \< 0.7) * adequate communication with or without corrective hearing aids * no intensive pr for at least 6 months before recruitment * an ability to provide informed consent. exclusion criteria: * presence of comorbidities that could have prevented communication or safe exercise

Keywords: dance copd maintenance program pulmonary rehabilitation
intra articular injection of ozone with corticosteroids vs prgf in knee osteoarthritis

Conditions: Intra Articular Injection; Corticosteroids; Plasma Rich in Growth Factors; Knee Osteoarthritis

Summary: the aim of this study is to compare the effectiveness of intra-articular injection of ozone with corticosteroids versus plasma rich in growth factors (prgf) in improvement of pain in knee osteoarthritis patients.

knee osteoarthritis (oa) is a prevalent degenerative condition in which functional impairment is caused by mechanical and chemical stress against the joint, resulting in pain and decreased range of motion (rom) . a multiplicity of treatments has been suggested for this disease : some of which include patient education, medication, exercise prescription, conventional and physical agent modalities such as pulsed radio-frequency, and surgical management , the current therapeutic options available for knee osteoarthritis are not effective and satisfactory for patients, and pain has been complained of by at least 40% of those cases who underwent surgical arthroplasty , also old age prevalence of knee osteoarthritismake treatment with analgesics has several side effects over stomach, liver and kidney and also surgical one has high morbidities and mortalities for old ages. therefore, in the last two decades, a large body of work has been performed to develop non-operative or minimally invasive interventions to decrease knee osteoarthritis symptoms or slow down its progression. among these modalities of intra-articular injections the autologous prp has gained more attention in the treatment of patients with knee osteoarthritis in recent years. several studies is supporting the use of prp injection as an effective method for knee osteoarthritis, as latelet-rich plasma (prp) could serve as anti-nociceptive and induce cell proliferation. also intra-articular injection of latelet-rich plasma modulates joint environment, promote chondrogenesis and inhibits the destruction of knee joint probably by reducing the production of pro-inflammatory mediators by the supra-physiologic concentrations of biological molecules and growth factors exist in in the granules of the platelets which could potentially reverse the catabolic environment in osteoarthritis, balancing the homeostasis of the joint, and subsequently stimulate the repair of damaged cartilage.

Eligibility criteria:
inclusion criteria: * patients aged 21-65 years * both genders * who will be diagnosed with stage 2-3 knee osteoarthritis according to the kellgren-lawrence classification system (k-l) \[10\], with womac score \> 2 for pain stiffness and physical disabilities * having symptoms of knee oa at least 3 months after getting usual conservative treatment like oral analgesics and therapeutic exercises. exclusion criteria: * patient refusal. * patients who will be diagnosed with stage 1or 4 oa according to k-l classification * history of knee trauma within the past month * history of malignancies * any surgical intervention of the knee * local or systemic infection * any patient with sever renal impairment * bleeding disorders
implementation of a cardiometabolic disease staging system in primary care

Conditions: Diabetes; Obesity; Overweight

Summary: the aim of this study is to develop a clinical decision support system (cdss) that incorporates obesity management guidelines and treatment options for use by physicians and, potentially, patients based on a cardiometabolic disease staging system. this cdss will be designed using primary care provider input through a qualitative approach to explore physician preferences for the appearance, electronic location, treatment options, referrals, accessibility and other user requirements of the cdss.

to provide appropriate medical management of obesity and facilitate the diabetes risk assessments of people with excess adiposity, a comprehensive staging system that establishes five stages of cardiometabolic disease risk-the cardiometabolic disease staging system (cmds) has been developed. this staging is based on adult treatment panel iii metabolic syndrome risk factors to guide decision making for selection of treatment modality and intensity in the management of obesity. hence, the study hypothesis is that providing cmds to pcps through a cdss for medical management of obesity can aid in reducing the barriers of obesity and diabetes treatment at the pcp-level and improve the quality of obesity and diabetes care. in this pilot and feasibility study, investigators will implement an early-phase cdss in a pragmatic pcp setting to assess the uptake and acceptability of cdss delivered to pcps implementing cmds at the point of care, and collect pilot data to assess change in patient engagement and satisfaction. investigators will conduct initial focus groups with pcps and a series of iterative design steps targeting pcp and patient needs. these qualitative interviews would be used to determine the initial set of requirements and prototypes of the user interfaces and notifications.

Eligibility criteria:
inclusion criteria: * primary care physicians employed at the university of alabama at birmingham exclusion criteria: * physicians ≥19
the results of middle-sized posterior malleolus fractures: fixed versus non-fixed

Conditions: Ankle Fractures; Posterior Malleolus Fractures

Summary: this study aims prospective randomized comparison of mid-term clinical outcomes of middle-sized posterior malleolus fractures with and without internal fixation.

although it has traditionally been accepted that posterior malleolar fractures involving less than 25 percent of the tibial plafond do not require operative fixation, this belief is now questioned. a number of studies have shown that the posterior malleolus contributes significantly to ankle stability. many studies have also demonstrated that postoperative outcomes are better in patients who underwent posterior malleolar reduction and fixation. the aim of this prospective study is to show how internal fixation of the posterior malleolus, including middle-sized posterior malleolar fractures (10-25%), affects clinical outcomes and to discuss the factors that may lead to arthritis of the ankle joint. the groups will be randomized. the clinical and functional results of 40 patients who will be operated between 2015-2019 due to ankle fracture will be evaluated at short to mid-term (2-5 years). according to our hypothesis, internally fixed posterior malleolar fractures give better clinical and functional scores.

Eligibility criteria:
inclusion criteria: patients who were operated for ankle fractures, including posterior malleolar fracture between 10-25% of the joint surface. two to five years of follow-up with functional and radiological outcome. exclusion criteria: posterior malleolar fractures accompanied by severe soft tissue damage after high-energy injury will not be included in the study. written consent will be obtained from all participants, patients who do not give consent or refuse to participate will not be included in the study. patients who left the study voluntarily will not be included in the statistics. patients with additional joint disorders, previous surgery or previous history of ankle complaint will not be included.

Keywords: ankle fracture posterior malleolus fixation
study to evaluate safety, tolerability, and pharmacokinetics of ub-621 in healthy volunteers

Conditions: Herpes Simplex Virus Infection

Summary: the purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics of a single subcutaneous dose of ub-621 in healthy volunteers.

this is a phase i, open-label, single dose, dose escalation study in healthy volunteers. eligible volunteers will be sequentially enrolled into four escalating-dose cohorts at a study site. after administration of a single subcutaneous dose of ub-621, subjects will be followed for 70 or 112 days and monitored for safety, tolerability, and pharmacokinetics parameters.

Eligibility criteria:
inclusion criteria: * with age between 20 and 55 years. * with no clinically relevant abnormalities in vital signs, and clinical laboratory tests at screening visit judged by investigator. * body weight: \<85 kg. * subject has signed the written informed consent form. exclusion criteria: * with significant active infection (acute or chronic) within 28 days prior to the screening visit. * evidence or history of clinically significant hematological, renal, endocrine, pulmonary, gastrointestinal, cardiovascular, hepatic, psychiatric, neurologic or allergic diseases, or clinical findings within 3 months prior to the screening visit. * positive serology for hiv antibody, hcv antibody or hbv surface antigen. * female subjects who are breastfeeding, pregnant, and planning to become pregnant during the study period. * the clinical investigator considers that the subject is not in the condition to participate in this study.
evaluation of gas propagation in snow during breathing of subjects under simulated avalanche snow

Conditions: Accident Caused by Snow Avalanche

Summary: the study is aimed at investigation of respiratory gases propagation in snow. the study involves volunteers breathing into the snow whereas concentrations of gases are measured at various positions in the snow.

the study is aimed at investigation of respiratory gases (oxygen and carbon dioxide) propagation in the simulated avalanche snow. the study involves volunteers breathing into the snow whereas concentrations of gases are measured at various positions in the snow. a special apparatus was created for sampling and measurement of gas concentrations and to assure stable geometry of the experiment required for data evaluation.

Eligibility criteria:
inclusion criteria: * healthy and fit volunteers, classified as the american society of anesthesiologists class i * without a smoking history exclusion criteria: * tiffeneau index less than 0.70 * any cardiovascular or respiratory condition

Keywords: snow avalanche hypercapnia diffusion