Kornimate/medical-research-clean huggingface dataset:
a study to compare safety and efficacy of high doses of eteplirsen in participants with duchenne muscular dystrophy (dmd) (mis51on)

Conditions: Muscular Dystrophy, Duchenne

Summary: part 1 (dose escalation) will evaluate the safety and tolerability of 2 doses (100 milligrams/kilogram \[mg/kg\] and 200 mg/kg) of eteplirsen in approximately 10 participants with dmd; part 2 (dose finding and dose comparison) will evaluate the efficacy and safety of the high doses (100 mg/kg and 200 mg/kg) of eteplirsen compared with that of the 30 mg/kg dose of eteplirsen, in approximately 144 participants with genetically confirmed deletion mutations amenable to treatment by skipping exon 51.


Eligibility criteria:
inclusion criteria: * be a male with an established clinical diagnosis of dmd and an out-of-frame deletion mutation of the dmd gene amenable to exon 51 skipping. * ambulatory participant, able to perform ttrise in 10 seconds or less at the time of screening visit. * able to walk independently without assistive devices. * have intact right and left biceps muscles or an alternative upper arm muscle group. * have been on a stable dose or dose equivalent of oral corticosteroids for at least 12 weeks prior to randomization and the dose is expected to remain constant (except for modifications to accommodate changes in weight and stress-related needs as per the recently published guidelines throughout the study. * for ages 7 years and older, has stable pulmonary function (forced vital capacity ≥50 percent (%) of predicted and no requirement for nocturnal ventilation). for ages 4 to 6 years, does not require support from ventilator or non-invasive ventilation at time of screening. exclusion criteria: * use of any pharmacologic treatment (other than corticosteroids) within 12 weeks prior to randomization. * current or previous treatment with any other experimental pharmacologic treatment for dmd or any prior exposure to antisense oligonucleotide, gene therapy or gene editing; except the following: ezutromid in the last 12 weeks prior to first dose; drisapersen in the last 36 weeks prior to first dose; suvodirsen in the last 12 weeks prior to first dose; vamorolone in the last 12 weeks prior to first dose; eteplirsen (previous or current use); and tamoxifen in the last 4 weeks prior to first dose. * major surgery within 3 months prior to randomization. * presence of any other significant neuromuscular or genetic disease other than dmd. * presence of any known impairment of renal function and/or other clinically significant illness. * has evidence of cardiomyopathy, as defined by left ventricular ejection fraction less than \<50% on the screening echocardiogram or fridericia's correction formula (qtcf) ≥450 millisecond based on the screening electrocardiograms (ecgs). other inclusion/exclusion criteria apply.

Keywords: duchenne muscular dystrophy exon skipping exon 51 north star ambulatory assessment ambulatory dmd pediatric duchenne exondys mis51on
impact of immune checkpoint inhibitor therapy on type 2 inflammation

Conditions: Patients Recieving Anti-PD-1 or Anti-PD-L1 Immunotherapies

Summary: the primary objective of this study is to determine the impact of anti-checkpoint immunotherapy on type 2 inflammation via a retrospective analysis of % eosinophilia.


Eligibility criteria:
inclusion criteria: * electronic patient file "dxcare" at the university hospitals of montpellier * patient has received anti-pd-1 immunotherapy --and/or -- anti-pd-l1 immunotherapy according to central pharmacy records exclusion criteria: * no mention of % eosinophilia in the patient file
colisurg : exploratory analysis of sexual function and the impact of biotherapies on postoperative morbidity.

Conditions: Ulcerative Colitis

Summary: the surgical treatment of the ulcerative colitis (uc) remains associate to a significant morbidity (up to 60%). anastomotic fistula and pelvic sepsis are the most severe complications which could dramatically compromise the surgical issue and functional status. thanks to the current therapeutic arsenal and the evolution of health care paradigms, the quality of life of patients plays a key role in the modern global management of these medical conditions. biotherapies (e.g anti-tnf) are widely used to treat patients with uc. anti-tnf and anti-integrins have an effect on the immune response and can theoretically aggravate the infectious disease. their potential impact on postoperative complications after ileo anal anastomosis (aia) remains debated. very few studies have looked at other biotherapies including vedolizumab. all studies are retrospective series with small sample size. here again the conclusion remain contradictory. lightner et al. showed an increased risk of surgical site infection for patients preoperatively exposed to vedolizumab (37% vs. 10%, p \<0.001). in a dedicated cohort to the rch, the same author found a risk of increased pelvic abscess (31.3% vs 5.9%, ns) but the difference was not statistically significant probably for lack of power. other studies did not find any impact of vedolizumab on the risk of postoperative complications. to clearly determine within a large prospective cohort the impact of anti-tnf agents and biotherapies on the postoperative complications seems to be essential in order to adapt and to optimize the therapeutic strategy, especially the surgical sequences, in patients with ucr whom benefit a surgery.


Eligibility criteria:
inclusion criteria: age ≥ 13 ans - patients with ulcerative colitis requiring surgical treatment with ileal pouch-anal anastomosis exclusion criteria: * age \< 13 ans * under any administrative or legal supervision

Keywords: surgical ileal pouch-anal anastomosis
exercise and shoe orthoses in treatment of posterior tibial tendon dysfunction

Conditions: Tendinopathy

Summary: posterior tibial tendon dysfunction (pttd) is the most common cause of painful and debilitating acquired flatfoot deformity in adults. the dysfunction is often progressive and may result in attenuation of the ptt and eventually collapse of the plantar arch or even rupture of the tendon. current therapeutic management of pttd is multipronged and includes management with foot orthoses, stretching, and strengthening exercises. evidence drawn from research related to the management of painful chronic achilles tendinosis suggests that eccentric strengthening of the posterior tibialis may lead to superior results compared to concentric. the purpose of this research is to determine the effectiveness of treatment interventions used in the management of pttd.

all study participants receive a pair of custom made orthoses and perform daily calf-stretches. in addition to the orthoses two groups will perform tibialis posterior specific exercises; one as concentric and the second as eccentric activation. intervention will last 12 weeks.

Eligibility criteria:
inclusion criteria: * pain (\> 3 months duration) * pain localized to the medial ankle and foot * swelling at the medial ankle * able to perform a single controlled heel lowering exclusion criteria: * bilateral posterior tibial tendon dysfunction * fixed foot deformities * previous foot surgery * presence of any other concurrent foot pathology besides posterior tibial tendon dysfunction * ability to walk only with assistive device * neurological disorders * cognitive dysfunction * uncontrolled cardiovascular disease * evidence of cord compression * uncontrolled hypertension * infection * severe respiratory disease * pregnancy * current or recent history of low back pain * known rheumatic joint disease * peripheral vascular disease with sensory loss of the foot.

Keywords: tibialis posterior tendinopathy
iv acetaminophen in children undergoing palatoplasty

Conditions: Cleft Palate

Summary: this project is being used to assess the utility of intravenous acetaminophen in the pediatric population undergoing palate repairs. this study is a prospective randomized double blinded placebo controlled study. the investigators will have two arms, one group of patients will receive intravenous acetaminophen and one group that will receive a placebo at a total of four doses over 24 hours. the results of this study may affect the intraoperative anesthetic management as well as post-operative surgical management of this specific patient population. the goal is to determine the benefit of adding a non-opioid analgesic and hypothesizing that it may reduce the amount of narcotic consumption in the post anesthesia care unit as well as during the post-operative stay.


Eligibility criteria:
inclusion criteria: * asa physical status i or ii * presenting for repair of palatoplasty with drs. kirschner or pearson exclusion criteria: * asa physical status \> ii * underlying syndrome * significant co-morbid diseases (cardiac, pulmonary, neurological disease) * patients having concomitant procedures (tonsillectomy, adenoidectomy, etc.)
quantitative analysis of outcomes in breast reconstruction

Conditions: Breast Cancer

Summary: the first objective of this study is to assess the accuracy of digital image-processing techniques to provide measurements of human breast physical dimensions. the ultimate usefulness of these techniques is to develop quantitative outcome measures of the visual appearance of breast after local treatments for breast cancer. these measures will be used to improve clinical outcomes from multimodality therapy that includes surgery and radiotherapy and form the basis for the future development of patient decision making aids. the second objective is to evaluate the relationship between patient reported body image outcomes and measurement of human breast physical dimensions for patients with breast cancer.

if you agree to take part in this study, you will be asked to fill out a brief questionnaire packet. this packet contains questions about how you feel about your appearance and your overall well-being. you will be asked to have photographs of your breast(s) taken with two different types of digital cameras (2d and 3d). these are the same type of photographs routinely taken for all patients having breast reconstructive surgery. the photographs and measurements will be taken during your routine clinic visit. you will not be identifiable in the photographs. the photographs will be used to develop automated methods for evaluating the appearance and shape of the breasts. you may be asked to complete additional follow-up visits for this study. these visits would take place at a regularly-scheduled clinic visit that takes place just before a surgical procedure is planned. at these visits you would be asked to fill out another questionnaire packet and to allow the research the research staff to repeat the photographs. the number of follow-up visits needed would depend on your individual treatment plan. a final study visit would take place once you feel you are finished with your breast reconstructive process. at this point, your participation in the study would be completed. breast reconstruction is different for each patient. the length of time of the reconstructive process depends on the procedure(s) that you and your surgeon have agreed upon. these options will be discussed at your regularly scheduled clinic appointments. this is an investigational study. a total of up to 300 patients will take part in this study. all will be enrolled at utmdacc.

Eligibility criteria:
inclusion criteria: 1. female 2. age greater than 21 years old. 3. candidate who has had or is planning to have breast reconstructive surgery and has at least one breast mound. 4. willing to participate in the study and able to complete informed consent. 5. proficient at reading and speaking english. exclusion criteria: 1. women who have undergone previous bilateral mastectomy without reconstruction. 2. women who are unable to stand unassisted for 2 minutes. 3. diagnosis of serious mental illness (e.g. schizophrenia) or cognitive impairment.

Keywords: breast cancer breast reconstruction digital imaging questionnaire survey
macular pigment optical density in healthy subjects

Conditions: Age Related Macular Degeneration

Summary: the macular pigment (mp) in humans consists of the yellow, blue-absorbing carotenoids lutein and zeaxanthin, which are contained in vegetables and fruits. the highest concentrations of lutein and zeaxanthin are found in the fovea. since light entering the eye passes through the mp before reaching the photo receptors it absorbs a significant portion of short-wavelength light. there is evidence that these absorbing properties of the mp as well as the ability of inactivating highly reactive oxygen species are protective for the retina. measurement of macular pigment is difficult and the investigators have recently proposed a way of measuring macular pigment optical density (mpod) based on optical reflectometry. there is increased interest in these measurements in the recent years, because a number of studies has provided evidence that low fruit and vegetable consumption increases the risk of age related macular degeneration (amd). the present study investigates mp optical density (od) in healthy volunteers and is used to form a database. furthermore the nutritional habits and the influence on mp density will be evaluated.


Eligibility criteria:
inclusion criteria: * healthy subjects without any signs of amd * age between 18 and 90 years, (20 subjects aged between 18 and 40, 20 subjects aged between 41 and 50, 20 subjects aged between 51 and 60, 20 subjects aged between 61 and 70, 20 subjects aged between 71 and 80, 20 subjects aged between 81 and 90) * clear non-lenticular ocular media * visual acuity \> 0.6 exclusion criteria: * any sign of amd, or another retinal or optic nerve head disease * ocular surgery within the last 6 months * treatment with photosensitizing drugs

Keywords: macular pigment to measure macular pigment in the retina
toripalimab based induction chemotherapy followed by de-escalation protocols in hpv-related opscc

Conditions: Oropharyngeal Carcinoma; Immune Checkpoint Inhibitor; De-escalation

Summary: human papillomavirus (hpv)-related oropharyngeal carcinoma are exquisitely radiosensitive. several studies attempted to reduce the toxicities of treatments through reduced-dose radiation and showed promising results, but all data were collected from non-chinese areas. like nasopharyngeal carcinoma (npc), oropharyngeal carcinoma may have different biological behavior and relationship with hpv infection. so the investigators studied whether toxicities reducing treatment with reduced radiation dose and omitted concurrent chemotherapy after good response to induction chemotherapy would maintain survival outcomes while improving tolerability for patients with hpv-positive oropharyngeal carcinoma. immune checkpoint inhibitors (icis) have proved to improve outcomes of head and neck cancers including ebv-related npc. oropharyngeal carcinoma was considered to be similar with npc in terms of immune environment. so we added anti-pd-1 antibody toripalimab to induction chemotherapy in order to achieve better response rates to receive de-escalation chemoradiotherapy followed.


Eligibility criteria:
inclusion criteria: * histological diagnosis of squamous cell carcinoma of oropharynx with ihc p16 * positive or pcr hpv16 positive * t1-2/n1-3m0（except t1n1m0 and single ln\<3cm）or t3-4n0-3m0 according to uicc/ajcc 8th staging system * age ≥18 * no prior anti-tumor treatment * karnofsky performance score (kps)≥70 * adequate blood supply * informed consent obtained exclusion criteria: * cannot take contrast-mri imaging * pregnant * combined with other malignant tumor (except basal cell carcinoma of skin)
addressing discrimination and mistrust among black men: phase iii

Conditions: HIV

Summary: the purpose of this research study is to test a group program aimed at improving the quality of life of hiv+ black men who have sex with men. the group will address ways to cope with discrimination and will help men examine mistrust that they may have about healthcare, including doctors and other healthcare providers and hiv medications. the aim is to provide a supportive community for hiv+ black men.

the aim of the study is to examine whether the pilot intervention can reduce mistrust and improve coping responses to discrimination among hiv positive black men who have sex with men (msm). we will conduct a small randomized pilot trial to test the feasibility of evaluation methods. all participants will attend a baseline, a first follow-up visit, and a final follow-up visit (each 3 months apart) at which they will take computer assessments. participants who are assigned to the intervention will attend 9 weekly intervention sessions after baseline and before the first follow-up. using a wait-list control design, control group participants from the first two cohorts will be offered the intervention after they complete the final follow-up visit. control group participants from the third cohort will be offered the intervention after they complete the first final-up visit and before they complete their final follow-up due to timing and budgetary restraints. we will hold three consecutive groups for our small randomized control trial. this means that we will be recruiting continuously; once we finish recruiting for the first group, we will start recruiting for the second group, and so on. each group has about 12 or 13 intervention and 7-11 control participants.

Eligibility criteria:
inclusion criteria: * 18 years-old or older * biologically male at birth * currently self-identifies as male * self-identifies as black or african american (mixed-race individuals will be eligible if they primarily identify as black or african american) * hiv-positive * reported having sex with men in lifetime * able and willing to provide informed consent exclusion criteria: * does not meet all of the inclusion criteria (e.g., if they are unable to provide proof of their hiv-positive status) * unable to provide informed consent * reports or presents evidence of severe mental health problems that require immediate treatment (e.g., psychotic symptoms, active suicidality at time of enrollment) or diagnosed mental disorder that would limit the ability to participate (e.g., dementia) * reports or presents evidence of cognitive impairments that limit the ability to comprehend the informed consent or to fully participate in the various parts of the study (assessments, intervention, homework).

Keywords: hiv discrimination coping medical mistrust hiv outcomes cognitive behavioral therapy
activity restriction for women with arrested preterm labor

Conditions: Preterm Birth

Summary: to test the hypothesis that activity restriction in women with singleton gestations and with arrested ptl would reduce the rate of ptb.

preterm birth (ptb) is a major cause of perinatal morbidity and mortality. worldwide, about 15 million babies are born too soon every year, causing 1.1 million deaths, as well as short- and long-term disability in countless survivors. preterm labor (ptl) is the final pathway for about 50% of all ptb. tocolytic agents are drugs that can slow or stop labor contractions in the attempt to delay births preceded by ptl. primary tocolysis is defined as tocolysis given on initial presentation of women with ptl. in most of these women, ptl stops, but as their risk of ptb remains high, some have advocated use of maintenance tocolysis, i.e. tocolysis after arrested ptl. so far, no maintenance tocolytic agent has been shown to be beneficial in preventing ptb. some authors have also advocated the use of activity restriction in women with arrested ptl to reduce the risk of ptb. activity restriction is defined as the following forms of activity restriction: pelvic rest and prohibition of sexual activity, reduction of work and/or non work activity. so far, the benefit of activity restriction in women with arrested ptl is still subject of debate and no randomized controlled trial (rct) has been undertaken. thus, the aim of this trial is to test the hypothesis that activity restriction in women with singleton gestations and with arrested ptl would reduce the rate of ptb.

Eligibility criteria:
inclusion criteria: * singleton gestations; * arrested ptl; * tvu cl \< 25 mm; * 18 years to 50 years. exclusion criteria: * multiple gestations; * symptoms of ptl (not arrested ptl); * tvu cl ≥ 25mm; * premature rupture of amnion and chorion membranes (prom) at the time of randomization; * cerclage in situ at the time of randomization; * pessary in situ at the time of randomization; * vaginal bleeding at the time of randomization; * women who are unconscious, severly ill, mentally handicapped; * women under the age of 18 years or over the age of 50 years.
hand-10 questionnaire in children with unilateral cerebral palsy

Conditions: Cerebral Palsy (CP); Hand Functions

Summary: a turkish version of the hand-10 (unabbreviated) questionnaire has been developed, but its validity and reliability in cerebral palsy has not yet been proven. in particular, questioning daily life activities with visual content can make a significant contribution to evaluating hand functions and independence. therefore, the aim of this planned study was to investigate the validity and reliability of the hand-10 questionnaire in cerebral palsy.

the hand-10 questionnaire was developed to evaluate upper extremity problems. it is a clear, short, and visual questionnaire that the individual fills out on their own. it includes shoulder, elbow, wrist, and hand functions. the developers of the questionnaire stated that it can be easily applied to children due to its visual explanations. pictorial materials make the survey more understandable for the elderly and children. visual content makes participation more enjoyable, especially for children. due to its visual content, hand10 has been proven to be applicable to the elderly and children. for its high accuracy, it is recommended to analyze its validity in specific diseases in previous studies. however, there are only two studies to date that have shown its validity for patients with lateral epicondylitis and trigger finger. due to its clarity and visuals, it can make a significant contribution to the assessment methods used especially in children with cerebral palsy. therefore, investigating its validity and reliability in children with cerebral palsy can provide support to the literature and professionals working in this field.

Eligibility criteria:
inclusion criteria: * children between the ages of 6-15, diagnosed with unilateral cerebral palsy, * with sufficient cognitive status to understand assessment instructions * with reading skills exclusion criteria: * those with health problems not related to cerebral palsy, * neglect of the affected extremity, * serious visual problems, * undergoing botox or orthopedic surgery in the last 6 months, * not willing to participate in the study

Keywords: cerebral palsy hand functions questionnaire validation disability