Kornimate/medical-research-clean huggingface dataset:
phase 2 study of edg-5506 in children and adolescents with duchenne muscular dystrophy previously treated with gene therapy

Conditions: Duchenne Muscular Dystrophy

Summary: the fox study is a 2-part, multicenter, phase 2 study of safety, pharmacokinetics, and biomarkers in children and adolescents with duchenne muscular dystrophy previously treated with gene therapy including a randomized, double-blind, placebo-controlled part a, followed by an open-label part b.

the edg-5506-215 protocol was amended to include an additional dose cohort and enroll additional participants across select cohorts. fox is a 2-part, multi-center, phase 2 study to evaluate the effect of sevasemten (edg-5506) on safety, pharmacokinetics and biomarkers of muscle damage in approximately 48 children and adolescents with duchenne muscular dystrophy treated with oral, once-daily sevasemten. this study will have up to a 4-week screening period, a 12-week randomized double-blind, placebo-controlled treatment period (part a), followed by a 40-week open-label extension period (part b) . approximately forty-eight (48) participants aged 6 to 17, inclusive, will be randomized to sevasemten or placebo in a 2:1 ratio. three dose cohorts (cohort 1, cohort 2 and cohort 3) of approximately 12 participants each will be enrolled. approximately 12 additional participants may be added to 1 of these cohorts following data monitoring committee (dmc) review of emerging data.

Eligibility criteria:
key inclusion criteria: * aged 6 to 17 with a documented mutation on the dmd gene and phenotype consistent with dmd. * prior receipt of an aav-based gene therapy (≥ 2 years after study drug administration in an open-label study or ≥ 3 years after randomization in a randomized study). * able to complete stand from supine in ≤ 8 seconds at the screening visit and able to perform the 4-stair climb in \< 10 seconds at the screening visit. * body weight ≥ 15 kg at the screening visit. * treatment with a stable dose of corticosteroids for a minimum of 6 months prior to the baseline visit. key exclusion criteria: * medical history or clinically significant physical exam/laboratory result that, in the opinion of the investigator, would render the participant unsuitable for the study. this includes venous access that would be too difficult to facilitate repeated blood sampling. * screening visit cardiac echocardiography showing left ventricular ejection fraction (lvef) \< 40%. * receipt of an investigational drug (other than the aav-based gene therapy per inclusion criteria) within 30 days or 5 half-lives (whichever is longer) of the screening visit in the present study. * receipt of an exon-skipping therapy within 6 months prior to the screening visit.

Keywords: duchenne muscular dystrophy
amodiaquine-artesunate & artemether-lumefantrine efficacy in burkina faso

Conditions: Malaria

Summary: this is a two-arm study aiming at recruiting 150 patients to assess the efficacy of amodiaquine-artesunate (asaq) and artemether-lumefantrine (al) in patients with a microscopy positive diagnosis of malaria in nanoro, burkina faso and assess the performance of the rapid diagnosis tests (rdts) compared to the microscopy.

study background and purpose * resistance to usual drugs was widespread and has required a change of the malaria treatment by several countries * several countries have changed their first-line treatments to acts; mainly al and asaq. \& have adopted the use of rdts prior to treatment * indeed, this contributes to decrease the number of unnecessary treatments and thus improve the management of malaria cases. * in february 2005, burkina faso changed its national drug policy from chloroquine to amodiaquine-artesunate (asaq) and artemether-lumefantrine (al) * the country has also implemented the strategy of using the rapid diagnosis tests (rdts) for the diagnosis of malaria prior to treatment * though endemic countries are being encouraged to implement rdts, choosing a particular rdt is not easy as several brands are available on the market. * in addition, little information on the performance of rdts in africa is available and recently quality problems have been reported with some rdts. * in this context, it is important to locally assess the performance of rdts compared with the microscopy, which is the gold standard for the malaria diagnosis and to assess the efficacy of the new drugs used for malaria treatment this is a phase iv two-arm randomized open-label study aiming at recruiting 150 patients to assess the efficacy of asaq and al in patients with a microscopy positive diagnosis of malaria in nanoro, burkina faso and assess the performance of the rdt compared to the microscopy

Eligibility criteria:
inclusion criteria: * age above 6 months, * eight above 5 kg; * positive blood slide (parasitaemia ≥ 2,000/μl to 200,000/μl) with plasmodium falciparum monospecific infection ; * fever (axillary temperature above 37.5 °c) or history of fever in the preceding 24 hours; * haemoglobin value above or equal 5.0 g/dl * signed informed consent; * willingness and ability to comply with the study protocol for the duration of the trial. exclusion criteria: * participation in any other investigational drug study (antimalarial or others) during the previous 30 days * known hypersensitivity to the study drugs * severe malaria * danger signs: not able to drink or breast-feed, vomiting (\> twice in 24hours), recent history of convulsions (more than 1 in 24h), unconscious state, unable to sit or stand; * known intercurrent illness or any condition (cardiac, renal, hepatic diseases) which would place the subject at undue risk or interfere with the results of the study. * severe malnutrition (defined as weight for height less than 70% of the median nchs/who reference) * known pregnancy

Keywords: treatment, diagnosis
a study of ranpirnase(onconase®) to pemetrexed plus carboplatin in patients with non-squamous non-small cell lung cancer

Conditions: Non-Small Cell Lung Cancer

Summary: the purpose of the trial is to determine the rate of improvement in objective tumor response, following the addition of ranpirnase to ongoing pemetrexed-carboplatin chemotherapy, for patients with sd or pr following 2 cycles of doublet chemotherapy.


Eligibility criteria:
inclusion criteria: * age greater than 18 years * ecog less than 2 * estimated survival of greater than 12 weeks exclusion criteria: * prior systemic chemotherapy for locally advanced or metastatic nsclc * use of any investigational agent within 28 days prior to registration * known hypersensitivity to any of the study drugs * brain metastases or leptomeningeal disease * history of prior malignancy within the past three years

Keywords: non small cell lung cancer, nsclc, lung cancer previously untreated non-squamous, non-small cell lung cancer
cognitive behavior therapy for somatization disorder

Conditions: Somatoform Disorders

Summary: this study will examine the long-term effects of cognitive behavioral therapy on the physical symptoms, functioning, and health care utilization of people with multiple medically unexplained physical symptoms.

somatization disorder is a chronic psychological condition that causes numerous physical complaints for which no underlying physical problem can be identified. the disorder often lasts for several years and results in substantial functional impairment. the physical complaints most frequently involve chronic pain and problems with the digestive, nervous, and reproductive systems. neither pharmacological nor psychosocial treatments for this disorder have been successful in suppressing symptoms. cognitive behavioral therapy (cbt) is a treatment that focuses on maladaptive patterns of thinking and the beliefs that underlie such thinking. this study will examine the long-term effects of cbt on the physical symptoms, functioning, and health care utilization of people with somatization disorder. participants in this open label study will be randomly assigned to receive either cbt supplemented with augmented standard medical care (asmc) as indicated by a psychiatric consultation letter or asmc alone. participants assigned to cbt plus asmc will receive cbt for 10 weeks. somatic symptomatology, functional impairment, and health care costs will be assessed at study visits at baseline and months 3, 9, and 15. the visits at months 9 and 15 will assess specifically the long-term efficacy of the treatment.

Eligibility criteria:
inclusion criteria: * meets dsm-iv criteria for somatization disorder * available for follow-up over the ensuing 15 months * english-speaking exclusion criteria: * meets dsm-iv criteria for a psychotic disorder, organic brain syndrome, or psychoactive substance dependence * active suicidal ideation * unstable major medical condition * plans to engage in additional psychotherapy during the first 3 months after study entry * current use of any medication that has not been stabilized for the 2 months prior to study entry

Keywords: somatization disorder multiple medically unexplained symptoms cognitive behavior therapy mental health
first in human safety study of fx-322 in adults undergoing cochlear implantation

Conditions: Hearing Loss

Summary: this is a phase 1 safety study performed in male or female adult participants with an established diagnosis of severe to profound sensorineural hearing loss that meets the criteria for cochlear implantation and the participant has already chosen to undergo cochlear implant surgery.

approximately 12 participants will be enrolled in the study. assessed will be safety and tolerability of fx-322 administered by intratympanic injection. also assessed will be the fx-322 concentration in cochlear fluid (perilymph), the pharmacokinetic (pk) profile of fx-322 to determine the systemic exposure to fx-322.

Eligibility criteria:
inclusion criteria: 1. male or female adult subjects with an established diagnosis of severe to profound sensorineural hearing loss audiometric threshold of 80 db hl or poorer at 500 hz that meets the criteria for cochlear implantation and the subject has already chosen to undergo cochlear implant surgery. 2. willingness and ability to comply with scheduled visits, ear examination, drug administration plan, auditory and laboratory tests, study restrictions, and all study procedures. other protocol-defined inclusion criteria may apply exclusion criteria: 1. persistent perforation of tympanic membrane or other tympanic membrane disorder that would interfere with the delivery and safety assessment of an intra -tympanic medication or reasonably be suspected to affect tympanic membrane healing after injection. 2. any conductive component defined as air-bone gaps \>10 db at two or more frequencies. other protocol-defined exclusion criteria may apply
establishing a physical activity referral scheme for people with noncommunicable diseases in the german healthcare system

Conditions: Non-Communicable Chronic Diseases

Summary: the bewegtversorgt project involved co-producing a physical activity referral scheme (pars) for inactive persons with underlying non-communicable diseases. various relevant actors of the german healthcare system (e.g., representatives of physicians, health insurance providers, sports organizations, exercise representatives of exercise professions, representatives of patients) participated in the co-production process. the purpose of this study is to evaluate the effectiveness of co-produced pars in the erlangen-nuremberg-fürth region. local general practitioners and exercise professionals will carry out the implementation of the pars in standard care. the cluster-randomized study includes two intervention arms; one group will receive specific support from physical activity experts to increase physical activity (pars). the control group will receive only the physician's advice and then continue to engage in physical activity on their own (paa). the participants will be followed up at 12 and 24 weeks.


Eligibility criteria:
inclusion criteria: * 18 years and older; living in the region of erlangen-nürnberg-fürth, germany; * at least one of the following existing non-communicable diseases (controlled type 2 diabetes mellitus; cardiovascular diseases; obesity (bmi ≥ 30 kg/m2); arthrosis in knee and/or hip); * not meeting german pa recommendations (less than 150 minutes/week of moderate-intensity aerobic activity or less than 75 minutes/week of vigorous-intensity aerobic activity or any other equivalent combination); * the person can safely participate in physical activities based on general practitioners' clinical judgment. exclusion criteria: * the person plans to leave the region of erlangen-nürnberg-fürth during the study period; * the person participating in another study with similar content; * the person is or plans to be absent for more than four weeks during the 12-week-intervention period; * person has cognitive impairments that prevent an effective communication with the general practitioner and the therapist; * persons with mental illness such as psychotic, substance abuse, mood, personality disorders; * unstable clinical situation or serious health impairments that prevent from undertaking physical activity safely (e.g., acute myocardial infarction, unstable angina pectoris, fever, terminal tumor diseases

Keywords: physical activity promotion physical activity referral scheme physician-initiated behaviour change
sdd for eradicating crkp carriage

Conditions: Carriers of Carbapenem-resistant Klebsiella Pneumonia

Summary: there is an urgent need to control the current national outbreak of carbapenem-resistent klebsiella pneumonia (crkp). in israel, the death rate among crkp carriers is 3.5 times higher than in carbapenem-sensitive klebsiella pneumonia carriers (44% vs. 12.5%, respectively). in the investigators' previous study: a randomized, double-blind, placebo-controlled trial of selective digestive decontamination (sdd) using oral gentamicin and oral polymyxin e for eradication of crkp carriage (infect control hosp epidemiol. 2012;33:14-19) the investigators have shown that the investigators' sdd regimen is effective for decolonization patients colonized with crkp. the investigators' assumption is that a higher dose of polymyxin e together with gentamicin (sdd drugs) for a prolonged period is needed to overcome the likelihood of a high rate of drug inactivation in the gut, thereby reaching crkp carriage eradication.


Eligibility criteria:
inclusion criteria: * provide a sighed and dated written informed consent document indicating that the subject (or a legally accepted representative) has been informed of all pertinent aspects of the study * hospitalized men or women at least 18 years of age * rectal swab culture positive for crkp in the last week * will be willing to initiate and remain on treatment, except for adverse events occurring exclusion criteria: * age less than 18 years. * pregnant women, lactating women. * a know allergy to one of the study drugs. * renal failure with creatinine clearance less than 50ml/min. * current treatment with iv gentamicin and/or iv polymyxin e * any safety, behavioral, clinical, or administrative reasons that, in the investigator's judgment, would potentially compromise study compliance or the ability to evaluate safety/efficacy
reduction of trauma-induced intrusions and amygdala hyperreactivity via non-invasive brain stimulation

Conditions: Intrusive Thoughts; Post Traumatic Stress Disorder

Summary: the study will focus on the modulation of intrusive memories via functional magnetic resonance imaging (fmri)-guided repetitive transcranial magnetic stimulation of the dorsolateral prefrontal cortex (dlpfc) directly after exposure to a traumatic video.

traumatic experiences can lead to posttraumatic stress disorder (ptsd) with clinical manifestations including intrusions, avoidance behavior, and hyperarousal. unlike most other psychological disorders, a ptsd diagnosis requires a clearly identifiable inciting event. as such, preventive interventions in recently traumatized individuals seem promising. in this randomized, placebo-controlled study the investigators explore the potential of individualized transcranial magnetic stimulation (tms) to reduce trauma-induced intrusive thoughts by altering functional connectivity between the dorsolateral prefrontal cortex (dlpfc) and amygdala. subjects will undergo a functional magnetic resonance imaging (fmri) session consisting of a resting state scan, an emotion recognition task and an anatomical scan at the beginning of the study (day 1). resting state data will be used to determine individualized tms targets for every subject, depending on functional connectivity between the dlpfc and amygdala. subsequently, the analogue trauma model will be used to induce intrusions in healthy subjects. subjects will be confronted with a video clip from the movie "irreversible" and they will complete online questionnaires in the following three days to measure intrusive thoughts, trauma disclosure (i.e. duration of conversations about the aversive movie) and sleeping quality. intermittent theta-burst stimulation (itbs) will be administered directly after the video clip and on the following two days (day 2-4). cognitive tasks will be applied before and after itbs sessions to examine short-term effects of itbs on a behavioral level. finally, subjects will undergo a second fmri session similar to the first one, to probe itbs-induced changes in functional connectivity and emotional processing (day 5).

Eligibility criteria:
inclusion criteria: * healthy subjects exclusion criteria: * current psychiatric illness * current psychiatric medication or psychotherapy * past ptsd diagnosis * mri contraindication (e.g. metal in body, claustrophobia) * pregnancy

Keywords: amygdala fmri intrusive thoughts post traumatic stress disorder tms
is reduces rib fracture complications

Conditions: Hemothorax; Traumatic; Rib Fractures; Rib Trauma

Summary: the incentive spirometer (is) is a mechanical device that helps lung expansion. it is commonly used to prevent postoperative lung atelectasis and decreased pulmonary complications in patients who received cardiac, lung, or abdomen surgery.this study aimed to explore the effect of the is on the improvement of lung function and decrease in pulmonary complication rate in rib fractures patients.

adult patients (\>18 years old) seen at our institution between june, 2014 until may, 2017 with traumatic rib fractures were included. for inclusion, a patient had to have at least one rib fracture as detected by chest x-ray (cxr) or computed tomography scan. we excluded patients with unconsciousness, history of chronic obstructive pulmonary disease, asthma, or an injury severity score (iss) ≥ 16. patients were divided into two groups using a randomized envelope technique: in the study group, patients were advised to use the is and in the control group patients did not received the is. all patients were managed with the same oral analgesic protocol. a cxr was obtained on the 1st and 5th day of admission and performed by a radiologist. the pulmonary function test (pft) was administered on the 2nd and 7th day of admission and performed by a pulmonologist. a numeric rating scale (nrs) was used to define the severity of chest pain on the 1st and 5th day of admission.

Eligibility criteria:
inclusion criteria: * at least one rib fracture as detected by chest x-ray (cxr) or computed tomography scan exclusion criteria: * unconsciousness, * history of chronic obstructive pulmonary disease * asthma * injury severity score (iss) ≥ 16

Keywords: incentive spirometer hemothorax; traumatic trauma rib fractures
online wellness intervention in medical students

Conditions: Stress, Psychological; Mind Body Therapy; Wellbeing

Summary: medical students are at high risk of stress. this project will test how well a 12-week stress reduction program works to reduce stress, anxiety and depression and improve well-being. the program is delivered online and each week is made of an introduction video, 7-8 minutes of yoga, 5-7 minutes of meditation and nutrition tips. at the beginning and the end of the 12-week research study, we will be using surveys to ask students about their stress, anxiety, depression, sense of control over their own life, quality of life, and pain levels. after the program, the research team will conduct interviews with the medical student to allow them to share their other feedback about the program. the researchers will also send surveys to the medical students one month after the program ends to asses their continued satisfaction with and adherence to the program.


Eligibility criteria:
inclusion criteria: - medical students in any year of medical school exclusion criteria: * hospital anxiety and depression scale (hads) depression score \>10 * active psychosis * post-traumatic stress disorder or frequent suicidal ideation * major medical co-morbidity * inability to provide written informed consent
efficacy of herbal galactagogue silitidil after preterm birth

Conditions: Breast Milk Production; Human Milk Feeding; Hypogalactia; Preterm Infant Nutrition

Summary: after preterm birth mothers often suffer from hypogalactia. herbal galactogogues can be used in order to increase milk production. in this double-blind, randomized, placebo-controlled clinical trial the effect of silitidil, an extract from milk thistle, on the postpartal milk production in mothers of very premature newborns is investigated.

mothers of preterm infants born at a gestational age \<= 32 wg are asked to participate in this double-blind, randomized, placebo-controlled interventional trial, where they will receive a preparation of the herbal galactogogue silitidil or placebo over an intervention period of 3 weeks. the influence of the galactagogue will be investigated by monitoring daily milk production before, during and 1 week after cessation of the intervention. as secondary outcome parameters weight gain, the progress of enteral feeding, and the percentage of human milk feeding on total enteral feeding volumes will be monitored in the newborn infants. also the breast milk composition (in terms of fatty acids, protein and carbohydrates content) will be investigated and compared between study groups. saliva and urine probes will be collected at specific time points to survey hormonal reactions during the various phases of lactation and the relation of maternal hormone levels on milk production.

Eligibility criteria:
inclusion criteria: * mothers after preterm birth \<= 32 weeks of gestation * age of the newborn 1 - 5 days * human milk feeding intended exclusion criteria: * ingestion of other galactagogues * maternal chronical disease requiring therapy (e.g. severe hypothyreosis, preexistent arterial hypertension, type i or 2 diabetes, chronic inflammatory bowel disease, severe bronchial asthma, pollinosis) * medication influencing prolactine levels * mastitis at time of enrollment * mammary tumors or surgery influencing milk production

Keywords: galactagogue milk thistle silymarin silitidil