Kornimate/medical-research-clean huggingface dataset:
natural history study of patients with centronuclear myopathies

Conditions: Centronuclear Myopathy

Summary: this is a prospective, longitudinal study of the natural disease course intended to recruit approximately 60 patients with centronuclear myopathies (cnm) in europe and the united states. the duration of the study, including the enrollment period, will be approximately 4 years. data from the study will be used to characterize the natural disease course of cnm, to identify prognostic variables of the disease and to determine the best outcome measure(s) for the evaluation of future therapeutic approaches.


Eligibility criteria:
inclusion criteria: * 1. clinically symptomatic male or female subjects of all ages (newborns included) with a cnm resulting from a documented mutation in the mtm1 or dnm2 gene. * 2. a written, signed and dated informed consent must be provided to participate in the study. for subjects \<18 years, consent of parent(s)/legal guardian(s) is required; informed assent can be obtained from the child according to local regulations. * 3. willing and able to comply with all protocol requirements and procedures. exclusion criteria: * 1. participation in any other interventional study. participation in a previous study should be completed at least 4 weeks before the first study visit. * 2. currently undergoing or has undergone previous gene therapy or other therapy for cnm. * 3. current or past abuse of alcohol or recreational/narcotic drugs (with the exception of caffeine and nicotine), which in the investigator's opinion would compromise the subject's safety and/or compliance with the study procedures. * 4. current or relevant history of physical or psychiatric illness, that would make the subject unlikely to comply with the study procedures. (note: subjects in a wheelchair are not to be excluded). * 5. subject is mentally incapacitated, or parent(s)/legally-authorized representative are legally incapacitated or have limited legal capacity, or have lack of mental capacity to fully understand the protocol requirements and complete all study required procedures.

Keywords: muscular diseases myopathies, structural, congenital musculoskeletal diseases neuromuscular diseases nervous system diseases
early fluid resuscitation with balanced hes 130/0.4 [6%] in severe burn injury

Conditions: Burns

Summary: rct colloids versus cristalloids only in severe burn victims. * trial with medicinal product


Eligibility criteria:
inclusion criteria: * deep burn injury more than 15% of total body surface area (2nd and 3rd degree burns) * informed consent (via deferred consent, if necessary, according to swiss hmg § 55 and 56). * patients or relatives can understand the study information and the provided information in german language exclusion criteria: * patients expected to succumb within the next 24 to 36 hours, i.e. whole body burn trauma, palliative care situation * pregnancy * no informed consent * known allergic reaction to hes * patients or patient's relatives, who do not understand the german language and therefore the study information, can not be provided in a legally correct manner. * patients with contraindications for balanced 6% hes 130/0.4, i.e. heart failure, pulmonary edema, intracerebral bleeding, acute renal failure, severe hypernatraemia and other severe electrolyte imbalances, severe von-willebrand syndrome and acute liver failure (see "arzneimittelkompendium der schweiz® - documed")
comparison of duodenal stenting vs transpyloric and duodenal stenting for malignant obstruction

Conditions: Duodenal Obstruction

Summary: malignant duodenal obstruction in patients not fit for surgery is treated by placing enteral stents during endoscopy. these patients may also have poor gastric motility. hence bridging the pyloric opening with the stent along with the duodenal obstruction may deliver better symptomatic improvement. both approaches are commonly clinically practiced but no formal comparative studies have been done to compare which one is better.

enteral self-expanding metal stents are routinely used to palliate malignant gastric outlet obstruction (pancreas cancer, duodenal cancer, gastric cancer and metastasis) in patients not fit for surgical bypass. the technical success in placing these stents approaches \~100% and many of these procedures can be performed in an outpatient setting. however the functional success (patient's ability to eat) is much lower than the technical success. one of the major reasons for this discrepancy is these patients are on narcotics, which are known to be associated with poor gastric motility. at the discretion of the gastroenterologist, fda approved enteral stents are placed either completely within the duodenum bridging the obstruction or placed across the pyloric opening besides bridging the duodenal obstruction. the significance of this study is to determine if trans-pyloric extension of an intra-duodenal stent facilitates better gastric emptying compared to an intra-duodenal stent without trans-pyloric extension.

Eligibility criteria:
inclusion criteria: 1 - confirmed diagnosis of cancer 2. evidence of a single small bowel obstruction 3. considered palliative (can be on narcotics, chemotherapy, and/or radiation therapy) 4. not a surgical candidate 5. \>18 years of age 6. able to give consent 7. eligible for endoscopy (medically fit) 8. able to traverse past obstruction with a guidewire exclusion criteria: 1 - \<18 years of age 2. unable to give consent 3. pregnant 4. have evidence of multiple sites of obstruction in the small bowel 5. have evidence of duodenal obstruction secondary to gastric cancer 6. ineligible for endoscopy (due to comorbidities or acuity of illness) 7. unable to traverse past obstruction with a guidewire
newborn genomic sequencing pilot study

Conditions: Genetic Disease

Summary: the goal of this clinical trial is to test a new method for newborn screening using whole genome sequencing, called beginngs. newborns who are not suspected of having genetic diseases and who are admitted to the nicu at rady children's hospital, san diego, will be enrolled. the main questions this study aims to answer are: * what is the diagnostic yield of diagnostic whole genome sequencing (dwgs) in this population? * what is the diagnostic sensitivity and specificity of beginngs and whole exome sequencing (wes) as compared to dwgs? * what are the potential issues related to implementing dwgs in this population? enrolled newborns will have a blood sample taken and will receive three tests: * dwgs * beginngs * wes

newborn screening (nbs) by testing dried blood spots (dbs) identifies newborns with a few diseases for which effective treatments are available to enable treatment at or before symptom onset. because nbs improves outcomes in these diseases, it is performed on almost all us babies. the current federal recommended nbs list is limited to 35 conditions and identifies \~6,600 affected children per year. in genetic diseases not screened by nbs, however, outcomes remain poor because of delays in diagnosis and treatment. the investigators recently developed a system for nbs for 434 severe, childhood genetic diseases for which effective treatments are available using whole genome sequencing (wgs), called beginngs. retrospective studies showed beginngs to have a true negative rate (specificity) of 99.7% and true positive rate (sensitivity) of 88.8%. the investigators now propose to undertake a first prospective study in newborns admitted to the neonatal intensive care unit (nicu) at rady children's hospital, san diego (rchsd) to compare the sensitivity and specificity of beginngs with that of standard, diagnostic rapid whole genome sequencing (dwgs) and whole exome sequencing (wes). this study is in preparation for larger, future clinical trials.

Eligibility criteria:
inclusion criteria: * neonates less than or equal to 10 days old who are admitted to the rchsd nicu. exclusion criteria: 1. neonates who have enrolled in another clinical study at rady children's institute for genomic medicine or in whom dwgs has been ordered or is being considered. 2. neonates whose mother is less than 18 years of age. 3. neonates who are wards of the state. 4. neonates whose parent/legal guardian is unable to provide consent.
exploratory efficacy study of guaifenesin in upper back pain

Conditions: Upper Back Pain

Summary: explore the clinical efficacy of 7-day treatment regimens of guaifenesin in order to investigate the evidence that guaifenesin is an effective treatment for upper back/neck/shoulder muscle spasm/stiffness and pain.


Eligibility criteria:
inclusion criteria: * paticipant presents with a new episode of acute upper back/neck/shoulder pain and muscle spasm that: 1. is at least 30 days from previous episode. 2. has an onset occurred within 48 hours of visit 1. 3. has symptoms consistent with a clinical diagnosis as per the physical examination at visit 1. 4. has a subject-rated muscle spasm score and a pain score of at least 40 out of 100 on a 100mm vas. * paticipant has a normal neurological examination. exclusion criteria: * medical history: a) paticipant has a known or suspected intolerance or hypersensitivity to guaifenesin or methocarbamol (or closely related compounds) or any of their stated ingredients. b) paticipant has history of upper back pain with active hypersensitive spots - trigger point in the muscles of upper back/neck/shoulder/maxillofacial regions. c) paticipant has a current or recent history of liver and/or kidney disease, neck/back/shoulder injuries, spinal disc disease, myocardial infarction (\<12 months from visit 1) and osteoporosis. d) subjects have history of other chronic pain e.g. headache, osteoarthritis, arthritis, lower back pain etc. * medications: a) paticipant has used a muscle relaxant or narcotic within 2 weeks of visit 1. b) paticipant has used monoamine oxidase inhibitors or selegiline within 2 weeks of visit 1 c) currently using tricyclic antidepressants, lithium, anticoagulants, or tramadol. * paticipant is involved in a workers compensation case. * paticipant is unlikely to refrain from using analgesics, nsaids, and other muscle relaxants for the duration of the study, with the exception of cardioprotective doses of aspirin (i.e., ≤ 325 mg/day). * paticipant is unlikely to refrain from physical therapy, massage therapy, acupuncture, heat treatment, spas, etc for the duration of the study.

Keywords: upper back/neck/shoulder muscle spasm
phototherapy remediation of black stain

Conditions: Dental Deposits; Tooth Discoloration; Dental Prophylaxis

Summary: this study investigates the remediation of black-stained tooth surfaces using antimicrobial photodynamic therapy. in this crossover trial, a cohort of patients received standard dental prophylactic cleaning to remove the black stain. later, following the washout period, (reappearance of the black stain) received photodynamic therapy in addition to the standard cleaning. stain-free period in each of the arms was the main outcome variable. additional microbial plaque testing was also carried out.

the standard treatment practice for black dental stain is dental prophylaxis, however this is often harmful to the enamel surface and can result in tooth sensitivity. in this study we aim to explore the application of antimicrobial photodynamic therapy (apdt) for the treatment of adult black dental stain. apdt has demonstrated positive results in the treatment of periodontitis. patients received an oral examination and the extent of black stain was scored (score 1, pigmented spots or incomplete lines parallel to the gingival margin, score 2: solid lines pigmented, readily observable and limited to the cervical third of the tooth surface, score 3: pigmentation extending beyond the cervical third). the patients' oral cavity was photographed in sextants, along with a complete picture of the jaw (lingual plane) and the maxillary arch (palatal plane). for bacterial analysis, 1mg of black plaque was collected by scraping with a plastic scaler from buccal tooth surfaces taking care to avoid unnecessary removal of enamel hydroxyapatite. the first dental prophylaxis was performed to remove the black stain. patients were reviewed monthly and in each visit, were photographed as previously described, and the level of pigmentation scored according to the mentioned criteria. the monthly visits were continued until the black stain score returned to the initial value. the period between the first and the last appointment was noted. at this point, each volunteer received dental prophylaxis once again, followed by the application of apdt. antimicrobial photodynamic therapy was applied to 31 orally healthy volunteers with black stained teeth, using the glo home whitening device (glo™ science llc, new york, usa) in 2 cycles of 8 minutes. outcome variables were area, color and time taken to reappearance of black stain following dental prophylaxis with and without antimicrobial photodynamic therapy. differences in bacterial composition of black stain (specifically aggregatibacter actinomycetemcomitans, tannerella forsythia and porphyromonas gingivalis) were analyzed dna extracted from plaque samples. following the application of apdt, patients were reviewed monthly until the black stain score returned to the initial value.

Eligibility criteria:
inclusion criteria: * volunteers with black stained (plaque on) teeth exclusion criteria: * children * pregnant women * volunteers having completed antibiotic therapy during 15 days prior to the start of the study

Keywords: photodynamic therapy black-stain pigmented bacteria tannerella forsythia
evaluate, safety and tolerability of intraprostatic prx302 administration, low to intermediate risk prostate cancer

Conditions: Prostate Cancer

Summary: to evaluate safety, tolerability and potential efficacy of prx302 effect on clinically significant localised low to intermediate prostate cancer.

a single center, open label, phase iia, idea, development, exploration, assessment, long-term follow-up (ideal) prospective development study. the study will treat approximately 20 men who meet the eligibility criteria, and give written informed consent. safety and tolerability will be assessed post-treatment at 2 days (phone call), 2 weeks, 6 weeks, 12 weeks, 24 weeks and 26 weeks. potential efficacy will be assessed by biopsy at 24 weeks and imaging (mri) at 2 weeks, 12 weeks and 24 weeks and psa outcomes at 12 and 24 weeks.

Eligibility criteria:
inclusion criteria: * men aged ≥40 years and life expectancy of ≥10 years. * serum prostate-specific antigen (psa) ≤15 ng/ml. * transperineal prostate biopsy within 12 months prior to dosing, with a clinically significant lesion correlating with an mpmri visible lesion. * radiological stage t1-t2 n0 mx/m0 disease. * a visible lesion on mpmri that is accessible to prx302 transperineal injection. exclusion criteria: * previous radiation therapy to the pelvis. * androgen suppression or anti-androgen therapy within the 12 months prior to dosing, for prostate cancer. * use of a 5-alpha reductase inhibitor within the 3 months prior to dosing. * evidence of metastatic disease or nodal disease outside the prostate on bone scan or cross-sectional imaging. * inability to tolerate a transrectal ultrasound (trus). * known allergy to latex or gadolinium (gd). * previous electroporation, radiofrequency ablation, high-intensity focused ultrasound (hifu), cryosurgery, thermal or microwave therapy to treat cancer of the prostate. * unable to have pelvic mri scanning (severe claustrophobia, permanent cardiac pacemaker, metallic implant, etc. likely to contribute significant artifact to images).

Keywords: mri lesion transperineal injection prostate biopsies
treadmill training effects on cardiorespiratory endurance and constipation in children with cerebral palsy

Conditions: Cardiopulmonary

Summary: this study examines the impact of treadmill training on cardiorespiratory endurance and constipation in children with cerebral palsy (cp). treadmill training is evaluated for its potential to improve cardiovascular fitness and alleviate constipation through its rhythmic movements. the findings shed light on the broader health benefits of treadmill interventions beyond motor function improvement in children with cp

this research investigates the effects of treadmill training on two distinct yet interconnected aspects of health in children with cerebral palsy (cp): cardiorespiratory endurance and constipation. the research employs a longitudinal design, with participants undergoing a structured treadmill training program tailored to their individual needs and abilities. outcome measures include objective assessments of cardiorespiratory fitness (e.g., exercise stress tests, spirometry) and subjective evaluations of constipation symptoms (e.g., bowel diaries, symptom questionnaires). additionally, the study examines potential correlations between improvements in cardiorespiratory endurance and changes in constipation severity, exploring the interplay between physical fitness and gastrointestinal function in children with cp. by investigating the effects of treadmill training on both cardiorespiratory endurance and constipation, this research aims to provide a comprehensive understanding of the broader health benefits associated with exercise interventions in children with cerebral palsy. the findings may inform the development of integrated rehabilitation programs that address multiple aspects of health and well-being in this population, ultimately improving overall quality of life and functional outcomes.

Eligibility criteria:
inclusion criteria: * age 4 to 12 years. * children having 1, or 2 on gmfcm. * children with spastic diplegic cerebral palsy. * children with well cognitive behavior. * children having 1 to 2 level on constipation assessment scale. * defecation frequency less than 3 time a week. exclusion criteria: * misdiagnosed or not conform to cerebral palsy. * children should be medically stable uncontrolled seizures. * the children with mental retardation or other neurological disorders. * children with taking medication like muscle relaxant or laxative

Keywords: motor function
open pilot trial of tes for depression

Conditions: Major Depression

Summary: this study will investigate whether using oscillating tes (random noise stimulation) or intermittent tdcs will have greater antidepressant effects in depressed subjects, compared to standard tdcs.


Eligibility criteria:
inclusion criteria: * subject meets criteria for a dsm-iv major depressive episode. exclusion criteria: * diagnosis (as defined by dsm-iv) of: any psychotic disorder (lifetime); bipolar disorder; eating disorder (current or within the past year); obsessive compulsive disorder (lifetime); post-traumatic stress disorder (current or within the past year); mental retardation. * subject has not responded adequately to standard tdcs or is not appropriate for trial of standard tdcs (eg depression too chronic in duration) * total madrs depression score of 20 or more * history of drug or alcohol abuse or dependence (as per dsm-iv criteria) within the last 3 months (except nicotine and caffeine). * inadequate response to ect in the current episode of depression. * subject is on regular benzodiazepine medication which it is not clinically appropriate to discontinue. * subject requires a rapid clinical response due to inanition, psychosis or high suicide risk. * neurological disorder or insult, e.g., recent stroke (cva), which places subject at risk of seizure or neuronal damage with tes. * subject has metal in the cranium, skull defects, or skin lesions on scalp (cuts, abrasions, rash) at proposed electrode sites. * female subject who is pregnant. however, women of child bearing age are eligible if they have the pregnancy test but will be excluded if they are sexually active and not using reliable contraception (urine test for pregnancy will be used if appropriate).

Keywords: depression transcranial direct current stimulation (tdcs) random noise stimulation oscillatory tdcs intermittent tdcs
lung cancer case registry and tissue resource

Conditions: Lung Cancer

Summary: potential participants in the lung cancer case registry will be all patients diagnosed or evaluated for possible treatment of lung cancer at the cancer research and treatment center (crtc) and new mexico veterans health care system (nmvhcs).

lung cancer is the most common cause of cancer death in the us in both men and women. this research study will enroll lung cancer patients into a registry to study changes in dna (or genetic material) that may be associated with development of lung cancer or other lung diseases such as chronic obstructive pulmonary disease (copd). we will also study the role of eating habits on lung cancer diagnosis and treatment, as well as the effects of the disease and treatment on quality of patient's lives.

Eligibility criteria:
inclusion criteria: * potential participants in the lung cancer case registry will be all patients diagnosed or evaluated for possible treatment of lung cancer at the crtc and nmvhcs. recruiting will primarily be performed through the multidisciplinary chest clinics held weekly at each hospital. these clinics are staffed by the principal investigator (pi) and reza mehran, md, a thoracic surgeon at both hospitals and a co-investigator on this submission. approximately 90% of all lung cancer cases diagnosed at the nmvhcs, and a somewhat smaller proportion of cases treated in the cancer center, are seen in these clinics. additional cases will be identified through review of new patient visits to each hospital with a diagnosis of lung cancer; drs. crowell and mehran also maintain weekly contact with pathology and oncology services at both facilities, which will facilitate case identification at nmvhcs and university hospital. finally, patient contact records will be correlated with tumor registry records at both hospitals, which will identify any cases not captured through the above mechanisms. although these cases may be identified several weeks after initial diagnosis, all attempts will still be made to contact the patient and family for enrollment into the registry. most spanish-speaking patients served by the crtc and the nmvhcs are bilingual, and a language barrier has not been a problem in other similar studies conducted by the pi. however, spanish-speaking personnel are available to obtain consent and answer any questions if necessary. exclusion criteria: * patients will be excluded if they cannot provide written informed consent.

Keywords: lung diseases cancer