Kornimate/medical-research-clean huggingface dataset:
reliability and validity of the active-mini for quantifying movement in infants with spinal muscular atrophy

Conditions: Spinal Muscular Atrophy

Summary: the purpose of this study is to investigate the measurement properties of the ability captured through interactive video evaluation-mini (active-mini) for quantifying movement in infants with spinal muscular atrophy (sma). specifically, i will investigate within-day and between-day test-retest reliability and calculate the minimal detectable change of the active-mini. additionally, i will determine the concurrent validity of the active-mini with the children's hospital of philadelphia infant test for neuromuscular disease (chop intend) and the construct validity of the active-mini in infants with sma using a known group methodology.

this study was a cross-sectional, repeated measure design, investigating the test-retest within- and between-day reliability and the convergent construct validity and the known-groups method construct validity of the active-mini. the group variable included two levels (non-rolling infants with sma and function matched non-rolling typically developing infants). the dependent variables included a predicted chop intend score determined by data captured by the active-mini and the chop intend extremity score. the dependent variables were collected at two time points over two days at a minimum of 24 hours and no more than 30 days between collections. variables were collected in a standard order of assessment. all participants underwent all assessments as set out in the procedures. based on results from pilot data, the conclusion was made that the active-mini could quantify infant movement parameters well enough to warrant further investigation. these data were important in establishing the feasibility of the specific aims of this research, because they provide initial validation of the ability of active-mini to quantify basic infant movement parameters

Eligibility criteria:
inclusion criteria: - participants will be included in this study if they: * are non-rolling at time of enrollment * age birth to 25 months * have no concomitant system pathology that would limit clinical evaluation. these inclusion criteria are set for both affected babies and healthy controls. exclusion criteria: participants will be excluded from this study if they: * demonstrate evidence of renal dysfunction, central nervous system damage, neuro-degenerative or neuromuscular disease other than sma type i or ii; * require mechanical ventilation of any type \> 16 hours per day.
acarbose and older adults with postprandial hypotension

Conditions: Other Specified Hypotension; Syncope

Summary: the current proposal will determine if blocking carbohydrate intake in the small intestine with acarbose can be a possible therapy for older adults with (pph) post prandial hypotension (a drop of blood pressure after eating), which can result in falls.

blocking the absorption of carbohydrates at the brush border of the small intestine with acarbose (an alpha-glucosidase inhibitor) seems a promising possibility as a potential therapeutic agent. although designed as a second-line diabetes drug, this medication has very little risk of hypoglycemia in older adults. in fact the risk of hypoglycemia is extremely low even in patients concurrently taking concurrent hypoglycemia agents (including insulin), and there is almost no risk of hypoglycemia in subjects not on other diabetes medications. acarbose suppresses postprandial glycemia by slowing small intestinal digestion and absorption of carbohydrate, and has been shown to slow gastric emptying acarbose has yet to be examined in a prospective fashion in older adults, despite the prevalence of pph in this patient population. preliminary, pilot work done in our laboratory on older adults with pph has demonstrated that the hypotensive response over 90 minutes to a standardized meal was significantly reduced by the administration of acarbose

Eligibility criteria:
inclusion criteria: * be 65 years of age or older, * be a non-smoker for at least 5 years * be referred to the falls clinic at vancouver general hospital * have a folstein test of cognition \> 25/30 to ensure meal log-book compliance exclusion criteria: * no oral or swallowing issues that would prevent a meal test * subject requiring dialysis due to end-stage renal failure will be excluded * subjects with evidence on history, physical or blood work of hepatic disease will be excluded since elevated serum transaminases are a potential adverse effect of acarbose * cannot currently be taking an alpha-glucosidase inhibitor * cannot have had allergic reactions to alpha-glucosidase inhibitors in the past * due to the fact that acarbose is renally excreted, all subjects must have a creatine clearance of greater than 25 ml/min * subjects with a past history of inflammatory bowel disease, intestinal obstruction, ileus and peptic ulcer disease will be excluded * subjects taking carbohydrate-splitting enzymes (such as amylase) will be excluded * subjects with chronic respiratory issues requiring treatment will be excluded

Keywords: acarbose pph (post prandial hypotension) falls syncope elderly
contingency management for smoking in substance abusers

Conditions: Cigarette Smoking

Summary: patients (n=102) who meet diagnostic criteria for alcohol, cocaine, marijuana, or opiate abuse or dependence will meet with research staff on two days for quit preparation sessions (2 per day). these sessions include testing a breath sample for evidence of smoking twice each day (separated by at least 5 hours), counseling based on public health service (phs) guidelines for quitting smoking during the second session each day, and setting a quit date. after these sessions, participants will be randomly assigned to one of two 4-week conditions: (a) standard care or (b) standard care plus prize cm for smoking abstinence with the opportunity to win prizes for submitting samples that meet smoking abstinence criteria (e.g., co ≤ 6ppm; cotinine ≤ 30ng/ml). nicotine withdrawal and related measures will be assessed throughout the intervention. follow-up data will be collected through 6-months post-quit date.


Eligibility criteria:
inclusion criteria: * current alcohol, cocaine, marijuana, or opiate abuse or dependence * smoke ≥ 10 cigarettes per day * expired co \> 8 ppm * self-reported interest in quitting smoking, * ≥ 18 years old * willing to provide names, addresses and phone numbers of individuals to assist in locating the participant for follow-up evaluations, * english speaking exclusion criteria: * currently receiving behavioral therapy or pharmacotherapy for smoking plan to use pharmacotherapy in this quit attempt * serious, uncontrolled psychiatric illness (e.g., acute schizophrenia, suicide risk) * cognitive impairment * in recovery for pathological gambling

Keywords: contingency management cigarette smoking cessation substance abusers
financing strategies of influenza vaccination in china

Conditions: Influenza Vaccines; Influenza, Human; Cost of Illness; Health Knowledge, Attitudes, Practice; Immunization Programs

Summary: this trial focuses on influenza vaccination among three recommended groups in china. data concerning the economic burden of influenza-like illness (ili), vaccine hesitancy, discrete choice experiment, immunization records and willingness to pay for vaccines will be collected via questionnaires, and the financing strategies of influenza vaccines in china will be further analyzed. besides, the knowledge, attitude/belief and practice (kap) of medical staff will also be asked in a supplementary questionnaire.

this trial focuses on influenza vaccination among three recommended groups in china, including children (aged between 6 to 59 months), the elderly (aged 60 years old or above), and adult patients with chronic diseases (aged below 60 years old). the questionnaires will collect data on the economic burden of influenza-like illness (ili), vaccine hesitancy, discrete choice experiment, immunization records and willingness to pay for vaccines, and the financing strategies of influenza vaccines in china will be further analyzed accordingly. besides the three recommended groups, vaccination and health care personnel will also be asked about their knowledge, attitude/belief and practice (kap) in a supplementary questionnaire.

Eligibility criteria:
inclusion criteria: * (for children group) children aged between 6 to 59 months. * (for the elderly group) the elderly aged 60 years old or above. * (for chronic disease patient group) adult patients with chronic diseases aged below 60 years old. * (for medical staff) general practitioners and vaccination staff. * interviewees with informed consent. exclusion criteria: * those cannot be surveyed for physical reasons. * those with communication barriers. * those who do not agree to participate in the survey.
enms with balance feedback for post-stroke ankle-foot rehabilitation

Conditions: Cerebrovascular Stroke; Foot Drop; Foot Inversion Deformity

Summary: in this work, a novel hybrid ankle-foot robot is designed for gait rehabilitation after stroke, i.e., exoneuromusculoskeleton with balance sensing feedback (enms-bsf) by integrating the advantages of soft pneumatic muscle, functional electrical stimulation, exoskeleton and foot balance feedback in one system. with the assistance of the enms-bsf the foot drop and foot inversion could be corrected with improved muscle coordination in the paretic lower limb. the device is wearable and light-in-weight for unilateral application during walking. it is hypothesized that with the intervention of the enms-bsf the gait pattern of persons after stroke can be improved with long-term rehabilitative effects.


Eligibility criteria:
inclusion criteria: * at least 6 months after the onset of stroke * have sufficient cognition to follow simple instructions, as well as understand the content and purpose of the experiment (mini-mental state examination (mmse) score \>21). * have mild-to-moderate motor impairment in the affected lower limb with foot drop but be capable of standing and walking without manual assistance. * fugl-meyer assessment (fma), total score on the lower limb \<20 * functional ambulatory category (fac) ≥4 * berg balance scale (bbs) ≥40 exclusion criteria: * the exclusion criteria will be server spasticity at the ankle joint measured by modified ashworth score (mas) \>3. * receiving other lower limb rehabilitative interventions at the same time

Keywords: robot stroke lower limb
a treatment extension study of mucopolysaccharidosis type iiib

Conditions: Mucopolysaccharidosis Type IIIB; MPS III B

Summary: the primary objectives of this study are to evaluate the long-term safety and tolerability of ax 250 administered to subjects with mps iiib by an implanted icv reservoir and catheter and to evaluate the impact of long-term ax 250 treatment on cognitive function in patients with mps iiib as assessed by developmental quotient (dq).


Eligibility criteria:
inclusion criteria: * must have completed 48 weeks in part 2 of study 250-201 and enter 250-202 within 8 weeks of study completion * written informed consent from parent or legal guardian and assent from subject, if required * has the ability to comply with protocol requirements, in the opinion of the investigator * males and females who are of reproductive age should practice true abstinence, defined as no sexual activity, during the study and for 6 months after the study has been completed (or withdrawal from the study). if sexually active and not practicing true abstinence, males and females of reproductive age must use a highly effective method of contraception while participating in the study. * if female with childbearing potential, must have a negative pregnancy test at the screening visit and be willing to have additional pregnancy tests during the study. exclusion criteria: * has both (1) a cognitive aeq score ≤ 18 months, and (2) a dq score ≤ 20 * would not benefit from enrolling in the study in the opinion of the investigator * has received stem cell, gene therapy or ert (other than ax 250) for mps iiib * has contraindications for neurosurgery (eg, congenital heart disease, severe respiratory impairment, or clotting abnormalities) * has contraindications for mri scans (eg, cardiac pacemaker, metal fragment or chip in the eye, or aneurysm clip in the brain) * has a history of poorly controlled seizure disorder * is prone to complications from intraventricular drug administration, including patients with hydrocephalus or ventricular shunts * has received any investigational medication other than ax 250 within 30 days prior to the baseline visit or is scheduled to receive any investigational drug during the course of the study * has a medical condition or extenuating circumstance that, in the opinion of the investigator, might compromise the subject's ability to comply with protocol requirements, the subject's well-being or safety, or the interpretability of the subject's clinical data. * is pregnant at any time during the study

Keywords: sanfilippo syndrome type b
ecg ai-guided screening for low ejection fraction

Conditions: Asymptomatic Left Ventricular Systolic Dysfunction (Disorder); Heart Failure

Summary: this is a randomized controlled trial (rct) to test a novel artificial intelligence (ai)-enabled electrocardiogram (ecg)-based screening tool for improving the diagnosis and management of left ventricular systolic dysfunction.


Eligibility criteria:
inclusion criteria: * primary care clinicians who are part of a participating care team that care for adult patients and have the ability to order ecg and tte (this includes physicians, nurse practitioners, and physician assistants). exclusion criteria: * primary care clinicians working in pediatrics, acute care, nursing homes, and resident care teams.
correlation of different ultrasonographic indices with clinical parameters in carpal tunnel syndrome

Conditions: Carpal Tunnel Syndrome

Summary: the aim of this study is to investigate the relationship between different ultrasonographic indices used in patients with carpal tunnel syndrome and clinical parameters. the main questions it aims to answer are: * to what extent are the ultrasonographic parameters used in the diagnosis of carpal tunnel syndrome related to the patient's clinical complaints? * to what extent are the different ultrasonographic parameters used in the diagnosis of carpal tunnel syndrome correlated with each other?

carpal tunnel syndrome (cts) is the most common entrapment neuropathy and the main mechanism is compression of the median nerve under the transverse ligament at the wrist level. in cts, neuropathic complaints such as numbness, burning and tingling in the palmar face of the first two fingers, which are the innervation area of the median nerve, and fatigue in the hand are the most common symptoms. in the diagnosis of cts, the diagnosis is made electrodiagnostically along with typical clinical findings. however, in recent years, ultrasound has become one of the most preferred methods in the diagnosis of cts because it is non-invasive, rapid and correlates with electrodiagnostic methods. diameter, cross-sectional area and echogenicity of the median nerve at the carpal tunnel entrance are the most frequently evaluated parameters in ultrasonographic examination. in different studies, it is aimed to increase the power of ultrasonography in the diagnosis of cts with various indices created in addition to standardized measurements. the most commonly used ultrasonographic indices, which have been shown to be correlated with electrodiagnostic diagnostic methods, are the wrist-forearm median nerve cross-sectional area ratio, the ratio of median nerve cross-sectional area to carpal tunnel area at the carpal tunnel entry level, and echogenicity assessment. although the relationship of these indexes, which are reported to have sufficient diagnostic sensitivity and specificity, with the patient's clinical complaints is shown on a measurement basis, there is not enough data on the comparison of different formulas and their correlation with the patient's clinical complaints, including neuropathic pain.based on this, this study aimed to investigate the relationship between ultrasonographic indices used in patients diagnosed with cts and clinical parameters.

Eligibility criteria:
inclusion criteria: * being diagnosed with carpal tunnel syndrome based on clinical and electrodiagnostic findings * agreeing to participate in the study exclusion criteria: * concomitant history of diabetes, systemic inflammatory disease, active infection and malignancy * having a disease with neuropathic pain such as polyneuropathy, radiculopathy,multiple sclerosis * not agreeing to participate in the study * history of surgery due to cts

Keywords: carpal tunnel syndrome ultrasound echogenicity
a study to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of m281 administered to adults with generalized myasthenia gravis

Conditions: Generalized Myasthenia Gravis

Summary: the purpose of this study is to evaluate the safety, tolerability, and efficacy of m281 administered to participants with generalized myasthenia gravis (gmg) who have an insufficient clinical response to ongoing standard of care therapy.


Eligibility criteria:
participants must be ≥18 years of age with a documented history of generalized myasthenia gravis (gmg) and clinical signs/symptoms of gmg, not pregnant or breastfeeding, and no history of any neurologic disorder other than mg that might interfere with the accuracy of study assessments. additional, more specific criteria are defined in the protocol.

Keywords: m281 generalized myasthenia gravis
simple psychological interventions and cryoanalgesia

Conditions: Arterial Puncture

Summary: this study aims to explore the effect of using simple psychological interventions and cryoanalgesia for reducing arterial punctures pain and anxiety in critically ill patients

the present study will be carried out in two interventions patients who received cryoanalgesia: patients subjects underwent arterial puncture. patients in the intervention group had a small plastic bag filled with 12 oz of crushed ice applied to the radial artery puncture site for 3 min (timed with a stopwatch) without external compression or massage. psychological interventions using verbal signals of the impending procedure, music distraction, visual distraction, and breathing interventions be used during arterial puncture in adults

Eligibility criteria:
inclusion criteria: * adult patients of both genders. * consciousness patient required arterial puncture exclusion criteria: * unconsciousness patients patients with complicated site from arterial puncture

Keywords: cryoanalgesia arterial puncture anxiety
safety study of plx4032 in patients with solid tumors

Conditions: Malignant Melanoma; Colorectal Carcinoma

Summary: the primary objective of this fih study is to assess the safety and pharmacokinetics of plx4032 in patients with solid tumors. the secondary objective is to assess the pharmacodynamic activity in paired biopsy specimens obtained from patients with malignant melanoma who have the v600e braf oncogenic mutation.

activating mutations of the braf gene have been observed in a variety of cancers, including 55-68% of malignant melanomas. in general, oncogenic mutations of braf correlate with a poor outcome. plx4032 is a compound that selectively inhibits oncogenic b-raf kinase. two extension cohorts of patients with confirmed v600e mutations will be recruited, consisting of advanced melanoma and metastatic colorectal carcinoma.

Eligibility criteria:
inclusion criteria: * solid tumors confirmed histologically whose tumors are refractory to standard therapy, or for whom standard or curative therapy does not exist * patients from whom paired melanoma biopsies are planned must have a v600e+ braf mutation confirmed prior to the administration of plx4032 * previous chemotherapy, immunotherapy, or radiation therapy must have been completed at least 2 weeks prior to starting plx4032 therapy, and all associated toxicity must be resolved prior to administration of plx4032 * patients in the extension cohorts (melanoma or adenocarcinoma of the colon or rectum) must have both a v600e+ braf mutation and measurable disease (by recist v 1.0 criteria) prior to the administration of plx4032. all patients enrolled must provide archival or fresh melanoma tumor biopsy for confirmation of v600e+ braf mutation status by taqman assay * ecog performance status 0 or 1 * life expectancy ≥ 3 months * adequate hematologic, hepatic, and renal function exclusion criteria: * brain metastases that are progressing or have been documented to be stable for less than 3 months, or for which systemic corticosteroids are required * investigational drug use within 28 days of the first dose of plx4032 * uncontrolled intercurrent illness * refractory nausea and vomiting, malabsorption, or significant bowel resection that would preclude adequate absorption
hct+f vs. hct alone in critically ill medical septic shock patients

Conditions: Septic Shock

Summary: this study will be conducted as a single-center, prospective, open-label, randomized trial that will evaluate adult patients admitted with septic shock to the medical critical care unit (mcc). the objective of the study is to determine if the use of hydrocortisone plus fludrocortisone is associated with a faster resolution of shock (defined as 24 hours vasopressor free) when compared to the use of hydrocortisone alone in medical, critically ill septic shock patients.

for three consecutive months, side 1 of the medical critical care unit (mcc1) will receive hydrocortisone alone and side 2 (mcc2) will receive the combination of hydrocortisone and fludrocortisone. after the initial three months, this will be flipped and patients admitted to mcc1 will receive the combination while patients in mcc2 will receive hydrocortisone alone for three consecutive months. this change in group assignments will occur to account for the difference in number of beds between mcc1 and mcc2, and to minimize the potential differences in patient acuity by location.

Eligibility criteria:
inclusion criteria: * critically ill medical patients requiring addition of stress dose steroid therapy (hydrocortisone) in addition to pressors for septic shock management during icu stay * fludrocortisone administered within 24 hours after initiation of hydrocortisone if in combination therapy group exclusion criteria: * use of fludrocortisone and/or hydrocortisone for any reason other than septic shock management during icu stay * fludrocortisone/hydrocortisone initiated by any service other than critical care medicine * prior use of fludrocortisone/hydrocortisone at time of admission (home or outside facility) * patients not appropriate for study inclusion as determined by provider discretion * patients receiving steroid therapy not in accordance with assigned group per location (mcc1 or mcc2) * patients re-admitted to the mcc during the same admission and restarted on vasopressor therapy will be noted during data collection and only the initial admission will be included for analysis * any patient receiving greater than one dose of hydrocortisone 100 mg * physical or medical contraindication to receiving po or per ft (per feeding tube) fludrocortisone