Kornimate/medical-research-clean huggingface dataset:
acceptability study of a new paediatric form of vigabatrin in infants and children with infantile spasms or pharmacoresistant partial epilepsy

Conditions: Infantile Spasms

Summary: the sponsor is developing a new paediatric formulation of vigabatrin to better adjust the dose to body weight and to limit waste of unused drug. the currently marketed vigabatrin (sabril™) form only exists as 500 mg film coated tablets (for adults and children above 6 years) and 500 mg granules for oral solution sachets (for infants and children below 6 years). sabril™ is not adapted for administration to infants when a fraction of the sachet is needed. manual splitting of the sachet or lengthy and error-prone dilutions are often required. this study is a descriptive, non-randomized, open label multi-centric acceptability study in infants and children affected with infantile spasms. the primary objective is to describe the adherence to the new formulation. secondary objectives include: * evaluation of the palatability and user-friendliness of the new treatment, * evaluation of the pharmacokinetic parameters of the new formulation, * pk parameters, * evaluation of the tolerance, * measurement of taurine plasma levels. this study will recruit up to 40 patients with infantile spasms and pharmacoresistant partial epilepsy aged 1 month to 6 years in 23 clinical sites in france.


Eligibility criteria:
inclusion criteria: * patients with diagnosed infantile spasms (is) or pharmacoresistant partial onset seizures (pos). * infants \> 1 month and \ 6 months and \ 2 years and \< 6 years. * patients under sabril® or naive patients. * patients under a twice-a-day posology of sabril® or patients for whom vigabatrin will be given twice daily. non inclusion criteria: * use of more than 2 other antiepileptic drugs as concomitant treatment (including steroids). ketogenic diet can be in addition to these 2 other antiepileptic drugs. * subjects receiving vigabatrin through a gastric tube. * weight \< 1.750 kgs. * any planned major surgery within the duration of the trial. * participation in any other clinical trial within 3 months prior to v1.

Keywords: infantile spasms vigabatrin soluble tablets acceptability adherence pharmacokinetics
nir hypoxia imaging of breast tumor response to neoadjuvant chemotherapy in vivo

Conditions: Breast Cancer

Summary: the purpose of this study is to determine whether an experimental alternative imaging method, near infrared spectroscopy (abbreviated as nir), can be used with a controlled respiratory system to examine the breasts for cancer. the nir system uses light beams to produce an image or picture of the inside of the breast. the respiratory system will help researchers get more information about the breast tissue by changing the amount of oxygen the tissue receives during nir imaging.


Eligibility criteria:
inclusion criteria: * normal cohort 1. breast size and epithelial integrity adequate to allow nir imaging exams 2. no serious associated psychiatric illnesses 3. female, ≥25 and \<76 years old 4. written informed consent * under treatment cohort 1. locally advanced breast cancer, with or without metastatic disease, defined as being clinically appropriate for neoadjuvant therapy 2. breast size and epithelial integrity adequate to allow nir imaging exams 3. no serious associated psychiatric illnesses 4. female, ≥25 and \<76 years old 5. written informed consent exclusion criteria: * both cohorts 1. pre-existing respiratory conditions: 1. severe chronic obstructive pulmonary disease (including chronic bronchitis and/or emphysema) 2. other respiratory or lung conditions which would place the patient at risk 3. presence of any other significant cardiac or pulmonary symptoms, such as moderate or severe dyspnea on exertion, orthopnea, or paroxysmal nocturnal dyspnea 2. congestive heart failure 3. intolerance of hyperoxia or hypercarbia as delivered by the respiract breathing circuit 4. pregnancy
proper nutrition and its relation to oral diseases in covid -19 era

Conditions: Diet, Healthy

Summary: effect of proper diet and vitamins on the oral health and the regeneration of the taste and smell in covid 19 patients

effect of proper diet and vitamins on the oral health ( bleeding gums, ulcers, bad breath) also the effect of proper diet on the regeneration of the taste and smell in covid 19 patients a diet will be followed for 2 weeks then the progress will be evaluated by making another questionnaire

Eligibility criteria:
inclusion criteria: - exclusion criteria: * chronic diseases

Keywords: proper diet taste oral health corona
cardiopulmonary exercise testing in girls (8-18y) with turner sydrome.

Conditions: Turner Syndrome

Summary: the goal of this clinical trial is to have a beter insight in the exercise tolerance in girls with turner syndrome aged 8-18 years . the main question it aims to answer is: is there a difference in vo2 max comparing turner syndrome girls with standard values? how do cardiovascular parameters change during exercise (heart rate, bloodpressure, ...) participants will perform a cyclo-ergometry in a standardised way.


Eligibility criteria:
inclusion criteria: * turner syndrome girls aged 8-18 years old exclusion criteria: * severe mental impairement making it impossible to perform an exercise test

Keywords: cardiopulmonary exercise test
uark 2009-32 compassionate use study of carfilzomib

Conditions: Multiple Myeloma

Summary: this is a compassionate use, open-label, ind study for the purpose of providing carfilzomib to patients with relapsed or resistant refractory multiple myeloma.

the protocol has been designed for relapsing or refractory multiple myeloma patients. a maximum of 30 patients may be treated on this protocol. since this is a compassionate use study of an investigational agent, the size is determined by agreement with the manufacturer of carfilzomib. therefore, the time period for enrollment and accrual number will be limited to n=30.

Eligibility criteria:
inclusion criteria: * participants must have histologically documented multiple myeloma which is relapsing or resistant after stem cell transplantation, or when other conventional therapies have failed or are contraindicated * serum creatinine \ 15 ml/min calculated or measured clearance is ≥15 ml/min * anc \> 1000/mm3 (may be supported with growth factors) * platelet count \> 30,000/mm3 (may receive transfusion) * female participant of child-bearing potential has agreed to use dual methods of contraception for the duration of the study * male participant has agreed to use a barrier method of contraception if sexually active with a female of child-bearing potential * patients must have signed and irb-approved written informed consent form and demonstrate willingness to meet follow-up schedule and study procedure obligations exclusion criteria: * active infection requiring systemic treatment * requires concomitant use of approved or investigative anticancer therapeutic treatment with activity against multiple myeloma, other than dexamethasone * concomitant use of other investigative agents (e.g., antibiotics or antiemetics) * pregnant or breast-feeding

Keywords: mm
effect of community mobile outreach approach compared to facility based directly observed treatment short course on treatment outcome among tuberculosis patients in jeddah: a randomized controlled trial

Conditions: Tuberculosis

Summary: to compare the effectiveness of the community mobile outreach approach in improving treatment outcomes (success rate) among tuberculosis patients with those being treated with facility based directly observed treatment short course (dots) in jeddah region.


Eligibility criteria:
inclusion criteria: * newly diagnosed tb cases exclusion criteria: * visitors having tb especially pilgrims
patient transitioning from exubera® inhalation powder to technosphere® insulin inhalation powder

Conditions: Type 1 Diabetes; Type 2 Diabetes

Summary: the purpose of this study is to allow patients with specific needs for inhaled insulin to continue with inhaled insulin therapy using technosphere insulin after exubera was withdrawn from the market.

a phase 3, open label, multicenter, safety follow up trial to convert the treatment of subjects currently using exubera to treatment with ti inhalation powder

Eligibility criteria:
inclusion criteria: * subject has type 1 or type 2 diabetes mellitus and is currently being treated with or has been treated with exubera. * subject has a severe phobia to sc injections of insulin (needle phobia) preventing conventional treatment or has impaired sc insulin absorption exclusion criteria: * smoking in the previous 6 months * history of asthma or chronic obstructive pulmonary disease (copd) or any other significant pulmonary disease, or exposure to pulmonary toxins. * clinically significant pulmonary abnormalities on chest high-resolution computed tomography (hrct). * pft results prior to transferring to ti inhalation powder showing any of the following: fev1 \< 70% of predicted, fvc \< 70% of predicted, dlco \< 70% of predicted, tlc \< 80% of predicted. * allergy to insulin
probiotics enhance the treatment of pd-1 inhibitors in patients with liver cancer

Conditions: Liver Cancer

Summary: human microbes have been called "the second genome of humanity".on may 13,2016,the white house launched the national microbiome initiative (nmi), with an estimated investment of us $521 million, to elevate microbiome research to a national strategic status. the gut is the largest microecological environment in the human body. the research in the field of intestinal microbiome has become one of the most advanced and hot research directions in the scientific field of the world today. at present, more than 50 diseases have been found to be related to intestinal microbiome disorders. pd-1 (programmed death receptor 1) is an important immunosuppressive molecule.it regulates the immune system and promotes tolerance by down-regulating the immune system's response to human cells and by suppressing t cell inflammatory activity. in the past, the research team and colleagues in related fields have found a strong correlation between gut microbiome and the efficacy of anti-pd-1 immunotherapy in cancer patients.this protects against autoimmune diseases, but it also prevents the immune system from killing cancer cells. as more and more scientific evidence shows that intervention of human intestinal flora may improve the efficacy of anti-pd-1 immunotherapy in tumor patients, intestinal flora, as the most effective way to intervene human intestinal flora, has been mentioned by many research institutions and international drug manufacturers in combination with anti-pd-1.our previous study showed that the abundance of beneficial bacteria such as lactic acid bacteria, bifidobacteria and akkermansia muciniphila was significantly correlated with pd-1 inhibitor response, and regulating the intestinal flora content could improve the effect of pd-1 inhibitor on mouse tumors, indicating that microbial flora was involved in regulating cancer immunotherapy.


Eligibility criteria:
inclusion criteria: * karnofsky score ≥90 in patients receiving immunotherapy for liver cancer at our cancer center exclusion criteria: * any immune system disease under high risk to antimicrobial agents such as diabetes,infection disease or drug allergy to v9
study of systemchange-hiv

Conditions: HIV

Summary: the purpose of this study is to assess a new behavioral intervention to help how people living with hiv/aids practice self-management skills. specifically, we want to see if a new educational intervention can improve physical activity, sleep, mental wellness and quality of life in hiv(human immunodeficiency virus)-infected men and women. we hypothesize that those who are in the intervention group will practice more self-management skills than those in the control group.

sample: the study sample will include 40 hiv+ men and women. inclusion criteria include: a documented hiv diagnosis; adult (18 years old or greater); and english speaking. design: the systemchange-hiv study will be a 15-week, randomized, two-group experimental design including a 10-week behavioral intervention. in the experimental group, one half of the participants (n=20) will receive the intervention; and in the control group, the other half of the participants will undergo usual care. both the experimental and control group will complete the same psychometric instrument packet, three times, to give a comparison of self-management before and after intervention and at a four-week follow up visit. this packet includes information on medical history, demographics, hiv self-management,physical activity, social support, social capital, mental wellness, and quality of life.additionally, all participants will wear an actigraph for seven consecutive days, at each data collection point, to assess sleep. analysis:preliminary data analysis will include descriptive statistics and scatter plots to examine the distribution of each outcome at each time point, as well as each covariate of interest. although our randomization scheme will ensure balance of baseline variables between the two arms (other than that due to chance alone), we will estimate the standardized magnitude of each baseline difference as well as the correlation between each baseline variable and each outcome of interest at 10 weeks or at 15 weeks to objectively determine baseline variables that will be needed to estimate the unbiased effect of the intervention on each outcome. the primary analysis will be based on intent to treat approach and will investigate the effect of the intervention on continuous outcomes at 10 weeks or 15 weeks. in particular, an analysis of covariance model will be used that adjusts for treatment arm and the baseline level of the outcome variable. such a model will allow us to estimate the effect of the intervention on each outcome, adjusting for the baseline outcome.in addition, linear mixed models will be used to determine if the effect of the intervention on the outcome at 10 weeks differs from the effect of the intervention on the outcome at 15 weeks. if needed, all analyses will be repeated using an as-treated approach. all analyses will be performed using a two-sided significance of 0.05.

Eligibility criteria:
inclusion criteria: * documented hiv diagnosis * adult (21 years old or greater) * english speaking exclusion criteria:

Keywords: hiv/aids adult behavioral intervention sleep physical activity
a study of imr-687 in subjects with sickle cell disease

Conditions: Sickle Cell Disease

Summary: a study to evaluate the safety and efficacy of imr-687 in subjects with sickle cell disease

a phase 2b, randomized, double-blind, placebo-controlled, multicenter study of subjects with sickle cell disease (scd; homozygous sickle hemoglobin \[hbss\], sickle-β0 \[hbsβ0\] thalassemia, or sickle-β+ \[hbsβ+\] thalassemia) to evaluate the safety and efficacy of the phosphodiesterase type 9 (pde9) inhibitor, imr-687, administered once daily (qd) for 52 weeks.

Eligibility criteria:
inclusion criteria: 1. confirmed diagnosis of scd (hbss, hbsb0 thalassemia, or hbsb+ thalassemia) 2. hemoglobin of \>5.5 and \<10.5 g/dl; hb values within 21 days post-transfusion will be excluded. 3. subjects must have had at least 2 and no more than 12 documented episodes of vocs in the past 12 months at the time of informed consent signing and at randomization (day 1). 4. subjects receiving hu must have received it continuously for at least 6 months prior to signing informed consent, and must have been on a stable dose for at least 3 months prior to signing the informed consent, with no anticipated need for dose adjustments during the study including the screening period, in the opinion of the investigator. 5. female subjects must not be pregnant or breastfeeding and be highly unlikely to become pregnant. male subjects must be unlikely to impregnate a partner. 6. must be willing and able to complete all study assessments and procedures, and to communicate effectively with the investigator and site staff. exclusion criteria: 1. hospital discharge for sickle cell crisis or other vaso-occlusive event within the 4 days prior to randomization (day 1). 2. subjects participating in a chronic/prophylactic rbc transfusion program (i.e., regularly scheduled rbc transfusions); any transfusions within 21 days of screening or baseline hb measurements 3. subjects with hbf \>25% at screening. 4. significant kidney disease (egfr \ 3x upper limit of normal. 5. body mass index (bmi) \ 35 kg/m2. 6. subjects with known active hepatitis a, hepatitis b, or hepatitis c, with active or acute event of malaria, or who are known to be positive for human immunodeficiency virus (hiv). 7. stroke requiring medical intervention within 24 weeks prior to randomization (day 1). 8. prior exposure to imr-687. 9. subjects taking direct acting oral anti-coagulants (apixaban, dabigatran, rivaroxaban, edoxaban, or ticagrelor) or taking warfarin unless they stopped the treatment at least 28 days prior to randomization (day 1). 10. a history of use of crizanlizumab (adakveo®) or voxelotor (oxbryta®) within 6 months prior to signing the informed consent. 11. receipt of erythropoietin, luspatercept (reblozyl®)or other hematopoietic growth factor treatment within 3 months of signing the icf or anticipated need for such agents during the study. 12. prior gene therapy.

Keywords: sickle cell disease
safety and pharmacokinetics of sad/mad oral doses of srp-3d (da)

Conditions: Pain

Summary: this is a two-part randomized, double-blind, placebo-controlled study.

this is a two-part randomized, double-blind, placebo-controlled study. the study comprises a sad (part 1) assessment which will include a food effect assessment that contributes data to inform a subsequent mad (part 2) dose-ranging study. safety measurements will be collected throughout the study for all subjects. blood samples will be collected to determine the pk parameters of srp-3d (da).

Eligibility criteria:
inclusion criteria: 1. healthy male or female. females must not be pregnant or breastfeeding. 2. is between 18 and 55 years of age (inclusive). 3. able to speak and understand english or spanish. 4. agrees to comply with testing procedures. 5. has a body mass index (bmi) between 18.0 and 32.0 kg/m2 (inclusive). 6. the subject meets good health criteria. 7. females of non-childbearing potential or agree to use birth control. 8. male subjects must be surgically sterile or agree to the use birth control. 9. agree to the confinement period and return for the outpatient visits. 10. has vital signs at screening within appropriate ranges. exclusion criteria: 1. history or presence of clinically significant diseases. 2. abnormal diet 4 weeks preceding the first dose of study medication. 3. received any investigational product in a clinical study. 4. previously been administered imp in this study. 5. taking any prescribed or otc drug. 6. taking moderate or strong inhibitors/inducers of cytochrome p450. 7. history of hypersensitivity to acetaminophen or similar chemical entities. 8. presence or history of allergy or blood or plasma donation. 9. blood or plasma donation 10. smokers and those who have smoked within the last 12 months. 11. current users of e-cigarettes and nicotine replacement products. 12. consumption of prohibited beverages or foods. 13. prior history of substance abuse or treatment. 14. regular alcohol consumption. 15. positive alcohol urine test at screening or admission. 16. is a female with a positive pregnancy test result. 17. positive urine screen for drugs of abuse. 18. positive test for hepititus b or c, or hiv. 19. active infection, periodontal disease,. certain dental appliances. 20. glucose-6-phosphate-dehydrogenase (g6pd) deficiency. 21. significant serious skin disease. 22. cohort 3 only: history of cholecystectomy or gall stones. 23. have poor venous access that limits phlebotomy 24. evidence of current sars-cov-2 infection 25. clinically significant abnormal clinical chemistry, hematology or urinalysis. 26. immediate family members of a study site or sponsor employee. 27. failure to satisfy the investigator of fitness to participate.

Keywords: pain
use of non invasive hemodynamic cardiovascular monitoring to evaluate emergency department patients

Conditions: COPD; Congestive Heart Failure; Trauma; Sepsis; Stroke

Summary: this study is an observational prospective pilot trial that utilizes finger cuff non invasive hemodynamic monitoring (nexfinhd monitor) to assess 4 different groups (chf/copd, trauma, sepsis, stroke) of patients on arrival to the emergency department and to document the changes seen in these hemodynamics with acute therapies.

there has been little very early (after emergency department presentation) hemodynamic monitoring of acutely ill or injured patients secondary to the lack of non invasive technologies that could be reliably applied to this patient population.thus all assessments of hemodynamics have been made by physicians on clinical information including intermittent pulse and blood pressure measurements. it is not known what the underlying continuous cardiac output, systemic vascular resistance, etc are in these patients and how these change with current emergency medicine therapeutic interventions. currently there is available a finger cuff devise (nexfinhd monitor, bmeye, amsterdam) that can measure these hemodynamics on a beat to beat basis and that can be easily applied to acutely ill individuals. this prospective pilot study of 48 patients (4 groups of 12 each) will document the hemodynamics of patients on arrival and continuously for 2 hours, blinded to the treating physician. treating physicians will be asked to estimate these hemodynamic parameters at various time points. the study will characterize the hemodynamics of patients, compare them to the estimates of treating physicians and determine whether they predict the need for life saving interventions.

Eligibility criteria:
inclusion criteria: * patients with acute chf/copd (n=12) * patients with acute trauma and a trauma iss\>15 (n=12) * patients with acute sepsis (n=12) * patients with acute stroke (n=12) exclusion criteria: * patients in cardiopulmonary arrest * patients with stemi * patients with known peripheral vascular disease * pregnant patients * age\<18 * excessively agitated patients * interference with current standard of acre

Keywords: non-invasive hemodynamic monitoring emergency department patients trauma with an iss>15 non-invasive continuous hemodynamic monitoring
a clinical study to assess the safety of a disposable intra-vaginal device for stress urinary incontinence

Conditions: Stress Urinary Incontinence

Summary: this study will evaluate the safety of the pessary device by objective evaluation of vaginal wall integrity and subjective assessment of comfort during in-use conditions.


Eligibility criteria:
inclusion criteria: * be female 18 years or older; * provide written informed consent prior to study participation and been given a signed copy; * be in generally good health as determined by the investigator; * have a ≥ 3 month history of experiencing stress urinary incontinence (sui) per week (self reported); * be willing to use the pessary investigational device to control stress urinary incontinence; * be willing to comply with study requirements and instructions; exclusion criteria: * are pregnant, lactating, or planning to become pregnant during the study; * within 3 months post partum; * intrauterine device (iud) placement of less than 6 months; * has self-reported difficulty emptying her bladder; * a history of toxic shock syndrome (tss) or symptoms consistent with tss; * experience difficulty inserting or wearing an intra-vaginal device, including a tampon; * vaginal surgery, perineal surgery, uterine surgery, or abortion (spontaneous or induced) within the past 3 months; * has any screening laboratory value outside the laboratory reference range considered clinically significant by the investigator and could impact the safety of the subject or the outcome of the study; or * for any reason, the investigator decides that the subject should not participate in the study.

Keywords: stress urinary incontinence, sui, pessary