cyrilzakka/clinical-trials huggingface dataset:
Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older With the G551D Mutation

The purpose of this study was to evaluate the efficacy and safety of ivacaftor in subjects with cystic fibrosis aged 12 years and older who have the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Ivacaftor is a potent and selective CFTR potentiator of wild-type, G551D, F508del, and R117H forms of human CFTR protein. Potentiators are pharmacological agents that increase the chloride ion transport properties of the channel in the presence of cyclic AMP-dependent protein kinase A (PKA) activation.

This was a phase 3 study in subjects with cystic fibrosis (CF) age 12 years and older who have a G551D-CFTR mutation and percent predicted forced expiratory volumn in 1 second (FEV1) between 40% and 90%.

Based on in vitro studies and pharmacologic, pharmacokinetic (PK), and safety profiles, ivacaftor was selected for clinical development as a possible treatment for patients with CF. Patients with the G551D mutation were the targeted population for this study because ivacaftor is a potentiator of the gating function of the CFTR protein, and the most prevalent mutation with a gating defect in CF is the G551D mutation.

This study was designed to further evaluate the efficacy of ivacaftor in subjects with CF who have a G551D-CFTR gene mutation and to evaluate safety in this population over a longer period than previously studied.

Eligibility Criteria:
Inclusion Criteria:

* Confirmed diagnosis of cystic fibrosis (CF) and G551D mutation in at least 1 allele
* Forced expiratory volume in 1 second (FEV1) of 40% to 90% (inclusive) of predicted normal for age, gender, and height at Screening.
* No clinically significant abnormalities that would have interfered with the study assessments, as judged by the investigator
* Willing to use highly effective birth control methods during the study

Exclusion Criteria:

* History of any illness or condition that might confound the results of the study or pose an additional risk in administering study drug to the subject
* Acute respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 4 weeks of Day 1 of the study
* History of alcohol, medication or illicit drug abuse within one year prior to Day 1
* Abnormal liver function ≥ 3x the upper limit of normal
* Abnormal renal function at Screening
* History of solid organ or hematological transplantation
* Pregnant, planning a pregnancy, breast-feeding, or unwilling to follow contraception requirements
* Ongoing participation in another therapeutic clinical study or prior participation in an investigational drug study within 30 days prior to Screening
* Use of inhaled hypertonic saline treatment
* Concomitant use of any inhibitors or inducers of cytochrome P450 3A4 (CYP 3A4)
Evaluation of Different Strategies of Pericardial Drainage After Aortic Valvular Surgery

The incidence of pericardial effusion and late cardiac tamponade after aortic and valvular surgery is higher than after other cardiac surgical procedures. The aim of this study is to evaluate the clinical safety and efficacy of prolonged mediastinal drainage using small, soft silastic drains (Blake drain, Ethicon USA) versus conventional mediastinal drainage using large chest tubes. A prospective randomized trial.

The incidence of pericardial effusion and late cardiac tamponade after aortic and valvular surgery is higher than after other cardiac surgical procedures. The aim of this study is to evaluate the clinical safety and efficacy of prolonged mediastinal drainage using small, soft silastic drains (Blake drain, Ethicon USA) versus conventional mediastinal drainage using large chest tubes. Patients undergoing aortic and / or valvular surgery will be randomized in two groups. In group A, mediastinal drainage will be accomplished using a 28F or 32F chest tube in the anterior mediastinum and a 19F Blake drain located in the posterior pericardial cavity. In group B, mediastinal drainage will be accomplished using two 28F or 32F chest tubes located in the anterior mediastinum. In both groups, conventional chest tubes will be removed on the first postoperative day, while patients in the group A will have prolonged drainage using the Blake drain until output is less than 50 ml over 24 hour. Patients will be followed during their postoperative course for occurrence of significant pericardial effusion as detected on routine echocardiogram and late cardiac tamponade requiring reintervention.

Eligibility Criteria:
Inclusion Criteria:

* Patients aged between 18 and 90 years old, undergoing either surgery of the ascending and/or transverse aorta, or surgery of the mitral and/or aortic valves
* Availability for follow-up at the Montreal Heart Institute Exclusion criteria

Exclusion Criteria:

* Emergency surgery
* Unavailability for follow-up at the Montreal Heart Institute
Exenatide and Impaired Hypoglycaemic Awareness in Type 1 Diabetes

Approximately 25% of patients with type 1 diabetes have lost the capacity to timely detect hypoglycaemia, a condition referred to as impaired hypoglycaemic awareness (IHA) that causes a six-fold higher risk of severe, potentially hazardous, hypoglycaemia. IHA is usually the end-result of a process of habituation to recurrent hypoglycemia that is potentially reversible. Treatment with glucagon-like peptide (GLP)-1 Receptor Agonists (1RAs) in addition to insulin therapy may decrease the incidence of hypoglycaemia in patients with type 1 diabetes. This study will test the hypothesis that treatment with the GLP-1RA, exenatide, added to basal-bolus insulin therapy will improve awareness of hypoglycaemia in patients with type 1 diabetes and IHA. In a randomized doubleblind placebo-controlled cross-over trial, patients will be treated for 6 weeks with exenatide (or placebo), after which hypoglycemic symptoms and counterregulatory hormone responses will be examined during a hyperinsulinemic hypoglycemic glucose clamp study.


Eligibility Criteria:
Inclusion Criteria:

* Type 1 diabetes, disease duration \>1 year
* Age \>18 years, \<70 years
* Insulin treatment according to basal-bolus insulin regimen (injections or insulin pump)
* Impaired hypoglycaemic awareness as assessed by a score of 3 or more on the modified Dutch translation of the Clarke questionnaire
* Glycated haemoglobin (HbA1c) ≥42 mmol/mol (6%) and ≤75 mmol/mol (9.0%)
* Ability to provide informed consent

Exclusion Criteria:

* Treatment with incretin-based therapy
* Known intolerance to GLP-1RAs (including allergy)
* Treatment with glucose-modifying or immune-modifying agents, e.g. prednisolon
* Recent history of myocardial infarction or stroke (past year) or laser coagulation for proliferative retinopathy (past 6 months)
* Proliferative retinopathy
* Symptomatic diabetic neuropathy
* Diabetic nephropathy as reflected by albumin-creatinin ratio \>30 mmol/mg or estimated Glomerular Filtration Rate (eGFR) \<60 ml/min/1.73 m2
* Known heart failure
* History of pancreatitis (acute or chronic) or pancreatic cancer
* Body-mass index \>40 kg/m2
* Use of premixed insulin or of long-acting insulin alone
* Total daily insulin dose requirements \<20 units unless on pump treatment
* Pregnancy or unwillingness to undertake measures for birth control
Quantifying Energetic Demands of Walking for People With Cerebral Palsy

The purpose of this study is to examine the underlying mechanisms that contribute to high energy costs for people with cerebral palsy (CP) as they walk.

The investigators will characterize the cost landscape of children with CP, quantifying the magnitude of cost (net nondimensional oxygen consumption) associated with walking and common sub-tasks of walking, such as supporting and stabilizing the body.

Children with CP use large amounts of energy to walk and move, leading to fatigue and limiting participation. For people with CP the energetic cost of walking is on average over two times higher than typically-developing (TD) peers. This means that for people with CP, walking is as tiring as jogging or climbing stairs. An energetic cost of this magnitude restricts activities of daily living (ADL) and causes exhaustion.

While the investigators and many others have sought to reduce these energetic costs through surgical interventions, rehabilitation, orthotics, or other assistive devices, these strategies have failed to result in meaningful reductions in energy. To design strategies that successfully reduce walking costs, the investigators must first understand the underlying mechanisms contributing to elevated cost in people with CP. This basic-science research will provide the foundation to create evidence-based strategies to decrease energy costs, minimize fatigue, and increase quality of life for people with CP and other neurologic injuries.

Eligibility Criteria:
Inclusion Criteria for Cases:

* 8-17 years old
* Diagnosed with bilateral cerebral palsy
* Gross Motor Function Classification System II
* No surgery within the last 9 months
* No current baclofen pump or explantation in the last 2 months
* No botulinum toxin or phenol injections in the last 2 months
* Had a prior gait and motion analysis at Gillette Children's Specialty Healthcare
* Participant and caregiver able to speak and read English
* Ability to follow instructions

Inclusion Criteria for Controls:

* 8-17 years old
* Participant and caregiver able to speak and read English
* Ability to follow instructions
Tamoxifen Therapy for Myotubular Myopathy

This is a phase 1 / 2, randomized, double-blinded, single cross-over study, with a washout period between treatment regimens, to test the efficacy and safety of tamoxifen therapy to improve motor and respiratory function in males with XLMTM.

Pre-clinical studies in Mtm1 knockout mice (a model of XLMTM) demonstrated prolonged survival, increased motor function (including muscle strength), and improved muscle histopathology with tamoxifen treatment. Based on these data, and the known safety profile of the drug in humans, we hypothesize that tamoxifen treatment will be safe and will improve motor and respiratory function in XLMTM patients. This is a randomized, double-blinded, single crossover clinical trial to test this hypothesis and determine the safety and efficacy of tamoxifen in improving motor and respiratory function in MTM patients. Each subject will serve as his own control during the placebo phase of the study. As treatments for XLMTM are current not available, this study addresses a critical unmet need by testing a therapy that, if effective, may serve as a primary treatment, or in the future as an adjunct to other therapies in development.

Eligibility Criteria:
INCLUSION CRITERIA

1. Male
2. Patients ages 6 months and older may participate.
3. XLMTM resulting from a confirmed mutation in the Myotubularin 1 (MTM1) gene
4. Patients over 18 years of age and parent(s)/legal guardian(s) of patients \<18 years of age must provide written informed consent prior to participating in the study and informed assent will be obtained from minors, or at least 7 years of age when required by regulation.
5. Willing and able to comply with all protocol requirements and procedures.

EXCLUSION CRITERIA

1. Other disease which may significantly interfere with the assessment of myotubular myopathy (MTM) and is clearly not related to the disease, at the discretion of the qualified investigator.
2. Has undergone surgery or hospitalization \< 3 months before starting TAM4MTM (at t = -3 months), or has surgery scheduled during the 18 months of participation in TAM4MTM, which will impede motor assessments in the opinion of the Investigator.
3. Has a history of thromboembolic events
4. Currently enrolled in a treatment study for XLMTM or receiving treatment with an experimental therapy other than pyridostigmine.
5. Treatment with pyridostigmine for \< 6 weeks duration (must be greater than 6 weeks to be included in TAM4MTM).
6. Use of concomitant medication known to inhibit CYP2D6 and/or CYP3A4, including clarithromycin, erythromycin, diltiazem, itraconazole, ketoconazole, ritonavir, verapamil, goldenseal and grapefruit, paroxetine, troleandomycin, rifampin, phenobarbital, aminoglutethimide, medroxyprogesterone, amiodarone, haloperidol, indinavir, ritonavir, quinidine, rifampicin, or any selective serotonin reuptake inhibitor (SSRI).
7. Subject has a contraindication to tamoxifen or its ingredients
Carotid Artery Stent Implantation in the Treatment of Vulnerable Plaque

To investigate the efficacy and safety of carotid stenting for vulnerable carotid plaques. All patients with carotid artery stenosis underwent carotid arterial contrast-enhanced ultrasonography before operation. According to the examination results, they were divided into two groups: vulnerable plaque group and stable plaque group. The incidence of perioperative stroke events in the two groups was compared. The incidence of stroke events in the two groups within 1 year was compared.

1. To enroll 100 cases of patients suffering from carotid artery stenosis。the investigators perform contrast enhanced ultrasound on patients for identifying the vulnerable plagues
2. The investigators divide patients into the stable group and the vulnerable group based on the results of CEUS inspection.
3. All patients undergo carotid stent. To analysis the incidence of postoperative complications such as stroke and mortality within 30 days and to compare the differences between the two groups.

Eligibility Criteria:
Inclusion Criteria:

* The patients with ischemic symptom whose carotid stenosis were more than 50%
* The patients without ischemic symptom whose carotid stenosis were more than 70%

Exclusion Criteria:

* • Confirmed with severe intracranial vascular lesions

  * Suffering from the large area cerebral infarction or critical stroke sequela
  * Restenosis after CEA or CAS
  * Suffering from severe coronary heart disease, respiratory failure,the hypertension difficult to controled
  * Patients with malignant tumor or expected life \< 2 years
Prognostic Value of New Cardiac Biomarkers in Elderly Patients Undergoing Cardiac Surgery

To evaluate the use of new cardiac biomarker for the prediction of in-hospital cardiovascular complications in elderly patients undergoing elective cardiac surgery.

Plasma N-terminal pro-brain natriuretic peptide (NT-proBNP, cardiac troponin T (cTnT), pregnancy-associated plasma protein A (PAPP-A), ischemia-modified albumin (IMA), myeloperoxidase (MPO), metalloproteinase (MMP) soluble CD 40 ligand (sCD40L), and inflammatory cytokine levels will be measured in 100 elderly patients undergoing elective cardiac surgery with cardiopulmonary bypass before induction of anesthesia and 12 h, 24 h, and 48 h after surgery. Optimum discriminators for prediction of postoperative cardiovascular complications wil be determined by the use of receiver operating characteristics (ROC) curves analyses.

Eligibility Criteria:
Inclusion Criteria:

* patients aged 75 years or older undergoing elective cardiac surgery with CPB

Exclusion Criteria:

* positive cardiac troponin T test within the week before surgery
* patients undergoing emergent procedures
* patients with preoperative mechanical cardiac support
* patients undergoing hemodialysis
Effects of Yoga on Menopausal Symptoms in Women With Breast Cancer

Women with breast cancer and breast cancer survivors are often bothered by menopausal symptoms to a special degree. Treatment options for those symptoms are currently limited as hormone replacement therapy is often prohibited. Yoga has been shown to reduce menopausal symptoms as well as symptoms associated with breast cancer. Therefore, this study aims to investigate whether a 12-week yoga intervention can reduce menopausal symptoms in women with breast cancer and breast cancer survivors.


Eligibility Criteria:
Inclusion Criteria:

* Breast cancer (Stage I-III)
* After completion of surgical therapy
* After completion of adjuvant radiotherapy and chemotherapy
* Score of 5 or greater on the Menopausal Rating Scale
* Physical and cognitive capacity to participate in the yoga intervention
* Willingness to participate in 10 or more of the 12 yoga interventions
* signed informed consent

Exclusion Criteria:

* Regular yoga practice in the past 12 months
* Planned surgery in the study period
* Diagnosed and medically treated psychosis
* Hormone replacement therapy
Safety of GlaxoSmithKline (GSK) Biologicals Hepatitis A Vaccine in Korean Population

GlaxoSmithKline Korea has submitted a registration file for its hepatitis A vaccine. The present study will collect clinical data in the local target population as per the requirement of Korean Food and Drugs administration (FDA).

All subjects will be administered one dose of the hepatitis A vaccine.

Eligibility Criteria:
Inclusion criteria

* Subjects who the investigator believes that they and/or their parents/guardians can and will comply with the requirements of the protocol (e.g. be reachable over the phone) should be enrolled in the study.
* Free of obvious health problems as established by medical history and clinical examination before entering into the study.
* Written informed consent is obtained from the subject/ subjects' parents or guardians.

Exclusion criteria

* History of allergic disease or reactions likely to be exacerbated by any component of the vaccine.
* Acute disease at the time of enrolment. (Acute disease is defined as the presence of a moderate or severe illness with or without fever. All vaccines can be administered to persons with a minor illness such as diarrhea, mild upper respiratory infection with or without low-grade febrile illness, i.e., Oral temperature \< 37.5°C (99.5°F) / Axillary temperature \< 37.5°C (99.5°F) / Rectal temperature \< 38°C (100.4°F) / Tympanic temperature on oral setting \< 37.5°C (99.5°F) / Tympanic temperature on rectal setting \< 38°C (100.4°F).
Postural and Muscle Fatigue Analysis of Endodontic Residents

Musculoskeletal disorders (MSDs) are a significant occupational health problem for dentists and have been linked to a decline in quality of life, often leading to stress-related illnesses and early retirement. Poor posture, poor movement or imbalances in the neck or shoulders can lead to the three most common pain syndromes in dentistry. The objectives of this study were to evaluate the working position adopted during an endodontic procedure and to determine the perception of symptoms of musculoskeletal disorders in first and second year students of the Master in Advanced Endodontics at the European University of Madrid (UEM), using the standardised Nordic Kuorinka questionnaire supplemented by a form with socio-demographic variables. This study has a descriptive cross-sectional observational design and included 10 students selected by convenience sampling. A video camera was used to record the procedures from three different angles for a maximum of 5 minutes. The information was processed using Microsoft Excel spreadsheet software, and the video recordings were assessed using Rodgers' Muscular Fatigue Analysis (RMFA) and Rapid Upper Limb Assessment (RULA).

The unit of analysis for this study consisted of the first and second year students of the Master's in Endodontics programme at the European University of Madrid (UEM) who completed the two years of annual clinical practice during the years 2022/24. A total of 10 students were enrolled over the two years of training, treating 30 patients (3 patients per student) in the first year and another 30 patients in the second year (for a total of 22 months). A video camera was used to record the procedures from three different angles for a maximum of 5 minutes (two side views - right and left - and one frontal view) on different patients, following the National Institute for Occupational Safety and Health (NIOSH) Protocol for Video Recording for Job Analysis and Risk Assessment. The selected endodontic procedure focused exclusively on upper or lower first molars during the opening and ultrasonic search for the fourth canal in upper first molars or the search for the mesiobuccal canal in lower first molars. The standardised Nordic Kuorinka Musculoskeletal Symptom Perception Questionnaire was used as the measuring instrument. Questionnaire data were collected using a spreadsheet program (Microsoft Excel).

Eligibility Criteria:
Inclusion Criteria:

- Students of the Master's Degree in Endodontics of the European University of Madrid (UEM), who have completed the two years of the annual teaching period of clinical practice.

Exclusion Criteria:

* Subjects previously diagnosed with a musculoskeletal disorder.
* Subjects who had suffered previous trauma affecting the musculoskeletal system.
* Pregnant women in their third trimester.
* Subjects who refused to participate.
Exercise, Oxidative Stress and HIV

The purpose of this study is to compare oxidative stress markers and the immunologic characteristics of HIV-infected and non-HIV subjects during a bout of moderate aerobic exercise followed by resistance exercises


Eligibility Criteria:
Inclusion Criteria:

* HIV Group:without contraindications for exercise training; to highly active antiretroviral therapy (HAART) for at least 6 months before enrollment; be classified as low level of physical activity by international physical activity questionnaire (IPAQ) and did not use antioxidant supplements.
* Control Group:negative result for HIV-1 test and the other criteria described above

Exclusion Criteria:

* Subjects with previous history of cardiovascular and neurological disease, diabetes, dyslipidemia, tobacco using, physical disability and pregnant women were excluded from the study.
A Study Comparing User Experience of Different Delivery Devices for Glucagon

The purpose of the study is to learn more about different devices for delivering glucagon during simulations of severe hypoglycemic (low blood sugar) emergencies. The study will be carried out using medical manikins to simulate real life scenarios. No drug will be administered to humans. Part A will last approximately 17 days for Trained Users and Participants with Diabetes (PWD). Part B will last approximately nine (9) days for Untrained Users. Each part will be separated by approximately 7 days.


Eligibility Criteria:
Inclusion Criteria:

All participants (Trained Users, Participants with Diabetes \[PWDs\] and Untrained Users):

* Are able to understand the purpose and procedure of the study and to give written informed consent to show willingness to participate in the study

Trained Users only:

* Are participants who are not diagnosed with Type 1 Diabetes Mellitus (T1DM) or Type 2 Diabetes Mellitus (T2DM)
* Are close friends/relatives of a patient with T1DM or T2DM on insulin
* Have not previously administered any rescue medications (e.g. epinephrine, glucagon, narcan or seizure medications)

Untrained Users only:

* Have not been diagnosed with T1DM or T2DM, and are not a Trained User of a person with T1DM or T2DM
* Have not previously administered any rescue medications (e.g. epinephrine, glucagon, narcan or seizure medications)

Exclusion Criteria:

All participants (Trained Users, PWDs and Untrained Users):

* In the opinion of the investigator or sponsor, are unsuitable for inclusion in the study

Trained Users and Untrained Users only (i.e. the participants involved in the simulations):

* Are currently enrolled in a clinical study involving any type of medical research judged not to be scientifically or medically compatible with this study
* Are judged by the investigator as being likely to have difficulty performing the administration due to physical, cognitive and/or severe psychiatric disorder
* Are judged by the investigator as being trained or experienced in performing rescue drug administration
* Are judged by the investigator as being trained or experienced in performing high-fidelity simulations of drug administration
* Have received formal training in a medical field, and/or worked in this field within the prior 5 years; or otherwise are judged by the investigator as being trained or experienced as a first responder