cyrilzakka/clinical-trials huggingface dataset:
Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis

This is an open-label extension study for participants who completed a Phase 3, placebo-controlled study of ataluren in participants with nonsense mutation cystic fibrosis (nmCF) not receiving chronic inhaled aminoglycosides.

The primary objective of this Phase 3 extension study will be to obtain long-term safety data to augment the overall safety database. The secondary objectives will be to augment the efficacy data collected in the double-blind study (PTC124-GD-021-CF; NCT02139306).

Eligibility Criteria:
Inclusion Criteria:

* Completion of study treatment (placebo or active) in the previous Phase 3, double-blind study protocol (Protocol PTC124-GD-021-CF)
* Evidence of signed and dated informed consent/assent document(s) indicating that the participant (and/or the participant's parent/legal guardian) has been informed of all pertinent aspects of the trial.

Exclusion Criteria:

* Known hypersensitivity to any of the ingredients or excipients of the study drug.
* Ongoing participation in any other therapeutic clinical trial.
DAPHNE Study: Direct Anticoagulant PHarmacogeNEtic

New/direct oral anticoagulants (NOAC/DOAC), like apixaban and rivaroxaban, are an interesting alternative to unfractionated or low molecular weight heparin relayed by oral anti-vitamin K anticoagulants (VKA) for the treatment of venous thromboembolism and atrial fibrillation. This new generation of anticoagulants directly inhibit a factor in the blood coagulation pathway and have a wide therapeutic range overcoming several practical issues associated with VKA therapy including the need of routine coagulation monitoring potentially simplifying patient management. However, despite this wide therapeutic range, a large interindividual dose variability related to factors such as age, body surface, smoking, concomitant diseases as well as differences in drug metabolism, could put susceptible patients at risk for uncontrolled bleeding. Both rivaroxaban and apixaban are cleared primarily via cytochrome P450 (CYP) mediated hepatic metabolism, mainly CYP3A, and renal excretion, involving the P-glycoprotein (P-gp). Both CYP3A and P-gp activity show important interindividual variations due to drug interactions and/or genetic polymorphisms in corresponding genes.

The aim of the current study is to evaluate the impact of cytochrome activity and relevant polymorphisms on rivaroxaban/apixaban dosage regimen or treatment efficacy in a hospital setting. The safety issue in this context is particularly relevant, since hospitalisation is linked to a modification of the patient's treatment with often an increase in the number of medications. The resulting changes in metabolism due to modified cytochrome and transporter activities could affect rivaroxaban/apixaban blood concentrations. Our central hypothesis is that genotype and/or phenotype in CYP3A4/5/7 or P-gp may influence the rivaroxaban/apixaban plasma concentration and increase the risk of thrombotic or hemorrhagic events. Thus, investigating how the patient's genotype and/or phenotype for CYP3A4/5/7 and P-gp could potentially alter the bio-disponibility of rivaroxaban and apixaban and therefore the risk to develop adverse events or inefficacy would be of particular interest.


Eligibility Criteria:
Inclusion Criteria:

* Understanding of French or English language and provide signed and dated informed consent form.
* Willing to comply with all study procedures and be available for the duration of the study.
* Male or female, aged 18 years or above.
* Diagnosed with atrial fibrillation, deep-vein thrombosis or pulmonary embolism and under rivaroxaban or apixaban drug treatment.

Exclusion Criteria:

* Participation in a clinical study that may interfere with participation in this study.
* Under rivaroxaban or apixaban for prophylaxis of deep-vein thrombosis and pulmonary embolism in patients undergoing knee or hip replacement surgery.
* Anything that would place the individual at increased risk or preclude the individual's full compliance with or completion of the study.
* Known allergy to midazolam or to fexofenadine
Community Pediatric Acceptability Study (CPAS)

Ear infections, or otitis media, are a leading cause of health expenditures and antimicrobial prescriptions in children. Diagnosis of otitis media requires the ability to view the tympanic membrane. An instrument called an otoscope with a light source and a magnifying lens with a plastic tip that conforms to the ear canal is currently the standard of care for examining the tympanic membrane. Interpretation of otoscopic examinations is operator-dependent and cannot be seen by anyone other than the person holding the otoscope. A pocket size attachment that uses the technology and light source of a smartphone to capture images of the ear canal and tympanic membrane facilitates image documentation of the otic examination. In previous studies with the device, the investigators have shown that image quality of photographs of the tympanic membrane taken with the smartphone otoscope are equivalent to those taken with a camera-fitted conventional otoscope.

In this study, the Community Provider Assessment Study (CPAS), the investigators will perform a cross-sectional study in which they will randomly assign 3-4 pediatricians to use a smartphone otoscope as the standard of care device for all ear examinations and 3-4 pediatricians to use a conventional otoscope for alternating 1 month periods for 6 months. The parents of children examined during the study period with both devices will be invited to participate in 3 telephone surveys assessing parental satisfaction with the device and antimicrobial use by their child for the otic complaint. The results of this study have the potential to improve diagnosis and management of otitis media, thus improving patient care, reducing costs, and decreasing the opportunity for the development of antimicrobial resistance.


Eligibility Criteria:
Inclusion Criteria:

* Clinical diagnosis of an otic complaint
* Willing to undergo otoscopy

Exclusion Criteria:

* Unwilling or unable to to complete study telephone questionnaires
* Do not meet inclusion criteria
Olfactory and Taste Disturbances in Sjogren's Syndrome

Sjögren syndrome is an autoimmune disease, responsible for xerostomia and xerophthalmia. Other organs and tissues can be affected: the skin, vaginal and nasal mucous membranes. As well, olfactory disorders have been also described in Sjögren syndrome.

Xerostomia often causes significant functional impairment of taste function. Impairment of taste function has been poorly evaluated in Sjögren syndrome like olfactory or (intra-nasal and oral) trigeminal disorders.

The aim of the study is to evaluate olfactory, taste (intra-nasal and oral) trigeminal functions in patients with Sjogren's syndrome compared to patients without sicca syndrome.

Eligibility Criteria:
Inclusion Criteria:

* group 1 : patients with Sjogren's syndrome
* group 2: patients without sicca syndrome
* older than 18 years old
* consenting to participate to the study

Exclusion Criteria:

* smoking patients
* patients with stomatitis
* patients with upper respiratory tract infection
A Study of Efruxifermin in Subjects With Histologically Confirmed Nonalcoholic Steatohepatitis (NASH)

This is a multi-center evaluation of efruxifermin (EFX) in a randomized, double-blind, placebo-controlled study administered for 16 weeks in subjects with biopsy proven F1 - F4 NASH.


Eligibility Criteria:
Key Inclusion Criteria:

* Males and non-pregnant, non-lactating females between 18 - 80 years of age inclusive, based on the date of the screening visit.
* Main Study only: Body mass index (BMI) \> 25 kg/m\^2 (unless the patient has biopsy-proven NASH documented within the last 2 years).
* Main Study only: Must have confirmation of ≥ 10% liver fat content on magnetic resonance imaging- proton density fat fraction (MRI-PDFF) at screening.
* Main Study only: Biopsy-proven NASH. Must have had a liver biopsy within 180 days of randomization with fibrosis stage 1 to 3 and a non-alcoholic fatty liver disease (NAFLD) activity score (NAS) of ≥ 4 with at least a score of 1 in each of the following NAS components:

  * Steatosis (scored 0 to 3),
  * Ballooning degeneration (scored 0 to 2), and
  * Lobular inflammation (scored 0 to 3)
* Cohort C only: FibroScan® measurement \> 13.1 kPa.
* Cohort C only: Cirrhosis due to NASH. Liver biopsy consistent with F4 fibrosis according to the NAS system, confirmed by the central or local reader.

Exclusion Criteria:

* Weight gain or loss \> 5% in the 3 months prior to randomization or \> 10% in the 6 months prior to screening.
* Type 1 and insulin-dependent Type 2 diabetes.
* Poorly controlled hypertension (blood pressure \> 160/100).
Efficacy and Tolerability of Idebenone in Boys With Cardiac Dysfunction Associated With Duchenne Muscular Dystrophy

Idebenone is a synthetic analogue of coenzyme Q10 and is a powerful antioxidant and essential constituent of the process of energy production on the cellular level. It can protect mitochondria from oxidative damage and boost their impaired function. It is thought that this mechanism will slow decline in heart function that is part of the disease process of Duchenne Muscular Dystrophy (DMD). It is possible that patients may benefit in terms of muscle strength and respiratory function. This pilot trial is designed to investigate this.


Eligibility Criteria:
Inclusion Criteria:

* Patients 8 - 16 years of age at time of enrolment
* Male
* Presence of cardiac involvement/dysfunction, defined by abnormal peak systolic strain in left ventricle (LV) inferolateral wall
* Confirmed diagnosis of DMD (out of frame dystrophin gene deletion OR absent/\<5% dystrophin protein on muscle biopsy; clinical picture consistent of typical DMD)
* If on chronic glucocorticosteroids treatment (deflazacort, prednisone) for DMD (or any other disease) (i.e. concomitant medication): dosage must be stable (unchanged) 6 months prior to inclusion
* If on chronic medication for DMD associated cardiomyopathy (β-blocker, diuretics): dosage must be stable (unchanged) 3 months prior to inclusion
* Ability to provide reproducible repeat quantitative muscle testing (QMT) upper limb score within 15% of first assessment score (at Visit1/Day 1 versus Screening Visit

Exclusion Criteria:

* Symptomatic cardiomyopathy or heart failure
* Asymptomatic but severe cardiac dysfunction on baseline (Screening) evaluation: Fractional shortening (FS) \< 20% and/or Ejection fraction (EF) \< 40%
* Use of ACE-inhibitors
* Previous history of ventricular arrhythmias (other than isolated ventricular extrasystole); ventricular arrhythmias presented at Screening
* Previous (6 months or less) participation in any other therapeutic trial for DMD
* Use of coenzymeQ10, idebenone, creatine, glutamine, oxatomide, or any herbal medicines within the last 6 months
* History of significant concomitant illness or significant impairment of renal or hepatic function
* Known individual hypersensitivity to idebenone
Hypnosis and Virtual Reality in Bone Marrow Examination

The goal of this clinical trial is to compare the effects of hypnosis, virtual reality or their combination in patients undergoing a bone marrow procedure.

The main question it aims to answer is whether such interventions may relieve anxiety and other symptoms described by patients undergoing the procedure.

Before the bone marrow procedure and after being explained on the study and signing informed consent, the nurse will measure vital signs and participants will fill-out a 2-minutes' questionnaire and will be assigned to one of 3 groups:

* Hearing a 7-minutes hypnotic script via earphones
* Seeing and hearing virtual images and sounds via a virtual reality device during 7 minutes
* None of these After these interventions or 10 minutes after assignation for patients not receiving intervention, patients will fill questionnaires again (about 5 minutes for filling the 2 questionnaires) and vital signs will be measured again. Then the bone marrow procedure will be performed. After the bone marrow procedure, the participants will fill-out another 2-minutes questionnaire and vital signs will be measured by the nurse.

Researchers will compare hypnotic script, virtual reality, their combination or none of them to see if they can affect anxiety and other complaints in patients undergoing a bone marrow procedure.


Eligibility Criteria:
Inclusion Criteria:

1. Age 18 or older
2. Candidate for a BM examination at the hematology unit
3. Ability to answer questionnaires in Hebrew, Arabic or Russian
4. Informed consent form signing

Exclusion Criteria:

1. Major hearing impairment
2. Major visual impairment.
A Study in Subjects With Retinal Detachment

The purpose of this trial is to access the tolerability of INS37217 Intravitreal Injection when administered intravitreally in subjects with macula-on or macula-off rhegmatogenous retinal detachment (RRD).


Eligibility Criteria:
Inclusion Criteria:

* have macula-on or macula-off rhegmatogenous retinal detachment
* are eligible for pneumatic retinopexy, with identifiable retinal breaks
* are phakic or pseudo-phakic

Exclusion Criteria:

* have detachments not of a rhegmatogenous nature
* currently have blood in the vitreous, corneal opacity, or other conditions which limit the view of peripheral retina
* have evidence of intraocular inflammation (uveitis)
* have a history of current condition of uncontrollable, elevated IOP or open-angle glaucoma
* have breaks not conducive to a single procedure of pneumatic retinopexy treatment
* have proliferative vitreoretinopathy of type C or D
* have previously had a vitrectomy or require one
* have previously had a scleral buckle procedure
Mortality of Sepsis in Swedish Intensive Care Units 2008-2016

A study of short and long term mortality in patients treated with or without a diagnosis of sepsis in Swedish intensive care units (ICU) 2008 - 2016. The cohort is characterized with regard to chronic comorbidity, severity of acute illness and demographic factors, and outcome is adjusted for these factors. Changes in mortality during the period are studied.

All adult patients admitted to a general ICU included in the Swedish Intensive Care Registry (a majority of all ICUs in Sweden) between 2008-01-01 and 2016-10-18 are followed for one year and any deaths are registered. Data on illness severity scores, age, sex, admitting hospital and duration of ICU stay are recovered. Data are linked with the inpatient registry of the Swedish Board of Health and Welfare (Socialstyrelsen), and any discharge diagnoses from the last 5 years preceding ICU admission are recovered. These are used to assess chronic comorbidity.

Mortality, adjusted for severity of illness, comorbidity and patient factors, is studied and any changes over time during the inclusion period are assessed.

The data in the Swedish ICU Registry are entered by the treating ICU physicians and include severity of illness (Simplified Acute Physiology Score (SAPS)3), selected chronic comorbidity, sex and date of birth, ICU stay and selected interventions and complications. Relevant International Classification of Diseases (ICD)-10 diagnoses are required and assessment of the occurrence of severe sepsis or septic shock, during the inclusion period defined according to the American College of Chest Physicians/Society of Critical Care Medicine (ACCP/SCCM) (sepsis 2) criteria, is mandatory when entering data.

Eligibility Criteria:
Inclusion Criteria:

* Admission to a Swedish general ICU (excluding specialized cardiothoracic, neurosurgical, pediatric and burns units) between 2008-01-01 and 2016-10-18
* Episode registered in Swedish Intensive Care Registry

Exclusion Criteria:

* Age \< 18 years
* Readmission (only first episode during the period included)
A Cohort Study of Incretin-based Therapy Combined With Insulin in Type 2 Diabetic Patients for 5 Years

The use of dipeptidyl-peptidase 4 (DPP-4) inhibitors and glucagon like peptide 1 (GLP1) analogues for the treatment of diabetic mellitus (DM) type 2 is growing (1,2). Currently, some of these agents have been approved in combination with insulin. The potential for combined use with insulin has garnered increasing attention due to reduce side effects associated with insulin therapy and improve glycemic control. Some investigators reported that GLP-1 analogue combined with insulin reduces HbA1c and weight with low risk of hypoglycemia and high treatment satisfaction (3). However, their duration of treatment was short time with less than a mean of 3.0 years and the alterations of chronic diabetic complications by combination with incretin-based and insulin therapies are not known.

We evaluated the long effects of adding incretin-based therapy (DPP-4 inhibitors or GLP-1 analogues) to insulin therapy on glycated hemoglobin (HbA1c) as glycemic control, body mass index (BMI), blood pressure (BP), insulin dosage, frequency of hypoglycemia, and chronic diabetic complications for 5 years-treatments.

Patients were treated with diet, exercise, and/or oral conventional pharmacotherapy combined with insulin. Oral conventional pharmacotherapy consisted of α-glycosidase inhibitors (α-GI), sulfonylurea (SU), biguanide (BG), thiazolidinedione (TZD), or combinations of these agents. Insulin therapy consisted of subcutaneous injections of long-acting insulin analogues prior to sleep and bolus subcutaneous injections of rapid-acting insulin analogues in multiple daily injections, or subcutaneous injections of mixed insulin analogues twice a day. In spite of the treatments, when the physician in charge judged that their values of HbA1c were inappropriate, the physician added the pharmacotherapy combined the insulin to incretin-based therapy. 2.3.2. For ethical reasons, patients were treated with various anti-hypertensive, anti-diabetic, anti-dyslipidemia and/or anti-hypercoagulation agents during the course of the study by the patients' own doctors as a part of continuing standard medical care. As to the drugs that have been used for the treatment of other disorders and its complication since the time before the study, the content should not be changed during the study in principle unless the complication is cured. If any new complication occurs during the study period, an appropriate treatment is given by the judgment of investigator.

Eligibility Criteria:
Inclusion Criteria:

After a detailed baseline examination, 500 Japanese subjects with type 2 diabetes were followed up for all-cause mortality and morbidity. All participants visited our clinic regularly. All patients were fully informed about the purposes and procedures for the study and provided oral consent at enrolment.

Exclusion Criteria:

Patients participating in other clinical study. Other than the above, patients judged inappropriate as the subjects of this study by the investigator.
Anti-thrombotic and Glucose Lowering Therapy in Diabetic Patients Undergoing PCI

Diabetes mellitus (DM) is one of the main risk factors for ischemic events in patients with coronary artery disease (CAD) and diabetes is a factor in several post-PCI (Percutaneous Coronary Intervention) risk scores. However, until recently, there were almost no studies performed specifically in the diabetic population of patients undergoing PCI. This study aims to describe the anti-thrombotic regimens, clinical outcomes and current diabetes medical treatment in an unselected consecutive population of patients with DM undergoing PCI.

Diabetes is one of the main risk factors for ischemic events in patients with coronary artery disease and diabetes is a factor in several post-PCI risk scores (including the commonly used DAPT score). However, until recently, there were almost no studies performed specifically in the diabetic population of patients undergoing PCI. At large, results from randomized trials assessing the duration of DAPT have produced conflicting results and there is uncertainty about the best anti-thrombotic strategy in patients with diabetes. Further assessment of the patterns of use and their clinical effects, including those related to prolonged DAPT is needed, in diabetic patients, especially in less selected "real world" populations.

Eligibility Criteria:
Inclusion Criteria:

* PCI with stent implantation, performed in at least one major coronary artery in the context of stable coronary artery disease or acute coronary syndrome
* Type 2 Diabetes mellitus (previously diagnosed or diagnosed at the index admission)
* Informed consent signed
* Patient not simultaneously participating in any interventional study

Exclusion Criteria:

* Patients with Type 1 Diabetes mellitus
* Patients whose survival is expected to be lower than 1 year at hospital discharge
* Patients not whiling to participate
Understanding Post-translational Modifications to Circulating Histones Via Mass Spectrometry in Pregnant Women Developing Pre-eclampsia: A Retrospective Study

This is a descriptive pilot study on a ready-constituted biobank (outside the Jardé Law). It is an ancillary study to the "GrossPath" cohort (RCB ID number: 2014-A01120-47).

Pregnancy generates an increased risk of thrombosis, and placenta-mediated diseases constitute a risk factor for cardiovascular pathologies responsible for significant maternal-fetal morbidity and mortality. Understanding and exploring the cellular and molecular mechanisms of dysfunctions of the vascular-placental interface could provide arguments to understand the systemic vascular risk, characterize it and finally detect it on the basis of new markers, thus opening the way for targeted preventive management to reinforce the general principles of precision medicine.

Netosis is a process of activation of neutrophils, which then generate filaments containing DNA, enzymes and extracellular histones. Netosis occurs in pregnancy and is increased in vascular-placental complications. It can be studied by measuring circulating histones, particularly the citrullinated histone H3. Levels of this modified histone H3, as well as those of two other modifications, have recently been shown to increase during pregnancy. These levels have also been shown to be even greater in pregnancy complications.

The aim of this study is to complete this mapping in order to obtain a precise signature for all post-translational histone modifications in normal pregnancies and pregnancies complicated by pre-eclampsia from the "GrossPath" cohort in order to propose new circulating biomarkers for placental vascular pathologies.

The post-translational histone modification profiles (mapping) of pregnant women with normal pregnancies will be compared with those developing pre-eclampsia.


Eligibility Criteria:
Inclusion Criteria:

* Not applicable as this study is on samples from a biobank. The inclusion criteria for the "GrossPath" cohort were:
* pregnant women followed at Nîmes University hospital for normal pregnancy or pregnancy with placental vascular pathology (pre-eclampsia and/or intra-uterine growth retardation).
* The patient must have given her free and informed consent and signed the consent form.
* The patient must be a member or beneficiary of a health insurance plan
* Only women are included
* Patients are at least 18 years old

Exclusion Criteria:

* Not applicable as this is a study on samples from a biobank. The non-inclusion criteria of the "GrossPath" cohort were :
* twin pregnancies.