cyrilzakka/clinical-trials huggingface dataset:
Mechanisms of Cell Death in Spinal Muscular Atrophy

Spinal muscular atrophy is a genetically based disease that affects motor neurons in the spinal cord and leads to muscle wasting and weakness. The gene found to be responsible for the underlying disease is called the SMN or survival motor neuron gene. Individuals with SMA are either missing a copy of the gene or have a mutation in the gene. Although the gene has been identified, how it actually causes the motor neurons to die and leads to muscle wasting and weakness is not completely understood. The investigators have found that skin cells from children with SMA tend to be more susceptible to cell death when exposed to cell death inducing agents. In this protocol, The investigators wish to study the mechanisms by which these cells die when exposed to these agents and how this may be related to the gene defect and the disease.

Spinal muscular atrophy (SMA) is a neuromuscular disease characterized by degeneration of motor neurons and progressive muscle atrophy. The disease is one of the most common genetic causes of infant death. The gene responsible for SMA, survival motor neuron (SMN), exists in humans as two nearly identical copies (SMN1 and SMN2). Only deletion or mutation(s) of the telomeric copy of the gene (SMN1) causes the disease. The SMN protein has been known to function in assembly of the RNA splicing complex, however, the mechanism(s) by which SMN-deficiency causes cell death in SMA are not clear. The long-term goal is to understand the mechanism(s) of motor neuron death in SMA and develop a means of prevention. SMN protein has been reported to have some survival promoting functions in cultured cells. Preliminary studies show that skin fibroblasts from SMA patients are more sensitive to certain death promoting stimuli than control fibroblasts. The investigators hypothesize that the SMN protein is directly involved in cell survival and that loss of this survival function of SMA results in motor neuron death in SMA. The investigators will use fibroblasts from SMA patients, fibroblasts from controls without SMA, motor neuron-like cell lines (such as NSC-34) and rodent primary motor neuron cultures as model systems to test our hypothesis. The investigators will determine the effect of expression of SMN protein in regulating cell death of SMA fibroblasts. The investigators will further investigate the role of SMN in neuronal cell survival. Finally, the investigators will determine biological pathway(s) of SMN-mediated cell protection. Results from the proposed studies will provide insight into the mechanism(s) by which SMN protects cells from death and how a decrease in SMN function leads to the SMA phenotype. Ultimately, the obtained information could lead to develop therapeutic strategies for SMA.

Eligibility Criteria:
Inclusion Criteria:

* Diagnosis of SMA confirmed by neurologist

Exclusion Criteria:

* Not seen as a patient at a participating Nemours facility
Using Practice Facilitation in Primary Care Settings to Reduce Risk Factors for Cardiovascular Disease

The primary purpose of this study is to evaluate the effectiveness of practice facilitation as a quality improvement strategy for implementing the Million Hearts' ABCS treatment guidelines for reducing cardiovascular disease (CVD) among high-risk patients who receive care in primary care practices in New York City. The ABCS refer to the following: Aspirin in high-risk individuals (A), Blood pressure control and management (B), Cholesterol management (C), and Smoking cessation (S). The long-term goal is to create a robust infrastructure to disseminate and implement evidence based practice guidelines (EBPG) findings in primary care practices and improve practices' capacity to receive and implement other EBPG findings in the future.


Eligibility Criteria:
Inclusion Criteria:

Provider and staff eligibility inclusion criteria.

* Eligibility includes working full or part time at the study site.

Stakeholder eligibility inclusion criteria.

* Steering committee members or other key stakeholder from the following groups: Health Plan Chief Medical Officer, State health officials in the Chronic Disease Program, and leadership from relevant national associations (American Heart Association), members of Advisory Board of CHCANYS (these are physician leaders).

Patient eligibility inclusion criteria:

* at least one of the ABCS risk factors for CVD (i.e., hypertension, hyperlipidemia, eligible for aspirin and/or is a current smoker)
* must have received care at the clinic for at least 12 months
* Patients eligible for aspirin are those with a documented ICD-9 code for ischemic vascular disease in the last 12 months. Similarly, patients with a diagnosis of hypertension and/or hyperlipidemia will have a documented ICD-9 code for the targeted risk factor
* Smokers will be identified by a documented ICD-9 code, prescription for a cessation medication in the last 12 months or documentation in the chart (e.g. meaningful use measure) during the last 12 months (see outcome measures)
Double-masked Study of PG324 Ophthalmic Solution in Patients With Glaucoma or Ocular Hypertension

To evaluate ocular hypotensive efficacy and safety of PG324 Ophthalmic Solution compared to Netarsudil (AR-13324) Ophthalmic Solution and Latanoprost Ophthalmic Solution.

Following completion of the Month 12 study visit procedures, subjects will be offered the opportunity to participate in a 2-month observational (i.e., non-interventional) trial extension.


Eligibility Criteria:
Inclusion Criteria:

1. 18 years of age or older
2. Diagnosis of open angle glaucoma or ocular hypertension in both eyes
3. Unmedicated intraocular pressure \>20mmHg and \<36mmHg in both eyes at 2 qualification visits. Both eyes had to qualify at all qualification visits
4. Corrected visual acuity equivalent to 20/200 Snellen or better
5. Able to give informed consent and follow study instructions

Exclusion Criteria:

1. Clinically significant ocular disease
2. Pseudoexfoliation or pigment dispersion component glaucoma, history of angle closure or narrow angles
3. Unmedicated Intraocular pressure ≥36mmHg
4. Use of more than 2 ocular hypotensive medications within 30 days of screening
5. Known hypersensitivity to any component of the formulation
6. Previous glaucoma surgery or refractive surgery
7. Ocular trauma within 6 months prior to screening
8. Any ocular surgery or non-refractive laser treatment within 3 months prior to screening
9. Recent or current ocular infection or inflammation in either eye
10. Used ocular medication in either eye of any kind within 30 days of screening
11. Mean central corneal thickness \>620µm at screening
12. Any abnormality preventing reliable applanation tonometry of either eye
13. Clinically significant abnormalities in lab tests at screening
14. Clinically significant systemic disease
15. Participation in any investigational study within 60 days prior to screening
16. Used any systemic medication that could have a substantial effect on intraocular pressure within 30 days prior to screening
17. Women who are pregnant, nursing, planning a pregnancy or not using a medically acceptable form of birth control
Study of Percutaneous Coronary Intervention in Left Main Coronary Artery Disease in Patients With Acute Coronary Syndrome at Sohag University Hospitals

Acute coronary syndromes (ACS) encompass a spectrum of cardiovascular disorders characterized by the sudden onset of myocardial ischemia. They are primarily caused by atherosclerotic plaque rupture or erosion, leading to partial or complete occlusion of the coronary arteries. Among the various coronary arteries affected, left main coronary artery disease (LMCAD) is of particular concern due to its high anatomical significance and potential for adverse outcomes

The left main coronary artery (LMCA) is responsible for supplying a substantial portion of the left ventricular myocardium, including the interventricular septum and the anterior and lateral walls. Any obstruction or compromise in blood flow within this critical artery can have severe consequences, including myocardial infarction, heart failure, or even sudden cardiac death. The management of LMCAD associated with ACS represents a significant clinical challenge, necessitating prompt and optimal treatment strategies

Among the various coronary arteries affected by atherosclerosis, left main coronary artery disease (LMCAD) holds particular clinical significance due to its anatomical location and the vital role it plays in supplying a substantial portion of the left ventricular myocardium. The left main coronary artery (LMCA) typically bifurcates into the left anterior descending artery (LAD) and the left circumflex artery (LCX), which together provide blood supply to the majority of the left ventricle, including the interventricular septum and the anterior and lateral walls

The selection of an appropriate treatment strategy for LMCAD associated with ACS is a complex decision that requires careful consideration of multiple factors, including patient characteristics, coronary anatomy, severity of ischemia, and procedural expertise. The emergence of several clinical trials and observational studies exploring the efficacy and safety of different revascularization strategies has further complicated the decision-making process


Eligibility Criteria:
Inclusion Criteria:

* Age \> 18 years.
* Acute coronary syndromes (ACS).
* Left main coronary artery disease (LMCAD) on CA.

Exclusion Criteria:

* Patients with contraindications to coronary angiography or invasive procedures.
* Prior history of percutaneous coronary intervention (PCI) with left main stenting.
* Known allergy or intolerance to the medications or devices commonly used during PCI procedures.
* Severe comorbidities or conditions that may limit life expectancy or impact the ability to follow the study protocol.
* Pregnancy or lactation.
* Refusal to participate
* Contra indication to antiplatelet.
Repetitive Assessement of PRECISE-DAPT Score

Patients treated with percutaneous coronary intervention (PCI) require dual antiplatelet therapy (DAPT). Preliminary assessment of the PRECISE-DAPT score is mandatory in order to assess the risk of bleeding while on DAPT. The score takes into consideration age, creatinine clearance, haemoglobin, white blood- cell count and previous spontaneous bleeding. One should consider, however, that some of the variables included in the PRECISE-DAPT score might change with time. As a consequence, the PRECISE-DAPT score should not be considered a static score as it might vary after the initial computation.

It remains unknown, however, if the use of the delta PRECISE-SCORE, which reflects the change in score between baseline and follow-up, might help to improve the management of PCI patients in order to decrease the bleeding risk during follow-up.

The RE-SCORE trial is a multicenter study aimed at comparing usual care of patients treated with PCI with a novel strategy of change of therapy during follow-up on the base of the Delta PRECISE-DAPT score, as assessed every 3 months in PCI patients receiving DAPT. The primary end-point of the study is the occurrence of bleeding, as defined according to the criteria of the Bleeding Academic Research Consortium (BARC). The second end-point is a composite of cardiac death, myocardial infarction, stroke, definite or probable stent thrombosis (ST), or BARC criteria type 2, 3 or 5 bleeding.

Eligibility Criteria:
Inclusion Criteria:

* Consecutive patients undergoing percutaneous coronary intervention
* Indication to dual antiplatelet therapy for at least 3 months

Exclusion Criteria:

- Contraindications to dual antiplatelet therapy lasting more than 3 months
Efficacy Study of Oxygenated Glycerol Triesters to Treat Xerostomia

The objective of the trial was to evaluate the efficacy and clinical safety and acceptability of Oxygenated Glycerol Triesters in the relief of symptoms of xerostomia versus a reference comparator.

Xerostomia is due to inadequate (hyposialia) or inexistent (asialia) saliva secretion.

There are may causes. Transitory xerostomia may occur in the presence of anxiety (stage fright, fear or dehydration). Prolonged xerostomia is most often related to a systemic disease, certain medicines or to radiotherapy of the head or neck.

Salivation depends upon the autonomic (sympathetic and parasympathetic) nervous system for its production and on the central nervous system for stimuli-dependant excretion. Substances acting on alpha an beta sympathetic receptors, whether agonists or antagonists, may induce hyposialia. Atropine, the main antagonist of the parasympathetic system, is present in many medicines used for pulmonary, ophthalmic or neurological purposes, potentially causing hyposialia. Similarly, imipramine antidepressants, phenothiazine neuroleptics, antihistamines and disopyramide predispose to the onset of hyposialia.

Xerostomia is often accompanied by buccal signs such as impaired sense of taste, halitosis and buccal ulcers and interferes with functions such as speech, chewing and swallowing. Because of reduced salivary secretion , there is a disturbance of the microbial colonization of the buccal cavity. Xerostomia has significant untoward effects on the buccal cavity and the quality of life of patients.

The basis of the treatment of xerostomia involves the use of saliva substitutes and/or saliva stimulants. Oxygenated Glycerol Triesters is neither a replacement for nor stimulant of saliva. It is a lubricant and protective solution for endobuccal spray.

Eligibility Criteria:
Inclusion Criteria:

* men or women over 18 years of age
* with a diagnosis requiring the prescription of psychotropies (antidepressants, tranquilizers, etc) for more than 6 weeks
* having received no palliative treatment for xerostomia for a minimum of the past 2 weeks
* having xerostomia meeting the questionnaire selection criteria and the sialometer method parameters
* patients legally competent to give their consent
* capable of and accepting to participate in the trial and capable of and accepting to answer trial questionnaires

Exclusion Criteria:

* combined treatments incompatible with the trial, un particular alcoholic solutions
* diabetes, any buccal condition requiring antimicrobial treatment
* Sjögren's syndrome or othermedical causes of xerostomia
* current participation in other clinical trials
* patient suffering from cancer
* patients with allergy to or known intolerance of any of the constituents of the investigational products
* patients treated with pilocarpine
Effect of PechaKucha (PK)Presentation Method on Nursing Students' Satisfaction Level With Deep Breathing and Cough Exercise Skills and Education Method

ABSTRACT

Aim: The aim of this study is to examine the effect of the PK presentation method on nursing students' deep breathing and cough exercise skills and the students' level of satisfaction with this method.

Design: The sample of the study consisted of students studying in the 2nd year of the Nursing Department of a foundation university and taking the Fundamentals of Nursing course (n=96). Experimental (n=48) and control (n=48) groups were determined using a randomization program. While the students in the experimental group were taught deep breathing and cough exercises with the PK presentation method, the control group was trained with a traditional Power Point presentation. As data collection tools, student information form, deep breathing and cough exercise skill checklist and Visual Analog Scale measurement tool were used to determine the level of satisfaction with the training method.

Keywords: PechaKucha, Nursing students, respiratory exercise, satisfaction


Eligibility Criteria:
Inclusion Criteria:

* Inclusion criteria Taking the Fundamentals of Nursing course for the first time attending the course on the day of the course

Exclusion Criteria:

* Students taking the course for the second time and those who did not attend the course on the day of the training were excluded from the study.
Comparison of Tai Chi and Pulmonary Rehabilitation on the Effect of Indacaterol in Treatment naïve COPD Participants

Tai-Chi may be a beneficial form of rehabilitation which is acceptable to the Chinese population but no data exist concerning metabolic responses to Tai-Chi in COPD patients. Investigators conduct an Randomized controlled trial to evaluate the synergistic effect of a Long acting β2-agonists with Tai-Chi as a culturally acceptable form of PR in the Asian population. Classical western style pulmonary rehabilitation will serve as a comparator Investigators propose a prospective randomized controlled trial in which 120 bronchodilator naïve participants (Age 40-80 with GOLD II-IV COPD (post-bronchodilator FEV1 ≥ 25% and \<80 % of the predicted normal, and a post-bronchodilator FEV1/FVC \< 0.70) are randomized to receive a 26 weeks course of Indacaterol with either Tai-Chi or conventional Pulmonary Rehabilitation. Only participants who are residents in Xingning city (Guangdong Province, China) will be recruited.

Both Tai-Chi and pulmonary rehabilitation will be given by qualified instructors at a rural location in southern China (Xingning). A qualified UK Physiotherapist will also be involved in the management of pulmonary rehabilitation program to further make sure the high quality of pulmonary rehabilitation has been applied. Both arm participants will also receive in an open label fashion Indacaterol 150µg qd. for 6 months giving 2 therapy groups (Tai-Chi/Indacaterol, pulmonary rehabilitation/Indacaterol). The primary endpoint of this study is change in SGRQ between Tai-Chi/Indacaterol and pulmonary rehabilitation/Indacaterol at 14 weeks after entry and the secondary endpoints are change in FEV1% and six minute walk distance. the planned recruitment will be 120 with a view to obtaining 100 completers. The investigators propose the study starts on 31 Dec 2014 and completes on 30 June, 2016.


Eligibility Criteria:
Inclusion Criteria:

1. bronchodilator naïve patients
2. Age 40-80 with GOLD II-IV COPD (post-bronchodilator FEV1 ≥ 25% and \<80 % of the predicted normal, and a post-bronchodilator FEV1/FVC \< 0.70)
3. patients who are residents in Xingning city (Guangdong Province, China) will be recruited.

Exclusion Criteria:

1. Patients currently or previous on any type of Tai Chi exercise or pulmonary rehabilitation
2. Patients with a history of malignancy of any organ system treated or untreated, within the past 5 years.
3. Patients with clinically significant renal, cardiovascular, neurological, metabolism, immunological, psychiatric, gastrointestinal, hepatic, or haematological abnormalities.
4. Patients with concomitant pulmonary disease (e.g. asthma, lung fibrosis, primary bronchiectasis, sarcoidosis, interstitial lung disorder, tuberculosis)
5. Patients with obesity (BMI\> 40 kg/m2).
6. Patients requiring long term oxygen therapy (\> 12 h a day) on a daily basis for chronic hypoxemia or recovering from acute exacerbation less than 6 weeks.
7. Patients with Asthma.
Diagnostic DNA Methylation Signature in Diagnosing Thyroid Cancer After Fine-Needle Aspiration in Patients With Thyroid Nodules

This trial studies how well the diagnostic deoxyribonucleic acid (DNA) methylation signature works in detecting thyroid cancer in patients with thyroid nodules. Using diagnostic testing, such as the diagnostic DNA methylation signature, may be a less invasive way to check for thyroid cancer.

PRIMARY OBJECTIVES:

I. To validate a novel epigenetic thyroid nodule diagnostic test, diagnostic DNA methylation signature (DDMS) in a retrospective-prospective study with leftover of fine needle aspiration biopsies.

OUTLINE:

Previously collected FNA aspiration specimen samples are analyzed via DDMS assay. Participants undergo FNA aspiration for collection of tissue samples for analysis via DDMS assay.

After completion of study, patients are followed up yearly for 5 years.

Eligibility Criteria:
Inclusion Criteria:

* All patients undergoing FNA for thyroid nodule diagnosis.
* Any race and ethnicity are eligible for the study.
* Patient should have thyroid nodule(s) with indeterminate diagnosis after FNA (Bethesda category of indeterminate FNA \[III-V\]).
The Psychiatric and Cognitive Phenotypes in Velocardiofacial Syndrome

The purpose of this study is to investigate the Psychiatric and Cognitive Phenotypes in Velocardiofacial Syndrome (VCFS), Williams Syndrome (WS)and Fragile X Syndrome Characterization, Treatment and Examining the Connection to Developmental and Molecular Factors


Eligibility Criteria:
Inclusion Criteria:

* chromosomal deletion proven by FISH examination

Exclusion Criteria:
Perceptual Retraining to Reduce Suicide Risk

The study will use a prospective design consisting of three phases. The phases are:

1. the development of the perceptual retraining treatment (PRT),
2. the evaluation of the treatment's feasibility and acceptability, and
3. an assessment of its efficacy.

During the third phase, a pilot study will be conducted in which participants will be randomly assigned to either PRT or a waitlist control group in order to assess efficacy. Diagnostic information and eligibility criteria will be assessed using the Structured Clinical Interview for Diagnostic and Statistical Manual (DSM) 4th ed. (SCID) and the Clinician Administered PTSD Scale for DSM-5 (CAPS-5).

This study will use clinical interviews, self-report measures, and behavioral assessments to evaluate the efficacy of the PRT protocol. The study will recruit approximately 30 veterans. The primary inclusionary criterion will be that participants must show some evidence of olfactory perceptual bias toward military-relevant stimuli (e.g., burning rubber, decay, fuel). Investigators are not requiring current suicidal ideation as an entry criterion because investigators believe that a fair percentage of veterans will have some suicidal ideation/intent but making this an entry requirement will limit the investigators ability to recruit given the tight timeline for the study.

The study will consist of the development and evaluation of a novel intervention. The essential feature of this intervention is the use of a gradient of triggering smells (e.g., burning rubber, decay, fuel, etc.), to which participants will be systematically exposed in an effort to facilitate habituation to these odors. The PRT protocol will be administered over two 1-hour sessions. Participants will return 1 month after their final PRT protocol session to complete a battery of follow-up measures, as well as the CAPS-5.

The primary outcome measures of interest are: olfactory perceptual bias (as measured by the olfactory bias assessment), suicidal ideation and behaviors (as measured by the Beck Suicide Scale and Depressive Symptom Inventory- Suicidality Subscale), and symptoms related to suicide, such as anxiety and trauma (as measured by the Beck Anxiety Inventory and PTSD Checklist for DSM-5, respectively).

Eligibility Criteria:
Inclusion Criteria:

* evidence of olfactory perceptual bias toward military-relevant stimuli (e.g., burning rubber, decay, fuel).
* military combat exposure in Operation Iraqi Freedom, Enduring Freedom, or New Dawn.
* Age 18-60.

Exclusion Criteria:

* Significant medical illness that compromises olfactory perception or tolerance (e.g., significant olfactory disease or dysfunction, cardiovascular disease, respiratory disorders, renal disease, epilepsy, stroke, and uncontrolled hypertension or migraines).
* Evidence of imminent suicidal intent
* Evidence of current or past psychotic-spectrum disorders, or uncontrolled bipolar disorder.
* English speakers.