cyrilzakka/clinical-trials huggingface dataset:
Nucleotide Protein -3 in Epileptic Children

Epilepsy is one of common serious neurological malfunction, characterized by recurrent unprovoked seizures. It always accompanied with multitude of complications as cognitive, behavioral, and psychiatric disorders.

Experimental studies and clinical evidence obtained in animal models of epilepsy and human brain specimen from various drug-resistant forms of epilepsy show the activation of the innate and adaptive immunity mechanisms and the induction of the associated inflammatory processes in the epileptogenic foci.

Epilepsy affects approximately 1% of the world population. Lifetime prevalence of childhood and adolescence epilepsy (children \<18 years) in Upper Egypt was 9.7/1000, with higher prevalence among children \<12 years (10.8/1000).

There is a clear cause for epilepsy in only a minority of the cases, while in up to70% of all case of epilepsy in adults and children, no cause can be discovered. Some of the main causes of epilepsy include: Low oxygen during birth, head injuries that occur during birth or from accidents during youth or adulthood, brain tumors, infections such as meningitis or encephalitis, stroke or any other type of damage to the brain.

A role of inflammatory molecules in the generation of seizures had been first investigated when selected anti-inflammatory treatments, in particular, steroids, immuno-globulins, and adrenocorticotropic hormone (ACTH), were shown to control seizures in pediatric epilepsies refractory to conventional anticonvulsive drugs. In addition, specific epileptic disorders have been associated with the presence of neuronal antigen-directed antibodies in plasma or cerebrospinal fluid (CSF).

A nucleotide-binding oligomerization domains (NODs) are cytosolic proteins that include key regulators of apoptosis and pathogen resistance in mammals and plants. A large number of NODs contain leucine-rich repeats (LRRs), hence referred to as NOD-LRR proteins. The NLRP3 gene provides instructions for making a protein called cryopyrin. Cryopyrin is a member of a family of proteins called nucleotide-binding domain and leucine-rich repeat containing (NLR) proteins. NLR family have two common features: the first is a nucleotide-binding oligomerization domain which is bound by ribonucleotide-phosphates (rNTP) and is important for self-oligomerization. The second is a C-terminal leucine-rich repeat, which serves as a ligand-recognition domain for other receptors (e.g. Toll like receptor (TLR)) or microbial ligands, while NLRP3 has been identified in microglial cells.

Eligibility Criteria:
Inclusion Criteria:

* Epileptic children in Assiut university hospital, pediatric department, neurology unit.

Exclusion Criteria:

* Children with other chronic disease such as liver, kidney or heart disease
* patient who have apparent infection
High Flow Nasal Cannula Rates in Pediatric Asthma

This is a randomized, open-label, 3-armed feasibility trial will examine conventional oxygen therapy (COT) vs high flow nasal cannula at 4L/min flow vs HFNC at 2L/kg/min flow (max 60L/min) in moderate to severe pediatric asthma exacerbations.

The purpose of this feasibility trial is to examine the safety and efficacy of high flow nasal cannula in moderate to severe pediatric asthma exacerbations. Eligible patients will be admitted to the Pediatric Intensive Care Unit (PICU) after initial presentation to the emergency department (ED) or as a transfer from an outside hospital ED. Patients will be admitted to the PICU on standard asthma therapy (continuous albuterol and systemic corticosteroids) based on the discretion of the treating ED physician. Upon PICU admission, eligible patients will be consented, enrolled, and randomized into the study. All patients will be continued on standard asthma therapy of continuous albuterol at 20mg/hr and systemic intravenous corticosteroids. Patient will be randomized into one of three groups: (1) conventional oxygen therapy at 2L/min via standard facemark (control group), (2) high flow nasal cannula at 4L/min, and (3) high flow nasal cannula at 2L/kg/min (max 60L/min). Pediatric Respiratory Assessment Measure (PRAM) scores and vital signs will be taken at baseline and hourly for two hours. After the two-hour long study is completed, patients will be placed on a respiratory support modality as per the treating physician's discretion. Patients will be followed longitudinally after study completion.

Eligibility Criteria:
Inclusion Criteria:

* Age 2-17 years old with prior clinical diagnosis of asthma
* Admitted to PICU on standard asthma therapy defined as continuous albuterol and systemic corticosteroids
* Admitted to PICU on either COT or HFNC
* Admitted to PICU from either Golisano Children's Hospital ED or as a transport from an outside hospital ED within 6 hours of initial presentation
* PRAM score greater than or equal to 4 after completion of initial ED therapy

Exclusion Criteria:

* Admitted to the PICU on NIV or invasive mechanical ventilation
* Admitted to the PICU from general floors or \> 6 hours from initial presentation
* Presence of a tracheostomy or baseline NIV requirement
* Pregnancy
* Immunocompromised State
Safety of Diclofenac Sodium Gel in Knee Osteoarthritis

This study will test the efficacy and safety of topical Voltaren in the treatment of knee osteoarthritis.


Eligibility Criteria:
Key Inclusion criteria

• Osteoarthritis of the knee

Key Exclusion criteria

* Other rheumatic disease, such as rheumatoid arthritis
* Active gastrointestinal ulcer during the last year
* Known allergy to analgesic drugs
Bio-Banking of Specimens for Advanced Lung Disease and Lung Transplant Research

A major goal of this protocol is to support biomarker studies in advanced lung diseases, lung transplantation care, and to improve our understanding of the effects of viral and other infectious exposures to outcomes in our lung transplant and ALD patient populations.

The identification of biomarkers in a patient's blood or tissue that are specific for particular medical conditions (such as interstitial lung disease, chronic obstructive lung disease (COPD), and post-lung transplant rejection) is important for the prevention and early detection of the disease, as well as to advance our understanding of targeted therapies. Availability of biomarkers for diagnosis and for the prediction of patient prognosis and therapy promises personalized medicine. Patients may be selected based on the presence of particular gene mutations or circulating protein levels to receive personalized treatment. Furthermore, knowledge regarding genetic risk and susceptibility to infectious diseases as well as structural lung disease is rapidly growing-as whole genome sequencing and genome-wide association studies have become easier to do, genetic risk has emerged as increasingly important in understanding why and how some patients develop chronic lung disease. This is true for the spectrum of lung diseases treated in the ALD program, from those with airways diseases such as COPD to diffuse parenchymal lung diseases such as idiopathic pulmonary fibrosis (IPF). The availability of human biological specimens for research purposes is crucial for the advancement of medical knowledge of understanding, diagnosing, and treating chronic lung diseases and optimizing post-lung transplant care.

Eligibility Criteria:
Inclusion Criteria:

1. All individuals aged 18 years and older
2. Persons who have undergone lung transplantation at Baylor University Medical Center in the previous 10 years
3. Individual is able to understand and sign the informed consent form

Exclusion Criteria:

1. Significant documented anemia (hemoglobin \<8 g/dL)
2. Blood transfusions within past 3 weeks
3. Active cancer (non-skin cancers)
4. Enrollment against doctor recommendation
5. Patient not able to provide informed consent
Elucidating TAAR-1, Dopamine, and Norepinephrine in Binge Eating Disorder Using Solriamfetol

ENGAGE (Elucidating TAAR-1, Dopamine, and Norepinephrine in Binge Eating Disorder Using Solriamfetol) is a Phase 3, randomized, double-blind, placebo-controlled, multicenter trial to assess the efficacy and safety of solriamfetol for the treatment of binge eating disorder (BED) in adults.

Eligible subjects must have a diagnosis of BED according to the Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-5) criteria. Subjects will be randomized in a 1:1:1 ratio to receive solriamfetol (150 or 300 mg) or placebo, once daily for 12 weeks.

Eligibility Criteria:
Inclusion Criteria:

* Primary diagnosis of BED according to DSM-5 criteria.
* Provides written informed consent to participate in the study before the conduct of any study procedures.
* Male or female, aged 18 to 55 inclusive.

Exclusion Criteria:

* Prior exposure to solriamfetol/Sunosi, through either a clinical study or prescription.
* Unable to comply with study procedures.
* Medically inappropriate for study participation in the opinion of the investigator.
Safety of Dabigatran Etexilate in Blood Clot Prevention in Children

This open-label, single arm prospective cohort study will assess the safety of dabigatran etexilate in secondary prevention of venous thromboembolism in paediatric patients. Children from 0 to less than 18 years of age will be eligible to participate.


Eligibility Criteria:
Inclusion criteria:

* Male or female subjects 0 to less than 18 years of age at the time of informed consent / assent
* Previously documented objective diagnosis of VTE, followed by completed course of initial VTE treatment for at least 3 months (in case of VKA - intended INR between 2 and 3) or completed study treatment (i.e. reached Visit 8) in the 1160.106 trial. Patients, who during the treatment phase of 1160.106 trial were switched from dabigatran etexilate to SOC arm for any reason, are not eligible for this study.
* Presence of an unresolved clinical risk factor requiring further anticoagulation for secondary VTE prevention (e.g. central venous line, underlying disease, thrombophilia, etc.)
* Written informed consent form (ICF) provided by the patient's parent or legal guardian and assent provided by the patient (if applicable) at the time of ICF signature according to local regulations.
* Further inclusion criteria apply

Exclusion criteria:

* Conditions associated with an increased risk of bleeding
* Renal dysfunction (eGFR \< 50 mL/min/1.73m\^2 using the Schwartz formula) or requirement for dialysis. eGFR retesting during the screening period is allowed (once).
* Active infective endocarditis
* Subjects with a heart valve prosthesis requiring anticoagulation.
* Hepatic disease: Active liver disease, including known active hepatitis A, B or C or Persistent alanine aminotransferase (ALT) or aspartate transaminase (AST) or alkaline phosphatase (AP) \> 3 × upper limit of normal (ULN) within 3 months of screening
* Pregnant or breast feeding females. Females who have reached menarche and are not using an acceptable method of birth control, or do not plan to continue using this method throughout the study and / or do not agree to adhere to pregnancy testing required by this protocol
* Patients in age group 0 to \< 2 years with gestational age at birth \< 37 weeks or with body weight lower than the 3rd percentile
* Anemia (hemoglobin \< 80g/L) or thrombocytopenia (platelet count \< 80 x 109/L) at screening. Transfusions during the screening period are allowed, provided that a satisfactory hemoglobin or platelet level is attained prior to visit 2
* Patients who have taken restricted medication prior to first dose of study medication
* Patients who have received an investigational drug in the past 30 days prior to screening, except patients who have completed the treatment period (up to Visit 8) in 1160.106 trial
* Patients who are allergic/sensitive to any component of the study medication including solvent
* Patients or parents/legal guardians considered unreliable to participate in the trial per investigator judgment or any condition which would present a safety hazard to the patient based on investigator judgment
* Further exclusion criteria apply
Efficacy of Botulinum Toxin A in the Treatment of Bruxism-related Symptomatology

Bruxism is defined as a parafunction that consists of a clenching of the jaw and/or grinding of the teeth. It can occur when an individual is sleeping or awake or it can be mixed.

Most often, it is primary or idiopathic bruxism, however when it is related to a cause it is qualified as secondary.

Primary bruxism is a condition that is not well known to the general public, but I is very widespread (12% of the adult population). In addition, it can alter quality of life, as a result of associated sleep disorders, concentration disorders, daytime fatigue, chronic pain, etc.

The current treatment for this condition is only symptomatic. Currently, one of the most effective treatments is the intramuscular injection of botulinum toxin A in the muscles of mastication. The progressive anticholinergic action results in a reduction of functional signs (dental pain, periodontal pain, temporomandibular joint pain, tension headaches, neck pain) and physical signs (reduction of the mass of the injected muscles, alba linea, tongue scalloping, limitation or stability of dental damage).

However, these clinical data are practically not objectified in daily practice. Objective criteria would make it possible to measure the effectiveness of the treatment, to follow the patient, and to identify the early symptoms in order to prevent a recurrence.

The aim of the prospective study is to concretely measure the strength and electromyographic activity of the muscles of mastication before and after botulinum toxin injection.


Eligibility Criteria:
Inclusion Criteria:

* Patient who has given written consent.
* Patient between 18 and 64 years of age.
* Patient who has never received botulinum toxins
* Symptomatic patient in the context of bruxism in failure to the usual treatments (analgesic, physiotherapy, relaxation technique and maxillary retainer)
* Woman of childbearing age using an effective method of contraception
* Symptomatic patients in the context of bruxism, without a maxillary disocclusion retainer.

Exclusion Criteria:

* Protected Adult
* Patient not affiliated to the national health insurance system
* Pregnant or breastfeeding woman
* Refusal to take part in the study
* Neuromuscular diseases, including Myasthenia gravis, Myopathies, Amyotrophic Lateral Sclerosis. Lambert-Eaton Syndrome, patients with peripheral motor neuropathies, patients with underlying neurological disorders.
* Patients with a history of cardiovascular disease
* Patients with epilepsy or a previous seizure episode
* Neurogenic impairment of the face (including facial paralysis, polyradiculoneuritis)
* History of dysphagia or pulmonary aspiration
* Injection site infection (masseter and temporal)
* Hypersensitivity or allergy to botulinum toxin or any of its excipients.
* Treatment with aminoglycosides or anticholinesterase agents.
Stimulating Fat Tissue Storage with Niacin to Reduce Fat Accumulation in the Liver.

Metabolic dysfunction-associated steatotic liver disease (MASLD) (aka non-alcoholic fatty liver disease), commonly occurring in individuals with obesity and type 2 diabetes can lead to liver inflammation/ fibrosis. MASLD results from fat being disproportionately deposited in the liver.

The goal of this mechanistic study is to investigate metabolic response in patients aged 50 to 80 years with non-alcoholic fatty liver disease, after niacin (vitamin B3) treatment.

The main questions it aims to answer are:

* Does Niacin lower the fat deposition in the liver?
* Does Niacin raise White Adipose Tissue storage of dietary fatty acids?

Researchers will compare Niacin to a placebo (a look-alike substance that contains no drug) to compare the metabolic response.

Duration of study per participant: Up to 28 weeks

It will be a randomized crossover study with two 12-week treatment phases (niacin vs. placebo) with a 4-week washout period between the two treatment phases.

The two 12-week treatment phases will be performed in random order. The treatment will be administered once daily, at the end of the largest meal. There will be a 3-week dose escalation: from 250mg (the first week) to 750mg from week 3 onward.

The outcomes will be assessed at the end of each of these two treatment phases in all participants with metabolic visit A and B (i.e., a total of 4 metabolic visits).

Each metabolic visit will last 9 hours: it will be a test meal with perfusion of stable tracers, blood sampling, PET acquisitions using radiopharmaceuticals (18FTHA and 11C-palmitate) and MRI acquisitions.

The two visits A and B will be performed without and with acute administration of niacin with the test meal, respectively, to determine acute niacin-induced reduction in hepatic fatty acid flux.

The two visits will be performed at four to seven-day interval, in random order during the last week of each of the treatment phase.

Eligibility Criteria:
Inclusion Criteria:

* aged 50 to 80 years;
* diagnosed with MASLD, defined as the presence of liver steatosis + abdominal obesity (as defined by the International Diabetes Federation country/ethnic group-specific criteria;
* all women will be post-menopausal.

Exclusion Criteria:

1. Presence of advanced fibrosis (i.e., ≥ F3 based on liver stiffness \> 10kPa) using vibration-controlled transient elastography (FibroScan), serum ALT \> 3 times the normal upper limit, or signs of portal hypertension \[106-109\].
2. Other hepatic disease.
3. Previous diagnosis of diabetes.
4. Overt cardiovascular or renal disease, cancer (other than non-melanoma skin cancer), or other uncontrolled medical conditions.
5. Any contraindication to MRI.
6. Previous intolerance or allergy to nicotinic acid.
7. Having participated to a research study with exposure to radiation in the last two years before the start of the study.
8. Being allergic to eggs
9. Smoking (\>1 cigarette/day) and/or consumption of \>2 alcoholic beverages per day.
Pilot Study of the Effect of Baclofen and Bromocriptine on Luteinizing Hormone Secretion in Pubertal Children

OBJECTIVES: I. Evaluate the effect of bromocriptine on luteinizing hormone (LH) secretion by suppression of prolactin and stimulation of dopaminergic receptors in early pubertal boys and girls.

II. Evaluate the effect of baclofen on LH secretion by stimulation of gamma-aminobutyric acid receptors in early pubertal boys and girls.

PROTOCOL OUTLINE:

Participants receive bromocriptine or baclofen followed by nocturnal measurements of luteinizing hormone, growth hormone, and sex steroids. Pituitary reserve is evaluated with the administration of gonadotropin-releasing hormone, and in children undergoing assessment of short stature, standard provocative stimuli.

Eligibility Criteria:
* Short stature and/or constitutional growth delay
* No epilepsy or risk of epilepsy
Three-dimensional Ultrasonography Thyroid Volume Measurement.

The purpose of this clinical trial is to investigate the accuracy of applying three-dimensional ultrasound on thyroid gland patients when determining a gland volume.

The method will be compared to conventional b-mode ultrasound where three axis measurements (length, width, and depth) are evaluated in the ellipsoid model.

The three dimensional (3D) method is utilizing optical tracking connected to the ultrasound image to form cross-sectional imaging. Patients enrolled in the study are set for complete thyroidectomies enabling a true volume of the gland by water displacement after excision.

The aim is to find if this 3D method is more accurate in volume estimation than the ellipsoid model.


Eligibility Criteria:
Inclusion Criteria:

* Patients referred to Rigshospitalet to undergo a complete thyroidectomy including patients with previous hemithyroidectomy
* Adults, 18 or older.
* Ability to give written informed consent

Exclusion Criteria:

* Thyroid glands that are too enlarged causing visualization/measurement impairment with ultrasonography.
* Thyroid cancer with preoperative suspected extracapsular spread.
* Thyroids or lymph nodes that need frozen section examination.
Shared Decision Making for Choosing Autosomal DOminant Polycystic Kidney Disease Treatment

The purpose of this study is to generate clinical evidence and assess the feasibility of a shared decision-making (SDM) model for the selection of Tolvaptan in patients with ADPKD.

Autosomal dominant polycystic kidney disease (ADPKD) patients, eligible to taking tolvaptan, have to decide whether to take tolvaptan regarding critical factors such as water intake, side effects, and their personal values and preferences. Our study aims to develop and validate a shared decision-making (SDM) model for ADPKD patients considering tolvaptan therapy.

The SDM model we developed include a patient decision aid, educational materials and self-monitoring tools, all provided by a mobile application. This model was built to deliver enough knowledge and considerations during a decision-making process, which would be helpful for patients to reach a decision on tolvaptan.

Through the multi-center hybrid randomized controlled trial, the model's effectiveness and feasibility will be evaluated. Improvements in treatment outcomes, patient satisfaction, adherence, and quality of life are anticipated. This study can propose a structured, patient-centered framework for therapeutic decision-making in ADPKD patients.

Eligibility Criteria:
Inclusion Criteria:

* Participants between the age of 18 to 80
* Participants with a diagnosis with CKD stage 2 or 3
* Participants with a diagnosis with ADPKD classified as 1C, 1D, or 1E according to Mayo classification
* Participants with the ability to communicate and use digital devices
* Participants with no severe visual impairment and cognitive dysfunction

Exclusion Criteria:

* Participants who were previously exposured to tolvaptan
* Participants who are contraindicated to tolvaptan
* Participants who were judged ineligible by the investigator
Biomarkers and Molecular Mechanism Study of Hepatocellular Carcinoma After Radical Resection and Conversion Therapy

Blood samples will be tested to identify circulating tumor DNA and plasma protein levels to potentially improve prediction of long term prognosis and guide treatment options of patients with hepatocellular carcinoma underwent surgical resection.


Eligibility Criteria:
Inclusion Criteria:

* Patients with a first diagnosis of HCC.
* Patients with HCC who are to undergo surgical treatment, with or without neoadjuvant therapy.
* Ability to obtain tissue sample for ctDNA analysis and detectable baseline ctDNA level.

Exclusion Criteria:

* Patients with secondary liver cancer, or intrahepatic cholangiocarcinoma.
* Any other concurrent malignancy.
* History of organ transplant or hepatic encephalopathy.