cyrilzakka/clinical-trials huggingface dataset:
Myotubular and Centronuclear Myopathy Patient Registry

The Myotubular and Centronuclear Myopathy Patient Registry (also referred to as the 'MTM and CNM Registry') is an international, patient-reported database specific to these conditions.

More details and online registration are available at www.mtmcnmregistry.org.

The Myotubular and Centronuclear Myopathy (MTM \& CNM) Patient Registry is managed and operated by the John Walton Muscular Dystrophy Research Centre at Newcastle University, in partnership with the Myotubular Trust, and is part of the TREAT-NMD Neuromuscular Network. The registry has been developed in partnership with a number of leading neuromuscular researchers, and is jointly funded by the Myotubular Trust, Muscular Dystrophy UK and Astellas Gene Therapies.

Participants register online and must provide consent before accessing the registry questionnaire. The clinical data and genetic or biopsy reports are provided by the participants and their doctors.

The MTM \& CNM Registry aims to:

* Help identify patients for relevant clinical trials as they become available.
* Encourage further research into myotubular and centronuclear myopathy.
* Provide researchers with specific patient information to support their research.
* Assist doctors and other health professionals by providing them with up-to-date information on managing myotubular and centronuclear myopathy, to help them deliver better standards of care for their patients.

The investigators welcome the registration of:

* All patients with a myotubular myopathy or centronuclear myopathy diagnosis, which has been confirmed via genetic testing or muscle biopsy.
* Any carrier females of x-linked myotubular myopathy, especially if they have manifested myotubular myopathy type symptoms.
* Any patient who is deceased, but who had a confirmed diagnosis.

This is an online registry and is hosted on the RDRF (Rare Disease Registry Framework) by Murdoch University.

More details and online registration are available at www.mtmcnmregistry.org.

Eligibility Criteria:
Inclusion Criteria:

* Patients with a myotubular myopathy or centronuclear myopathy diagnosis, which has been confirmed via genetic testing or muscle biopsy.
* Any carrier females of x-linked myotubular myopathy, especially if they have manifested myotubular myopathy type symptoms.
* Any patient who is deceased, but who had a confirmed diagnosis.

Exclusion Criteria:

- None
Fish Oil Supplementation in Late-life Depression

To investigate whether fish oil supplementation, compared to placebo (olive oil), could have better effects on depression course and cognitive function in older people with major depression.

Patients with late-life depression were reported cognitive impairment, especially in information-processing speed, working memory, attention and episodic memory, even after depressive symptoms subside and some of them further progress to dementia in two to four years. Several epidemiological studies, fatty acid comparison studies, animal studies, and clinical trials found that omega-3 polyunsaturated fatty acids (PUFAs) were significantly associated with the major depression, cognitive decline in general population and Alzheimer's disease. Until now, there is no study investigating the effects of n-3 PUFAs on depressive symptoms and cognition in patients with late-life depression. Thus, the aims of this study were to investigate whether fish oil supplementation, compared to placebo (olive oil), could have better effects on depression course and cognitive function in older people with major depression.

Eligibility Criteria:
Inclusion Criteria:

* An age range of 60 years old or over;
* A previous diagnosis of major depressive disorder according to the Chinese version of Structured Clinical Interview for DSM IV-TR Axis-I Disorder;
* Depressive symptoms were stable for at least three consecutive weeks and the 17-item Hamilton Depression Rating Scale score less or equal to 10.
* Capacity to provide informed consent.

Exclusion Criteria:

* People with severe or acute medical illness ( such as metastatic cancer, brain tumor, decompensated cardiac, hepatic, or renal failure, or myocardial infarction or stroke) within the 3 months preceding the study.
* Those who had neurological disorders involving central nervous system, such as delirium, Parkinson's disease, aphasia or multiple sclerosis)
* Prominent cognitive impairment, defined as the Chinese version of Mini-Mental State examination score less than 17.
* People with alcoholism, defined by a score of 8 or higher in male and of 6 or higher in female on the Chinese version of the Alcohol Use Disorders Identification Test (AUDIT) questionnaire.
A Study of LY3509754 in Healthy Non-Japanese and Japanese Participants

The main purpose of this study in healthy participants is to learn more about the safety of LY3509754 and any side effects that might be associated with it. Blood tests will be performed to check how much LY3509754 gets into the bloodstream and how long it takes the body to eliminate it.


Eligibility Criteria:
Inclusion Criteria:

* Overtly healthy males or females, as determined by medical history and physical examination.
* Body mass index (BMI) within the range of 18 to 35 kilograms per meter squared (kg/m²) in Parts A, B, and C. In Part D (Japanese participants), body weight between 50 and 85 kg and BMI within the range of 18 to 28 kg/m².

Exclusion Criteria:

* Have previously completed or withdrawn from this study or any other study investigating LY3509754, and have previously received LY3509754.
* Women of childbearing potential are excluded from the trial.
Protein Balance in Protein Supplemented Elderly Patients: Acute Study With Protein-tracer-techniques

The aim of this study is to investigate potential protein synthesis effects of whey protein high in leucine compared to whey protein with normal leucin content in elderly patients in a acute crossover setting. We will use the method of a 2 X 3 h tracer infusing protocol with 15Nphenylalanine along with blood samples and muscle biopsies.

Background: Improved muscle function/strength will enhance the quality of life of the more frail elderly population. Dietary proteins and amino acids act as anabolic substrates in muscles in elderly people. In particular the amino acid leucine is suggested to have an impact on muscle protein synthesis. In collaboration with the Danish dairy company Arla Foods we will use their conventional Whey protein and leucine enriched Whey protein in an acute study on elderly patients.

Our aim is to examine the acute effects of supplementation with whey protein high in leucine compared to whey protein with normal leucine content on muscle protein synthesis and protein balance in elderly patients.

Methods: We include 10 elderly patients, age 60-85 years in a randomized crossover study. The subjects will be recruited from the Osteoporosis Section at the Department of Endocrinology at Aarhus University Hospital. The subjects are their own controls in 2 x 1 day interventions A: whey protein high in leucine and B: whey protein with normal leucine content. Interventions are conducted with a 4 week washout period between them. On the intervention day postprandial protein synthesis rate will be measured over 2 X 3h using a tracer infusing protocol with 15Nphenylalanine along with blood samples and muscle biopsies. For analyzing steps of signaling in muscle protein synthesis initiated by leucine mammalian target of rapamycin (mTor) and sites downstream from mTor is quantified using western blotting. Whole body protein kinetics are calculated using the formula Q=i\*Ei/Ep-i

Eligibility Criteria:
Inclusion criteria:

- Healthy elder people

Exclusion criteria:

* Liver and kidney disease.
* Known diabetes or Hgb1c ≥6,5% (≥48mmol/l).
* Severe heart disease (NYHA-Class \>2).
* Oral corticosteroid treatment within the last 3 month.
* participation in other intervention studies within the last 4 weeks.
Investigate the Role of microRNA in Chronic Rhinosinusitis From DC-Th Axis

This study aimed to evaluate the upstream events of Th17/Treg imbalance in CRS and their immune regulatory factors. Then the investigators aimed to explore the regulatory role of miRNA on DC-Th axis and its dysfunction in the pathogenesis of CRS, in order to determine the miRNA expression profile in CRS and clarify the role of miRNA in the pathogenesis of CRS by regulating the DC-Th axis.

Chronic rhinosinusitis (CRS), commonly encountered in the ﬁeld of clinical otorhinolaryngology, is still a challenging proposition for doctors because of its high incidence and the unsatisfactory treatment outcomes. Nowadays, studies on the pathogenesis of NP are still attached great importance by researchers from each country. NP has become a global health problem with a considerable socioeconomic burden. Recently, research showed that the impaired balance of Th17/Treg was the significant basis of NP. Nevertheless, the pathogenesis of Th17/Treg imbalance was unclear. In this study, DC-Th axis was designed as the main line, and the regulation of miRNA on DC was designed as the entry point. This study aimed to evaluate the upstream events of Th17/Treg imbalance in CRS and their immune regulatory factors. Then the investigators aimed to explore the regulatory role of miRNA on DC-Th axis and its dysfunction in the pathogenesis of CRS, in order to determine the miRNA expression profile in CRS and clarify the role of miRNA in the pathogenesis of CRS by regulating the DC-Th axis. This study will play an important role in clarifying the pathogenesis of CRS eventually and will fill the blanks of the research between miRNA and CRS in the investigators'country. This study is with important clinical value for establishing control strategies for CRS.

Eligibility Criteria:
Inclusion Criteria:

1. CRS diagnosis is accord with diagnostic criteria of The European nasal sinusitis, nasal polyps guidelines (EPOS 2012) .
2. Selected subjects are stopped using systemic or topical corticosteroids in one month before experiment.
3. All the patients have sinus CT and nasal endoscopy and allergen Skin Prick Test in preoperative.
4. Aged from 18 to 75 years old.
5. Woman or man.

Exclusion Criteria:

1. Choanal polyp, fungal nasal sinusitis, cystic fibrosis.
2. Acute upper respiratory tract infection and other diseases associated with the body.
Early Detection of Ventilator-associated Pneumonia (VAP)

The study goal consists in the sequential collection of exhaled breath condensates (EBC) from patients receiving invasive ventilation. Subsequent Raman spectroscopy aims at the identification of putative biomarkers that could enable rapid early distinction of Ventilator-associated pneumonia.

Diagnostics of VAP relies on two complementary strategies: meeting pre-defined clinical criteria and evidence of pathogens in the lower respiratory tract. Mere reliance on clinical symptoms, such as radiologic evidence, systemic signs of inflammation and indices of compromised pulmonary function reportedly lead to misdiagnosis. Current diagnostic procedures recommend gathering of evidence for new or progressive lung infiltrates from serial chest radiographies, together with co-existing clinical signs of impaired respiratory function. Microbiological confirmation of pathogen presence requires examination of airway secretions collected by invasive bronchoalveolar lavage which demands specific technical skills and staff training. Although verification of causal pathogens is essential for the introduction of targeted antibiotic therapy, the procedure is time-consuming and qualitative in reportedly less than 30 % of the cases. Furthermore, the currently adopted diagnostic approaches are based on sporadic information sampling and do not support continuous monitoring and evaluation of the effect of therapeutic interventions. All above-listed flaws and shortcomings emphasize the need for rapid, reliable and non-invasive recognition of VAP, preferably in a setting that would permit continuous bedside monitoring and timely introduction of targeted drug therapy.

The study objective consists in sequential collection of exhaled breath condensates (EBC) in ICU patients receiving invasive ventilation. The EBC samples will be subjected to determination of volatile organic compound (VOC) profiles by Stimulated Raman Spectroscopy (SRS) which, upon individual matching to routinely collected clinical parameters, may become putative biomarkers for the early recognition of VAP. Evidence has accumulated in support of the assumption that certain metabolites in EBC might display significant profile differences as to their size, hydrophobicity and electrical charge. Identification of VAP-specific profiles of VOC will provide the basis for the generation of a data base enabling the construction and standardization of a bedside device for continuous real-time point-of-care monitoring of VAP hazard in patients on invasive ventilation.

Eligibility Criteria:
Inclusion Criteria:

* ICU patients on invasive ventilation
* Signed informed consent

Exclusion Criteria:

* Glucocorticoid dosage above 0.3 mg/kg Prednisolone equivalent over more than 3 weeks
* Treatment with recognized T cell immunosuppressant (cyclosporine, Tumor Necrosis Factor antagonists) or nucleoside analogues during the preceding 90 days
* Inherited severe immunodeficiency
* Solid organ or stem cell transplant recipients
* Anti-cancer chemotherapy during the preceding 90 days
Endothelial Disfunction Treatment With Glimepiride/Metformin Combination (Glimetal) in Type 2 Diabetes Patients

The Purpose of this study was to evaluate the effect of the combination glimepiride/metformin over endothelial dysfunction (ED) in asymptomatic patients with type 2 diabetes mellitus (DM) using 13N-ammonia-positron emission tomography (PET).


Eligibility Criteria:
Inclusion Criteria:

* Both genders
* At least 18 years old
* Type 2 diabetes mellitus diagnosis
* Signed Informed Consent

Exclusion Criteria:

* History of smoking, hypertension, dyslipidemia, ischemic heart disease or autoimmune rheumatic diseases
* Pregnancy or lactation
* History of abuse and/or substance dependence within 6 months preceding the survey.
* History of glimepiride or metformin allergy
Effect of Remifentanil on Hemodynamic Stability During Placement of a Mayfield Head Fixation Device for Craniotomy

To identify a better method using a combination of routine anesthetic drugs to improve hemodynamic stability during Mayfield head pinning for craniotomy surgery.

A randomized, placebo-controlled, double-blinded comparative effectiveness study to test hypothesis that addition of remifentanil bolus to a standard anesthetic drug regimen will better attenuate the hemodynamic response to the noxious stimulus of Mayfield head pin insertion than the standard anesthetic drug regimen.

Eligibility Criteria:
Inclusion Criteria:

* Adult non-pregnant patients
* undergoing general anesthesia for a craniotomy
* requiring a Mayfield head fixation device.

Exclusion Criteria:

* Patients under 18 years of age,
* non-English speaking,
* pregnancy,
* opioid tolerance,
* illegal drug use or alcohol abuse.
Response to SARS-CoV-2 Vaccine in Stem Cell Transplant and Cellular Therapy Patients

For many patients with hematologic disorders and bone marrow failure, hematopoietic stem cell transplantation (HSCT) or cellular therapy (CART) offers a curative treatment option. Patients after SCT or CART have a variable period of immune deficiency in the post-treatment period. The response to vaccination may affect the outcome of the transplant patients. the immunogenicity of vaccines in this immunosuppressed population is uncertain and variable. HSCT and CAR-T recipients are in a COVID-19 high-risk group and conferring immunity by vaccination at the earliest effective timepoint is desirable. At present, the immunogenicity and efficacy of SARS-CoV-2 vaccines in immune-impaired patients including autologous and allogeneic HSCT recipients is unknown. Furthermore, the impact of GvHD and IST on SARS-CoV-2 vaccine immunogenicity is unknown. the investigators aim to evaluate the vaccination response to COVID vaccines after SCT and CART

so for participants with SCT or CART who decide to get their vaccines, the investigators will measure response by therapy by measuring COVID-19 serology IgG at the following schedule: Baseline +5 days after vaccination; 1 month +/- 10 days (after the first dose), 2 months +/- 10 days (after the 2nd dose), 3 months +/- 10 days, 6 months +/- 10 days and 12 months +/- 10 days In addition, also the investigators will collect CD4 and Total IgG which is the standard of care of these patients post SCT and CART

Eligibility Criteria:
Inclusion Criteria:

* any SCT or CART post COVID vaccine

Exclusion Criteria:

* none
Clinical Course of Cirrhotic Patients With Portal Vein Thrombosis Treated With TIPS

Portal vein thrombosis (PVT) is defined as an obstruction of the portal vein. The prevalence of PVT is 10-25% and incidence is about 16% in cirrhotic patients. PVT leads to increased intrahepatic resistance, decreased portal velocities, splanchnic vasodilatation, and stagnant flow.

Portal vein recanalization (PVR) with transjugular intrahepatic portosystemic shunt (TIPS) is aimed at restoring main portal vein (PV) flow in chronic PVT. In this study, we will review the safety and outcomes of this approach.

Portal vein thrombosis (PVT) is a fairly common complication of liver cirrhosis. Importantly, occlusive PVT might influence the prognosis of patients with cirrhosis. Transjugular intrahepatic portosystemic shunt (TIPS) has been reported to be an effective treatment of PVT in cirrhosis, with the advantage of avoiding the risk of bleeding linked to anticoagulation. In this study, we will evaluate the outcome of TIPS for PVT in patients with and without cirrhosis and determinate survival.

Eligibility Criteria:
Inclusion Criteria:

* Written informed consent
* Liver cirrosis or non-cirrhotic patients
* Portal vein thrombosis (degree of vessel obstruction \> 50%)

Exclusion Criteria:

* Active variceal bleeding
* Prior history of TIPS placement or shunt surgery
* Concomitant renal insufficiency
* Severe cardiopulmonary diseases
* Uncontrolled sepsis Serious medical conditions which may reduce the life expectancy
* Contraindications for TIPS
Evaluating the Effect of Injectable Platelet-rich Fibrin on Accelerating Orthodontic Tooth Movement

One of the main goals of orthodontic treatment is the reduction of treatment time through faster tooth movement. The previous studies evaluating platelet-rich fibrin (PRF) and orthodontic tooth movement (OTM) are limited, which makes the results difficult to generalize.

One of the main goals of orthodontic treatment is the reduction of treatment time through faster tooth movement. The previous studies evaluating platelet-rich fibrin (PRF) and orthodontic tooth movement (OTM) are limited, which makes the results difficult to generalize. To this end, we set up this Randomized experiment to evaluate the effect of the injectable form of PRF (i-PRF) on OTM, using nonadditive containing i-PRF, which has higher levels of regenerative cells and growth factors compared with other PRF types because of the use of low centrifugation spee. Accordingly, we aimed to evaluate the effect of i-PRF on the rate of maxillary incisors leveling during comprehensive orthodontic treatment.

Eligibility Criteria:
Inclusion Criteria:

* The patient's age (18-26). Severe or very severe irregularity of the upper incisors greater than (7 mm) according to Little's index, so it is recommended to extract the upper first premolar.

  * Class I or Class II malocclusion, first model according to Angle, with skeletal class I or II and a normal or mild vertical growth pattern.
  * All permanent upper teeth, up to the first molar, are present, with the possibility of attaching brackets to all teeth in a correct position.
  * The patient has good oral health.

Exclusion Criteria:

* • The presence of any systemic disease that affects orthodontic dental movement.

  * Severe malalignment of one of the teeth (palatal quadrant, ectopic canine, ectopic premolar).
  * The patient has undergone previous orthodontic treatment.
  * The patient is subject to any drug treatment that may affect orthodontic dental movement (cortisone, non-steroidal anti-inflammatory drugs).
  * The patient has poor oral health.
  * Commitment to periodic follow-up appointments.
Trial Measuring the Clinical Impact of the FilmArray® Pneumonia Panel in Critically Ill

The objective of this study is to measure how the use of Film-Array Pneumonia (FA-PNEU) on respiratory samples of adult patients suspected of acute care pneumonia in the intensive care unit of Saint-Luc University Hospital can accelerate / improve the initiation of the optimal antibiotic treatment.

The main objective of this trial is to confirm/attest/prove the following statement:

The use of the FilmArray Pneumonia panel on respiratory samples of critically ill suspected with pneumonia, enables a faster microbiological pneumonia diagnosis and an improved therapeutic management of the patient.

Eligibility Criteria:
Inclusion Criteria:

* Adult patients 18 years, remaining at the intensive care of the Saint-Luc University Hospital
* Recently transferred from emergency care with Community acquired Pneumonia of grade 4 suspicion
* With a Ventilation acquired Pneumonia suspicion

Suspicion will be based on symptoms, clinical, biological and radiological criteria.

Exclusion Criteria:

* Patients from whom no respiratory sample can be obtained
* Patients benefitting from palliative care
* Insufficient volume of the respiratory sample after routine testing to perform the FA-PNEU test.
Safety and Efficacy of Human Bone Marrow Stem Cells for Treatment of HBV-related Liver Cirrhosis

HBV related Liver disease is a common medical problem in China. An estimated 7.18% of the Chinese (about 93 million) is infected with hepatitis B, and most of the HBV- related hepatitis can developed into liver cirrhosis.

Liver transplantation is the only available life saving treatment for patients with end stage liver disease. However, lack of donors, surgical complications, rejection, and high cost are serious problems.

stem cells(SCs) possess plasticity and have the potential to differentiate into hepatocyte; Thus, SCs hold great hope for therapeutic applications. Adult bone marrow is the most common source of SCs for clinical applications.Previous study showed that bone marrow derived stem cells (BMSCs) replace hepatocytes in injured liver, and effectively rescue experimental liver failure and contribute to liver regeneration. In this study, the patients with HBV-related liver cirrhosis will undergo administration of human autologous BMSCs via hepatic artery to evaluate the safty and efficacy of human autologous BMSCs treatment for these patients.


Eligibility Criteria:
Inclusion Criteria:

1. Aged 18-65 years
2. HBV-related liver cirrhosis
3. Child-Pugh score 9-15
4. Written consent

Exclusion Criteria:

1. Hepatocellular carcinoma or other malignancies
2. Severe problems in other vital organs(e.g.theheart,renal or lungs)
3. Pregnant or lactating women
4. Severe bacteria infection
5. Anticipated with difficulty of follow-up observation
6. Other candidates who are judged to be not applicable to this study by doctors