cyrilzakka/clinical-trials huggingface dataset:
L-leucine in Diamond Blackfan Anemia Patients

Diamond-Blackfan anemia (DBA) is a rare congenital syndrome associated with physical anomalies, short stature, red cell aplasia, and an increased risk of malignancy.

Mutations affecting genes encoding ribosomal proteins cause DBA. Genetic studies have identified heterozygous mutations in at least one of eight ribosomal protein genes in up to 50% of cases.

25% of patients carry a mutation in the ribosomal protein (RP)S19 gene, whereas mutations in RPS24, RPS17, RPL35A, RPL11, and RPL5 are rare.

p53 activation has been identified as a key component in the pathophysiology of DBA after cellular and molecular studies. Other potential mechanisms that warrant further investigation include impaired translation as the result of ribosomal insufficiency, which may be ameliorated by Leucine supplementation.

Despite significant improvements in understanding of the pathophysiology of Diamond Blackfan anemia (DBA), there have been few advances in therapy. The cornerstones of treatment remain corticosteroids,chronic red blood cell transfusions, and hematopoietic stem cell transplantation, each of which is fraught with complications. Other treatments have been shown to be effective in only a few patients or in individual case reports : IL-3, cyclosporine (alone or in combination with steroids), metaclopramide. Gene therapy is still a part of research programs.

There are some indications that the Amino Acid (AA) L-leucine, a translation enhancer, may have some efficacy in DBA and 5q-syndrome, which has the same altered ribosome functions as the DBA. L-leucine is an essential AA that is unique among the branched-chain AA acting as a nutrient regulator of protein synthesis in skeletal muscle and adipose tissue.

Several preclinical studies with DBA lymphocytes exposed to various L-leucine doses, have demonstrated that protein synthesis can be increased by using high doses L-leucine.

Recent clinical data on L-leucine therapeutic use have demonstrated increase the hemoglobin level and transfusion independence in patients with DBA and 5q-syndrom.

These data support the rationale for clinical trial on L-leucine use as a therapeutic agent for DBA patients.

Experimental: one arm L-leucine , dose- 700mg/m2 , per os, 3 time a day, 6 months

Eligibility Criteria:
Inclusion Criteria:

* signed Informed Consent Form
* diagnosed Diamond Blackfan Anemia
* transfusion dependenсe
* adequate renal function
* adequate liver function
* negative B-HCG and adequate contraception

Exclusion Criteria:

* known hypersensitivity to branched chain amino acids
* diagnosed AA metabolism disorder
* prior HSCT
* pregnancy or planning to become pregnant
Ultrasound Patellar Tendon Assessment

448kilohertz capacitive resistive monopolar radiofrequency is a novel technique in physiotherapy and its usefulness and clinical relevance is still to be investigated at both clinical practice and also in performance. Current studies show promising results in different musculoskeletal disorders, however there no studies in the assessment of quality soft tissue in sporty population after the used of this technique.

Patellar tendon injuries is is one of the most common musculoskeletal disorders, presenting a high prevalence in sport populations. Many factors have been proposed as the cause of pain and dysfunction, however there are no studies analysing possibilities of preventing patellar tendon injuries by using a radiofrequency stimulus.

The use of 448kilohertz capacitive resistive monopolar radiofrequency as the focus of the treatment looking for improve the quality of the tissue is still to be explored.

The hypothesis of the present project is that focus treatments on 448kilohertz capacitive resistive monopolar radiofrequency on the patellar tendon will produce better outcomes in terms of ultrasound assessment measured by quantified elastography, and this will decreased the possibilities of get injured.

The stimulus will be carried out on the patellar tendon region. All interventions will be developed by the same examiner, who is a physiotherapist with 6 years of clinical experience.

Eligibility Criteria:
Inclusion Criteria:

* Age between 18-65 years.
* Sporty people with at least 3 days of training per week

Exclusion Criteria:

* Suffer from knee pain.
* Sedentary people
Feasibility Study on the Model School Pediatric Health Initiative Pilot Project

This is a prospective cross sectional study to assess the feasibility of a school based health centre (SBHC) at Sprucecourt Public School, an inner-city elementary school in Toronto. The SBHC allows inner-city children who face barriers to accessing health care the opportunity to receive health care in their school.The primary aim of this study is to determine utilization of the SBHC. Establishing feasibility of the SBHC would support further development and expansion of this program into other inner city schools.

A chart review of the first year of the SBHC was conducted. Feasibility metrics included the number of children who enrolled at the SBHC. Efficacy metrics included the proportion of enrolled children who enrolled at SBHC. Efficacy metrics included the proportion of enrolled children who attended and received new diagnoses and treatment plans

Eligibility Criteria:
Inclusion Criteria:

* All students at Sprucecourt Public School and the surrounding fourteen schools, as well as their siblings (ages 0-12 years), who are enrolled at and have consent to attend the SBHC
* All infants and preschool aged children attending the on-site Parenting and Family Literacy Centre who are enrolled at the SBHC

Exclusion Criteria:

* N/A
A Re-licensing Study to Assess the Efficacy of Inflexal V Formulated With WHO Recommended 2008/2009 Influenza Virus Strains for the Northern Hemisphere

A study to assess whether the Northern Hemisphere 2008/2009 season influenza vaccine Inflexal V fulfills the EMEA requirements for re-registration of influenza vaccines


Eligibility Criteria:
Inclusion criteria:

* Healthy female and male adults
* Aged ≥18 to ≤60 years or \>60 years on Day 1
* Written informed consent

Exclusion criteria:

* Acute exacerbation of bronchopulmonary infection (cough, sputum, lung findings) or other acute disease
* Acute febrile illness (≥38.0 °C)
* Prior vaccination with an influenza vaccine in the past 330 days
* Known hypersensitivity to any vaccine component
* Previous history of a serious adverse reaction to influenza vaccine
* History of egg protein allergy or severe atopy
* Known blood coagulation disorder
* Chronic (longer than 14 days) administration of immunosuppressants or other immune-modifying drugs within 6 months before the first dose of study vaccine; oral corticosteroids in dosages of ≥0.5 mg/kg/d prednisolone or equivalent are excluded; inhaled or topical steroids are allowed
* Known immunodeficiency (incl. leukemia, cancer, HIV seropositivity)
* Investigational medicinal product received in the past 3 months (90 days)
* Treatment with immunoglobulins or blood transfusion(s) received in the past 3 months (90 days)
* Pregnancy or lactation
* Participation in another clinical trial
* Employee at the investigational site, or relative or spouse of the investigator
* Suspected non-compliance
Effectiveness of Yangxue Qingnao Granules in Preventing Post Thrombolytic Hemorrhage Transformation in Patients With Acute Cerebral Infarction

Evaluate the effectiveness of Yangxue Qingnao Granules in preventing post thrombolytic hemorrhage transformation in patients with acute cerebral infarction, and explore its possible mechanism


Eligibility Criteria:
Inclusion Criteria:

* Age ≥ 50 years old, regardless of gender
* After the onset of this disease, the National Institutes of Research Stroke Scale score was 2 ≤ NIHSS ≤ 20 points
* Patients who have developed symptoms within 4.5 hours and can receive rt PA intravenous thrombolysis treatment
* According to the 2019 Diagnosis Points for Major Cerebrovascular Diseases in China, patients with ischemic stroke were diagnosed as having a good recovery from the first or last onset (mRS score ≤ 1 point before this onset)
* Obtain the patient or their legal representative to voluntarily sign an informed consent form approved by the ethics committee.

Exclusion Criteria:

* Intracranial hemorrhagic diseases seen on cranial imaging: hemorrhagic stroke, epidural hematoma, intracranial hematoma, ventricular hemorrhage, subarachnoid hemorrhage, etc; If it is only oozing blood, the researcher can determine whether it is suitable for enrollment
* Severe consciousness disorder: NIHSS scored\>1 point in the 1a consciousness level item
* After controlling the patient's blood pressure, the systolic blood pressure remains ≥ 180mmHg or the diastolic blood pressure remains ≥ 100mmHg
* Blood glucose\<2.8mmol/L (random blood glucose measurement device can be used)
* Patients with severe mental disorders and dementia
* Diagnosed with severe active liver diseases, such as acute hepatitis, chronic active hepatitis, cirrhosis, etc; Or ALT or AST\>2.0 x ULN
* Diagnosed with severe active kidney disease and renal insufficiency; Or serum creatinine\>1.5 × ULN
* Concurrent malignant tumors or ongoing anti-tumor treatment; For subjects diagnosed with malignant tumors after enrollment, the decision to continue participating in the study can be based on the researcher's judgment and the subject's willingness
* Suffering from severe systemic diseases, with an estimated survival time of\<90 days
* History of major surgery within 4 weeks prior to enrollment
* Participated in other clinical studies within the first 30 days of randomization, or is currently participating in other clinical studies
* The researcher believes that it is not suitable to participate in this clinical study
* Patients who are allergic to any ingredient in the study drug
Bilevel Positive Airway Pressure (BiPAP) for the Treatment of Moderate to Severe Acute Asthma Exacerbations

Bilevel Positive Airway Pressure (BiPAP) is increasingly being reported as an effective and safe method of respiratory support for children with severe asthma exacerbations unresponsive to standard therapies and with impending respiratory failure. Much of the evidence base supporting its use comes from retrospective observational studies, and there is currently a lack of data from randomized controlled trials to inform this practice.

The investigators hypothesize that the use of BiPAP in children with moderate to severe asthma exacerbations could reduce the length of hospital stay, need for invasive ventilation, and use of intravenous bronchodilators. The investigators aim to test this hypothesis by randomizing children attending the Emergency Department with a moderate to severe clinical severity score refractory to inhaled bronchodilators to receive either BiPAP in addition to standard asthma care, or standard care alone.

Children aged 2 - 18 years presenting to the Emergency Department (ED) with a moderate or severe asthma exacerbation (Pediatric Respiratory Assessment Measure (PRAM) of \> 3) who fail to improve clinically with standard ED management with inhaled salbutamol and ipratropium will be randomized to receive either standard asthma management according to our local severe asthma guideline or management with BiPAP in addition to standard care. Both groups will receive a comparable dose of systemic steroid and hourly salbutamol inhalers with subsequent weaning according to PRAM score. Patients randomized to receive BiPAP will be admitted to the Pediatric Intensive Care Unit (PICU) and those randomized to the control group will be admitted to the medical ward. Both groups will be monitored with 3-hourly PRAM scoring through the duration of their admission.

Eligibility Criteria:
Inclusion Criteria:

* 2-18 years old
* Admitted to BC Children's Hospital with a clinical diagnosis of an acute asthma exacerbation
* PRAM score of \>3 following initial treatment with three rounds of inhaled salbutamol and ipratropium bromide, and one dose of systemic steroid
* Parents willing and able to sign consent
* Children over the age of 6 willing to provide assent

Exclusion Criteria:

* Clinical suspicion of co-existing bacterial pneumonia: focal crackles or bronchial breathing, and/or major chest x-ray findings
* Impending respiratory failure at presentation requiring direct PICU admission
* Receiving maintenance dose of oral steroid at time of hospital admission
* Any contraindication to BiPAP use including altered mental status, recent bowel surgery, intractable vomiting, or inability to protect airway
* Current tracheostomy, home ventilation (IPPV or NIPPV) or home oxygen requirement
* History of congenital heart disease or chronic respiratory disease (including bronchopulmonary dysplasia, cystic fibrosis, pulmonary hypertension)
* Craniofacial abnormality precluding the use of a tight fitting facial mask
Assessment of Skeletal Maturity Using Proximal Femoral Epiphysis in Patients With Scoliosis

This study aims to explore the use of proximal femoral head for the assessment of skeletal maturity in patients with idiopathic scoliosis. The aim is to validate the use of the stages of proximal femoral epiphyseal closure in assessing pubertal growth landmarks in this patient cohort. The grading system of this skeletal maturity index will be established and its reliability and reproducibility in clinical use will be examined.

There are multiple skeletal maturity measures currently in use which were established with reliability in assessing growth. The advantages and shortcomings of each index was previously examined. This study aims to explore the use of proximal femoral epiphysis, which is in close proximity of the radiographic field of the spine, in assessing the growth status of patients with idiopathic scoliosis. The stages of the proximal femoral epiphyseal ossification has been established with specific definitions. The reliability and reproducibility of using these gradings will be examined by orthopaedic surgeons. Subsequently, the assessment of different pubertal growth landmarks using these gradings will be validated, and to see whether these gradings can provide indications of peak growth and growth cessation. Finally, the relationships of such growth assessment using this proximal femoral epiphysis ossification (PFEO) system and the curve progression in idiopathic scoliosis will be studied.

Eligibility Criteria:
Inclusion Criteria:

* Idiopathic scoliosis
* Open triradiate cartilage or open distal radius epiphysis without capping

Exclusion Criteria:

* Non-idiopathic scoliosis
* Patients with growth hormone deficiency or developmental delay
* Patient has not reached skeletal maturity
* Defaulted and lost to follow-up
Symphion® System In-Office Study

The purpose of this study is to determine the safety and feasibility of using the Symphion system in an office setting.

The purpose is to determine the safety and feasibility of using an intrauterine tissue resection system in the office setting, as well as to expand the national discussion regarding in-office hysteroscopic procedures as it relates to the highest standards of care and cost containment.

Eligibility Criteria:
Inclusion Criteria:

* Subjects must meet the approved Instructions for Use for the Symphion® bipolar hysteroscopic tissue resection system
* Confirmation hysteroscopic evaluation to access appropriate pathology type and size meets the Protocol guidelines for inclusion
* Subject has signed written Informed Consent

Exclusion Criteria:

* Subjects who are pregnant
* Subjects who have an active genital tract infection (as assessed by the physician)
* Subjects who have cervical malignancies
* Subjects who have previously been diagnosed with endometrial cancer
* Subjects who have Type 2 intracavitary myomas
* Type 0 or 1 intracavitary myoma greater than 3.0 cm
A Randomized, Double Masked, Clinical Study of Subjects With Dry Eye Syndrome

The objective of this study is to evaluate the safety, tolerability, and pharmacodynamic activity of ADX-102 Ophthalmic Solutions and ADX-102 Ophthalmic Lipid Solution in subjects with dry eye syndrome.


Eligibility Criteria:
Inclusion Criteria:

* Be at least 18 years of age of either gender and any race;
* Have a reported history of dry eye for at least 6 months prior to Visit 1;
* Have a history of use or desire to use eye drops for dry eye symptoms within 6 months of Visit 1;

Exclusion Criteria:

* Have any clinically significant slit lamp findings at Visit 1 that may include active blepharitis, meibomian gland dysfunction (MGD), lid margin inflammation, or active ocular allergies that require therapeutic treatment, and/or in the opinion of the investigator may interfere with study parameters;
* Be diagnosed with an ongoing ocular infection (bacterial, viral, or fungal), or active ocular inflammation at Visit 1;
* Have worn contact lenses within 7 days of Visit 1 or anticipate using contact lenses during the study;
* Have previously had laser-assisted in situ keratomileusis (LASIK) surgery within the last 12 months;
* Have used cyclosporine 0.05% or lifitegrast 5.0% ophthalmic solution within 45 days of Visit 1;
* Have any planned ocular and/or lid surgeries over the study period or any ocular surgery within the last 6 months;
* Be using or anticipate using temporary punctal plugs during the study that have not been stable within 30 days of Visit 1;
Respiratory Monitoring of Intrathecal Baclofen- a Feasibility Study

The methodology is a feasibility study to determine the feasibility of assessing people in the community with a neurological condition before proceeding towards a full powered community trial evaluating the influence of intrathecal baclofen.

The purpose of this proposed research will evaluate a feasible method to assess the respiratory status of people with neurological conditions receiving or scheduled to receive Intrathecal Baclofen. The use of Intrathecal Baclofen to manage severe spasticity is a developing treatment option with a limited research base, particularly when evaluating the influence of intrathecal baclofen on the respiratory system. The proposed research is derived from clinical practice and involves joint collaboration between clinical team, research professionals and patients.

In order for the study to evaluate the influence of Intrathecal baclofen has on the respiratory system, the proposed research will need to develop and evaluate a method of assessing people in the community ensuring it's feasible for people with neurological conditions. Prior to implementing a full powered community trial to evaluate the influence of intrathecal baclofen on people with neurological conditions.

Eligibility Criteria:
Inclusion Criteria:

* -Adults over the age of 18
* A person who is able to provide consent or a family member/ close friend who is able to assent on behalf of the participant
* A person or a family member/ close friend who is able to understand English (understand written English and spoken instructions)
* A person currently receiving Intrathecal Baclofen or is scheduled to receive Intrathecal Baclofen (awaiting fitting of pump) therapy to manage spasticity
* A patient of the Solent/ University Hospitals Southampton NHS Foundation Trust Intrathecal baclofen service

Exclusion Criteria:

* -Pre-existing use of overnight non-invasive ventilation (NIV) and is compliant with NIV treatment
* A person who is unable to consent to the study and does not have a family/ close person to assent
* The study will exclude a person who is unable to independently remove the polysomnography and is also without supervision from another person (carer or partner) during the night. This ensures the participant is able to take off the polysomnography (or assisted by a carer) if it becomes uncomfortable and/or causes distress.
Effectiveness of Iguratimod Versus Placebo to Treat Early Rheumatoid Arthritis on MRI

The purpose of this study is to evaluate the efficacy of Iguratimod versus placebo on synovial inflammation,bone erosion and bone edema as measured by MRI of wrist and Metacarpophalangeal joints in patients with early Rheumatoid Arthritis (RA).

Magnetic resonance imaging (MRI) is a powerful imaging modality that is now widely used in both scientific research and clinical settings to visualise joints of patients with RA. MRI is able to image structural damage and soft tissue changes, ie, synovitis, bone oedema, damage to cartilage and bone, as well as tendon pathology. MRI is more responsive to change in joint damage and can be used to monitor disease progression.

In this studly,MRI of the dominant wrist was performed at baseline ,24 weeks and at 52 weeks, using a 1.5T or 3.0T MRI with a dedicated high-resolution wrist phased array coil.The same scanner and wrist coil were used for three examinations. The hand was placed in the wrist coil at the patient's side with the coil anchored to the base tray to reduce motion artefacts. The MRI sequences in this study included the OMERACT recommended MRI core set of sequences.

Eligibility Criteria:
Inclusion Criteria:

* Subjects with a diagnosis of RA according to the diagnostic criteria of the American College of Rheumatology (ACR) (revised in 1987)
* Rheumatoid Arthritis for 3 months to 2 years from the time of the initial diagnosis
* Functional Class II-III
* Subjects have active RA at the time of screening
* Must have a negative Pregnancy test and use adequate method of contraception throughout the trial
* Must have at least 4 tender joints and 4 swollen joints(involved in DAS28) and among these,at least one swollen Metacarpophalangeal joint is essential
* Has a C-reactive protein ≥ 10 mg/L OR Erythrocyte Sedimentation Rate (ESR) ≥ 28 mm/hr
* Written informed consent

Exclusion Criteria:

* Preceding treatment with DMARDs, immunosuppressants (cyclophosphamide, cyclosporine, azathioprine, etc.), biological agents or tripterygium within 12 weeks prior to study entry
* Chest x-ray abnormalities, such as tuberculosis, interstitial pulmonary fibrosis, etc
* ALT \>1.5×ULN, AST \>1.5×ULN, Cr \>135umol/L or Cr \>1.5mg
* WBC\<4×109/L，HGB\<85g/L，PLT\<100×109/L
* Subjects with serious cardiovascular, renal, hematologic,endocrine diseases or malignant
* Subjects with immunodeficiency, uncontrolled infection and active gastrointestinal disease
* Pregnant, intend to become pregnant, or are breastfeeding
* Subjects with other rheumatological diseases such as SLE, mixed connective tissue disease, scleroderma, polymyositis, etc
* Subjects with inflammatory arthritis, such as gout, reactive arthritis, psoriatic arthritis, seronegative spondyloarthropathy, Lyme disease
* Subjects with intra-articular corticosteroid injections within 6 weeks prior to study entry
* Allergic to any of the study drugs
* History of alcoholism
* Subjects with mental illness
* Subjects receiving live vaccines recently
* Subjects participating in other clinical study within 3 months prior to study entry