cyrilzakka/clinical-trials huggingface dataset:
A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia

Study 111-209 is a Phase 2 randomized, open-label clinical trial of BMN 111 in infants and young children with a diagnosis of Achondroplasia at a heightened risk of requiring cervicomedullary decompression surgery


Eligibility Criteria:
Inclusion Criteria:

* Parent(s) or guardian(s) willing and able to provide signed informed consent after the nature of the study has been explained and prior to performance of any research related procedure.
* Have ACH, documented by genetic testing.
* Are willing and able to perform all study procedures as physically possible.
* Age 0 to ≤ 12 months, at study entry (Day 1). Given that any potential impact of vosoritide therapy on the foramen magnum is dependent on treating as early as possible and as long as possible while the synchondroses at the base of the skull are still open. For subjects \> 6 months of age at enrollment, a discussion between the investigator and the Medical Monitor should occur with the goal of limiting the number of subjects in the range of \> 6 months to ≤ 12 months of age.
* Parent(s) or caregiver(s) are willing to administer daily injections to the subject and complete the required training.
* Have evidence of CMC that "may" require surgical intervention

Exclusion Criteria:

* Have hypochondroplasia or short-stature condition other than achondroplasia (eg, trisomy 21, pseudoachondroplasia, etc).
* Have CMC that either does not require surgical intervention (for example foramen magnum narrowing with preservation of the cerebrospinal fluid space) or does require immediate surgical intervention .
* Have any of the following: Untreated congenital hypothyroidism or maternal history of hyperthyroidism, Insulin-requiring neonatal diabetes mellitus, Autoimmune inflammatory disease, Inflammatory bowel disease, Autonomic neuropathy.
* Have a history of any of the following:Renal insufficiency, Chronic anemia,Baseline systolic blood pressure below age and gender specified normal range or recurrent symptomatic hypotension (defined as episodes of low blood pressure generally accompanied by symptoms eg, pallor, cyanosis, irritability, poor feeding) and Cardiac or vascular disease.
* Have a clinically significant finding or arrhythmia that indicates abnormal cardiac function or conduction or QTc-F ≥ 450 msec on screening ECG.
* Have been treated with growth hormone, insulin-like growth factor 1, or anabolic steroids in the 6 months prior to Screening, or long-term treatment (\> 3 months) at any time.
* Have ever had prior cervicomedullary decompression surgery.
* Have had a fracture of the long bones or spine within 6 months prior to Screening.
Heterogeneity of Metastatic Neuroendocrine Tumors as Determined With 18F-dihydroxyphenylalanine-PET /CT, a Retrospective Analysis

In an individual cancer patient extensive genetic and phenotypic variation exists between and within tumor lesions. Tumour load in NET patients can be measured with fluorine-18-L-dihydroxyphenylalaninepositron emission tomography (18F-DOPA-PET) scan.

This aim of the study is to investigate heterogeneity between tumor lesions within patients with intestinal NET by determining the differences in tracer uptake measured on a 18F- DOPA-PET scan retrospectively.

In an individual cancer patient extensive genetic and phenotypic variation exists between and within tumor lesions. Tumour load in NET patients can be measured with fluorine-18-L-dihydroxyphenylalanine positron emission tomography (18F-DOPA-PET) scan.

This aim of the study is to investigate heterogeneity between tumor lesions within patients with intestinal NET by determining the differences in tracer uptake measured on a 18F- DOPA-PET scan in retrospect.

Eligibility Criteria:
Inclusion Criteria:

* age ≥18 years
* diagnosis of intestinal NET diagnosed by a dedicated NET specialist
* more than one positive lesion on the 18F-DOPA-PET scan.
* available CT-scan within 6 months before or after the 18F-DOPA-PET-scan.

Exclusion Criteria:

* no clinical data available at the UMCG, besides the results of the DOPA PET scan.
Breathing-swallowing Interaction in Chronic Obstructive Pulmonary Disease Patients - Impact of Non Invasive Ventilation

In the investigators' knowledge there are no data about the impact of non invasive mechanical ventilation on the breathing-swallowing interaction.

Our main objective is to evaluate breathing-swallowing interaction in Chronic Obstructive Pulmonary Disease (COPD) patients hospitalized in intensive care unit for an acute exacerbation, and evaluate the impact of using non invasive mechanical ventilation (NIV)

In healthy subjects previous studies showed that most swallows started during expiration and were followed by expiration, a pattern believed to contribute to airway protection during swallowing. However In healthy individuals, the occurrence of inspiration after swallows was increased by hypercapnia or application of an inspiratory elastic load.

In a previous study the investigators have demonstrated that patients with neuromuscular disorders exhibited piecemeal deglutition leading to an increase in the time needed to swallow a water bolus, as well as occurrence of inspiration after nearly half the swallows. These abnormalities which increased with the decreasing of respiratory muscle performances may explain feeding difficulties. However in tracheostomized patients who could breathe spontaneously, piecemeal deglutition and swallowing time per bolus were diminished by the use of mechanical ventilation.

In the investigators' knowledge there are no data about the impact of non invasive mechanical ventilation on the breathing-swallowing interaction.

The investigators' main objective is to evaluate breathing-swallowing interaction in Chronic Obstructive Pulmonary Disease (COPD) patients hospitalized in intensive care unit for an acute exacerbation, and evaluate the impact of using non invasive mechanical ventilation (NIV)

Eligibility Criteria:
Inclusion Criteria:

* Patients with Chronic Obstructive Pulmonary Disease
* Age \> 18 years
* Hospitalized in Intensive care for an acute exacerbation
* Requiring Non invasive mechanical ventilation
* Able to breath spontaneously without non invasive ventilation more than 4h/day
* Without bulbar dysfunction

Exclusion Criteria:

* Hemodynamic instability
* Absence of consent
* Severe Hypoxemia
* pH \< 7,30
* No cooperation of the patient
A Trial of Daily Ultraviolet Therapy to Reduce Cardiovascular Risk Factors

The investigators have shown that a single dose of ultraviolet irradiation (as found in sunlight) will lower blood pressure for around one hour. They are now testing whether daily UVA for two weeks will produce a sustained fall in BP in patients with high blood pressure.

They will also measure the effect of daily UVA on other cardiovascular risk factors.

Epidemiological studies suggest that sunlight reduces all cause mortality, and particularly cardiovascular mortality.

The investigators have previously shown vasodilatation and a transient fall in blood pressure following irradiation of human volunteers with 2 standard erythemal doses of UVA radiation. This was independent of vitamin D and temperature rise and correlated with a nitric oxide synthase independent mobilisation of NO stores from the skin to the systemic circulation.

In this randomised, sham-controlled, cross-over double blind study, they will measure whether twice daily UVA administration can produce a sustained fall in BP and other cardiovascular risk factors in a cohort of pre-hypertensive patients.

Eligibility Criteria:
Inclusion Criteria:

* Pre-hypertensives (BP 120-139/80-89).
* Fitzpatrick skin types 2 and above (i.e. the ability to tan)

Exclusion Criteria:

* History of skin cancer.
* Fitzpatrick type 1 skin (always burns, never tans).
* Red hair.
* Family history of melanoma in first degree relative.
* Atypical naevus syndrome.
* Planned holiday or foreign travel during and for 4 weeks before the period of the study.
* Concurrent administration of:

  * anti-hypertensive medication,
  * photosensitising medication,
  * systemic immunosuppressive medication.
A Study of CRV431 Dosed Once Daily in NASH Induced F2 and F3 Subjects

This is a randomized, single-blind, placebo-controlled, once daily (QD) dose study of CRV431 in presumed NASH F2/F3 subjects.

This is a randomized, single-blind, placebo-controlled, once daily (QD) dose study of CRV431 in presumed NASH F2/F3 subjects. Study will evaluate the safety and tolerability of a once daily 75mg dose and 225mg of CRV431 compared to placebo over 28 days of dosing. Pharmacokinetic parameters of CRV431 and its major metabolites and fraction unbound will also be evaluated. Non-invasive antifibrotic bio-markers will be collected and quantified from presumed NASH F2/F3 subjects dosed with 75mg CRV431 or placebo.

Eligibility Criteria:
Key Inclusion Criteria:

* Male or female between 18 and 75 years of age (inclusive).
* Capable of giving written informed consent and able to effectively communicate with the investigator and study personnel.
* Presumed F2/F3 NASH to include: AST \>20 IU/L, Pro-C3 \>15.5 ng/mL, enhanced liver fibrosis (ELF) score \>9.8, and FibroScan \>8.5 kPa values.

Key Exclusion Criteria:

* Pregnant or breastfeeding or planning to become pregnant during the study period.
* Known allergy to CRV431, cyclosporine, or any of their inactive ingredients.
* Positive test for hepatitis B surface antigen (HBsAg), hepatitis C virus antibodies (HCVAb) or human immunodeficiency virus antibodies (HIVAb).
* Well documented causes of chronic liver disease according to standard diagnostic procedures to include any history or presence of decompensated cirrhosis.
* Subjects with a platelet count \<150,000/mL.
* Subjects with hemoglobin A1c(HbA1c) \>9.5%.
* Weight loss of more than 5% within 3 months prior to randomization.
* Subjects with a blood pressure to include a systolic pressure \>150 or a diastolic pressure \>90.
* At Screening, an estimated glomerular filtration rate (eGFR) \<60 mL/min/1.73 mL (calculated by the Chronic Kidney Disease Epidemiology Collaboration \[CKD-EPI\] method) and/or a Kidney Disease Improving Global Outcomes (KDIGO) category of \>G2.
* Subjects with a history of organ transplantation. Corneal transplantation will be allowed.
Assessment of EyeArt Performance With Retinal Imaging Devices

The study evaluates the performance of the EyeArt system for detecting diabetic retinopathy from images captured using multiple retinal imaging devices and operators.


Eligibility Criteria:
Inclusion Criteria:

* A diagnosis of diabetes mellitus;
* Understanding of study and provision of written informed consent; and
* 22 years of age or older.

Exclusion Criteria:

* Persistent visual impairment in one or both eyes;
* History of macular edema, severe non-proliferative retinopathy, proliferative retinopathy, or retinal vascular (vein or artery) occlusion;
* History of ocular injections, laser treatment of the retina, or intraocular surgery other than cataract surgery without complications;
* Subject is contraindicated for fundus photography (for example, has light sensitivity);
* Subject has contraindications for mydriatic medications or is unwilling or unable to dilate;
* Subject is currently enrolled in an interventional study of an investigational device or drug; or
* Subject has a condition or is in a situation which in the opinion of the Investigator, might confound study results, may interfere significantly with the subject's participation in the study, or may result in ungradable dilated fundus photographs.
Renal Hemodynamics, Energetics and Insulin Resistance: A Follow-up Study

The current protocol plans to enroll participants with youth-onset Type 2 Diabetes (T2D) as well as obese and lean controls from the Renal-HEIR - Renal Hemodynamics, Energetics and Insulin Resistance in Youth Onset Type 2 Diabetes Study (n=100) \[COMIRB #16-1752\] in a prospective investigation that seeks to 1) define the changes in kidney function by gold standard techniques and energetics by functional Magnetic Resonance Imaging (MRI) in adolescents with and without T2D as they transition to young adulthood; 2) quantify kidney oxidative metabolism by 11C-acetate Positron Emission Tomography (PET) in a subset of participants who are ≥18 years of age with youth-onset T2D and/or obesity; 3) determine peripheral arterial stiffness by SphygmoCor. Mechanistic insight will be provided by transcriptomic analyses of repeat biopsies 3-years after their initial biopsy for eligible participants with youth-onset T2D, as well as molecular analysis of tissue obtained from J-wire endovascular biopsies. This study will also leverage this well-characterized cohort of youths to define youth-onset T2D-related changes in brain morphology and function by structural MRI and resting-state functional MRI and through the assessment of cognitive function (fluid and crystallized intelligence) using the NIH Toolbox Cognitive Battery (NIHTB-CB), as an exploratory objective. All enrollees in Renal-HEIR have consented to be contacted for future research opportunities.


Eligibility Criteria:
Inclusion Criteria:

* Obese youth with and without Type 2 Diabetes + lean controls who were previously enrolled in the Renal HEIR Study
* Participants who will undergo a PET Scan: ≥ 18 years

Exclusion Criteria:

* Anemia
* Seafood, iodine, or penicillin allergy
* Pregnancy
* MRI Scanning contraindications (claustrophobia, implantable devices, \>550 lbs)
* History of bleeding or clotting disorders, thrombocytopenia, warfarin and/or heparin use
* Taking sulfonamides, procaine, thiazolsulfone or probenecid
A Study of MCI-196 in Chronic Kidney Disease Subjects on Dialysis With Hyperphosphatemia and Dyslipidaemia

This is a phase III multi-centre study in two periods: the first period is a phosphate binder and lipid lowering drugs washout for 8 weeks, the second period is a double-blind, randomised, parallel group, fixed dose, for 12 weeks.


Eligibility Criteria:
Inclusion Criteria:

* Male or female, 18 years of age or over
* Clinically stable haemodialysis or peritoneal dialysis
* Stable phosphate control
* On a stabilised phosphorus diet
* Female and of child-bearing potential have a negative serum pregnancy test
* Male subjects must agree to use appropriate contraception

Exclusion Criteria:

* Current clinically significant medical comorbidities, which may substantially compromise subject safety, or expose them to undue risk, or interfere significantly with study procedures and which, in the opinion of the Investigator, makes the subject unsuitable for inclusion in the study.
* A a serum albumin level\<30.0g/L
* A PTH level \>1000pg/mL
* A body mass index (BMI)\<= 16.0kg/㎡ or =\>40.0kg/㎡
* A serum LDL-C level \>4.94mmol/L(190mg/dL)
* A serum triglycerides level \>6.76mmol/L (600mg/dL)
* A History of significant gastrointestinal motility problems
* A positive test for HIV 1 and 2 antibodies
* A history of substance or alcohol abuse within the last year
* Seizure disorders
* A history of drug or other allergy
* A temporary catheter as a vascular access
* Participated in a clinical study with any experimental medication in the last 30 days or an experimental biological product within the last 90 days prior to signing of informed consent
Eisenmenger Quality Enhancement Research Initiative

Eisenmenger Quality Enhancement Research Initiative (QuERI) is a multi-center, observational, US-based longitudinal program, with enrollment of consecutive Eisenmenger patients who meet enrollment. Patient data will be collected prospectively for three years. Individual physician feedback will be provided on data collected with the purpose of improving the management of patients - quality enhancement research initiative (QuERI) process.

Approximately 200 male and female adult patients with a history of Eisenmenger will be recruited from approximately 50 cardiology practices over a period of 18 months and will be followed up every six months for the period of three years. Consecutive patients in each practice meeting inclusion and exclusion criteria should be considered for the study. Participating sites will be asked to maintain a screening log to identify which inclusion or exclusion criteria was not met thus excluding them from the study.

Eligibility Criteria:
Inclusion Criteria:

* Male and female patients
* Diagnosis of Eisenmenger syndrome based on right heart catheterization data
* Right to Left intra-cardiac shunting
* PAH
* Need for comprehensive management according to guidelines and peer-reviewed evidence
* Ability and desire to execute the consent for follow up

Exclusion Criteria:

* Poor mental function, drug, or substance (e.g.,alcohol) abuse, or unstable psychiatric illness, which, in the opinion of the investigator, may interfere with optimal participation in the study
* Prior inclusion in this program
Efficacy of Pioglitazone Hydrochloride and Metformin Hydrochloride Tablets on the Patients With Newly Diagnosed Type 2 Diabetes Mellitus Combined With Non-alcoholic Fatty Liver Disease

This is an multicenter, randomized, double-blind, parallel-controlled study to evaluated the efficacy of pioglitazone hydrochloride and metformin hydrochloride tablets on the patients with newly diagnosed type 2 diabetes mellitus combined with non-alcoholic fatty liver disease.

Taking metformin monotherapy as a control, we evaluated the efficacy of pioglitazone hydrochloride and metformin hydrochloride tablets on hepatic fat ultrasound and liver enzyme levels, and observed whether the drug can improve fatty liver in patients with newly diagnosed type 2 diabetes combined with non-alcoholic fatty liver disease. This is an multicenter, randomized, double-blind, parallel-controlled study.

Eligibility Criteria:
Inclusion Criteria:

1. To meet the new diagnosed type 2 diabetes patients, never received oral hypoglycemic drugs or insulin therapy;
2. In accordance with the nonalcoholic fatty liver (NAFLD) criteria for the diagnosis of patients.
3. the level of HbA1c was 7 -10.0%;
4. age 18-70, body mass index 21-35kg/m2;
5. the subjects informed consent and signed the informed consent.

Exclusion Criteria:

1. type 1 diabetes or secondary diabetes;
2. suffering from other liver diseases, such as hepatitis, self - free liver, etc.
3. abnormal thyroid function (in the active period), or the need for long-term oral and intravenous glucocorticoids to treat patients;
4. patients with severe renal dysfunction or renal disease (eGFR\<60);
5. in those with abnormal liver function, Alanine transaminase(ALT) or Aspartate transaminase(AST) was more than 3 times the normal upper limit.
6. people with serious gastrointestinal diseases such as peptic ulcers and chronic diarrhea;
7. patients with severe cardiopulmonary disease, cerebrovascular disease or stents;
8. hemopoietic system diseases such as serious primary diseases, hemoglobin \< 100g/L or need regular transfusion treatment;
9. pregnant, breast-feeding, women of childbearing age who are unwilling to contraception during the study period;
10. chronic cardiac insufficiency, the classification of heart function III level and above;
11. uncontrolled malignant tumor, and the history of bladder cancer.
12. acute complications of diabetes;
13. the use of other drugs for diabetes and liver disease;
14. patients who had participated in other clinical studies within three months;
15. people who have known allergies to this kind of drugs are known.
16. it is impossible to guarantee the effect or the safety judgment of the drug or the person who is unable to cooperate with the mental illness.
Impact of the Mandibular Advancement Device on Sleep Apnea During CPAP Withdrawal

Obstructive sleep apnea hypopnea syndrome (OSAS) is a frequent disease with neuropsychological and cardiovascular (CV) consequences. Continuous positive pressure (CPAP), the main treatment for OSAHS, is effective on the majority of symptoms but restrictive, which can promote non-compliance. Treatment interruptions are often observed in connection with intercurrent events such as nasal obstructions or even when patients are on the move. However, randomized trials have shown that stopping treatment, even for a short time, leads to a recurrence of symptoms and significant CV disturbances (increase in blood pressure, endothelial dysfunction, cardiac repolarization disorders). It seems important to consider strategies that promote therapeutic continuity. The mandibular advancement device (MAD) is an interesting tool in this regard. MAD is as effective as CPAP on symptoms and CV data. The investigators want to assess its effectiveness as a complementary treatment during treatment discontinuation on the main consequences of OSAHS.

Patients are recruited during sleep consultations in the Angers University Hospital pneumology department among patients followed for severe OSAHS and treated with CPAP. All of the scheduled examinations are carried out in the sleep laboratory of the CHU d'Angers.

Patients meeting the inclusion criteria and not having any non-inclusion criteria are randomized to the "MAD" group or to the "control" group. For patients in the "MAD" group, an appointment with a stomatologist is organized to make and adjust a thermo-molded type MAD.

An initial assessment is carried out for all patients during a day hospitalization specific to the study (study of endothelial function, blood pressure, Osler test, ECG, venous and urinary sampling, and completion of the study questionnaires).

Patients are then asked to stop CPAP treatment for two weeks. Patients in the OAM group use OAM during this period. Patients in the control group do not receive any specific treatment for their OSAHS during this period.

After 2 weeks of stopping CPAP, a second assessment, identical to the initial assessment, is carried out for all patients. This assessment is carried out during one night of hospitalization which also allows a PSG to be carried out under OAM or without treatment.

At the end of this assessment, the study is terminated and the patients resume their usual use of CPAP.

Eligibility Criteria:
Inclusion Criteria:

* Patient accepting MAD treatment
* Patient with an apnea-hypopnea index (AHI) greater than 30 events per hour on the initial recording
* Patient with excessive daytime sleepiness during initial treatment (ESE\>10)
* Patient treated for more than 12 months by CPAP with average compliance greater than 5 hours per night

Exclusion Criteria:

* Initial severe daytime sleepiness characterized
* OSAS with ≥ 5 central apneas per hour of sleep at baseline recording
* Previously diagnosed severe cardiac and/or respiratory pathology:
* Body mass index ≥ 35 kg/m2
* Known contraindication to OAM treatment
PRECISION GRX Post-Market Study - Japan

To collect data on the routine patterns of use, safety and effectiveness, including the clinical and technical performance of the CorPath GRX System, in the delivery and manipulation of coronary guidewires and stent/balloon catheters, and manipulation of guide catheters during PCI procedures in the first 231 patients treated with the CorPath device in Japan.

This is a single-arm, open-label, multi-center patient registry in Japan of the CorPath GRX System to examine its performance during PCI procedures and patient outcomes through 72 hours post-procedure or hospital discharge, whichever occurs first.

Eligibility Criteria:
Inclusion Criteria:

1. Age ≥ 20 years;
2. Patients with coronary artery disease with clinical indication for PCI;
3. Patient deemed appropriate for robotic-assisted PCI; and
4. The subject has been informed of the nature of the study, agrees to its provisions and has provided written informed consent, unless the EC has waived informed consent.

Exclusion Criteria:

1. Failure/inability/unwillingness to provide informed consent, unless the EC has waived informed consent; or
2. The Investigator determines that the patient or the coronary anatomy is not suitable for robotic-assisted PCI.