cyrilzakka/clinical-trials huggingface dataset:
Liver Biopsy Following Gene Therapy For Hemophilia

This observational study will obtain liver biopsy samples and evaluate the long-term effect of adeno-associated virus (AAV)-mediated gene therapy on the liver tissue in adult patients with hemophilia A or hemophilia B who have previously been treated with a factor VIII or factor IX gene-containing AAV-vector for liver-targeted gene transfer. Participants are from a cohort of patients treated with AAV-mediated gene transfer and at least 6 months after vector infusion.

To better understand the effect of AAV-mediated gene transfer on the liver, eligible participants will have a one-time, standard transjugular liver biopsy (TJLB) under moderate sedation. Or, no intervention if prior liver biopsy tissue is available.

Participants are from a cohort of patients treated with AAV-mediated gene transfer and at least 6 months after vector infusion expressing at least 1% of FVIII or FIX activity, respectively.

Evaluations will be done on the liver tissue samples regarding transduction frequency, morphology, gene expression patterns, vector genome integrations, epigenetic signature, and consequences of transgene expression on hepatocytes.

Eligibility Criteria:
Inclusion Criteria:

* Age ≥18 to 80 years
* Patients, who were enrolled and treated in one of the following clinical trials:

  * AGT4HB (EudraCT number: 2005-005711-17; NCT00979238) - FIX AAV gene therapy trial (sponsor: St. Jude Children's Research Hospital)
  * GO8 (EudraCT number:2016-000925; NCT03001830) - FVIII AAV gene therapy trial (sponsor: University College, London)
* Patients with endogenous expression of FVIII/FIX at \>1% after gene transfer that is stably maintained for more than six months after vector infusion
* Able to give informed consent
* Able to comply with study requirements

Exclusion Criteria:

* Any condition that, in the opinion of the investigator or sponsor of the ongoing clinical trial in which the patient is participating in, would prevent the patient from fully complying with the requirements of the clinical trial and/or would influence or interfere with evaluation and interpretation of subject safety or efficacy result of that ongoing clinical trial
* Platelet count \<100x10\^9/L
* INR \>1.5
* Abnormal kidney function with estimated GFR \<50 mL/min (calculated using the CKD-EPI equation)
* Known allergy to iodine-based intravenous contrast agents
* Known allergy to local or general anesthetics
* Known allergic reaction to FVIII/FIX concentrate infusions
* Presence of FVIII/FIX inhibitor
* Evidence of any bleeding disorder other than hemophilia A or B
Hürthle Cell Carcinoma and Follicular Thyroid Carcinoma

The goal of this retrospective study is to evaluate in patients with HCC and FTC who underwent lobectomy. The main questions it aims to answer are:

* The necessity of completion thyroidectomy based on the clinicopathological characteristics
* Prognosis Participants underwent thyroid lobectomy Researchers compared HCC and FTC groups to see the prognosis after lobectomy.

Background The prognosis of Hürthle cell carcinoma (HCC) and follicular thyroid carcinoma (FTC) is poorly understood. These types of cancers are usually confirmed after diagnostic lobectomy. We aimed to evaluate the clinicopathological characteristics and prognoses of patients with HCC and FTC who did not undergo completion thyroidectomy.

Materials and Methods We screened 221 patients diagnosed with FTC or HCC after thyroid lobectomy at Yonsei Severance Hospital between January 2000 and December 2020. After excluding patients who underwent complete thyroidectomy, 184 patients with FTC and 37 patients with HCC were included.

Eligibility Criteria:
Inclusion Criteria:

* Follicular thyroid carcinoma
* Hurthle cell carcinoma
* Lobectomy

Exclusion Criteria:

* Completion thyroidectomy
* Total thyroidectomy
Tumor Markers Study in Gastric Cancer for Cancer Immunotherapy (ONCO-RD 010 CRT)

Gastric cancer is one of the leading causes of cancer-related deaths worldwide. Despite advances in therapeutic intervention, the mortality from this disease remains high. As a result, most patients are now offered varying combinations of surgery, chemotherapy, and radiation therapy in order to derive benefits from a multidisciplinary approach. Unfortunately, therapies are often toxic or debilitating and therapeutic responses can vary. Thus new therapeutic strategy is required for the treatment of gastric cancer.

In the early 1980s, the discovery of cytolytic T cells (cytolytic T lymphocytes, CTLs) directed against an antigen presented on tumor cells was published. They can kill many invasive cells by recognizing the tumor specific antigens on the major histocompatibility complex (MHC) molecules. It is now generally agreed that although tumors express tumor antigens, they usually lack immunogenicity because of their inability to activate the immune response. The current view is that tumor cells are antigenic, but not immunogenic. If we can artificially activate the immune system against the tumor, it may be possible to eradicate the tumor. This approach is the basis of cancer immunotherapy.

Many tumor antigens have been defined in terms of multiple solid tumors: MART-1/Melan-A, gp100, carcinoembryonic antigen (CEA), HER-2, mucins (i.e., MUC-1), prostate-specific antigen (PSA), and prostatic acid phosphatase (PAP) are just a short list. Some immune-based therapies targeting these tumor antigens are in phase III trials assessing whether immunizing against these antigens affects overall survival. In gastric cancer, some tumor antigens such as MAGE-A3, NY-ESO-1, and WT-1 have been reported to be expressed in substantial proportion of cases. They have the potential to be the targeting molecules for immunotherapy in the future. In this study, we aim to explore the expression levels of the four tumor markers (MAGE-A3, NY-ESO-1, WT-1 and PRAME) in gastric cancers of Korean patients and to examine the correlations of the expression levels of the four markers to clinic-patholoical factors.


Eligibility Criteria:
Inclusion Criteria:

* gastric carcinoma
* received gastrectomy

Exclusion Criteria:

* neoadjuvant therapy
Improving Driving in Young People With Autism Spectrum Disorders

This study will examine the effects of treatment with the anti-anxiety medicine buspirone on driving performance (eye tracking) in individuals with high-functioning autism spectrum disorder (HF-ASD).

The study consists of an Assessment Visit at Massachusetts General Hospital (MGH), as well as two Driving Simulation visits that will take place at Massachusetts Institute of Technology (MIT). Subjects will be given buspirone and asked to take the medication for the two days preceding the Driving Simulation Visit.


Eligibility Criteria:
Inclusion Criteria:

* Males and females, ages 18-24, with a diagnosis of DSM-V Autism Spectrum Disorder
* Has a valid Driver's License

Exclusion Criteria:

* Major sensorimotor handicaps (e.g. deafness, blindness)
* Individuals who have never held a valid driver's license
* Intellectual Deficiency (Verbal Comprehension Index \< 80)
* Inadequate command of the English language
* Subjects with any clinically meaningful medical or psychiatric condition as determined by the investigator
* Individuals who are currently taking a monoamine oxidase inhibitor (MAOI) for any reason
* Pregnant
Impact of Modified Specific Carbohydrate Diet on Bronchial Asthma Control in Children

The aim of this study will explore if the specific carbohydrate diet has an effect on asthma control in children and if it will affect the frequency, severity, and duration of asthma and thus provide it as a potential complementary treatment option for them.

This study will be carried out on 30 children with moderate persistent asthma presented to the outpatient clinic of the Pulmonology Unit, Pediatric Department, Tanta University Hospital.

All children at the start of the study will be subjected to full history taking including, full nutritional assessment, laboratory tests, Pulmonary function tests, and Anthropometric measurements

Eligibility Criteria:
Inclusion Criteria:

* Asthmatic children with moderate persistent asthma according to the definition of National Heart, Lung, and Blood Institute guidelines, if they have daily symptoms, nighttime awakenings more than 1 time/week, but not nightly, daily use of short-acting beta-agonist for symptom control, some limitations of normal activities, and Forced Expiratory Volume in 1 second (FEV1)60-80%.
* Children aged from 6 to 18 years.
* The children are included only if parents and children are strongly motivated to try a dietary intervention (mSCD) as a complementary treatment.

Exclusion Criteria:

* Children with chronic lung disease, immunodeficiency, major thoracic deformities, neuromuscular, cardiovascular, digestive, rheumatic, osteoarticular, or genetic syndromes or any adverse health conditions that can affect nutritional status were excluded from the study.
* Children excluded if parents and children are not motivated to try a dietary intervention (mSCD) as a complementary treatment or who failed to have regular outpatient follow-up visits.
Evaluation of Objective Perimetry Using Chromatic Multifocal Pupillometer

Objective perimetry can better monitor visual field defects in retinal dystrophy and Glaucoma patients than conventional subjective perimetry. The PLR ( Pupil Light Reflex to short and long wavelength stimuli should be significantly lower compared to healthy participants in areas of visual field defects in retinal dystrophy and Glaucoma patients.

Pupil light reflex (PLR) will be measured by a chromatic multifocal pupillometer in response to short and long wavelength light with small spot stimulus in 76 points of the 30 degree visual field.

A computerized infrared video pupillometer will be used to record changes in pupil diameter in response to short- and long-wavelength stimuli (peak 485 nm and 620 nm, respectively) presented by 76 LEDs, 1.8mm spot size, at light intensities of 10-3000 cd/m2 and duration of 1-3 sec at different points of the 30 degree visual field

Eligibility Criteria:
Inclusion Criteria:

* Age 18-80 years old.
* Signing informed consent.
* Pupillary reflex to light.

Exclusion Criteria:

1. Cloudy corneas.
2. Surgical intraocular ophthalmic procedure within the past 30 days.
3. No reactive pupils.
4. Synechia of the iris to the lens after surgery or inflammation.
5. Neovascularization.
6. Axenfeld-Rieger Syndrome.
7. Iris atrophy (ICE syndrome).
8. Iris coloboma.
9. Sphincter damage due to ischemia.
10. Sphincter damage due to trauma (tears of sphincter or diffuse damage to muscle).
11. Sphincter damage due to Herpes Zoster Uveitis.
12. Sphincter damage due to high intraocular pressure.
13. Iris tumor or cyst.
14. Ectropion uvae.
15. Adie's pupil.
16. Third nerve aberrant regeneration of the iris sphincter.
17. RP patients with Optic neuropathy with the potential of producing a positive RAPD (Relative Afferent Pupillary Defect).
18. Chronic use of myotics or mydriatics.
19. Systemic medication that have affect on pupillary reflex .
20. Any condition preventing accurate measurement or examination of the pupil.
Impact of Obesity on the Efficacy of Endocrine Therapy With Aromatase Inhibitors

To see the impact of obesity on the efficacy of adjuvant endocrine therapy with aromatase inhibitors in postmenopausal patients with early breast cancer in terms of:

i) Locoregional recurrence ii) Distant metastases iii) Disease-free survival iv) Overall survival

The relationship between obesity and breast cancer is a complex one. Obesity is a risk factor for the development of breast cancer in postmenopausal women and has been linked to an increased risk of recurrence and decreased survival as compared to patients with normal weight.

The hypothesis that led to this study is that the amount of total-body aromatization capacity indicated by body mass index (BMI). In postmenopausal women and in premenopausal women with ovarian suppression, the major source of serum estrogens is the fat tissue, in which precursors are metabolized to estrogens by the enzyme aromatase. Thus, an increase in BMI leads to an increase in total-body aromatization and, consequently, an increase in oestrogen serum levels, which impact on breast cancer. Taken together, this suggests that BMI may serve as a useful surrogate parameter for total-body aromatization and eventually may be a practicable tool to tailor aromatase inhibitors (AIs) therapy for individual patients.

The study will include 360, postmenopausal patients with early breast cancer who have hormones receptor positive tumour as defined by the expression of oestrogen receptor (ER) and/or progesterone receptor (PR). Patients will be randomly assigned to receive tamoxifen 20 mg once daily or AIs (letrozole 2.5mg/ anastrozole 1mg/exemestane 25mg) once daily for five years. Patients with a tumour stage IB, IC, or II irrespective of nodal stage (\<10 positive nodes) will be included. Weight and height will be taken at baseline for calculation of BMI according to the WHO criteria. The frequency of adverse events will be used to assess safety throughout the study.

The primary end point will be disease-free survival (DFS). Secondary end points will be recurrence-free survival and overall survival (OS). The data will be analyzed for DFS and OS according to the BMI subgroups as well as two treatment arms (tamoxifen v AIs). The frequency of adverse events will be used to assess safety throughout the study.

Eligibility Criteria:
Inclusion Criteria:

* postmenopausal patients with breast cancer who have hormones receptor positive tumour as defined by the expression of oestrogen receptor (ER) and/or progesterone receptor (PR).
* patients with a tumour stage IB, IC, or II irrespective of nodal stage (\< 10 positive nodes)

Exclusion Criteria:

* premenopausal patients,
* ER/PR negative
Effect of Iron Status on Occurrence of Non-transferrin-bound Iron (NTBI) in Serum in Response to an Oral Iron Load

This study aims to investigate a possible effect of iron status on temporary build-up of non-transferrin bound iron (NTBI) in healthy volunteers upon iron supplementation.

For Visit 1, blood samples will be collected to assess iron status. For Visit 2, after an overnight fast, iron supplement will be provided to participant before breakfast. Blood samples will be collected right before and 2 h after iron supplement consumption for NTBI analysis.

NTBI concentration before and after iron supplement consumption will be compared. Dependence of NTBI formation on markers of iron status (serum iron, transferrin, transferrin saturation, serum ferritin and hemoglobin) will be evaluated.

Eligibility Criteria:
Inclusion Criteria:

* Apparently healthy volunteers (21 - 55 years old)
* C-reactive protein (CRP) \< 5

Exclusion Criteria:

* Blood donation or significant blood loss (e.g. surgery) within the past 4 months
* Regular intake of iron and other nutrient supplements within the past 4 months
* Regular intake of medication except oral contraceptives
* Acute or recent inflammatory or infectious symptoms
* Chronic gastrointestinal disorders or metabolic diseases
* Pregnancy or lactation
* Coeliac disease or gluten-related disorders
* Iron overload conditions (serum ferritin concentration \> 200 ng/mL for females, \> 300 ng/mL for males)
* Non iron deficiency anemia \[serum ferritin concentration within normal range (15-200 ng/mL for females, 30-300 ng/mL for males) but low hemoglobin (\< 12 g/dL for females, \< 14 g/dL for males)\]
The Acute Effects of Interesterification of Commercially Used Fats on Postprandial Fat Metabolism

The purpose of this study is to investigate whether there are differences in postprandial metabolic indices following interesterified fats used commercially versus the corresponding un-interesterified blend.

Aim: The current study aims to investigate the acute effects of commercially relevant interesterified 'hardstock' versus the corresponding un-interesterified blend on 8 h postprandial fat metabolism.

Hypothesis: Interesterification of a palm kernel and palm stearin fat blend, to produce a fat with a higher proportion of palmitic acid in the middle position of the triglyceride (TAG; but the same fatty acid composition), will alter postprandial lipid metabolism.

Subjects: Participants will include 24 healthy male and female (postmenopausal) volunteers aged between the ages of 45 and 75 years (since during this age metabolic changes start to take place).

Power calculation: Based on previous studies carried out by our group at King's College London, a sample size of 10 males and 10 females is required to detect a (clinically relevant) difference (for males and females separately) between means of 0.3 mmol/L maximal difference in 8 h plasma TAG concentration (standard deviation 0.33 mmol/L, significance level (alpha) of 0.05 and 80% power). 12 males and 12 females will be recruited to allow for a 20% drop out rate.

Expected value:The study will provide novel information on the acute effects of commercially relevant processed fats on postprandial lipaemia.

Eligibility Criteria:
Inclusion Criteria:

* Males and females aged 45-75 years
* Healthy (free of diagnosed diseases listed in exclusion criteria)
* Able to understand the information sheet and willing to comply with study protocol
* Able to give informed consent

Exclusion Criteria:

* Medical history of myocardial infarction, angina, thrombosis, stroke, cancer, liver or bowel disease or diabetes
* Body mass index \< 20 kg/m2 or \> 35 kg/m2
* Plasma cholesterol ≥7.5 mmol/L
* Plasma triacylglycerol \> 3 mmol/L
* Plasma glucose \> 7 mmol/L
* Blood pressure ≥140/90 mmHg
* Current use of antihypertensive or lipid lowering medications
* Premenopausal (for women)
* Alcohol intake exceeding a moderate intake (\> 28 units per week)
* Current cigarette smoker (or quit withint the last 6 months)
* ≥ 20% 10-year risk of CVD as calculated using a risk calculator
Study to Evaluate Real-world Pharmacoeconomics of Panitumumab in Metastatic Colorectal Cancer Patients

This is a phase IV multicenter trial to evaluate real-world health outcomes and economic impact of panitumumab versus standard-of-care (SOC) in the treatment of patients with chemotherapy-refractory metastatic colorectal cancer (mCRC).

The study will enable real-life health economics and outcome research (HEOR) to assess the impact of panitumumab in the Quebec population.

The primary objective is to evaluate real-world health outcomes and economic impact of panitumumab in the treatment of patients with chemotherapy-refractory mCRC in comparison with SOC. The secondary objectives are to confirm survival data, to assess the quality of life of patients and to assess the health care resource utilization of patients.

Patients with a mutated KRAS gene will be treated with standard-of-care (SOC) and patients with a non-mutated (wild type) KRAS gene will be treated with panitumumab.

During the course of the study, data will be collected on quality of life and work productivity. Patients will be asked to fill a set of questionnaires at their recruitment in the study and at every 3 months after treatment initiation.


Eligibility Criteria:
Inclusion Criteria:

* Patients with a histologically confirmed diagnosis of mCRC.
* Immunohistochemical evidence of EGFR expression.
* ECOG performance status of 0, 1 or 2.
* Patients refractory to fluoropyrimidine, oxaliplatin, and irinotecan chemotherapy regimens
* Patients with a wt KRAS scheduled to receive panitumumab as a single agent for the third-line treatment of mCRC or patients with a mt KRAS scheduled to receive SOC for third-line treatment of mCRC.
* Signed and dated IRB-approved informed consent document.
* Ability to read and understand English or French.
* 18 years of age or older.
Comparison of Intratechal Levobupivacaine and Adding With Sufetanil and Fentanyl in Ceserean Section

45 pregnant women undergoing cesarean section were enrolled in the study in november 2006 to march 2007. 2 ml of 0.5% levobupivacaine was added to 1 ml of saline in group I, 1 ml of 15 µcg of fentanyl in group II and 1 ml of 1,5 µcg sufentanil in group III by intratechal administration. Hemodynamic parameters, characteristics of sensory and motor blockade, peri-operative and postoperative visual analogue scale (VAS) pain scores, the time to the first analgesic requirement and adverse effects were recorded.

45 pregnant women undergoing cesarean section were enrolled in the study in november 2006 to march 2007. Using CSE technique, 2 ml of 0.5% levobupivacaine was added to 1 ml of saline in group I, 1 ml of 15 µcg of fentanyl in group II and 1 ml of 1,5 µcg sufentanil in group III by intratechal administration. Hemodynamic parameters, characteristics of sensory and motor blockade, peri-operative and postoperative visual analogue scale (VAS) pain scores, the time to the first analgesic requirement and adverse effects were recorded.

Eligibility Criteria:
Inclusion Criteria:

* pregnant women with a single live fetus aged 18-45 years old and classified as ASA I, undergoing elective cesarean section, were included the study in november 2006 to march 2007

Exclusion Criteria:

* Multiple pregnancies,
* preterm labor,
* eclampsia,
* preeclampsia,
* concomitant diabetes mellitus,
* allergy to local anesthetics, fentanyl or sufentanil,
* contraindication for regional anesthesia were the exclusion criteria.
Effectiveness of Intraoperative Cell Salvage in Aseptic Revision Total Hip Arthroplasty.

Intraoperative cell salvage is an important measure of patient blood management but its effectiveness in patients undergoing revision total hip arthroplasty remains unclear. Over the last decade, we have used intraoperative cell salvage systematically in this group of patients. However, since the use of cell salvage has a cost and requires additional resources, we decided to retrospectively investigate its usefulness in this particular indication.


Eligibility Criteria:
Inclusion Criteria:

* Eligible patients were adults undergoing elective aseptic revision of hip arthroplasty at the department of orthopedic surgery of the University Hospital of Liege between 01 January 2011 and 31 December 2020.

Exclusion Criteria:

* Patients undergoing revision for infection or with local malignancies were excluded from this study.