cyrilzakka/clinical-trials huggingface dataset:
In Vitro Models From Pediatric Brain Tumors

Tumors of the Central Nervous System (CNS) represent the leading cause of cancer-related deaths in children. Current treatment options are not curative for most malignant histologies, and intense preclinical and clinical research are necessary to develop more effective therapeutic interventions against these tumors, most of which meet the FDA definition for orphan diseases. The majority of malignant CNS tumors in children and adolescents belong to two broad histologic tumor entities: those of glial origin, such as high-grade glioma (HGG)and ependymoma (EPN), and those of neuronal origin, also identified as embryonal tumors, that include medulloblastoma and AT/RT(1). Over the last few years, whole-genome sequencing, gene-expression profiling and genome-wide methylation studies have greatly deepened our understanding of the biology and genetics of these tumors, allowing for robust stratification in clinically relevant molecular subgroups. The advancement of single-cell omics over the last decade have highlighted the enormous heterogeneity of tumors, a complex mixture of co-existing cancer subclones and supportive normal cell populations.

However, current treatments have remained largely static, and 5-year survival rate for children with malignant CNS tumors only achieves a modest 57.5%.

More effective treatment strategies should include novel chemotherapeutic agents that take into account high intrinsic tumor heterogeneity as well as the complex regulations of transcriptional and translational mechanisms that control protein expression. Identification of novel drugs and treatment strategies is further limited by the paucity of appropriate preclinical models, which mirror the molecular characteristics of distinct tumor subgroups.

We propose to establish patient-derived in vitro models to predict chemotherapeutic drug sensitivity/resistance in malignant pediatric CNS tumors. Next, we propose to perform molecular analyses in tissues of pediatric CNS tumors to determine whether in vitro findings have clinical correlates.


Eligibility Criteria:
Inclusion Criteria:

* Children and young adults who undergo standard surgical resection for suspected primary brain tumors or recurrent brain tumors
* signed written informed consent

Exclusion Criteria:

* no informed consent
A Phase 2 Study to Assess Efficacy and Safety of LB54640 in Patients With Genetic Obesity

The purpose of the study was to assess the efficacy of LB54640 in participants with rare genetic disorders of obesity

Patients with obesity due to POMC, PCSK1 or LEPR deficiency will be enrolled to receive LB54640 by oral administration for 16 weeks. After 16 weeks of treatment, the participant may enter a long term extension phase and receive treatment for up to 1 year.

Eligibility Criteria:
Inclusion Criteria:

* Participants aged ≥12 years at the time of enrollment and diagnosed with genetic obesity due to POMC, PCSK1, or LEPR deficiency mutations.
* Obesity is defined as BMI ≥30 kg/m2 for participants ≥18 years of age or BMI ≥95th percentile for age and gender for participants \<18 years of age.

Exclusion Criteria:

* Participants with recent (within 2 months, prior to Screening and up to enrollment) intensive diet/exercise regimen that resulted in \>2% weight loss, use of medication to treat obesity (within 3 months of first dose of LB54640 with certain exceptions), or weight loss surgery within 6 months prior to Screening or prior surgery which resulted in \>10% weight loss with no evidence of weight regain are excluded
* History of major surgical procedure
* Weight loss surgery within the previous 6 months
* Any history of a suicide attempt, or any suicidal behavior
* HbA1c \>10.9%
* Fasting glucose level \>270 mg/dL
Nudges on Hospitalist Behavior and Patient Satisfaction

Physician behavior plays an important role on patient perception of their care team and on patient satisfaction. Though studies have attempted to impact how physicians interact with their patients through various initiatives, the effect of more subtle nudges on physician behavior have not been studies. As such, the investigators propose a randomized controlled deception trial to evaluate the impact of nudges on hospitalist behavior and patient satisfaction.


Eligibility Criteria:
Inclusion Criteria:

* Hospitalist Physicians at Parkland Memorial Hospital who are willing to participate

Exclusion Criteria:

* None
Initial Local Anesthetic Dose With Continuous Interscalene Analgesia

Objective: To determine a minimally effective initial local anesthetic bolus required to provide satisfactory analgesia using continuous brachial plexus infusion following arthroscopic shoulder surgery using a double-blind, randomized, study comparing 3 initial doses.

Aims:

1. To compare pain ratings and supplemental analgesic requirements at discharge from PACU, 24 hours, and 48 hours and 12 weeks following 5, 10, and 20 ml boluses.
2. To compare adverse events including clinical dysphonia, Horner's syndrome, dyspnea, unexpected hospitalization, evidence of local anesthetic toxicity, and hand weakness at discharge from PACU following 5, 10, and 20 ml boluses.
3. To compare impairment in diaphragmatic excursion at discharge from PACU following 5, 10, and 20 ml boluses.
4. To compare patients' satisfaction with analgesia at 24 and 48 hours following 5, 10, and 20 ml boluses.
5. To compare patient rating of functional outcome at baseline and at 12 weeks following 5, 10, and 20 ml boluses.
6. To compare the rate of general anesthesia required following 5, 10, and 20 ml boluses.

Eligibility Criteria:
Inclusion Criteria:

* Provide written informed consent prior to participation in the study.
* Is scheduled to undergo Arthroscopic Rotator Cuff Repair, defined as arthroscopically assisted repair of any of the muscles or tendons comprising the rotator cuff.
* If subject is a female of childbearing potential, have a negative pregnancy test on the day of surgery per standard of care
* Be at least 18, but not more than 80 years of age
* Have an ASA risk class of I, II, or III according to the American Society of Anesthesiologists (ASA).
* Have the ability to read and understand the study procedures and the use of the pain scales. Must have the ability to communicate meaningfully with the Study Investigator and staff.
* Be free of other physical, mental, or medical conditions, which in the opinion of the Investigator, makes study participation inadvisable.

Exclusion Criteria:

* Subjects requiring daily opioids equivalent to greater than 40 mg oxycodone for over 2 weeks prior to the study.
* Has significant preexisting pulmonary (respiratory/breathing) disease, diaphragmatic paralysis or history of phrenic nerve injury (problems with the diaphragm causing breathing difficulties).
* Has significant medical disease(s), laboratory abnormalities or condition(s) that in the Investigator's judgment could compromise the subject's welfare, ability to communicate with the study staff, complete study activities, or would otherwise contraindicate study participation.
* Has known significant hypersensitivity to opioids, Ropivacaine, or the inactive ingredients (excipients) of the study medications.
* Has known or suspected history of alcohol or drug abuse or dependence within the previous 2 years.
* Has clinically significant liver disease, or other condition (e.g., alcoholism, cirrhosis, or hepatitis) based on medical history and examination.
* Has participated in another clinical Study (investigational or marketed product within 30 days of surgery.
A Dose-Finding, Efficacy and Safety Study of YY-351 in Patients With Type 2 Diabetes

The purpose of this trial is to determine the dosage selected and to evaluate the efficacy and safety of YY-351.

Ginseng has been widely studied for treatment of diabetes, dyslipidemia and obesity. Interestingly, in addition to ginseng root, ginseng berry and leaf were also shown to reduce blood glucose in diabetic models. In our recent study, ginsam, vinegar extraction from Panax ginseng, which is enriched in the ginsenoside Rg3, has distinct beneficial effects on glucose metabolism and body weight control in an obese animal model of insulin resistance by changing the expression of genes involved in glucose and fatty acid metabolism. Our group has also published that Rg3 improves insulin signaling and glucose uptake primarily by stimulating the expression of IRS-1 and GLUT4. Thus, we have evaluated the efficacy, dose-response relationships and safety of a ginsam, a vinegar extract from Panax ginseng.

Eligibility Criteria:
Inclusion Criteria:

* Previous diagnosis of Type 2 diabetes(more than 3months)
* Patients aged over 18 years
* FPG levels in the range : 126 - 270mg/dL or HbA1c : 7.0 - 12.0%

Exclusion Criteria:

* Pregnant women, Breast feeding, or actively trying to be come pregnant
* Patients with Type 1 DM, gestational diabetes or secondary diabetes
* FPG levels in the range : ≥ 270mg/dL HbA1c : \< 7.0, \>12.0%
* Patient who take the medicine which may affect to blood sugar control (i.e.systemic glucocorticoid)
* Patients with diabetic complications or the history of a case that would affect to efficacy and safety evaluation (i.e. Thyroid disorder, Cushing's Syndrome, Multiple ovarian cystoma, pheochromocytoma)
* Patients with Chronic hepatitis, hepatitis B, C (except healthy HBV carrier) or Liver diseases (AST or/and ALT \>2 × ULN(upper limit normal))
* Patients with Kidney disorder (Cr\>2.0)
To Assess the Level of Adherence of Subjects Receiving SAIZEN® Via Easypod™ (ECOS Taiwan, Indonesia, Singapore)

This is an Multinational, Multicenter, Observational Study to evaluate Adherence and Long Term Outcomes of Therapy in pediatric subjects using easypod™ electromechanical device for growth hormone treatment and to assess the level of adherence of subject receiving SAIZEN® via easypod™.

Subjects will be enrolled in a multicenter longitudinal observational study. Parents/subjects will provide their Informed Consent/assent to upload their data for population-based analyses and, ultimately, clinical outcomes. Adherence data will be primarily derived from the easypod™ device combined with physician data entry of outcome measures. Data will be collected both retrospectively and prospectively. This will allow the establishment of adherence profiles and explore the hypothesis that patient adherence support programs improve adherence and subsequent clinical outcomes. Collected data will be also analyzed in a multinational pooled analysis of comparable national studies.

Primary Objective:

• To assess the level of adherence of subjects receiving SAIZEN® via easypod™

Secondary Objectives:

* To describe the impact of adherence on clinical outcomes for subject receiving SAIZEN® via easypod™
* To identify adherence subject profiling
* To assess the impact of adherence on Insulin-like Growth Factor 1 (IGF1) (that is, above, below or within normal ranges)

Eligibility Criteria:
Inclusion Criteria:

* Administered growth hormone via the easypod™ electromechanical device according to Summary of Product Characteristic (SmPC)
* Over the age of 2 years
* Under 18 years of age, or over 18 without fusion of growth plates
* Parent's or guardian's written informed consent, given before entering data into the observational study, with the understanding that the subject or parent/guardian may withdraw consent at any time without prejudice to future medical care. If the child is old enough to read and write, a separate assent form will be given as defined in the appropriate jurisdiction of each country

Exclusion Criteria:

* Subjects taking growth hormone in whom growth plates have fused (that is, for taking growth hormone for it's metabolic effects)
* Contra-indications to SAIZEN® as defined in the SmPC
* Use of an investigational drug or participation in another interventional clinical study
Quality-Of-Life Assessment in Patients With Head and Neck Cancer Treated With Radiation Therapy

RATIONALE: Gathering information over time about patients' quality-of-life and satisfaction with care may help doctors plan the best treatment and help patients live more comfortably.

PURPOSE: This randomized clinical trial is studying quality-of-life assessment in patients with head and neck cancer treated with radiation therapy.

OBJECTIVES:

Primary

* To evaluate the impact of quality-of-life assessment in routine oncology practice on satisfaction with care, health-related quality-of-life, and toxicity in patients with primary nonmetastatic head and neck cancer treated with radiotherapy.

Secondary

* To evaluate the feasibility of using quality-of-life tools in routine oncology practice.
* To evaluate the impact on the occurrence of toxicity.
* To determine the impact on overall survival.
* To study the concordance between the toxicity reported by the clinician and those reported by the patient quality-of-life questionnaires.

OUTLINE: Patients are stratified according to sex, tumor localization, and TNM stage. Patients are randomized to 1 of 2 arms.

* Arm I: Patients complete quality-of-life questionnaires (EORTC tools only, including EORTC QLQ-SAT32, EORTC QLQ-C30, and specific module QLQ-H\&N35/Euroqol EQ-5D) before each consultation with the clinician for one year.
* Arm II: Patients undergo standard follow-up care comprising consultation with the clinician for one year.

After completion of study, patients are followed up every 3 months for 1 year and then at 2 years.

Eligibility Criteria:
DISEASE CHARACTERISTICS:

* Diagnosis of primary nonmetastatic head and neck cancer
* No other primary cancer localizations
* No second tumor localization at the time of diagnosis
* Must be planning to be treated initially with radiotherapy

PATIENT CHARACTERISTICS:

* No cognitive impairment or psychiatric history
* No history of other cancer

PRIOR CONCURRENT THERAPY:

* No prior chemotherapy or radiotherapy
Development of Normative Ultrasound Databases of Tongue Surface Configuration

Our past ultrasound research has indicated a need for baseline normative tongue data against which to compare our disordered speech populations. Subjects with normal speech and hearing who represent different ages and sexes, will be studied with respect to tongue shape and movement patterns. Recently developed parameters such as curve fitting, curve averaging, and polar coordinates will be used to create baseline data categories. In addition, new parameters and improved instruments will be developed as previously to produce a data base which can be used across disciplines.

Our past ultrasound research has indicated a need for baseline normative tongue data against which to compare our disordered speech populations. Subjects with normal speech and hearing who represent different ages and sexes, will be studied with respect to tongue shape and movement patterns. Recently developed parameters such as curve fitting, curve averaging, and polar coordinates will be used to create baseline data categories. In addition, new parameters and improved instruments will be developed as previously to produce a data base which can be used across disciplines.

Eligibility Criteria:
Healthy normal subjects.

No reconstructive tongue surgery.

No radiation for head and neck malignancy.
Changes in Physical Activity Status: Comparison of Older Adults With Versus Without a History of Covid-19 Infection

The negative impact of the Covid-19 pandemic on the physical activity behavior of overall adults has been reported. But much less is known in older adults. Alterations in physical activity among Covid-19 survivors have not been investigated so far. The present study aimed to evaluate the physical activity status of older adults in terms of a history of Covid-19 infection.


Eligibility Criteria:
Inclusion Criteria:

* 65 years non-dementia community dwelling older adults

Exclusion Criteria:

* The exclusion criteria were: (1) intensive care unit admission due to Covid-19 infection, (2) neurodegenerative or psychotic disease, (3) delirium, (4) terminal illness (malignancy, advanced organ failure), and (5) hearing impairment that disrupts conducting the telephone survey
Differential Intervention Trial by Standard Therapy Versus Pitavastatin in Patients With Chronic Hemodialysis (DIALYSIS)

The purpose of this study is to verify whether pitavastatin prevents from cardiovascular events and improves the mortality in chronic hemodialysis patients with hypercholesteremia.

It was already proved by the clinical trials that statins prevent from death and cardiovascular events. However, the efficacy of statins in patients with chronic hemodialysis has not been proved yet.

Eligibility Criteria:
Inclusion Criteria:

* patients under hemodialysis
* patients with hypercholesterolemia as defined by any of following parameters:

  * LDL-C ≧ 100 mg / dL
  * TC ≧ 180 mg / dL
  * patients required cholesterol-lowering treatment by investigators.
* patients aged 20-75 years
* patients with written consent by their own volition after being provided sufficient explanation for the participation into this clinical trial

Exclusion Criteria:

* patients taking statins or fibrates
* patients enrolled to the other trials using contraindication drugs of pitavastatin
* patients who had acute myocardial infarction within six months before the day of the agreement acquisition
* patients scheduled PCI and CABG within six months after the day of the agreement acquisition
* Patients who had diagnosis or doubt of malignant tumor
* patients corresponded to "Contraindications" of pitavastatin
* Familial hypercholesterolemia patients
* patients judged ineligible by investigators
An Organ Preservation Strategies After Chemoradiotherapy Combined With Immunotherapy for Esophageal Cancer (PALACE3).

Patients with locally advanced esophageal squamous cell carcinoma will randomly assigned to receive neoadjuvant chemo-radiotherapy combined with immunotherapy post organ preservation strategy (experimental group) or neoadjuvant chemo-radiotherapy followed by surgery (control group).

The 3-year overall survival rate is the primary outcome.

The investigators will conduct the open, multicenter, prospective, randomized controlled clinical study(PALACE3). Patients with locally advanced esophageal squamous cell carcinoma will randomly assigned to either receive neoadjuvant synchronous radiotherapy and chemotherapy combined with immunotherapy followed by organ preservation strategy (experimental group, Arm1) or neoadjuvant concurrent radiotherapy and chemotherapy followed by radical surgery (control group,Arm 2). Collect relevant data on preoperative treatment, re examination after neoadjuvant therapy, perioperative and long-term follow-up of patients, and evaluate the clinical treatment effects (cCR rate), surgical pathological results (pCR rate, R0 resection rate, tumor regression grade, lymph node positivity rate), neoadjuvant therapy and perioperative complications, long-term oncological effects (total survival, disease-free survival), and quality of life of the two treatment plans through statistical analysis.

Eligibility Criteria:
Inclusion Criteria:

* A patient will be eligible for inclusion in this study only if ALL of the following criteria apply:

  * Histologically confirmed cT2-T4a,N0-N+,M0 resectable esophageal squamous cell carcinoma.
  * Eastern Cooperative Oncology Group (ECOG) performance status 0-1
  * Patients approve and sign the informed consent

Exclusion Criteria:

* Patients with active autoimmune disease or history of autoimmune disease.
* Patients who have a condition requiring systemic treatment with either corticosteroids or other immunosuppressive medications.
* Subjects with a history of symptomatic interstitial lung disease.
* History of allergy to study drug components.
* Women must not be pregnant or breast-feeding.
* Men with female partners (WOCBP) that are not willing to use contraception.
* Patient has received prior chemotherapy, radiotherapy, target therapy and immune therapy for this malignancy or for any other past malignancy.
* medical conditions that, in the Investigator's opinion, will make the administration of study drug hazardous or obscure the interpretation of toxicity or adverse events
Gut Bacteria and Brain of the Baby

The project is a prospective French population based cohort. A hundred moderate preterms born at GA30-326/7 will be enrolled for a longitudinal follow-up up to 2 years of corrected age. Eligible neonates will be those born at 30-326/7 weeks of gestation admitted to the neonatal intensive care unit of Rennes Hospital, including transferred infants during the first day of life. The infants will be followed up to the corrected age of 2 years by pediatricians.


Eligibility Criteria:
Inclusion Criteria:

* Preterms born at gestational age of 30-326/7 weeks of amenorrhea
* Preterms born in Rennes hospital or preterms born in another hospital but moved to Rennes hospital during the first 24 h of postnatal life
* Parents or holder of the parental authority who give in writing their free, prior and informed consent for recruitment of their baby who has a social security system
* Mother of the baby who gives in writing her free, prior and informed consent to be involved in the present study
* Mother of the baby who has a social security system

Exclusion Criteria:

* Preterms born with a genetic disease
* Preterms born with a digestive defect
* Uncompetent parents under the protection of guardianship or tutelage services
Goal Attainment and Physical Activity in People With Hemophilia A

This is a Phase 4, multi-center, observational study conducted in patients aged 12 to 50 years with moderate or severe hemophilia A who are newly starting prophylaxis with efa in the US and Japan.

This study aims to enroll 35 patients.


Eligibility Criteria:
Inclusion Criteria:

* At enrollment, newly starting prophylaxis therapy with efanesoctocog alfa according to usual clinical practice, with efanesoctocog alfa initiated within 6 months after the enrollment visit
* Diagnosis of moderate (endogenous FVIII activity between 1% to 5% of normal) or severe (endogenous FVIII activity \<1% of normal) hemophilia A
* Aged 12 to 50 years at time of enrollment, inclusive
* Access to a smartphone device (Android version 12.0 or higher; or iOS 13 or higher) with Bluetooth 4.0 (minimum) or 5.0 (recommended) capabilities for compatibility with physical activity tracker
* Availability of home-based access to internet for electronic patient-reported outcome (ePRO)/diary assessments
* Willingness to utilize the activity tracking device

Exclusion Criteria:

* Current diagnosis of a FVIII inhibitor, defined as inhibitor titer ≥ 0.60 BU/mL
* Use of efanesoctocog alfa for prophylaxis in the 6 months prior to enrollment

NOTE: Other Inclusion/Exclusion criteria may apply. The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.