cyrilzakka/clinical-trials huggingface dataset:
Growth Hormone Treatment for the Prevention of Short Stature in Young Girls With Turner Syndrome Before the Age of 4 Years

The objective of this study is to evaluate the efficacy and safety of recombinant human growth hormone (r-hGH) treatment in girls with Turner Syndrome under the age of 4 years. After 4 years of treatment, height in these girls will be compared with an historical control group of untreated girls with Turner Syndrome, matched for age and height at baseline.


Eligibility Criteria:
Inclusion Criteria:

* Young girls with turner syndrome proved by karyotype
* Growth hormone secretion confirmed with ornithin stimulation test
* Normal glucidic metabolism confirmed by assessment of HbA1c
* None associated severe pathology which could have impact on growth (i.e. renal insufficiency, decompensated heart failure)
* No previous or associated treatment with anabolic or sexual steroids
* Known parental height

Exclusion Criteria:

* Severe associated pathology with impact on growth
* Concomitant treatment with impact on growth
* Previous or associated treatment with anabolic steroids
* Associated growth hormone deficiency
Safety, Efficacy, and Patient Preference of Split-Dose Crystalline Lactulose as a Preparation for Colonoscopy in Adults

The purpose of this study is to determine the efficacy of a split-dose regimen of crystalline lactulose for cleansing of the colon as a preparation for colonoscopy, as assessed by the physician's determination of the cleanliness of the colon using the Boston Bowel Prep Scale (BBPS).

This is a single center, open-label, pilot study to determine the safety, efficacy, and patient preference of a split-dose regimen of Crystalline Lactulose for cleansing of the colon prior to colonoscopy. Safety will be assessed by the occurrence of any treatment emergent adverse events. Efficacy will be determined by the endoscopist's rating of the cleanliness of the colon and the incidence of treatment failure (insufficient evacuation of the bowel). Patient preference will be determined by the patient's responses on a patient questionnaire.

Eligibility Criteria:
Inclusion Criteria:

* Patients requiring bowel evacuation for colonoscopy

Exclusion Criteria:

* Patients with galactosemia (galactose-sensitive diet)
* Patients known to be hypersensitive to any of the components of Crystalline Lactulose
* Patients with a history of a failed bowel preparation
* Patients with severe constipation, defined as those patients taking daily prescription or over-the-counter laxatives
* Patients with possible bowel obstruction, previous colonic surgery, gastric retention, bowel perforation, toxic colitis, toxic megacolon, or ileus
* Patients on lactulose therapy or receiving any treatment for chronic constipation
* Patients less than 18 years of age
* Inability to understand the requirements of the study or be unwilling to provide written informed consent (as evidenced by signature on an informed consent document approved by an Institutional Review Board \[IRB\]) and agree to abide by the study restrictions
* Be pregnant or nursing
* Be otherwise unsuitable for the study, in the opinion of the Investigator
Clinical Trial to Evaluate the Long-term Safety of Udenafil in Patient With PAH

This study evaluates the long-term safety of Udenafil in Patient with Pulmonary Arterial Hypertension(PAH). All the patients will receive Udenafil for 1-year(48-weeks).

Pulmonary arterial Hypertension is characterized by a progressive incerease in pulmonary vascular resistance(PVR) leading to right ventricualar failure.

Udenafil inhibits PDE 5, an enzyme that metabolizes cGMP, enhancing the cGMP mediated relaxation and growth inhibition of vascular smooth muscle cells, including those in the lung.

Eligibility Criteria:
Inclusion Criteria:

* Patients who completed the initial study(DA8159_PAH_II), 12-week, double-blind, placebo-controlled trial

Exclusion Criteria:

-
Everolimus in Breast Cancer Patients After Pre-operative Chemotherapy

This is a randomized controlled pilot study investigating signals involved in drug resistance to chemotherapy.

Patient will be randomized to undergo observation or to receive Everolimus after completion of neoadjvuant treatment (anthracycline and/or taxane-based) and before surgery. The patients will be monitored for efficacy and safety.


Eligibility Criteria:
Inclusion Criteria:

* Women aged ≥ 18 years;
* Histologically and/or cytologically confirmed invasive ductal or lobular breast cancer;
* Tumor of 3 cm or greater at time of diagnosis
* Measurable primary tumor after neoadjuvant treatment before randomization
* No prior chemotherapy for breast cancer;
* ECOG performance status ≤1 or Karnofsky performace status ≥ 70%
* Adequate liver/renal function
* Able to swallow whole tablets.
* Able to give written informed consent
* Able to follow prescription instructions reasonably well

Exclusion Criteria:

* Male patient
* Prior history of other malignancy within 5 years of study entry, aside from contralateral breast carcinoma, appropriately treated cervical carcinoma in situ, or adequately treated basal or squamous cell carcinoma of the skin
* Distant metastasis, including skin involvement beyond the breast area
* Other severe acute or chronic medical or psychiatric condition, or laboratory abnormality that would impart, in the judgment of the investigator, excess risk associated with study participation or study drug administration, or which, in the judgment of the investigator, would make the patient inappropriate for entry into this study
Prehabilitation for Kidney Transplant Candidates

This interventional trial seeks to determine the feasibility of wearable sensors to provide data from patients while undergoing supervised exercise.

This pilot study is a prospective, non-randomized, interventional trial of measures of prehabilitation in kidney transplant candidates who are frail. Kidney transplant candidates who meet criteria for the study will have wearable sensors placed for monitoring during the exercise session of their prehabilitation. The sensors will capture data during the single exercise session and transfer the data to a mobile health (mHealth) platform. The study aims to use the information from the sensors to develop future studies where the exercise is adjusted based on the sensor reports.

Eligibility Criteria:
Inclusion Criteria:

1. Adult Frail patients who are being evaluated for kidney transplant at Houston Methodist Hospital
2. Male or Female, aged 55 or older
3. Provision of signed and dated informed consent by the participant
4. Willingness to comply with study procedures and the duration of the study

Exclusion Criteria:

1. Inability or contraindication to exercise
2. Refusal to participate
3. Inability to consent or effectively communicate
Early Feasibility Study of Transcutaneous Upper Airway Stimulation in Obstructive Sleep Apnea

Early feasibility study to estimate the functionality of a novel intervention based on non-invasive transcutaneous electrical stimulation of the hypoglossal nerve to reduce the Apnea Hypopnea Index (AHI).


Eligibility Criteria:
Inclusion Criteria:

* Provision of signed and dated informed consent form
* Stated willingness to comply with all study procedures and availability for the duration of the study
* Male or female, aged 18 years or older
* In good general health, as evidenced by medical history and diagnosed with moderate or severe OSA
* In-lab PSG, within the last 6 months, with an AHI of 15-50/hour. In addition, \<25% of the respiratory events on the diagnostic PSG can be attributed to central apneas
* BMI less than or equal to 32

Exclusion Criteria:

* No or mild OSA (AHI \<15/h) or very severe OSA (AHI \>50/hour), exclusively postural sleep apnea, or isolated Rapid-Eye-Movement (REM) sleep associated OSA.
* Patients who are cachectic (BMI \<18.5 kg/m2) or obese (BMI \>32 kg/m2).
* Patients should not have enlarged tonsils (size 3-4) and/or adenoids, nasal polyps, neuromuscular disease, hypoglossal nerve palsy, abnormal pulmonary function tests, severe pulmonary hypertension, valvular heart disease, heart failure (New York Heart Association, NYHA III-IV), recent myocardial infarction, significant cardiac arrhythmias, atrial fibrillation, stroke, opioid usage, uncontrolled hypertension, known allergic reaction to adhesives or latex, active psychiatric disease, co-existing non-respiratory sleep disorder, pregnancy or significant metal implants or cardiac/other pacemakers.
* Patients with facial hair that affects the correct placement of the electrodes, if they are unwilling to shave.
Duloxetine - Warfarin Pharmacodynamic Study

To evaluate the blood clotting effects of multiple doses of warfarin at steady state when taken at the same time as multiple doses of duloxetine as measured by changes in the International Normalised Ratio. All subjects will begin dosing with warfarin, then after approximately 12 days, duloxetine 60 mg is added and subjects with stabilized INR will either continue dosing at 60 mg or have a dose increase to 120 mg.


Eligibility Criteria:
Inclusion Criteria:

Healthy men or women between ages 18 and 64 years.

Exclusion Criteria:

Have a personal history, family history of, or current evidence of: a bleeding disorder, have Positive Faecal Occult Blood (FOB) sample at screening, significant neuropsychiatric disease (including a history of suicide attempts or who have exhibited suicidal ideation or who are at significant risk to commit suicide, as judged by the investigator), significant active respiratory, hepatic, renal, gastrointestinal, endocrine, hematological, or neurological disorders.

Use or intended use of aspirin or NSAIDs within 2 weeks prior to first dosing.

Subjects who have an average weekly alcohol intake that exceeds 28 units per week (males) and 21 units per week (females). Subjects who smoke more than 5 cigarettes per day.

Use or intended us of a drug that inhibits or induces CYP1A2 or inhibits CYP2D6 within 2 weeks prior to first dosing occasion or during the study.

Have received any drug that acts as a monoamine oxidase inhibitor (MAOI) within 2 weeks prior to first dosing occasion or have a potential need to use an MAOI during the conduct of this study or within 2 weeks after discontinuation of study drug.
Glucagon-like Peptide 2 - a Glucose Dependent Glucagonotropic Hormone?

Infusion of glucagon-like peptide 2 (GLP-2) during euglycaemia, hypoglycaemia and hyperglycaemia and evaluation of the effect on glucagon secretion in healthy subjects.

This is a cross-over, randomized, double-blinded study in which 10 participants will undergo 6 experimental days each. The participants will have their plasma glucose clamped at either euglycaemia (fasting level), hypoglycemia (2,5 mmol/l) or hyperglycemia (10 mmol/l) during infusion of GLP-2 or saline (placebo)

The six days will be as follows:

* Euglycaemia + Placebo
* Euglycaemia + GLP-2
* Hyperglycaemia + Placebo
* Hyperglycaemia + GLP-2
* Hypoglycaemia + Placebo
* Hypoglycaemia + GLP-2

The primary endpoint will be levels of plasma glucagon. Secondary endpoint will be levels of GLP-1, GLP-2, Insulin/C-peptide, OXM, Amino acids, CCK, Gastrin, norepinephrine, bile acids, FGF-19/FGF-21. Secondary endpoints will also include ultrasound scan of the gallbladder.

Eligibility Criteria:
Inclusion Criteria:

* Caucasian ethnicity
* Body mass index (BMI): 18.5-27 kg/m2
* Glycated haemoglobin (HbA1c) \< 42 mmol/mol
* Normal haemoglobin (8.3-10.5 mmol/l)
* Informed and oral and written consent

Exclusion Criteria:

* Prediabetes, type 1 diabetes or type 2 diabetes or first-degree relatives with type 1 or type 2 diabetes
* Nephropathy (eGFR \< 60 and/or albuminuria)
* Known liver disease and/or alanine transaminase (ALAT) or aspartate transaminase (ASAT) \> 2 × upper normal limit
* Bilirubin \> 25 μmol/l
* Known intestinal disease or previous surgery of the intestines
* Active or recent malignant disease
* Treatment with medicine that cannot be paused for 12 hours
* Any condition considered incompatible with participation by the investigators
Same Day Ambulatory Appendectomy (SAMBA)

The potential benefit of outpatient care for this common digestive emergency is considerable, both for the patients themselves and for the public health system:

1. Optimization of the care pathway, reducing the length of stay in hospital (a major issue in the context of the COVID-19 (coronavirus disease) pandemic) liberating patient beds and staff, and reducing the risk of nosocomial exposure.
2. Improved patient satisfaction compared to waiting for hours in the emergency department due to lack of hospital beds.
3. Non-inferiority of care in an outpatient unit in terms of quality and safety in day hospitalization.
4. Significant decrease in the overall cost of this pathology as a result of a reduction in the hospital stay.


Eligibility Criteria:
Inclusion Criteria:

* Patients aged 15-74 years
* BMI ≤ 30 kg/m2
* Uncomplicated acute appendicitis confirmed by imaging (ultrasound and/or CT and/or MRI)

  * Temperature ≤ 38,1°C and \> 35,5°C
  * Appendix diameter \> 6mm and ≤ 15mm
  * Without effusion or with only localized peri-appendicular effusion
  * Infiltration of peri-appendicular fat without abscess or plastron
  * No sign of perforation
  * Leukocytes ≤ 15,000G/L AND
  * CRP (C reactive protein) ≤ 50mg/L
* If pain, calmed by level 2 analgesic at maximum
* Ambulatory criteria

  * Availability of monitoring by a relative during the 12 hours after discharge from the hospital
  * Residence located less than 20 minutes by car from a health center (hospital or clinic)
  * Access to a telephone mobile or fixed in case of problems
* Signature of the written informed consent form by the patient
* If the patient is a minor, signature of the written informed consent form by both parents or their legal representative
* Affiliation to a French health insurance scheme or equivalent

Exclusion Criteria:

* Criteria that exclude ambulatory care such as an ASA score (Physical status score) \> 2, severe or uncontrolled comorbidities, severe pulmonary disease including obstructive sleep apnea, anticoagulation or antiplatelet drug or contraindication to ambulatory surgery such as intubation difficulties
* Presence of active cancer, a malignant hemopathy, drug addiction, coagulopathy, immunosuppressive treatment
* Non-acute or interval appendectomy, i.e. after antibiotic treatment of a complicated appendicitis of the plastron or drainage of an appendicular abscess;
* History of pelvic surgery
* Vulnerable people: pregnant or breast-feeding women (patients will undergo a pregnancy test: plasmatic β-hCG (human chorionic gonadotropin) or urinary test), adult under guardianship or deprived of freedom. Pregnant women are considered to have a full stomach, with risk of inhalation at anesthetic induction and represent a contraindication to ambulatory surgery. In addition, the need to perform abdominal surgery on a pregnant woman requires obstetric monitoring that is difficult to reconcile with management in an outpatient surgery unit (need for obstetric ultrasound or monitoring).
* Suspicion of a tumor of the appendix : Mucocele and pseudomyxoma, Carcinoid tumor, Adenocarcinoma of the appendix, Another type of tumor
International Validation of a Delirium Prediction Model for ICU Patients

Recently our ICU research group developed and validated a delirium prediction model in the NetherlandsThis. This model has a high predictive value. We want to examine what the predictive value is in ICUs of other European countries.

Delirium is a serious and common disorder in ICU patients. It is increasingly recognized that screening of ICU patients is a valuable tool for early detection and treatment of delirium, which may reduce its incidence, severity and duration. Apart from treatment of delirium, delirium prevention (pharmacological and nursing interventions)is an important issue. However, it is time consuming to take preventive measures in all ICU patients. The recently developed prediction model facilitates the conduct of preventive measures focused on high risk groups. This prediction model is developed and validated in the Netherlands. The aim of our study is to investigate the predictive value of the model in ICUs in other countries.

Eligibility Criteria:
Inclusion Criteria:

* patients 18 years or older

Exclusion Criteria:

* delirious state within the first 24hours after ICU admission
* sustained comatose during complete ICU stay
* admission duration less than twelve hours;
* serious auditory or visual disorders
* unable to understand the language of the included centre
* severely mentally disabled
* serious receptive aphasia
* or if there are missing or incomplete delirium screenings during admission to the ICU
Breastfeeding Success and Time of First Meconium Passage

To investigate the relationship between breastfeeding success and the time of first meconium passage in term newborns.

The timing of the first meconium passage in neonates is a useful indicator of distal colon function. Delayed passage may signal congenital distal bowel dysfunctions such as hirschsprung disease (HD), anorectal malformations, distal intestinal atresia, prematurity, or hypothyroidism. Several factors may influence the timing of the first meconium passage, including gestational age, birth weight, sex, mode of delivery, and presence of respiratory distress. Another significant factor is breastfeeding. Effective breastfeeding is known to contribute to the maturation of the gut microbiota and has long-term positive outcomes for both mother and infant. However, the literature lacks studies examining the relationship between breastfeeding and the timing of meconium passage.This study aimed to investigate the relationship between breastfeeding success and the timing of first meconium passage in term newborns. The following hypotheses were tested: (a) There is a relationship between breastfeeding success and the timing of meconium passage in term newborns; (b) Term newborns with higher breastfeeding success will pass meconium earlier.

Eligibility Criteria:
Inclusion Criteria:

* Healthy term newborns (≥37 weeks gestation) who were exclusively breastfeed

Exclusion Criteria:

* Newborns who received any feeding other than direct breastfeeding (e.g., bottle, cup, etc.)
Liver Transplant European Study Into the Prevention of Fungal Infection

Prevention of invasive fungal infection in high risk patients following liver transplant.

After receiving liver transplant, subjects will be randomized to one of the two treatment arms.

Study drugs will be administered for a period of 21 days, or until hospital discharge, whichever occurs first.

Additionally, mortality data will be collected at the Long-term Follow-up.

Eligibility Criteria:
Inclusion Criteria:

* Undergoing orthotopic whole or split liver allograft transplantation
* Patients at 'high risk' of invasive fungal infection due to the presence of at least one of the following risk factors:

  * Re-transplantation
  * Acute liver failure
  * Pre- or post-operative renal impairment (defined as creatinine clearance ≤40 ml/min) or need for renal replacement therapy
  * Admission to Intensive Care Unit (ICU) for greater than 48 hours prior to liver transplant
  * Re-operation (abdominal surgery) within 5 days of liver transplant
  * Presence of choledocojejunostomy
  * Perioperative colonization with fungi, defined as two or more positive clinical site surveillance cultures for Candida spp., obtained within 96 hours before or after liver transplant
  * Need for prolonged mechanical ventilation for greater than 48 hours following liver transplant
  * Transfusion intraoperatively of 20 or more units of cellular blood products
* Female subject of childbearing potential must have a negative urine or serum pregnancy test prior to randomization and must agree to maintain effective birth control during the study

Exclusion Criteria:

* Any systemic antifungal therapy (excluding fluconazole or oral nystatin for a maximum of 7 days) within 14 days prior to randomization
* Evidence of documented ('proven' or 'probable') or suspected ('possible') IFD (according to the EORTC/MSG criteria)
* Allergy, hypersensitivity, or any serious reaction to an echinocandin antifungal, or any of the study drugs or their excipients
Safety and Efficacy of HMI-103 in Participants With Classical PKU Due to PAH Deficiency

This is an open-label, sequential ascending dose-escalation, Phase 1 study to evaluate the safety and efficacy of a single intravenous (I.V.) administration of HMI-103, a gene editing development candidate, in adult participants aged 18 to 55 years, inclusive, with classical PKU due to PAH deficiency who have uncontrolled disease despite Phe restricted dietary management.

This is an open-label, sequential ascending dose-escalation, Phase 1 study to evaluate the safety and efficacy of a single intravenous administration of HMI-103, a gene editing development candidate, in adult participants aged 18 to 55 years, inclusive, with classical PKU due to PAH deficiency who have uncontrolled disease despite Phe-restricted dietary management. Up to 3 dose levels of HMI-103 may be investigated. At a given dose level, 3 participants are planned to be enrolled and dosed. Participant dosing will be staggered.

Eligibility Criteria:
Inclusion Criteria:

* Adults 18-55 years of age at the time of informed consent
* Diagnosis of classical phenylketonuria (PKU) due to PAH deficiency
* Four baseline plasma Phe values with a concentration of ≥ 600 μmol/L and at least one historical value ≥ 600 μmol/L in the preceding 24 months.
* Participants must have uncontrolled classical PKU disease (despite Phe-restricted dietary management) in the judgment of the investigator and confirmed by the independent DMC at the end of the Screening period.
* Participant has the ability and willingness to maintain their baseline diet, for the duration of the trial, unless otherwise directed

Exclusion Criteria:

* Subjects with PKU that is not due to PAH deficiency
* Presence of anti-AAVHSC15 neutralizing antibodies
* Participants who are well controlled on a Phe-restricted diet.
* Hemoglobin A1c \>6.5% or fasting glucose \>126 mg/dL
* Liver function tests \> ULN
* International normalized ratio (INR) \> 1.2
* Hematology values outside of the normal range
* Previously received gene therapy for the treatment of any condition.