cyrilzakka/clinical-trials huggingface dataset:
IGF-1 Treatment for Individuals With Short Stature Due to PAPP-A2 Deficiency

With this study we want to investigate the pharmacokinetic (PK) effect of a single injection of rhIGF-1 in patients with PAPP-A2 mutations compared to heterozygous carriers and healthy controls. This will be followed by treatment of PAPP-A2 deficient patients with IGF-1 for a period of one-year to assess growth velocity. Additionally, we want to further describe the phenotypic characteristics of patients with PAPP-A2 deficiency.

The 24-hour pharmacokinetic response of free and total IGF-1 and IGF binding protein-3 (IGFBP-3) to a single dose of rhIGF-1 (120 mcg/kg) in three patients with PAPP-A2 mutation compared to up to four unaffected heterozygous relatives and 2 healthy adult controls.

One-year trial of rhIGF-1 at standard dose given to the two youngest males with PAPP-A2 mutation. The primary end point of this trial will be first year height velocity. Secondary outcomes will include height standard deviation score (SDS), height velocity, and whole body and lumbar spine bone mineral density assessment. The study was amended to extend the treatment period to continue until the subject has stopped growing (or elects to withdraw). All study procedures remain the same. Important note: the treatment phase continues to follow the youngest affected male. The older affected male developed an adverse event that resulted in discontinuation of treatment.

A post-treatment follow up visit (either in-person or remote) will be completed for the study participant who remained on Increlex approximately one-year after their discontinuation of therapy.

Description of additional phenotypic characteristics of patients with PAPP-A2 mutation will be studied by collecting information on glucose and insulin metabolism, body composition, bone geometry and bone density before and after treatment with rhIGF-1. These measures will be collected at the 12-month time period, and every year thereafter until the completion of the study. All three affected siblings will take part in the phenotyping activities.

Eligibility Criteria:
PAPP-A2 deficient

Inclusion Criteria:

* Defect in PAPP-A2 (heterozygous or homozygous mutation)

Exclusion Criteria:

* None

Healthy Volunteers

Inclusion Criteria:

* Between the ages of 18 and 30
* In general good health

Exclusion Criteria:

* Any medications (with the exception of contraceptives)
* Pregnancy
A Dose Escalation Study of Intravenous L-citrulline in Steady-state Sickle Cell Disease

The purpose of this study is to assess the maximum tolerated dose, safety and pharmacokinetics of an investigational drug, intravenous (IV) citrulline, in subjects in steady-state sickle cell disease.


Eligibility Criteria:
Inclusion Criteria:

* Sickle cell disease genotypes (HbSS, HbS/β° thalassemia, HbS/β+thalassemia, HbSC)

Exclusion Criteria:

* Presence of any acute illness defined by fever \>100.4° F within the past 48 hours
* Presence of sickle cell pain crisis defined by the presence of pain requiring oral or parental opioid therapy.
* Presence of acute chest syndrome or presence of any other complication related to sickle cell disease requiring hospitalization such as splenic sequestration, hepatic sequestration, stroke, avascular necrosis of the hip/shoulder, acute priapism, and patients with diabetes etc.
* Severe anemia (hemoglobin \< 5g/dL)
* History of red blood cell transfusion within the last 14 days
* Systemic steroid therapy within the last 48 hours
* Pregnant (as confirmed by a negative urine pregnancy test) or lactating female
* Alanine/aspartate transferase \>2x upper limit of normal laboratory range for age.
* Elevated serum creatinine \>1.5mg/dL
* Patients with an inability to give consent will be excluded
* Medications that are known to be contra-indicated with use of L-citrulline (concurrent use of hydroxyurea will be allowed).
* History of diabetes due to risk of electrolyte imbalance
Analysis of Biomarkers for Acute Graft-versus-Host Disease (GVHD)

Samples of blood and urine will be analyzed for biomarkers to check their predictivity of Graft-versus-Host Disease (GVHD) outcomes.

To determine if serum biomarkers of inflammation correlate with onset and severity of acute GVHD, particularly GVHD of the gut.

Eligibility Criteria:
Inclusion Criteria:

* 18 years old or older patients
* Undergoing allogeneic stem cell transplant

Exclusion Criteria:

None
Caudal Epidural Block for Postoperative Analgesia in Adolescents Following Major Hip Surgery.

This retrospective chart review will demonstrate the feasibility of performing caudal epidural blockade in adolescents undergoing major orthopedic hip surgery and provide information regarding the feasibility of this technique's use on a regular basis.


Eligibility Criteria:
Inclusion Criteria:

* Adolescents that underwent major orthopedic hip surgery.

Exclusion Criteria:

* None.
Web-Based Epilepsy Education Program for Adolescents and Parents

This study aims to develop a web-based epilepsy education program for adolescents with epilepsy and their parents and to evaluate the efficacy of eb-based epilepsy education program on health management.eb-based epilepsy education program has been proved to be a useful and reliable educational website for the development of knowledge, attitude, self-efficacy and e-health literacy of adolescents with epilepsy and their parents.

This study aims to develop a web-based epilepsy education program for adolescents with epilepsy and their parents and to evaluate the efficacy of web-based epilepsy education program on health management.

This randomized controlled study was conducted with 69 adolescents with epilepsy aged between 9-18 and their parents (n=71) who were accepted to Pediatric Neurology Policlinic of Antalya Training and Research Hospital of Health Science University between November 2017 and April 2018. The study sample was divided into two groups as study (35 adolescents, 37 parents) and control (34 adolescents, 34 parents) groups using simple randomization method. The web-based epilepsy training program which was developed during the preparation stage of the study was evaluated by 13 experts using the Quality of Criteria for Consumer Health Information measurement instrument. The data collection materials (Seizure Self efficacy scale, E-Health literacy scale, Child Attitude Toward Illness Scale, Website Analysis and MeasureMent Inventory Scale) were administered by the researcher to the adolescents and parents in both groups before applying eb-based epilepsy education program. Following the administration of eb-based epilepsy education program to the initiative group, all participants were observed for three months. The data collection materials were reapplied to all the participants at the end of the third month. In addition, the adolescents and their parents in the study group were applied Website Analysis and Measurement Inventory measurement tool for the evaluation of usability of the site.

Eligibility Criteria:
Inclusion Criteria:

* Adolescents without mental disability,
* Followed-up for at least six months with epilepsy diagnosis,
* No chronic disease other than epilepsy,
* No photosensitive epilepsy diagnosis,
* Internet access and use, and parents of these adolescents with internet access and use - were included in the study.

Exclusion Criteria:

* Adolescents and parents who do not use the Internet
Efficacy of Repaglinide in Subjects With Type 2 Diabetes

This trial is conducted in Europe. The aim of this trial is to compare the efficacy of repaglinide to glyburide and placebo on hepatic glucose metabolism in subjects with type 2 diabetes.


Eligibility Criteria:
Inclusion Criteria:

* Type 2 diabetes
* HbA1c (glycosylated haemoglobin A1c) above 7 %
* BMI (body mass index) below 32 kg/m\^2
* FBG (fasting blood glucose) after wash out period 130-220 mg/dl
* Currently treated with diet or OHA (oral hypoglycaemic agent)

Exclusion Criteria:

* Current systemic treatment with concomitant medication
* Known or suspected history of drug or alcohol dependence
* Any other significant concomitant disease such as cerebrovascular or symptomatic peripheral vascular disease, malignant, disease or severe treated or untreated hypertension
* Hepatic disease
* Cardiac problems
* Active proliferative retinopathy
* Known or suspected allergy to trial product or related products
* Women in fertile age and women having the intention of becoming pregnant
* Body Mass Index (BMI) above 32 kg/m\^2
Product Feasibility of a New Silicone Hydrogel Contact Lens

The objective of this study is to determine the clinical feasibility and to evaluate the product performance of investigational contact lens.


Eligibility Criteria:
Inclusion Criteria:

* Subjects must have clear central corneas and be free of any anterior segment disorders.
* Subjects must be adapted wearers of soft contact lenses, wear a lens in each eye, and each lens must be of the same manufacture and brand.
* Subjects must be myopic and require lens correction

Exclusion Criteria:

* Subjects with any systemic disease affecting ocular health.
* Subjects using any systemic or topical medications that will, in the Investigator's opinion, affect ocular physiology or lens performance.
* Subjects with an active ocular disease or are using any ocular medication.
* Subjects with any "Present" finding during the slit lamp examination that, in the Investigator's judgment, interferes with contact lens wear.
Early Feeds in Gastroschisis

This study aims to evaluate the feasibility of initiating early feeds in neonates with gastroschisis, a condition where infants are born with their intestines outside the body.


Eligibility Criteria:
Inclusion Criteria:

* Neonates with presumed simple gastroschisis
* Born at ≥ 34 weeks' gestation
* Hemodynamically stable
* Consented within 48 hours after abdominal closure
* Mothers who are ≥16 years old
* Mothers who speak English or Spanish

Exclusion Criteria:

* Neonates with evidence of complex gastroschisis at time of closure (any ischemic bowel, intestinal perforation, or obvious atresia with mesenteric defect)
* Presence of any major congenital anomalies
* Neonates who are receiving ionotropic medications
* Neonates who are wards of the state
* Neonates whose care is considered to be futile or those undergoing re-direction of care
* Neonates participating in another interventional trial
* Any patient deemed unfit for participation by study investigator(s)
Clinical and Dynamic Floor MRI Evaluation Before and After Prolapse Surgery

Pelvic organ prolapsed, associated with defecation disorders and urinary tracts symptoms are common and affect up to 25% of the population, mostly parous women. The pelvic floor must be seen as one entity, with multiple anatomical and physiological interactions between the various compartments (rectum, vagina, uterus and bladder) which are embedded in the same anatomical region. The often complex pathologies of this region should therefore be treated in a multidisciplinary setting.

Besides clinical evaluation, functional dynamic imaging of anorectal and pelvic floor disfunctions has an important role in the diagnosis and management of these disorders. Although the colpocystodefecography is still considered to be the golden standard in imaging this complex anatomical region, there is clearly a need for more precise imaging of the structural details, preferentially without any irradiation. Transperineal ultrasound is an option but the investigators have chosen to evaluate the use of dynamic magnetic resonance imaging. In contrast to colpocystodefecography, dynamic pelvic floor magnetic resonance imaging is an evolving technology and its precise role in functional imaging of the pelvic floor still remains to be determined.

Prolapse surgery is commonly performed and therefore it is important to assess the efficacy of the operations in correcting the anatomical defects and the symptoms associated without creating new, pelvic floor related symptoms. Few studies exist today allowing the assessment of the anatomical changes and symptoms after surgery, through abdominal or perineal approach.

This study will evaluate the reliability of the dynamic pelvic floor imaging, done in a sitting position, compared to colpocystodefecography, done in a sitting position. It will also compare clinical objective and subjective results related to pelvic floor abnormalities with imaging. Finally, it will evaluate the anatomical changes in correlation with the clinical results, organ position and inter-compartments relationships after surgery.

This study will allow to understand and explain some relapses and failures and could lead to an improvement of the indications for surgery and surgical techniques used.


Eligibility Criteria:
Inclusion Criteria:

All patients who will undergo internal or external rectal prolapse, enterocele or urogenital prolapse surgery in CHU Brugmann and CHU St Pierre.

Exclusion Criteria:

* MRI contra-indications
* Patients with prior pelvic floor surgery
A Study of Milvexian in Participants After a Recent Acute Coronary Syndrome

The purpose of this study is to evaluate that milvexian is superior to placebo, in addition to standard-of-care, in reducing the risk of major adverse cardiovascular event (MACE) (the composite of cardiovascular \[CV\] death, myocardial infarction \[MI\], and ischemic stroke).


Eligibility Criteria:
Inclusion Criteria:

* Participants must have an index event that meets all 3 of the following criteria within 7 days prior to randomization: a) clinical syndrome consistent with spontaneous cardiac ischemia, b) diagnosis of acute coronary syndrome (ACS) (that is, ST-elevation myocardial infarction \[STEMI\], non-STEMI, or unstable angina \[UA\]), c) cardiac biomarker elevation (example, troponin I, troponin T, creatine kinase-MB \[CK-MB\]) above the upper limit of normal as determined by the local laboratory
* Participants must have at least 2 of the following risk factors:a) age 65 or older, b) diabetes mellitus, c) history of a prior myocardial infarction (MI) (other than index ACS event), d) multivessel coronary artery disease (CAD), e) history of coronary artery bypass graft (CABG) surgery prior to index ACS event, f) history of peripheral artery disease (PAD) or cerebrovascular disease (example, carotid atherosclerosis, intracranial artery stenosis, g) conservative management (that is, no percutaneous intervention \[PCI\] or CABG after index ACS event), h) Any one or more of the following high-risk angiographic features i) total stent length of greater than (\>) 30 millimeters (mm), ii) thrombotic target lesion, iii) bifurcation lesion treated with more than one stent, iv) calcified target lesion treated with atherectomy, v) treatment of obstructive left main or proximal left anterior descending artery for index ACS (or clinical diagnosis of an anterior STEMI)
* All female participants of childbearing potential must have a negative highly sensitive serum beta-human chorionic gonadotropin (hCG) or urine test at screening
* A female participant must not be pregnant, breastfeeding, or planning to become pregnant until 4 days (5 half-lives) after the last dose of study intervention

Exclusion Criteria:

* MI secondary to ischemia due to either increased oxygen demand or decreased supply (Type 2 MI) or periprocedural MI as the index ACS event
* Planned CABG or staged PCI after randomization
* Any condition that requires chronic anticoagulation at the discretion of the investigator and/or local guidelines
* Conditions with a significant increased risk of bleeding (example, clinically significant bleeding within previous 3 months, known bleeding diathesis, et cetera)
PRospective Evaluation Complementing Investigation With Acurate Neo Device

The study collects real-world data of patients who were treated with the Acurate Neo TAVI System and evaluates early and midterm clinical outcomes.

The study collects real-world data of patients who were treated with the Acurate Neo TAVI System and evaluates early and midterm clinical outcomes.

Eligibility Criteria:
Inclusion Criteria:

* ≥18 years.
* Severe aortic stenosis with indication of implantation of aortic valve prosthesis in which an Acurate Neo device is implanted according to the indications for use.
* Has signed the Patient Informed Consent Form.

Exclusion Criteria:

* Severe aortic stenosis without indication of implantation of aortic valve prosthesis in which an Acurate Neo device is implanted according to the indications for use.
The Adding Value of Parents to Nursing Care in the Control of FiO2

The purpose of this study is to determine whether saturation control by parents in children younger than 32 weeks or under 1500 g versus conventional care performed by nursing increases the time in which these patients remain at optimal saturation range. If the hypothesis of this study is confirmed premature infants could prevent episodes of hypoxia-hyperoxia and also give more relevance to the role of parents in caring for their children admitted in a neonatal intensive care unit.

Both episodes of hyperoxia and hypoxemia in preterm infants increase morbidity and mortality. Nowadays the adjustment of FiO2 is manually controlled, which means it depends on the staff, needing multiple adjustments throughout the day.

The investigators's hypothesis is that the FiO2 control by parents, improve the percentage of time in range of optimum saturation at least 10% compared to control by nurses.

The aim of this study is to compare the proportion of time with oxygen saturation (SpO2) within the assigned target range, in the preterm infants whose parents modify the fraction of inspired oxygen (FiO2) versus the nursing staff modifying group.

This is a randomized controlled clinical study in the neonatal intensive care unit at Hospital 12 de Octubre. Patients will be randomized in two groups, Experimental Group: parents controlling the saturation ranges, they will manually adjust FiO2 versus the Control Group: controlled by nursing staff (standard procedure). Saturations will be registered and adjustments of FiO2 will be filmed for nonconsecutive 20-hours periods. Twenty-three patients will be enrolled in each group.

Eligibility Criteria:
Inclusion Criteria:

* Related to child
* Preterm neonates less than 32 weeks or less than 1500 g hospitalized in the Neonatal Intensive Care Unit at Hospital 12 de Octubre.
* Receiving supplemental oxygen ≤0.4 administered by CPAP or conventional nasal cannula or high flow or children with BiPAP (flow generator with double level pressure)
* Minimum age 7 days old.
* Related to the mother:
* Absence of health problems that allows her to perform the task.
* Availability to frequently visit her baby with the father or with a companion, at least for 20 hours, not consecutive.
* Understanding of the study and the Spanish language by the mother and father or companion.
* Signed informed consent.

Exclusion Criteria:

* Evidence of pneumothorax or pneumomediastinum.
* Non-compliance with study protocol.
* Requiring treatment with vasoactive drugs.
Involvement of Viral Infections in the Pathogenesis of Chronic Lymphocytic Thyroiditis

Autoimmune diseases represent a heterogeneous group of pathologies whose etiopathogenic mechanisms are most often unknown. Autoimmune diseases are the leading cause of morbidity and mortality in young women and autoimmune thyroid diseases are the most common.

Viral infections are the main environmental factors suspected of triggering autoimmune diseases. Several viruses are certainly involved, all of which are possibly capable of triggering an autoimmune response. However, the precise identification of the viruses involved remains to be established.

It has been shown for the first time by the 2005 PHRC that enteroviral RNA is present in perioperative specimens of thyroid tissue. However, this case-control study did not show any difference between the thyroid group and the group other thyroid pathologies It has been recently published that Parvovirus is possibly involved in thyroiditis: the parvoviral genome is present in the thyroid tissue of Hashimoto thyroiditis operated and more precisely is present within the thyrocytes itself.


Eligibility Criteria:
Inclusion Criteria:

* Patients participating in PHRC 2005 "involvement of enteroviruses in TLC pathogenesis"
* Inclusion of TLC: presence of a significant lymphocyte infiltrate within the thyroid parenchyma defined by the presence of clusters of more than 30 to 50 lymphocytes
* Inclusion of controls: absence of significant lymphocyte infiltrate

Exclusion Criteria:

* Persons refusing to participate in research
Clinical Efficacy of a Virtual Reality Tool for the Treatment of Obesity

The objective of the study, which is framed within European Union's H2020 project titled SOCRATES, is to assess the clinical efficacy of a Virtual Reality (VR) embodiment tool for treating obesity and to compare it to usual care, through a Randomized Controlled Trial


Eligibility Criteria:
Inclusion Criteria:

* BMI ≥ 30 kg/m2 and ≤ 55 kg/m2.
* Receiving ambulatory treatment at the Vall d´Hebron University Hospital.
* No concurrent involvement in other treatment related to the obesity condition.
* Minimal digital skills and able to use a proper digital device (Smartphone, tablet, computer).
* Oral and written understanding of the Spanish language to complete the questionnaires and use the system.
* Acceptance to sign the informed consent to participate.

Exclusion Criteria:

* Body Mass Index \> 45.
* Presence of an Eating Disorder during the last 2 years.
* Non-stabilised severe mental disorder that could interfere with the successful implementation of the research protocol (i.e. psychosis, depression with suicidal risk, alcohol or drug abuse, psychotic or manic symptoms).
* Auditory or visual complications that might affect the participant during exposure to the VR platform.
* Intellectual disability or any major illness seriously affecting cognitive performance (i.e. neurological disorders).
* Personal history of epilepsy.
Long-term Study to Evaluate Different Dental Implant Alloys in Straumann 3.3mm Diameter Bone Level Implants

Patients with an edentulous mandible restored with implants who have participated in the core study CR 04/07 will be recruited for this extension study. No further interventions will be made for this extension study other than a clinical follow-up examination and questionnaires. Patients will not receive an investigational treatment in this study. Patients will be followed at 5 to 10 years from the date of the original implant loading

The primary study objective is to compare implant survival with the Titanium Zirconium (Roxolid™) implant compared the Titanium grade IV (SLActive®) implant between baseline (surgery) and 10 years.

The following outcome parameters will be evaluated at 5 and 10 years post surgery for the two different implant types Straumann® Bone Level SLActive, diameter 3.3 mm, Titanium-Zirconium (Roxolid®) vs. Straumann® Bone Level SLActive implant, diameter 3.3 mm, Titanium Grade IV:

* Implant survival
* Implant success according to Buser et al. (1990)
* Crestal bone level change measured by Orthopantomogram (OPT)
* Soft tissue health
* Clinical measurements
* Prosthetic parts assessments: Success and maintenance
* Oral Health Related Quality of Life (OHRQoL)
* Product Safety (Adverse events and device deficiencies)

Eligibility Criteria:
Inclusion Criteria:

* Subjects must have voluntarily signed the informed consent form
* Patients must be committed to participate in the study until the ten year follow-up examinations
* Subjects must have received treatment in the CR 04/07 core study
* Subjects must have completed 3 year visit of core study

Exclusion Criteria:

* Physical handicaps that would interfere with the ability to perform adequate oral hygiene or attend follow up visits
* Use of any investigational drug or device during the study
* Conditions or circumstances, in the opinion of the investigator, which would prevent completion of study participation or interfere with analysis of study results, such as history of non-compliance, unreliability