Masan-si, Korea, Republic of

Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome

This Phase 3, open-label, multi-center extension study will have two groups of participants: Cohort 1 (participants who participated in Study A4250-012 [NCT04674761; ASSERT] and meet the entry criteria for this study) and Cohort 2 (infants under 12 months of age) with ALGS. The study will consist of 2 or 3 periods: 1. A 'Treatment period' of 72 weeks (cohort 1) or 12 weeks (cohort 2). Participants will visit the clinic every 4 to 12 weeks and will receive a dose of 120 μg/kg odevixibat daily. 2. An 'Optional extension period' where participants who wish to continue receiving odevixibat after the 'treatment period' will have the opportunity to remain on treatment with visits every 16 weeks until the drug is commercially available. The optional extension is available provided continued use is supported by the risk-benefit profile, the participant has not been previously withdrawn or discontinued from the study, and the study is not terminated by the Sponsor. 3. A 'Safety follow-up period' of 4 weeks (cohort 1) or 2 weeks (cohort 2). The Safety Follow-up Period will not occur for those who remain on treatment in the optional extension period. Participants will need to complete an e-diary and questionnaires throughout the study (cohort 1 only). Participants will undergo blood samplings, urine collections (cohort 1 only), physical examinations, and clinical evaluations. They may continue some other medications, but the details need to be recorded.

The Effect of Airway and Chest Wall Oscillation on Respiratory Functions in COPD Patients in Acute Exacerbation

Chronic obstructive pulmonary disease (COPD); It is a common, treatable and preventable disease characterized by progressive and irreversible airflow limitation and has systemic effects with respiratory system involvement. Increased respiratory workload, decreased work of breathing in patients are important factors that cause respiratory and peripheral muscle strength and functional capacity exercise intolerance. It is defined as an exacerbation if there is an increase in shortness of breath, cough, sputum amount and purulence, which necessitates a change in treatment in the COPD patients. In the guides; Individualized exercise training for disease management is an essential component of these programs. In the literature; Although there are many studies showing the beneficial effects of traditional exercise training programs in patients with COPD, physiotherapy applications used in the exacerbation period are recommended, but there are not many studies examining the device technologies used in this area. In this period, devices that provide chest wall oscillation and airway oscillation can be preferred for the symptomatic treatment of increasing cough and sputum complaints. There is no study comparing these two methods that we know of. Our study will be carried out on 33 patients hospitalized in the Pulmonology Service of the Health Sciences University Gülhane Training and Research Hospital, followed up with the diagnosis of COPD exacerbation, who met the inclusion criteria and accepted to participate in the study on a voluntary basis. Patients will be randomized into 3 groups as 2 experiments and 1 control. Measurements of patients admitted to the ward will be made at the beginning and end of the study. Socio-demographic data of the patient, chest circumference measurement, COPD assessment test, mMRC dyspnea scale, BODE index, pulmonary function test, arterial blood gas, chest X-ray, 30-second sit and stand test, Richard-Campbell Sleep Scale, Clinical COPD Questionnaire, Charlson comorbidity index will be applied. Pursed-lip breathing, active breathing techniques cycle, bronchial hygiene techniques, mobilization, calisthenic exercises and patient education (teaching relaxation positions and energy conservation techniques) will be applied to the patients in the study and control groups during their hospitalization. Chest wall oscillation will be practiced together with chest physiotherapy in the 1st experimental group, and airway oscillation will be practiced in the 2nd experimental group with the same physiotherapy program. In the study, the clinical use and technical features of the devices will be examined.

A Phase III Study to Investigate the Efficacy and Safety of Baxdrostat in Combination With Dapagliflozin on CKD Progression in Participants With CKD and High Blood Pressure.

A Study to Compare the Pharmacokinetics, Efficacy, Safety, and Immunogenicity of Pembrolizumab (SB27, EU Sourced Keytruda, and US Sourced Keytruda) in Subjects with Stage II-IIIA NSCLC Following Complete Resection and Adjuvant Platinum-based Chemotherapy

Alexander Technique on Labor Pain and Anxiety

Pain during childbirth has been described as the most severe pain women experience throughout their life course. Labor pain is a result of physiologic changes in the uterus. Pharmacologic and nonpharmacologic methods are used in the management of pain, which is a physiologic process. Nonpharmacological methods help women to feel comfortable in labor and contribute to women to have a positive birth experience by providing relaxation and reducing pain perception through cognitive, psychological and physical methods (Adil et al., 2020; Thompson et al. 2019). In addition, midwives who are with the woman during the birth process use non-pharmacological methods in an independent role in the management of labor pain, and this is also effective in alleviating the burden on current health expenditures. Alexander Technique appears as a non-pharmacologic method. In the literature, there is no study on the application of the Alexander Technique in the management of labor pain. It is assumed that the current posture and position to be provided with the Alexander Technique will be effective on pain and anxiety during the birth process. It is necessary to conduct studies on the use of the Alexander Technique in the birth process and evaluate the results. This study will be the first study on the Alexander Technique to be applied to women in labor. The research to be conducted is planned as a randomized controlled experimental study. The population of the study consists of 68 primiparous pregnant women who applied to Istanbul Provincial Health Directorate Başakşehir Çam and Sakura City Hospital, D-Block Maternity Hospital between November 2023 and May 2024. Simple random sampling method will be used for the sample of the study. Pregnant women included in the study will be informed about the study and 'Informed Consent Form' and 'Voluntary Consent Form' will be filled. 'Personal Information Form', 'Visual Analog Scale (VAS)', 'Beck Anxiety Scale (BAS)' and 'Oxford Birth Anxiety Scale (OWLS)' will be applied to the pregnant women included in the experimental group. After the initial data is collected, the principles in the Alexander Technique will be applied when the woman's cervical dilatation is 0-4 cm. The duration of the application is approximately 15 minutes and the researcher will take an active role with the woman accompanied by a counselor during this period. The second application will be applied when the cervical dilatation is 5-7 cm and the third application will be applied when the cervical dilatation is 8-10 cm. 'Visual Analog Scale (VAS)' will be applied after each application. 'Visual Analog Scale (VAS)', 'Beck Anxiety Scale (BAS)' and 'Oxford Birth Anxiety Scale (OWLS)' will be applied at the 2nd hour after delivery.

Effectiveness of TTNS in MS Patients With Lower Urinary Track Symptoms

This is a single-centered, prospective, single blind, randomized controlled study of patients with MS who suffer lower urinary track symptoms. All patients will be informed of the details of all the procedures and of the details of the study. After the written informed consent is obtained, patients will be distributed to two equal groups using randomized number table. At the beginning and end of the study, post-void residue (PVR) will be calculated with ultrasonography, International Consultation on Incontinence Questionnaire Short Form (ICIQ-SF), Incontinence Quality of Life( I-QOL), 3 day bladder diary (number of urination, urge, incontinence, nocturia) forms will be administered. Patients will be evaluated via PVR, urodynamic measurements, ICIQ-SF, I-QOL and bladder diary.

A Phase 1/2 Study to Investigate CRB-701 in Solid Tumors

This is a three-part open-label, Phase 1/2 clinical trial designed to evaluate the safety, PK, and efficacy of CRB-701 in participants with advanced solid tumors expressing nectin-4. Part A will include solid tumor types known to express nectin-4. Dose escalation will be guided by the Bayesian optimal interval (BOIN) design to determine the MTD of CRB-701. Four (4) dose groups are pre-determined. Dose escalation/de-escalation decisions are made based on the occurrence of DLT. Part B will determine the RP2D of CRB-701 by evaluating two dose levels of CRB-701 by using a time-to-event Bayesian optimal Phase 2 (TOP) study design to optimize the dose of CRB-701 in one or more separate cohorts of participants with nectin-4-positive tumors. During Part C, the RP2D dose of CRB-701 will be evaluated in five planned expansion cohorts using Simon's optimal two-stage design.

Association of Urine BDNF and NGF With Lower Urinary System Parameters

As the demographic structure of societies changes, benign prostatic hyperplasia (BPH) is one of the most important health problems for older men, especially in developed countries. Histologically, BPH is a benign proliferative process involving both epithelial and stromal elements and is characterised by progressive enlargement of the prostate. BPH is a lifelong chronic disease with an incidence of approximately 8% in men aged 31-40 years, which increases rapidly with age and reaches 90% in the 9th decade. Symptom complex including increased frequency of urination, sudden feeling of urge to urinate, nocturia, difficulty in urinating, feeling of incomplete emptying of the bladder, decreased flow rate and intermittent urination are called lower urinary tract symptoms (LUTS). The most important cause of LUTS in men is BPH. Many structural and physiological changes occur in the lower urinary system with bladder outlet obstruction. Detrusor hypertrophy and bladder hyperactivity may occur due to bladder outlet obstruction. Although the density of afferent and efferent nerves in the bladder decreases after urethral obstruction, enlargement of their trunks indicates that changes occur in these nerves. In addition, changes also occur in the neural pathways of the central nervous system following lower urinary tract obstruction. Nerve growth factor (NGF) and brain derived neurotropin factor (BDNF) are trophic proteins that act as retrograde messengers between peripheral effector tissue and the nerves that innervate it. In peripheral tissues, the source of NGF and BDNF is presumed to be the target tissues innervated by nerves. Smooth muscle cells, fibroblasts, astrocytes and other cells synthesise NGF and BDNF in culture medium. NGF is required for survival, development and neurotransmitter synthesis regulation of dorsal root ganglion and sympathetic cells in embryonic and postnatal life . NGF receptor contains two subunits; the low affinity subunit is called p75 and the high affinity tyrosine kinase subunit is called tyrosine kinase A which is responsible for the growth and survival effects of NGF. Many potential stimuli that increase NGF in the lower urinary system have been identified. These are denervation, inflammation and mechanical tension. This information has led to the idea that autonomic innervation changes in the bladder may be related with changing NGF levels. Altered afferent and adrenergic innervation in the obstructed bladder increases the possibility that NGF plays an important role in this neural growth because this type of nerves are highly sensitive to this neurotrophin. Clinical and experimental data have shown a relationship between urinary NGF and overactive bladder. Overactive bladder (OAB) is a complex of uncomfortable symptoms accompanied by a feeling of urgency, frequent urination and nocturia, with or without urge incontinence. Overactive bladder is thought to occur as a result of an inflammatory process occurring in the bladder. The reason for this is shown as high levels of inflammation mediators in bladder biopsies and urine of OAB patients. NGF, which is one of the inflammation mediators, was found to be high in OAB patients in studies and it was observed that NGF level decreased after antimuscarinic treatment or botulinum neurotoxin injection. In this study, we investigated NGF ve BDNF levels in urine samples obtained before surgery (Transurethral Prostate Resection, Prostate Enucleation with Holmium Laser and Prostate Enucleation with Thulium Fibre Laser) and after removal of obstruction in patients with bladder outlet obstruction secondary to benign prostatic enlargement using ELISA method, We aimed to determine the role of NGF and BDNF in bladder outlet obstruction and bladder changes secondary to obstruction by comparing with control patients without obstruction.

Pediatric-Quiz Game Behaviour Guidance in Children

Behavior orientation is the cornerstone of the success of pediatric dentistry. The use of behavior management techniques enables children to learn appropriate behavior and coping skills, reduce anxiety, and facilitate the delivery of adequate oral health care. Better communication can be achieved when children feel the freedom to express themselves. In the same way, parents may state that in cases where there is no coercion and verbal communication, the children are uncomfortable and they have problems during their second-third dentist visits. However, in some cases, success may not be achieved even if different behavior management techniques are applied. Activities such as diverting attention in children to other areas, starting the communication in the waiting room before entering the practice environment can lead to self-confidence, relaxation and confidence. The method of rewarding can also be very effective in directing behavior in children. For this reason, it communicates with a combination of some techniques without depending on only one technique and can lead to applications in dentistry practice. Based on this idea, some behavior guidance systems have been tried to be created. On the basis of the magic game, it has been tried to move the children away from the current real situation, the reality of dental treatment, to move forward in communication and successful results have been obtained. With this information, the aim of our study is to experience a new behavior management technique in children. The main thing in the technique is to start the communication with the child in the waiting room of the dentist and to reduce the anxiety of the child by improving the self-efficacy perception of the child. In this direction, in order to increase the child's self-efficacy perception, simple questions are asked to the child about colors, fruits and animals, if necessary, clues are given to give correct answers, and verbal praise for correct answers aims to increase self-efficacy perception and reduce anxiety. After the communication is started in the waiting room, the dental examination, dental chair and planned procedures will be introduced to the child, together with the tell-show-do method, as always practiced in the dental chair. The aim of this study is to investigate the effectiveness of question-answer behavior guidance technique in dental treatment in pediatric patients. Children's pain and anxiety levels were assessed by various tests designed to purpose. The Visual Analogue Scale (VAS), a self-reporting scale,was preferred to evaluate children's experience of pain. The scale displayed a colored spectrum on the front side ranging from no pain (white) to severe pain (red), which indicates relevant pain scores ranging from 0 to 10 on the reverse side.10

A Study to Evaluate the Efficacy and Safety of Subcutaneous Amlitelimab on Background Topical Corticosteroids Therapy in Participants Aged 12 Years and Older With Moderate-to-severe AD Who Have Had an Inadequate Response to Prior Biologic Therapy or an Oral JAK Inhibitor