Active clinical trials for "Congenital Abnormalities"

Phase 1 and 2 trial to study the safety, pharmacokinetics, and efficacy of TAS0953/HM06 in patients with advanced solid tumors with RET gene abnormalities. Phase 1 aims to determine the Maximum Tolerated Dose (MTD) and identify the Recommended Phase 2 Dose (RP2D) to be used in phase 2.

The main objectives of this study are to evaluate overall clinical performance and safety of the Persona Ti-Nidium implant in total knee arthroplasty.

Supervised exercise is an essential component of contemporary center-based cardiac rehabilitation (CR) programs. Despite the proven effectiveness, uptake and adherence to supervised exercise-based CR (exCR) remains suboptimal (approximately 50%), especially in rural and remote areas of Sweden. The main reasons for low participation rate in centre-based exCR are different accessibility barriers i.e. long distances, transportation problems or employment commitments. In this randomised clinical trial, the overall aim is to evaluate the effectiveness of, home-based, remotely monitored and coached exCR using the REMOTE-CR system compared to home-based exCR based on individualized information regarding current exercise recommendations but without monitoring and coaching (usual care) to improve exercise capacity. Our hypothesis is that remotely monitored and coached exCR will improve exercise capacity to a greater extent than home-based exCR without monitoring and coaching (usual care).

The study in question is a post market clinical follow up (PMCF) study to evaluate the safety and performance of bioabsorbable headless compression screw Inion CompressOn in selected fracture, osteotomy and arthrodeses operations of the foot and ankle. The study recruits 125 adult patients who meet the acceptance criteria. The follow-up time for each study patient is 4 years. The study is a single center single group study which is conducted in Tampere University Hospital (TAYS) in Finland. The main focus of the study in terms of performance is to evaluate the bone healing/ossification of operated areas. The main focus of the study in terms of safety is to evaluate the occurrence of adverse events and required revision surgeries that are or might be related to the study device.

The aim of the BEST study is to examine the feasibility of assessing patient and clinician reported outcome of a single electrosclerotherapy treatment of vascular malformations. Sclerotherapy = injection of Bleomycin into vascular birthmarks to seal off abnormal vascular channels Electroporation = application of an electric field to vessels treated with a handheld needle electrode Electrosclerotherapy = a combination of Bleomycin sclerotherapy and electroporation

Objective of the study is to determine possibilities of intraoperative sonography in detecting of various brain mass lesions, assessing extent of their resection and define indications to use ultrasound-guided needle or ultrasound wire-guided port.

Spinal muscular atrophy (SMA) is a serious neuromuscular disease characterized by the degeneration of alpha motor neurons in the spinal cord, resulting in progressive proximal muscle atrophy and denervation. The main problems are posture disorders, scoliosis, pelvic curvature, contracture, hip dislocation, foot and chest deformities. In this study, examining the effectiveness of trunk support used to alleviate the progression of scoliosis in children diagnosed with SMA Type I will contribute to the current literature.In addition to Individualized Trunk Exercises (ITE), Individualized Pulmonary Rehabilitation (IPR) and Chest Care (CC) Programme, the use of thoracolumbosacral spinal orthosis in Type I children will be used for the first time in our country and in the world literature. SMA. Our aim in the project is to examine the effectiveness of this treatment program on the motor functions, scoliosis Cobb angle, pelvic curvature and chest deformity of children with Type I SMA.The project is planned to be carried out with children diagnosed with Type I SMA who are followed up at Medipol Mega University Hospital Pediatric Chest Diseases Polyclinic.In evaluating the development of scoliosis as the primary outcome measure; Radiological evaluation (Cobb Angle) and examination of chest deformity; Lung X-ray (Basal Chest Wall Upper-Lower Ratio Measurement) will be used. As secondary outcome measures, the Children's Hospital of Philadelphia Infant Test for Neuromuscular Disorders and the Hammersmith Functional Motor Scale Expanded were used to assess motor functions and examine the level of motor development; In the World Health Organization Motor Development Scale body posture assessment; Supine Trunk Rotation Angle Test and Pelvic Curvature Test, pulse oximetry to assess oxygenation; In determining the level of satisfaction with orthosis use; Children/families' information will be questioned through the Quebec Assistive Technology User Satisfaction Evaluation Survey and Personal Information Form.The active control group will receive the ITE, IPR and CC program as a home program and once a week in the outpatient clinic for 8 weeks, 7 days a week, once a day, each session being 50-60 minutes. In the ITE-IPR-CC + spine orthosis group, in addition to the control group program, a thoracolumbosacral spine orthosis specially designed for the child will be used every day of the week and 8 hours a day for 8 weeks. Evaluations will be made at baseline and at week 8.

The purpose of this open-label study is to evaluate the safety and efficacy of cobimetinib in extracranial AVM.

In this study, The investigators aims to evaluate the efficacy and safety of topical hyaluronic acid nanoparticles as a novel nanodermatological tool in periocular rejuvenation as a replacement approach of invasive dermal filler injections.

The prognosis of patients undergoing spinal deformity surgery is often compromised by perioperative anemia due to iron deficiency. The aim of this randomized, controlled trial was to evaluate whether postoperative ferric derisomaltose intravenous injection may improve anemia and prognosis in patients undergoing spinal deformity surgery comparing with oral iron. Participants will be randomly assigned to the treatment group (intravenous ferric derisomaltose) and the control group (oral iron). Changes in hemoglobin concentration, percentage of anemia correction, changes in iron indicators, patient quality of life, and incidence of adverse events will be analyzed to evaluate the efficacy and safety of iron isomaltoside infusion.