Active clinical trials for "Spinal Dysraphism"

The purpose of this investigation is to evaluate maternal and fetal outcomes following fetoscopic repair of fetal spina bifida at the Johns Hopkins Hospital. The hypothesis of this study is that fetoscopic spina bifida repair is feasible and has the same effectiveness as open repair of fetal spina bifida, but with the benefit of significantly lower maternal and fetal complication rates. The fetal benefit of the procedure will be the prenatal repair of spina bifida. The maternal benefit of fetoscopic spina bifida repair will be the avoidance of a large uterine incision. This type of incision increases the risk of uterine rupture and requires that all future deliveries are by cesarean section. The use of the minimally invasive fetoscopic surgical technique may also lower the risk of preterm premature rupture of membranes and preterm birth compared to open fetal surgery. Finally, successful fetoscopic spina bifida repair also makes vaginal delivery possible.

Researchers are studying a new minimally invasive technique (fetoscopic repair) for repair of spina bifida (MMC) during the second trimester of pregnancy. Researchers are trying to determine if this less invasive surgical approach will have less risk to the mother and at the same time adequate closure of the fetal spina bifida defect.

The purpose of the study is to evaluate the maternal and fetal outcomes of a new technique for the fetoscopic repair of fetal MMC at Texas Children's Hospital Pavilion for Women. The investigators hypothesis is that this minimally invasive technique is feasible, and that this approach will have the same efficacy as open fetal surgery for MMC, but with significantly less maternal-fetal risk. Both mother and baby will benefit from the surgery. The fetus will have a repaired MMC defect, and the mother will not have a uterine incision (hysterotomy). A hysterotomy increases the risk of uterine rupture and requires that all subsequent deliveries are by cesarean section. There may also be a decreased risk of Pre-term Premature Rupture Of Membranes (PPROM) and prematurity when compared with the current open operation. Finally, a vaginal delivery is possible following the fetoscopic fetal surgery if the baby is shown to have a skin covered repair.

Crouch gait/walking, characterized by an 'over-flexed' knee when the leg is supporting body weight, is common in children with diagnoses of cerebral palsy, spina bifida and other incomplete spinal cord injuries. The "Agilik" is a leg exoskeleton device that aims to improve how children with crouch gait walk. In this study the investigators will quantify the improvement that the Agilik facilitates in children with crouch gait in two ways: 1) the difference the Agilik makes when the participants start using it, and 2) any 'training effects' that can be seen in barefoot walking after six sessions of training with the Agilik.

For children who use a power wheelchair, a powered wheelchair standing device (PWSD) may be considered for daily use. A PWSD allows a child to electronically move between sitting and standing and can be driven in either position. Existing published PWSD research in pediatrics is limited to boys with Duchenne muscular dystrophy (DMD).(1, 2) While these studies provide some insights into PWSD use in boys with DMD, they do not reflect PWSD use in children with other conditions. The purpose of this exploratory study is to determine the feasibility of a research protocol exploring use of a PWSD in children who have neurodevelopmental conditions other than DMD.

Performing surgery in utero on fetuses with certain birth defects has led to significant improvements in outcomes after birth; however, many of these infants are born preterm which can decrease the effectiveness of these procedures. The investigators aim to understand the effects of surgery on the maternal and fetal immune system and whether immune activation may be causing some of these infants to be born prematurely.

Urinary continence is still a difficult goal to achieve in neurogenic bladder patients. Conservative treatment of neurogenic urinary incontinence using clean intermittent catheterization in conjunction with pharmacological therapy, notably anticholinergic medication +/- adrenergic agents, may produce continence in a variable number of patients. In other patients, reconstructive surgery of the bladder and/or bladder neck is necessary to achieve urinary continence. Surgical treatment options to increase bladder outlet resistance in patients with neurogenic bladder include injection of bulking agents around the bladder neck, bladder neck reconstruction, fascial sling procedures and Artificial Urinary Sphincter. The Adjustable Continence Therapy system (ACT) or periurethral adjustable balloons are a minimally invasive device consisting of two volume-adjustable balloons implanted periurethrally at the bladder neck as a method of augmenting titration for urethral coaptation. Adjustable means that such system would be adaptable to the individual clinical condition. Originally conceived and developed as a treatment for female stress urinary incontinence, the technique has been then adapted and balloons were globally developed for the use in postprostatectomy incontinence. The published success rate in male after prostatectomy and in women was respectively 56 to 92% and 60 to 83%. The investigators hypothesize that the use of ACT for treating incontinence in children secondary to neurogenic sphincter incontinence could compress the urethra or the bladder neck, acting as an extrinsic occlusive system increasing passive and dynamic urethral and bladder resistance. Goal of the study: To prospectively assess the efficacy and safety of periurethral adjustable balloons in the treatment of neurogenic incontinence in children with spinal dysraphism. Material and methods : A prospective study will be performed at La Timone Enfants hospital and La Nord hospital in Marseille, France. Boys and girls at least at school age (5 or 6 years) with neurogenic incontinence due to outlet issues (low detrusor leak point pressure and low stress leak point pressure) with spinal dysraphism will be recruited. The ACT balloon is an implantable medical device developed and furnished for free by Uromedica (Irvine, CA, USA). The procedure is performed under general anaesthesia using the same implantation technique as published in adult population.

The goal of this clinical trial is to learn about how a participation-based intervention builds capacity of youth with physical disabilities to pursue activities of their choice in the community. The investigators plan to examine in what ways working with a therapist to set up and engage in an 8-week self-chosen community-based activity builds capacity of youth with physical disabilities to pursue a new activity of their choice in the community without the support of a therapist. During this study, participants will be followed for 26 weeks. Youth will work with an occupational therapist (OT). In the first week, the OT will meet with youth to set a community-based leisure goal. Examples of activities could include music, sports, cooking lessons, painting, or photography, in the youth's community. The OT will work with youth to identify and remove barriers. They will also adapt the activity to help youth do the activity for 8 weeks. During this time, the OT will perform site visits to consult and support youths' involvement as needed. (Weeks #1-8) Youth will have a four-week break after completing their first activity. (Weeks #9-12). Then, youth will be asked to choose a second (new) activity. They will try to start this activity for 8 weeks without the OT. (Weeks #13-20) At the end of these 8 weeks, the same therapist will help the youth for 6 weeks if needed to do their second activity. (Weeks #21-26) Youth will be asked to complete the following online: A standard demographic questionnaire (during the first meeting). Rate their perceived performance in the chosen activity once a week. A questionnaire about their daily participation in the community. This will be done at the start and end of the study. A questionnaire about how well they feel they are able to do things. This will be done three times. Share steps they take to participate in the activity. This will be done through a weekly diary entry. In addition, three one-on-one interviews (for about an hour each) will be done remotely (using Microsoft TEAMS) to share their experience pursuing their selected activities. Interviews will be done before starting their second (new) activity, after 8 weeks of pursuing the new activity on their own, and after 6 weeks with OT support. These interviews will be video, and audio recorded and transcribed. This study examines 'real-life' experiences and participation outcomes of youth with physical disabilities after a participation-based capacity-building intervention.

The purpose of the study is to develop a comprehensive program to increase early initiation of self-catheterization in children with Spina Bifida. This will be a prospective open label randomized control trial. Patients and caregivers/guardians in the intervention arm will be enrolled in a comprehensive program that utilizes behavioral economic theory to incentivize initiation of independent CIC. The study population will be patients aged 4 to 12 years old with diagnosis of spina bifida, including meningocele and myelomeningocele, as defined by International Classification of Diseases, 10th revision (ICD10). This study will look at behavioral interventions. There are no additional physical risks to participation in the study. Patient demographics will be reported using descriptive statistics. Study team will use Kaplan-Meier curves to demonstrate and compare the probability of achieving independent CIC over time between the study arms.

Participation in community-based activities is essential to the health and well-being of youth with physical disabilities; yet, it is extremely restricted. Emerging treatment approaches aimed at improving participation have shifted from focusing only on impaired body functions towards the performance of functional meaningful activities within the youth's natural environment. Investigators' initial results from studies in Quebec show that targeting intervention at the activity/participation level can result in improvement of impaired body functions (e.g., balance, attention, anxiety) - important components to address in rehabilitation. Investigators' team aims to continue studying the impact of participation by launching a larger more rigorous study. Investigators have partnered with major organizations providing rehabilitation services for youth as well as key community-based stakeholders including youth, clinicians, and managers, and together investigators plan to further examine whether engaging in an 8-week community-based activity individually chosen by the youth (e.g., sledge hockey, drawing, playing a musical instrument) can lead to a significant improvement in three key body functions: motor, behavioral and emotional. One hundred and fifty youth with physical disabilities living in Quebec and Ontario will participate and engage in an activity of choice. Changes in their body functions (e.g., movement, attention, mood) will be measured multiple times before, during and after engagement in the chosen activity. Findings of this study can guide clinicians, families and policy-makers to select effective approaches that not only promote participation but also facilitate additional motor and mental benefits from a single intervention. Such 'real-world' treatment approaches involving activities of choice can also increase motivation, compliance and reduce burden on the healthcare system and on the youth and families.