Stanmore

Researchers are investigating cavovarus feet deformities, which involve complex changes in multiple planes and present in different forefoot types. The study focuses on adults with Charcot-Marie-Tooth disease, aiming to find a reliable way to identify forefoot morphotypes without using specialized software. This is important because current methods are complex and not easily applied outside specialist centers. Participants will undergo clinical examinations as part of their routine care to assess foot deformities. The study will explore the effectiveness of manual measurements and clinical exams combined with weightbearing CT scans to distinguish between forefoot morphotypes. The research includes both prospective and retrospective elements, examining existing records and new assessments. Participants will be monitored for reproducibility of clinical examination results over 6 months. Data such as clinical findings, imaging, and manual measurements will be collected and analyzed. The study aims to provide practical and reproducible methods for identifying foot deformities in patients with Charcot-Marie-Tooth disease, lasting until the study completion date in late 2027.

This research aims to improve pelvic rehabilitation for adults with chronic, supra-sacral spinal cord injury SCI by evaluating the use of epidural spinal cord stimulation eSCS combined with pelvic floor muscle training. Up to 20 volunteers will participate, all having confirmed neurogenic detrusor overactivity NDO and responsiveness to dorsal genital nerve stimulation. The study focuses on assessing bladder, bowel, sexual function, quality of life, motor function, and spasticity over time. The study includes two main phases. In Phase I, participants will have a Senza implantable pulse generator placed under the skin, with electrodes positioned to target bladder, bowel, and pelvic floor muscles. Mapping sessions will identify effective stimulation settings for home use. Participants will learn pelvic floor muscle exercises to perform alongside eSCS. Phase II involves a 12-week home trial using these stimulation programs and exercises, with regular clinic visits and weekly phone calls for support and progress review. Participants will undergo extensive assessments including urodynamics, anorectal physiology, bladder and bowel diaries, and various questionnaires before and after the intervention, as well as at 3 and 6 months follow-up. They will be offered the choice to keep or have the eSCS device removed after the trial. Researchers will continue to monitor all participants long-term to evaluate changes in bladder capacity and other key outcomes, ensuring comprehensive safety and effectiveness tracking throughout the study period.

Researchers are conducting an international, multicenter, prospective observational registry to study patients with X-Linked Hypophosphatemia XLH. This registry aims to collect detailed data on the natural history, treatment patterns, progression, and long-term outcomes of XLH in both adults and children. The study includes patients regardless of whether they are currently receiving treatment for XLH. Participants will be enrolled in a non-interventional setting without any additional treatments or procedures beyond their standard clinical care. Data will be collected retrospectively and prospectively using a web-based system. Physicians will update patient information during routine visits, with reminders to update data approximately every 12 months. Some sites may also participate in a post-authorization safety study PASS to monitor treatment-related adverse events. Participants will provide informed consent or assent according to age and national guidelines. The study team will collect baseline and ongoing clinical information, including treatment details and adverse events, over a period extending to 10 years. No fixed follow-up schedule is required, but data updates align with patients usual healthcare visits. Researchers will review adverse events related to treatment as a primary outcome measure throughout the study period.

Researchers are conducting a global, prospective, multi-center observational study to gather real-world data on the long-term effectiveness, economic value, and technical performance of Boston Scientifics commercially approved neurostimulation systems for pain management. The study focuses on patients using these neurostimulation devices according to their approved instructions for use in routine clinical practice. Participants will first undergo a neurostimulation trial period using any commercially approved Boston Scientific neurostimulator for pain. Those who experience a positive outcome from this trial may then receive a permanent implant of the neurostimulation system. Treatment customization will be guided by the investigators judgment and routine care practices at each site. During the study, participants will be observed to collect information on clinical outcomes, technical device performance, and economic factors related to neurostimulation therapy. The research team will monitor participants over the long term to assess how well the therapy manages pain and its associated benefits. The study is expected to continue until May 2035, with ongoing follow-up and data collection throughout this period.

Knee cartilage injuries can cause pain and limit movement, affecting daily activities like walking and sports. Since cartilage heals poorly on its own, surgery such as autologous chondrocyte implantation ACI is often needed. This study aims to understand how knee function changes after ACI surgery, helping doctors and patients know what to expect and identify issues that physiotherapy might improve. The research is important because untreated cartilage damage can lead to arthritis, a painful condition later in life. Participants in this study will all undergo ACI surgery, which involves two procedures first, harvesting cartilage cells from the patient, then growing these cells in a lab before implanting them to repair the knees damaged cartilage. The study will take place at seven hospitals, with patients invited to attend two appointments at their hospitalone before surgery and one six months afterto assess knee function. During these visits, researchers will measure knee range of motion using a goniometer and analyze walking patterns using motion capture technology. They will also evaluate balance and walking ability. The studys results will provide detailed information about knee movement and function after ACI surgery, which could help improve patient care and treatment outcomes. The entire participation spans about six months with assessments before and after surgery.

Researchers are evaluating the drug INCB000928 in people diagnosed with fibrodysplasia ossificans progressiva FOP, a rare condition that causes bone to form outside the skeleton. This Phase 2 study is designed to assess how well INCB000928 works, its safety, tolerability, and how the body processes the drug. The study includes a placebo comparison and aims to provide important information on treatment effects in participants with FOP. Participants aged 2 years and older are divided into three age cohorts and randomly assigned to receive either INCB000928 or a placebo daily by mouth for a 24-week double-blind period. Those who complete this initial phase may continue in an open-label extension where all participants receive INCB000928 for an additional 292 weeks. Imaging scans excluding the head are used to monitor changes in bone formation during the study. During the trial, participants will have assessments to detect new bone lesions, measure their volume, and track flare-ups related to FOP at regular intervals up to week 48 and beyond. The study also monitors treatment side effects and the drugs pharmacokinetics at multiple time points. Overall participation can last several years due to the long extension phase, with ongoing safety and efficacy evaluations throughout.