Clinical Research Directory

Repurposing 5-Azacytidine for the Treatment of Muscle Contractures in Children With Cerebral Palsy

In this controlled dose-escalation study, we will study the initial safety, biological properties, and potential efficacy of 5-azacytidine (AZA). Our overarching aspiration is for AZA to evolve into an approved pharmacological treatment, fostering muscle growth and enhancing body movement, ultimately contributing to an improved quality of life in children with CP. The main questions this study aims to answer are: 1. What is the optimal dose of AZA injection that can be used safely in children with CP? 2. Can the optimal safe dose of AZA improve the function of muscle-generating stem cells in children with CP? Each participant will have up to five research visits over the course of the study duration, in which they will participate in: blood draws, pregnancy test(s) (if applicable), medical assessments, and a muscle biopsy during a surgery for muscle contractures. Researchers will compare participants with four different dosages of AZA injections to those with four different dosages of placebo injections. A placebo is a look-alike substance that contains no active drug. They will see if a single injection of AZA at a standard concentration currently approved by the FDA to treat myelodysplastic syndromes, can also safely improve muscle growth and function in children with CP.

Effectiveness of Rehabilitation Protocol in Patients With Shortened Posterior Leg Muscles

The goal of this clinical study is to develop a specialized rehabilitation exercise protocol designed for patients diagnosed with foot pathologies and calf muscle shortening, regardless of their choice to undergo minimally invasive ultrasound-guided surgery. The primary focus is to assess disparities in outcomes, particularly in the recovery of ankle mobility degrees. Additionally, for patients opting for minimally invasive ultrasound-guided surgery, the study aims to evaluate the resumption of both sporting activities and daily routines using the aforementioned specific rehabilitation protocol. The primary questions this study aims to answer are: * How does the proposed rehabilitation protocol impact ankle mobility recovery for patients with foot pathologies and calf muscle shortening? * What are the differences observed in the return to sporting activities and daily life among patients undergoing minimally invasive ultrasound-guided surgery, following the prescribed protocol? Participants enrolled in this study will be engaged in: Undertaking the specified rehabilitation exercises tailored for foot pathologies and calf muscle shortening. Those opting for minimally invasive ultrasound-guided surgery will follow the same rehabilitation protocol post-surgery to assess its impact on their return to normal activities. If there exists a comparison group: Researchers will compare participants who undergo minimally invasive ultrasound-guided surgery against those who choose other treatment options to discern any differential effects on ankle mobility recovery and resumption of activities.

Percutaneous Needle Fasciotomy +/- Corticosteroid Injection for Dupuytren's Contracture

Comparing percutaneous needle fasciotomy +/- corticosteroid injection for Dupuytren's contracture affecting metacarpophalangeal joints. A clinician-initiated, multicenter, randomized controlled trial.