Clinical Trial: Muscle Derived Cell Therapy for Bladder Exstrophy Epispadias Induced Incontinence

Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Interventional

Official Title: A Phase I Clinical Protocol to Study the Safety and Tolerability of Endoscopic Injection of Autologous Muscle Derived Cells (MDC) in Children With Exstrophy-epispadias Complex

Brief Summary: The aim of this study is to study safety, tolerability and efficacy of muscle derived cell (MDC) therapy in children with bladder exstrophy epispadias induced urinary incontinence.

Detailed Summary:

Bladder exstrophy patients who have undergone primary bladder closure, but have a bladder capacity too low for bladder neck reconstruction (Group 1), have limited additional surgical options that permits urethral voiding and urine storage within a native bladder. Previous studies have demonstrated a positive correlation between bladder capacity and success of bladder neck reconstruction. Likewise, patients who have undergone bladder neck reconstruction but continue to have urinary incontinence (Group 2) are also faced with limited options. Often, both groups are considered for augmentation cystoplasty with closure of the bladder neck requiring intermittent catheter voiding through a surgically constructed continent catheterizable channel. Such major reconstruction has significant associated short and long-term morbidities. Endoscopic injection of MDCs for the treatment of urinary sphincter insufficiency is a potential alternative therapy for these patients. By increasing the outflow resistance and rhabdosphincter contractility, MDC injection may permit more efficient bladder cycling and bladder expansion in Group 1 patients allowing them to proceed on to bladder neck reconstruction. This same increase in resistance and sphincter contractility may allow Group 2 patients to attain urinary continence and avoid any further reconstructive surgical procedures.

Eligible and consented patients would undergo rectus muscle biopsy and immediately transferred to the Cell Therapy Lab for tissue processing and MDC expansion.The MDC expansion process takes approximately 21 days after which cells are harvest and cryopreserved for future injection.Each vial will be filled with cells at a concentration of approximately 2.0 x 107 cells/ml. At the time of planned MDC injection, enrolled patients will return for cystoscopy under anesthesia. At that time, aliquots of MDCs will be removed from
Sponsor: Johns Hopkins University

Current Primary Outcome: The ability to inject the MDC product in a safe and tolerable manner in a clinical setting using a descriptive approach. [ Time Frame: Post op day 1, day 40, monthly afterwards for 36 months ]

Original Primary Outcome: Same as current

Current Secondary Outcome: changes in urinary incontinence levels and bladder growth in enrolled participants after undergoing autologous MDC injection using cystograms, urodynamic studies, 24-hour pad weight test, continence score, and maximum day & nighttime dry interval. [ Time Frame: Monthly voiding diary. All other parameters measure at baseline and every 6 months for 36 months. ]

Original Secondary Outcome: Same as current

Information By: Johns Hopkins University

Dates:
Date Received: November 10, 2009
Date Started: July 2013
Date Completion: June 2017
Last Updated: July 1, 2013
Last Verified: July 2013