Muscle Derived Cell Therapy for Bladder Exstrophy Epispadias Induced Incontinence
Stopped: Due to participants withdrawal prior to anticipated end of study.
United States2 participantsStarted 2017-02-01
Plain-language 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.
Who can participate
Age range2 Years
SexALL
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Inclusion criteria
✓. Group 1 - Males and females at least 2 years of age with:
✓. Group 2 - Males and females greater than 5 years of age with:
✓. Screening labs obtained less than 30 days prior to MDC injection meeting the following criteria:
✓. Parent or legal guardian who is, in the opinion of the investigator, reliable and willing to make themselves and patient available for the duration of the study and are willing to follow study procedures and unit policies.
✓. Parent or legal guardian able to complete and sign the informed consent document.
✓. Negative pregnancy test for sexual active female teenagers. If able to conceive and sexually active, participants must agree to use barrier contraceptives from the time of study enrollment until 6 months after the last MDC injection. Male participants who are able to conceive and are sexually active must agree to use protection as well from the time of study enrollment until 6 months after the last MDC injection.
Exclusion criteria
✕. Urodynamic study demonstrating severe uninhibited bladder contractions.
✕. Severe urethral or bladder neck stricture demonstrated during screening cystoscopy or cystogram
✕. Cystography at the time of screening demonstrating Grade IV vesicoureteral reflux (high-grade reflux with dilation of the renal pelvis and blunting or the fornices) or Grade V vesicoureteral reflux (Grade IV findings plus loss of the papillary impression and ureteral tortuosity).
What they're measuring
1
The ability to inject the MDC product in a safe and tolerable manner in a clinical setting using a descriptive approach.
Timeframe: Post op day 1, day 40, monthly afterwards for 36 months
. Any degree of renal scarring at the time of screening as demonstrated by DMSA or MAG3 renal scintigraphy in the presence of any grade of vesicoureteral reflux (VUR)
✕. Renal ultrasound demonstrating Society of Fetal Urology Grade III hydronephrosis (widely split renal pelvis, renal calices uniformly dilated, no parenchymal thinning) or Grade IV hydronephrosis (Grade III dilation plus parenchymal thinning). See Appendix D.
✕. Previous injection of bulking agents at the level of the bladder neck (bovine collagen or DEFLUX™)
✕. Positive urine culture resistant to preoperative oral antibiotic therapy
✕. Need for chronic or pulse steroids or history of other congenital or acquired condition that results in immunocompromise