Neurogenic bladder secondary to spina bifida MedDRA version: 9.1 Level: LLT Classification code 10029279 Term: Neurogenic bladder MedDRA version: 9.1 Level: LLT Classification code 10041524 Term: Spina bifida
Conditions
Interventions
Product Name: Autologous Neo-Bladder Construct
Pharmaceutical Form: Implant
INN or Proposed INN: Autologous urothelial and smooth muscle cells
Sponsors
Tengion Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Male and female subjects between the ages of 3 and 21 years of age with myelodysplasia (spina bifida) 2. Willing and able to give signed informed consent, or have a legally authorized representative who is willing and able to give consent. Informed assent will be required for children =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Female subjects who are pregnant, lactating (breast feeding) or planning a pregnancy during the course of the study, or who are of child bearing potential and not using a highly effective method of birth control (including sexual abstinence). A highly effective method of birth control is defined as one that results in a low failure rate (i.e., less than 1 percent per year) when used consistently and correctly, such as implants, injectables, combined oral contraceptives, some intrauterine devices (IUDs), sexual abstinence, or a vasectomised partner. Subjects must be willing to continue to use one of these methods throughout the duration of the study. 2. Recent (3 times the upper limit of normal 15. Subjects with an albumin value <3.0 g/dL 16. Subjects with a history of an anaphylactic or a severe systemic reaction to the biodegradable polymers glycolic acid (PGA) and lactic-co-glycolic acid (PLGA) 17. Subjects with acute or chronic abdominal skin infections and/or acute or chronic inflammatory bowel disease 18. Subjects with uncontrolled diabetes, unstable cardiac and/or pulmonary disorders, or bleeding disorders 19. Subjects who have received Botulinum Toxin A injections into the bladder within the previous 12 months.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The objective of this study is to demonstrate that augmentation cystoplasty using an autologous neo-bladder construct will significantly increase bladder compliance in subjects with neurogenic bladder secondary to spina bifida (myelodysplasia) that is refractory to medical treatment. Primary outcome measure • Percent change from baseline in bladder compliance as assessed by standardized urodynamic measurements at 12 months;Secondary Objective: Secondary Outcome Measures in the Primary Analysis Phase Safety • Overall safety assessment Efficacy • Percent change from baseline in bladder capacity as assessed by standardized urodynamic measurements at 12 months • Percent change from baseline in bladder compliance and capacity as assessed by standardized urodynamic measurements at 6, 9, 36 and 60 months • End filling pressure at age-related nomogram-derived bladder capacity at baseline, 6, 9, 12, 30 and 60 months ;Primary end point(s): Percent change from baseline in bladder compliance as assessed by standardized urodynamic measurements at 12 months | — |
Countries
United Kingdom
Outcome results
None listed