An open-label, multicentre study of augmentation cystoplasty using an autologous Neo-Bladder construct in subjects with spina bifida.

• Conditions: eurogenic bladder secondary to spina bifida; MedDRA version: 9.1Level: LLTClassification code 10029279Term: Neurogenic bladder; MedDRA version: 9.1Level: LLTClassification code 10041524Term: Spina bifida

• Registration Number: EUCTR2007-006433-13-GB
• Lead Sponsor: Tengion Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2008-03-07
• Last Update Posted: 19 March 2012

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 10

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 < 18 years of age as appropriate or warranted.

3. Ability of subject and/or care provider to be successfully trained in clean intermittent catheterization and bladder cycling

4. Uilization of maximally-tolerated dose and regimen of medical therapy (e.g. anticholinergics) or failure to tolerate /contraindication to such agents

5. Medical need for bladder augmentation, as defined by the presence of:
a. Decreased and inadequate bladder compliance with a bladder pressure =40 cmH20 at or below estimated bladder capacity for age, OR
b. New-onset of upper urinary tract changes (hydronephrosis, vesicoureteral reflux) in the last 12 months

Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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 (< 12 months) urological or intraperitoneal surgery or device implantation; recent (<6 month neurological surgery-brain or spine)

3. Any prior bladder augmentation procedure

4. Requirement of concomitant urological surgical procedures in addition to augmentation (e.g., ureteral reimplantation). Minor surgical correction for bladder neck incompetence (i.e., fascial sling) is permitted.

5. Known or suspected limitation to obtaining omentum for implantation procedure (e.g., extensive intraperitoneal adhesions)

6. Any contraindication to general anaesthesia

7. Any contraindication or known anaphylactic or severe systemic reaction to either human blood products or materials of bovine origin

8. Immunocompromised subjects or subjects receiving immunosuppressive agents (inhaled corticosteroids and chronic low-dose corticosteroids [=0.25 mg/kg prednisone or equivalent per day] are permitted). Brief pulsed corticosteroids for intermittent symptoms (e.g., asthma) are permitted.

9. Known history of hypersensitivity to aminoglycosides or fluoroquinolones.

10. Use of any investigational product within 3 months

11. Prior participation in the study

12. Unwillingness, inability, or unlikely compliance with study related procedures

13. Any circumstance in which the investigator deems participation in the study is not in the subject’s best interest

14. Subjects with an ALT or AST value >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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified