Augmentation Cystoplasty Using an Autologous Neo-Bladder

An Open-Label Multicenter Study of Augmentation Cystoplasty Using an Autologous Neo-Bladder Construct in Subjects With Spina Bifida

Subjects with neurogenic bladder secondary to spina bifida refractory to medical treatment will be enrolled. The hypothesis is that augmentation cystoplasty using an autologous neo-bladder construct will significantly increase bladder compliance.

Study Overview

• Status: Terminated
• Conditions: Neurogenic Bladder
• Intervention / Treatment: Biological: Autologous neo bladder construct

Detailed Description

Subjects with neurogenic bladder secondary to spina bifida (myelodysplasia) that is refractory to medical treatment and require augmentation cystoplasty will be enrolled. The hypothesis is that augmentation cystoplasty using an autologous neo-bladder construct will significantly increase bladder compliance.

• Study Type: Interventional
• Enrollment (Actual): 10
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Boston Childrens Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 3 years to 21 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• subjects with neurogenic bladders secondary to myleodysplasia

Exclusion Criteria:

• prior augment procedures
• recent urologic surgery
• requires concomitant urologic intervention

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Neo-bladder construction; Surgical implantation of autologous neo-bladder construct	Biological: Autologous neo bladder construct; augmentation cystoplasty with autologous neo-bladder construct

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Responders as Assessed by Compliance	Efficacy of the autologous neo-bladder construct as assessed by a responder analysis (comparing each patient's baseline with 12 month data). Improvement in compliance (i.e. improved bladder pressure/volume relationship) was measured by urodynamics at predetermined bladder pressure points. This was coupled with an assessment of clinical benefit and used to determine responders versus non-responders	12 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall Safety Profile - Number of Participants Experiencing an Adverse Event	clinical evaluation of adverse events, laboratory parameters and urinary imaging to assess any safety issues emerging from this technology and to allow a comparison of a safety profile with standard of care enterocystoplasty. Please refer to the Adverse Event section for detailed information.	periodically within first 12 months as well as during long term follow up out to 5 years

Collaborators and Investigators

• Sponsor: Tengion
• Investigators: Study Director: Sunita Sheth, MD, Tengion, Inc.

Study record dates

Study Major Dates

• Study Start: December 1, 2006
• Primary Completion (Actual): October 1, 2008
• Study Completion (Actual): April 1, 2011

Study Registration Dates

• First Submitted: January 4, 2007
• First Submitted That Met QC Criteria: January 4, 2007
• First Posted (Estimate): January 8, 2007

Study Record Updates

• Last Update Posted (Estimate): November 30, 2015
• Last Update Submitted That Met QC Criteria: November 2, 2015
• Last Verified: November 1, 2015

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Urologic Diseases; Urinary Bladder Diseases; Neurologic Manifestations; Urinary Bladder, Neurogenic
• Other Study ID Numbers: TNG-CL003