Neurogenic Bladder
Conditions
Brief summary
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.
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.
Interventions
BIOLOGICALAutologous neo bladder construct
augmentation cystoplasty with autologous neo-bladder construct
Sponsors
Tengion
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
3 Years to 21 Years
Healthy volunteers
No
Inclusion criteria
* subjects with neurogenic bladders secondary to myleodysplasia
Exclusion criteria
* prior augment procedures * recent urologic surgery * requires concomitant urologic intervention
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Responders as Assessed by Compliance | 12 months | 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 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Overall Safety Profile - Number of Participants Experiencing an Adverse Event | periodically within first 12 months as well as during long term follow up out to 5 years | 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. |
Countries
United States
Participant flow
Recruitment details
11 patients were recruited between Dec 2006 and July 2007. Patients were recruited from 4 academic centers across the US. One patient withdrew from the study prior to implantation of the Neo-bladder construct and is included in the safety analysis, but not in the efficacy analysis (all implanted population).
Pre-assignment details
This was an open label single arm study.
Participants by arm
| Arm | Count |
|---|---|
| Implanted Patients Patients implanted with Tengion Neo-Bladder Augment | 10 |
| Total | 10 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Long Term Follow up Period | study ongoing | 10 |
Baseline characteristics
| Characteristic | Implanted Patients |
|---|---|
| Age, Categorical <=18 years | 10 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants |
| Age, Continuous | 8.2 years STANDARD_DEVIATION 4.29 |
| Region of Enrollment United States | 10 participants |
| Sex: Female, Male Female | 6 Participants |
| Sex: Female, Male Male | 4 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 11 / 11 |
| serious Total, serious adverse events | 6 / 11 |
Outcome results
Primary
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
Time frame: 12 months
Population: The analysis population of the primary outcome measure is the all implanted population (Intent to Treat - ITT). There was no imputation of analysis measures.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Implanted Patients | Number of Responders as Assessed by Compliance | 6 participants |
Secondary
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.
Time frame: periodically within first 12 months as well as during long term follow up out to 5 years
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Implanted Patients | Overall Safety Profile - Number of Participants Experiencing an Adverse Event | 10 participants |