Trial Outcomes & Findings for Augmentation Cystoplasty Using an Autologous Neo-Bladder (NCT NCT00419120)

NCT ID: NCT00419120

Last Updated: 2015-11-30

Results Overview

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

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

10 participants

Primary outcome timeframe

12 months

Results posted on

2015-11-30

Participant Flow

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).

This was an open label single arm study.

Participant milestones

Participant milestones
Measure	Implanted Patients Patients implanted with Tengion Neo-Bladder Augment
Primary Study Period STARTED	10
Primary Study Period COMPLETED	10
Primary Study Period NOT COMPLETED	0
Long Term Follow up Period STARTED	10
Long Term Follow up Period COMPLETED	0
Long Term Follow up Period NOT COMPLETED	10

Reasons for withdrawal

Reasons for withdrawal
Measure	Implanted Patients Patients implanted with Tengion Neo-Bladder Augment
Long Term Follow up Period study ongoing	10

Baseline Characteristics

Augmentation Cystoplasty Using an Autologous Neo-Bladder

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Implanted Patients n=10 Participants Patients implanted with Tengion Neo-Bladder Augment
Age, Categorical <=18 years	10 Participants n=93 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=93 Participants
Age, Categorical >=65 years	0 Participants n=93 Participants
Age, Continuous	8.2 years STANDARD_DEVIATION 4.29 • n=93 Participants
Sex: Female, Male Female	6 Participants n=93 Participants
Sex: Female, Male Male	4 Participants n=93 Participants
Region of Enrollment United States	10 participants n=93 Participants

PRIMARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Implanted Patients n=10 Participants Patients implanted with Tengion Neo-Bladder Augment
Number of Responders as Assessed by Compliance	6 participants

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Implanted Patients n=10 Participants Patients implanted with Tengion Neo-Bladder Augment
Overall Safety Profile - Number of Participants Experiencing an Adverse Event	10 participants

Adverse Events

Safety Population

Serious events: 6 serious events

Other events: 11 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Safety Population n=11 participants at risk All patients undergoing screeing and meeting inclusion/exclusion criteria
Metabolism and nutrition disorders Dehydration	18.2% 2/11 • Number of events 2 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
General disorders Pyrexia	18.2% 2/11 • Number of events 2 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Injury, poisoning and procedural complications Ventriculoperitoneal Shunt Malfunction	27.3% 3/11 • Number of events 3 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Infections and infestations Urinary Tract Infection	18.2% 2/11 • Number of events 3 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Gastrointestinal disorders Diarrhoea	9.1% 1/11 • Number of events 1 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Gastrointestinal disorders Faecaloma	9.1% 1/11 • Number of events 1 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Infections and infestations Pyelonephritis	9.1% 1/11 • Number of events 1 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Injury, poisoning and procedural complications Complication of Device Insertion	9.1% 1/11 • Number of events 1 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Injury, poisoning and procedural complications Post Procedural Urine Leak	9.1% 1/11 • Number of events 1 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.

Other adverse events

Other adverse events
Measure	Safety Population n=11 participants at risk All patients undergoing screeing and meeting inclusion/exclusion criteria
Blood and lymphatic system disorders Anemia	9.1% 1/11 • Number of events 1 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Gastrointestinal disorders Nausea	45.5% 5/11 • Number of events 5 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Gastrointestinal disorders Vomiting	45.5% 5/11 • Number of events 5 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Gastrointestinal disorders Diarrhoea	9.1% 1/11 • Number of events 1 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Gastrointestinal disorders Abdominal Pain	9.1% 1/11 • Number of events 1 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Gastrointestinal disorders Flatulence	9.1% 1/11 • Number of events 1 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Gastrointestinal disorders Ileus	9.1% 1/11 • Number of events 1 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
General disorders Pyrexia	45.5% 5/11 • Number of events 5 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
General disorders Implant Site Inflammation	9.1% 1/11 • Number of events 1 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
General disorders Pain	9.1% 1/11 • Number of events 2 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Infections and infestations Urinary Tract Infection	90.9% 10/11 • Number of events 37 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Infections and infestations Nasopharyngitis	27.3% 3/11 • Number of events 4 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Infections and infestations Clostridial Infection	18.2% 2/11 • Number of events 2 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Infections and infestations Bronchitis	9.1% 1/11 • Number of events 1 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Infections and infestations Ear Infection	9.1% 1/11 • Number of events 1 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Infections and infestations Enterococcal Infection	9.1% 1/11 • Number of events 1 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Infections and infestations Molluscum Contagiosum	9.1% 1/11 • Number of events 1 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Infections and infestations Otitis Media	9.1% 1/11 • Number of events 1 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Infections and infestations Pharyngitis Streptococcal	9.1% 1/11 • Number of events 1 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Infections and infestations Sinusitis	9.1% 1/11 • Number of events 1 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Infections and infestations Skin infection	9.1% 1/11 • Number of events 1 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Injury, poisoning and procedural complications Skin Laceration	9.1% 1/11 • Number of events 1 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Injury, poisoning and procedural complications Thermal Burn	9.1% 1/11 • Number of events 1 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Investigations Hemoglobin Decreased	9.1% 1/11 • Number of events 1 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Metabolism and nutrition disorders Hypervolaemia	9.1% 1/11 • Number of events 1 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Metabolism and nutrition disorders hypokalaemia	9.1% 1/11 • Number of events 1 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Musculoskeletal and connective tissue disorders back pain	9.1% 1/11 • Number of events 1 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Musculoskeletal and connective tissue disorders Musculoskeletal Pain	9.1% 1/11 • Number of events 1 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Musculoskeletal and connective tissue disorders Neck Pain	9.1% 1/11 • Number of events 1 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Nervous system disorders Headache	18.2% 2/11 • Number of events 4 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Nervous system disorders Convulsion	9.1% 1/11 • Number of events 1 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Nervous system disorders Dizziness	9.1% 1/11 • Number of events 2 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Psychiatric disorders Attention Deficit/Hyperactivity Disorder	9.1% 1/11 • Number of events 1 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Psychiatric disorders Hallucination	9.1% 1/11 • Number of events 1 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Renal and urinary disorders Hydronephrosis	9.1% 1/11 • Number of events 1 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Respiratory, thoracic and mediastinal disorders Cough	18.2% 2/11 • Number of events 2 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Respiratory, thoracic and mediastinal disorders Atelectasis	9.1% 1/11 • Number of events 1 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Respiratory, thoracic and mediastinal disorders Pharyngolaryngeal Pain	9.1% 1/11 • Number of events 1 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Skin and subcutaneous tissue disorders Pruritus	18.2% 2/11 • Number of events 2 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Skin and subcutaneous tissue disorders Rash	18.2% 2/11 • Number of events 2 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Skin and subcutaneous tissue disorders Blister	9.1% 1/11 • Number of events 1 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Skin and subcutaneous tissue disorders Decubitus Ulcer	9.1% 1/11 • Number of events 1 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.
Vascular disorders Hypertension	9.1% 1/11 • Number of events 1 Adverse events are reported for 11 subjects who were enrolled in the study. One patient withdrew prior to implantation with the neo-bladder construct and is included in the safety population but not in the all implanted population used for efficacy analysis.

Additional Information

Sunita Sheth, MD - Chief Medical Officer

Tengion, Inc.

Phone: 267-960-4805

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The investigator is free to individually communicate, orally present or publish in scientific journals or other scholarly media the study information at the conclusion of the study without the prior approval of the sponsor provided that 1)the results of the study in its entirely have been publically disclosed by or with the consent of the sponsor 2)18 months after the conclusion of the study at all sites whichever is first to occur.
Publication restrictions are in place

Restriction type: OTHER