Urinary Incontinence, Stress
Conditions
Keywords
Urinary Incontinence, Stress, Tissue Therapy (Cell Therapy), Transplantation, Autologous
Brief summary
This is a clinical trial to study the safety and effectiveness of Autologous Muscle Derived Cells for Urinary Sphincter Repair (AMDC-USR) for the treatment of female stress urinary incontinence (SUI).
Detailed description
Study comparing intrasphincteric injection of AMDC-USR with placebo. Subjects unblinded after 12 months visits, but followed for 2 years. Subjects randomized to placebo could elect to receive open-label AMDC-USR after completing 12 months visit.
Interventions
BIOLOGICALPlacebo
Placebo treatment
BIOLOGICALAMDC-USR
AMDC-USR Treatment
Sponsors
Cook MyoSite
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, Investigator)
Eligibility
Sex/Gender
FEMALE
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Female patient has primary symptoms of SUI, as confirmed by patient medical history and clinical symptoms, including a focused incontinence evaluation.
Exclusion criteria
* Patient has symptoms of pure urge incontinence as confirmed by basic evaluation of etiology from a patient medical history, including a focused incontinence history. * Patient has symptoms of mixed urinary incontinence where urge incontinence is the predominant factor. * Patient has had stress urinary incontinence symptoms less than 6 months prior to signing the informed consent. * Patient has not previously attempted conservative treatment for at least 1 month prior to signing the informed consent. (Examples of conservative treatment include behavior modifications, bladder exercises, biofeedback, etc.) * Patient has more than 2 episode of awakening to void during normal sleeping hours. * Patient cannot be maintained on a stable dose and/or frequency of medication (including diuretics) known to affect lower urinary tract function, including but not limited to, anticholinergics, tricyclic antidepressants or alpha-adrenergic blockers, for at least 2 weeks prior to randomization or is likely to change during the course of the study. * Patient is pregnant, lactating, or plans to become pregnant during the course of the study. * Patient refuses to provide written informed consent. * Patient is not at least 18 years of age. * Patient is not available for the follow-up evaluations as required by the protocol.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants With Response (Based on Stress IEF, or In-office Pad Weight Test, or 24-hour Pad Weight Test) at 12 Months | 12 months | A composite primary endpoint of responder rate was used, where a subject was considered a responder if she had ≥ 50% reduction from baseline in stress Incontinence Episode Frequency (stress IEF; reported stress leaks from 3-day diary) or ≥ 50% reduction in leakage from baseline as determined by either the in-office pad weight test or the 24-hour pad weight test. |
Countries
Canada, Germany, United Kingdom
Participant flow
Recruitment details
Subjects were screened and enrolled at 10 sites globally; 8 sites in Canada, 1 site in Germany, and 1 site in United Kingdom.
Pre-assignment details
227 subjects consented to study participation and were screened for eligibility, of whom 150 subjects were enrolled (underwent biopsy procedure) and 143 subjects underwent at least 1 study treatment AMDC-USR (93 subjects) or placebo (50 subjects). Analysis population is based on 143 subjects that underwent at least 1 study treatment.
Participants by arm
| Arm | Count |
|---|---|
| AMDC-USR Subjects received 1 or 2 treatments of 150 million AMDC-USR delivered via transurethral intrasphincteric injection. | 93 |
| Placebo Subjects received 1 or 2 treatments of placebo delivered via transurethral intrasphincteric injection. | 50 |
| Total | 143 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Protocol Violation | 0 | 3 |
| Overall Study | Withdrawal by Subject | 0 | 1 |
Baseline characteristics
| Characteristic | AMDC-USR | Placebo | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical >=65 years | 11 Participants | 7 Participants | 18 Participants |
| Age, Categorical Between 18 and 65 years | 82 Participants | 43 Participants | 125 Participants |
| Age, Continuous | 51.4 Years STANDARD_DEVIATION 11.1 | 51.7 Years STANDARD_DEVIATION 9.9 | 51.5 Years STANDARD_DEVIATION 10.6 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 1 Participants | 1 Participants | 2 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 2 Participants | 2 Participants |
| Race (NIH/OMB) More than one race | 1 Participants | 0 Participants | 1 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 91 Participants | 47 Participants | 138 Participants |
| Region of Enrollment Canada | 91 Participants | 49 Participants | 140 Participants |
| Region of Enrollment Germany | 1 Participants | 1 Participants | 2 Participants |
| Region of Enrollment United Kingdom | 1 Participants | 0 Participants | 1 Participants |
| Sex: Female, Male Female | 93 Participants | 50 Participants | 143 Participants |
| Sex: Female, Male Male | 0 Participants | 0 Participants | 0 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 93 | 0 / 50 |
| other Total, other adverse events | 32 / 93 | 13 / 50 |
| serious Total, serious adverse events | 0 / 93 | 0 / 50 |
Outcome results
Primary
Percentage of Participants With Response (Based on Stress IEF, or In-office Pad Weight Test, or 24-hour Pad Weight Test) at 12 Months
A composite primary endpoint of responder rate was used, where a subject was considered a responder if she had ≥ 50% reduction from baseline in stress Incontinence Episode Frequency (stress IEF; reported stress leaks from 3-day diary) or ≥ 50% reduction in leakage from baseline as determined by either the in-office pad weight test or the 24-hour pad weight test.
Time frame: 12 months
Population: Analysis population is based on subjects that received at least 1 treatment of AMDC-USR or placebo at the 12 months follow-up..
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| AMDC-USR | Percentage of Participants With Response (Based on Stress IEF, or In-office Pad Weight Test, or 24-hour Pad Weight Test) at 12 Months | 82 Percentage of subjects |
| Placebo | Percentage of Participants With Response (Based on Stress IEF, or In-office Pad Weight Test, or 24-hour Pad Weight Test) at 12 Months | 90 Percentage of subjects |