Trial Outcomes & Findings for Autologous Muscle Derived Cells Female Stress Urinary Incontinence Clinical Study (NCT NCT01382602)
NCT ID: NCT01382602
Last Updated: 2018-05-17
Results Overview
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.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
150 participants
Primary outcome timeframe
12 months
Results posted on
2018-05-17
Participant Flow
Subjects were screened and enrolled at 10 sites globally; 8 sites in Canada, 1 site in Germany, and 1 site in United Kingdom.
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.
Participant milestones
| Measure |
AMDC-USR
Subjects received 1 or 2 treatments of 150 million AMDC-USR delivered via transurethral intrasphincteric injection. After completing 12 months follow-up subjects were unblinded. Subjects were followed for 2 years after initial AMDC-USR treatment. Analysis population is based on subjects that received at least 1 treatment of AMDC-USR at the 12 months follow-up.
|
Placebo
Subjects received 1 or 2 treatments of placebo delivered via transurethral intrasphincteric injection. After completing 12 months follow-up, subjects were unblinded and could elect to receive open-label AMDC-USR treatment. Subjects that received open-label AMDC-USR treatment were followed for 2 years after initial placebo treatment. Analysis population is based on subjects that received at least 1 treatment of placebo at the 12 months follow-up.
|
|---|---|---|
|
Overall Study
STARTED
|
93
|
50
|
|
Overall Study
COMPLETED
|
93
|
46
|
|
Overall Study
NOT COMPLETED
|
0
|
4
|
Reasons for withdrawal
| Measure |
AMDC-USR
Subjects received 1 or 2 treatments of 150 million AMDC-USR delivered via transurethral intrasphincteric injection. After completing 12 months follow-up subjects were unblinded. Subjects were followed for 2 years after initial AMDC-USR treatment. Analysis population is based on subjects that received at least 1 treatment of AMDC-USR at the 12 months follow-up.
|
Placebo
Subjects received 1 or 2 treatments of placebo delivered via transurethral intrasphincteric injection. After completing 12 months follow-up, subjects were unblinded and could elect to receive open-label AMDC-USR treatment. Subjects that received open-label AMDC-USR treatment were followed for 2 years after initial placebo treatment. Analysis population is based on subjects that received at least 1 treatment of placebo at the 12 months follow-up.
|
|---|---|---|
|
Overall Study
Protocol Violation
|
0
|
3
|
|
Overall Study
Withdrawal by Subject
|
0
|
1
|
Baseline Characteristics
Autologous Muscle Derived Cells Female Stress Urinary Incontinence Clinical Study
Baseline characteristics by cohort
| Measure |
AMDC-USR
n=93 Participants
Subjects received 1 or 2 treatments of 150 million AMDC-USR delivered via transurethral intrasphincteric injection.
|
Placebo
n=50 Participants
Subjects received 1 or 2 treatments of placebo delivered via transurethral intrasphincteric injection.
|
Total
n=143 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
82 Participants
n=5 Participants
|
43 Participants
n=7 Participants
|
125 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
11 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
18 Participants
n=5 Participants
|
|
Age, Continuous
|
51.4 Years
STANDARD_DEVIATION 11.1 • n=5 Participants
|
51.7 Years
STANDARD_DEVIATION 9.9 • n=7 Participants
|
51.5 Years
STANDARD_DEVIATION 10.6 • n=5 Participants
|
|
Sex: Female, Male
Female
|
93 Participants
n=5 Participants
|
50 Participants
n=7 Participants
|
143 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
91 Participants
n=5 Participants
|
47 Participants
n=7 Participants
|
138 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
Canada
|
91 Participants
n=5 Participants
|
49 Participants
n=7 Participants
|
140 Participants
n=5 Participants
|
|
Region of Enrollment
United Kingdom
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Region of Enrollment
Germany
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 12 monthsPopulation: Analysis population is based on subjects that received at least 1 treatment of AMDC-USR or placebo at the 12 months follow-up..
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.
Outcome measures
| Measure |
AMDC-USR
n=91 Participants
Subjects received 1 or 2 treatments of 150 million AMDC-USR delivered via transurethral intrasphincteric injection.
|
Placebo
n=50 Participants
Subjects received 1 or 2 treatments of placebo delivered via transurethral intrasphincteric injection.
|
|---|---|---|
|
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
Interval 73.0 to 90.0
|
90 Percentage of subjects
Interval 78.0 to 97.0
|
Adverse Events
AMDC-USR
Serious events: 0 serious events
Other events: 32 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 13 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
AMDC-USR
n=93 participants at risk
Subjects received 1 or 2 treatments of 150 million AMDC-USR delivered via transurethral intrasphincteric injection. Analysis population is based on subjects that underwent at least 1 treatment of AMDC-USR, at the 12 month follow-up.
|
Placebo
n=50 participants at risk
Subjects received 1 or 2 treatments of placebo delivered via transurethral intrasphincteric injection. Analysis population is based on subjects that underwent at least 1 treatment of placebo, at the 12 month follow-up.
|
|---|---|---|
|
Infections and infestations
Urinary tract infection
|
12.9%
12/93 • Adverse events were monitored from consent through study exit. Reporting groups are based on adverse events that occurred between initial blinded injection treatment and unblinding of subjects after 12-month follow-up.
Adverse events that occurred between initial blinded injection and unblinding of subjects after 12-month follow-up were compared between AMDC-USR and Placebo groups.
|
10.0%
5/50 • Adverse events were monitored from consent through study exit. Reporting groups are based on adverse events that occurred between initial blinded injection treatment and unblinding of subjects after 12-month follow-up.
Adverse events that occurred between initial blinded injection and unblinding of subjects after 12-month follow-up were compared between AMDC-USR and Placebo groups.
|
|
Renal and urinary disorders
Dysuria
|
5.4%
5/93 • Adverse events were monitored from consent through study exit. Reporting groups are based on adverse events that occurred between initial blinded injection treatment and unblinding of subjects after 12-month follow-up.
Adverse events that occurred between initial blinded injection and unblinding of subjects after 12-month follow-up were compared between AMDC-USR and Placebo groups.
|
6.0%
3/50 • Adverse events were monitored from consent through study exit. Reporting groups are based on adverse events that occurred between initial blinded injection treatment and unblinding of subjects after 12-month follow-up.
Adverse events that occurred between initial blinded injection and unblinding of subjects after 12-month follow-up were compared between AMDC-USR and Placebo groups.
|
|
Renal and urinary disorders
Pollakiuria
|
2.2%
2/93 • Adverse events were monitored from consent through study exit. Reporting groups are based on adverse events that occurred between initial blinded injection treatment and unblinding of subjects after 12-month follow-up.
Adverse events that occurred between initial blinded injection and unblinding of subjects after 12-month follow-up were compared between AMDC-USR and Placebo groups.
|
8.0%
4/50 • Adverse events were monitored from consent through study exit. Reporting groups are based on adverse events that occurred between initial blinded injection treatment and unblinding of subjects after 12-month follow-up.
Adverse events that occurred between initial blinded injection and unblinding of subjects after 12-month follow-up were compared between AMDC-USR and Placebo groups.
|
|
Gastrointestinal disorders
Nausea
|
8.6%
8/93 • Adverse events were monitored from consent through study exit. Reporting groups are based on adverse events that occurred between initial blinded injection treatment and unblinding of subjects after 12-month follow-up.
Adverse events that occurred between initial blinded injection and unblinding of subjects after 12-month follow-up were compared between AMDC-USR and Placebo groups.
|
0.00%
0/50 • Adverse events were monitored from consent through study exit. Reporting groups are based on adverse events that occurred between initial blinded injection treatment and unblinding of subjects after 12-month follow-up.
Adverse events that occurred between initial blinded injection and unblinding of subjects after 12-month follow-up were compared between AMDC-USR and Placebo groups.
|
|
General disorders
Injection site pain
|
5.4%
5/93 • Adverse events were monitored from consent through study exit. Reporting groups are based on adverse events that occurred between initial blinded injection treatment and unblinding of subjects after 12-month follow-up.
Adverse events that occurred between initial blinded injection and unblinding of subjects after 12-month follow-up were compared between AMDC-USR and Placebo groups.
|
2.0%
1/50 • Adverse events were monitored from consent through study exit. Reporting groups are based on adverse events that occurred between initial blinded injection treatment and unblinding of subjects after 12-month follow-up.
Adverse events that occurred between initial blinded injection and unblinding of subjects after 12-month follow-up were compared between AMDC-USR and Placebo groups.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place