Trial Outcomes & Findings for Autologous Muscle Derived Cells for Underactive Bladder (NCT NCT02463448)

NCT ID: NCT02463448

Last Updated: 2022-01-19

Results Overview

Safety of Autologous Muscle Derived Cells in the treatment of underactive bladder as measured by occurrences of study-related adverse events (AEs) reported by 6 months after enrollment. Number of reports of each adverse event is listed for events determined to be definitely or possibly related to study activities.

• Recruitment status: COMPLETED
• Study phase: PHASE1
• Target enrollment: 23 participants
• Primary outcome timeframe: 6 months
• Results posted on: 2022-01-19

Participant Flow

Participant milestones

Measure	Autologous Muscle Derived Cells Autologous Muscle Derived Cells are obtained by needle biopsy from the subject's own thigh muscle. These cells are sent to a special lab for growth and processing. When ready the cells are sent to the treatment location for injection into the bladder wall. Autologous Muscle Derived Cells: A small sample of the subject's own thigh muscle is obtained by needle biopsy. The biopsy sample is sent to Cook Myosite, Inc. for growth and processing. When the sample cell dosage is achieved the cells are frozen and sent back to the treatment site. The cells are thawed, diluted and injected under lighted instrumentation and visualization into about 30 sites in the bladder wall.
Enrollment Through 6 Months STARTED	23
Enrollment Through 6 Months COMPLETED	20
Enrollment Through 6 Months NOT COMPLETED	3
6 Months Through 12 Months STARTED	20
6 Months Through 12 Months COMPLETED	18
6 Months Through 12 Months NOT COMPLETED	2
12 Months Through End of Study STARTED	18
12 Months Through End of Study COMPLETED	17
12 Months Through End of Study NOT COMPLETED	1

Reasons for withdrawal

Measure	Autologous Muscle Derived Cells Autologous Muscle Derived Cells are obtained by needle biopsy from the subject's own thigh muscle. These cells are sent to a special lab for growth and processing. When ready the cells are sent to the treatment location for injection into the bladder wall. Autologous Muscle Derived Cells: A small sample of the subject's own thigh muscle is obtained by needle biopsy. The biopsy sample is sent to Cook Myosite, Inc. for growth and processing. When the sample cell dosage is achieved the cells are frozen and sent back to the treatment site. The cells are thawed, diluted and injected under lighted instrumentation and visualization into about 30 sites in the bladder wall.
Enrollment Through 6 Months Withdrawal by Subject	3
6 Months Through 12 Months Lost to Follow-up	1
6 Months Through 12 Months Withdrawal by Subject	1
12 Months Through End of Study Lost to Follow-up	1

Baseline Characteristics

Autologous Muscle Derived Cells for Underactive Bladder

Baseline characteristics by cohort

Measure	Autologous Muscle Derived Cells n=20 Participants Autologous Muscle Derived Cells are obtained by needle biopsy from the subject's own thigh muscle. These cells are sent to a special lab for growth and processing. When ready the cells are sent to the treatment location for injection into the bladder wall. Autologous Muscle Derived Cells: A small sample of the subject's own thigh muscle is obtained by needle biopsy. The biopsy sample is sent to Cook Myosite, Inc. for growth and processing. When the sample cell dosage is achieved the cells are frozen and sent back to the treatment site. The cells are thawed, diluted and injected under lighted instrumentation and visualization into about 30 sites in the bladder wall.
Age, Continuous	61.45 years STANDARD_DEVIATION 12.00 • n=5 Participants
Sex: Female, Male Female	4 Participants n=5 Participants
Sex: Female, Male Male	16 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	20 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	20 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	20 participants n=5 Participants

PRIMARY outcome

Timeframe: 6 months

Population: Participants may have reported more than one AE during this study period.

Safety of Autologous Muscle Derived Cells in the treatment of underactive bladder as measured by occurrences of study-related adverse events (AEs) reported by 6 months after enrollment. Number of reports of each adverse event is listed for events determined to be definitely or possibly related to study activities.

Outcome measures

Measure	Autologous Muscle Derived Cells n=20 Participants Autologous Muscle Derived Cells are obtained by needle biopsy from the subject's own thigh muscle. These cells are sent to a special lab for growth and processing. When ready the cells are sent to the treatment location for injection into the bladder wall. Autologous Muscle Derived Cells: A small sample of the subject's own thigh muscle is obtained by needle biopsy. The biopsy sample is sent to Cook Myosite, Inc. for growth and processing. When the sample cell dosage is achieved the cells are frozen and sent back to the treatment site. The cells are thawed, diluted and injected under lighted instrumentation and visualization into about 30 sites in the bladder wall.
Number of Participants With Study-related Adverse Events at 6 Months Bruise	2 participants
Number of Participants With Study-related Adverse Events at 6 Months Pain	1 participants
Number of Participants With Study-related Adverse Events at 6 Months Scrotum discomfort	1 participants
Number of Participants With Study-related Adverse Events at 6 Months Scrotum swelling	1 participants
Number of Participants With Study-related Adverse Events at 6 Months Urinary tract infection	1 participants
Number of Participants With Study-related Adverse Events at 6 Months Vasovagal response	1 participants

SECONDARY outcome

Timeframe: Between 6 and 12 months

Population: Subjects may have reported more than one adverse event during this study period

Long term safety of Autologous Muscle Derived Cells in the treatment of underactive bladder as measured by occurrences of study-related adverse events (AEs) reported between 6 and 12 months after enrollment. Number of reports of each adverse event is listed for events determined to be definitely or possibly related to study activities.

Outcome measures

Measure	Autologous Muscle Derived Cells n=20 Participants Autologous Muscle Derived Cells are obtained by needle biopsy from the subject's own thigh muscle. These cells are sent to a special lab for growth and processing. When ready the cells are sent to the treatment location for injection into the bladder wall. Autologous Muscle Derived Cells: A small sample of the subject's own thigh muscle is obtained by needle biopsy. The biopsy sample is sent to Cook Myosite, Inc. for growth and processing. When the sample cell dosage is achieved the cells are frozen and sent back to the treatment site. The cells are thawed, diluted and injected under lighted instrumentation and visualization into about 30 sites in the bladder wall.
Number of Participants With Study-related Adverse Events Between 6 and 12 Months Urine leakage	1 participants
Number of Participants With Study-related Adverse Events Between 6 and 12 Months Bleeding at site of injection	1 participants

SECONDARY outcome

Timeframe: 12 months

Population: 17 subjects remaining in study at 12 months submitted data for analysis

The efficacy of Autologous Muscle Derived Cells in the treatment of underactive bladder as measured by changes to subject completed GRA questionnaire. The GRA measures the subjects perceived level of change in underactive bladder symptoms at 12 months compared to before they were treated. The scale ranges from: Markedly worse, Moderately worse, Slightly worse, No change, Slightly improved, Moderately improved, to Markedly improved. Subjects select one response.

Outcome measures

Measure	Autologous Muscle Derived Cells n=17 Participants Autologous Muscle Derived Cells are obtained by needle biopsy from the subject's own thigh muscle. These cells are sent to a special lab for growth and processing. When ready the cells are sent to the treatment location for injection into the bladder wall. Autologous Muscle Derived Cells: A small sample of the subject's own thigh muscle is obtained by needle biopsy. The biopsy sample is sent to Cook Myosite, Inc. for growth and processing. When the sample cell dosage is achieved the cells are frozen and sent back to the treatment site. The cells are thawed, diluted and injected under lighted instrumentation and visualization into about 30 sites in the bladder wall.
Number of Participants With Improvement in Underactive Bladder Symptoms at 12 Months. Markedly Improved	0 Participants
Number of Participants With Improvement in Underactive Bladder Symptoms at 12 Months. Moderately improved	3 Participants
Number of Participants With Improvement in Underactive Bladder Symptoms at 12 Months. Slightly improved	9 Participants
Number of Participants With Improvement in Underactive Bladder Symptoms at 12 Months. No change	5 Participants
Number of Participants With Improvement in Underactive Bladder Symptoms at 12 Months. Slightly worse	0 Participants
Number of Participants With Improvement in Underactive Bladder Symptoms at 12 Months. Moderately worse	0 Participants
Number of Participants With Improvement in Underactive Bladder Symptoms at 12 Months. Markedly worse	0 Participants

SECONDARY outcome

Timeframe: 12 months

Population: 17 subjects remaining in study at 12 months submitted data for analysis

The efficacy of Autologous Muscle Derived Cells in the treatment of underactive bladder as measured by changes to subject reported degree of change in urinary symptoms on the PGI-I at 12 months, compared to how their symptoms were before they began the study. Subjects select one response. The response scale ranges from: Very much better, Much better, A little bit better, No change, A little worse, Much worse, Very Much worse.

Outcome measures

Measure	Autologous Muscle Derived Cells n=17 Participants Autologous Muscle Derived Cells are obtained by needle biopsy from the subject's own thigh muscle. These cells are sent to a special lab for growth and processing. When ready the cells are sent to the treatment location for injection into the bladder wall. Autologous Muscle Derived Cells: A small sample of the subject's own thigh muscle is obtained by needle biopsy. The biopsy sample is sent to Cook Myosite, Inc. for growth and processing. When the sample cell dosage is achieved the cells are frozen and sent back to the treatment site. The cells are thawed, diluted and injected under lighted instrumentation and visualization into about 30 sites in the bladder wall.
Number of Participants With Improvement in Underactive Bladder Symptoms at 12 Months. Very much better	0 Participants
Number of Participants With Improvement in Underactive Bladder Symptoms at 12 Months. Much better	3 Participants
Number of Participants With Improvement in Underactive Bladder Symptoms at 12 Months. A little bit better	9 Participants
Number of Participants With Improvement in Underactive Bladder Symptoms at 12 Months. No change	5 Participants
Number of Participants With Improvement in Underactive Bladder Symptoms at 12 Months. A little worse	0 Participants
Number of Participants With Improvement in Underactive Bladder Symptoms at 12 Months. Much worse	0 Participants
Number of Participants With Improvement in Underactive Bladder Symptoms at 12 Months. Very much worse	0 Participants

SECONDARY outcome

Timeframe: 12 months

Population: 17 subjects remaining in the study at 12 months had data analyzed for this outcome: 3 female and 14 male.

The efficacy of Autologous Muscle Derived Cells in the treatment of underactive bladder as measured by changes to subject reported urinary symptoms at 12 months from baseline. The ICIQ has a separate female (ICIQ-FLUTS Long form) and male (ICIQ-MLUTS long Form) set of questions to assess urinary symptoms in each sex. The total score range for ICIQ-FLUTS is 0-69. The total score range for ICIQ-MLUTS is 0-84. The sum of all the question scores equal a total score. Each question has a degree of bother scale associated with it. The degree of bother ranges from 0 (none at all) to 10 ( a great deal). Degree of bother scores are not incorporated in the overall score but indicate impact of the individual symptom. The difference taken in this analysis is the difference between the total score at 12 months minus the total score at baseline. If the difference is negative, it indicates improvement in the symptoms; 0 is no change; and a positive difference indicates a worsening of the symptoms.

Outcome measures

Measure	Autologous Muscle Derived Cells n=17 Participants Autologous Muscle Derived Cells are obtained by needle biopsy from the subject's own thigh muscle. These cells are sent to a special lab for growth and processing. When ready the cells are sent to the treatment location for injection into the bladder wall. Autologous Muscle Derived Cells: A small sample of the subject's own thigh muscle is obtained by needle biopsy. The biopsy sample is sent to Cook Myosite, Inc. for growth and processing. When the sample cell dosage is achieved the cells are frozen and sent back to the treatment site. The cells are thawed, diluted and injected under lighted instrumentation and visualization into about 30 sites in the bladder wall.
Change in Incontinence Questionnaire (ICIQ) Long Form Score From Baseline at 12 Months. Female (ICIQ-FLUTS) long form	0.6667 units on a scale Standard Deviation 5.1
Change in Incontinence Questionnaire (ICIQ) Long Form Score From Baseline at 12 Months. Male (ICIQ-MLUTS) long form	1.071 units on a scale Standard Deviation 9.600

SECONDARY outcome

Timeframe: 12 months

Population: 17 subjects remaining at 12 months submitted data for analysis

The efficacy of Autologous Muscle Derived Cells in the treatment of underactive bladder as measured by subject reported changes to urinary symptoms at 12 months from baseline on the UABQ. The UABQ is a set of questions to assess urinary symptoms associated with underactive bladder. The total score ranges between 0 to 16. A lower score indicates a better outcome. A higher score indicates a worse outcome.

Outcome measures

Measure

Autologous Muscle Derived Cells n=17 Participants Autologous Muscle Derived Cells are obtained by needle biopsy from the subject's own thigh muscle. These cells are sent to a special lab for growth and processing. When ready the cells are sent to the treatment location for injection into the bladder wall. Autologous Muscle Derived Cells: A small sample of the subject's own thigh muscle is obtained by needle biopsy. The biopsy sample is sent to Cook Myosite, Inc. for growth and processing. When the sample cell dosage is achieved the cells are frozen and sent back to the treatment site. The cells are thawed, diluted and injected under lighted instrumentation and visualization into about 30 sites in the bladder wall.

Change in Underactive Bladder Questionnaire (UABQ) Score From Baseline at 12 Months

5.7 score on a scale Standard Deviation 2.7

SECONDARY outcome

Timeframe: 12 months

Population: 10 subjects had data available for both timepoints

The efficacy of Autologous Muscle Derived Cells in the treatment of underactive bladder as measured by changes to subject reported number of independent daytime voiding occurrences per day on the 3-day diary at 12 months compared to baseline. Positive numbers reflect a larger number of independent voids per day reported at baseline, and negative number represent a larger number of independent voids per day reported at 12 months.

Outcome measures

Measure

Autologous Muscle Derived Cells n=10 Participants Autologous Muscle Derived Cells are obtained by needle biopsy from the subject's own thigh muscle. These cells are sent to a special lab for growth and processing. When ready the cells are sent to the treatment location for injection into the bladder wall. Autologous Muscle Derived Cells: A small sample of the subject's own thigh muscle is obtained by needle biopsy. The biopsy sample is sent to Cook Myosite, Inc. for growth and processing. When the sample cell dosage is achieved the cells are frozen and sent back to the treatment site. The cells are thawed, diluted and injected under lighted instrumentation and visualization into about 30 sites in the bladder wall.

The Change in Number of Independent Voiding Occurrences Per Day on the 3-day Bladder Diary From Baseline at 12 Months.

-0.665 number of voids/day Standard Deviation 5.3

SECONDARY outcome

Timeframe: 12 months

Population: 19 subjects had data reported for both timepoints

The efficacy of Autologous Muscle Derived Cells in the treatment of underactive bladder as measured by changes to subject reported number of daytime voiding occurrences per day using a catheter on the 3-day diary at 12 months compared to baseline. Positive numbers reflect a larger number of voiding occurrences per day reported at baseline, and negative numbers represent a larger number of voiding occurrences per day reported at 12 months.

Outcome measures

Measure

Autologous Muscle Derived Cells n=19 Participants Autologous Muscle Derived Cells are obtained by needle biopsy from the subject's own thigh muscle. These cells are sent to a special lab for growth and processing. When ready the cells are sent to the treatment location for injection into the bladder wall. Autologous Muscle Derived Cells: A small sample of the subject's own thigh muscle is obtained by needle biopsy. The biopsy sample is sent to Cook Myosite, Inc. for growth and processing. When the sample cell dosage is achieved the cells are frozen and sent back to the treatment site. The cells are thawed, diluted and injected under lighted instrumentation and visualization into about 30 sites in the bladder wall.

The Change in the Number of Voiding Occurrences Per Day Using a Catheter Recorded on the 3-day Bladder Diary From Baseline at 12 Months.

-0.2094 number of voids/day Standard Deviation 1.6

SECONDARY outcome

Timeframe: 12 months

Population: 10 subjects had data reported for both timepoints

The efficacy of Autologous Muscle Derived Cells in the treatment of underactive bladder as measured by changes to subject reported average volume voided independently per day over the 3 day period on the 3-day diary at 12 months compared to baseline. Volumes are measured in milliliters. Positive numbers reflect a larger volume voided per day at baseline, and negative numbers represent a larger volume voided per day at 12 months.

Outcome measures

Measure

Autologous Muscle Derived Cells n=10 Participants Autologous Muscle Derived Cells are obtained by needle biopsy from the subject's own thigh muscle. These cells are sent to a special lab for growth and processing. When ready the cells are sent to the treatment location for injection into the bladder wall. Autologous Muscle Derived Cells: A small sample of the subject's own thigh muscle is obtained by needle biopsy. The biopsy sample is sent to Cook Myosite, Inc. for growth and processing. When the sample cell dosage is achieved the cells are frozen and sent back to the treatment site. The cells are thawed, diluted and injected under lighted instrumentation and visualization into about 30 sites in the bladder wall.

The Change in the Average Volume Voided Independently Per Day Measured in Milliliters Over the 3 Day Period Recorded on the 3-day Bladder Diary From Baseline at 12 Months.

-156.66 milliliters/day Standard Deviation 1100.0

SECONDARY outcome

Timeframe: 12 months

Population: 14 subjects had data for both timepoints

The efficacy of Autologous Muscle Derived Cells in the treatment of underactive bladder as measured by changes to subject reported average volume voided using a catheter per day over the 3 day period on the 3-day diary at 12 months compared to baseline. Volumes are measured in milliliters. Positive numbers reflect a larger volume voided per day at baseline, and negative numbers represent a larger volume voided per day at 12 months.

Outcome measures

Measure

Autologous Muscle Derived Cells n=14 Participants Autologous Muscle Derived Cells are obtained by needle biopsy from the subject's own thigh muscle. These cells are sent to a special lab for growth and processing. When ready the cells are sent to the treatment location for injection into the bladder wall. Autologous Muscle Derived Cells: A small sample of the subject's own thigh muscle is obtained by needle biopsy. The biopsy sample is sent to Cook Myosite, Inc. for growth and processing. When the sample cell dosage is achieved the cells are frozen and sent back to the treatment site. The cells are thawed, diluted and injected under lighted instrumentation and visualization into about 30 sites in the bladder wall.

The Change in the Average Volume in Milliliters Voided Per Day Using a Catheter Per Day Over the 3 Day Period Recorded on the 3-day Bladder Diary From Baseline at 12 Months.

-894.9 milliliters/day Standard Deviation 2100

SECONDARY outcome

Timeframe: 12 months

The efficacy of Autologous Muscle Derived Cells in the treatment of underactive bladder as measured by changes to subject reported number of bladder leaks over the 3 day period on the 3-day diary at 12 months compared to baseline. Positive numbers reflect a larger number of leaks reported at baseline, and negative numbers represent a larger number of leaks reported at 12 months.

Outcome measures

Measure

Autologous Muscle Derived Cells n=20 Participants Autologous Muscle Derived Cells are obtained by needle biopsy from the subject's own thigh muscle. These cells are sent to a special lab for growth and processing. When ready the cells are sent to the treatment location for injection into the bladder wall. Autologous Muscle Derived Cells: A small sample of the subject's own thigh muscle is obtained by needle biopsy. The biopsy sample is sent to Cook Myosite, Inc. for growth and processing. When the sample cell dosage is achieved the cells are frozen and sent back to the treatment site. The cells are thawed, diluted and injected under lighted instrumentation and visualization into about 30 sites in the bladder wall.

The Change in the Number of Bladder Leaks Over the 3 Day Period Recorded on the 3-day Bladder Diary From Baseline at 12 Months.

0 bladder leaks per day Standard Deviation 0

Adverse Events

Autologous Muscle Derived Cells

Serious events: 4 serious events

Other events: 20 other events

Deaths: 0 deaths

Serious adverse events

Measure	Autologous Muscle Derived Cells n=20 participants at risk Autologous Muscle Derived Cells are obtained by needle biopsy from the subject's own thigh muscle. These cells are sent to a special lab for growth and processing. When ready the cells are sent to the treatment location for injection into the bladder wall. Autologous Muscle Derived Cells: A small sample of the subject's own thigh muscle is obtained by needle biopsy. The biopsy sample is sent to Cook Myosite, Inc. for growth and processing. When the sample cell dosage is achieved the cells are frozen and sent back to the treatment site. The cells are thawed, diluted and injected under lighted instrumentation and visualization into about 30 sites in the bladder wall.
Cardiac disorders Aortic stenosis	5.0% 1/20 • Number of events 1 • 18 months
Musculoskeletal and connective tissue disorders Closed fracture of tibia	5.0% 1/20 • Number of events 1 • 18 months
Renal and urinary disorders Pyelonephritis	5.0% 1/20 • Number of events 1 • 18 months
Musculoskeletal and connective tissue disorders Fracture of Humerus	5.0% 1/20 • Number of events 1 • 18 months
Musculoskeletal and connective tissue disorders Fracture of femoral neck	5.0% 1/20 • Number of events 1 • 18 months

Other adverse events

Measure	Autologous Muscle Derived Cells n=20 participants at risk Autologous Muscle Derived Cells are obtained by needle biopsy from the subject's own thigh muscle. These cells are sent to a special lab for growth and processing. When ready the cells are sent to the treatment location for injection into the bladder wall. Autologous Muscle Derived Cells: A small sample of the subject's own thigh muscle is obtained by needle biopsy. The biopsy sample is sent to Cook Myosite, Inc. for growth and processing. When the sample cell dosage is achieved the cells are frozen and sent back to the treatment site. The cells are thawed, diluted and injected under lighted instrumentation and visualization into about 30 sites in the bladder wall.
Renal and urinary disorders Urinary tract infection	50.0% 10/20 • Number of events 20 • 18 months
Respiratory, thoracic and mediastinal disorders Influenza	15.0% 3/20 • Number of events 3 • 18 months
Skin and subcutaneous tissue disorders Bruise	10.0% 2/20 • Number of events 2 • 18 months
General disorders Fall	10.0% 2/20 • Number of events 2 • 18 months
General disorders Fatigue	10.0% 2/20 • Number of events 2 • 18 months
General disorders Pain	15.0% 3/20 • Number of events 3 • 18 months

Additional Information

Jennifer Giordano

Beaumont Hospitals

Phone: 248-551-3517

Email: jennifer.geiordano@beaumont.org

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place