Autologous Muscle Derived Cells for Underactive Bladder

• ClinicalTrials.gov Identifier: NCT02463448
• Recruitment Status: Completed
• First Posted: June 4, 2015
• Results First Posted: January 19, 2022
• Last Update Posted: January 19, 2022
• Sponsor: Jason Gilleran
• Information provided by (Responsible Party): Jason Gilleran, William Beaumont Hospitals

• Study Type: Interventional
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Conditions: Urinary Retention; Urinary Incontinence
• Intervention: Biological: Autologous Muscle Derived Cells
• Enrollment: 23

Participant Flow

Recruitment Details
Pre-assignment Details

Arm/Group Title	Autologous Muscle Derived Cells
Arm/Group Description	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.
Period Title: Enrollment Through 6 Months
Started	23
Completed	20
Not Completed	3
Reason Not Completed
Withdrawal by Subject	3
Period Title: 6 Months Through 12 Months
Started	20
Completed	18
Not Completed	2
Reason Not Completed
Lost to Follow-up	1
Withdrawal by Subject	1
Period Title: 12 Months Through End of Study
Started	18
Completed	17
Not Completed	1
Reason Not Completed
Lost to Follow-up	1

Baseline Characteristics

Arm/Group Title		Autologous Muscle Derived Cells
Arm/Group Description		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.
Overall Number of Baseline Participants		20
Baseline Analysis Population Description		Of initial 23 subjects consented, 3 subjects withdrew from study prior to collection of initial data. Demographic data is not available for these 3 subjects.
Age, Continuous; Mean (Standard Deviation); Unit of measure: Years
	Number Analyzed	20 participants
		61.45 (12.00)
Sex: Female, Male; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	20 participants
	Female	4 20.0%
	Male	16 80.0%
Ethnicity (NIH/OMB); Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	20 participants
	Hispanic or Latino	0 0.0%
	Not Hispanic or Latino	20 100.0%
	Unknown or Not Reported	0 0.0%
Race (NIH/OMB); Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	20 participants
	American Indian or Alaska Native	0 0.0%
	Asian	0 0.0%
	Native Hawaiian or Other Pacific Islander	0 0.0%
	Black or African American	0 0.0%
	White	20 100.0%
	More than one race	0 0.0%
	Unknown or Not Reported	0 0.0%
Region of Enrollment; Measure Type: Number; Unit of measure: Participants
United States	Number Analyzed	20 participants
		20

Outcome Measures

1. Primary Outcome

Title	Number of Participants With Study-related Adverse Events at 6 Months
Description	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.
Time Frame	6 months

Outcome Measure Data

Analysis Population Description

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

Arm/Group Title	Autologous Muscle Derived Cells
Arm/Group Description:	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.
Overall Number of Participants Analyzed	20
Measure Type: Number; Unit of Measure: participants
Bruise	2
Pain	1
Scrotum discomfort	1
Scrotum swelling	1
Urinary tract infection	1
Vasovagal response	1

2. Secondary Outcome

Title	Number of Participants With Study-related Adverse Events Between 6 and 12 Months
Description	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.
Time Frame	Between 6 and 12 months

Outcome Measure Data

Analysis Population Description

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

Arm/Group Title	Autologous Muscle Derived Cells
Arm/Group Description:	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.
Overall Number of Participants Analyzed	20
Measure Type: Number; Unit of Measure: participants
Urine leakage	1
Bleeding at site of injection	1

3. Secondary Outcome

Title	Number of Participants With Improvement in Underactive Bladder Symptoms at 12 Months.
Description	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.
Time Frame	12 months

Outcome Measure Data

Analysis Population Description

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

Arm/Group Title	Autologous Muscle Derived Cells
Arm/Group Description:	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.
Overall Number of Participants Analyzed	17
Measure Type: Count of Participants; Unit of Measure: Participants
Markedly Improved	0 0.0%
Moderately improved	3 17.6%
Slightly improved	9 52.9%
No change	5 29.4%
Slightly worse	0 0.0%
Moderately worse	0 0.0%
Markedly worse	0 0.0%

4. Secondary Outcome

Title	Number of Participants With Improvement in Underactive Bladder Symptoms at 12 Months.
Description	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.
Time Frame	12 months

Outcome Measure Data

Analysis Population Description

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

Arm/Group Title	Autologous Muscle Derived Cells
Arm/Group Description:	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.
Overall Number of Participants Analyzed	17
Measure Type: Count of Participants; Unit of Measure: Participants
Very much better	0 0.0%
Much better	3 17.6%
A little bit better	9 52.9%
No change	5 29.4%
A little worse	0 0.0%
Much worse	0 0.0%
Very much worse	0 0.0%

5. Secondary Outcome

Title	Change in Incontinence Questionnaire (ICIQ) Long Form Score From Baseline at 12 Months.
Description	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.
Time Frame	12 months

Outcome Measure Data

Analysis Population Description

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

Arm/Group Title		Autologous Muscle Derived Cells
Arm/Group Description:		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.
Overall Number of Participants Analyzed		17
Mean (Standard Deviation); Unit of Measure: units on a scale
Female (ICIQ-FLUTS) long form	Number Analyzed	3 participants
		0.6667 (5.1)
Male (ICIQ-MLUTS) long form	Number Analyzed	14 participants
		1.071 (9.600)

6. Secondary Outcome

Title	Change in Underactive Bladder Questionnaire (UABQ) Score From Baseline at 12 Months
Description	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.
Time Frame	12 months

Outcome Measure Data

Analysis Population Description

17 subjects remaining at 12 months submitted data for analysis

Arm/Group Title	Autologous Muscle Derived Cells
Arm/Group Description:	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.
Overall Number of Participants Analyzed	17
Mean (Standard Deviation); Unit of Measure: score on a scale
	5.7 (2.7)

7. Secondary Outcome

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

Outcome Measure Data

Analysis Population Description

10 subjects had data available for both timepoints

Arm/Group Title	Autologous Muscle Derived Cells
Arm/Group Description:	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.
Overall Number of Participants Analyzed	10
Mean (Standard Deviation); Unit of Measure: number of voids/day
	-0.665 (5.3)

8. Secondary Outcome

Title	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.
Description	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.
Time Frame	12 months

Outcome Measure Data

Analysis Population Description

19 subjects had data reported for both timepoints

Arm/Group Title	Autologous Muscle Derived Cells
Arm/Group Description:	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.
Overall Number of Participants Analyzed	19
Mean (Standard Deviation); Unit of Measure: number of voids/day
	-0.2094 (1.6)

9. Secondary Outcome

Title	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.
Description	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.
Time Frame	12 months

Outcome Measure Data

Analysis Population Description

10 subjects had data reported for both timepoints

Arm/Group Title	Autologous Muscle Derived Cells
Arm/Group Description:	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.
Overall Number of Participants Analyzed	10
Mean (Standard Deviation); Unit of Measure: milliliters/day
	-156.66 (1100.0)

10. Secondary Outcome

Title	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.
Description	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.
Time Frame	12 months

Outcome Measure Data

Analysis Population Description

14 subjects had data for both timepoints

Arm/Group Title	Autologous Muscle Derived Cells
Arm/Group Description:	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.
Overall Number of Participants Analyzed	14
Mean (Standard Deviation); Unit of Measure: milliliters/day
	-894.9 (2100)

11. Secondary Outcome

Title	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.
Description	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.
Time Frame	12 months

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Autologous Muscle Derived Cells
Arm/Group Description:	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.
Overall Number of Participants Analyzed	20
Mean (Standard Deviation); Unit of Measure: bladder leaks per day
	0 (0)

Adverse Events

Time Frame	18 months
Adverse Event Reporting Description	[Not Specified]
Arm/Group Title	Autologous Muscle Derived Cells
Arm/Group Description	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.
All-Cause Mortality
	Autologous Muscle Derived Cells
	Affected / at Risk (%)
Total	0/20 (0.00%)
Serious Adverse Events
	Autologous Muscle Derived Cells
	Affected / at Risk (%)	# Events
Total	4/20 (20.00%)
Cardiac disorders
Aortic stenosis †	1/20 (5.00%)	1
Musculoskeletal and connective tissue disorders
Closed fracture of tibia † [1]	1/20 (5.00%)	1
Fracture of Humerus † [1]	1/20 (5.00%)	1
Fracture of femoral neck † [1]	1/20 (5.00%)	1
Renal and urinary disorders
Pyelonephritis †	1/20 (5.00%)	1
† Indicates events were collected by systematic assessment; [1] with fall
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	5%
	Autologous Muscle Derived Cells
	Affected / at Risk (%)	# Events
Total	20/20 (100.00%)
General disorders
Fall †	2/20 (10.00%)	2
Fatigue †	2/20 (10.00%)	2
Pain †	3/20 (15.00%)	3
Renal and urinary disorders
Urinary tract infection †	10/20 (50.00%)	20
Respiratory, thoracic and mediastinal disorders
Influenza †	3/20 (15.00%)	3
Skin and subcutaneous tissue disorders
Bruise †	2/20 (10.00%)	2
† Indicates events were collected by systematic assessment

Limitations and Caveats

[Not Specified]

More Information

Certain Agreements

All Principal Investigators ARE employed by the organization sponsoring the study.

Results Point of Contact

• Name/Title: Jennifer Giordano
• Organization: Beaumont Hospitals
• Phone: 248-551-3517
• EMail: jennifer.geiordano@beaumont.org

• Responsible Party: Jason Gilleran, William Beaumont Hospitals
• ClinicalTrials.gov Identifier: NCT02463448
• Other Study ID Numbers: 2015-134
• First Submitted: May 27, 2015
• First Posted: June 4, 2015
• Results First Submitted: September 18, 2021
• Results First Posted: January 19, 2022
• Last Update Posted: January 19, 2022