Autologous Cell Therapy for Stress Urinary Incontinence in Males Following Prostate Surgery

• ClinicalTrials.gov Identifier: NCT02291432
• Recruitment Status: Completed
• First Posted: November 14, 2014
• Results First Posted: October 26, 2021
• Last Update Posted: November 1, 2021
• Sponsor: Cook MyoSite
• Information provided by (Responsible Party): Cook MyoSite

• Study Type: Interventional
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Conditions: Urinary Incontinence; Stress; Urination Disorders; Urologic Diseases; Lower Urinary Tract Symptoms; Urological Manifestations
• Intervention: Biological: autologous muscle-derived cells (AMDC)
• Enrollment: 25

Participant Flow

Recruitment Details
Pre-assignment Details

Arm/Group Title	AMDC-USR
Arm/Group Description	Single intraurethral injection of 150 x 10^6 Autologous Muscle-Derived Cells (AMDCs)
Period Title: Overall Study
Started	25
Muscle Biopsy	25
AMDC Injection	23
1-Month Follow Up	23
3-Month Follow Up	23
6-Month Follow Up	23
12-Month Follow Up	23
Completed	23
Not Completed	2
Reason Not Completed
Withdrawal by Subject	2

Baseline Characteristics

Arm/Group Title		AMDC-USR
Arm/Group Description		Single intraurethral injection of 150 x 10^6 autologous muscle-derived cells (AMDCs).
Overall Number of Baseline Participants		25
Baseline Analysis Population Description		[Not Specified]
Age, Categorical; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	25 participants
	<=18 years	0 0.0%
	Between 18 and 65 years	8 32.0%
	>=65 years	17 68.0%
Age, Continuous; Median (Full Range); Unit of measure: Years
	Number Analyzed	25 participants
		67; (49 to 78)
Age, Customized; Mean (Standard Deviation); Unit of measure: Years
Participant Age	Number Analyzed	25 participants
		67.2 (5.8)
Sex: Female, Male; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	25 participants
	Female	0 0.0%
	Male	25 100.0%
Race (NIH/OMB); Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	25 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	1 4.0%
	White	24 96.0%
	More than one race	0 0.0%
	Unknown or Not Reported	0 0.0%
Region of Enrollment; Measure Type: Count of Participants; Unit of measure: Participants
United States	Number Analyzed	25 participants
		25 100.0%
Urinary Incontinence Type; Measure Type: Count of Participants; Unit of measure: Participants	Number Analyzed	25 participants
Pure Stress Urinary Incontinence		4 16.0%
Mixed Urinary Incontinence, Stress Predominant		17 68.0%
Mixed Urinary Incontinence, Urge Predominant		4 16.0%
24-hour Pad Test Weight [1]; Median (Full Range); Unit of measure: Grams
	Number Analyzed	25 participants
		109.1; (4.1 to 431)
		[1] Measure Description: A test in which the participant wears specific absorbent pads dispensed by the clinic. The pads are weighed to record the pre-test weight prior to dispensing to the participant. The participant must wear the absorbent pads for 24 hours. The participant returns the used absorbent pads to the clinic, and the clinic records the post-test weight to determine urinary leakage for the 24 hour period. The difference in weights represent the amount of urine leaked.
Incontinence Quality of Life (I-QOL) [1]; Median (Full Range); Unit of measure: Scores on scales
	Number Analyzed	25 participants
		59.1; (0 to 87.5)
		[1] Measure Description: The I-QOL questionnaire was a validated, 22-item tool used to assess QOL of participants with urinary incontinence. Scored 0 to 100, with higher scores indicating a better QOL.
International Consultation on Incontinence Questionnaire (ICIQ) [1]; Median (Full Range); Unit of measure: Scores on scales
	Number Analyzed	25 participants
		13; (7 to 18)
		[1] Measure Description: The ICIQ questionnaire was a validated 4-item tool used to assess symptom severity of participants with urinary incontinence. Scored 0 to 21, with lower scores indicating a better symptom severity.
International Prostate Symptom Score (IPSS) [1]; Median (Full Range); Unit of measure: Scores on scales
	Number Analyzed	25 participants
		9; (1 to 33)
		[1] Measure Description: The I-PSS was a validated questionnaire used to assess the severity of three urine storage symptoms (frequency, nocturia, urgency), four voiding symptoms (feeling of incomplete emptying, intermittency, straining, and a weak stream) and the degree of bother associated with those symptoms. A score of 0 to 7 indicates mild symptoms, 8 to 19 indicates moderate symptoms, and 20 to 35 indicates severe symptoms.
International Index of Erectile Function (IIEF) [1]; Median (Full Range); Unit of measure: Scores on scales
	Number Analyzed	25 participants
		13; (5 to 25)
		[1] Measure Description: The IIEF-5 questionnaire was a validated 5-item tool used to assess to presence and severity of erectile dysfunction (ED). Scored from 5 to 25, with lower scores indicating less ED symptoms.
Patient Global Impression of Severity (PGI-S) Questionnaire [1]; Measure Type: Count of Participants; Unit of measure: Participants	Number Analyzed	25 participants
Normal		0 0.0%
Mild		8 32.0%
Moderate		13 52.0%
Severe		4 16.0%
		[1] Measure Description: The PGI-S was a global assessment of symptom severity compared with severity before treatment started. Ratings that could be selected were: 1-normal, 2-mild, 3-moderate, and 4-severe.

Outcome Measures

1. Primary Outcome

Title	Rate of Product-related, Biopsy Procedure-related, and Injection Procedure-related Adverse Events
Description	Safety of AMDC-USR following treatment of SUI in male patients who have undergone prior prostate surgery was determined by the frequency and severity of adverse events related to study procedures and study product through 24 months following treatment of SUI in male patients who have undergone prior prostate surgery.
Time Frame	24 months

Outcome Measure Data

Analysis Population Description

All subjects enrolled (biopsied)

Arm/Group Title	AMDC-USR
Arm/Group Description:	Single intraurethral injection of 150 x 10^6 Autologous Muscle-Derived Cells (AMDCs)
Overall Number of Participants Analyzed	25
Measure Type: Number; Unit of Measure: events
Study product-related serious adverse events	0
Study Product-related adverse events	2
Injection procedure-related adverse events	0
Biopsy procedure-related adverse events	12

2. Primary Outcome

Title	Volume of Post-void Residual (PVR) Urine
Description	Post void residual volume (PVR) was assessed through 12 months post-treatment to monitor potential retention or obstruction.
Time Frame	1, 3, 6, and 12 months

Outcome Measure Data

Analysis Population Description

All subjects injected

Arm/Group Title	AMDC-USR
Arm/Group Description:	Single intraurethral injection of 150 x 10^6 Autologous Muscle-Derived Cells (AMDCs)
Overall Number of Participants Analyzed	23
Median (Full Range); Unit of Measure: millileters
Post void residual volume 1 month	0; (0 to 52)
Post void residual volume 3 months	0; (0 to 37)
Post void residual volume 6 months	0; (0 to 142)
Post void residual volume 12 months	0; (0 to 22)

3. Secondary Outcome

Title	Change From Baseline in Amount of Urine Leakage (Median 24 Hour Pad Weight)
Description	Change in the amount of urine leakage from baseline was assessed by 24-hour pad test at 1, 3, 6, and 12 months post-treatment. Amount of urine leakage experienced by subject at home during a 24-hour period; all pads used during test period are weighed before and after use and differences in weights represent the amount of urine leaked.
Time Frame	1, 3, 6, and 12 months

Outcome Measure Data

Analysis Population Description

All subjects injected

Arm/Group Title	AMDC-USR
Arm/Group Description:	Single intraurethral injection of 150 x 10^6 Autologous Muscle-Derived Cells (AMDCs)
Overall Number of Participants Analyzed	23
Median (Full Range); Unit of Measure: grams
24 hour pad weight at 1 month	101.2; (5.3 to 674.3)
24 hour pad weight at 3 months	59.5; (18.1 to 357)
24 hour pad weight at 6 months	104.9; (9.2 to 359.5)
24 hour pad weight at 12 months	85.7; (13.5 to 319.7)

4. Secondary Outcome

Title	Number of Participants With a Change From Baseline in Amount of Urine Leakage (Categorial ≥50% Reduction in 24-hour Pad Weight)
Description	Change in the amount of urine leakage from baseline was assessed by a 24-hour pad test at 1, 3, 6, and 12 months post-treatment. Amount of urine leakage experienced by subject at home during a 24-hour period; all pads used during test period are weighed before and after use and differences in weights represent the amount of urine leaked.
Time Frame	1, 3, 6, and 12 months

Outcome Measure Data

Analysis Population Description

All subjects injected

Arm/Group Title	AMDC-USR
Arm/Group Description:	Single intraurethral injection of 150 x 10^6 Autologous Muscle-Derived Cells (AMDCs)
Overall Number of Participants Analyzed	23
Measure Type: Count of Participants; Unit of Measure: Participants
≥50% reduction in 24 hour pad weight at 1 month	2 8.7%
≥50% reduction in 24 hour pad weight at 3 months	4 17.4%
≥50% reduction in 24 hour pad weight at 6 months	4 17.4%
≥50% reduction in 24 hour pad weight at 12 months	7 30.4%

5. Secondary Outcome

Title	Median Change From Baseline in Patient-reported Quality of Life (QOL) - Incontinence Quality of Life (I-QOL) Questionnaire
Description	Median change from baseline in patient-reported quality of life (QOL) was assessed by the Incontinence Quality of Life (I-QOL) questionnaire at 1, 3, 6 and 12 months post-treatment. The I-QOL questionnaire was a validated, 22-item tool used to assess QOL of participants with urinary incontinence. Scored 0 to100, with higher scores indicating a better QOL.
Time Frame	1, 3, 6, and 12 months

Outcome Measure Data

Analysis Population Description

All subjects injected

Arm/Group Title	AMDC-USR
Arm/Group Description:	Single intraurethral injection of 150 x 10^6 Autologous Muscle-Derived Cells (AMDCs)
Overall Number of Participants Analyzed	23
Median (Full Range); Unit of Measure: points
Change in I-QOL total score at 1 month	-6.8; (-14.8 to 6.8)
Change in I-QOL total score at 3 months	0; (-27.3 to 9.1)
Change in I-QOL total score at 6 months	-9.1; (-37.5 to 12.5)
Change in I-QOL total score at 12 months	-9.1; (-36.4 to 14.8)

6. Secondary Outcome

Title	Median Change From Baseline in Patient-reported Symptom Severity- International Consultation on Incontinence Questionnaire (ICIQ)
Description	Median change from baseline in patient-reported symptom severity was assessed by the International Consultation on Incontinence Questionnaire (ICIQ) questionnaire at 1, 3, 6, and 12 months post-treatment. The ICIQ questionnaire was a validated 4-item tool used to assess symptom severity of participants with urinary incontinence. Scored 0 to 21, with lower scores indicating a better symptom severity.
Time Frame	Baseline, 1, 3, 6, and 12 months

Outcome Measure Data

Analysis Population Description

All subjects injected

Arm/Group Title	AMDC-USR
Arm/Group Description:	Single intraurethral injection of 150 x 10^6 Autologous Muscle-Derived Cells (AMDCs)
Overall Number of Participants Analyzed	23
Median (Full Range); Unit of Measure: scores on a scale
Change in ICIQ score at 1 month	0; (-2 to 7)
Change in ICIQ score at 3 months	0; (-3 to 7)
Change in ICIQ score at 6 months	0; (-3 to 7)
Change in ICIQ score at 12 months	1; (-1 to 16)

7. Secondary Outcome

Title	Median Change in Patient-reported Incontinence Symptom Severity - International Prostate Symptom Score (I-PSS) Questionnaire
Description	Patient-reported incontinence symptom severity was assessed by questionnaires at 1, 3, 6, and 12 months post-treatment using the International Prostate Symptom Score (I-PSS) questionnaire. The I-PSS was a validated questionnaire used to assess the severity of three urine storage symptoms (frequency, nocturia, urgency), four voiding symptoms (feeling of incomplete emptying, intermittency, straining, and a weak stream) and the degree of bother associated with those symptoms. A score of 0 to 7 indicates mild symptoms, 8 to 19 indicates moderate symptoms, and 20 to 35 indicates severe symptoms.
Time Frame	Baseline, 1, 3, 6, and 12 months

Outcome Measure Data

Analysis Population Description

All subjects injected

Arm/Group Title	AMDC-USR
Arm/Group Description:	Single intraurethral injection of 150 x 10^6 Autologous Muscle-Derived Cells (AMDCs)
Overall Number of Participants Analyzed	23
Median (Full Range); Unit of Measure: scores on a scale
Change in I-PSS score at 1 month	1; (-4 to 8)
Change in I-PSS score at 3 months	1; (-8 to 10)
Change in I-PSS score at 6 months	1; (-6 to 15)
Change in I-PSS score at 12 months	1; (-3 to 10)

8. Secondary Outcome

Title	Distribution of Patient-reported Incontinence Symptom Severity Scores - Patient Global Impression of Severity (PGI-S) Questionnaire
Description	Patient-reported incontinence symptom severity was assessed by questionnaires at 1, 3, 6, and 12 months post-treatment using the Patient Global Impression of Severity (PGI-S) questionnaire. The PGI-S was a global assessment of symptom severity compared with severity before treatment started. Ratings that could be selected were: 1-normal, 2-mild, 3-moderate, and 4-severe. Percentages of participants in each category were determined at each visit.
Time Frame	1, 3, 6, and 12 months

Outcome Measure Data

Analysis Population Description

All subjects injected

Arm/Group Title		AMDC-USR
Arm/Group Description:		Single intraurethral injection of 150 x 10^6 Autologous Muscle-Derived Cells (AMDCs)
Overall Number of Participants Analyzed		23
Measure Type: Count of Participants; Unit of Measure: Participants
PGI-S score at 1 month	Normal	1 4.3%
	Mild	3 13.0%
	Moderate	13 56.5%
	Severe	6 26.1%
PGI-S score at 3 months	Normal	0 0.0%
	Mild	8 34.8%
	Moderate	12 52.2%
	Severe	3 13.0%
PGI-S score at 6 months	Normal	0 0.0%
	Mild	9 39.1%
	Moderate	11 47.8%
	Severe	3 13.0%
PGI-S score at 12 months	Normal	1 4.3%
	Mild	6 26.1%
	Moderate	13 56.5%
	Severe	3 13.0%

9. Secondary Outcome

Title	Distribution of Patient-reported Incontinence Symptom Severity Scores - Patient Global Impression of Improvement (PGI-I) Questionnaire
Description	Patient-reported incontinence symptom severity was assessed by questionnaires at 1, 3, 6, and 12 months post-treatment using the Patient Global Impression of Improvement (PGI-I) questionnaire. The PGI-I was a global assessment of symptom severity in which participants selected the following ratings: 1-Very much better, 2-Much better, 3-A little better, 4-No change, 5-A little worse, 6-Much worse, and 7-Very much worse. Percentages of participants in each category were determined at each visit.
Time Frame	1, 3, 6, and 12 months

Outcome Measure Data

Analysis Population Description

All subjects injected

Arm/Group Title		AMDC-USR
Arm/Group Description:		Single intraurethral injection of 150 x 10^6 Autologous Muscle-Derived Cells (AMDCs)
Overall Number of Participants Analyzed		23
Measure Type: Count of Participants; Unit of Measure: Participants
PGI-I score at 1 month	Very much better	1 4.3%
	Much better	0 0.0%
	A little better	4 17.4%
	No change	18 78.3%
	A little worse	0 0.0%
PGI-I score at 3 months	Very much better	0 0.0%
	Much better	1 4.3%
	A little better	12 52.2%
	No change	9 39.1%
	A little worse	1 4.3%
PGI-I score at 6 months	Very much better	0 0.0%
	Much better	3 13.0%
	A little better	11 47.8%
	No change	7 30.4%
	A little worse	2 8.7%
PGI-I score at 12 months	Very much better	0 0.0%
	Much better	4 17.4%
	A little better	11 47.8%
	No change	5 21.7%
	A little worse	3 13.0%

10. Secondary Outcome

Title	Median Change From Baseline in Patient-reported Erectile Dysfunction (ED) - Index of Erectile Function (IIEF-5) Questionnaire
Description	SUI and ED can be comorbidities that develop following prostate surgery; median change from baseline in patient-reported Erectile Dysfunction (ED) was assessed by the 5-Item International Index of Erectile Function (IIEF-5). The IIEF-5 questionnaire was a validated 5-item tool used to assess to presence and severity of erectile dysfunction. Scored from 5 to 25, with lower scores indicating less ED symptoms.
Time Frame	Baseline, 1, 3, 6, and 12 months

Outcome Measure Data

Analysis Population Description

All subjects injected

Arm/Group Title	AMDC-USR
Arm/Group Description:	Single intraurethral injection of 150 x 10^6 Autologous Muscle-Derived Cells (AMDCs)
Overall Number of Participants Analyzed	23
Median (Full Range); Unit of Measure: scores on a scale
Change in IEEF-5 score at 1 month	0; (-9 to 8)
Change in IEEF-5 score at 3 months	0; (-7 to 8)
Change in IEEF-5 score at 6 months	0; (-11 to 4)
Change in IEEF-5 score at 12 months	0; (-8 to 7)

Adverse Events

Time Frame	Adverse events were monitored from enrollment through study exit - 24 months total.
Adverse Event Reporting Description	All adverse events were collected at baseline, 1 month, 3 months, 6 months, 12 months, and 24 months post-treatment. Any adverse events occurring in ≥ 5% of participants are reported. Adverse events assessed as AMDC product-related or biopsy procedure-related were reported regardless of frequency threshold. No injection procedure-related adverse events were reported.
Arm/Group Title	AMDC-USR
Arm/Group Description	Single intraurethral injection of 150 x 10^6 Autologous Muscle-Derived Cells (AMDCs)
All-Cause Mortality
	AMDC-USR
	Affected / at Risk (%)
Total	0/25 (0.00%)
Serious Adverse Events
	AMDC-USR
	Affected / at Risk (%)	# Events
Total	4/25 (16.00%)
Cardiac disorders
Myocardial infarction † 1	1/25 (4.00%)	1
Infections and infestations
Pneumonia † 1	1/25 (4.00%)	1
Musculoskeletal and connective tissue disorders
Spinal column stenosis † 1	1/25 (4.00%)	1
Spondylolisthesis † 1	1/25 (4.00%)	1
Nervous system disorders
Cerebrovascular accident † 1	1/25 (4.00%)	1
Renal and urinary disorders
Nephrolithiasis † 1	1/25 (4.00%)	2
1 Term from vocabulary, MedDRA version 15.0; † Indicates events were collected by systematic assessment
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	0%
	AMDC-USR
	Affected / at Risk (%)	# Events
Total	9/25 (36.00%)
Gastrointestinal disorders
Abdominal Pain † 1 [1]	2/25 (8.00%)	2
Infections and infestations
Sinusitis † 1 [1]	2/25 (8.00%)	7
Laceration † 1 [1]	2/25 (8.00%)	2
Injury, poisoning and procedural complications
Post procedural contusion † 1 [2]	2/25 (8.00%)	2
Post procedural swelling † 1 [3]	1/25 (4.00%)	1
Procedural hypotension † 1 [3]	1/25 (4.00%)	1
Procedural nausea † 1 [3]	1/25 (4.00%)	1
Procedural pain † 1 [2]	3/25 (12.00%)	3
Fall † 1 [1]	2/25 (8.00%)	2
Musculoskeletal and connective tissue disorders
Arthralgia † 1 [4]	2/25 (8.00%)	2
Nervous system disorders
Hypoaesthesia † 1 [3]	1/25 (4.00%)	1
Renal and urinary disorders
Micturition urgency † 1 [5]	1/25 (4.00%)	1
Pollakiuria † 1 [5]	1/25 (4.00%)	1
Skin and subcutaneous tissue disorders
Erythema † 1 [3]	1/25 (4.00%)	1
Hyperhidrosis † 1 [3]	1/25 (4.00%)	1
1 Term from vocabulary, MedDRA version 15.0; † Indicates events were collected by systematic assessment; [1] AEs Not Related to Study Product or Procedures with ≥5% Frequency; [2] Biopsy Procedure-Related Adverse Event, AEs with ≥5% Frequency; [3] Biopsy Procedure-Related Adverse Event; [4] 1 Event Biopsy Procedure-Related Adverse Event, 1 Event Not Related to Study Product or Procedures, AEs with ≥5% Frequency; [5] Study Product-Related Adverse Event

Limitations and Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

Results Point of Contact

• Name/Title: Ron Jankowski, PhD
• Organization: Cook MyoSite Incorporated
• Phone: 412-963-7380
• EMail: Ron.Jankowski@CookMyosite.com

• Responsible Party: Cook MyoSite
• ClinicalTrials.gov Identifier: NCT02291432
• Other Study ID Numbers: 13-11
• First Submitted: November 11, 2014
• First Posted: November 14, 2014
• Results First Submitted: August 26, 2021
• Results First Posted: October 26, 2021
• Last Update Posted: November 1, 2021