Trial Outcomes & Findings for An Investigation of the Safety of 4 Different Doses of Autologous Muscle Derived Cells as Therapy for Stress Urinary Incontinence (NCT NCT00847535)

Results Overview

Biopsy was required to generate AMDC products. Biopsy procedure-related events were defined as systemic responses to the biopsy procedure or injury at the biopsy site. Since biopsy occurred prior to AMDC treatment, results are presented independent of AMDC dose received. All biopsy procedure-related events either self-resolved or were easily treated.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

66 participants

Primary outcome timeframe

at biopsy or between biopsy and treatment

Results posted on

2023-09-08

Participant Flow

66 participants were biopsied, 64 participants received injections.

Participant milestones

Participant milestones
Measure	Part I: Transurethral Injection Autologous Muscle-Derived Cells (AMDC) are intended for use in the treatment of stress urinary incontinence (SUI) in women. Following skeletal muscle biopsy of the thigh to obtain starting material for production of AMDC, patients underwent intrasphincteric injection of AMDC.	Part I: Periurethral Injection Autologous Muscle-Derived Cells (AMDC) are intended for use in the treatment of stress urinary incontinence (SUI) in women. Following skeletal muscle biopsy of the thigh to obtain starting material for production of AMDC, patients underwent intrasphincteric injection of AMDC.	Part II: Transurethral Injection Autologous Muscle-Derived Cells (AMDC) are intended for use in the treatment of stress urinary incontinence (SUI) in women. Following skeletal muscle biopsy of the thigh to obtain starting material for production of AMDC, patients underwent intrasphincteric injection of AMDC.
Overall Study STARTED	24	24	16
Overall Study 10 Million AMDC	8	8	0
Overall Study 50 Million AMDC	8	8	0
Overall Study 100 Million AMDC	8	8	8
Overall Study 200 Million AMDC	0	0	8
Overall Study COMPLETED	22	22	15
Overall Study NOT COMPLETED	2	2	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Part I: Transurethral Injection Autologous Muscle-Derived Cells (AMDC) are intended for use in the treatment of stress urinary incontinence (SUI) in women. Following skeletal muscle biopsy of the thigh to obtain starting material for production of AMDC, patients underwent intrasphincteric injection of AMDC.	Part I: Periurethral Injection Autologous Muscle-Derived Cells (AMDC) are intended for use in the treatment of stress urinary incontinence (SUI) in women. Following skeletal muscle biopsy of the thigh to obtain starting material for production of AMDC, patients underwent intrasphincteric injection of AMDC.	Part II: Transurethral Injection Autologous Muscle-Derived Cells (AMDC) are intended for use in the treatment of stress urinary incontinence (SUI) in women. Following skeletal muscle biopsy of the thigh to obtain starting material for production of AMDC, patients underwent intrasphincteric injection of AMDC.
Overall Study Withdrawal by Subject	2	1	1
Overall Study Lost to Follow-up	0	1	0

Baseline Characteristics

An Investigation of the Safety of 4 Different Doses of Autologous Muscle Derived Cells as Therapy for Stress Urinary Incontinence

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Patients Treated With AMDC n=64 Participants Autologous Muscle-Derived Cells (AMDC) are intended for use in the treatment of stress urinary incontinence (SUI) in women.
Age, Continuous	54 years STANDARD_DEVIATION 1 • n=5 Participants
Sex: Female, Male Female	64 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: at biopsy or between biopsy and treatment

Population: During the study, 66 patients underwent a total of 78 biopsies.

Biopsy was required to generate AMDC products. Biopsy procedure-related events were defined as systemic responses to the biopsy procedure or injury at the biopsy site. Since biopsy occurred prior to AMDC treatment, results are presented independent of AMDC dose received. All biopsy procedure-related events either self-resolved or were easily treated.

Outcome measures

Outcome measures
Measure	Patients With Biopsy n=66 Participants Autologous Muscle-Derived Cells (AMDC) are intended for use in the treatment of stress urinary incontinence (SUI) in women.
Number of Participants That Experienced Biopsy Procedure-related Adverse Events	3 participants

PRIMARY outcome

Timeframe: at biopsy or between biopsy and treatment

Biopsy was required to generate AMDC products. Biopsy procedure-related events were defined as systemic responses to the biopsy procedure or injury at the biopsy site. Since biopsy occurred prior to AMDC treatment, results are presented independent of AMDC dose received. All biopsy procedure-related events either self-resolved or were easily treated.

Outcome measures

Outcome measures
Measure	Patients With Biopsy n=66 Participants Autologous Muscle-Derived Cells (AMDC) are intended for use in the treatment of stress urinary incontinence (SUI) in women.
Biopsy Procedure-related Adverse Events Wound hematoma	2 Number of events
Biopsy Procedure-related Adverse Events Post procedural hemorrhage	1 Number of events
Biopsy Procedure-related Adverse Events Joint swelling	1 Number of events
Biopsy Procedure-related Adverse Events Feeling hot	1 Number of events
Biopsy Procedure-related Adverse Events Procedural dizziness	1 Number of events
Biopsy Procedure-related Adverse Events Hyperhidrosis	1 Number of events

PRIMARY outcome

Timeframe: 30 days

Population: Sixty-four patients underwent intrasphincteric injection of AMDC.

AMDC treatment was administered via intrasphincteric injection. Injection procedure-related events were defined as systemic responses to the injection procedure or genitourinary events occurring within 30 days of the injection procedure that could be attributed to cystoscopy or catheterization. Since these events could be attributed to the injection procedure, results are considered independent of AMDC dose received. All injection procedure-related events self-resolved or were easily treated.

Outcome measures

Outcome measures
Measure	Patients With Biopsy n=64 Participants Autologous Muscle-Derived Cells (AMDC) are intended for use in the treatment of stress urinary incontinence (SUI) in women.
Number of Participants That Experienced Injection Procedure-related Adverse Events	8 participants

PRIMARY outcome

Timeframe: 30 days

AMDC treatment was administered via intrasphincteric injection. Injection procedure-related events were defined as systemic responses to the injection procedure or genitourinary events occurring within 30 days of the injection procedure that could be attributed to cystoscopy or catheterization. Since these events could be attributed to the injection procedure, results are considered independent of AMDC dose received. All injection procedure-related events self-resolved or were easily treated.

Outcome measures

Outcome measures
Measure	Patients With Biopsy n=64 Participants Autologous Muscle-Derived Cells (AMDC) are intended for use in the treatment of stress urinary incontinence (SUI) in women.
Injection Procedure-related Adverse Events Dysuria	3 Number of events
Injection Procedure-related Adverse Events Vulvovaginal pruritis	3 Number of events
Injection Procedure-related Adverse Events Pelvic/abdominal pain	2 Number of events
Injection Procedure-related Adverse Events Hematuria	2 Number of events
Injection Procedure-related Adverse Events Vulvovaginal burning sensation	1 Number of events
Injection Procedure-related Adverse Events Sensation of foreign body	1 Number of events
Injection Procedure-related Adverse Events Pollakiuria	1 Number of events
Injection Procedure-related Adverse Events Micturition urgency	1 Number of events

PRIMARY outcome

Timeframe: 12 months

If an immune response after injection or any urinary retention occurred and seemed suspicious, the physicians were consulted to determine whether the effect was likely related to the AMDC product. No adverse events reported during the study were adjudicated as AMDC product-related.

Outcome measures

Outcome measures
Measure	Patients With Biopsy n=64 Participants Autologous Muscle-Derived Cells (AMDC) are intended for use in the treatment of stress urinary incontinence (SUI) in women.
Number of Participants That Experienced AMDC Product-related Adverse Events	0 participants

Adverse Events

Patients

Serious events: 8 serious events

Other events: 18 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Patients n=66 participants at risk Autologous Muscle-Derived Cells (AMDC) are intended for use in the treatment of stress urinary incontinence (SUI) in women
Immune system disorders Biopsy to treatment: Drug hypersensitiviy	1.5% 1/66 • Number of events 1 • Biopsy to treatment, 12 months Post-treatment During the study, 66 patients underwent a total of 78 biopsies, and 64 patients underwent intrasphincteric injection of AMDC.
Musculoskeletal and connective tissue disorders Biopsy to treatment: Femoral neck fracture	1.5% 1/66 • Number of events 1 • Biopsy to treatment, 12 months Post-treatment During the study, 66 patients underwent a total of 78 biopsies, and 64 patients underwent intrasphincteric injection of AMDC.
Cardiac disorders Post-treatment: Angina unstable	1.6% 1/64 • Number of events 1 • Biopsy to treatment, 12 months Post-treatment During the study, 66 patients underwent a total of 78 biopsies, and 64 patients underwent intrasphincteric injection of AMDC.
Cardiac disorders Post-treatment: Cardiac failure congestive	1.6% 1/64 • Number of events 1 • Biopsy to treatment, 12 months Post-treatment During the study, 66 patients underwent a total of 78 biopsies, and 64 patients underwent intrasphincteric injection of AMDC.
Gastrointestinal disorders Post-treatment: Enterocolitis infectious	1.6% 1/64 • Number of events 1 • Biopsy to treatment, 12 months Post-treatment During the study, 66 patients underwent a total of 78 biopsies, and 64 patients underwent intrasphincteric injection of AMDC.
Surgical and medical procedures Post-treatment: Intervertebral disc operation	1.6% 1/64 • Number of events 1 • Biopsy to treatment, 12 months Post-treatment During the study, 66 patients underwent a total of 78 biopsies, and 64 patients underwent intrasphincteric injection of AMDC.
Musculoskeletal and connective tissue disorders Post-treatment: Intervertebral disc protrusion	1.6% 1/64 • Number of events 1 • Biopsy to treatment, 12 months Post-treatment During the study, 66 patients underwent a total of 78 biopsies, and 64 patients underwent intrasphincteric injection of AMDC.
Cardiac disorders Post-treatment: Myocardial infarction	1.6% 1/64 • Number of events 1 • Biopsy to treatment, 12 months Post-treatment During the study, 66 patients underwent a total of 78 biopsies, and 64 patients underwent intrasphincteric injection of AMDC.
Gastrointestinal disorders Post-treatment: Small intestinal obstruction	1.6% 1/64 • Number of events 1 • Biopsy to treatment, 12 months Post-treatment During the study, 66 patients underwent a total of 78 biopsies, and 64 patients underwent intrasphincteric injection of AMDC.

Other adverse events

Other adverse events
Measure	Patients n=66 participants at risk Autologous Muscle-Derived Cells (AMDC) are intended for use in the treatment of stress urinary incontinence (SUI) in women
Renal and urinary disorders Biopsy to treatment: Urinary tract infection	6.1% 4/66 • Number of events 4 • Biopsy to treatment, 12 months Post-treatment During the study, 66 patients underwent a total of 78 biopsies, and 64 patients underwent intrasphincteric injection of AMDC.
Respiratory, thoracic and mediastinal disorders Post-treatment: Bronchitis	6.2% 4/64 • Number of events 4 • Biopsy to treatment, 12 months Post-treatment During the study, 66 patients underwent a total of 78 biopsies, and 64 patients underwent intrasphincteric injection of AMDC.
Renal and urinary disorders Post-treatment: Dysuria	7.8% 5/64 • Number of events 5 • Biopsy to treatment, 12 months Post-treatment During the study, 66 patients underwent a total of 78 biopsies, and 64 patients underwent intrasphincteric injection of AMDC.
Renal and urinary disorders Post-treatment: Pollakiuria	6.2% 4/64 • Number of events 4 • Biopsy to treatment, 12 months Post-treatment During the study, 66 patients underwent a total of 78 biopsies, and 64 patients underwent intrasphincteric injection of AMDC.
Renal and urinary disorders Post-treatment: Urinary tract infection	7.8% 5/64 • Number of events 9 • Biopsy to treatment, 12 months Post-treatment During the study, 66 patients underwent a total of 78 biopsies, and 64 patients underwent intrasphincteric injection of AMDC.

Additional Information

Ron Jankowski, PhD

Cook MyoSite, Inc.

Phone: 412-963-7380

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: GT60