Autologous Muscle Derived Cells for Underactive Bladder
Study Details
Study Description
Brief Summary
This is a prospective, open-label, Phase 1, single center study evaluating the safety and efficacy of Autologous Muscle Derived Cells as a treatment for chronic Underactive Bladder.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 1 |
Detailed Description
Subject that meet inclusion and exclusion criteria will be consented and enrolled in this study. Initial screening includes blood and urine testing, questionnaires, physical examination, specialized bladder function testing and review of medical history. At the next visit a small sample of each subject's own thigh muscle is obtained by needle biopsy. This cell biopsy is sent to a specialized lab for growth and processing. Approximately ten weeks later the frozen cells will be sent back to the treatment site for thawing, diluting and injection under lighted instrumentation into the subject's bladder wall. The subject will be followed for 6 months and at that time may be eligible for a second injection of cells. The study doctor will determine if the second injection may be beneficial to the subject. All subjects will be followed for up to 12 months. The follow-up assessments at 12 months will include questionnaires, urine and blood testing, specialized bladder function testing and visualizing the inside of the bladder with a lighted instrument.
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: 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. |
Biological: 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.
|
Outcome Measures
Primary Outcome Measures
- Number of Participants With Study-related Adverse Events at 6 Months [6 months]
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.
Secondary Outcome Measures
- Number of Participants With Study-related Adverse Events Between 6 and 12 Months [Between 6 and 12 months]
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.
- Number of Participants With Improvement in Underactive Bladder Symptoms at 12 Months. [12 months]
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.
- Number of Participants With Improvement in Underactive Bladder Symptoms at 12 Months. [12 months]
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.
- Change in Incontinence Questionnaire (ICIQ) Long Form Score From Baseline at 12 Months. [12 months]
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.
- Change in Underactive Bladder Questionnaire (UABQ) Score From Baseline at 12 Months [12 months]
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.
- The Change in Number of Independent Voiding Occurrences Per Day on the 3-day Bladder Diary From Baseline at 12 Months. [12 months]
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.
- 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. [12 months]
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.
- 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. [12 months]
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.
- 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. [12 months]
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.
- 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. [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.
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Males and females, at least 18 years of age
-
History of Underactive Bladder (UAB) for at least 6 months documented in the medical record
-
Recurring UAB symptoms
-
Subjects unresponsive to relief symptoms of UAB with previous use of medications and/or other treatments
-
Voiding difficulty (complains of difficulty emptying the bladder)
-
Post void residual greater than or equal to 150 mL
-
Total UAB Questionnaire Score greater than or equal to 3
-
Females of child-bearing potential agree to use a reliable form of birth control for the entire study duration
-
Willing and capable of understanding and complying with all requirements of the protocol, including proper completion of the voiding diaries and self-administered questionnaires
Exclusion Criteria:
-
Pregnant, plans to become pregnant or lactating
-
History of bleeding diathesis, uncorrectable coagulopathy, or would refuse a blood transfusion
-
Currently on anticoagulant therapy
-
Obvious neurological impairment
-
Known allergy or hypersensitivity to bovine proteins or allergens, gentamicin sulfate, ampicillin, and/or lidocaine that medically warrants exclusion as determined by the physician
-
Simultaneously participating in another investigational drug or device study or use of any investigational drug(s) or therapeutic device(s) within 3 months preceding enrollment
-
Has been treated with an investigational device, drug, or procedure for UAB within the last 6 months.
-
Medical condition or disorder that may limit life expectancy or that may cause protocol deviations (e.g. unable to perform self-evaluations and/or accurately report medical history, urinary symptoms, and/or data)
-
History of cancer in pelvic organs, ureters, or kidneys or any cancer that has undergone treatment within the past 12 months
-
Compromised immune system due to disease state, chronic corticosteroid use, or other immunosuppressive therapy
-
History of radiation therapy to the bladder
-
Tests positive for Hepatitis B (Hepatitis B Surface Antigen), and Anti-Hepatitis B Core Total Antibody, Hepatitis C (Anti-Hepatitis C virus enzyme immunoassay, Human Immunodeficiency Virus (HIV Type I and 2 Antibodies), and/or Syphilis
-
Abnormal renal function
-
An active urinary tract infection as evidenced by positive urine culture
-
Taking medication(s) that affect urination (e.g. medically necessary, stable drugs) such as prescription drugs, over-the-counter drugs, or dietary supplements, including herbal supplements and those taken with teas
-
Requires concomitant use of or treatment with immunosuppressive agents
-
Pelvic organ prolapse beyond the introitus (e.g., cystocele, rectocele)
-
Abnormal bladder capacity (i.e., less than 100 mL)
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Beaumont Health | Royal Oak | Michigan | United States | 48073 |
Sponsors and Collaborators
- Jason Gilleran
Investigators
- Principal Investigator: Jason Gilleran, MD, Beaumont Health
Study Documents (Full-Text)
More Information
Publications
None provided.- 2015-134
Study Results
Participant Flow
| Recruitment Details | |
|---|---|
| Pre-assignment Detail |
| 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 |
| Period Title: Enrollment Through 6 Months | |
| STARTED | 20 |
| COMPLETED | 18 |
| NOT COMPLETED | 2 |
| Period Title: Enrollment Through 6 Months | |
| STARTED | 18 |
| COMPLETED | 17 |
| NOT COMPLETED | 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 Participants | 20 |
| Age (years) [Mean (Standard Deviation) ] | |
| Mean (Standard Deviation) [years] |
61.45
(12.00)
|
| Sex: Female, Male (Count of Participants) | |
| Female |
4
20%
|
| Male |
16
80%
|
| Ethnicity (NIH/OMB) (Count of Participants) | |
| Hispanic or Latino |
0
0%
|
| Not Hispanic or Latino |
20
100%
|
| Unknown or Not Reported |
0
0%
|
| Race (NIH/OMB) (Count of Participants) | |
| American Indian or Alaska Native |
0
0%
|
| Asian |
0
0%
|
| Native Hawaiian or Other Pacific Islander |
0
0%
|
| Black or African American |
0
0%
|
| White |
20
100%
|
| More than one race |
0
0%
|
| Unknown or Not Reported |
0
0%
|
| Region of Enrollment (participants) [Number] | |
| United States |
20
100%
|
Outcome Measures
| 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. |
| Measure Participants | 20 |
| Bruise |
2
10%
|
| Pain |
1
5%
|
| Scrotum discomfort |
1
5%
|
| Scrotum swelling |
1
5%
|
| Urinary tract infection |
1
5%
|
| Vasovagal response |
1
5%
|
| 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. |
| Measure Participants | 20 |
| Urine leakage |
1
5%
|
| Bleeding at site of injection |
1
5%
|
| 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. |
| Measure Participants | 17 |
| Markedly Improved |
0
0%
|
| Moderately improved |
3
15%
|
| Slightly improved |
9
45%
|
| No change |
5
25%
|
| Slightly worse |
0
0%
|
| Moderately worse |
0
0%
|
| Markedly worse |
0
0%
|
| 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. |
| Measure Participants | 17 |
| Very much better |
0
0%
|
| Much better |
3
15%
|
| A little bit better |
9
45%
|
| No change |
5
25%
|
| A little worse |
0
0%
|
| Much worse |
0
0%
|
| Very much worse |
0
0%
|
| 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. |
| Measure Participants | 17 |
| Female (ICIQ-FLUTS) long form |
0.6667
(5.1)
|
| Male (ICIQ-MLUTS) long form |
1.071
(9.600)
|
| 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. |
| Measure Participants | 17 |
| Mean (Standard Deviation) [score on a scale] |
5.7
(2.7)
|
| 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. |
| Measure Participants | 10 |
| Mean (Standard Deviation) [number of voids/day] |
-0.665
(5.3)
|
| 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. |
| Measure Participants | 19 |
| Mean (Standard Deviation) [number of voids/day] |
-0.2094
(1.6)
|
| 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. |
| Measure Participants | 10 |
| Mean (Standard Deviation) [milliliters/day] |
-156.66
(1100.0)
|
| 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. |
| Measure Participants | 14 |
| Mean (Standard Deviation) [milliliters/day] |
-894.9
(2100)
|
| 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. |
| Measure Participants | 20 |
| Mean (Standard Deviation) [bladder leaks per day] |
0
(0)
|
Adverse Events
| Time Frame | 18 months | |
|---|---|---|
| Adverse Event Reporting Description | ||
| 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 (%) | # Events | |
| Total | 0/20 (0%) | |
Serious Adverse Events |
||
| Autologous Muscle Derived Cells | ||
| Affected / at Risk (%) | # Events | |
| Total | 4/20 (20%) | |
| Cardiac disorders | ||
| Aortic stenosis | 1/20 (5%) | 1 |
| Musculoskeletal and connective tissue disorders | ||
| Closed fracture of tibia | 1/20 (5%) | 1 |
| Fracture of Humerus | 1/20 (5%) | 1 |
| Fracture of femoral neck | 1/20 (5%) | 1 |
| Renal and urinary disorders | ||
| Pyelonephritis | 1/20 (5%) | 1 |
Other (Not Including Serious) Adverse Events |
||
| Autologous Muscle Derived Cells | ||
| Affected / at Risk (%) | # Events | |
| Total | 20/20 (100%) | |
| General disorders | ||
| Fall | 2/20 (10%) | 2 |
| Fatigue | 2/20 (10%) | 2 |
| Pain | 3/20 (15%) | 3 |
| Renal and urinary disorders | ||
| Urinary tract infection | 10/20 (50%) | 20 |
| Respiratory, thoracic and mediastinal disorders | ||
| Influenza | 3/20 (15%) | 3 |
| Skin and subcutaneous tissue disorders | ||
| Bruise | 2/20 (10%) | 2 |
Limitations/Caveats
More Information
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
There is NOT 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 | Jennifer Giordano |
|---|---|
| Organization | Beaumont Hospitals |
| Phone | 248-551-3517 |
| jennifer.geiordano@beaumont.org |
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