Autologous Muscle Derived Cells for Underactive Bladder
20 december 2021 bijgewerkt door: Jason Gilleran
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.
Studie Overzicht
Toestand
Voltooid
Conditie
Interventie / Behandeling
Gedetailleerde beschrijving
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.
Studietype
Ingrijpend
Inschrijving (Werkelijk)
23
Fase
- Fase 1
Contacten en locaties
In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.
Studie Locaties
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Michigan
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Royal Oak, Michigan, Verenigde Staten, 48073
- Beaumont Health
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Deelname Criteria
Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.
Geschiktheidscriteria
Leeftijden die in aanmerking komen voor studie
18 jaar en ouder (Volwassen, Oudere volwassene)
Accepteert gezonde vrijwilligers
Nee
Geslachten die in aanmerking komen voor studie
Allemaal
Beschrijving
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)
Studie plan
Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.
Hoe is de studie opgezet?
Ontwerpdetails
- Primair doel: Behandeling
- Toewijzing: NVT
- Interventioneel model: Opdracht voor een enkele groep
- Masker: Geen (open label)
Wapens en interventies
Deelnemersgroep / Arm |
Interventie / Behandeling |
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Experimenteel: 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.
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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.
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Wat meet het onderzoek?
Primaire uitkomstmaten
Uitkomstmaat |
Maatregel Beschrijving |
Tijdsspanne |
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Number of Participants With Study-related Adverse Events at 6 Months
Tijdsspanne: 6 months
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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.
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6 months
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Secundaire uitkomstmaten
Uitkomstmaat |
Maatregel Beschrijving |
Tijdsspanne |
|---|---|---|
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Number of Participants With Study-related Adverse Events Between 6 and 12 Months
Tijdsspanne: Between 6 and 12 months
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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.
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Between 6 and 12 months
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Number of Participants With Improvement in Underactive Bladder Symptoms at 12 Months.
Tijdsspanne: 12 months
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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.
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12 months
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Number of Participants With Improvement in Underactive Bladder Symptoms at 12 Months.
Tijdsspanne: 12 months
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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.
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12 months
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Change in Incontinence Questionnaire (ICIQ) Long Form Score From Baseline at 12 Months.
Tijdsspanne: 12 months
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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.
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12 months
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Change in Underactive Bladder Questionnaire (UABQ) Score From Baseline at 12 Months
Tijdsspanne: 12 months
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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.
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12 months
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The Change in Number of Independent Voiding Occurrences Per Day on the 3-day Bladder Diary From Baseline at 12 Months.
Tijdsspanne: 12 months
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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.
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12 months
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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.
Tijdsspanne: 12 months
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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.
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12 months
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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.
Tijdsspanne: 12 months
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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.
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12 months
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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.
Tijdsspanne: 12 months
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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.
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12 months
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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.
Tijdsspanne: 12 months
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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.
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12 months
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Medewerkers en onderzoekers
Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.
Sponsor
Onderzoekers
- Hoofdonderzoeker: Jason Gilleran, MD, Beaumont Health
Studie record data
Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.
Bestudeer belangrijke data
Studie start (Werkelijk)
16 november 2016
Primaire voltooiing (Werkelijk)
1 december 2019
Studie voltooiing (Werkelijk)
1 november 2020
Studieregistratiedata
Eerst ingediend
27 mei 2015
Eerst ingediend dat voldeed aan de QC-criteria
3 juni 2015
Eerst geplaatst (Schatting)
4 juni 2015
Updates van studierecords
Laatste update geplaatst (Werkelijk)
19 januari 2022
Laatste update ingediend die voldeed aan QC-criteria
20 december 2021
Laatst geverifieerd
1 december 2021
Meer informatie
Termen gerelateerd aan deze studie
Trefwoorden
Aanvullende relevante MeSH-voorwaarden
Andere studie-ID-nummers
- 2015-134
Plan Individuele Deelnemersgegevens (IPD)
Bent u van plan om gegevens van individuele deelnemers (IPD) te delen?
NEE
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