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Multisystem Cell Therapy for Improvement of Urinary Continence (MUSIC)

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ClinicalTrials.gov Identifier: NCT03439527
Recruitment Status : Recruiting
First Posted : February 20, 2018
Last Update Posted : January 22, 2020
Sponsor:
Collaborators:
University Hospital Tuebingen
Salzburger Landeskliniken
Information provided by (Responsible Party):
University of Zurich

Tracking Information
First Submitted Date  ICMJE December 18, 2017
First Posted Date  ICMJE February 20, 2018
Last Update Posted Date January 22, 2020
Actual Study Start Date  ICMJE January 22, 2020
Estimated Primary Completion Date December 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: February 19, 2018)
Incidence of clinically relevant Adverse Events of MPCs or related to cell injection [ Time Frame: at 3 months post-implantation ]
Number of clinically relevant findings related to cell injection
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: February 19, 2018)
  • Feasibility of MPC injection [ Time Frame: day of implantation ]
    Percentage of subjects with successful injection
  • Efficacy of MPC injection measured by post-void residual volume [ Time Frame: at baseline, day of implantation, 1 month, 3 months and 6 months post-implantation ]
    post-void residual volume
  • Efficacy of MPC injection measured by Uroflowmetry [ Time Frame: at baseline, day of implantation, 1 month, 3 months and 6 months post-implantation ]
    Uroflowmetry: urinary flow pattern
  • Efficacy of MPC injection measured by Urodynamic Evaluation [ Time Frame: at baseline, 3 months and 6 months post-implantation ]
    2 Filling-Cystometries
  • Efficacy of MPC injection measured by Urodynamic Evaluation [ Time Frame: at baseline, 3 months and 6 months post-implantation ]
    2 Pressure-Flow studies
  • Efficacy of MPC injection measured by Urodynamic Evaluation [ Time Frame: at baseline, 3 months and 6 months post-implantation ]
    Urethal-Pressure-Profile
  • Efficacy of MPC injection measured by 1h pad test [ Time Frame: at baseline, 1 month, 3 months and 6 months post-implantation ]
    1h pad test
  • Efficacy measured by average Incontinence Score [ Time Frame: at baseline, 1 month, 3 months and 6 months post-implantation ]
    Incontinence Score. Range from 0 (no incentinence) to 21 (highly incontinent)
  • Efficacy measured by average score of Visual Analog Scale of degree of suffering [ Time Frame: at baseline, 1 month, 3 months and 6 months post-implantation ]
    Visual Analog Scale of degree of suffering. Range 0 (worst) to 10 (best)
  • Efficacy measured by average score of Quality of Life [ Time Frame: at baseline, 1 month, 3 months and 6 months post-implantation ]
    Quality of Life Score, using SF-36v2™ Health Survey
  • Efficacy [ Time Frame: 6 months post-implantation ]
    Number for (planned) subsequent incontinence surgery
  • Number of patients with abnormal laboratory values and/or Adverse Events that are related to MPC injection [ Time Frame: at baseline, day of implantation, 1 month, 3 months and 6 months post-implantation ]
    Safety measures (ultrasound, physical examination, laboratroy)
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Multisystem Cell Therapy for Improvement of Urinary Continence
Official Title  ICMJE Open-label, Monocentric, First-in-man Trial to Assess Safety and Tolerability of a New Therapeutic Strategy for Stress Urinary Incontinence Based on the Implantation of Muscle Precursor Cells (MPCs)
Brief Summary The rising success of cell therapies places an increasing burden on health care costs. Consequently, the need to reduce production costs while maintaining quality has been widely acknowledged. In addition, the demand for high-quality products with an optimal safety profile is increasing. The proposed cell treatment is the first therapeutical option with the possibility to revert the underlying condition. The investigators expect that this healing response will be achieved with minimal side effects justifying the addional costs and complexity.
Detailed Description

muscle precursor cells (MPCs) will be isolated from biopsies of the lower leg of patients, seeded, expanded and cultivated in vitro in a GMP facility to generate vital muscle cells dedicated for implantation.

These muscle cells exhibit myogenic phenotype (IHC and flow cytometry) and therefore morphological and histological prerequisites approximating the properties of native skeletal muscle tissue. The muscle progenitor cells shall be applied in external sphincter muscle for the treatment of patients with stress urinary incontinence.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Intervention Model Description:

Open-label, monocentric, first-in-man trial to assess safety and tolerability of a new therapeutic strategy for stress urinary incontinence based on the implantation of muscle precursor cells (MPCs) 40 female patients suffering from urinary incontinence since at least 6 months (predominant clinical diagnosis of SUI) and who are candidate for a surgical treatment will be included .

All patients are treated by the same product: Autologous MPCs. After treatment, the patients will be randomized 1:1 in the MPC-group or NMES+MPC-group to investigate the benefits of an additional physiotherapy.

Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Urinary Stress Incontinence
Intervention  ICMJE Other: Muscle Precursor Cells (MPCs), ATMP
The advanced therapeutic investigational product, the MPCs, is a living, autologous cell-product isolated from a biopsy of the patient's own musculus soleus.
Study Arms  ICMJE
  • Experimental: MPC-group
    All patients are treated by the same product: Autologous MPCs
    Intervention: Other: Muscle Precursor Cells (MPCs), ATMP
  • NMES+MPC-group
    After treatment, the patients will be randomized 1:1 in the MPC-group or NMES+MPC-group to investigate the benefits of an additional physiotherapy (Neuromuscular Electromagnetic Stimulation, NMES)
    Intervention: Other: Muscle Precursor Cells (MPCs), ATMP
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: February 19, 2018)
40
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 2022
Estimated Primary Completion Date December 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

Adult women 20y-60y

  • Incontinence >/= grade I since at least 6 months
  • Predominant clinical diagnosis of SUI
  • Candidate for a surgical treatment (artificial urinary sphincter, synthetic compressive tapes or adjustable balloons).
  • Post void residual volume of <100 ml (exclusion of overflow incontinence)
  • Can independently use toilet without difficulty
  • Capacity to answer the questionnaires of evaluation
  • Negative blood test for: Human immunodeficiency virus (HIV 1/2), Hepatitis B HBsAg, Anti HBc, Hepatitis C Anti-HCV-Ab, Syphilis
  • Competent to comprehend, sign and date informed consent form before any study-specific procedure is performed

Exclusion Criteria:

  • History of anti-incontinence or prolapse surgery.
  • Previous diagnosis of any of the following conditions, disorders, or diseases of the urinary tract:

    • Clinically significant cystocele or rectocele
    • Ureteric bladder, urethral or rectal fistula
    • Uncorrected congenital abnormality leading to urinary incontinence
    • Interstitial cystitis
  • Urinary urgency that results in leakage (as a predominant symptom)
  • Adult enuresis
  • Urodynamically proven detrusor instability
  • Sensory urgency defined as first sensation of bladder fill (urge to void) of <100 ml; bladder capacity of <300 ml
  • No sensation at any time during the simple filling cystometry procedure
  • Known urethral stenosis (ureterocystoscopy) or urethral diverticulum
  • History of urogenital cancer or history of pelvic radiotherapy
  • Women who are pregnant, breast feeding or <12 months postpartum Note: Female subjects who are surgically sterilized, hysterectomized or post-menopausal for longer than 2 years are not considered as having child bearing potential. No pregnancy test will be performed for this population.
  • Untreated symptomatic urinary tract infection
  • Fever (as defined by ≥ 38,5°C, axillar measurement), any infectious disease, cold or flu within the last 7 days
  • Unstable severe systemic disease including uncontrolled hypertension, unstable angina, or myocardial infarction, severe coagulation disorders, bleeding diathesis, emboli, thrombophlebitis, infectious diseases, poor wound healing, and poorly controlled diabetes mellitus within 6 months before enrolment
  • Any organic or psychiatric disorder that, in the opinion of the investigator, might prevent the subject from completing the study or interfere with the interpretation of the study results.
  • Known allergy or intolerance of at least one of the active ingredients or excipients of the investigational products
  • Known allergy or intolerance of Penicillin or Streptomycin
  • Known genetically determined or acquired muscular disease.
  • Known Neurological disorder (Parkinson's disease, multiple sclerosis, spina bifida, medullary traumatism).
  • Medication regimen including estrogens, anti-estrogens or diuretics where dose and/or frequency has not been stable for at least the past 12 weeks or is anticipated to change during the course of the study.
  • Chronic use of any of the following drugs and not stopped for at least 2 weeks prior to inclusion into the study: selective serotonin and norepinephrine reuptake inhibitor antidepressant (SSNRI), alpha-receptor antagonists/agonists, beta-3-receptor agonists or anticholinergic/-muscarinic drugs.
  • Chronic use of any of the following drugs and not stopped for at least 6 months prior to inclusion into the study: Antidepressants or neuroleptic drugs.
Sex/Gender  ICMJE
Sexes Eligible for Study: Female
Gender Based Eligibility: Yes
Gender Eligibility Description: due to injection technique
Ages  ICMJE 20 Years to 60 Years   (Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Daniel Eberli, Prof +41432530264 daniel.eberli@usz.ch
Contact: Deana Mohr-Haralampieva, Dr +4144 255 9008 deana.haralampieva@usz.ch
Listed Location Countries  ICMJE Switzerland
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03439527
Other Study ID Numbers  ICMJE H2020-SC1-2016-2017
731377 ( Other Grant/Funding Number: European Commission )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Undecided
Responsible Party University of Zurich
Study Sponsor  ICMJE University of Zurich
Collaborators  ICMJE
  • University Hospital Tuebingen
  • Salzburger Landeskliniken
Investigators  ICMJE
Principal Investigator: Daniel Eberli, Prof University of Zurich
PRS Account University of Zurich
Verification Date January 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP