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Multisystem Cell Therapy for Improvement of Urinary Continence (MUSIC)

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ClinicalTrials.gov Identifier: NCT03439527
Recruitment Status : Not yet recruiting
First Posted : February 20, 2018
Last Update Posted : May 8, 2018
Sponsor:
Collaborators:
University Hospital Tuebingen
Salzburger Landeskliniken
Information provided by (Responsible Party):
University of Zurich

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.

Condition or disease Intervention/treatment Phase
Urinary Stress Incontinence Other: Muscle Precursor Cells (MPCs), ATMP Phase 1

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 : Interventional  (Clinical Trial)
Estimated Enrollment : 40 participants
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
Official Title: 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)
Estimated Study Start Date : January 2019
Estimated Primary Completion Date : June 2022
Estimated Study Completion Date : December 2022

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: MPC-group
All patients are treated by the same product: Autologous MPCs
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.

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)
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.




Primary Outcome Measures :
  1. 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


Secondary Outcome Measures :
  1. Feasibility of MPC injection [ Time Frame: day of implantation ]
    Percentage of subjects with successful injection

  2. 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

  3. 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

  4. Efficacy of MPC injection measured by Urodynamic Evaluation [ Time Frame: at baseline, 3 months and 6 months post-implantation ]
    2 Filling-Cystometries

  5. Efficacy of MPC injection measured by Urodynamic Evaluation [ Time Frame: at baseline, 3 months and 6 months post-implantation ]
    2 Pressure-Flow studies

  6. Efficacy of MPC injection measured by Urodynamic Evaluation [ Time Frame: at baseline, 3 months and 6 months post-implantation ]
    Urethal-Pressure-Profile

  7. 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

  8. 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)

  9. 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)

  10. 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

  11. Efficacy [ Time Frame: 6 months post-implantation ]
    Number for (planned) subsequent incontinence surgery

  12. 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)



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Ages Eligible for Study:   20 Years to 60 Years   (Adult)
Sexes Eligible for Study:   Female
Gender Based Eligibility:   Yes
Gender Eligibility Description:   due to injection technique
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03439527


Contacts
Contact: Daniel Eberli, Prof +41432530264 daniel.eberli@usz.ch
Contact: Regina Grossmann, Dr +41432530264 regina.grossmann@usz.ch

Sponsors and Collaborators
University of Zurich
University Hospital Tuebingen
Salzburger Landeskliniken
Investigators
Principal Investigator: Daniel Eberli, Prof University of Zurich

Responsible Party: University of Zurich
ClinicalTrials.gov Identifier: NCT03439527     History of Changes
Other Study ID Numbers: H2020-SC1-2016-2017
731377 ( Other Grant/Funding Number: European Commission )
First Posted: February 20, 2018    Key Record Dates
Last Update Posted: May 8, 2018
Last Verified: May 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by University of Zurich:
Muscle Precursor Cells

Additional relevant MeSH terms:
Urinary Incontinence, Stress
Urinary Incontinence
Urination Disorders
Urologic Diseases
Lower Urinary Tract Symptoms
Urological Manifestations
Signs and Symptoms