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Multisystem Cell Therapy for Improvement of Urinary Continence

Phase 1
Completed
Conditions
Urinary Stress Incontinence
Interventions
Other: Muscle Precursor Cells (MPCs), ATMP
Registration Number
NCT03439527
Lead Sponsor
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.

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.

Recruitment & Eligibility

Status
COMPLETED
Sex
Female
Target Recruitment
10
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.

Study & Design

Study Type
INTERVENTIONAL
Study Design
SEQUENTIAL
Arm && Interventions
GroupInterventionDescription
MPC-groupMuscle Precursor Cells (MPCs), ATMPAll patients are treated by the same product: Autologous MPCs
NMES+MPC-groupMuscle Precursor Cells (MPCs), ATMPAfter 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)
Primary Outcome Measures
NameTimeMethod
Incidence of clinically relevant Adverse Events of MPCs or related to cell injectionat 3 months post-implantation

Number of clinically relevant findings related to cell injection

Secondary Outcome Measures
NameTimeMethod
Efficacy measured by average Incontinence Scoreat baseline, 1 month, 3 months and 6 months post-implantation

Incontinence Score. Range from 0 (no incentinence) to 21 (highly incontinent)

Efficacy6 months post-implantation

Number for (planned) subsequent incontinence surgery

Feasibility of MPC injectionday of implantation

Percentage of subjects with successful injection

Efficacy of MPC injection measured by post-void residual volumeat baseline, day of implantation, 1 month, 3 months and 6 months post-implantation

post-void residual volume

Efficacy of MPC injection measured by Uroflowmetryat baseline, day of implantation, 1 month, 3 months and 6 months post-implantation

Uroflowmetry: urinary flow pattern

Efficacy of MPC injection measured by Urodynamic Evaluationat baseline, 3 months and 6 months post-implantation

Urethal-Pressure-Profile

Efficacy of MPC injection measured by 1h pad testat baseline, 1 month, 3 months and 6 months post-implantation

1h pad test

Efficacy measured by average score of Visual Analog Scale of degree of sufferingat baseline, 1 month, 3 months and 6 months post-implantation

Visual Analog Scale of degree of suffering. Range 0 (worst) to 10 (best)

Number of patients with abnormal laboratory values and/or Adverse Events that are related to MPC injectionat baseline, day of implantation, 1 month, 3 months and 6 months post-implantation

Safety measures (ultrasound, physical examination, laboratroy)

Efficacy measured by average score of Quality of Lifeat baseline, 1 month, 3 months and 6 months post-implantation

Quality of Life Score, using SF-36v2™ Health Survey

Trial Locations

Locations (1)

University Hospital Zurich

🇨🇭

Zürich, Switzerland

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