Safety of Human Embryonic Stem Cell (hESC)-Derived Mesenchymal Stem Cells in Interstitial Cystitis

Study Overview
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Brief Summary
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This study primarily aimed to evaluate the safety of human embryonic stem cell (hESC)-derived mesenchyma stem cells in interstitial cystitis.

Official Title
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Safety of Human Embryonic Stem Cell (hESC)-Derived Mesenchymal Stem Cells in Interstitial Cystitis; Open-labelled, Single Center, Phase 1 Study

Conditions
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Interstitial Cystitis, Stem Cell Transplant, Mesenchymal Stem Cell

Intervention / Treatment
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* Drug: MR-MC-01


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Female, aged >= 20 years Interstitial cystitis symptom duration more than 6 months Presence of Hunner lesions in outpatient cystoscopy (within one months of screening), with size < 2cm, number <= 2 VAS (Visual pain analogue scale) >=4 in screening symptom questionnaire Those who are suitable for stem cell transplantation normal laboratory findings (hematological, chemical) no history of drug abuse negative HIV, HBV, HCV serology tests No history of malignancies willing to contraception no plan for blood, tissue donation Who can understand consent form and willing to participate in the study Exclusion Criteria: recurrent urinary tract infection ( more than twice per past six months or more than three times per past one year) or active urinary tract infection any active or past history of tuberculosis or systemic infection Anatomical abnormality of lower urinary tract History of following procedures stem cell transplantation In past 6 months, transurethral resection/fulguration of Hunner lesion or hydrodistension of bladder intravesical instillation of ialuril hysterectomy, anti-incontinence surgery, transvaginal surgery, pelvic organ prolapse repair, vagina delivery or C/sec any neurological conditions including cerebrovascular disease, multiple sclerosis, spinal cord injury, Parkinson disease indwelling Foley catheter or intermittent catheterization any plans for electrostimulation, neuromodulation, physiotherapy or operation for other organs any history of malignancy history of myocardiac infarction in past 12 months Uncontrolled diabetes (HbAlc >= 7.2%) or diabetes requiring insulin injection Uncontrolled hypertension (systolic >170mmHg or <90mmHg, diastolic >100mmHg or <50mmHg) Immunodeficiency Positive HBV, HCV, HIV, syphilis pregnant or on breast feeding any history of drug, alcohol abuse. mis-use Any significant signs, symptoms or previous diagnosis of psychological disorder Impossible to follow scheduled visits Currently participating or participated in other clinical studies within past 3 months Allergic to protein products (serum), antibiotics (gentamicin), DMSO (Dimethyl sulfoxide) Any circumstances that is not suitable for participating or continuing clinical study or participants who clinical investigator considers not suitable for participation

Ages Eligible for Study
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Minimum Age: 20 Years

Sexes Eligible for Study
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Female

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Stem cell group<br>Interstitial cystitis patients who receive submucosal injection of hESC-MSCs | Drug: MR-MC-01<br>* Submucosal injection of 2.0 x 10,000,000/5ml (1mL * 5 sites)<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of Treatment-Emergent Adverse Events (TEAEs) | Any TEAEs reported by patients or assessed by laboratory and image work up | One month after stem cell injection | 
| Incidence of Treatment-Emergent Adverse Events | Any TEAEs reported by patients or assessed by laboratory and image work up | Three months after stem cell injection | 
| Incidence of Treatment-Emergent Adverse Events | Any TEAEs reported by patients or assessed by laboratory and image work up | Six months after stem cell injection | 
| Incidence of Treatment-Emergent Adverse Events | Any TEAEs reported by patients or assessed by laboratory and image work up | Nine months after stem cell injection | 
| Incidence of Treatment-Emergent Adverse Events | Any TEAEs reported by patients or assessed by laboratory and image work up | Twelve months after stem cell injection | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Changes of pain after stem cell injection | Assessed by VAS (Visual Analog Scale) | Changes of Pain from baseline, 1, 3, 6, and 12 months after stem cell injection | 
| Changes of PUF (Pelvic Pain and Urgency/Frequency) scores after stem cell injection | Assessed by PUF questionnaire | Changes of PUF score from baseline, 1, 3, 6, 12 months after stem cell injection | 
| Changes of ICQ (O'Leary-Sant interstitial cystitis symptom index/problem index) scores after stem cell injection | Assessed by ICQ questionnaire | Changes of ICQ scores from baseline, 1, 3, 6, 12 months after stem cell injection | 
| Changes of voiding profiles after stem cell injection | Assessed by voiding diary (frequency, nocturia, urgency, urge incontinence and mean voided volume) | Changes of voiding profiles from baseline, 1, 3, 6, 12 months after stem cell injection | 
| Changes of Hunner lesion after stem cell injection | Assessed by cystoscopy (number, sized, location) | Changes of Hunner lesion from baseline, 1, 3, 6, 12 months after stem cell injection |