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

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

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

Study Overview

• Status: Unknown
• Conditions: Stem Cell Transplant; Interstitial Cystitis; Mesenchymal Stem Cell
• Intervention / Treatment: Drug: MR-MC-01

• Study Type: Interventional
• Enrollment (Anticipated): 3
• Phase: Phase 1

Contacts and Locations

Study Locations

• Korea, Republic of
  • Seoul, Korea, Republic of, 138-736
    • Recruiting
    • Department of Urology, Asan Medical Center
    • Contact: Myung-Soo Choo, professor; Phone Number: 82-2-3010-3735; Email: mschoo@amc.seoul.kr

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Female

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

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

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Asan Medical Center
• Collaborators: MIRAE CELL BIO
• Investigators: Principal Investigator: Myung-Soo Choo, M.D, Ph.D, Professor, Asan Medical Center

Study record dates

Study Major Dates

• Study Start (Actual): October 20, 2020
• Primary Completion (Anticipated): October 1, 2022
• Study Completion (Anticipated): December 1, 2022

Study Registration Dates

• First Submitted: October 19, 2020
• First Submitted That Met QC Criteria: October 26, 2020
• First Posted (Actual): October 30, 2020

Study Record Updates

• Last Update Posted (Actual): October 30, 2020
• Last Update Submitted That Met QC Criteria: October 26, 2020
• Last Verified: October 1, 2020

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Urologic Diseases; Urinary Bladder Diseases; Cystitis; Cystitis, Interstitial
• Other Study ID Numbers: 2019-1451

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No