Role of UC-MSC and CM to Inhibit Vision Loss in Retinitis Pigmentosa Phase I/II

Study Overview
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Brief Summary
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The study will perform UC-MSCs and CM transplantation. There are two groups with different dosages. The first group will be transplanted with 1.5 million cells, meanwhile, the second group is 5 million cells. Each group consists of 30 subjects. All groups will be transplanted via the peribulbar route. All groups will be observed until six months.

Detailed Description
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The eyes we will give the transplant should be given an aseptic and antiseptic technique to prevent contamination from the inside and outside. Sterile cover attached to other parts of the face except for the eyes. 1.8 ml Umbilical Cord-derived Mesenchymal Stem Cell (UC-MSC) preparations are suspended in Conditioned Medium (CM) until it reaches a 2 ml volume of cell suspension. Two dosages will be prepared, 1.5 million and 5 million of UC-MSC. Stem cell suspension will be injected by peribulbar, and patients will be given a quinolone antibiotic if the injection is done. On day 1st and day 7th after therapy, patients will be observed the presence of infection, inflammation, and increased eye pressure. On day 7th, day 30th, and day 90th after therapy, patients will do a visual field test, visual acuity test, electroretinography, funduscopy, and Optical Coherence Tomography examination. The observations will be written in the table and analyzed by a statistic.

Official Title
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Role of Umbilical Cord-derived Stem Cell Transplantation and Conditioned Medium to Inhibit Vision Loss in Retinitis Pigmentosa Phase I/II

Conditions
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Retinitis Pigmentosa

Intervention / Treatment
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* Biological: 1.5 x 10^6 UC-MSC + CM
* Biological: 5 x 10^6 UC-MSC + CM


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Visus more than 20/100 Have more than 0.68 uV on conical receptor cell amplitude checked by ERG Visual field equivalent diameter more than 10o Willing to sign informed consent as research subjects Willing to do the peribulbar injection with mesenchymal stem cells isolated from umbilical cord tissue Willing to do visual field checks with Humphrey's perimetry, vision tests with Snellen boards, Optical Coherent Tomography (OCT) examinations, electroretinogram examinations, and fill out a quality of life questionnaire Exclusion Criteria: Pregnant or nursing women Positive result of HIV test Have a history of eye tumors Under immunosuppressive treatment or other drugs that can affect the growth of transplanted stem cells Have another eye disease such as diabetic retinopathy, uveitis, cataract, and glaucoma Do not come to control according to the schedule determined by the researcher (loss to follow up)

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 65 Years

Sexes Eligible for Study
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All

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
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: EYESTEM 001-X<br>1.8 ml cell preparations are suspended in Conditioned Media (CM) until it reaches a 2 ml volume of cell suspension. Umbilical Cord Mesenchymal Stem Cell (UC-MSC) suspension will be injected into the peribulbar. | Biological: 1.5 x 10^6 UC-MSC + CM<br>* 1.5 million cells of UC-MSC in 2 ml of Conditioned Medium (CM)<br>|
| Experimental: EYESTEM 001-XF<br>1.8 ml cell preparations are suspended in Conditioned Media (CM) until it reaches a 2 ml volume of cell suspension. Umbilical Cord Mesenchymal Stem Cell (UC-MSC) suspension will be injected into the peribulbar. | Biological: 5 x 10^6 UC-MSC + CM<br>* 5 million cells of UC-MSC in 2 ml of Conditioned Medium (CM)<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incident of Adverse Events | assessment of infection, inflammation, eye pressure and patients complaints | 1 day after injection | 
| Incident of Adverse Events | assessment of infection, inflammation, eye pressure and patients complaints | 1 week after injection | 
| Frequency of Adverse Events | assessment of infection, inflammation, eye pressure and patients complaints | 1 day after injection | 
| Frequency of Adverse Events | assessment of infection, inflammation, eye pressure and patients complaints | 1 week after injection | 
| Visual Acuity Test | a test to see the sharpness of vision using Snellen Chart which is read at a distance of 6 meters. Normal eye test results are stated in 6/6. | 1 week after injection | 
| Visual Acuity Test | a test to see the sharpness of vision using Snellen Chart which is read at a distance of 6 meters. Normal eye test results are stated in 6/6. | 1 month after injection | 
| Visual Acuity Test | a test to see the sharpness of vision using Snellen Chart which is read at a distance of 6 meters. Normal eye test results are stated in 6/6. | 3 months after injection | 
| Visual Acuity Test | a test to see the sharpness of vision using Snellen Chart which is read at a distance of 6 meters. Normal eye test results are stated in 6/6. | 6 months after injection | 
| Visual Field Test | a test to assess the breadth of views of the lateral, medial, superior and inferior aspects using Humphrey's perimetry. | 1 week after injection | 
| Visual Field Test | a test to assess the breadth of views of the lateral, medial, superior and inferior aspects using Humphrey's perimetry. | 1 month after injection | 
| Visual Field Test | a test to assess the breadth of views of the lateral, medial, superior and inferior aspects using Humphrey's perimetry. | 3 months after injection | 
| Visual Field Test | a test to assess the breadth of views of the lateral, medial, superior and inferior aspects using Humphrey's perimetry. | 6 months after injection | 
| Funduscopy | an examination to see and assess the abnormalities and conditions in the ocular fundus using an ophthalmoscope. This examination is carried out by an ophthalmologist. | 1 week after injection | 
| Funduscopy | an examination to see and assess the abnormalities and conditions in the ocular fundus using an ophthalmoscope. This examination is carried out by an ophthalmologist. | 1 month after injection | 
| Funduscopy | an examination to see and assess the abnormalities and conditions in the ocular fundus using an ophthalmoscope. This examination is carried out by an ophthalmologist. | 3 months after injection | 
| Funduscopy | an examination to see and assess the abnormalities and conditions in the ocular fundus using an ophthalmoscope. This examination is carried out by an ophthalmologist. | 6 months after injection | 
| Electrorectinography | an examination to provide a picture of objectivity, a quantitative measure of retinal function to monitor the function of stem cells and ganglion cells in the eye. This examination is carried out by a trained ophthalmologist. | 1 week after injection | 
| Electrorectinography | an examination to provide a picture of objectivity, a quantitative measure of retinal function to monitor the function of stem cells and ganglion cells in the eye. This examination is carried out by a trained ophthalmologist. | 1 month after injection | 
| Electrorectinography | an examination to provide a picture of objectivity, a quantitative measure of retinal function to monitor the function of stem cells and ganglion cells in the eye. This examination is carried out by a trained ophthalmologist. | 3 months after injection | 
| Electrorectinography | an examination to provide a picture of objectivity, a quantitative measure of retinal function to monitor the function of stem cells and ganglion cells in the eye. This examination is carried out by a trained ophthalmologist. | 6 months after injection | 
| Optical Coherence Tomography (OCT) | is a diagnostic test that give the information about posterior segment structure, which is retina and optic nerve papillae so that it can be used to assess macular abnormalities. | 1 week after injection | 
| Optical Coherence Tomography (OCT) | is a diagnostic test that give the information about posterior segment structure, which is retina and optic nerve papillae so that it can be used to assess macular abnormalities. | 1 month after injection | 
| Optical Coherence Tomography (OCT) | is a diagnostic test that give the information about posterior segment structure, which is retina and optic nerve papillae so that it can be used to assess macular abnormalities. | 3 months after injection | 
| Optical Coherence Tomography (OCT) | is a diagnostic test that give the information about posterior segment structure, which is retina and optic nerve papillae so that it can be used to assess macular abnormalities. | 6 months after injection | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Patients Quality of Life | Patient's quality of life using National Eye Institute-Visual Functional Questioner 25 | 1 week after injection | 
| Patients Quality of Life | Patient's quality of life using National Eye Institute-Visual Functional Questioner 25 | 1 month after injection | 
| Patients Quality of Life | Patient's quality of life using National Eye Institute-Visual Functional Questioner 25 | 3 months after injection | 
| Patients Quality of Life | Patient's quality of life using National Eye Institute-Visual Functional Questioner 25 | 6 months after injection | 
| Pain evaluation | Evaluation on level of pain felt by patients | 6 months after injection | 
| Eye bump | Evaluation on appearance of eye bump | 6 months after injection | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Allogeneic Mesenchymal Stem Cell, Umbilical Cord Mesenchymal Stem Cell, Conditioned Medium, Allogeneic Umbilical Cord Mesenchymal Stem Cell