Back to resultsSafety and Tolerability of MA09-hRPE Cells in Patients With Stargardt's Macular Dystrophy(SMD)
Primary Purpose
Stargardt's Macular Dystrophy
Status
Unknown status
Phase
Phase 1
Locations
Korea, Republic of
Study Type
Interventional
Intervention
MA09-hRPE
Sponsored by
About this trial
This is an interventional treatment trial for Stargardt's Macular Dystrophy
Eligibility Criteria
Inclusion Criteria:
- Adult male or female over 20 years of age.
- Clinical diagnosis of advanced SMD.
- The visual acuity of the eye to receive the transplant will be no better than hand movement.
- The visual acuity of the eye that is not to receive the transplant will be no better than 24 (20/320) Early Treatment of Diabetic Retinopathy Study (ETDRS) letters.
Exclusion Criteria:
- History of malignancy.
- History of myocardial infarction in previous 12 months.
- History of diabetes mellitus.
- Any immunodeficiency.
- Any current immunosuppressive therapy other than intermittent or low dose cortico steroids.
- Serologic evidence of infection with Hepatitis B, Hepatitis C, or HIV.
- Current participation in any other clinical trial.
- Participation within previous 6 months in any clinical trial of a drug by ocular or systemic administration.
- Any other sight-threatening ocular disease.
- Any chronic ocular medications. Any history of retinal vascular disease (compromised blood-retinal barrier). Glaucoma. Uveitis or other intraocular inflammatory disease. Significant lens opacities or other media opacity. Ocular lens removal within previous 3 months
Sites / Locations
- CHA Bundang Medical Center
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
50,000 cells
Arm Description
Biological: MA09-hRPE Cellular therapy
Outcomes
Primary Outcome Measures
safety and tolerance of transplantation
The transplantation of hESC-derived RPE cells MA09-hRPE will be considered safe and tolerated in the absence of:
Any grade 2 (NCI grading system) or greater adverse event related to the cell product
Any evidence that the cells are contaminated with an infectious agent
Any evidence that the cells show tumorigenic potential
Secondary Outcome Measures
Evidence of successful engraftment
Evidence of successful engraftmentEvidence of successful engraftment will consist of:
Structural evidence (OCT imaging, fluorescein angiography, autofluorescense photography, slit-lamp examination with fundus photography) that cells have been implanted in the correct location Electroretinographic evidence (mfERG) showing enhanced activity in the implant location
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01625559
Brief Title
Safety and Tolerability of MA09-hRPE Cells in Patients With Stargardt's Macular Dystrophy(SMD)
Official Title
A Phase I, Open-Label, Prospective Study to Determine the Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial(MA09-hRPE) Cells in Patients With Stargardt's Macular Dystrophy(SMD)
Study Type
Interventional
2. Study Status
Record Verification Date
August 2012
Overall Recruitment Status
Unknown status
Study Start Date
September 2012 (undefined)
Primary Completion Date
March 2015 (Anticipated)
Study Completion Date
June 2015 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
CHABiotech CO., Ltd
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
The purpose of this study is:
To evaluate the safety and tolerability of RPE cellular therapy in patients with SMD Group
When-MA09-hRPE cell transplantation to evaluate the safety of surgical procedures.
In future studies intended to assess the number of transplanted hRPE cells.
In the past, MA09-hRPE cell therapy used in the study was to evaluate the validity of the potential.
Homologous retinal pigment epithelial cells derived from embryonic stem cells, future studies of drugs that are used in representing the potential validity to evaluate the optimal dose.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Stargardt's Macular Dystrophy
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
3 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
50,000 cells
Arm Type
Experimental
Arm Description
Biological: MA09-hRPE Cellular therapy
Intervention Type
Biological
Intervention Name(s)
MA09-hRPE
Intervention Description
MA09-hRPE: 50,000 cells
Primary Outcome Measure Information:
Title
safety and tolerance of transplantation
Description
The transplantation of hESC-derived RPE cells MA09-hRPE will be considered safe and tolerated in the absence of:
Any grade 2 (NCI grading system) or greater adverse event related to the cell product
Any evidence that the cells are contaminated with an infectious agent
Any evidence that the cells show tumorigenic potential
Time Frame
18 months
Secondary Outcome Measure Information:
Title
Evidence of successful engraftment
Description
Evidence of successful engraftmentEvidence of successful engraftment will consist of:
Structural evidence (OCT imaging, fluorescein angiography, autofluorescense photography, slit-lamp examination with fundus photography) that cells have been implanted in the correct location Electroretinographic evidence (mfERG) showing enhanced activity in the implant location
Time Frame
18 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
20 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Adult male or female over 20 years of age.
Clinical diagnosis of advanced SMD.
The visual acuity of the eye to receive the transplant will be no better than hand movement.
The visual acuity of the eye that is not to receive the transplant will be no better than 24 (20/320) Early Treatment of Diabetic Retinopathy Study (ETDRS) letters.
Exclusion Criteria:
History of malignancy.
History of myocardial infarction in previous 12 months.
History of diabetes mellitus.
Any immunodeficiency.
Any current immunosuppressive therapy other than intermittent or low dose cortico steroids.
Serologic evidence of infection with Hepatitis B, Hepatitis C, or HIV.
Current participation in any other clinical trial.
Participation within previous 6 months in any clinical trial of a drug by ocular or systemic administration.
Any other sight-threatening ocular disease.
Any chronic ocular medications. Any history of retinal vascular disease (compromised blood-retinal barrier). Glaucoma. Uveitis or other intraocular inflammatory disease. Significant lens opacities or other media opacity. Ocular lens removal within previous 3 months
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Wonkyung Song, MD. PhD.
Organizational Affiliation
CHA Bundang Medical Center
Official's Role
Principal Investigator
Facility Information:
Facility Name
CHA Bundang Medical Center
City
Seongnam-si
State/Province
Gyeonggi-do
ZIP/Postal Code
463-712
Country
Korea, Republic of
12. IPD Sharing Statement
Citations:
PubMed Identifier
32727729
Citation
Sung Y, Lee MJ, Choi J, Jung SY, Chong SY, Sung JH, Shim SH, Song WK. Long-term safety and tolerability of subretinal transplantation of embryonic stem cell-derived retinal pigment epithelium in Asian Stargardt disease patients. Br J Ophthalmol. 2021 Jun;105(6):829-837. doi: 10.1136/bjophthalmol-2020-316225. Epub 2020 Jul 29.
Results Reference
derived
Learn more about this trial
Safety and Tolerability of MA09-hRPE Cells in Patients With Stargardt's Macular Dystrophy(SMD)
We'll reach out to this number within 24 hrs