Clinical Research of Human Retinal Pigment Epithelial (HuRPE) Cell Injection on Atrophy of High Myopia Macular Area
Overview
- Phase
- Not Applicable
- Sponsor
- Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
- Enrollment
- 9
- Primary Endpoint
- Number of subjects with Local and systemic adverse events
Overview
Brief Summary
To evaluate the safety and tolerability of human retinal pigment epithelial (HuRPE) cell injection subretinal transplantation for atrophy of high myopia macular area, and to explore the maximum tolerated dose (MTD).
Detailed Description
This study is a perspective, single-arm and open-labeled investigation of the safety and preliminary efficacy of unilateral subretinal transplantation of HuRPE cells in subjects with Macular degeneration caused by high myopia. The investigators will recruit and enroll 9 patients based on specific inclusive/exclusive criteria. Experimental and self-controlled eye will be determined based on best-corrected visual acuity (BCVA). The eye with BCVA between 5 and 60 ETDRS letters will be determined as experimental eye, which will be divided into 3 groups and undergo subretinal injection of 3 different dosages of HuRPE cells (300.000, 500,000 or 1,000,000) respectively, while the other one as control eye, will not receive the surgery.
HuRPE cells will be obtained from Eyecure Therapeutics, Inc.(Jiangsu) located in WuXi, Jiangsu Province. The obtained HuRPE cells will meet its quality standards and conform to Good manufacturing practices (GMP). HuRPE cells will be transplanted by a board-certified vitreoretinal surgeon, which will be administered into the subretinal space of experimental eye through a standard surgical approach.
Immunosuppressive agents will be administered orally to all subjects after transplantation. Dosage and time duration of immunosuppressive agents will be regulated strictly relying on the condition of immune rejection. Subjects will be monitored with ophthalmologic and systemic examinations frequently at regular post-transplant intervals after HuRPE cells transplantation.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 18 Years to — (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •A patient who is able (or with the assistance of his or her legal representative) to read and understand the informed consent form and is willing to sign a written informed consent;
- •age ≥ 18 years old, male or female;
- •myopic diopter ≥ -6D or visual axis length ≥ 26mm;
- •study the best corrected visual acuity ≥ 5 letters, ≤ 60 letters (using ETDRS eye chart);
- •According to the researcher's judgment, the patient's high myopia macular atrophy is the main reason for the patient's vision loss;
- •If both eyes meet the inclusion criteria, the selected disease is more severe; the eyes are the same, and the right eye is the study eye.
Exclusion Criteria
- •The study eye has intravitreal hemorrhage within 2 months before the group visit;
- •the study of the eye involved in the foveal scar, fibrosis, suggesting a serious irreversible vision loss;
- •The study eye diagnosis is a pupillary afferent defect (APD);
- •the study eye has significant refractive interstitial opacity, and moderate to severe cataract, may cause interference to OCT and fundus examination;
- •any eye diagnosed as infectious blepharitis, keratitis, scleritis or conjunctivitis; or there are currently serious systemic infectious diseases;
- •Uncontrollable glaucoma, defined as a patient who is adequately treated with medication, with an intraocular pressure still above 25 mmHg, or who has undergone previous or filtering surgery;
- •Study the eye with other diseases that affect vision;
- •traumatic macular hole, idiopathic macular hole, combined with other parts of the retinal tear;
- •single eye blind patients;
- •The study eye is an aphakic eye (except for the intraocular lens);
Outcomes
Primary Outcomes
Number of subjects with Local and systemic adverse events
Time Frame: six months following transplantation
Descriptive analysis of frequency and types of adverse events experienced by each subject during the study period.
Secondary Outcomes
- Mean change of Best-Corrected Visual Acuity (BCVA)(six months following transplantation)
- Change in NEI VFQ-25 Total Score(six months following transplantation)
- Evidence of successful engraftment(six months following transplantation)
Investigators
Xiaodong Sun
Professor
Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine