Retinitis pigmentosa
Conditions
Interventions
Starting dose group:Subretinal injection of starting dose of RM-101 in one eye
High dose group:Subretinal injection of high dose of RM-101 in one eye
Sponsors
Xiamen Eye Centre affiliated to Xiamen University
Eligibility
Sex/Gender
All
Age
18 Years to 60 Years
Inclusion criteria
Inclusion criteria: 1. Male for female, with age = 18 years old and = 60 years old at the time of consent. 2. Diagnosed with RP based on clinical examination and genetic testing. 3. A molecular diagnosis of homozygotes or compound heterozygotes for one or more pathogenic mutations in exon 13 of the USH2A gene, based on genetic analysis. 4. BCVA = 20/63 and = 20/4000 Snellen equivalent in the study eye (hand motion [HM] ~ 60 ETDRS letters) . 5. Female subjects of childbearing potential or male subjects with partners of childbearing potential must agree to use effective contraception from signing of informed consent until at least 6 months after study treatment; surgical sterilization or postmenopausal women. 6. Volunteer to participate in this study, sign informed consent form and be willing to comply with the protocol and accept an additional long-term follow-up of approximately 4 years.
Exclusion criteria
Exclusion criteria: 1. For subjects with compound heterozygotes for mutations in exon 13, presence of additional non-exon 13 USH2A pathogenic mutation(s) on the USH2A allele carrying the exon 13 mutation. 2. For subjects with homozygous for mutations in exon 13, presence of non-exon 13 USH2A pathogenic mutation(s) on both USH2A alleles. 3. Presence of pathogenic mutations in genes (other than the USH2A gene) associated with RP or other inherited retinal degenerative diseases or syndromes. 4. Lack of sufficient viable retinal photoreceptor cells in the target eye based on the examination results in opinion of investigator. 5. Unable to complete perimetry. 6. Spherical equivalent < -8.00 D in either eye. 7. Axial length = 28 mm or = 21 mm in either eye. 8. History or presence of ocular herpetic disease (herpes simplex virus or varicella zoster infection) in either eye. 9. Presence of active ocular inflammation or periocular acute infection in either eye. 10. Presence of contraindications of dilation of pupil (e.g., narrow anterior chamber angle) in either eye. 11. History of ocular surgery in the study eye within 3 months. 12. History of retinal laser photocoagulation in the study eye within 1 month. 13. History of vitreoretinal surgery in the study eye. 14. History of amblyopia with previous BCVA < 20/80 in the study eye. 15. Having other ophthalmic diseases other than RP in the study eye, such as age-related macular degeneration, diabetic retinopathy, optic neuropathy, obvious lens opacities, ocular hypertension or glaucoma, retinal detachment and other eye diseases that, in the opinion of the investigator, may interfere with the assessment of the safety or efficacy of the investigational drug, interfere with surgical procedures or increase the risk of surgery. 16.AAV neutralizing antibody titer = 1:1000. 17.Positive etiological tests for active hepatitis B virus, hepatitis C virus, human immunodeficiency virus, treponema pallidum. 18.Prior treatment with gene therapy or cell therapy. 19. Oral administration of drugs that cause retinal toxicity and cause severe impairment of visual function, such as hydroxychloroquine, chloroquine, etc. 20. Participation in any drug or medical device clinical trial within 3 months. 21. Having live attenuated vaccine within 3 months. 22. Known hypersensitivity to any component of the investigational product and concomitant medications (such as, anesthetics, cycloplegics, glucocorticoids) or history of hypersensitivity (history of hypersensitivity to two or more drugs or foods). 23. Presence of contraindication to corticosteroids. 24. Complicated with systemic diseases that may alter ocular function, including the disease or the corresponding treatments for the disease (e.g., malignancy, diabetes, rheumatoid arthritis, systemic lupus erythematosus). 25. History of malignancy, end-stage vital organ disease, heart failure, serious cardiac arrhythmia, stroke or transient ischemic attack, diabetes, immunosuppressive status, autoimmune diseases, major mental disorders, epilepsy, chronic obstructive pulmonary disease, renal failure, or any chronic systemic disease requiring continuous systemic steroid, anticoagulant, or immunosuppressive therapy. 26.Having abnormal laboratory test, ECG or chest X-ray findings, which are judged as clinically significant and inappropriate for participation by the investigator. 27.Pregnant and lactating female. 28.History of addiction to alcohol or illicit drugs. 29.Any other condition that, in the o
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence and severity of ocular and non-ocular adverse events and serious adverse events; | — |
Secondary
| Measure | Time frame |
|---|---|
| Incidence and severity of ocular and non-ocular adverse events and serious adverse events;Full-field stimulus threshold;Best corrected visual acuity;Static perimetry;Microperimetry;Contrast sensitivity;Quality of life assessment ;Immunogenicity;Biodistribution and vector shedding; | — |
Countries
China
Contacts
Public ContactLi Xiaoxin
Xiamen Eye Centre affiliated with Xiamen University
Outcome results
None listed