Primary blindness and total blindness caused by retinal degeneration
Conditions
Interventions
Treatment group:Intravitreal injection of GA001
Sponsors
Beijing Tiantan Hospital, Capital Medical University
Eligibility
Sex/Gender
All
Age
18 Years to 80 Years
Inclusion criteria
Inclusion criteria: 1.Age between 18 and 80, of any gender; 2.Participants must voluntarily sign a written informed consent form; 3.Confirmed diagnosis of advanced retinitis pigmentosa; 4.Best-corrected visual acuity in the study eye during screening is worse than 1.9 LogMAR on the ETDRS visual acuity test; 5.Presence of retinal atrophy and nerve fiber layer on optical coherence tomography or abnormal visual evoked potential in the study eye during screening as determined by the investigator; 6.Normal laboratory values during screening; 7.Normal liver function tests during screening; 8.Male or female participants of reproductive potential must agree to use effective contraception from the time of signing the informed consent form through at least 6 months after dosing.
Exclusion criteria
Exclusion criteria: 1.Participants who have received any gene therapy during the screening or baseline period; 2.Refusal or inability to undergo mobility testing; 3.Presence of narrow angles in both eyes during screening, with contraindication to dilation; 4.Presence of underlying diseases in both eyes during screening; 5.Presence of refractive errors in both eyes during screening, as judged by the investigator to potentially interfere with visual acuity and other ocular assessments; 6.Presence of significant vitreoretinal pathologies such as vitreomacular adhesion or traction, macular folds and macular holes, retinal detachment, as determined by the investigator to significantly impact central visual acuity, observed through slit-lamp examination, color fundus photography, and/or OCT imaging during screening; 7.Underwent procedures within the three months prior to screening and during the screening period, as judged by the investigator to potentially cause interference; 8.Currently using hydroxychloroquine, chloroquine, or any related retinal toxic drugs during the screening period; 9.Presence of active ocular inflammation, or a history of idiopathic or autoimmune uveitis recurrence during the screening period; 10.History of retinal repair surgery or any stem cell therapy prior to screening; 11.Presence of any complex systemic diseases during the screening period; 12.Positive results for any of the four infection tests during the screening or baseline period; 13.Poor compliance during the screening period, with an anticipated difficulty in completing the study; 14.Any other conditions as judged by the investigator to potentially interfere with safety and efficacy assessments.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The safety and tolerance of GA001; | — |
Secondary
| Measure | Time frame |
|---|---|
| The eficacy of GA001;Immunogenicity of GA001;Pharmacokinetic characteristics of GA001; | — |
Countries
China
Contacts
Public ContactXuxiang Zhang
Beijing Tiantan Hospital, Capital Medical University
Outcome results
None listed