Neovascular Age-related Macular Degeneration
Conditions
Brief summary
A Phase I/IIa, Dose-escalation and Dose-expansion Study to Evaluate the Safety and Efficacy of Single Subretinal Injection of RRG001 in Subjects With Neovascular Age-related Macular Degeneration
Detailed description
This study adopts a multicenter, single-arm, open-label phase I/IIa seamless study design and comprises two stages, i.e., phase I 3+3 dose escalation study and a phase IIa dose expansion study. Four dose groups are preset for the phase I dose escalation study; 3-6 subjects will be enrolled for each dose group, and it is planned to enroll 12-24 subjects. The phase IIa dose expansion study will be conducted successively at the doses after safety assessments of dose 2 and/or dose 3 in phase I are completed. Twelve subjects are expected to be enrolled for each dose group, with a total of 24 subjects will be enrolled overall. The sponsor will assess whether or not to adjust the dose(s) and sample size for the exploration study based on the results in the phase I exploration study.
Interventions
DRUGRRG001
Administered via Subretinal injection. Dosage form: injection solution.
Sponsors
Shanghai Refreshgene Technology Co., Ltd.
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
dose escalation
Eligibility
Sex/Gender
ALL
Age
50 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Subjects that are willing and able to follow study procedures. * Patinets ≥50 years old. * Clinically diagnosed with CNV secondary to nAMD. * BCVA of the study eye is ≥5 letters and ≤73 letters(Snellen 20/800\ 20/40), as well as that of the other eye ≥19 letters(Snellen 20/400). * Responding to anti-VEGF
Exclusion criteria
* Presence of any other eye diseases other than nAMD in study eye that would affect the treatment or confusing the interpretation of the study results , as judged by the investigator. * CNV or macular edema in the study eye secondary to any causes other than AMD. * Uncontrolled glaucoma. * Uncontrolled hypertension despite medication. * Pregnant or lactating women or individuals with reproductive potential who are unwilling to take effective contraception during the study period.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety and tolerability | Week 52 | incidence of AEs and SAEs |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Efficacy after RRG001 injection | Week 52 | change in best-corrected visual acuity (BCVA)； |
Countries
China
Contacts
Primary ContactXiaorong Li, Master
Outcome results
None listed