Gene therapy for neovascular age-related macular degeneration
A Phase I/IIa, Dose-escalation and Dose-extension Study to Evaluate the Safety and Efficacy of Single Subretinal Injection of RRG001 in Subjects With Neovascular Age-related Macular Degeneration
PHASE1; PHASE2 · Shanghai Refreshgene Technology Co., Ltd. · NCT06141460
This study is testing a new gene therapy injection to see if it can safely improve vision in people with a specific type of age-related eye disease.
Quick facts
| Phase | PHASE1; PHASE2 |
|---|---|
| Study type | Interventional |
| Enrollment | 48 (estimated) |
| Ages | 50 Years and up |
| Sex | All |
| Sponsor | Shanghai Refreshgene Technology Co., Ltd. (industry) |
| Locations | 1 site (Tianjin, Tianjin Municipality) |
| Trial ID | NCT06141460 on ClinicalTrials.gov |
What this trial studies
This study evaluates the safety and efficacy of a single subretinal injection of RRG001 in patients with neovascular age-related macular degeneration (nAMD). It employs a multicenter, single-arm, open-label design, consisting of a Phase I dose-escalation study followed by a Phase IIa dose-expansion study. The Phase I component will enroll 3-6 subjects per dose group, while the Phase IIa will expand to 12 subjects per group based on safety assessments. The goal is to determine the optimal dosing and assess the treatment's effectiveness in improving vision.
Who should consider this trial
Good fit: Ideal candidates are individuals aged 50 and older with clinically diagnosed nAMD and specific visual acuity criteria.
Not a fit: Patients with other eye diseases or conditions that could interfere with the study results will not benefit from this treatment.
Why it matters
Potential benefit: If successful, this gene therapy could significantly improve vision and quality of life for patients with nAMD.
How similar studies have performed: While gene therapy for eye conditions is a novel approach, similar studies have shown promising results in treating other retinal diseases.
Eligibility criteria
Show full inclusion / exclusion criteria
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.
Where this trial is running
Tianjin, Tianjin Municipality
- Tianjin Medical University Eye Hospital — Tianjin, Tianjin Municipality, China (RECRUITING)
Study contacts
- Study coordinator: Xiaorong Li, Master
- Email: xiaorong.li@163.com
- Phone: 8602223346434
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Neovascular Age-related Macular Degeneration