Safety and Efficacy of RRG001 Gene Therapy in Subjects With Neovascular Age-related Macular Degeneration (nAMD)

Shanghai Refreshgene Technology

Status and phase: Enrolling; Phase 2; Phase 1

Conditions: Neovascular Age-related Macular Degeneration

Treatments: Drug: RRG001

Study type: Interventional

Funder types: Industry

Identifiers

NCT06141460

RRG001-101

Details and patient eligibility

About

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

Full 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.

Enrollment: 48 estimated patients

Sex: All

Ages: 50+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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48 participants in 4 patient groups

RRG001 Dose1

Experimental group

Description:

Frequency of administration: one time injection.

Treatment:

Drug: RRG001

RRG001 Dose2

Experimental group

Description:

Frequency of administration: one time injection.

Treatment:

Drug: RRG001

RRG001 Dose3

Experimental group

Description:

Frequency of administration: one time injection.

Treatment:

Drug: RRG001

RRG001 Dose4

Experimental group

Description:

Frequency of administration: one time injection.

Treatment:

Drug: RRG001