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

NCT ID: NCT06141460

Last Updated: 2024-11-14

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 48 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-12-12
• Study Completion Date: 2030-12-31

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.

Conditions

Neovascular Age-related Macular Degeneration

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

RRG001 Dose1

Frequency of administration: one time injection.

Group Type: EXPERIMENTAL

RRG001

Intervention Type: DRUG

Administered via Subretinal injection. Dosage form: injection solution.

RRG001 Dose2

Frequency of administration: one time injection.

Group Type: EXPERIMENTAL

RRG001

Intervention Type: DRUG

Administered via Subretinal injection. Dosage form: injection solution.

RRG001 Dose3

Frequency of administration: one time injection.

Group Type: EXPERIMENTAL

RRG001

Intervention Type: DRUG

Administered via Subretinal injection. Dosage form: injection solution.

RRG001 Dose4

Frequency of administration: one time injection.

Group Type: EXPERIMENTAL

RRG001

Intervention Type: DRUG

Administered via Subretinal injection. Dosage form: injection solution.

Interventions

RRG001

Administered via Subretinal injection. Dosage form: injection solution.

Intervention Type: DRUG

Eligibility 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.

• Minimum Eligible Age: 50 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Shanghai Refreshgene Technology Co., Ltd.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Tianjin Medical University Eye Hospital

Tianjin, Tianjin Municipality, China

Site Status: RECRUITING

Countries

China

Central Contacts

Xiaorong Li, Master

Role: CONTACT

xiaorong.li@163.com

8602223346434

Facility Contacts

Li Xiaorong, Professor

Role: primary

xiaorli@163.com

8602223346434

Other Identifiers

RRG001-101

Identifier Type: -

Identifier Source: org_study_id