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

Sponsor
Shanghai Refreshgene Technology Co., Ltd. (Industry)
Overall Status
Not yet recruiting
CT.gov ID
NCT06141460
Collaborator
(none)
48
Enrollment
4
Arms
85.2
Anticipated Duration (Months)

Study Details

Study Description

Brief Summary

RRG001 is a adeno-associated virus (AAV) vector-based gene therapy for subretinal injection. The long-term, stable therapeutic protein after one time injection for nAMD could potentially reduce the treatment burden and maintain vision.RRG001 is designed to reduce the current treatment burden which often results in undertreatment and vision loss in patients with nAMD receiving anti-VEGF therapy in clinical practice.

Condition or Disease Intervention/Treatment Phase
Phase 1/Phase 2

Detailed Description

This clinical trial is designed to evaluate the safety and preliminary efficacy of single subretinal injection of RRG001 gene therapy in nAMD.

Study Design

Study Type:
Interventional
Anticipated Enrollment :
48 participants
Allocation:
Non-Randomized
Intervention Model:
Sequential Assignment
Intervention Model Description:
dose escalationdose escalation
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
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
Anticipated Study Start Date :
Nov 24, 2023
Anticipated Primary Completion Date :
Dec 31, 2027
Anticipated Study Completion Date :
Dec 31, 2030

Arms and Interventions

Arm Intervention/Treatment
Experimental: RRG001 Dose1

Frequency of administration: one time injection.

Drug: RRG001
Administered via Subretinal injection. Dosage form: injection solution.
Experimental: RRG001 Dose2

Frequency of administration: one time injection.

Drug: RRG001
Administered via Subretinal injection. Dosage form: injection solution.
Experimental: RRG001 Dose3

Frequency of administration: one time injection.

Drug: RRG001
Administered via Subretinal injection. Dosage form: injection solution.
Experimental: RRG001 Dose4

Frequency of administration: one time injection.

Drug: RRG001
Administered via Subretinal injection. Dosage form: injection solution.

Outcome Measures

Primary Outcome Measures

  1. Safety and tolerability [Week 52]

    incidence of AEs and SAEs

Secondary Outcome Measures

  1. Efficacy after RRG001 injection [Week 52]

    change in best-corrected visual acuity (BCVA)；

  2. Efficacy after RRG001 injection [Week 52]

    change in central retinal thickness (CRT)

Eligibility Criteria

Criteria

Ages Eligible for Study:
50 Years and Older
Sexes Eligible for Study:
All
Accepts 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.

Contacts and Locations

Locations

No locations specified.

Sponsors and Collaborators

  • Shanghai Refreshgene Technology Co., Ltd.

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Shanghai Refreshgene Technology Co., Ltd.
ClinicalTrials.gov Identifier:
NCT06141460
Other Study ID Numbers:
  • RRG001-101
First Posted:
Nov 21, 2023
Last Update Posted:
Nov 21, 2023
Last Verified:
Nov 1, 2023
Studies a U.S. FDA-regulated Drug Product:
No
Studies a U.S. FDA-regulated Device Product:
No
Additional relevant MeSH terms:
Macular Degeneration
Wet Macular Degeneration

Study Results

No Results Posted as of Nov 21, 2023