The Preliminary Safety and Efficacy of RRG001 After Vitrectomy in Subjects With Proliferative Diabetic Retinopathy (PDR)

An Exploratory ITT（Investigator Initiated Study) Study to Evaluate the Preliminary Safety and Efficacy of Single Subretinal Injection of RRG001 at the End of or After Vitrectomy in Subjects With PDR

This study aims to slow down disease progression, reduce postoperative complications and decrease retreatment frequency in subjects with proliferative diabetic retinopathy (PDR) by administering a single subretinal injection of RRG001 gene therapy after vitrectomy.

Study Overview

• Status: Recruiting
• Conditions: PDR - Proliferative Diabetic Retinopathy
• Intervention / Treatment: Drug: RRG001

• Study Type: Interventional
• Enrollment (Estimated): 6
• Phase: Early Phase 1

Contacts and Locations

Study Locations

• China
  • Tianjin, China
    • Recruiting
    • Tianjin Medical University Eye Hospital
    • Contact: Xiaorong Li, Pro.; Phone Number: 18622818042; Email: xiaorli@163.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Able and willing to provide informed consent
• Age >= 20 years
• Diagnosis of diabetes mellitus (type 1 or type 2)
• The study eye diagnosed with PDR, in the opinion of the investigator, requires vitrectomy and postoperative anti-VEGF therapy, or that has undergone vitrectomy and still requires postoperative anti-VEGF therapy
• Based on the ETDRS chart, the best-corrected visual acuity (BCVA) of the study eye is ≥ hand motion and ≤ 63 letters

Exclusion Criteria:

• Active infection or inflammation in either eye
• Previous gene therapy in either eye
• Uncontrolled blood pressure（defined as systolic ≥160mmHg or diastolic ≥ 110mmHg by anti-hypertensive treatment)
• HbA1c >12% for diabetes patients at screening
• Previous condition not eligible for study drug
• History of major ocular surgery (except for PDR) or severe trauma
• Currently enrolled in another clinical trial or planning to enroll during the study
• Pregnant or lactating women
• Other conditions that, in the opinion of the investigator, would preclude participation in the study

Study Eye-Exclusion Criteria：

• Ocular disorders that, in the opinion of the investigator, would confound the interpretation of study results or affect the administration
• CNV or macular edema secondary to any causes other than diabetic retinopathy
• Ocular condition considered by the investigator to contraindicate subretinal injection
• Diagnosed primary or secondary glaucoma
• History of intraocular corticosteroid treatment

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: RRG001; Frequency of administration: one time injection.	Drug: RRG001; Administered by subretinal injection. Dosage form: injection.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To evaluate the incidence of DLTs (dose limiting toxicity analysis) at most 6 subjcts	To evaluate the incidence of DLTs (dose limiting toxicity analysis) at most 6 subjcts	Within 28 days after RRG001 administration
To evaluate the incidence of AEs(Adverse Event) as assessed CTCAE 5.0 at most 6 subjcts	To evaluate the incidence of AEs as assessed CTCAE 5.0 at most 6 subjcts	Within 24 weeks after RRG001 administration

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To evaluate the disease progression as assessed the vitreous hemorrhage of the study eye at most 6 subjects	To evaluate the disease progression as assessed the vitreous hemorrhage of the study eye at most 6 subjects	Within 24 weeks after RRG001 administration
To evaluate the disease progression as assessed the retinal detachment of the study eye at most 6 subjects	To evaluate the disease progression as assessed the retinal detachment of the study eye at most 6 subjects	Within 24 weeks after RRG001 administration
To evaluate the frequency for rescue therapy of the study eye at most 6 subjects	To evaluate the frequency for rescue therapy of the study eye at most 6 subjects	Within 24 weeks after RRG001 administration
To evaluate the change from baseline in BCVA(Best Corrected Visual Acuity) of the study eye as assessed by ETDRS chart at most 6 subjects	To evaluate the change from baseline in BCVA of the study eye as assessed by ETDRS chart at most 6 subjects	Within 24 weeks after RRG001 administration
To evaluate the anatomical changes of the study eye by OCT(optical coherence tomography) at most 6 subjects	To evaluate the anatomical changes of the study eye by OCT(optical coherence tomography) at most 6 subjects	Within 24 weeks after RRG001 administration
To evaluate the anatomical changes of the study eye by CFP(colour fundus photography）at most 6 subjects	To evaluate the anatomical changes of the study eye by CFP(colour fundus photography）at most 6 subjects	Within 24 weeks after RRG001 administration
To evaluate the anatomical changes of the study eye by OCTA（optical coherence tomography angiography) at most 6 subjects	To evaluate the anatomical changes of the study eye by OCTA（optical coherence tomography angiography) at most 6 subjects	Within 24 weeks after RRG001 administration

Collaborators and Investigators

• Sponsor: Li Xiaorong
• Collaborators: Shanghai Refreshgene Technology Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): December 20, 2023
• Primary Completion (Estimated): December 31, 2024
• Study Completion (Estimated): December 31, 2028

Study Registration Dates

• First Submitted: April 24, 2024
• First Submitted That Met QC Criteria: May 9, 2024
• First Posted (Actual): May 14, 2024

Study Record Updates

• Last Update Posted (Actual): May 14, 2024
• Last Update Submitted That Met QC Criteria: May 9, 2024
• Last Verified: May 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Eye Diseases; Endocrine System Diseases; Diabetic Angiopathies; Diabetes Complications; Diabetes Mellitus; Retinal Diseases; Diabetic Retinopathy
• Other Study ID Numbers: RRG001-P01

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No