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

Early Phase 1

Recruiting

• Conditions: PDR - Proliferative Diabetic Retinopathy
• Interventions: Drug: RRG001

• Registration Number: NCT06412224
• Lead Sponsor: Li Xiaorong

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2023-12-20
• Study First Submitted: 2024-04-24
• Status Verified: 2024-05
• Study First Submitted QC: 2024-05-09
• Last Update Submitted: 2024-05-09
• Study First Posted: 2024-05-14
• Last Update Posted: 2024-05-14
• Primary Completion: 2024-12-31
• Study Completion: 2028-12-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 6

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
RRG001	RRG001	Frequency of administration: one time injection.

Primary Outcome Measures

Name	Time	Method
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 DLTs (dose limiting toxicity analysis) at most 6 subjcts
To evaluate the incidence of AEs(Adverse Event) as assessed CTCAE 5.0 at most 6 subjcts	Within 24 weeks after RRG001 administration	To evaluate the incidence of AEs as assessed CTCAE 5.0 at most 6 subjcts

Secondary Outcome Measures

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

Trial Locations

Locations (1)

Tianjin medical university eye hospital; 🇨🇳 Tianjin, China