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

NCT06412224 | Recruiting Early Phase 1

• 1 other identifier: RRG001-P01
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

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.

Conditions

PDR - Proliferative Diabetic Retinopathy

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (7)

• 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

  Within 24 weeks after RRG001 administration

• 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

  Within 24 weeks after RRG001 administration

• To evaluate the anatomical changes of the study eye by OCT(optical coherence tomography) at most 6 subjects

  Within 24 weeks after RRG001 administration

Study Arms (1)

RRG001

EXPERIMENTAL

Frequency of administration: one time injection.

Drug: RRG001

Interventions

RRG001 DRUG

Administered by subretinal injection. Dosage form: injection.

RRG001

Eligibility Criteria

• Age: 20 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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
• 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

Sponsors & Collaborators

• Li Xiaorong: lead
• Shanghai Refreshgene Technology Co., Ltd.: collaborator

Study Sites (1)

Tianjin medical university eye hospital

Tianjin, China

RECRUITING

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Assistant Investigator

Study Record Dates

• First Submitted: April 24, 2024
• First Posted: May 14, 2024
• Study Start: December 20, 2023
• Primary Completion: December 31, 2024
• Study Completion (Estimated): December 31, 2028
• Last Updated: May 14, 2024

Record last verified: 2024-05

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)