Clinical trial NCT06412224: The Preliminary Safety and Efficacy of RRG001 After Vitrectomy in Subjects With Proliferative Diabetic Retinopathy (PDR)
Official title: 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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: EARLY_PHASE1
Conditions: PDR - Proliferative Diabetic Retinopathy
Lead sponsor: Tianjin Medical University Eye Hospital
Sponsor class: OTHER
Collaborators: name: Shanghai Refreshgene Technology Co., Ltd.
First posted: 2024-05-14
Enrollment: 6
Sex: ALL
Minimum age (years): 20
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: To evaluate the incidence of DLTs (dose limiting toxicity analysis) at most 6 subjcts
timeFrame: Within 28 days after RRG001 administration
measure: To evaluate the incidence of AEs(Adverse Event) as assessed CTCAE 5.0 at most 6 subjcts
timeFrame: Within 24 weeks after RRG001 administration
Secondary outcomes:
measure: To evaluate the disease progression as assessed the vitreous hemorrhage of the study eye at most 6 subjects
timeFrame: Within 24 weeks after RRG001 administration
measure: To evaluate the disease progression as assessed the retinal detachment of the study eye at most 6 subjects
timeFrame: Within 24 weeks after RRG001 administration
measure: To evaluate the frequency for rescue therapy of the study eye at most 6 subjects
timeFrame: Within 24 weeks after RRG001 administration
measure: 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
timeFrame: Within 24 weeks after RRG001 administration
measure: To evaluate the anatomical changes of the study eye by OCT(optical coherence tomography) at most 6 subjects
timeFrame: Within 24 weeks after RRG001 administration
measure: To evaluate the anatomical changes of the study eye by CFP(colour fundus photography）at most 6 subjects
timeFrame: Within 24 weeks after RRG001 administration
measure: To evaluate the anatomical changes of the study eye by OCTA（optical coherence tomography angiography) at most 6 subjects
timeFrame: Within 24 weeks after RRG001 administration
Other outcomes:
Locations:
city: Tianjin
country: China
facility: Tianjin medical university eye hospital
Study officials:
References: