Safety and Efficacy Study of NGGT001 in Bietti Crystalline Corneoretinal Dystrophy

Clinical Trial on the Safety and Effectiveness of Subretinal Injection of NGGT001 for Treating Crystalline Retinal Degeneration

Evaluate the safety and tolerability of NGGT001 subretinal injection for the treatment of crystalline retinal degeneration (BCD)

Study Overview

• Status: Active, not recruiting
• Conditions: Bietti's Crystalline Dystrophy
• Intervention / Treatment: Genetic: NGGT001

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

Contacts and Locations

Study Locations

• China
  • Fujian
    • Xiamen, Fujian, China, 361000
      • Xiuju Chen

Participation Criteria

Eligibility Criteria

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

Description

Inclusion Criteria:

1. Age ≥ 18 years old;
2. Gender unlimited.
3. Diagnosed as crystalline retinal degeneration (BCD).
4. Molecular diagnosis confirms CYP4V2 mutation.
5. The best corrected visual acuity is less than 20/60.
6. Agree to take contraceptive measures within 1 year from the start of the study until administration.
7. Voluntarily sign an informed consent form.

Exclusion Criteria:

1. Insufficient number of photoreceptor cells in the retina, such as retinal thickness less than 100 μ m. Or no atrophy or pigmentation in the posterior pole area<3- Retinal disc.
2. The presence of choroidal neovascularization or other eye lesions caused by BCD, which researchers believe may affect surgical procedures or interfere with the interpretation of clinical endpoints.
3. The use of therapeutic drugs within the first 6 months of enrollment may affect experimental observation, such as Lucentis, Avastin, Conbercept, Triamcinolone acetonide, steroids, etc;
4. The treatment eye has undergone intraocular surgery, such as PDT, vitrectomy, periocular vascular bypass surgery, etc., or requires intraocular surgery during clinical research, such as cataract surgery, retinal laser therapy, etc;
5. Used or may use systemic medications that may cause eye damage, such as psoralen, tamoxifen, etc;
6. Highly sensitive or allergic to the ingredients in the experimental drug (with a history of allergies to two or more drugs or foods);
7. Abnormal and clinically significant physical examination, vital signs, laboratory tests (such as blood routine, urine routine, blood biochemistry, coagulation function, immunological tests, etc.), or abnormal indicators deemed clinically significant by researchers;
8. There are diseases or medical histories that may affect drug safety or internal processes, especially cardiovascular, liver, kidney, endocrine, digestive, lung, neurological, hematological, tumor, immune or metabolic disorders that researchers consider clinically significant.
9. Participated in clinical trials of other drugs or medical devices within 3 months prior to enrollment;
10. Female patients during pregnancy or lactation;
11. Other researchers believe that it is not suitable to participate in the study.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Dose level 1; Dose level 1 will be administered	Genetic: NGGT001; Safety and Effectiveness of subretinal injection of NGGT001 for treating crystalline retinal degeneration
Experimental: Dose level 2; Dose level 2 will be administered	Genetic: NGGT001; Safety and Effectiveness of subretinal injection of NGGT001 for treating crystalline retinal degeneration

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence and severity of adverse events (AEs) and serious adverse events (SAEs)	Incidence and severity of adverse events (AEs) and serious adverse events (SAEs)	1 year
BCVA	ETDRS visual acuity	1 year

Collaborators and Investigators

• Sponsor: Xiamen Ophthalmology Center Affiliated to Xiamen University
• Investigators: Principal Investigator: Li, Xiamen Ophthalmology Center Affiliated to Xiamen University

Study record dates

Study Major Dates

• Study Start (Actual): February 8, 2023
• Primary Completion (Estimated): May 29, 2024
• Study Completion (Estimated): May 29, 2028

Study Registration Dates

• First Submitted: February 18, 2024
• First Submitted That Met QC Criteria: March 7, 2024
• First Posted (Actual): March 12, 2024

Study Record Updates

• Last Update Posted (Actual): March 12, 2024
• Last Update Submitted That Met QC Criteria: March 7, 2024
• Last Verified: February 1, 2024

More Information

Terms related to this study

• Other Study ID Numbers: NGGT-BCD-P-2203

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