Safety and Efficacy Study of NGGT001 in Bietti Crystalline Corneoretinal Dystrophy Subjects

NCT ID: NCT06706427

Last Updated: 2025-05-25

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 12 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-03-21
• Study Completion Date: 2029-09-26

Brief Summary

The objective of this study is to evaluate the safety, tolerability, and efficacy of subretinal injection of NGGT001 in patients with Bietti Crystalline Corneoretinal Dystrophy (BCD) and to recommend the optimal dosage for future clinical administration.

Conditions

Bietti Crystalline Corneoretinal Dystrophy

Study Design

• Allocation Method: NA
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

NGGT001

Single Arm: This study is a single-arm design in which all participants receive the NGGT001 gene therapy administered via subretinal injection. Participants are divided into three dose-escalation groups to evaluate safety and efficacy.

Group Type: EXPERIMENTAL

NGGT001

Intervention Type: BIOLOGICAL

Using a recombinant adeno-associated virus (AAV) vector to deliver the gene CYP4V2 via subretinal injection for the treatment of crystalline retinal degeneration.

Interventions

NGGT001

Using a recombinant adeno-associated virus (AAV) vector to deliver the gene CYP4V2 via subretinal injection for the treatment of crystalline retinal degeneration.

Intervention Type: BIOLOGICAL

Eligibility Criteria

Exclusion Criteria

1. There are choroidal neovascularization or other ocular diseases caused by BCD, which are considered to affect the operation or interfere with the interpretation of clinical endpoint.

2. Patients with evidence of neovascularization or suspected neovascularization, and the presence of tubular reflectivity in the neuroepithelial layer as shown by OCT.

3. Those who had used any of the treatment drugs within 6 months before enrollment, such as Lucentis, Avastin, Conbercept, Triamcinolone acetonide, etc. These may affect the experimental observation.

4. The treated eyes have undergone intraocular surgery, such as photodynamic therapy (PDT), vitrectomy, periocular vascular bypass surgery, etc., or need intraocular surgery in the process of clinical research, such as cataract surgery, retinal laser therapy, etc.

5. Have used or may use systemic drugs that may cause eye damage, such as psoralen, tamoxifen, etc.

6. Highly sensitive or allergic to ingredients in the test drug (with allergic history of two or more drugs or food).

7. Physical examination, vital signs, and laboratory examination (such as blood routine, urine routine, blood biochemistry, coagulation function, immunology examination, etc.) are abnormal and clinically significant, or the investigators believe that the abnormal indicators have clinical significance.

8. There are diseases or medical histories that may affect drug safety or in vivo processes, especially cardiovascular, liver, kidney, endocrine, digestive tract, lung, nerve, blood, tumor, immune or metabolic disorders considered by investigators to be of clinical significance.

9. Participated in clinical trials of other drugs or medical devices within three months before enrollment.

10. Female patients who are pregnant or lactating.

11. Any other conditions which lead the investigator to determine the participant is unsuitable for this study.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

NGGT (Suzhou) Biotechnology Co., Ltd.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Southwest Hospital/Southwest Eye Hospital, Third Military Medical University (Army Medical University)

Chongqing, Chongqing Municipality, China

Xiamen Eye Center of Xiamen University

Xiamen, Fujian, China

Countries

China

Other Identifiers

NGGT001-P-2301

Identifier Type: -

Identifier Source: org_study_id