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

NCT06706427 | Active Not Recruiting Phase 1

• 1 other identifier: NGGT001-P-2301
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 2

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

Keywords

BCD; Bietti Crystalline Corneoretinal Dystrophy

Outcome Measures

Primary Outcomes (2)

• Incidence of adverse events (AEs) from baseline to 52 weeks.

  To evaluate the incidence and severity of AEs, including serious AEs (SAEs) of subretinal injection of NGGT001 in patients with BCD.

  52 weeks

• Evaluate the improvement in BCVA compared to baseline at Week 12, 26 and 52.

  To evaluate the BCVA in ETDRS test of subretinal injection of NGGT001 from baseline to W12, 26 and 52.

  Week 12, Week 26 and Week 52

Secondary Outcomes (4)

• Assessment of microperimetry changes in dB compared to baseline at Week 12, 26 and 52.

  Week 12, Week 26 and Week 52

• Assessment of contrast sensitivity (CS) changes in dB compared to baseline at Week 12, 26 and 52.

  Week 12, Week 26 and Week 52

• Assessment of Optical Coherence Tomography (OCT) retinal thickness changes compared to baseline at Week 12, 26 and 52.

  Week 12, Week 26 and Week 52

• Assessment of Multi-Luminance Mobility Test (MLMT) score changes compared to baseline at Week 12, 26 and 52.

  Week 12, Week 26 and Week 52

Other Outcomes (4)

• Assessment of Static Visual Fields (SVF) changes compared to baseline at Week 12, 26 and 52.

  Week 12, Week 26 and Week 52

• Assessment of Fundus Autofluorescence (FAF) retinal structural changes compared to baseline at Week 12, 26 and 52.

  Week 12, Week 26 and Week 52

• Assessment of Quality of Life Questionnaire score changes compared to baseline at Week 12, 26 and 52.

  Week 12, Week 26 and Week 52

Study Arms (1)

NGGT001

EXPERIMENTAL

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.

Biological: NGGT001

Interventions

NGGT001 BIOLOGICAL

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

NGGT001

Eligibility Criteria

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

You may not qualify if:

• 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.
• Patients with evidence of neovascularization or suspected neovascularization, and the presence of tubular reflectivity in the neuroepithelial layer as shown by OCT.
• 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.
• 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.
• Have used or may use systemic drugs that may cause eye damage, such as psoralen, tamoxifen, etc.
• Highly sensitive or allergic to ingredients in the test drug (with allergic history of two or more drugs or food).
• 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.
• 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.
• Participated in clinical trials of other drugs or medical devices within three months before enrollment.
• Female patients who are pregnant or lactating.
• Any other conditions which lead the investigator to determine the participant is unsuitable for this study.

Sponsors & Collaborators

• NGGT (Suzhou) Biotechnology Co., Ltd.: lead

Study Sites (2)

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

Chongqing, Chongqing Municipality, 400000, China

Location

Xiamen Eye Center of Xiamen University

Xiamen, Fujian, 361000, China

Location

MeSH Terms

Conditions

Bietti Crystalline Dystrophy

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 14, 2024
• First Posted: November 26, 2024
• Study Start: March 21, 2024
• Primary Completion: November 30, 2025
• Study Completion (Estimated): September 26, 2029
• Last Updated: May 25, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

CN (2)