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

Phase 1

Active, not recruiting

• Conditions: Bietti Crystalline Corneoretinal Dystrophy
• Interventions: Biological: NGGT001

• Registration Number: NCT06706427
• Lead Sponsor: NGGT (Suzhou) Biotechnology Co., Ltd.

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2024-03-21
• Status Verified: 2024-11
• Study First Submitted: 2024-11-14
• Study First Submitted QC: 2024-11-22
• Last Update Submitted: 2024-11-22
• Study First Posted: 2024-11-26
• Last Update Posted: 2024-11-26
• Primary Completion: 2025-03-26
• Study Completion: 2029-09-26

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 12

Inclusion Criteria

Not provided

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
NGGT001	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.

Primary Outcome Measures

Name	Time	Method
Incidence of adverse events (AEs) from baseline to 26 weeks.	26 weeks	To evaluate the incidence and severity of AEs, including serious AEs (SAEs) of subretinal injection of NGGT001 in patients with BCD.
Evaluate the improvement in BCVA compared to baseline at Week 12 and 26.	Week 12, Week 26	To evaluate the BCVA in ETDRS test of subretinal injection of NGGT001 from baseline to W12 and W26

Secondary Outcome Measures

Name	Time	Method
Assessment of microperimetry changes in dB compared to baseline at Week 12 and 26.	Week 12, Week 26	Retinal sensitivity will be assessed using the MP-3 Type Microperimeter. This will be measured in dB (decibels), and the retinal sensitivity analysis will be conducted based on these readings.
Assessment of contrast sensitivity (CS) changes in dB compared to baseline at Week 12 and 26.	Week 12, Week 26	Changes in contrast sensitivity (CS) will be evaluated, and the measurements will be analyzed in decibels (dB).
Assessment of Optical Coherence Tomography (OCT) retinal thickness changes compared to baseline at Week 12 and 26.	Week 12, Week 26	Retinal thickness will be measured using Optical Coherence Tomography (OCT), and the outcome will be expressed in micrometers (µm).
Assessment of Multi-Luminance Mobility Test (MLMT) score changes compared to baseline at Week 12 and 26.	Week 12, Week 26	Multi-Luminance Mobility Test (MLMT) will assess subjects' daily life quality across different lighting conditions. The results will be categorized by the different light intensity levels, and the aggregate scores will be used to assess mobility performance

Trial Locations

Locations (2)

Southwest Hospital/Southwest Eye Hospital, Third Military Medical University (Army Medical University); 🇨🇳 Chongqing, Chongqing, China

Xiamen Eye Center of Xiamen University; 🇨🇳 Xiamen, Fujian, China