Clinical trial NCT06706427: Safety and Efficacy Study of NGGT001 in Bietti Crystalline Corneoretinal Dystrophy Subjects
Official title: A Phase I/II Study for Subretinal Injection of NGGT001 in Patients With Bietti Crystalline Corneoretinal Dystrophy
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE1
PHASE2
Conditions: Bietti Crystalline Corneoretinal Dystrophy
Keywords: BCD
Bietti Crystalline Corneoretinal Dystrophy
Lead sponsor: NGGT (Suzhou) Biotechnology Co., Ltd.
Sponsor class: INDUSTRY
First posted: 2024-11-26
Enrollment: 12
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

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.


Eligibility criteria: 1. Age ≥ 18 years old.
2. Male or female.
3. Confirmed diagnosis of BCD.
4. Molecular diagnosis confirmed cytochrome P450 family 4 subfamily v member 2 (CYP4V2) mutation.
5. AAV2 neutralizing antibody titer ≤1:5120.
6. 0.05 ≤ Best Corrected Visual Acuity (BCVA) ≤ 0.3.
7. -6.00D ≤ Refractive error ≤ +3.00D.
8. Agree to take contraceptive measures from the start of the study until one year after medication administration.
9. Volunteer to participate in the study and sign informed consent.

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.

Primary outcomes:
measure: Incidence of adverse events (AEs) from baseline to 52 weeks.
timeFrame: 52 weeks
measure: Evaluate the improvement in BCVA compared to baseline at Week 12, 26 and 52.
timeFrame: Week 12, Week 26 and Week 52
Secondary outcomes:
measure: Assessment of microperimetry changes in dB compared to baseline at Week 12, 26 and 52.
timeFrame: Week 12, Week 26 and Week 52
measure: Assessment of contrast sensitivity (CS) changes in dB compared to baseline at Week 12, 26 and 52.
timeFrame: Week 12, Week 26 and Week 52
measure: Assessment of Optical Coherence Tomography (OCT) retinal thickness changes compared to baseline at Week 12, 26 and 52.
timeFrame: Week 12, Week 26 and Week 52
measure: Assessment of Multi-Luminance Mobility Test (MLMT) score changes compared to baseline at Week 12, 26 and 52.
timeFrame: Week 12, Week 26 and Week 52
Other outcomes:
measure: Assessment of Static Visual Fields (SVF) changes compared to baseline at Week 12, 26 and 52.
timeFrame: Week 12, Week 26 and Week 52
measure: Assessment of Fundus Autofluorescence (FAF) retinal structural changes compared to baseline at Week 12, 26 and 52.
timeFrame: Week 12, Week 26 and Week 52
measure: Assessment of Quality of Life Questionnaire score changes compared to baseline at Week 12, 26 and 52.
timeFrame: Week 12, Week 26 and Week 52
measure: Assessment of MNRead Test changes compared to baseline at Week 12, 26 and 52.
timeFrame: Week 12, Week 26 and Week 52
Locations:
city: Chongqing
country: China
facility: Southwest Hospital/Southwest Eye Hospital, Third Military Medical University (Army Medical University)
state: Chongqing
city: Xiamen
country: China
facility: Xiamen Eye Center of Xiamen University
state: Fujian
Study officials:
References: