Safety and Efficacy Study of NGGT001 in Bietti Crystalline Corneoretinal Dystrophy

NCT06302608 | Active Not Recruiting Early Phase 1

• 1 other identifier: NGGT-BCD-P-2203
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Bietti's Crystalline Dystrophy

Outcome Measures

Primary Outcomes (2)

• BCVA

  ETDRS visual acuity

  1 year

• 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

Study Arms (2)

Dose level 1

EXPERIMENTAL

Dose level 1 will be administered

Genetic: NGGT001

Dose level 2

EXPERIMENTAL

Dose level 2 will be administered

Genetic: NGGT001

Interventions

NGGT001 GENETIC

Safety and Effectiveness of subretinal injection of NGGT001 for treating crystalline retinal degeneration

Dose level 1; Dose level 2

Eligibility Criteria

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

You may qualify if:

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

You may not qualify if:

• 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.
• 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.
• 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;
• 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;
• Used or may use systemic medications that may cause eye damage, such as psoralen, tamoxifen, etc;
• Highly sensitive or allergic to the ingredients in the experimental drug (with a history of allergies to two or more drugs or foods);
• 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;
• 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.
• Participated in clinical trials of other drugs or medical devices within 3 months prior to enrollment;
• Female patients during pregnancy or lactation;
• Other researchers believe that it is not suitable to participate in the study.

Sponsors & Collaborators

• Xiamen Ophthalmology Center Affiliated to Xiamen University: lead

Study Sites (1)

Xiuju Chen

Xiamen, Fujian, 361000, China

Location

Related Publications (1)

• Chen X, Liu X, Cui S, Wang G, Liu Y, Qu G, Jiang L, Liu Y, Li X. Safety and Vision Outcomes Following Gene Therapy for Bietti Crystalline Dystrophy: A Nonrandomized Clinical Trial. JAMA Ophthalmol. 2025 Feb 1;143(2):126-133. doi: 10.1001/jamaophthalmol.2024.5619.

  PMID: 39786763 DERIVED

MeSH Terms

Conditions

Bietti Crystalline Dystrophy

Study Officials

• Li

  Xiamen Ophthalmology Center Affiliated to Xiamen University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 18, 2024
• First Posted: March 12, 2024
• Study Start: February 8, 2023
• Primary Completion: May 29, 2024
• Study Completion (Estimated): May 29, 2028
• Last Updated: March 12, 2024

Record last verified: 2024-02

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)