A Phase I/II Study for Subretinal Injection of NGGT001 in Patients With Bietti Crystalline Corneoretinal Dystrophy

NGGT (Suzhou) Biotechnology Co., Ltd.2 sites in 1 country12 target enrollmentMarch 21, 2024

ConditionsBietti Crystalline Corneoretinal Dystrophy

InterventionsNGGT001

DrugsNGGT001

Overview

Phase: Phase 1
Intervention: NGGT001
Conditions: Bietti Crystalline Corneoretinal Dystrophy
Sponsor: NGGT (Suzhou) Biotechnology Co., Ltd.
Enrollment: 12
Locations: 2
Primary Endpoint: Incidence of adverse events (AEs) from baseline to 52 weeks.
Status: Active, not recruiting
Last Updated: 11 months ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

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.

Registry

clinicaltrials.gov

Start Date

March 21, 2024

End Date

September 26, 2029

Last Updated

11 months ago

Study Type

Interventional

Study Design

Sequential

Sex

All

Investigators

Sponsor

NGGT (Suzhou) Biotechnology Co., Ltd.

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

Not provided

Exclusion Criteria

•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.

Arms & Interventions

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.

Intervention: NGGT001

Outcomes

Primary Outcomes

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

Time Frame: 52 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, 26 and 52.

Time Frame: Week 12, Week 26 and Week 52

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

Secondary Outcomes

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 microperimetry 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)