The Gene Replacement Therapy by rAAV2/8-hCYP4V2

• Status: Recruiting
• Trial end date: 2023-12-30
• Target enrollment: 0
• Participant gender: All

Summary

Primary Objectives: To evaluate the safety of The Gene Replacement Therapy by rAAV2/8-hCYP4V2 Following a Single Subretinal Administration in The Patients of Bietti's Crystalline Dystrophy (BCD).

Secondary Objectives: To explore the efficacy of The Gene Replacement Therapy by rAAV2/8-hCYP4V2.

• Phase: Early Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Beijing Tongren Hospital

Criteria

Inclusion Criteria:

• 1) Are 18 years and older;

• 2) Are diagnosed of Bietti's crystalline dystrophy (BCD);

• 3) Molecular diagnosis confirmed due to CYP4V2 mutations (two pathogenic mutation);

• 4) Are met with the target eye inclusion criterion;

• 5) Agree to take effective contraceptive measures from the beginning of the study to 1 year after the administration;

• 6) Are voluntarily participated in the study and signed the informed consent form.

Exclusion Criteria:

• 1) Have insufficient viable retinal photoreceptor cells. Retinal photoreceptor cells are less than 1D , or areas of retina with thickness measurements less than 100 µm；

• 2) Existing or pre-existing of choroidal neovascular (CNV) lesions that were secondary to BCD, or other eye conditions interfering with the surgery or the interpretation of the clinical endpoint, in the investigators' opinion;

• 3) Prior use of medicines which may affect the experimental observation within the 6 months before screening (e.g. Lucentis, Avastin Eylea, Conbercept, Triamcinolone acetonide, Corticosteroids)；

• 4) Prior intraocular surgery in the target eye (e.g. PDT, pars plana vitrectomy, periocular vascular bypass surgery), or requirement of intraocular surgery during the clinical study (e.g. cataract surgery, laser therapy of retina);

• 5) Use of or potentially require of systemic medications that may cause eye damage (e.g. psoralen, risselinic acid, tamoxifen)；

• 6) Known hypersensitivity to any ingredient of clinical trial medicine, or allergies (with an allergy history to two or more kinds of medicines or foods);

• 7) Abnormal physical examination, vital signs, laboratory examination (e.g. blood routine, urine routine, blood biochemistry, coagulation function, immunologic test, pregnacy test), or other related indicators which have a clinical significance considered by the investigators；

• 8) Have any medical conditions or medical history which may have an effect on the safety or the intracorporal process of drugs, especially medical history of angiocarpy, liver, kidney, internal secretion, digestive tract, lung, nerve, blood, tumor, immune or metabolic disorders, which have a clinical significance considered by the investigators；

• 9) Participation in any medicine or medical device clinical trials within 3 months prior to enrollment;

• 10) Neutralizing antibodies to rAAV> 1:1000 by immunologic test;

• 11) For females in pregnancy or lactation period;

• 12) Any other conditions which leads the investigator to determine the participant is unsuitable for this study.