Bietti Crystalline Corneoretinal Dystrophy, Bietti's Crystalline Dystrophy
Conditions
Keywords
Bietti's Crystalline Dystrophy, ZVS101e, AAV8-hCYP4V2, gene replacement treatment, Bietti Crystalline Corneoretinal Dystrophy
Brief summary
The goal of this clinical trial is to evaluate the safety and early effectiveness of ZVS101e in patients with Bietti crystalline dystrophy who meet the eligibility criteria for treatment under the translational application program in the Hainan Boao Lecheng International Medical Tourism Pilot Zone. Participants will: undergo screening and baseline assessments to confirm eligibility; receive a single subretinal injection of ZVS101e in the study eye; complete follow-up visits over 4 weeks after treatment for safety monitoring and assessment of early effectiveness.
Interventions
DRUGZVS101e
ZVS101e injection contains a recombinant adeno-associated virus serotype 8 (rAAV8) vector that expresses human CYP4V2 protein.
Sponsors
Chigenovo Co., Ltd
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Voluntarily participate in the research and sign the informed consent form, and be willing to complete the entire process according to the protocol requirements; 2. Clinically diagnosed with Bietti's Crystalline Dystrophy (BCD), age ≥ 18 years; 3. Confirmed by genetic testing to carry homozygous or compound heterozygous mutations in CYP4V2, and not complicated with other ophthalmic genetic diseases; 4. Target eye BCVA ≤ 60 ETDRS letters.
Exclusion criteria
Subjects will be excluded if they meet any 1 of the following
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The incidence and severity of adverse events and serious adverse events | From enrollment to the end of treatment at 52 weeks |
| Change from baseline in Best corrected visual acuity (BCVA) | From enrollment to the end of treatment at 52 weeks |
Countries
China
Contacts
CONTACTJinlu Zhang, MD
CONTACTTingting Wu, BS
Outcome results
None listed