- ICH GCP
- Amerikanska kliniska prövningsregistret
- Klinisk prövning NCT07653971
ZVS101e in Patients With Bietti's Crystalline Dystrophy (ZVS101e-BCD)
Clinical Translation and Application Protocol for ZVS101e in the Gene Therapy of Bietti Crystalline Corneoretinal Dystrophy
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.
Studieöversikt
Status
Intervention / Behandling
Studietyp
Inskrivning (Beräknad)
Fas
- Inte tillämpbar
Kontakter och platser
Studiekontakt
- Namn: Jinlu Zhang, MD
- Telefonnummer: +86-15810570898
- E-post: zhangjinlu@chinagene.cc
Studera Kontakt Backup
- Namn: Tingting Wu, BS
- Telefonnummer: +86-18500191916
- E-post: wutingting@chinagene.cc
Studieorter
-
-
Hainan
-
Qionghai, Hainan, Kina
- Boao Super Hospital
-
Qionghai, Hainan, Kina
- Hainan Boao Ophthalmology Center, Zhongshan Ophthalmic Center, Sun Yat-sen University
-
-
Deltagandekriterier
Urvalskriterier
Åldrar som är berättigade till studier
- Vuxen
- Äldre vuxen
Tar emot friska volontärer
Beskrivning
Inclusion Criteria:
- Voluntarily participate in the research and sign the informed consent form, and be willing to complete the entire process according to the protocol requirements;
- Clinically diagnosed with Bietti's Crystalline Dystrophy (BCD), age ≥ 18 years;
- Confirmed by genetic testing to carry homozygous or compound heterozygous mutations in CYP4V2, and not complicated with other ophthalmic genetic diseases;
- Target eye BCVA ≤ 60 ETDRS letters.
Exclusion Criteria:
Subjects will be excluded if they meet any 1 of the following exclusion criteria:
- The target eye currently has or has a history of macular lesions; suffers from ocular diseases that may hinder surgery or interfere with the interpretation of study endpoints;
- The target eye has previously undergone retinal reattachment surgery, vitrectomy, or any intraocular surgery within 3 months before enrollment;
- Within 1 month before enrollment, suffered from viral infectious diseases that may affect the evaluation of the efficacy and safety of the investigational drug, or received antiviral vaccines;
- The target eye has previously received gene therapy or stem cell therapy for BCD or other ocular diseases;
- Known allergy to the drugs planned for use in the study;
- The laboratory abnormalities are considered clinically significant;
- Pregnant or lactating females.
Studieplan
Hur är studien utformad?
Designdetaljer
- Primärt syfte: Behandling
- Tilldelning: N/A
- Interventionsmodell: Enskild gruppuppgift
- Maskning: Ingen (Open Label)
Vapen och interventioner
Deltagargrupp / Arm |
Intervention / Behandling |
|---|---|
|
Experimentell: ZVS101e Treatment Arm
|
ZVS101e injection contains a recombinant adeno-associated virus serotype 8 (rAAV8) vector that expresses human CYP4V2 protein.
|
Vad mäter studien?
Primära resultatmått
Resultatmått |
Tidsram |
|---|---|
|
Change from baseline in Best corrected visual acuity (BCVA)
Tidsram: From enrollment to the end of treatment at 52 weeks
|
From enrollment to the end of treatment at 52 weeks
|
|
The incidence and severity of adverse events and serious adverse events
Tidsram: From enrollment to the end of treatment at 52 weeks
|
From enrollment to the end of treatment at 52 weeks
|
Samarbetspartners och utredare
Sponsor
Publikationer och användbara länkar
Allmänna publikationer
- Jia R, Meng X, Chen S, Zhang F, Du J, Liu X, Yang L. AAV-mediated gene-replacement therapy restores viability of BCD patient iPSC derived RPE cells and vision of Cyp4v3 knockout mice. Hum Mol Genet. 2023 Jan 1;32(1):122-138. doi: 10.1093/hmg/ddac181.
- Wang J, Zhang J, Yu S, Li H, Chen S, Luo J, Wang H, Guan Y, Zhang H, Yin S, Wang H, Li H, Liu J, Zhu J, Yang Q, Sha Y, Zhang C, Yang Y, Yang X, Zhang X, Zhao X, Wang L, Yang L, Wei W. Gene replacement therapy in Bietti crystalline corneoretinal dystrophy: an open-label, single-arm, exploratory trial. Signal Transduct Target Ther. 2024 Apr 24;9(1):95. doi: 10.1038/s41392-024-01806-3.
- Jia R, Chen S, Li W, Zhang J, Qu B, Qiao J, Meng X, Yu S, Liu X, Xu B, Chen T, Shen X, Sun W, Dou H, Mahajan VB, Zhang Q, Yang L. Unravelling CYP4V2: Clinical features, genetic insights, pathogenic mechanisms and therapeutic strategies in Bietti crystalline corneoretinal dystrophy. Prog Retin Eye Res. 2025 Jul;107:101377. doi: 10.1016/j.preteyeres.2025.101377. Epub 2025 Jun 5.
- Yang L, Xing D, Zhang J, Liu B, Jiang X, Chen S, Li W, Qu B, Qiao J, Feng X, Jia H, Hu X, Yu S, Jia R, Wang L, Mahajan VB, Dou H, Lu F, Li X. Gene therapy for Bietti crystalline corneoretinal dystrophy: A phase 1/2 clinical trial. Mol Ther. 2026 Mar 4;34(3):1743-1755. doi: 10.1016/j.ymthe.2026.01.005. Epub 2026 Jan 10.
Studieavstämningsdatum
Studera stora datum
Studiestart (Beräknad)
Primärt slutförande (Beräknad)
Avslutad studie (Beräknad)
Studieregistreringsdatum
Först inskickad
Först inskickad som uppfyllde QC-kriterierna
Första postat (Faktisk)
Uppdateringar av studier
Senaste uppdatering publicerad (Faktisk)
Senaste inskickade uppdateringen som uppfyllde QC-kriterierna
Senast verifierad
Mer information
Termer relaterade till denna studie
Nyckelord
Ytterligare relevanta MeSH-villkor
Andra studie-ID-nummer
- ZYA-2025-003
Plan för individuella deltagardata (IPD)
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