Ignite Creation Date:
2024-05-06 @ 8:14 PM
Last Modification Date:
2024-10-26 @ 3:23 PM
Study NCT ID:
NCT06300476
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-03-08
First Post:
2024-03-03
Brief Title:
Safety and Efficacy of a Single Subretinal Injection of JWK006 Gene Therapy in Subjects With Stargardt DiseaseSTGD1
Sponsor:
West China Hospital
Organization:
West China Hospital
Study Overview
Official Title:
Safety and Efficacy of a Single Subretinal Injection of JWK006 Gene Therapy in Subjects With Stargardt DiseaseSTGD1
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2024-03
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The purpose of the study is to assess the safety and efficacy of JWK006 in Stargardt DiseaseSTGD1
JWK006 is packed by adeno-associated virus vector that expressing ABCA4 gene
JWK006 is packed by adeno-associated virus vector that expressing ABCA4 gene
Detailed Description:
This clinical study is a single-center non-randomized non-blinded self-controlled dose-escalation clinical study aiming to evaluate the safety tolerability and preliminary effectiveness of JWK006 injection in the treatment of approximately 12 patientsaged 10 to 18 years with Stargardt DiseaseSTGD1genotyped with ABCA4 gene mutations
In this study a 33 dose escalation trial was adopted with 3 doses low dose medium dose and high dose set up and 9 subjects were planned to be enrolled 3 in each dose group Following the principle of dose escalation starting from the low-dose treatment group one subject was enrolled in each dose group In order to fully protect the safety of subjects it is required to adopt an orderly enrollment method within each dose group with one subject enrolled at a time No obvious toxic or side effects are observed 28 days after the completion of the administration The next subject in the group At the same time 28 days after the last subject in each dose group is administered the researchers and others will jointly evaluate the safety results of the dose group before deciding whether to enter the next dose group
In this study a 33 dose escalation trial was adopted with 3 doses low dose medium dose and high dose set up and 9 subjects were planned to be enrolled 3 in each dose group Following the principle of dose escalation starting from the low-dose treatment group one subject was enrolled in each dose group In order to fully protect the safety of subjects it is required to adopt an orderly enrollment method within each dose group with one subject enrolled at a time No obvious toxic or side effects are observed 28 days after the completion of the administration The next subject in the group At the same time 28 days after the last subject in each dose group is administered the researchers and others will jointly evaluate the safety results of the dose group before deciding whether to enter the next dose group
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None