Ignite Creation Date:
2024-05-06 @ 7:35 PM
Last Modification Date:
2024-10-26 @ 3:10 PM
Study NCT ID:
NCT06066008
Status:
RECRUITING
Last Update Posted:
2024-02-21
First Post:
2023-09-25
Brief Title:
Safety and Efficacy Study of Novel Gene Therapy ZM-01 for X-linked Retinoschisis Patients
Sponsor:
Zhongmou Therapeutics
Organization:
Zhongmou Therapeutics
Study Overview
Official Title:
Prospective Dose-Escalating Investigator Initiated Trial to Evaluate the Safety and Efficacy of ZM-01 in 3-18 Year-old Male Subjects With X-linked Retinoschisis
Status:
RECRUITING
Status Verified Date:
2024-02
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:
This trial is meant to evaluate the safety and efficacy of ZM-01 of X-linked retinoschisis Unilateral intravitreal injections IVT will be given into the subjects Study Eye
Detailed Description:
X-linked retinoschisis XLRS is a rare inherited retinal disease caused by mutations in the RS1 gene Individuals affected by XLRS often experience progressive visual impairment from a young age potentially leading to legal blindness There is currently no established clinical treatment available We developed an innovative adeno-associated virus AAV-based gene therapy for individuals with XLRS Six to nine subjects with XLRS received a single unilateral intravitreal injection of ZM-01 at ascending doses
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