Safety and Efficacy Study of Novel Gene Therapy ZM-01 for X-linked Retinoschisis Patients

X-linked Retinoschisis Clinical Trial

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

Clinical Trial 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 subject's Study Eye.

Clinical Trial 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. ;

Related Conditions & MeSH terms

• Retinoschisis
• X-linked Retinoschisis

• NCT number: NCT06066008
• Study type: Interventional
• Source: Zhongmou Therapeutics
• Contact: Pei Cao
• Phone: +86 18707134160
• Email: zmt@simbaeye.com
• Status: Recruiting
• Phase: Early Phase 1
• Start date: September 25, 2022
• Completion date: October 2027