NCT06066008

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

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: Completed
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 2 (actual)
• Sponsor: Zhongmou Therapeutics · Industry
• Sex: Male
• Age: 3 Years – 18 Years
• Healthy volunteers: Not accepted

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.

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.

Conditions

• X-linked Retinoschisis

Interventions

Type	Name	Description
DRUG	ZM-01-L	rAAV-hRS1 intravitreal injection of low dose
DRUG	ZM-01-H	rAAV-hRS1 intravitreal injection of high dose

Timeline

• Start date: 2022-09-25
• Primary completion: 2023-10-31
• Completion: 2026-01-31
• First posted: 2023-10-04
• Last updated: 2026-03-31

Locations

1 site across 1 country: China