NCT06292650

Safety and Efficacy Study of Novel Gene Therapy ZM-02 for Retinitis Pigmentosa Patients

Prospective, Dose-Escalating, Investigator Initiated Trial to Evaluate the Safety and Efficacy of ZM-02 in Retinitis Pigmentosa

• Status: Recruiting
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 12 (estimated)
• Sponsor: Zhongmou Therapeutics · Industry
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

This is zM-02's safety, tOlerability, and efficacy in retinitis pigmentOsa first-in-humaN study (MOON). This trial is meant to evaluate the safety and efficacy of ZM-02 in Retinitis pigmentosa (RP) patients. Unilateral intravitreal injections (IVT) will be given into the subject's Study Eye.

Detailed description

Retinitis pigmentosa (RP) is the most common inherited retinal disease. Individuals affected by RP often experience progressive visual impairment, potentially leading to legal blindness. There is currently no established effective clinical treatment available. We developed an innovative adeno-associated virus (AAV)-based gene therapy for individuals with RP, regardless of their causative mutations. Eight to twelve subjects with RP will be recruited and six to nine of them will receive a single unilateral intravitreal injection of ZM-02 at ascending doses, while two to three receive sham injections as the control group.

Conditions

• Retinitis Pigmentosa

Interventions

Type	Name	Description
DRUG	ZM-02-L	rAAV-PsCatCh2.0 intravitreal injection of low dose
DRUG	ZM-02-H	rAAV-PsCatCh2.0 intravitreal injection of high dose
PROCEDURE	ZM-02-S	sham intravitreal injection of ZM-02 (not actual injection)

Timeline

• Start date: 2024-02-25
• Primary completion: 2027-12-25
• Completion: 2028-12-25
• First posted: 2024-03-05
• Last updated: 2026-03-31

Locations

1 site across 1 country: China