Prospective, Randomized, Sham-controlled, Dose-finding I/II Trial of Safety and Efficacy of Modified Optogenetic Gene Therapy (ZM-02 Injection)

NCT07282457 | Not Yet Recruiting Phase 1

• 1 other identifier: ZM-02-201
• Study Type: interventional
• Target: 21
• Locations: 0 countries
• Sites: N/A

Brief Summary

This is a Phase 1/2, multi-center, randomized, sham-controlled, dose-escalation study evaluating ZM-02 in patients with advanced retinitis pigmentosa (RP).

Conditions

Retinitis Pigmentosa (RP)

Keywords

gene therapy; AAV; RP; optogenetics

Outcome Measures

Primary Outcomes (1)

• Safety and tolerability of ZM-02

  Incidence of severity of ocular and systemic adverse events (AEs) and serious adverse events (SAEs) following a single intravitreal injection of ZM-02.

  Baseline to Week 52.

Secondary Outcomes (3)

• Change from Baseline in Best-Corrected Visual Acuity (BCVA) of Both Eyes

  Baseline to Week 52.

• Change from Baseline in Functional Vision (MLMT)

  Baseline to Week 52.

• Change from Baseline in Vision-Related Quality of Life (NEI VFQ-25)

  Baseline to Week 52.

Study Arms (4)

Low Dose ZM-02

EXPERIMENTAL

Genetic: ZM-02 (low dose)

High Dose ZM-02

EXPERIMENTAL

Genetic: ZM-02 (high dose)

ZM-02 (selected dose)

EXPERIMENTAL

Genetic: ZM-02 (selected dose)

Sham Comparator

SHAM COMPARATOR

Procedure: Sham injection

Interventions

ZM-02 (selected dose) GENETIC

Single unilateral IVT injection of selected-dose ZM-02 in the study eye

ZM-02 (selected dose)

Sham injection PROCEDURE

Sham IVT procedure in the study eye

Sham Comparator

ZM-02 (low dose) GENETIC

Single unilateral IVT injection of low-dose ZM-02 in the study eye

Low Dose ZM-02

ZM-02 (high dose) GENETIC

Single unilateral IVT injection of high-dose ZM-02 in the study eye

High Dose ZM-02

Eligibility Criteria

• Age: 6 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Male or female participants aged 6 to 60 years.
• Clinical diagnosis of advanced retinitis pigmentosa (RP).
• Best corrected visual acuity at or below the protocol-defined threshold in the study eye.
• Presence of sufficient retinal structure in the study eye as determined by screening assessments.
• Ability to understand and sign informed consent (and assent, when applicable).

You may not qualify if:

• Presence of other ocular conditions that could interfere with study assessments.
• History of significant ocular surgery in the study eye within a protocol-defined period.
• Active ocular infection or inflammation.
• Clinically significant systemic disease that could increase study risk or interfere with assessments.
• Positive screening for clinically significant infectious diseases, as defined in the protocol.
• Pregnant or breastfeeding individuals.
• Any condition that, in the investigator's judgment, would make the participant unsuitable for study participation.

Sponsors & Collaborators

• Zhongmou Therapeutics: lead

MeSH Terms

Conditions

Retinitis Pigmentosa

Interventions

salicylhydroxamic acid

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Retinal Dystrophies; Retinal Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Windy Zhou

CONTACT

+86 18986214263; zmt@zmtherapeutics.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 26, 2025
• First Posted: December 15, 2025
• Study Start (Estimated): June 25, 2026
• Primary Completion (Estimated): December 25, 2029
• Study Completion (Estimated): December 25, 2031
• Last Updated: April 13, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share