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

April 9, 2026 updated by: Zhongmou Therapeutics

A Prospective, Randomized, Sham-controlled, Dose-finding Phase 1/2 Two-part Trial to Evaluate the Safety, Tolerability, and Efficacy of ZM-02 Injection in Patients With Advanced Retinitis Pigmentosa

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

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

Detailed Description

Retinitis pigmentosa (RP) is the most common inherited retinal disease, characterized by progressive loss of visual function that can lead to severe visual impairment or blindness with no effective treatment.

This Phase 1/2 study is designed to evaluate the safety, tolerability, and preliminary efficacy of a single unilateral intravitreal injection of ZM-02 at escalating dose levels in patients with advanced RP. The study includes a sham-controlled design and will assess safety outcomes as well as multiple functional and structural measures of visual performance over long-term follow-up.

Study Type

Interventional

Enrollment (Estimated)

21

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria

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

Exclusion Criteria

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Sequential Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Sham Comparator: Sham Comparator
Procedure: Sham injection
Sham IVT procedure in the study eye
Experimental: Low Dose ZM-02
Genetic: ZM-02 (low dose)
Single unilateral IVT injection of low-dose ZM-02 in the study eye
Experimental: High Dose ZM-02
Genetic: ZM-02 (high dose)
Single unilateral IVT injection of high-dose ZM-02 in the study eye
Experimental: ZM-02 (selected dose)
Genetic: ZM-02 (selected dose)
Single unilateral IVT injection of selected-dose ZM-02 in the study eye

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Safety and tolerability of ZM-02
Time Frame: Baseline to Week 52.
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 Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change from Baseline in Best-Corrected Visual Acuity (BCVA) of Both Eyes
Time Frame: Baseline to Week 52.
Baseline to Week 52.
Change from Baseline in Functional Vision (MLMT)
Time Frame: Baseline to Week 52.
Change from baseline in the multi-luminance mobility test (MLMT) performance.
Baseline to Week 52.
Change from Baseline in Vision-Related Quality of Life (NEI VFQ-25)
Time Frame: Baseline to Week 52.
Baseline to Week 52.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Zhongmou Therapeutics

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

June 25, 2026

Primary Completion (Estimated)

December 25, 2029

Study Completion (Estimated)

December 25, 2031

Study Registration Dates

First Submitted

November 26, 2025

First Submitted That Met QC Criteria

December 2, 2025

First Posted (Actual)

December 15, 2025

Study Record Updates

Last Update Posted (Actual)

April 13, 2026

Last Update Submitted That Met QC Criteria

April 9, 2026

Last Verified

April 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ZM-02-201

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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