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Not yet recruiting NCT07282457

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

Phase I / Phase II Interventional Retinitis Pigmentosa (RP)

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: ZM-02 (low dose), ZM-02 (high dose), ZM-02 (selected dose), Sham injection.
Who it may be relevant to
Registry conditions: Retinitis Pigmentosa (RP). Basic parameters: 6 years — 60 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Center list to be confirmed — check the primary protocol.
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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

Overview

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

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.

Interventions

  • Genetic ZM-02 (low dose)
    Single unilateral IVT injection of low-dose ZM-02 in the study eye
  • Genetic ZM-02 (high dose)
    Single unilateral IVT injection of high-dose ZM-02 in the study eye
  • Genetic ZM-02 (selected dose)
    Single unilateral IVT injection of selected-dose ZM-02 in the study eye
  • Procedure Sham injection
    Sham IVT procedure in the study eye

Primary outcome measures

  • Safety and tolerability of ZM-02 [Time frame: Baseline to Week 52.]
Secondary outcome measures (3)
  • Change from Baseline in Best-Corrected Visual Acuity (BCVA) of Both Eyes [Time frame: Baseline to Week 52.]
  • Change from Baseline in Functional Vision (MLMT) [Time frame: Baseline to Week 52.]
  • Change from Baseline in Vision-Related Quality of Life (NEI VFQ-25) [Time frame: Baseline to Week 52.]

Eligibility criteria

Inclusion criteria

  • 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).

Exclusion criteria

  • 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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Randomized
Model
Sequential
Masking
Quadruple blind
Primary purpose
Treatment

Study locations

Center list to be confirmed — check the primary protocol.

Identifiers

NCT: NCT07282457 · ZM-02-201

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗