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

Zhongmou Therapeutics

Status and phase: Begins enrollment this month; Phase 2; Phase 1

Conditions: Retinitis Pigmentosa (RP)

Treatments: Procedure: Sham injection; Genetic: ZM-02 (low dose); Genetic: ZM-02 (high dose); Genetic: ZM-02 (selected dose)

Study type: Interventional

Funder types: Industry

Identifiers

NCT07282457

ZM-02-201

Details and patient eligibility

About

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

Full 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.

Enrollment: 21 estimated patients

Sex: All

Ages: 6 to 60 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Sequential Assignment

Masking: Quadruple Blind

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21 participants in 4 patient groups

Low Dose ZM-02

Experimental group

Treatment:

Genetic: ZM-02 (low dose)

High Dose ZM-02

Experimental group

Treatment:

Genetic: ZM-02 (high dose)

ZM-02 (selected dose)

Experimental group

Treatment:

Genetic: ZM-02 (selected dose)

Sham Comparator

Sham Comparator group

Treatment:

Procedure: Sham injection