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

ConditionRetinitis Pigmentosa (RP)
Trial statusNot yet recruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age6-60
SponsorZhongmou Therapeutics

About this trial

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

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

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.

Trial design

Design model

Sequential

Treatments tested in this trial

  • ZM-02 (low dose)

    Genetic

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

  • ZM-02 (high dose)

    Genetic

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

  • ZM-02 (selected dose)

    Genetic

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

  • Sham injection

    Procedure/Surgery

    Sham IVT procedure in the study eye

Treatment groups

21 Participants
are divided into 4 treatment groups
Group A: Low Dose ZM-02Experimental treatment 1 intervention
Group B: High Dose ZM-02Experimental treatment 1 intervention
Group C: ZM-02 (selected dose)Experimental treatment 1 intervention
Group D: Sham ComparatorSham comparator 1 intervention

Trial outcomes

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.

Time frame
Baseline to Week 52.

Secondary outcomes

1

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

Time frame
Baseline to Week 52.
2

Change from Baseline in Functional Vision (MLMT)

Change from baseline in the multi-luminance mobility test (MLMT) performance.

Time frame
Baseline to Week 52.
3

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

Time frame
Baseline to Week 52.

Other outcomes

Locations

This trial has no locations.

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Zhongmou Therapeutics

Lead sponsor

Source ClinicalTrials.gov

This trial is not recruiting at the moment. You can still explore other options: