Retinitis Pigmentosa (RP)Phase 1/2PRISMNCT07282457
Prospective, Randomized, Sham-controlled, Dose-finding I/II Trial of Safety and Efficacy of Modified Optogenetic Gene Therapy (ZM-02 Injection)
A Phase 1/2 study testing ZM-02 (low dose), ZM-02 (high dose) and ZM-02 (selected dose) in people with Retinitis Pigmentosa (RP), run by Zhongmou Therapeutics.
Trial 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). Read more about this study
- Who is it for?
- Ages 6 to 60 years
- Conditions the study is for
- Retinitis Pigmentosa (RP)
- What is being tested?
- ZM-02 (low dose), ZM-02 (high dose) and ZM-02 (selected dose)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 21 participants
- Where
- No sites listed yet
- Study dates
- 25 Jun 2026to 25 Dec 2031
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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).
Who cannot join
- 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.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
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
This is a Phase 1/2, multi-center, randomized, sham-controlled, dose-escalation study evaluating ZM-02 in patients with advanced retinitis pigmentosa (RP).
Read the 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.
- Conditions
- Retinitis Pigmentosa (RP)
- Being tested
- ZM-02 (low dose), ZM-02 (high dose), ZM-02 (selected dose), Sham injection
- Keywords
- gene therapy, AAV, RP, optogenetics
- Medical subject headings
- Retinitis Pigmentosa
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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 injectionProcedure or surgery
Sham IVT procedure in the study eye
| Study group | Role | What they receive |
|---|---|---|
| Low Dose ZM-02 | Experimental | Not described Receives: Genetic: ZM-02 (low dose) |
| High Dose ZM-02 | Experimental | Not described Receives: Genetic: ZM-02 (high dose) |
| ZM-02 (selected dose) | Experimental | Not described Receives: Genetic: ZM-02 (selected dose) |
| Sham Comparator | Sham comparator | Not described Receives: Procedure: Sham injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Baseline to Week 52.
Secondary outcomes (3)
Change from Baseline in Best-Corrected Visual Acuity (BCVA) of Both Eyes
Measured: Baseline to Week 52.
Change from Baseline in Functional Vision (MLMT)
Change from baseline in the multi-luminance mobility test (MLMT) performance.
Measured: Baseline to Week 52.
Change from Baseline in Vision-Related Quality of Life (NEI VFQ-25)
Measured: Baseline to Week 52.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Dec 2025
- Study start25 Jun 2026 (estimated)
- Primary completion25 Dec 2029 (estimated)
- Study completion25 Dec 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Retinitis Pigmentosa (RP)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 21 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Zhongmou Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07282457
- Sponsor's protocol ID
- ZM-02-201
- Acronym
- PRISM
- Status last verified
- Apr 2026
- First submitted
- 26 Nov 2025
- Last updated
- 13 Apr 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT07282457
- Acronym / sponsor ID
- PRISM / ZM-02-201
- Conditions
- Retinitis Pigmentosa (RP)
- Keywords
- gene therapy, AAV, RP, optogenetics
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 21 participants (estimated)
- Lead sponsor
- Zhongmou TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 25 Jun 2026 (estimated)
- Primary completion
- 25 Dec 2029 (estimated)
- Study completion
- 25 Dec 2031 (estimated)
- First posted
- 15 Dec 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| 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 |
Who may qualify
- Ages
- 6 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- 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).
Key exclusion criteria (7 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ZM-02 (low dose)Single unilateral IVT injection of low-dose ZM-02 in the study eye | Genetic | Low Dose ZM-02 |
| ZM-02 (high dose)Single unilateral IVT injection of high-dose ZM-02 in the study eye | Genetic | High Dose ZM-02 |
| ZM-02 (selected dose)Single unilateral IVT injection of selected-dose ZM-02 in the study eye | Genetic | ZM-02 (selected dose) |
| Sham injectionSham IVT procedure in the study eye | Procedure or surgery | Sham Comparator |
Primary outcome
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.
3 secondary outcomes
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.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Apr 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing ZM-02 (low dose), ZM-02 (high dose) and ZM-02 (selected dose) in people with Retinitis Pigmentosa (RP), run by Zhongmou Therapeutics.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 13 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Windy Zhou (+86 18986214263, zmt@zmtherapeutics.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and tolerability of ZM-02 (time frame: Baseline to Week 52.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Apr 2026.