Retinitis PigmentosaEarly Phase 1MOONNCT06292650
Safety and Efficacy Study of Novel Gene Therapy ZM-02 for Retinitis Pigmentosa Patients
A Early Phase 1 study testing ZM-02-L, ZM-02-H and ZM-02-S in people with Retinitis Pigmentosa, run by Augelux Therapeutics at 1 site in China.
Trial summary
This is zM-02's safety, tOlerability, and efficacy in retinitis pigmentOsa first-in-humaN study (MOON). This trial is meant to evaluate the safety and efficacy of ZM-02 in Retinitis pigmentosa (RP) patients. Unilateral intravitreal injections (IVT) will be given into the subject's Study Eye. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Retinitis Pigmentosa
- What is being tested?
- ZM-02-L, ZM-02-H and ZM-02-S
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 12 participants
- Where
- 1 siteChina
- Study dates
- 25 Feb 2024to 25 Dec 2028
- Phase
- Early Phase 1
- 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 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients who meet all of the following criteria can be selected as subjects:
- Clinically diagnosed with retinal pigment degeneration
- The visual acuity of study eye is no better than the finger counting, while the visual acuity of the study eye is not better than that of the contralateral eye
- The subject has had visual experience above the finger counting
- In the OCT examination of the tested eye, the disappearance of the ellipsoid zone is observed, but the inner nuclear layer and the nerve fiber layer of the retina are still present
- The refractive power of the tested eye is between -6.00 D and +6.00 D
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- Not infected with the Human Immunodeficiency Virus (HIV) and other acute and chronic infectious diseases
- Voluntarily sign an Informed Consent Form (ICF), and the age is not less than 18 years and not more than 65 years
- Able to fully understand and agree to cooperate with the implementation of the research protocol
Who cannot join
- Subjects who meet any one of the following exclusion criteria will be excluded from the study:
- Pregnant women, breastfeeding women, or male and female subjects who do not agree to contraception during the 12 months before and after medication
- Subjects with narrow anterior chamber angles or any other medical conditions that contraindicate pupil dilation
- Subjects allergic to corticosteroids, who are unable to tolerate the corticosteroid treatment described in the protocol, or have active 4. concurrent infections that contraindicate treatment
- Subjects with systemic diseases, or other medical or mental illnesses, or other safety concerns for the study
- Subjects with other symptoms and/or diseases or conditions that can alter visual function, including but not limited to glaucoma and central nervous system lesions (mild cataracts are not included in this restriction)
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- Eye diseases that may interfere with the assessment of vision during the study and/or interfere with other ocular assessments such as OCT
- Diseases that may affect the clinical trial, such as tumors, metabolic, immune-related diseases, etc.
- Subjects who have undergone major eye surgery within the last 3 months before screening
- Subjects with a history of malignant tumors within the last 5 years
- Subjects with other retinal diseases not suitable for this study, such as retinal detachment
- Patients undergoing or potentially undergoing immunosuppressive treatment for other diseases, excluding this study
- Participation in any clinical trials other than this study within the last 3 months
- Subjects who have received gene therapy outside of this study
- Other reasons deemed by the researcher as unsuitable for participation in this study
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Yin Shen contact
China1 site, 1 recruiting
- Recruiting
Beijing Tongren Hospital of Capital Medical University
Beijing, Beijing Municipality
Windy Zhou, +86 18986214263, zmt@zmtherapeutics.com
Yin Shen
About this study
Official title: Prospective, Dose-Escalating, Investigator Initiated Trial to Evaluate the Safety and Efficacy of ZM-02 in Retinitis Pigmentosa
This is zM-02's safety, tOlerability, and efficacy in retinitis pigmentOsa first-in-humaN study (MOON). This trial is meant to evaluate the safety and efficacy of ZM-02 in Retinitis pigmentosa (RP) patients. Unilateral intravitreal injections (IVT) will be given into the subject's Study Eye.
Read the detailed description
Retinitis pigmentosa (RP) is the most common inherited retinal disease. Individuals affected by RP often experience progressive visual impairment, potentially leading to legal blindness. There is currently no established effective clinical treatment available. We developed an innovative adeno-associated virus (AAV)-based gene therapy for individuals with RP, regardless of their causative mutations. Eight to twelve subjects with RP will be recruited and six to nine of them will receive a single unilateral intravitreal injection of ZM-02 at ascending doses, while two to three receive sham injections as the control group.
- Conditions
- Retinitis Pigmentosa
- Being tested
- ZM-02-L, ZM-02-H, ZM-02-S
- 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-LDrug
Also called rAAV-PsCatCh2.0e, rAAV-PsCatCh2.0
rAAV-PsCatCh2.0 intravitreal injection of low dose
ZM-02-HDrug
Also called rAAV-PsCatCh2.0e, rAAV-PsCatCh2.0
rAAV-PsCatCh2.0 intravitreal injection of high dose
ZM-02-SProcedure or surgery
Also called Sham injection
sham intravitreal injection of ZM-02 (not actual injection)
| Study group | Role | What they receive |
|---|---|---|
| group 1 | Experimental | IVT administration of a single low dose ZM-02 injection Receives: Drug: ZM-02-L |
| group 2 | Experimental | IVT administration of a single high dose ZM-02 injection Receives: Drug: ZM-02-H |
| group 3 | Sham comparator | Sham IVT injection Receives: Procedure: ZM-02-S |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of adverse events and serious adverse events
An adverse event (AE) is any untoward medical occurrence in a clinical investigation participant administered a product; the event will not need to have a causal relationship with the treatment.
A serious adverse event (SAE) is any untoward medical occurrence at any dose that leading to the following: Results in death; Life-threatening, refers to an event in which the patient is at risk of death at the time of the event; it does not refer to an event which hypothetically might have caused death if it were more severe; Significant or permanent disability/incapacity, where disability refers to a serious disruption and damage of a person's ability to perform normal life functions; Requires inpatient hospitalization or prolongation of existing hospitalization; Congenital anomaly or birth defect; Other medically important events.
Measured: baseline to day 3, week 1, 4, 12, 24, 36, 52
Changes in intraocular pressure (IOP) in Subjects
IOP refers to the fluid pressure inside the eye. Monitoring IOP ensures that interventions do not inadvertently increase IOP to dangerous levels. IOP will be measured using a clinical tonometry device.
Measured: baseline to day 3, week 1, 4, 12, 24, 36, 52
Secondary outcomes (3)
Change of MLMT level
Multi-Luminance Mobility Test (MLMT) is used to evaluate how well individuals with visual impairments can navigate and perform tasks in different lighting conditions. This test is particularly relevant for conditions that affect night vision or light adaptation, such as retinitis pigmentosa or other forms of inherited retinal dystrophies.
Measured: baseline to day 3, week 1, 4, 12, 24, 36, 52
Change of Quality of Life
Quality of Life will be measured using Visual Function Questionnaire (VFQ-25) or other similar questionnaires before and after treatment
Measured: baseline to day 3, week 1, 4, 12, 24, 36, 52
Change in best corrected visual acuity (BCVA)
BCVA of both eyes will be assessed using the early treatment of diabetic retinopathy study (ETDRS) chart or tumbling "E" chart or other avaliable measurement. This approach was chosen to facilitate visual acuity testing in subject who cannot recognize letters.
Measured: baseline to day 3, week 1, 4, 12, 24, 36, 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Mar 2024
- Study start25 Feb 2024
- Primary completion25 Dec 2027 (estimated)
- Study completion25 Dec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Retinitis PigmentosaStudies 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Care provider
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Augelux Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Wenbin Wei, PhD, Principal investigator, Beijing Tongren Hospital, CMU
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06292650
- Sponsor's protocol ID
- MOON-01
- Acronym
- MOON
- Status last verified
- Mar 2026
- First submitted
- 27 Feb 2024
- Last updated
- 31 Mar 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 31 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective, Dose-Escalating, Investigator Initiated Trial to Evaluate the Safety and Efficacy of ZM-02 in Retinitis Pigmentosa
- ClinicalTrials.gov ID
- NCT06292650
- Acronym / sponsor ID
- MOON / MOON-01
- Conditions
- Retinitis Pigmentosa
- Keywords
- gene therapy, AAV, RP, optogenetics
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Augelux TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 25 Feb 2024 (actual)
- Primary completion
- 25 Dec 2027 (estimated)
- Study completion
- 25 Dec 2028 (estimated)
- First posted
- 5 Mar 2024 (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 zM-02's safety, tOlerability, and efficacy in retinitis pigmentOsa first-in-humaN study (MOON). This trial is meant to evaluate the safety and efficacy of ZM-02 in Retinitis pigmentosa (RP) patients. Unilateral intravitreal injections (IVT) will be given into the subject's Study Eye.
Detailed description
Retinitis pigmentosa (RP) is the most common inherited retinal disease. Individuals affected by RP often experience progressive visual impairment, potentially leading to legal blindness. There is currently no established effective clinical treatment available. We developed an innovative adeno-associated virus (AAV)-based gene therapy for individuals with RP, regardless of their causative mutations. Eight to twelve subjects with RP will be recruited and six to nine of them will receive a single unilateral intravitreal injection of ZM-02 at ascending doses, while two to three receive sham injections as the control group.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, care provider)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| group 1 | Experimental | Drug: ZM-02-LIVT administration of a single low dose ZM-02 injection |
| group 2 | Experimental | Drug: ZM-02-HIVT administration of a single high dose ZM-02 injection |
| group 3 | Sham comparator | Procedure: ZM-02-SSham IVT injection |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Clinically diagnosed with retinal pigment degeneration
- The visual acuity of study eye is no better than the finger counting, while the visual acuity of the study eye is not better than that of the contralateral eye
- The subject has had visual experience above the finger counting
- In the OCT examination of the tested eye, the disappearance of the ellipsoid zone is observed, but the inner nuclear layer and the nerve fiber layer of the retina are still present
- The refractive power of the tested eye is between -6.00 D and +6.00 D
Key exclusion criteria (15 listed)
- Subjects who meet any one of the following exclusion criteria will be excluded from the study:
- Pregnant women, breastfeeding women, or male and female subjects who do not agree to contraception during the 12 months before and after medication
- Subjects with narrow anterior chamber angles or any other medical conditions that contraindicate pupil dilation
- Subjects allergic to corticosteroids, who are unable to tolerate the corticosteroid treatment described in the protocol, or have active 4. concurrent infections that contraindicate treatment
- Subjects with systemic diseases, or other medical or mental illnesses, or other safety concerns for the study
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-LAlso: rAAV-PsCatCh2.0e, rAAV-PsCatCh2.0rAAV-PsCatCh2.0 intravitreal injection of low dose | Drug | group 1 |
| ZM-02-HAlso: rAAV-PsCatCh2.0e, rAAV-PsCatCh2.0rAAV-PsCatCh2.0 intravitreal injection of high dose | Drug | group 2 |
| ZM-02-SAlso: Sham injectionsham intravitreal injection of ZM-02 (not actual injection) | Procedure or surgery | group 3 |
Primary outcome
Incidence of adverse events and serious adverse events
Time frame: baseline to day 3, week 1, 4, 12, 24, 36, 52
An adverse event (AE) is any untoward medical occurrence in a clinical investigation participant administered a product; the event will not need to have a causal relationship with the treatment. A serious adverse event (SAE) is any untoward medical occurrence at any dose that leading to the following: Results in death; Life-threatening, refers to an event in which the patient is at risk of death at the time of the event; it does not refer to an event which hypothetically might have caused death if it were more severe; Significant or permanent disability/incapacity, where disability refers to a serious disruption and damage of a person's ability to perform normal life functions; Requires inpatient hospitalization or prolongation of existing hospitalization; Congenital anomaly or birth defect; Other medically important events.
Changes in intraocular pressure (IOP) in Subjects
Time frame: baseline to day 3, week 1, 4, 12, 24, 36, 52
IOP refers to the fluid pressure inside the eye. Monitoring IOP ensures that interventions do not inadvertently increase IOP to dangerous levels. IOP will be measured using a clinical tonometry device.
3 secondary outcomes
Change of MLMT level
Time frame: baseline to day 3, week 1, 4, 12, 24, 36, 52
Change of Quality of Life
Time frame: baseline to day 3, week 1, 4, 12, 24, 36, 52
Change in best corrected visual acuity (BCVA)
Time frame: baseline to day 3, week 1, 4, 12, 24, 36, 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 31 Mar 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.
Yin Shen contact
Wenbin Wei, PhD principal investigator
Beijing Tongren Hospital, CMU
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing ZM-02-L, ZM-02-H and ZM-02-S in people with Retinitis Pigmentosa, run by Augelux Therapeutics at 1 site in China.
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 9 inclusion and 15 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: looking for participants now, as of the sponsor's last update on 31 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
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 phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
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?
Incidence of adverse events and serious adverse events (time frame: baseline to day 3, week 1, 4, 12, 24, 36, 52). The study lists 2 primary outcomes in all. 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 31 Mar 2026.