X-linked RetinoschisisEarly Phase 1NCT06066008
Safety and Efficacy Study of Novel Gene Therapy ZM-01 for X-linked Retinoschisis Patients
A Early Phase 1 study testing ZM-01-L and ZM-01-H in people with X-linked Retinoschisis, run by Augelux Therapeutics at 1 site in China.
Trial summary
This trial is meant to evaluate the safety and efficacy of ZM-01 of X-linked retinoschisis. Unilateral intravitreal injections (IVT) will be given into the subject's Study Eye. Read more about this study
- Who is it for?
- Ages 3 to 18 years, men only
- Conditions the study is for
- X-linked Retinoschisis
- What is being tested?
- ZM-01-L and ZM-01-H
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 1 siteChina
- Study dates
- 25 Sep 2022to 31 Jan 2026
- Phase
- Early Phase 1
- Are there results?
- None posted in the registry
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 3 to 18 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- Subjects who meet all of the following criteria will be enrolled into the study
- Diagnosis of X-linked retinoschisis consistent with the presence of RS1 gene mutation
- Male, aged between 3 and 18 years old, in overall good health except for XLRS condition
- Capable of undergoing visual and retinal function assessment.
- The visual acuity of the study eye not better than: 0.4 (68 ETDRS letters equivalent)
- No carbonic anhydrase inhibitors have been used at present and for 3 months before treatment
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- Laboratory tests meet the following criteria:
- Hemoglobin ≥ 11.0 g/dL
- White blood cell counts ranged from 3,300 to 12,000 cells /mm³;
- Platelet count 125,000-550,000 /mm³;
- Alanine aminotransferase (ALT) is not higher than 1.5 times the upper limit of the normal range of laboratory tests;
- Serum creatinine was no higher than 1.1 times the upper limit of the normal range for laboratory tests;
- Prothrombin time (PT) ≤14.5 seconds and partial thromboplastin time (PTT) ≤ 36.0 seconds.
- Willing to discontinue aspirin, aspirin-containing products, and any other medications that may alter clotting function at least 7 days before dosing.
- Be able to understand and sign informed consent.
Who cannot join
- Subjects who meet any of the following exclusion criteria before enrollment were excluded from the study
- Previously received any AAV gene therapy
- The following mutations in RS1 gene: R141H, C59S or C223S
- Pre-existing eye conditions that cause severe vision loss or increase the risk of intravitreal injections (e.g., advanced glaucoma, uveitis, or severe retinal detachment)
- Ocular diseases in which there is opacity of the lens, cornea, or other media, hindering adequate observation and examination of the retina
- Use anticoagulant or antiplatelet drugs within 7 days before dosing
- Use any experimental drug within 3 months prior to registration
- Presented any situation that causes the investigator to believe the subject might not adhere to the study protocol or that participation might pose an unacceptable risk to the subject
Trial locations and contacts
1 site in 1 country.
China1 site
Wuhan University Renmin Hospital affiliated with Hanchuan Hospital
Xiaogan, Hubei
About this study
Official title: Prospective, Dose-Escalating, Investigator Initiated Trial to Evaluate the Safety and Efficacy of ZM-01 in 3-18 Year-old Male Subjects With X-linked Retinoschisis
This trial is meant to evaluate the safety and efficacy of ZM-01 of X-linked retinoschisis. Unilateral intravitreal injections (IVT) will be given into the subject's Study Eye.
Read the detailed description
X-linked retinoschisis (XLRS) is a rare, inherited retinal disease caused by mutations in the RS1 gene. Individuals affected by XLRS often experience progressive visual impairment from a young age, potentially leading to legal blindness. There is currently no established clinical treatment available. We developed an innovative adeno-associated virus (AAV)-based gene therapy for individuals with XLRS. Six to nine subjects with XLRS received a single unilateral intravitreal injection of ZM-01 at ascending doses.
- Conditions
- X-linked Retinoschisis
- Being tested
- ZM-01-L, ZM-01-H
- Keywords
- gene therapy, AAV, RS1
- Medical subject headings
- Retinoschisis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ZM-01-LDrug
Also called rAAV-hRS1
rAAV-hRS1 intravitreal injection of low dose
ZM-01-HDrug
Also called rAAV-hRS1
rAAV-hRS1 intravitreal injection of high dose
| Study group | Role | What they receive |
|---|---|---|
| group 1 | Experimental | IVT administration of a single low dose ZM-01 injection Receives: Drug: ZM-01-L |
| group 2 | Experimental | IVT administration of a single high dose ZM-01 injection Receives: Drug: ZM-01-H |
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 7, month 1, 2
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. This approach was chosen to facilitate visual acuity testing in children who cannot recognize letters, which was more appropriate for this study.
Measured: baseline to day 7, month 1, 2
Secondary outcomes (7)
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 month 3, 4, 6, 9, 12
Change in Quality of Life
Quality of Life will be measured using Pediatric Eye Questionnaire (PedEyeQ) or other similar questionnaires before and after treatment
Measured: baseline to month 9, 12
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. This approach was chosen to facilitate visual acuity testing in children who cannot recognize letters, which was more appropriate for this study.
Measured: baseline to month 3, 4, 6, 9, 12
Change in visual field
Visual field will be assessed by Humphrey perimetry, changes in VFI, MD, PSD will be analyzed.
Measured: baseline to month 1, 2, 3, 4, 6, 9, 12
Change in electrophysiology result
The ERG measurement will be performed based on the standards of international society for clinical electrophysiology of vision (ISCEV).
Measured: baseline to month 1, 2, 3, 4, 6, 9, 12
Anti-AAV neutralizing antibody titer, Anti-RS1 neutralizing antibody titer
Peripheral blood samples were collected from each subjects to measure the AAV8 antibody levels and virus titers in the peripheral blood.
Measured: baseline to day 1, 7 and month 1, 2
Change in the retina cavity assessed by macular OCT
Optical coherence tomography (OCT) of the macula was performed in both eyes of each participant at each visit.
Measured: baseline to day 7, month 1, 2, 3, 4, 6, 9, 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Oct 2023
- Study start25 Sep 2022
- Primary completion31 Oct 2023
- Study completion31 Jan 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for X-linked RetinoschisisStudies 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
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Augelux Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Yin Shen, PhD, Study chair, Zhongmou Theraputics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06066008
- Sponsor's protocol ID
- ZM-01
- Status last verified
- Mar 2026
- First submitted
- 25 Sep 2023
- Last updated
- 31 Mar 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: publish research paper
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-01 in 3-18 Year-old Male Subjects With X-linked Retinoschisis
- ClinicalTrials.gov ID
- NCT06066008
- Acronym / sponsor ID
- ZM-01
- Conditions
- X-linked Retinoschisis
- Keywords
- gene therapy, AAV, RS1
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Augelux TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 25 Sep 2022 (actual)
- Primary completion
- 31 Oct 2023 (actual)
- Study completion
- 31 Jan 2026 (actual)
- First posted
- 4 Oct 2023 (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 trial is meant to evaluate the safety and efficacy of ZM-01 of X-linked retinoschisis. Unilateral intravitreal injections (IVT) will be given into the subject's Study Eye.
Detailed description
X-linked retinoschisis (XLRS) is a rare, inherited retinal disease caused by mutations in the RS1 gene. Individuals affected by XLRS often experience progressive visual impairment from a young age, potentially leading to legal blindness. There is currently no established clinical treatment available. We developed an innovative adeno-associated virus (AAV)-based gene therapy for individuals with XLRS. Six to nine subjects with XLRS received a single unilateral intravitreal injection of ZM-01 at ascending doses.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| group 1 | Experimental | Drug: ZM-01-LIVT administration of a single low dose ZM-01 injection |
| group 2 | Experimental | Drug: ZM-01-HIVT administration of a single high dose ZM-01 injection |
Who may qualify
- Ages
- 3 to 18 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (15 listed)
- Subjects who meet all of the following criteria will be enrolled into the study
- Diagnosis of X-linked retinoschisis consistent with the presence of RS1 gene mutation
- Male, aged between 3 and 18 years old, in overall good health except for XLRS condition
- Capable of undergoing visual and retinal function assessment.
- The visual acuity of the study eye not better than: 0.4 (68 ETDRS letters equivalent)
Key exclusion criteria (8 listed)
- Subjects who meet any of the following exclusion criteria before enrollment were excluded from the study
- Previously received any AAV gene therapy
- The following mutations in RS1 gene: R141H, C59S or C223S
- Pre-existing eye conditions that cause severe vision loss or increase the risk of intravitreal injections (e.g., advanced glaucoma, uveitis, or severe retinal detachment)
- Ocular diseases in which there is opacity of the lens, cornea, or other media, hindering adequate observation and examination of the retina
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-01-LAlso: rAAV-hRS1rAAV-hRS1 intravitreal injection of low dose | Drug | group 1 |
| ZM-01-HAlso: rAAV-hRS1rAAV-hRS1 intravitreal injection of high dose | Drug | group 2 |
Primary outcome
Incidence of adverse events and serious adverse events
Time frame: baseline to day 7, month 1, 2
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.
Change in best corrected visual acuity (BCVA)
Time frame: baseline to day 7, month 1, 2
BCVA of both eyes will be assessed using the early treatment of diabetic retinopathy study (ETDRS) chart or tumbling "E" chart. This approach was chosen to facilitate visual acuity testing in children who cannot recognize letters, which was more appropriate for this study.
7 secondary outcomes
Incidence of adverse events and serious adverse events
Time frame: baseline to month 3, 4, 6, 9, 12
Change in Quality of Life
Time frame: baseline to month 9, 12
Change in best corrected visual acuity (BCVA)
Time frame: baseline to month 3, 4, 6, 9, 12
Change in visual field
Time frame: baseline to month 1, 2, 3, 4, 6, 9, 12
Change in electrophysiology result
Time frame: baseline to month 1, 2, 3, 4, 6, 9, 12
Anti-AAV neutralizing antibody titer, Anti-RS1 neutralizing antibody titer
Time frame: baseline to day 1, 7 and month 1, 2
Change in the retina cavity assessed by macular OCT
Time frame: baseline to day 7, month 1, 2, 3, 4, 6, 9, 12
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Mar 2026.
- Individual participant dataSharing planned: No. publish research paper
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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. This study is not recruiting at the moment.
Yin Shen, PhD study chair
Zhongmou Theraputics
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-01-L and ZM-01-H in people with X-linked Retinoschisis, 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 15 inclusion and 8 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: finished, 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. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. 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 7, month 1, 2). 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.