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Clinical Trial Details — Status: Not yet recruiting

Administrative data

NCT number NCT06292650
Other study ID # ZM-02
Secondary ID
Status Not yet recruiting
Phase Early Phase 1
First received
Last updated
Start date February 25, 2024
Est. completion date December 25, 2028

Study information

Verified date February 2024
Source Zhongmou Therapeutics
Contact Pei Cao
Phone +86 18707134160
Email zmt@simbaeye.com
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

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.


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.


Recruitment information / eligibility

Status Not yet recruiting
Enrollment 12
Est. completion date December 25, 2028
Est. primary completion date December 25, 2027
Accepts healthy volunteers No
Gender All
Age group 18 Years to 65 Years
Eligibility Inclusion Criteria: Patients who meet all of the following criteria can be selected as subjects: 1. Clinically diagnosed with retinal pigment degeneration 2. The vision of the eye being tested is no better than the index value, while the vision of the opposite eye is not better than that of the tested eye 3. The subject has had visual experience above the index value 4. 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 5. The refractive power of the tested eye is between -6.00 D and +6.00 D 6. Not infected with the Human Immunodeficiency Virus (HIV) and other acute and chronic infectious diseases 7. Voluntarily sign an Informed Consent Form (ICF), and the age is not less than 18 years and not more than 65 years 8. Able to fully understand and agree to cooperate with the implementation of the research protocol Exclusion Criteria: Subjects who meet any one of the following exclusion criteria will be excluded from the study: 1. Pregnant women, breastfeeding women, or male and female subjects who do not agree to contraception during the 12 months before and after medication 2. Subjects with narrow anterior chamber angles or any other medical conditions that contraindicate pupil dilation 3. 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 4. Subjects with systemic diseases, or other medical or mental illnesses, or other safety concerns for the study 5. 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) 6. Eye diseases that may interfere with the assessment of vision during the study and/or interfere with other ocular assessments such as OCT 7. Diseases that may affect the clinical trial, such as tumors, metabolic, immune-related diseases, etc. 8. Subjects who have undergone major eye surgery within the last 3 months before screening 9. Subjects with a history of malignant tumors within the last 5 years 10. Subjects with other retinal diseases not suitable for this study, such as retinal detachment 11. Patients undergoing or potentially undergoing immunosuppressive treatment for other diseases, excluding this study 12. Participation in any clinical trials other than this study within the last 3 months 13. Subjects who have received gene therapy outside of this study 14. Other reasons deemed by the researcher as unsuitable for participation in this study

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
ZM-02-L
rAAV-PsCatCh2.0 intravitreal injection of low dose
ZM-02-H
rAAV-PsCatCh2.0 intravitreal injection of high dose
Procedure:
ZM-02-S
sham intravitreal injection of ZM-02 (not actual injection)

Locations

Country Name City State
China Beijing Tongren Hospital of Capital Medical University Beijing Beijing

Sponsors (1)

Lead Sponsor Collaborator
Zhongmou Therapeutics

Country where clinical trial is conducted

China, 

Outcome

Type Measure Description Time frame Safety issue
Other 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. baseline to day 3, week 1, 4, 16, 24, 36, 52
Other Change in visual field (VF) Visual field will be assessed by Humphrey perimetry, changes in VFI, MD, PSD will be analyzed. baseline to day 3, week 1, 4, 16, 24, 36, 52
Other Change in Fundus Autofluorescence (FAF) FAF is a non-invasive imaging technique that provides critical information about the health of the retina, which is vital for the functioning of the retina. baseline to day 3, week 1, 4, 16, 24, 36, 52
Other Change in central foveal thickness (CFT) using Optical Coherence Tomography (OCT) Central Foveal Thickness refers to the thickness of the retina at the fovea, the central part of the macula. Changes in CFT can indicate various retinal conditions, including macular edema, macular degeneration, and central serous retinopathy. baseline to day 3, week 1, 4, 16, 24, 36, 52
Other Change in central macular thickness (CMT) using Optical Coherence Tomography (OCT) Central Macular Thickness is the measurement of the thickness of the macula, which includes the fovea and the small surrounding area. The macula is responsible for central vision and visual acuity. baseline to day 3, week 1, 4, 16, 24, 36, 52
Other Changes in the fundus using fundus color photography Fundus photography is used to document and monitor changes in the eye over time. It's vital for assessing the conditions of retina and monitoring the effects of treatments. baseline to day 3, week 1, 4, 16, 24, 36, 52
Primary 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.
baseline to day 3, week 1, 4, 16, 24, 36, 52
Primary 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. baseline to day 3, week 1, 4, 16, 24, 36, 52
Secondary Change of FST outcome The Full Stimulus Test (FST) is a test to assess the functional integrity of the entire visual pathway, from the retina to the visual cortex. It often used in studies involving retinal dystrophies or certain neuro-ophthalmological conditions, FST might be used to assess the efficacy of a treatment or intervention. baseline to day 3, week 1, 4, 16, 24, 36, 52
Secondary 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. baseline to day 3, week 1, 4, 16, 24, 36, 52
Secondary 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 baseline to day 3, week 1, 4, 16, 24, 36, 52
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