Safety and Efficacy Study of Novel Gene Therapy ZM-02 for Retinitis Pigmentosa Patients
ConditionRetinitis Pigmentosa
Trial statusRecruiting
Trial phaseEarly Phase 1
Trial typeInterventional
Biological sexAll
Age18-65
SponsorZhongmou Therapeutics
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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
Disqualifiers
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
Trial design
Design model
Parallel
Treatments tested in this trial
ZM-02-L
DrugrAAV-PsCatCh2.0 intravitreal injection of low dose
ZM-02-H
DrugrAAV-PsCatCh2.0 intravitreal injection of high dose
ZM-02-S
Procedure/Surgerysham intravitreal injection of ZM-02 (not actual injection)
Treatment groups
12 Participants
are divided into 3 treatment groupsGroup A: group 1Experimental treatment 1 intervention
Group B: group 2Experimental treatment 1 intervention
Group C: group 3Sham comparator 1 intervention
Trial outcomes
Primary outcomes
1
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.
Time frame
baseline to day 3, week 1, 4, 12, 24, 36, 522
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.
Time frame
baseline to day 3, week 1, 4, 12, 24, 36, 52Secondary outcomes
1
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.
Time frame
baseline to day 3, week 1, 4, 12, 24, 36, 522
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
Time frame
baseline to day 3, week 1, 4, 12, 24, 36, 523
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.
Time frame
baseline to day 3, week 1, 4, 12, 24, 36, 52Other outcomes
1
Change in color discrimination
Color discrimination testing measures an individual's ability to perceive and differentiate hues across the visible spectrum under standard luminance level. Instead of relying on fine spatial detail, these tests challenge the visual system to detect subtle differences in wavelength-an ability that is often impaired in advanced retinal disease.
Time frame
baseline to day 3, week 1, 4, 12, 24, 36, 522
Change in visual field (VF)
Visual field will be assessed by Humphrey perimetry, changes in VFI, MD, PSD will be analyzed.
Time frame
baseline to day 3, week 1, 4, 12, 24, 36, 523
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.
Time frame
baseline to day 3, week 1, 4, 12, 24, 36, 524
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.
Time frame
baseline to day 3, week 1, 4, 12, 24, 36, 52Sponsors and contacts
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Source ClinicalTrials.gov