Retinitis Pigmentosa
Conditions
Keywords
gene therapy, AAV, RP, optogenetics
Brief 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.
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.
Interventions
DRUGZM-02-L
rAAV-PsCatCh2.0 intravitreal injection of low dose
DRUGZM-02-H
rAAV-PsCatCh2.0 intravitreal injection of high dose
PROCEDUREZM-02-S
sham intravitreal injection of ZM-02 (not actual injection)
Sponsors
Zhongmou Therapeutics
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Caregiver)
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
Patients who meet all of the following criteria can be selected as subjects: 1. Clinically diagnosed with retinal pigment degeneration 2. 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 3. The subject has had visual experience above the finger counting 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of adverse events and serious adverse events | 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 | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change of MLMT level | baseline to day 3, week 1, 4, 12, 24, 36, 52 | 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. |
| Change of Quality of Life | baseline to day 3, week 1, 4, 12, 24, 36, 52 | Quality of Life will be measured using Visual Function Questionnaire (VFQ-25) or other similar questionnaires before and after treatment |
| Change in best corrected visual acuity (BCVA) | baseline to day 3, week 1, 4, 12, 24, 36, 52 | 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. |
Countries
China
Contacts
CONTACTWindy Zhou
CONTACTYin Shen
PRINCIPAL_INVESTIGATORWenbin Wei, PhD
Beijing Tongren Hospital, CMU
Outcome results
None listed