RecruitingEarly Phase 1NCT06292650

Safety and Efficacy Study of Novel Gene Therapy ZM-02 for Retinitis Pigmentosa Patients

Prospective, Dose-Escalating, Investigator Initiated Trial to Evaluate the Safety and Efficacy of ZM-02 in Retinitis Pigmentosa

Sponsor

Zhongmou Therapeutics

Enrollment

12 participants

Start Date

Feb 25, 2024

Study Type

INTERVENTIONAL

Conditions

Retinitis Pigmentosa

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.

Eligibility

Min Age: 18 YearsMax Age: 65 Years

Inclusion Criteria9

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
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

Exclusion Criteria14

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)
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