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

NCT ID: NCT06292650

Last Updated: 2025-12-04

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: EARLY_PHASE1
• Total Enrollment: 12 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-02-25
• Study Completion Date: 2028-12-25

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.

Conditions

Retinitis Pigmentosa

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

group 1

IVT administration of a single low dose ZM-02 injection

Group Type: EXPERIMENTAL

ZM-02-L

Intervention Type: DRUG

rAAV-PsCatCh2.0 intravitreal injection of low dose

group 2

IVT administration of a single high dose ZM-02 injection

Group Type: EXPERIMENTAL

ZM-02-H

Intervention Type: DRUG

rAAV-PsCatCh2.0 intravitreal injection of high dose

group 3

Sham IVT injection

Group Type: SHAM_COMPARATOR

ZM-02-S

Intervention Type: PROCEDURE

sham intravitreal injection of ZM-02 (not actual injection)

Interventions

ZM-02-L

rAAV-PsCatCh2.0 intravitreal injection of low dose

Intervention Type: DRUG

ZM-02-H

rAAV-PsCatCh2.0 intravitreal injection of high dose

Intervention Type: DRUG

ZM-02-S

sham intravitreal injection of ZM-02 (not actual injection)

Intervention Type: PROCEDURE

Other Intervention Names

rAAV-PsCatCh2.0e; rAAV-PsCatCh2.0; rAAV-PsCatCh2.0e; rAAV-PsCatCh2.0; Sham injection

Eligibility Criteria

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

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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Zhongmou Therapeutics

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Wenbin Wei, PhD

Role: PRINCIPAL_INVESTIGATOR

Beijing Tongren Hospital, CMU

Locations

Beijing Tongren Hospital of Capital Medical University

Beijing, Beijing Municipality, China

Site Status: RECRUITING

Countries

China

Central Contacts

Pei Cao

Role: CONTACT

zmt@simbaeye.com

+86 18707134160

Yin Shen

Role: CONTACT

Facility Contacts

Pei Cao

Role: primary

zmt@simbaeye.com

+86 18707134160

Yin Shen

Role: backup

Other Identifiers

MOON-01

Identifier Type: -

Identifier Source: org_study_id