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

NCT06292650 | Recruiting Early Phase 1

• 1 other identifier: MOON-01
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

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.

Conditions

Retinitis Pigmentosa

Keywords

gene therapy; AAV; RP; optogenetics

Outcome Measures

Primary Outcomes (2)

• 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, 12, 24, 36, 52

• 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, 12, 24, 36, 52

Secondary Outcomes (3)

• Change of MLMT level

  baseline to day 3, week 1, 4, 12, 24, 36, 52

• Change of Quality of Life

  baseline to day 3, week 1, 4, 12, 24, 36, 52

• Change in best corrected visual acuity (BCVA)

  baseline to day 3, week 1, 4, 12, 24, 36, 52

Other Outcomes (9)

• Change in color discrimination

  baseline to day 3, week 1, 4, 12, 24, 36, 52

• Change in visual field (VF)

  baseline to day 3, week 1, 4, 12, 24, 36, 52

• Change in Fundus Autofluorescence (FAF)

  baseline to day 3, week 1, 4, 12, 24, 36, 52

Study Arms (3)

group 1

EXPERIMENTAL

IVT administration of a single low dose ZM-02 injection

Drug: ZM-02-L

group 2

EXPERIMENTAL

IVT administration of a single high dose ZM-02 injection

Drug: ZM-02-H

group 3

SHAM COMPARATOR

Sham IVT injection

Procedure: ZM-02-S

Interventions

ZM-02-L DRUG

rAAV-PsCatCh2.0 intravitreal injection of low dose

Also known as: rAAV-PsCatCh2.0e, rAAV-PsCatCh2.0

group 1

ZM-02-H DRUG

rAAV-PsCatCh2.0 intravitreal injection of high dose

Also known as: rAAV-PsCatCh2.0e, rAAV-PsCatCh2.0

group 2

ZM-02-S PROCEDURE

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

Also known as: Sham injection

group 3

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Zhongmou Therapeutics: lead

Study Sites (1)

Beijing Tongren Hospital of Capital Medical University

Beijing, Beijing Municipality, China

RECRUITING

MeSH Terms

Conditions

Retinitis Pigmentosa

Interventions

salicylhydroxamic acid

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Retinal Dystrophies; Retinal Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Wenbin Wei, PhD

  Beijing Tongren Hospital, CMU

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Windy Zhou

CONTACT

+86 18986214263; zmt@zmtherapeutics.com

Yin Shen

CONTACT

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, CARE PROVIDER
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 27, 2024
• First Posted: March 5, 2024
• Study Start: February 25, 2024
• Primary Completion (Estimated): December 25, 2027
• Study Completion (Estimated): December 25, 2028
• Last Updated: March 31, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)