Gene editing treatment for Retinitis Pigmentosa

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This study evaluates the safety, tolerability, and efficacy of a gene editing drug called ZVS203e administered through a subretinal injection in participants with Retinitis Pigmentosa caused by a specific RHO gene mutation. It is a single-arm, open-label study that will enroll up to 9 participants, who will receive escalating doses of the drug. The study aims to measure the safety and effectiveness of the treatment, as well as the characteristics of the gene delivery vector.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* 1\. Patients with clinical diagnosis of Retinitis Pigmentosa (RP) (age ≥ 18 years) ;
* 2\. Genetic test confirmed to carry a fix mutation of RHO and carry no pathogenic mutations of other ophthalmic genetic diseases;
* 3\. Meet the following target eye selection criteria： Best corrected visual acuity between 2.3 LogMAR and 0.5 LogMAR (including 2.3 LogMAR and 0.5 LogMAR, equivalent to Snellen visual acuity of hand move to 20/63) ;
* 4\. Agree to take effective contraceptive measures from the beginning of the study to 1 year after the administration;
* 5.Willingness to adhere to protocol as evidenced by written informed consent;

  * Definition of the study eye: The study eye is defined as the eye with worse visual acuity between the two eyes of each prospective subject. For all subjects, only one eye shall be designated as the "study eye" (i.e., the eye receiving treatment). If both eyes of a patient meet the eligibility criteria and have equal visual acuity, the investigator shall determine the study eye based on clinical judgment.

Exclusion Criteria:

* 1\. Existing or pre-existing of macular lesions such as retinoschisis or macular membrane, or other eye conditions interfering with the surgery or the interpretation of the clinical endpoint, in the investigators' opinion;
* 2\. The study eye has been treated with other drugs within 3 months that could affect the evaluation of the investigational drug;
* 3\. The study eye has been treated with the following intraocular procedures: retinal detachment surgery, vitrectomy;
* 4\. The presence of an ocular/visual disease, disorder or lesion known to cause, or to be associated with, vision loss, or whose associated treatment or therapy is known to cause, or to be associated with, vision loss；
* 5\. Currently taking or may require systemic medications that can cause ocular toxicity, such as psoralen, risedronate, or tamoxifen;
* 6\. Known allergy to the drug planned for use in the study；
* 7.Those with the following laboratory abnormalities which are clinically significant: Liver function: chronic liver disease, ALT increased \>2 times the upper limit of normal; With uncontrolled hypertension, mean systolic blood pressure ≥ 160 mmHg or mean diastolic blood pressure ≥ 100 mmHg; With uncontrolled diabetes, HbA1c\>10%; Patients with abnormal coagulation function (prothrombin time ≥ upper limit of normal (3 seconds' longer), activated partial thromboplastin time ≥ upper limit of normal (10 seconds' longer)); Serum virology test: Active hepatitis B, hepatitis C virus antibody (HCV-Ab), human immunodeficiency virus antibody (HIV-Ab) or syphilis antibody positive;
* 8\. Having any past or present medical history that may affect the safety of the trial or the in vivo process of the drug, especially the medical history of cardiovascular, hepatic, renal, endocrine, gastrointestinal, pulmonary, neurological, hematological, oncologic, immunological or metabolic disorders and others that are thought clinically significant by the investigator；
* 9\. Participation in any medicine or medical device clinical trials within 3 months prior to enrollment;
* 10\. Neutralizing antibodies to rAAV\> 1:1000 by immunologic test;
* 11\. For females in pregnancy or lactation period;
* 12\. Any other conditions which leads the investigator to determine the participant is unsuitable for this study.

Where it's running

• Peking University Third Hospital
  Beijing, Beijing Municipality, China Recruiting

Study contacts

• Principal investigator: Liping Yang, MD — Peking University Third Hospital
• Study coordinator: Liping Yang, MD
• Email: alexlipingyang@bjmu.edu.cn
• Phone: 010-82266595

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Other studies for the same condition or treatment. Recruiting studies are listed first.

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• ZVS203e eye gene-editing therapy for a specific RHO-related retinitis pigmentosa

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• Safety of a one-time DSP-3077 retinal sheet injection in adults with retinitis pigmentosa

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.