Yükleniyor... / Loading...
Safety and Efficacy Study of Novel Gene Therapy ZM-02 for Retinitis Pigmentosa Patients
MOON adı verilen bu erken Faz 1 (Early Phase 1) çalışma, ZM-02'nin retinitis pigmentoza (retinitis pigmentosa, RP) hastalarında insanda ilk kez denendiği araştırmadır ve güvenliliği, tolere edilebilirliği ile etkililiği değerlendiriyor. Katılımcılara çalışma gözüne tek taraflı göz içi (intravitreal) enjeksiyon yapılıyor; düşük doz (ZM-02-L), yüksek doz (ZM-02-H) ve sahte enjeksiyon (ZM-02-S) grupları bulunuyor. Çalışmaya, klinik olarak retinal pigment dejenerasyonu tanısı almış ve çalışma gözünün görme keskinliği parmak sayma düzeyinden daha iyi olmayan hastalar alınıyor.
Yukarıdaki özet, resmî kaydın sade dile aktarılmış halidir. Orijinal İngilizce başlık referans için gösterilir.
Kriterler ClinicalTrials.gov kaydından orijinal İngilizce haliyle alınmıştır. Uygunluk kararını yalnızca deneyi yürüten ekip verebilir.
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 exclusion criteria will be excluded from the study: 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