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The Safety and Efficacy of ZVS106e in the Treatment of IRDs Caused by Biallelic Mutations in ABCA4
Bu çalışma, ABCA4 genindeki iki alelli (biallelic) mutasyonların neden olduğu kalıtsal retina hastalıklarının (inherited retinal diseases) tedavisinde ZVS106e adlı ilacın güvenliliğini ve etkililiğini incelemektedir. Çalışmaya, genetik test ile ABCA4 iki alelli mutasyonlarına bağlı kalıtsal retina dejenerasyonu tanısı almış, 8 yaş ve üzeri hastalar alınmaktadır. Çalışma Erken Faz 1 aşamasında yürütülmektedir ve halen katılımcı almaktadır.
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: 1. Age ≥8 years old; 2. Patients diagnosed with hereditary retinal degeneration caused by ABCA4 biallelic mutations through genetic testing, and without other ophthalmic genetic diseases; 3. The target eye must meet the following requirements: the best corrected visual acuity is 0.5 to 2.0 LogMAR (including 0.5 and 2.0 LogMAR, equivalent to the decimal visual acuity index up to 0.3); 4. The subject and his or her spouse agree to take effective contraceptive measures during the trial period and for at least one year after administration. 5. Voluntarily participate in clinical trials and sign informed consent forms, and be able to complete all trial processes as required by the protocol. Exclusion Criteria: 1. The researchers determined that the target eye currently has or has previously had other macular lesions such as retinal schisis or epiretinal membrane. Or have other eye diseases that may hinder the surgery or interfere with the interpretation of the study endpoint; 2. Having received drug treatment that may affect the observation of the trial within the three months prior to screening; 3. The target eye has undergone the following intraocular surgeries: retinal repositioning and vitrectomy; 4. There are known eye/visual diseases, disorders or lesions that cause or are related to vision loss, or whose related treatments or therapies are known to cause or are related to vision loss; 5. Having suffered from a viral infectious disease that may affect the efficacy and safety evaluation of the investigational drug or having received an antiviral vaccine within one month prior to enrollment; 6. Systemic medications that are currently in use or may be required to cause eye toxicity, such as psoralen, risselinic acid or tamoxifen, etc. 7. Known to be allergic to the drugs planned to be used in the study; 8. Suffering from poorly drug-controlled hypertension: systolic blood pressure ≥160 mmHg or diastolic blood pressure ≥100 mmHg; 9. The following laboratory test abnormalities have clinical significance: Liver function: Chronic liver disease, elevation of ALT or AST \>2 times the upper limit of the normal value; Those with abnormal coagulation function (prothrombin time ≥ 3 seconds above the upper limit of the normal value, activated partial thromboplastin time ≥ 10 seconds above the upper limit of the normal value); Serum virological examination: Positive for active hepatitis B, hepatitis C virus antibody (HCV-Ab), human immunodeficiency virus antibody (HIV-Ab), or syphilis antibody; 10. There is any past or current medical history that may affect the safety of the trial or the in vivo process of the drug, especially a history of diseases such as cardiovascular, liver, kidney, endocrine, digestive tract, lung, nervous, hematological, tumor, immune or metabolic disorders that the investigators consider to have clinical significance; 11. Those who have participated in any clinical trials of drugs or medical devices within the three months prior to screening; 12. Pregnant or lactating women; According to the researcher's judgment, those who are deemed unsuitable to participate in this clinical trial for other reasons