Yükleniyor... / Loading...
CNTF Implants for CNGB3 Achromatopsia
Bu çalışma, CNGB3 genindeki mutasyonlara bağlı akromatopsi (achromatopsia, tam renk körlüğü) olan hastalarda CNTF salgılayan NT-501 implantını incelemektedir. Çalışmaya, iki CNGB3 gen mutasyonu taşıyan, 18 yaş ve üzeri hastalar alınmıştır. Çalışma Faz 1/Faz 2 aşamasında yürütülmüş ve tamamlanmıştır.
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: To be eligible, the following inclusion criteria must be met, where applicable. * Participant must be 18 years of age or older. * Participant must carry two alleles for CNGB3 gene mutations and no cyclic nucleotide-gated channel alpha 3 (CNGA3) sequence variations as confirmed in a Clinical Laboratory Improvement Amendments (CLIA)-certified laboratory. * Participant must understand and sign the protocol informed consent. * Both female participants of childbearing potential and male participants able to father children must have (or have a partner who has) had a hysterectomy or vasectomy, be completely abstinent from intercourse, or must agree to use contraception during the first six months following implantation. Acceptable forms of contraception include:hormonal contraception (i.e., birth control pills, injected hormones, dermal patch or vaginal ring), intrauterine device, barrier methods (diaphragm, condom) with spermicide, or surgical sterilization (tubal ligation). Exclusion Criteria: A participant is not eligible if any of the following exclusion criteria are present. * Participant has a history of other ocular disease likely to contribute significantly to visual loss (e.g., optic neuropathy, glaucoma, uveitis, or other retinal disease). * Participant is judged by the investigator as not sufficiently healthy to safely undergo ophthalmic surgery. * Participant is on anticoagulant therapy that cannot be safely stopped peri-operatively at the implant procedure. Patients on warfarin will always be excluded. Patients on aspirin will be asked to stop the medication at least seven days prior to the surgery (when not contraindicated by the underlying medical condition). The stoppage period for other anticoagulant medications is based on the best clinical judgment of the investigator surgeon and is variable depending on the patient's medical condition and the type of medication. * Participant has had diagnosis or treatment of a malignancy (excluding non-melanoma skin cancer) within the previous five years. * Participant has received investigational treatment in another clinical study related to an ocular condition in the last six months. * Participant is pregnant, lactating or planning to become pregnant in the first six months following implantation. Study Eye Eligibility Criteria: The participant must have at least one eye meeting all inclusion criteria and none of the exclusion criteria listed below. Study Eye Inclusion Criteria: The study eye must have a best-corrected Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity letterscore of ≤ 53 (i.e., ≤ 20/100). The visual acuity from the first baseline visit (Baseline 1) will be used for eligibility determination in case of a change in visual acuity at the second baseline visit (Baseline 2). Study Eye Exclusion Criteria: * The study eye has a choroidal nevus or ocular neoplasm with potential risk for malignant transformation. * The study eye is judged by the investigator, based on history or examination findings, as high-risk for retinal detachment, vitreous hemorrhage, infection, or uveitis. * The study eye has lens, cornea, or other media opacities precluding adequate visualization and testing of the retina. * The study eye has undergone intraocular surgery within 12 months prior to enrollment. Study Eye Selection Criteria in Cases of Bilateral Disease: * As this is a genetic condition that usually affects both eyes to a similar degree, if both eyes of a participant meet the study eye eligibility criteria and have comparable visual acuity, the study eye will be selected at the investigator's medical judgment after consultation with the participant. * In case of an eye with lower visual acuity, that eye will be selected as the study eye.