Leber Konjenital Amorozisin Tedavisinde AAV-RPE65 Vektörünün Güvenlik ve Etkinlik Çalışması (Faz 1/2)

Sade dilde özet

Bu çalışma, RPE65 geni mutasyonlarına bağlı Leber Konjenital Amoroz (Leber congenital amaurosis, doğuştan ağır görme kaybı) hastalarında bir gen terapisini (AAV taşıyıcı ile RPE65 geni aktarımı) inceliyor. RPE65 mutasyonu doğrulanmış, 6 yaş ve üzeri, görme ve retina testlerini yapabilen hastalar dahil edilmiştir. Faz 1/Faz 2 aşamasında yürütülmüş ve tamamlanmıştır.

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Deney bilgileri

Faz

PHASE1/PHASE2

Durum

Tamamlandı

Sponsor

Beacon Therapeutics

Müdahale

rAAV2-CB-hRPE65

Başlangıç

17.06.2009

Tahmini bitiş

22.09.2017

Ülkeler

United States

İlgili genler

Merkezler

• Worcester, United States
• Portland, United States

Uygunluk kriterleri

Kriterler ClinicalTrials.gov kaydından orijinal İngilizce haliyle alınmıştır. Uygunluk kararını yalnızca deneyi yürüten ekip verebilir.

Inclusion Criteria:

* Retinal disease consistent with a diagnosis of Leber congenital amaurosis and documented mutations in the RPE65 gene (including null mutations and mutations that code for abnormal RPE65 protein);
* At least 6 years of age;
* Good general health without significant physical examination findings or clinically significant abnormal laboratory results;
* Able to perform tests of visual and retinal function;
* Visual acuity not better than 20/60 and not worse than hand motion in both the treated eye and the fellow eye;
* Visible photoreceptor (outer nuclear) layer on a standard optical coherence tomography (OCT) scan;
* Acceptable hematology, clinical chemistry and urine laboratory parameters;
* For females of childbearing potential, a negative pregnancy test at screening and at baseline, and agreement to use effective contraception for 12 months after administration of rAAV2-CB-hRPE65, for sexual activity that could lead to pregnancy;
* For males of reproductive potential, agreement to use effective contraception for 12 months after administration of rAAV2-CB-hRPE65, for sexual activity that could lead to pregnancy

Exclusion Criteria:

* Pre-existing eye conditions that would preclude the planned surgery or interfere with interpretation of study endpoints or complications of surgery (e.g. glaucoma, corneal or lenticular opacities, or history or retinal detachment);
* Presence of epiretinal membrane on OCT;
* History of immunodeficiency or other medical conditions that might increase the risk of rAAV2-CB-hRPE65 administration;
* Use of anticoagulants or anti-platelet agents within 7 days prior to study agent administration;
* History of allergy or sensitivity to medications planned for use in the peri-operative period;
* For females of childbearing potential, a positive pregnancy test at screening or baseline (within 2 days before rAAV2-CB-hRPE65 administration);
* Females who are breast feeding;
* Use of any investigational agent, or systemic corticosteroids or other immunosuppressive drug(s), within 3 months prior to enrollment;
* Prior receipt of any AAV gene therapy product;
* Any condition which leads the investigator to believe that the participant cannot comply with the protocol requirements or that may place the participant at an unacceptable risk for participation.