Retina Altına (rAAV2-VMD2-hMERTK) Enjeksiyonu Çalışması

Sade dilde özet

Bu çalışma, MERTK geniyle ilişkili retinitis pigmentoza (RP) hastalarında rAAV2-VMD2-hMERTK gen aktarımının retina altına (subretinal) uygulanmasının güvenliğini değerlendirmiştir. Faz 1 (Phase 1) çalışmaya, MERTK ile ilişkili retina hastalığı olan, daha kötü gören gözünde görme keskinliği 20/100 veya daha düşük olan, görme ve retina işlev testlerini yapabilecek ve genel sağlık durumu iyi katılımcılar alınmıştır.

Yukarıdaki özet, resmî kaydın sade dile aktarılmış halidir. Orijinal İngilizce başlık referans için gösterilir.

Deney bilgileri

Faz

Faz 1 (Phase 1)

Durum

Tamamlandı

Sponsor

King Khaled Eye Specialist Hospital

Müdahale

Subretinal administration of rAAV2-VMD2-hMERTKRecombinant Adeno-Associated Virus

Başlangıç

01.08.2011

Tahmini bitiş

01.08.2019

Ülkeler

Saudi Arabia

İlgili genler

Merkezler

• Riyadh, Saudi Arabia

Uygunluk kriterleri

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

Inclusion Criteria:

* MERTK-associated retinal disease;
* VA: 20/100 or less in worse eye
* Ability to perform tests of visual and retinal function;
* Good general health based on a complete physical examination and hematology and chemistry studies performed at a pre-treatment evaluation;
* Ability to comply with research procedures;

Exclusion Criteria:

* Pre-existing eye conditions that would preclude the planned surgery or interfere with the interpretation of study endpoints or surgical complications (for example, glaucoma, corneal or lenticular opacities);
* Complicating systemic diseases (such as medical conditions causing immunosuppression) that would preclude the gene transfer, ocular surgery or known sensitivity or allergy to medications planned for use in the peri-operative period;
* Use of anti-platelet agents that may alter coagulation within 7 days prior to study agent administration;
* Use of immunosuppressive medications;
* Pregnancy or breastfeeding;
* Individuals (males and females) of childbearing potential who are unwilling to use effective contraception for 1 year following agent administration and barrier contraception for 3 months following agent administration;
* Any other condition that would prevent a subject from completing follow-up examinations during the course of the study and that, in the opinion of the investigator, makes the subject unsuitable for the study.
* Current, or recent (within the past 30 days, or 10 half lives of the drug) participation, in any other research protocol involving investigational agents or therapies.
* Recent (within past 6 months) receipt of an investigational biologic therapeutic agent.Subjects will not be excluded based on their gender, race or ethnicity.