Stargardt Hastalığı Olan Bireylerde Tek Doz Göz İçi vMCO-010 Optogenetik Tedavisinin Güvenliliği ve Etkileri

Sade dilde özet

Bu çalışma, Stargardt hastalığı (Stargardt disease) adı verilen kalıtsal bir göz hastalığı olan bireylerde, tek doz göz içine (intravitreal) uygulanan vMCO-010 adlı optogenetik gen terapisinin güvenliliğini ve etkilerini incelemektedir. Çalışmaya, klinik ya da genetik olarak Stargardt tanısı konmuş (ABCA4, ELOVL4 veya PROM1 genlerinde patojenik varyant taşıyan), 16 yaş ve üzeri, görme düzeyi belirli aralıkta olan bireyler katılabilmiştir. Çalışma Faz 2 aşamasında yürütülmüş ve tamamlanmıştır.

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

Deney bilgileri

Faz

Faz 2 (Phase 2)

Durum

Tamamlandı

Sponsor

Nanoscope Therapeutics Inc.

Müdahale

Gene Therapy-vMCO-010

Başlangıç

05.07.2022

Tahmini bitiş

28.09.2023

Ülkeler

United States

İlgili genler

Merkezler

• Miami, United States
• McAllen, United States

Uygunluk kriterleri

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

Inclusion Criteria:

1. ≥16 years of age
2. Able to comprehend and give informed consent.
3. Able to comply with testing and all protocol tests.
4. Documented clinical diagnosis of Stargardt disease (classic fleck phenotype and/or well-demarcated sub-foveal area of significantly reduced autofluorescence as imaged by FAF), or genetic diagnosis with pathogenic variants in ABCA4, ELOVL4, or PROM1
5. In the study eye: ETDRS BCVA in range of 1.3 logMAR (Approximate Snellen equivalent: 20/400) to 1.9 logMAR (Snellen equivalent: 20/1600), and ETDRS BCVA no better than 20/200 in the fellow eye.
6. Presence of retinal inner nuclear and nerve fiber layers on optical coherence tomography (OCT) testing in the study eye at screening

Exclusion Criteria:

1. Presence of any concurrent ocular disease that would affect study outcomes (e.g., severe cataracts; subjects can be enrolled 3 months after successful cataract surgery).
2. Received any of the following treatments: gene therapy, stem cell therapy, surgical implantation of prosthetic retinal chips (such as ARGUS-II) or sub-retinal injections.
3. Has taken non-approved items (supplement containing vitamin A or beta-carotene, liver-based products, or prescription oral retinoid medications) over the past 30 days
4. Participation in an interventional study of a vitamin A derivative ≤ 3 months prior to screening
5. Presence of significant cardiovascular or cerebrovascular disease, including stroke within 12 months of entry.
6. Resting heart rate outside specified limits upon repeated measurement.
7. History of uncontrolled diabetes, hepatitis, pancreatitis, cirrhosis, liver failure, uncontrolled thyroid disease or hypervitaminosis A.
8. Any intraocular surgery or thermal laser within 3 months of trial entry or any prior thermal laser in the macular region.
9. Any major surgical procedure within one month of trial entry or anticipated during the trial.
10. Clinically significant abnormal lab results at screening
11. Known serious allergies to the fluorescein dye used in angiography or intraocular pressure measurement, povidone iodine, or to the components of the vMCO-010 formulation
12. In the Investigator's opinion, any severe acute or chronic medical condition, psychiatric condition, physical examination finding or laboratory abnormality
13. Pre-existing conditions in the study eye such as glaucoma, diseases affecting the optic nerve causing significant visual field loss, history of uveitis, corneal or lenticular opacities).
14. Presence of any complicating systemic diseases such as malignancies whose treatment could affect central nervous system function..
15. Subjects who are positive for syphilis, hepatitis B, C, and human immunodeficiency virus (HIV) will be excluded..
16. Presence of narrow iridocorneal angles contraindicating pupillary dilation in the study eye.
17. Presence of disorders of the ocular media in the study eye which could interfere with visual acuity and other ocular assessments, including OCT, during the study period.
18. Presence of macular hole in the study eye, evident by ophthalmoscopy and/or by OCT examinations
19. Current evidence of retinal detachment in the study eye assessed by the Investigator that significantly affects central vision.
20. Current use of hydroxychloroquine, chloroquine, or any related retina-toxic compounds.
21. Active ocular inflammation or recurrent history of idiopathic or autoimmune associated uveitis.