Koroideremi İçin BIIB111 Uygulanan Retinal Gen Terapisinin Güvenlilik Çalışması

Sade dilde özet

Bu çalışma, koroideremi (choroideremia) adı verilen kalıtsal göz hastalığı olan hastalarda her iki göze uygulanan BIIB111 adlı retinal gen terapisinin güvenliliğini incelemektedir. Çalışmaya, genetik olarak doğrulanmış CHM tanısı bulunan ve belirli görme düzeyine sahip hastalar katılmıştır. 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

Biogen

Müdahale

BIIB111

Başlangıç

29.11.2017

Tahmini bitiş

29.06.2022

Ülkeler

United States, France, Germany

İlgili genler

Merkezler

• Miami, United States
• Boston, United States
• Cincinnati, United States
• Portland, United States
• Paris, France
• Tübingen, Germany

Uygunluk kriterleri

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

Key Inclusion Criteria:

1. Are willing and able to give informed consent for participation in the study to have both eyes treated.
2. Have documentation of a genetically-confirmed diagnosis of CHM.
3. Have active disease clinically visible within the macular region of both eyes.
4. Have a BCVA of ≥34 ETDRS letters (20/200 or better Snellen acuity) in both eyes, or in the untreated eye, if the other eye was previously treated with BIIB111\*

   \*If previously treated with BIIB111 in an antecedent study, participants may be eligible for participation following Sponsor approval.
5. For participants who received treatment with BIIB111 in an antecedent study, have biological samples available to complete an adequate immunology profile.

Key Exclusion Criteria:

1. Have a history of amblyopia or inflammatory disorder in either eye.
2. Are unwilling to use barrier contraception methods or abstain from sexual intercourse for a period of 3 months following treatment with BIIB111 in either eye.
3. Have had previous intraocular surgery performed within 3 months of the Screening Visit in either eye.
4. Have any other significant ocular or non-ocular disease/disorder which, in the opinion of the investigator, may either put the participants at risk because of participation in the study, or may influence the results of the study or the participant's ability to participate in the study. This includes but is not limited to a potential participants:

   * with a contraindication to oral corticosteroid (e.g., prednisolone/prednisone)
   * with clinically significant cataract in either eye
   * who, in the clinical opinion of the Investigator, is not an appropriate candidate for sub-retinal surgery.
5. Have participated in another research study involving an investigational product in the past 12 weeks or received a gene/cell-based therapy at any time previously, except if treated within an antecedent study with BIIB111.

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply