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Study to Evaluate QR-110 in Subjects With Leber’s Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene

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Clinical Trial

NCT03140969 Completed
Study to Evaluate QR-110 in Subjects With Leber’s Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene

Study to Evaluate QR-110 in Subjects With Leber’s Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene.

Phase 1/2
Interventional

Disease

Disease type

Paediatric clinical trials

Orphan drug recognition

NA

Patient type

Adult

Children

Inclusion / Exclusion

Opening date

16/10/2017

Closing date

01/12/2019

Inclusion criteria :

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Exclusion criteria :

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Funding

industry

Members involved

Main investigators

Pr Bart LEROY

Representative

Belgium

Genetic Diagnostics (TWG6), Low Vision Daily Life and Patients Groups (TWG5), National Integration (TWG9), Research (TWG8), Retinal Rare Eye Diseases (WG1)
Working Group Leader
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Others investigators

Pr Elfride DE BAERE

Substitute

Belgium

Genetic Diagnostics (TWG6), Retinal Rare Eye Diseases (WG1)
Working Group Leader
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ERN EYE member investigating site

HCP : Principal investigators