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

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

11 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-10-16

Study Completion Date

2019-10-02

Brief Summary

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The purpose of this study is to evaluate the safety and tolerability of QR-110 administered via intravitreal injection in subjects with LCA due to the CEP290 p.Cys998X mutation.

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Detailed Description

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The purpose of this study is to evaluate the safety and tolerability of QR-110 administered via intravitreal injection in subjects with LCA due to the CEP290 p.Cys998X mutation. Subjects will receive QR-110 in one eye every 3 months, for a maximum of 4 doses. Up to 3 dose levels of QR-110 will be evaluated.

Conditions

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Leber's Congenital Amaurosis

Study Design

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Allocation Method

NA

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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QR-110

Administered every 3 months

Group Type EXPERIMENTAL

QR-110

Intervention Type DRUG

RNA antisense oligonucleotide for intravitreal injection

Interventions

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QR-110

RNA antisense oligonucleotide for intravitreal injection

Intervention Type DRUG

Other Intervention Names

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Sepofarsen

Eligibility Criteria

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Inclusion Criteria

* Male or female, ≥ 6 years of age at Screening with a clinical diagnosis of LCA and a molecular diagnosis of homozygosity or compound heterozygosity for the CEP290 p.Cys998X mutation.
* Best-corrected visual acuity greater than or equal to light perception in both eyes and equal to or worse than LogMAR +1.0 (Snellen notation 20/200) in the worse eye and equal to or worse than LogMAR +0.7 (Snellen notation 20/100) in the contralateral eye.
* Detectable outer nuclear layer (ONL) in the area of the macula.
* An electroretinogram (ERG) result consistent with LCA.
* Clear ocular media and adequate pupillary dilation to permit good quality retinal imaging.

Exclusion Criteria

* Syndromic disease.
* Pregnant or breast-feeding female.
* Any clinically significant cardiac disease or defect.
* One or more coagulation parameters outside of the normal range.
* Any ocular disease or condition that could compromise treatment safety, visual acuity or interfere with assessment of efficacy and safety.
* Prior receipt of intraocular surgery or intravitreal injection within 3 months prior to study start or planned intraocular surgery or procedure during the course of the study.
* Use of any investigational drug or device within 90 days or 5 half-lives of Day 1, whichever is longer, or plans to participate in another study of a drug or device during the PQ-110-001 study period.
* Any prior receipt of genetic therapy for LCA

Minimum Eligible Age

6 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No