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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

NCT03140969 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 11

Last updated 2024-10-15

Study results available
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Summary

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.

Conditions

  • Leber's Congenital Amaurosis

Interventions

DRUG

QR-110

RNA antisense oligonucleotide for intravitreal injection

Sponsors & Collaborators

  • Sepul Bio

    collaborator INDUSTRY

  • Laboratoires Thea

    lead INDUSTRY

Principal Investigators

  • Sepul Bio Chief Medical Officer · Sepul Bio

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
6 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-10-16
Primary Completion
2019-10-02
Completion
2019-10-02
FDA Drug
Yes

Countries

  • United States
  • Belgium

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03140969 on ClinicalTrials.gov