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A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)

NCT03913143 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 36

Last updated 2022-03-17

No results posted yet for this study

Summary

The purpose of this double-masked, randomized, controlled, multiple-dose study is to evaluate the efficacy, safety, tolerability and systemic exposure of sepofarsen (QR-110) administered via intravitreal injection in subjects with Leber's Congenital Amaurosis (LCA) due to the CEP290 p.Cys998X mutation after 24 months of treatment

Conditions

  • Leber Congenital Amaurosis 10
  • Blindness
  • Leber Congenital Amaurosis
  • Vision Disorders
  • Sensation Disorders
  • Neurologic Manifestations
  • Eye Diseases
  • Eye Diseases, Hereditary
  • Eye Disorders Congenital
  • Retinal Disease

Interventions

DRUG

sepofarsen

RNA antisense oligonucleotide for intravitreal injection

OTHER

Sham

Sham-Procedure (no experimental drug administered)

Sponsors & Collaborators

  • ProQR Therapeutics

    lead INDUSTRY

Principal Investigators

  • ProQR Medical Monitor · ProQR Therapeutics

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
8 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-04-04
Primary Completion
2022-01-31
Completion
2023-03-31
FDA Drug
Yes

Countries

  • United States
  • Belgium
  • Brazil
  • Canada
  • France
  • Germany
  • Italy
  • Netherlands
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases

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 NCT03913143 on ClinicalTrials.gov