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An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene.

NCT04855045 · Status: UNKNOWN · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 15

Last updated 2022-03-25

No results posted yet for this study

Summary

PQ-110-005 (BRIGHTEN) is an open-label, dose escalation and double-masked, randomized, controlled study evaluating safety and tolerability of sepofarsen administered via intravitreal (IVT) injection in pediatric subjects (\<8 years of age) with LCA10 due to the c.2991+1655A\>G mutation over 24 months of treatment.

Conditions

  • Leber Congenital Amaurosis 10
  • Blindness
  • Leber Congenital Amaurosis
  • Vision Disorders
  • Sensation Disorders
  • Neurologic Manifestations
  • Eye Diseases
  • Eye Disorders Congenital
  • Retinal Disease
  • Retinal Degeneration
  • Retinal Dystrophies

Interventions

DRUG

sepofarsen

RNA antisense oligonucleotide for intravitreal injection

Sponsors & Collaborators

  • ProQR Therapeutics

    lead INDUSTRY

Principal Investigators

  • ProQR Medical Monitor · ProQR Therapeutics

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Model
SEQUENTIAL

Eligibility

Min Age
0 Years
Max Age
7 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-03-23
Primary Completion
2023-12-31
Completion
2023-12-31

Countries

  • Belgium
  • Brazil
  • Canada
  • 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 NCT04855045 on ClinicalTrials.gov