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Antisense Oligonucleotide

sepofarsen for Leber Congenital Amaurosis (ILLUMINATE Trial)

Phase 2 & 3

Waitlist Available

Research Sponsored by ProQR Therapeutics

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 12 and 24 months

Awards & highlights

ILLUMINATE Trial Summary

This trial is studying whether a gene therapy called sepofarsen is effective and safe in treating patients with Leber's congenital amaurosis (LCA) who have the CEP290 p.Cys998X mutation.

Eligible Conditions

Leber Congenital Amaurosis
Eye Diseases
Congenital Eye Disorders
Blindness
Retinal Disease
Sensory Disorders
Neurologic Symptoms
Vision Impairment

ILLUMINATE Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 12 and 24 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~12 and 24 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 and 24 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Change in BCVA

Secondary outcome measures

Change from baseline in BCVA ≤ -0.3 LogMAR

Change in BCVA based on FrACT

Change in FAF

+13 more

ILLUMINATE Trial Design

3Treatment groups

Experimental Treatment

Active Control

Placebo Group

Group I: Group 1: Dose 1 sepofarsen (QR-110)Experimental Treatment1 Intervention

Initial loading dose, followed by maintenance doses at month 3 and every 6 months there after, administered by intravitreal injection (24 months duration of treatment). After 12 months treatment of the contralateral eye may be initiated

Group II: Group 2: Dose 2 sepofarsen (QR-110)Active Control1 Intervention

Group III: Group 3: ShamPlacebo Group1 Intervention

Sham procedure (no experimental drug administered), Day 1, month 3 and every six months there after. After 12 months cross over to active study drug may be initiated

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

ProQR TherapeuticsLead Sponsor

11 Previous Clinical Trials

261 Total Patients Enrolled

3 Trials studying Leber Congenital Amaurosis

35 Patients Enrolled for Leber Congenital Amaurosis

ProQR Medical MonitorStudy DirectorProQR Therapeutics

7 Previous Clinical Trials

141 Total Patients Enrolled

2 Trials studying Leber Congenital Amaurosis

24 Patients Enrolled for Leber Congenital Amaurosis

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is this a new clinical trial?

"At this moment in time, there are three ongoing clinical trials for sepofarsen being conducted in 11 cities and 9 countries. The first trial happened in 2019 and was completed successfully by 36 participants. Since then, 114 more trials have been carried out."

Answered by AI

How many people are able to join this clinical study?

"This study is no longer recruiting patients. However, as of 3/2/2022, there were 1240 other clinical trials for retinal diseases and 3 studies involving sepofarsen that were still enrolling patients."

Answered by AI

Does a body of research exist for sepofarsen?

"There are 3 ongoing studies for sepofarsen, 2 of which are in Phase 3. The focus of the majority of these studies is in Strasbourg and Iowa; however, there are a total of 32 research sites."

Answered by AI

Are patients being actively recruited for this experiment right now?

"The clinical trial mentioned is not currently recruiting patients. This can be seen by the most recent update on clinicaltrials.gov being from 3/2/2022. Although this particular study isn't looking for candidates, there are 1243 other trials that are actively recruiting patients."

Answered by AI

In how many different medical institutions is this research project being conducted presently?

"This study is looking for participants from 8 different centres, which are located in Toronto, Miami, Houston and other cities. To limit travel as much as possible, patients should try to enroll at the site closest to them."

Answered by AI

What are the proposed benefits of this clinical trial?

"The primary outcome measure for this study, change in BCVA, will be assessed over the course of a year. Additionally, ProQR Therapeutics plans to evaluate secondary outcomes including changes in ophthalmic examination findings, frequency of non-ocular AEs, and change in mobility course score."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

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

2019. "A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03913143.