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RNA Antisense Oligonucleotide

QR421a RNA antisense oligonucleotide for intravitreal injection for Retinitis Pigmentosa (HELIA Trial)

Phase 2
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
Subjects who have participated in a preceding QR-421a study and who may derive benefit from continued treatment with QR 421a, and/or continued follow up, as assessed by the Investigator, in consultation and agreement with the Medical Monitor An adult (≥ 18 years) willing and able to provide informed consent for participation prior to performing any study related procedures, and suitable verbal, auditory, written and/or tactile sign language communication as to allow informed consent to be obtained, in the opinion of the Investigator.
OR A minor (12 to < 18 years) able to provide age-appropriate assent for study participation, and with a parent or legal guardian willing and able to provide written permission for the subject's participation prior to performing any study related procedures.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 24 months
Awards & highlights

HELIA Trial Summary

This trial is studying an experimental drug called QR 421a to see if it is safe and effective in treating people with a certain type of genetic eye disease.

Eligible Conditions
  • Retinitis Pigmentosa
  • Usher Syndrome

HELIA Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

HELIA Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~24 months
This trial's timeline: 3 weeks for screening, Varies for treatment, 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
Non-ocular Adverse Events (AEs)
Ocular Adverse Events (AEs)
Secondary outcome measures
Best Corrected Visual Acuity (BCVA)
Ellipsoid Zone (EZ) Area/Width by Spectral Domain Optical Coherence Tomography (SD-OCT)
Exposure of QR-421a in Serum
+3 more

HELIA Trial Design

1Treatment groups
Experimental Treatment
Group I: QR421a RNA antisense oligonucleotide for intravitreal injectionExperimental Treatment1 Intervention
There is only one treatment arm in the PQ-421a-002 study; All participants that are eligible to be dosed will receive QR-421a in an open label fashion.

Find a Location

Who is running the clinical trial?

ProQR TherapeuticsLead Sponsor
11 Previous Clinical Trials
276 Total Patients Enrolled
4 Trials studying Retinitis Pigmentosa
117 Patients Enrolled for Retinitis Pigmentosa
Medical MonitorStudy DirectorProQR Therapeutics
1,649 Previous Clinical Trials
979,704 Total Patients Enrolled

Frequently Asked Questions

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

How many separate sites are overseeing this clinical investigation?

"At present, there are 5 sites across the country where this study is hosted. Patients can access these locations in Portland, Ann Arbor and Boston as well as five other cities. To reduce travel time for participants, it is beneficial to select a site closer to home during enrollment."

Answered by AI

Is there still opportunity for participants to join this trial?

"As per the details hosted on clinicaltrials.gov, this research trial is not taking applications currently. Initially posted in September 2021 and with its most recent update being made in October of last year, it has no open slots for participants at present; however, 53 other relevant studies are actively enrolling patients."

Answered by AI

Does this treatment have the endorsement of a regulatory body?

"Our team at Power assessed the treatment's safety to be a 2 as this is only Phase 2, indicating that some data exists in regards to its security but none for efficacy."

Answered by AI

What is the intended outcome of this clinical research project?

"ProQR Therapeutics' clinical trial will evaluate the safety of their drug by monitoring adverse events over a two year period. Alongside this primary objective, researchers plan to assess secondary outcomes such as changes in SD-OCT ellipsoid zone area/width, serum QR-421a concentration levels, and static perimetry scores from baseline values."

Answered by AI

Who else is applying?

What state do they live in?
Massachusetts
Idaho
Texas
How old are they?
18 - 65
What site did they apply to?
Center for Clinical Research Operations, Massachusetts Eye and Ear
Casey Eye Institute, Oregon Health & Science University
Retina Foundation of the Southwest
What portion of applicants met pre-screening criteria?
Met criteria
Recent research and studies
clinicaltrials.govStudy
An Open-Label Extension Study to Evaluate Safety & Tolerability of QR-421a in Subjects With Retinitis Pigmentosa
2021. "An Open-Label Extension Study to Evaluate Safety & Tolerability of QR-421a in Subjects With Retinitis Pigmentosa". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05085964.
~6 spots leftby Apr 2025
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