A phase 2/3 to evaluate safety and efficacy of 421a in RP patients
Research type
Research Study
Full title
A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and Tolerability of QR-421a in Subjects with Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene with Advanced Vision Loss (Sirius)
IRAS ID
305425
Contact name
Susan Downes
Contact email
Sponsor organisation
ProQR Therapeutics IV B.V.
Eudract number
2021-002729-74
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 10 months, days
Research summary
Research Summary
This is a study designed to study the effectiveness, safety and tolerability of QR-421a in different dose levels in the participants with advanced vision loss due to mutations in exon 13 of USH2A gene. QR-421a is designed to specifically target mutations in exon 13 of USH2A gene which cause the Retinitis Pigmentosa (RP) and to overcome this genetic defect in the DNA and to restore this important protein, which may stop some effects of RP, or possibly reverse some effects. There are currently no approved therapies for the treatment of USH2A associated to RP. This study will compare the effect of QR-421a with the effects of sham procedure. The study drug will be injected into the participant's eye (Intravitreal (IVT) injection). Approximately 81 participants are planned to be recruited for this study worldwide. Participants will be 12 years or older. Study period is anticipated to be approximately 34 months and duration of the patient participation will be approximately 27 months (up to 12 weeks of screening and 24 months treatment).
Summary of Results
Who participated in the study?
Subjects with impaired vision due to a particular type of Retinitis Pigmentosa (RP) caused by one or more mutations (defects) in their DNA participated in this study.
What treatments or interventions did the participants take/receive?
The study investigated whether the study drug, ultevursen (QR-421a), could slow or stop the worsening of vision loss and whether the study drug is safe. The study drug was given in 2 different doses and was compared against a sham (dummy) procedure. The study drug was given through an injection in the eye.
What medical problems (adverse reactions) did the participants have?
None of the subjects who participated in the study had any serious health issues or side effects. No subjects stopped the study because of any changes in their medical condition or due to side effects. The majority of subjects reported mild side effects that were caused by the way the study drug was administered (i.e., injection in the eye). The subjects who received the dummy procedure did not report any side effects in the eye that was treated.
What happened during the study?
The study was stopped early by the Sponsor. The decision to stop the study was not due to any changes in the medical condition or health of the subjects who participated, or for any other safety issue. In total 7 subjects joined the study; 5 received the study drug and 2 received the dummy treatment.
What were the results of the study?
The study was stopped early and well before the last visit for any subject. Due to this reason, the effects of the study drug on the vision of subjects with the particular type of RP could not be assessed.
How has this study helped subjects and researchers?
Since the study was stopped early, limited information is available on the effect and safety of the study drug. More research will be needed to know if treatment with the study drug will be safe and may potentially slow down or stop the worsening of vision loss in subjects with a specific form of RP.
What are the details surrounding further research?
At present there is no future research planned. Subjects that participated in the study will be offered an opportunity to continue treatment in a post-trial access program, if allowed in their country, and if in their best interest.
Where can I learn more about this study?
You can find out more information on https://eur03.safelinks.protection.outlook.com/?url=http%3A%2F%2Fwww.clinicaltrials.gov%2F&data=05%7C01%7Capprovals%40hra.nhs.uk%7C6cdb3496b00d4d4eed7d08dacbc78097%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C638046354958099800%7CUnknown%7CTWFpbGZsb3d8eyJWIjoiMC4wLjAwMDAiLCJQIjoiV2luMzIiLCJBTiI6Ik1haWwiLCJXVCI6Mn0%3D%7C3000%7C%7C%7C&sdata=69rCm6tlqd0RqFmo5RtT9uIo%2BPqdgLMNMjUmHqMh0AA%3D&reserved=0 and search on study number: NCT05158296.REC name
East of England - Cambridge South Research Ethics Committee
REC reference
21/EE/0277
Date of REC Opinion
9 Feb 2022
REC opinion
Further Information Favourable Opinion