Actively Recruiting
A Phase II Randomized, Controlled, Double Masked Study of the Safety, Tolerability, and Efficacy of Intravitreal KIO-301 in Patients With Late-stage Retinitis Pigmentosa
Led by Kiora Pharmaceuticals, Inc. · Updated on 2026-04-30
36
Participants Needed
5
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety, tolerability, and effectiveness of up to three doses of KIO-301 given by injection into both eyes every six weeks in patients with late-stage retinitis pigmentosa (RP). The study includes patients who have no light perception or low vision. This Phase II trial aims to better understand how KIO-301 works in this population. Participants will receive either 50 micrograms or 100 micrograms of KIO-301 or a placebo injected into both eyes once every six weeks for a total of three injections. The study includes a screening period of up to 45 days, followed by a treatment and follow-up period lasting 12 weeks each. Those who received placebo and opt to join the open-label extension may participate for an additional 24 weeks. During the study, participants will attend visits every three weeks for safety, tolerability, and effectiveness checks, including blood tests and eye exams. Researchers will monitor the effects from baseline through 12 weeks after the last injection, totaling up to 30 weeks for the main study. Those in the extension phase will have ongoing assessments, with total participation depending on timing between study phases.
CONDITIONS
Brief Title
A Phase II Study of Intravitreal KIO-301 in Patients With Late-stage Retinitis Pigmentosa
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Be aged 18 years or older at the time of consent.
- Provide informed consent before any study procedures.
- Willing and able to follow study instructions and attend all visits.
- Have a clinical diagnosis of non-syndromic retinitis pigmentosa, including Usher's Syndrome Type II.
- Have no light perception or low vision in both eyes as measured by the Berkeley Rudimentary Vision Test.
- No use of intravitreal corticosteroids or other intravitreal medications during the study.
- For low vision participants, must pass specific multi-luminance functional vision tests at set light levels.
- Agree to follow contraception requirements during the study and for 3 months after the last dose.
You will not qualify if you...
- Pregnant or breastfeeding, or planning pregnancy during the study.
- Evidence of significant optic nerve disease.
- History of one or more retinal detachments.
- Significant ocular diseases or opacities that might interfere with study assessments.
- History of high myopia greater than 6 diopters.
- Uncontrolled severe glaucoma with high intraocular pressure despite medication.
- Previous intraocular surgery except certain cataract surgeries over 12 months prior.
- Aphakia, subluxed intraocular lens, or zonular weakness causing light obstruction.
- Psychiatric conditions that interfere with study compliance.
- Clinically significant abnormalities in medical tests or history.
- Any other medical condition or findings that may interfere with study or participant safety.
- Active ocular or systemic infection or fever at screening.
- Participation in other investigational studies recently.
- Known hypersensitivity to study drug components.
- Use of medications toxic to the retina or optic nerve.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - Up to 6 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person) for screening assessments
Duration - 12 weeks
Participants receive intravitreal injections of KIO-301 or placebo in both eyes every 6 weeks for a total of 3 injections while attending visits for pharmacokinetics, safety, tolerability, and efficacy assessments.
Visits every 3 weeks during treatment (total 5 visits including dosing visits)
Duration - 12 weeks
Participants are monitored for safety, tolerability, and efficacy assessments after the last injection.
Visits every 3 weeks during follow-up (total 4 visits)
Duration - 24 weeks
Participants who received placebo and choose to continue may participate in an additional 24-week open label extension with further assessments.
Visit schedule depends on extension protocol
Trial Site Locations
Total: 5 locations
1
Save Sight Institute
Sydney, New South Wales, Australia, 2000
Actively Recruiting
2
Queensland Eye Institute
Woolloongabba, Queensland, Australia, 4102
Actively Recruiting
3
Royal Adelaide Hospital
Adelaide, South Australia, Australia, 5000
Actively Recruiting
4
Cerulea Clinical Trials
East Melbourne, Victoria, Australia, 3002
Actively Recruiting
5
Lions Eye Institute
Nedlands, Western Australia, Australia, 6009
Actively Recruiting
Research Team
C
Claudia Gregorio-King
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here