Actively Recruiting
A Phase II Study of Intravitreal KIO-301 Injections for Safety and Tolerability in Patients with Late-stage Retinitis Pigmentosa
Led by Kiora Pharmaceuticals, Inc. · Updated on 2026-04-30
36
Participants Needed
5
Research Sites
100 weeks
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 injections into both eyes every six weeks in adults with late-stage retinitis pigmentosa (RP). This study includes patients with no light perception or low vision and is a Phase II, controlled study designed to better understand how this treatment may work in advanced RP cases. Participants diagnosed with non-syndromic RP, including Usher's Syndrome Type II, are eligible to join. Participants will receive either 50 micrograms or 100 micrograms of KIO-301, or a placebo consisting of sterile saline or balanced salt solution, administered by intravitreal injection. The treatment period lasts 12 weeks with injections every six weeks, followed by a 12-week follow-up for safety and efficacy evaluations. Those who received placebo may opt to join an open-label extension lasting an additional 24 weeks for further observation. During the study, participants will attend visits every three weeks for pharmacokinetic, safety, tolerability, and efficacy assessments. The total main study duration can be up to 30 weeks, including a screening period of up to 45 days. Researchers will monitor vision using specialized tests, track side effects, and analyze the body's response to the drug. Safety and tolerability will be closely followed from baseline through 12 weeks after the last injection.
CONDITIONS
Official 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.
- Be willing and able to attend all study visits and complete assessments.
- Have a clinical diagnosis of non-syndromic retinitis pigmentosa, including Usher's Syndrome Type II.
- Have vision classified as no light perception or low vision in both eyes as per study vision tests.
- Not receive intravitreal medications other than corticosteroids from screening until study end.
- For low vision participants, pass specified multi-luminance functional vision tests at certain light levels.
- Agree to follow contraception requirements from screening until three months after last dose of study drug, with specific conditions based on sex and reproductive potential.
You will not qualify if you...
- Pregnant, breastfeeding, or planning pregnancy during the study.
- Evidence of significant optic nerve disease.
- History of one or more retinal detachments.
- Have other significant eye diseases or opacities that interfere with study assessments.
- History of high myopia greater than 6 diopters.
- Have uncontrolled severe glaucoma despite medication.
- Previous eye surgery except certain cataract surgeries more than 12 months prior.
- Aphakia, subluxed intraocular lens, or zonular weakness causing light obstruction.
- Psychiatric conditions that affect study compliance or recent history of severe mental health issues.
- Significant abnormal medical findings that interfere with participation.
- Active infections or fever at screening.
- Recent participation in other investigational studies or use of investigational products.
- Known allergies to study drug components.
- Taking medications toxic to the retina or optic nerve.
AI-Screening
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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
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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
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