Intravitreal KIO-301 for people with late-stage retinitis pigmentosa

A Phase II, Randomised, Controlled, Double Masked, Multiple Dose Study of the Safety, Tolerability and Efficacy of Intravitreal KIO-301 in Patients With Late-stage Retinitis Pigmentosa

This trial will test whether injections of KIO-301 into both eyes are safe and can improve vision or light perception in adults with late-stage retinitis pigmentosa.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 36 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Kiora Pharmaceuticals, Inc. (Industry-sponsored)
• Locations: 5 sites (Sydney, New South Wales and 4 other locations)
• Trial ID: NCT06628947 on ClinicalTrials.gov

What this trial studies

This Phase II, randomized interventional trial gives up to three bilateral intravitreal injections of KIO-301 (50 µg or 100 µg) or placebo every 6 weeks, with clinic visits every 3 weeks for safety, pharmacokinetics, and efficacy monitoring. Treatment and follow-up periods together cover about 24 weeks in the main study, with screening up to 45 days and total main-study participation up to ~30 weeks. Participants include late-stage RP patients with either no light perception or low vision by the Berkeley Rudimentary Vision Test. Placebo recipients may opt into a 24-week open-label extension after the main study completes.

Who should consider this trial

Good fit: Adults 18 or older with non-syndromic late-stage RP (no light perception OU or low vision OU per BRVT), willing to attend frequent visits and avoid other intravitreal medications, are the intended participants.

Not a fit: People who do not meet the BRVT vision criteria, have recent intravitreal treatments (other than corticosteroids), active ocular infection, or other exclusionary ocular conditions are unlikely to qualify or benefit.

Why it matters

Potential benefit: If successful, KIO-301 injections could restore some light perception or improve residual vision for people with late-stage RP who currently have little or no useful vision.

How similar studies have performed: Related intravitreal pharmacologic and photoswitch approaches have shown promising preclinical and early human signals, but meaningful clinical benefit in patients with no light perception remains largely unproven.

Eligibility criteria

Inclusion Criteria:

1. Be aged 18 years or older at the time of consent.
2. Provide informed consent prior to any study procedures, as stipulated by local laws, Ethics Committee (EC) and Regulatory Authority (RA) guidelines.
3. Be willing and able to follow all study instructions, attend all study visits, and complete all study assessments.
4. Have a clinical diagnosis of non-syndromic RP, with the exception of Usher's Syndrome Type II (USH2) which is allowed.
5. Have a visual acuity as per the Berkeley Rudimentary Vision Test (BRVT) at Screening of:

   * NLP OU confirmed by inability to see pen torch light at 25 cm OD, OS, and OU (assigned logMAR of 4.0).
   * LV OU limited to logMAR \> 1.6 and \< 4.0.
6. Other than intravitreal corticosteroids, participants must not receive intravitreal concomitant medications from Screening until end of study.
7. For Low Vision (LV) OU participants only: must pass at least one multi-luminance functional vision (MLFV) test at two successive light levels (between 1 and 500 lux), or at 1400 lux. Additionally, they must fail the same test at 0.125 and 0.35 lux.
8. Must agree to follow appropriate contraception requirements from Screening until 3 months after the last dose of IMP.

   * Participants assigned female at birth who are of child-bearing potential (OCBP) must agree to a pregnancy test at Screening and use an acceptable method of birth control including oral, transdermal, injectable, or implantable hormonal contraception, intrauterine device, abstinence from intercourse with partner assigned male at birth, or surgical sterilisation of partner assigned male at birth. Participants assigned female at birth are not OCBP if they have had a hysterectomy, bilateral oophorectomy, bilateral tubal ligation, or are post-menopausal by at least 12 months.
   * Participants assigned male at birth with a partner OCBP must be surgically sterile for at least 3 months prior to starting study drug, or ensure their partner uses contraception as outlined above, and must use a male condom. Participants assigned male at birth must not donate sperm from Screening until 3 months after the last dose of IMP.
   * Participants who have practiced true abstinence for at least 1 year due to usual and preferred lifestyle choice are exempt from contraceptive requirements. If a participant who is abstinent becomes sexually active, they must agree to use appropriate contraception as described above.

Exclusion Criteria:

1. Pregnant or breast-feeding, or plan to become pregnant during the study.
2. Have, in the investigator's opinion, evidence of material/substantial optic nerve disease.
3. Have a history of one or more retinal detachments.
4. Other than RP related macular pathologies, have in the investigator's opinion, clinically significant ocular disease (e.g., corneal oedema, uveitis, severe keratoconjunctivitis sicca), or clinically significant opacities of the media which might interfere with the study assessments, or the ability of the participant to complete the study.
5. Have a history of high myopia (\> 6 diopters).
6. Have uncontrolled severe glaucoma defined as intraocular pressure (IOP) of \> 26 mmHg when on 2 or more IOP lowering medications and cup disc ratio of ≥ 0.8, as diagnosed by an ophthalmologist.
7. Have had a previous intraocular surgery (with the exception of phacoemulsification cataract surgery and YAG capsulotomy more than 12 months prior to first study drug administration, which is allowed).
8. Have aphakia or a subluxed intraocular lens, or have evidence of zonular weakness that in the opinion of the investigator would result in light obfuscation.
9. Have a psychiatric condition that, in the investigator's opinion, precludes compliance with the protocol; past or present psychoses; past or present bipolar disorder; disorder requiring lithium; or within five years prior to screening, a history of suicide plan.
10. Have any clinically significant abnormality at screening determined by medical history, vital signs, clinical biochemistry, haematology, urinalysis, or a 12-lead electrocardiogram (ECG), as assessed by the investigator, which might interfere with the study assessments or the ability of the participant to complete the study.
11. Have any other medical condition or significant co-morbidities, or any finding during screening, which in the view of the Investigator is likely to interfere with the study or put the Participant at risk, confound study data, or interfere significantly with study participation.
12. Have clinical signs of active ocular or systemic infection and/or a temperature greater than 38.0°C at the time of screening. Study entry must be deferred at least 14 days from resolution.
13. Have participated in any investigational study within 30 days prior to screening, prior exposure to an investigational product within 5 elimination half-lives, or planned used of an investigational product or device during the study.
14. Have known or suspected hypersensitivity to any of the study drug excipients.
15. Are taking any medications that are known to be toxic to the retina or optic nerve.

Where this trial is running

Sydney, New South Wales and 4 other locations

• Save Sight Institute — Sydney, New South Wales, Australia (Recruiting)
• Queensland Eye Institute — Woolloongabba, Queensland, Australia (Recruiting)
• Royal Adelaide Hospital — Adelaide, South Australia, Australia (Recruiting)
• Cerulea Clinical Trials — East Melbourne, Victoria, Australia (Recruiting)
• Lions Eye Institute — Nedlands, Western Australia, Australia (Recruiting)

Study contacts

• Principal investigator: Doron Hickey, MBChB — The Centre for Eye Research Australia (CERA)
• Study coordinator: Claudia Gregorio-King

Conditions Retinitis Pigmentosa; Late-stage RP with No Light Perception; Late-stage RP with Low Vision