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Updated May 2026 · ClinicalTrials.gov

RECRUITINGPhase 2INTERVENTIONAL

A Phase II Study of Intravitreal KIO-301 in Patients 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

A Phase II Study of Intravitreal KIO-301 in Patients With Late-stage Retinitis Pigmentosa (NCT06628947) is a Phase 2 interventional studying Retinitis Pigmentosa, sponsored by Kiora Pharmaceuticals, Inc.. RECRUITING as of the most recent ClinicalTrials.gov update. Talk to your doctor before contacting the trial site.

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

The goal of the study is to investigate the safety, tolerability and efficacy of up to 3 doses of KIO-301 administered by intravitreal (IVT) injection bilaterally every 6 weeks in patients with late-stage retinitis pigmentosa (RP). Late-stage RP patients will include those patients with No Light Perception (NLP), or Low Vision (LV).

What Stage of Research Is This?

Phase 2 trials evaluate whether a treatment actually works against Retinitis Pigmentosa and continue monitoring side effects. Phase 2 enrolls larger groups (typically 100–300 patients) and produces the first real efficacy signal. A successful Phase 2 readout is what unlocks the much larger Phase 3 confirmatory trials needed for FDA approval.

This trial is currently recruiting participants. The sponsor has registered the study with ClinicalTrials.gov as actively enrolling, which means new applicants who meet the eligibility criteria can be considered for screening. Trial status can change between updates — confirm current recruiting status with the study contact before traveling for a screening visit.

With a target enrollment of 36 participants, this is a small study — typical of early-phase research, rare-disease trials, or pilot studies designed to generate preliminary signal before a larger study is launched.

Who May Be Eligible (Plain English)

Who May Qualify: 1. Be aged 18 years or older at the time of consent. 2. Provide willing to sign a consent form 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. ...See full criteria on ClinicalTrials.gov Always talk to your doctor about whether this trial is right for you.

These are translations of the protocol\'s inclusion and exclusion criteria, simplified for patients and caregivers. The original clinical text appears below. Eligibility is ultimately confirmed by the trial site\'s screening process — this summary is a starting point for a conversation with your doctor, not a final determination.

Original Eligibility Criteria

View original clinical language
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.

Treatments Being Tested

OTHER

Placebo (Sterile Saline or Balanced Salt Solution)

A control 50 μl injection of clear sterile saline or balanced salt solution (BSS) liquid.

DRUG

100 μg KIO-301

KIO-301 drug product is an ophthalmic formulation of the drug substance KIO-300-Cl in sulfobutylether-β-cyclodextrin, sucrose, phosphate buffer salts and water suitable for IVT injection. The drug substance KIO-300-Cl is a quaternary ammonium chloride salt of the active compound KIO-300.

DRUG

50 μg KIO-301

KIO-301 drug product is an ophthalmic formulation of the drug substance KIO-300-Cl in sulfobutylether-β-cyclodextrin, sucrose, phosphate buffer salts and water suitable for IVT injection. The drug substance KIO-300-Cl is a quaternary ammonium chloride salt of the active compound KIO-300.

Locations (5)

Trial sites listed on ClinicalTrials.gov for this study. Site activation status can vary — confirm with the specific site before traveling for a screening visit.

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

How to Talk to Your Doctor About This Trial

Bring the printable summary of this trial — including the NCT ID (NCT06628947), the sponsor (Kiora Pharmaceuticals, Inc.), and the key eligibility criteria — to your next appointment. Your doctor can review the inclusion and exclusion criteria against your medical history, lab values, and current treatments to assess whether you are likely to qualify. They can also help you weigh whether trial participation makes sense alongside your existing care plan.

Useful questions to walk through together: What does the trial protocol require beyond standard care? How long is the active treatment phase, and how long is follow-up? Are there study visits at sites I can reach? Who pays for the trial-specific procedures, and who pays for standard-of-care portions? See our 25 questions to ask about clinical trials guide for a more complete checklist.

Authoritative Sources

The official record for this trial lives on ClinicalTrials.gov — the federal registry maintained by the National Library of Medicine at NIH. For background on how this trial fits into the FDA approval pathway, see the FDA drug approval process. For oncology-specific guidance for patients considering trials, the National Cancer Institute publishes patient-oriented overviews. International trial registries are aggregated by the WHO ICTRP.

Frequently Asked Questions

What is the NCT06628947 clinical trial studying?

The goal of the study is to investigate the safety, tolerability and efficacy of up to 3 doses of KIO-301 administered by intravitreal (IVT) injection bilaterally every 6 weeks in patients with late-stage retinitis pigmentosa (RP). Late-stage RP patients will include those patients with No Light Perception (NLP), or Low Vision (LV). The full protocol is registered on ClinicalTrials.gov and includes the primary outcome measures, eligibility criteria, and study endpoints.

Who can participate in NCT06628947?

Eligibility for this trial depends on the specific inclusion and exclusion criteria set by the sponsor. The plain-English summary above translates the most important criteria into accessible language; the official clinical text is preserved in the collapsible section underneath. Whether you fit any specific trial is a medical decision your doctor needs to confirm — bring the trial information to your treating physician for a full review against your medical history.

How do I contact the trial site for NCT06628947?

Contact information registered with ClinicalTrials.gov is shown in the sidebar of this page. Before reaching out, confirm with your treating physician that this trial is appropriate for your situation. The trial site will then walk you through the screening process to determine final eligibility.

Is participating in a clinical trial safe?

Clinical trials in the United States are regulated by the FDA and overseen by Institutional Review Boards (IRBs) that review the protocol for safety. Risk varies by trial — Phase 1 studies test new treatments in humans for the first time, while Phase 3 trials use treatments that have already passed earlier safety screening. The informed consent document for any specific trial details the known risks and what to expect. Discuss those risks with your physician before deciding whether to participate.

Where can I verify the data on this page?

Every detail on this page comes directly from the ClinicalTrials.gov API. Click "View on ClinicalTrials.gov" in the sidebar to see the official, unmodified record. The federal record is always authoritative; this page is a structured presentation with a plain-English eligibility translation. For background on how clinical trials are regulated, see the FDA drug approval process documentation.

How This Page Is Built

Every field on this page is pulled directly from the ClinicalTrials.gov API v2 — no estimates, no proxies. The plain-English eligibility translation is generated from the original protocol text and reviewed for fidelity to the underlying clinical criteria. The original clinical text remains visible in the collapsible section above so users and clinicians can verify the translation. Read the full methodology for the data pipeline and known limitations.

Source: ClinicalTrials.gov API v2 record for NCT06628947. Maintained by the National Library of Medicine at NIH. Public domain. Cite as: "TrialFinderData. NCT06628947. Data: ClinicalTrials.gov."

Medical disclaimer: This page is informational, not medical advice. Talk to your doctor about whether a clinical trial is right for you.

Last updated 2026-05-08 · Data from ClinicalTrials.gov.