Skip to main content
TTrialFinderData
TrialFinderData is for informational purposes only and does not provide medical advice. Always talk to your doctor.

Updated May 2026 · ClinicalTrials.gov

RECRUITINGPhase 1 / Phase 2INTERVENTIONAL

A Phase I/II Dose-escalating Study of the Safety, Tolerability and Efficacy of KIO-301 Administered Intravitreally to Patients With Retinitis Pigmentosa and Choroideremia (ABACUS)

A Phase I/II Dose-escalating Study of the Safety, Tolerability and Efficacy of KIO-301 Administered Intravitreally to Patients With Retinitis Pigmentosa and Choroideremia (ABACUS) (NCT05282953) is a Phase 1 / Phase 2 interventional studying Retinitis Pigmentosa and Choroideremia, 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

A phase I/II dose-escalating study of the safety, tolerability and efficacy of KIO-301 administered intravitreally to patients with retinitis pigmentosa and choroideremia (ABACUS).

What Stage of Research Is This?

Phase 1 trials test a new treatment for the first time in humans, focusing on safety, dosing, and how the body processes the drug. For Retinitis Pigmentosa, a Phase 1 study typically enrolls a small number of participants — often healthy volunteers or patients who have exhausted standard treatment options. Phase 1 results determine whether a treatment moves into larger Phase 2 efficacy studies.

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 48 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)

Main Study: Inclusion Criteria 1. Be aged 18 to 80 years at Visit 1 of either sex and of any race. 2. Be willing and able to provide willing to sign a consent form either written, or if the Participant is not able to read, provide consent as stipulated by local laws and Human Research Ethics Committee (HREC) guidelines. 3. Be willing and able to follow all instructions and attend all study visits. 4. Have a clinical diagnosis of retinitis pigmentosa (Cohorts 1 - 3) or choroideremia (Cohort 3 only). 5. Have a visual acuity of: 1. no light perception for Cohort 3 or 2. no light perception or bare light perception for Cohort 1 confirmed with a LogMar \> 2.9 using the Berkeley Rudimentary Vision Test (BRVT), or 3. count fingers or hand motion for Cohort 2 as confirmed by a LogMar ≤ 2.9 and \> 1.6 using the BRVT. 6. Have similar visual acuity in both eyes as defined as a LogMar difference between eyes of \< 0.05 using the BRVT. 7. Be willing to avoid disallowed medications and treatments for the duration of the study. 8. Contraception: - If female, agree to submit to a pregnancy test at Visit 1, or not be of childbearing potential. All participants of childbearing potential must agree to use an acceptable method of contraception for the duration of the study. Acceptable methods of birth control include oral, transdermal, injectable, or implantable contraception, intrauterine device, abstinence, and surgical sterilisation of partner. Female Participants are not of childbearing potential 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
Main Study: Inclusion Criteria 1. Be aged 18 to 80 years at Visit 1 of either sex and of any race. 2. Be willing and able to provide informed consent either written, or if the Participant is not able to read, provide consent as stipulated by local laws and Human Research Ethics Committee (HREC) guidelines. 3. Be willing and able to follow all instructions and attend all study visits. 4. Have a clinical diagnosis of retinitis pigmentosa (Cohorts 1 - 3) or choroideremia (Cohort 3 only). 5. Have a visual acuity of: 1. no light perception for Cohort 3 or 2. no light perception or bare light perception for Cohort 1 confirmed with a LogMar \> 2.9 using the Berkeley Rudimentary Vision Test (BRVT), or 3. count fingers or hand motion for Cohort 2 as confirmed by a LogMar ≤ 2.9 and \> 1.6 using the BRVT. 6. Have similar visual acuity in both eyes as defined as a LogMar difference between eyes of \< 0.05 using the BRVT. 7. Be willing to avoid disallowed medications and treatments for the duration of the study. 8. Contraception: * If female, agree to submit to a pregnancy test at Visit 1, or not be of childbearing potential. All participants of childbearing potential must agree to use an acceptable method of contraception for the duration of the study. Acceptable methods of birth control include oral, transdermal, injectable, or implantable contraception, intrauterine device, abstinence, and surgical sterilisation of partner. Female Participants are not of childbearing potential if they have had a hysterectomy, bilateral oophorectomy, bilateral tubal ligation, or are post-menopausal by at least 12 months. * Male participants will be surgically sterile for at least 3 months, or when sexually active with female partners of childbearing potential will be required to use a male condom from Screening until 3 months the last dose of study drug. Sexual intercourse with female partners who are pregnant, or breastfeeding should be avoided unless condoms (or double barrier method) are used from the time of the first dose until 3 months after the last dose of study drug. Male participants are required to refrain from donation of sperm from Screening until 3 months after the last dose of study drug. * Participants who have practiced true abstinence for at least 1 year because of the participant's lifestyle choice (i.e., the participant should not become abstinent just for the purpose of study participation) are exempt from contraceptive requirements. Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception. If a participant who is abstinent at the time of signing the ICF becomes sexually active they must agree to use contraception as described above and as outlined in the ICF. * For participants who are exclusively in same sex relationships, contraceptive requirements do not apply. If a participant who is in a same sex relationship at the time of signing the ICF becomes engaged in a heterosexual relationship, they must agree to use contraception as described above and as outlined in the ICF. Exclusion Criteria 1. Have evidence of material/substantial optic nerve disease. 2. Have a history of retinal detachments. 3. Have clinically significant ocular disease (e.g., corneal oedema, uveitis, severe keratoconjunctivitis sicca) which might interfere with the study or clinically significant opacities of the media. 4. Have high intraocular pressure (IOP) \>22 mm Hg. 5. Have had a previous intraocular surgery (excluding phakocataract surgery). 6. Have aphakia. 7. Have a psychiatric condition that 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. 8. Have any clinically significant abnormality at Screening determined by medical history, vital signs, blood chemistry, haematology, urinalysis or a 12-lead electrocardiogram (ECG), as assessed by the investigator. 9. 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. 10. Have clinical signs of active infection and/or a temperature greater than 38.0°C at the time of Screening. Study entry may be deferred at the discretion of the Principal Investigator (PI). 11. Have implanted pacemakers. 12. Have intracranial aneurysm clips. 13. Have cochlear implants. 14. Have implanted drug infusion and prosthetic devices. 15. Have certain intrauterine contraceptive devices. 16. Have any other type of metal implant or object such as shrapnel, pins, screws or wire mesh. Sub-study: Inclusion Criteria 1. Patients with RP, CHM, or other non-syndromic, rod-dominant IRDs, and a visual acuity of: 1. NLP OU confirmed by inability to see pen torch light at 25cm in OD, OS, and OU (assigned logMAR of 4.0 as per BRVT). 2. Light perception (LP), Ultra Low Vision (ULV), or Low Vision (LV) OU (logMAR \> 1.0 and \< 4.0) as determined by the BRVT or Early Treatment of Diabetic Retinopathy Study (ETDRS) chart. 2. Aged 18 years or older and of either sex and of any race. 3. Willing and able to provide informed consent either written, or if the Participant is not able to read, provide consent as stipulated by local laws and HREC guidelines. 4. Willing and able to attend all study visits and perform study assessments. Exclusion Criteria 1\. Have any evidence of any other material/substantial disease that, in the opinion of the investigator, is likely to interfere with the study objectives or put the Participant at risk.

Treatments Being Tested

DRUG

KIO-301

KIO-301 intravitreal injection at ascending doses

Locations (3)

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
Royal Adelaide Hospital
Adelaide, South Australia, Australia
Harley Eye Clinic
North Adelaide, South Australia, Australia

How to Talk to Your Doctor About This Trial

Bring the printable summary of this trial — including the NCT ID (NCT05282953), 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 NCT05282953 clinical trial studying?

A phase I/II dose-escalating study of the safety, tolerability and efficacy of KIO-301 administered intravitreally to patients with retinitis pigmentosa and choroideremia (ABACUS). The full protocol is registered on ClinicalTrials.gov and includes the primary outcome measures, eligibility criteria, and study endpoints.

Who can participate in NCT05282953?

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 NCT05282953?

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 NCT05282953. Maintained by the National Library of Medicine at NIH. Public domain. Cite as: "TrialFinderData. NCT05282953. 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.