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Retinitis PigmentosaPhase 1/2NCT05282953

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 1/2 study testing KIO-301 in people with Retinitis Pigmentosa and Choroideremia, run by Kiora Pharmaceuticals, Inc. at 3 sites in Australia.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

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). Read more about this study

Who is it for?
Ages 18 to 80 years
What is being tested?
KIO-301
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
32 participants
Where
3 sitesAustralia
Study dates
10 Nov 2022to 24 Mar 2026
Phase
Phase 1/2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 80 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Be aged 18 to 80 years at Visit 1 of either sex and of any race.
  • 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.
  • Be willing and able to follow all instructions and attend all study visits.
  • Have a clinical diagnosis of retinitis pigmentosa (Cohorts 1 - 3) or choroideremia (Cohort 3 only).
  • Have a visual acuity of:
  • no light perception for Cohort 3 or
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  • no light perception or bare light perception for Cohort 1 confirmed with a LogMar > 2.9 using the Berkeley Rudimentary Vision Test (BRVT), or
  • count fingers or hand motion for Cohort 2 as confirmed by a LogMar ≤ 2.9 and > 1.6 using the BRVT.
  • Have similar visual acuity in both eyes as defined as a LogMar difference between eyes of < 0.05 using the BRVT.
  • Be willing to avoid disallowed medications and treatments for the duration of the study.
  • 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.
  • Patients with RP, CHM, or other non-syndromic, rod-dominant IRDs, and a visual acuity of:
  • 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).
  • 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.
  • Aged 18 years or older and of either sex and of any race.
  • 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.
  • Willing and able to attend all study visits and perform study assessments.

Who cannot join

  • Have evidence of material/substantial optic nerve disease.
  • Have a history of retinal detachments.
  • 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.
  • Have high intraocular pressure (IOP) >22 mm Hg.
  • Have had a previous intraocular surgery (excluding phakocataract surgery).
  • Have aphakia.
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  • 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.
  • 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.
  • 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.
  • 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).
  • Have implanted pacemakers.
  • Have intracranial aneurysm clips.
  • Have cochlear implants.
  • Have implanted drug infusion and prosthetic devices.
  • Have certain intrauterine contraceptive devices.
  • Have any other type of metal implant or object such as shrapnel, pins, screws or wire mesh.
  • Sub-study:
  • 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.

Trial locations and contacts

3 sites in 1 country.

Australia3 sites

  • Royal Adelaide Hospital

    Adelaide, South Australia, 5000

  • Harley Eye Clinic

    North Adelaide, South Australia, 5006

  • Save Sight Institute

    Sydney, New South Wales, 2001

Other trials in Australia

About this study

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).

Read the detailed description

Main Study: assesses the safety and tolerability of single doses of KIO-301 when administered intravitreally to participants with retinitis pigmentosa and choroideremia.

Sub-study: determines the test/re-test reliability of functional vision assessments in participants with low visual acuity.

Being tested
KIO-301
Medical subject headings
Retinitis Pigmentosa, Choroideremia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • KIO-301Drug

    KIO-301 intravitreal injection at ascending doses

Study groups in NCT05282953
Study groupRoleWhat they receive
Patients with Retinitis Pigmentosa and ChoroideremiaExperimentalNot described
Receives: Drug: KIO-301

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Main Study Primary Outcome Measure

    Number of participants with treatment-related adverse events as assessed by overall changes in retinal thickness and appearance, clinical chemistry and hematology, and other eye assessments.

    Measured: 84 days.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted16 Mar 2022
  2. Study start10 Nov 2022
  3. Primary completion10 Aug 2023
  4. Study completion24 Mar 2026

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
32 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05282953
Sponsor's protocol ID
KIO-301-1101
Status last verified
Aug 2025
First submitted
8 Mar 2022
Last updated
4 May 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Casson RJ, Daniels E, Barras CD, Dwyer A, Strem BM, Wykoff CC, Gregorio-King C, Schuh C, Kramer RH, Van Gelder RN. Intravitreal photoswitch therapy in advanced retinitis pigmentosa: a phase 1 open-label trial. Nat Med. 2026 May;32(5):1865-1870. doi: 10.1038/s41591-026-04317-6. Epub 2026 Apr 14.

    PubMed 41981307

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing KIO-301 in people with Retinitis Pigmentosa and Choroideremia, run by Kiora Pharmaceuticals, Inc. at 3 sites in Australia.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 21 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 4 May 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 sites in Australia. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Main Study Primary Outcome Measure (time frame: 84 days.). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05282953, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 May 2026.

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