Retinitis PigmentosaPhase 2ABACUS-2NCT06628947
A Phase II Study of Intravitreal KIO-301 in Patients With Late-stage Retinitis Pigmentosa
A Phase 2 study testing 100 μg KIO-301 and 50 μg KIO-301 in people with Retinitis Pigmentosa, run by Kiora Pharmaceuticals, Inc. at 5 sites in Australia.
Trial summary
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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Retinitis Pigmentosa
- What is being tested?
- 100 μg KIO-301 and 50 μg KIO-301
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 36 participants
- Where
- 5 sitesAustralia
- Study dates
- 29 Aug 2025to Aug 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Be aged 18 years or older at the time of consent.
- Provide informed consent prior to any study procedures, as stipulated by local laws, Ethics Committee (EC) and Regulatory Authority (RA) guidelines.
- Be willing and able to follow all study instructions, attend all study visits, and complete all study assessments.
- Have a clinical diagnosis of non-syndromic RP, with the exception of Usher's Syndrome Type II (USH2) which is allowed.
- 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).
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- LV OU limited to logMAR > 1.6 and < 4.0.
- Other than intravitreal corticosteroids, participants must not receive intravitreal concomitant medications from Screening until end of study.
- 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.
- 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.
Who cannot join
- Pregnant or breast-feeding, or plan to become pregnant during the study.
- Have, in the investigator's opinion, evidence of material/substantial optic nerve disease.
- Have a history of one or more retinal detachments.
- 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.
- Have a history of high myopia (> 6 diopters).
- 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.
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- 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).
- 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.
- 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.
- 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.
- 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 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.
- 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.
- Have known or suspected hypersensitivity to any of the study drug excipients.
- Are taking any medications that are known to be toxic to the retina or optic nerve.
Trial locations and contacts
5 sites in 1 country. Contact the study team or a site near you to ask about taking part.
5 sites in 1 country
Australia5 sites, 5 recruiting
- Recruiting
Royal Adelaide Hospital
Adelaide, South Australia, 5000
Melanie Willoughby, +61 8 7074 2257, Melanie.Willoughby@sa.gov.au
Robert Casson
- Recruiting
Cerulea Clinical Trials
East Melbourne, Victoria, 3002
Lauren Moussallem, +61 3 9929 0118, lmoussallem@cerulea.org.au
Doron Hickey
- Recruiting
Lions Eye Institute
Nedlands, Western Australia, 6009
Tammy Corica, +61 8 9381 0829, tammy.corica@lei.org.au
Fred Chen
- Recruiting
Save Sight Institute
Sydney, New South Wales, 2000
John Grigg, +61 2 9382 7300, john.grigg@sydney.edu.au
John Grigg
- Recruiting
Queensland Eye Institute
Woolloongabba, Queensland, 4102
Brett Caldwell, +61 7 3239 5001, Brett.Caldwell@qei.org.au
Anthony Kwan
About this study
Official title: 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
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).
Read the detailed description
All enrolled and randomised study participants will attend study visits every 3 weeks during treatment (12 weeks) and follow-up (12 weeks) for PK, safety, tolerability, and efficacy assessments. The Screening period may be up to 45 days. Total duration of the main study may be up to 30 weeks.
For participants who received placebo in the main study and choose to participate in the open label (OL) extension, duration of participation will be a further 24 weeks with total participation dependent upon time between completion of the main study and initiation of the OL extension.
- Conditions
- Retinitis Pigmentosa
- Being tested
- Placebo (Sterile Saline or Balanced Salt Solution), 100 μg KIO-301, 50 μg KIO-301
- Keywords
- Late-stage RP with No Light Perception, Late-stage RP with Low Vision
- Medical subject headings
- Retinitis Pigmentosa
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Placebo (Sterile Saline or Balanced Salt Solution)Other
A control 50 μl injection of clear sterile saline or balanced salt solution (BSS) liquid.
100 μg KIO-301Drug
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.
50 μg KIO-301Drug
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.
| Study group | Role | What they receive |
|---|---|---|
| 50 μg KIO-301 | Experimental | 50 μg KIO-301 or placebo administered by IVT injection bilaterally (OU) once every 6 weeks for 3 administrations per participant. Receives: Other: Placebo (Sterile Saline or Balanced Salt Solution); Drug: 50 μg KIO-301 |
| 100 μg KIO-301 | Experimental | 100 μg KIO-301 or placebo administered by IVT injection OU once every 6 weeks for 3 administrations per participant. Receives: Other: Placebo (Sterile Saline or Balanced Salt Solution); Drug: 100 μg KIO-301 |
What is being measured
The primary outcome is the main question the study is designed to answer.
To assess the safety and tolerability of multiple doses of KIO-301 administered by IVT injection bilaterally of KIO-301 in patients with late-stage RP.
Change in ophthalmic and non-ophthalmic adverse events
Measured: Baseline (Week 1/pre-dose) to Week 25 (12 weeks post 3rd IVT administration of KIO-301)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Oct 2024
- Study start29 Aug 2025
- Primary completionAug 2027 (estimated)
- Study completionAug 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Retinitis PigmentosaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Cohort design: Cohort 1A: • RP who have NLP will be randomised (2:1) to receive 50 μg KIO-301 or placebo Cohort 1B: • RP who have LV will be randomised (2:1) to receive 50 μg KIO-301 or placebo Cohort 2A: • RP who have NLP will be randomised (2:1) to receive 100 μg KIO-301 or placebo Cohort 2B: • RP who have LV will be randomised (2:1) to receive 100 μg KIO-301 or placebo Cohort 3A: • RP who have NLP will be randomised (2:1) to receive 25 μg KIO-301 or placebo Cohort 3B: • RP who have LV will be randomised (2:1) to receive 25 μg KIO-301 or placebo
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 36 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Kiora Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Doron Hickey, MBChB, Principal investigator, The Centre for Eye Research Australia (CERA)Robert Casson, MBBS (Hons), Principal investigator, Royal Adelaide Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06628947
- Sponsor's protocol ID
- KIO-301-2101
- Acronym
- ABACUS-2
- Status last verified
- Jul 2025
- First submitted
- 3 Oct 2024
- Last updated
- 18 Jun 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 18 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT06628947
- Acronym / sponsor ID
- ABACUS-2 / KIO-301-2101
- Conditions
- Retinitis Pigmentosa
- Keywords
- Late-stage RP with No Light Perception, Late-stage RP with Low Vision
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 36 participants (estimated)
- Lead sponsor
- Kiora Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 29 Aug 2025 (actual)
- Primary completion
- Aug 2027 (estimated)
- Study completion
- Aug 2027 (estimated)
- First posted
- 8 Oct 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Detailed description
All enrolled and randomised study participants will attend study visits every 3 weeks during treatment (12 weeks) and follow-up (12 weeks) for PK, safety, tolerability, and efficacy assessments. The Screening period may be up to 45 days. Total duration of the main study may be up to 30 weeks.
For participants who received placebo in the main study and choose to participate in the open label (OL) extension, duration of participation will be a further 24 weeks with total participation dependent upon time between completion of the main study and initiation of the OL extension.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- Cohort design: Cohort 1A: • RP who have NLP will be randomised (2:1) to receive 50 μg KIO-301 or placebo Cohort 1B: • RP who have LV will be randomised (2:1) to receive 50 μg KIO-301 or placebo Cohort 2A: • RP who have NLP will be randomised (2:1) to receive 100 μg KIO-301 or placebo Cohort 2B: • RP who have LV will be randomised (2:1) to receive 100 μg KIO-301 or placebo Cohort 3A: • RP who have NLP will be randomised (2:1) to receive 25 μg KIO-301 or placebo Cohort 3B: • RP who have LV will be randomised (2:1) to receive 25 μg KIO-301 or placebo
| Arm | Type | Receives |
|---|---|---|
| 50 μg KIO-301 | Experimental | Other: Placebo (Sterile Saline or Balanced Salt Solution), Drug: 50 μg KIO-30150 μg KIO-301 or placebo administered by IVT injection bilaterally (OU) once every 6 weeks for 3 administrations per participant. |
| 100 μg KIO-301 | Experimental | Other: Placebo (Sterile Saline or Balanced Salt Solution), Drug: 100 μg KIO-301100 μg KIO-301 or placebo administered by IVT injection OU once every 6 weeks for 3 administrations per participant. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- Be aged 18 years or older at the time of consent.
- Provide informed consent prior to any study procedures, as stipulated by local laws, Ethics Committee (EC) and Regulatory Authority (RA) guidelines.
- Be willing and able to follow all study instructions, attend all study visits, and complete all study assessments.
- Have a clinical diagnosis of non-syndromic RP, with the exception of Usher's Syndrome Type II (USH2) which is allowed.
- Have a visual acuity as per the Berkeley Rudimentary Vision Test (BRVT) at Screening of:
Key exclusion criteria (15 listed)
- Pregnant or breast-feeding, or plan to become pregnant during the study.
- Have, in the investigator's opinion, evidence of material/substantial optic nerve disease.
- Have a history of one or more retinal detachments.
- 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.
- Have a history of high myopia (> 6 diopters).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Placebo (Sterile Saline or Balanced Salt Solution)A control 50 μl injection of clear sterile saline or balanced salt solution (BSS) liquid. | Other | 100 μg KIO-301, 50 μg KIO-301 |
| 100 μg KIO-301KIO-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 | 100 μg KIO-301 |
| 50 μg KIO-301KIO-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 |
Primary outcome
To assess the safety and tolerability of multiple doses of KIO-301 administered by IVT injection bilaterally of KIO-301 in patients with late-stage RP.
Time frame: Baseline (Week 1/pre-dose) to Week 25 (12 weeks post 3rd IVT administration of KIO-301)
Change in ophthalmic and non-ophthalmic adverse events
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jun 2026.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Doron Hickey, MBChB principal investigator
The Centre for Eye Research Australia (CERA)
Robert Casson, MBBS (Hons) principal investigator
Royal Adelaide Hospital
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing 100 μg KIO-301 and 50 μg KIO-301 in people with Retinitis Pigmentosa, run by Kiora Pharmaceuticals, Inc. at 5 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 13 inclusion and 15 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: looking for participants now, as of the sponsor's last update on 18 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 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 phase mean?
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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Claudia Gregorio-King (+61 421 992 076, clinical@kiorapharma.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To assess the safety and tolerability of multiple doses of KIO-301 administered by IVT injection bilaterally of KIO-301 in patients with late-stage RP. (time frame: Baseline (Week 1/pre-dose) to Week 25 (12 weeks post 3rd IVT administration of KIO-301)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 18 Jun 2026.