Macular EdemaPhase 2KLARITY-1NCT06825702
Safety, Tolerability and Efficacy of Intravitreal KIO-104 in Patients With Macular Edema
A Phase 2 study testing KIO-104 in people with Macular Edema, run by Kiora Pharmaceuticals, Inc. at 6 sites in Australia.
Trial summary
This is a multi-center, open label study to assess the safety, tolerability, and efficacy of KIO-104 administered by IVT injection to the study eye of eligible participants with macular edema. Read more about this study
- Who is it for?
- Ages 18 to 85 years
- Conditions the study is for
- Macular Edema
- What is being tested?
- KIO-104
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 28 participants
- Where
- 6 sitesAustralia
- Study dates
- 1 May 2025to Dec 2026
- 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 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participants must meet all the following criteria:
- Be aged 18 to 85 years inclusive 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 ME in the study eye secondary to non-infectious uveitis, retinal vein occlusion, diabetic retinopathy or cataract surgery.
- If currently receiving systemic corticosteroid therapy or immunosuppressive therapy (or any combination thereof), be on a stable dose of therapy for at least 3 months prior to Screening and during the study.
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- Have a Central Subfield Thickness (CST) of ≥ 350 μm.
- Have a Best Corrected Visual Acuity (BCVA) in the study eye of:
- ≤ 20/32 (6/9.5); logMAR ≥ 0.2; ≤ 75 ETDRS letters
- ≥ 20/800 (6/240); logMAR ≤ 1.6; ≥ 5 ETDRS letters
- Have media clarity and pupillary dilation sufficient for adequate visualization and assessment of the study eye.
- Be willing to avoid disallowed medications and treatments for the duration of the study.
- Agree to follow appropriate contraception requirements from screening until 3 months after the last dose of the study drug.
- Participants assigned female at birth who are of child-bearing potential (OCBP) must agree to a pregnancy test at Screening and prior to each dose of investigational medicinal product (IMP) 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. Post-menopausal status of amenorrheic female participants should be confirmed at Screening through testing of follicle-stimulating hormone (FSH) as per analysing laboratory threshold.
- 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
- Participants must not meet any of the following criteria:
- Have media opacities (cornea, anterior or posterior synechia, cataract, vitreous haze and others) of either eye that preclude investigation and documentation of the posterior pole and intravenous fluorescein angiography, or optical coherence tomography evaluation in the study eye.
- Receive local or systemic biologicals (i.e. tumour necrosis factor [TNF]-blockers, B-cell blockers, cytokines, cytokine-blockers, receptor antagonists) 90 days prior to Day 1 or planned during the study.
- Receive treatment with cyclophosphamide or chlorambucil during the study.
- Receive intravitreal injections (including but not limited to anti-vascular endothelial growth factors) 90 days prior to Day 1 or planned during the study.
- Receive a posterior subtenon's or orbital floor injection of steroids 90 days prior to Day 1 or planned during the study.
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- Have any implantable corticosteroid-eluting device (Ozurdex, Iluvien, Retisert, triamcinolone intravitreal implant, fluocinolone intravitreal implant) in the study eye, with the following exceptions:
- If the device had been removed more than 90 days prior to Day 1 of the study.
- If Ozurdex® had been implanted at least 6 months before Day 1 of the study.
- If Iluvien® or Retisert® had been implanted at least 3 years before Day 1 of the study.
- Use of topical steroids are permissible provided the participant is receiving a stable dose for at least 3 months prior to Screening and during the study.
- Have ocular surgery (including cataract extraction, laser surgery such as pan retinal photocoagulation, vitreoretinal or scleral buckling surgery) in the study eye, within 90 days prior to Day 1, or planned during the study.
- Have a capsulotomy in the study eye, within 30 days prior to Day 1, and during the study.
- Have Intraocular pressure (IOP) ≥ 25 mmHg in the study eye (glaucoma patients maintained on no more than one topical medication with IOP < 25 mmHg are allowed to participate).
- Have ocular hypotony (IOP < 6 mmHg) in the study eye.
- Have aphakia or anterior chamber lens in the study eye.
- Have visible scleral thinning, scleral ectasia or keratoconus in the study eye.
- Have presence of any ocular malignancy in either eye.
- Have evidence of any other clinically significant ocular disease that might interfere with the study assessments.
- Have ocular or periocular (either eye) or systemic infection and/or a temperature greater than 38.0°C, or the use of systemic or topical ocular antibiotics within 14 days of Day 1.
- 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 and ophthalmic history, vital signs, clinical biochemistry, hematology, 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 participated in any other investigational drug or device clinical trial within 90 days prior to Day 1 or planning to participate in other investigational drug or device clinical trials during the study and within 120 days following Day 1. This includes both ocular and non-ocular clinical trials.
- Have any clinically significant clotting abnormality as assessed by the Investigator, which might interfere with the collection of blood samples required by the study.
- Have a known allergy or hypersensitivity to the study medication, any component of the delivery vehicle, any corticosteroids, any diagnostic agents used during the study (e.g., fluorescein, dilation drops), or any other standard of care medications likely to be used during the study (e.g., antibiotic drops, povidone, rescue medications).
- Be pregnant or breast-feeding, or plan to become pregnant during the study.
Trial locations and contacts
6 sites in 1 country. Contact the study team or a site near you to ask about taking part.
6 sites in 1 country
Australia6 sites, 6 recruiting
- Recruiting
Royal Adelaide Hospital
Adelaide, South Australia, 5000
Jagjit (Jolly) Singh Gilhotra, +610870742257, jsgilhotra@gmail.com
Jagjit (Jolly) Singh Gilhotra
- Recruiting
Centre for Eye Research Australia
East Melbourne, Victoria, 3002
Marios Constantinou, +610399590058, MConstantinou@ceruleaclinicaltrials.org.au
Lyndell Lim
- Recruiting
Hobart Eye Surgeons
Hobart, Tasmania, 7000
Jacqueline Townley, +610362106000, jacqueline.townley@eyesurgeons.com.au
Nitin Verma
- Recruiting
Lions Eye Institute Limited
Nedlands, Western Australia, 6009
Amelia Jason, +61 08 6382 0582, lionsvisiontrials@lei.org.au
Mei-Ling Tay-Kearney
- Recruiting
Sydney Eye Hospital
Sydney, New South Wales, 2001
Elisa Cornish, +61413805583, elisa.cornish@sydney.edu.au
Elisa Cornish
- Recruiting
Sydney West Retina
Westmead, New South Wales, 2145
Honey Grace Paule, +610298939022, honeygrace.swr@gmail.com
Paul Mitchell
About this study
Official title: A Phase 2, Open-label, Multiple Dose Study of the Safety, Tolerability and Efficacy of Intravitreal KIO-104 in Patients With Macular Edema (KLARITY-1)
This is a multi-center, open label study to assess the safety, tolerability, and efficacy of KIO-104 administered by IVT injection to the study eye of eligible participants with macular edema.
Read the detailed description
In Part A of the study, the safety and efficacy of up to three doses (administrations) of KIO-104, administered every 2 weeks, will be investigated in 2 possible dose cohorts.
Cohort 1: Low dose KIO-104 administered to the study eye Cohort 2: High dose KIO-104 administered to the study eye Part B of the study will investigate the safety and efficacy of up to three doses (administrations) of KIO-104 with 2 different dosing regimens (every 2 weeks or every 4 weeks), in participants with macular edema at a dose selected from Part A.
Cohort 3A: KIO-104 administered to the study eye every 2 weeks Cohort 3B: KIO-104 administered to the study eye every 4 weeks
- Conditions
- Macular Edema
- Being tested
- KIO-104
- Keywords
- Macular Edema
- Medical subject headings
- Macular Edema
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
KIO-104Drug
KIO-104 is an ophthalmic formulation for IVT injection of the active pharmaceutical ingredient (API) KIO-100, a novel, non-steroidal, small molecule inhibitor of dihydroorotate dehydrogenase (DHODH).
| Study group | Role | What they receive |
|---|---|---|
| Low Dose KIO-104: 2-weekly | Experimental | 2-weekly dosing Receives: Drug: KIO-104 |
| High Dose KIO-104: 2-weekly | Experimental | 2-weekly dosing Receives: Drug: KIO-104 |
| Low or High Dose KIO-104: 2-weekly | Experimental | 2-weekly dosing Receives: Drug: KIO-104 |
| Low or High Dose KIO-104: 4-weekly | Experimental | 4-weekly dosing Receives: Drug: KIO-104 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and Tolerability
The primary objective is safety and tolerability. The incidence of AEs will be presented by System Organ Class (SOC) and Preferred Term (PT) by dose cohorts.
Measured: 23months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Feb 2025
- Study start1 May 2025
- Primary completionNov 2026 (estimated)
- Study completionDec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Macular EdemaStudies 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
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 28 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Kiora Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06825702
- Sponsor's protocol ID
- KIO-104-2100
- Acronym
- KLARITY-1
- Status last verified
- Sep 2026
- First submitted
- 4 Feb 2025
- Last updated
- 9 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Thurau S, Deuter CME, Heiligenhaus A, Pleyer U, Van Calster J, Barisani-Asenbauer T, Obermayr F, Sperl S, Seda-Zehetner R, Wildner G. A new small molecule DHODH-inhibitor [KIO-100 (PP-001)] targeting activated T cells for intraocular treatment of uveitis - A phase I clinical trial. Front Med (Lausanne). 2022 Oct 17;9:1023224. doi: 10.3389/fmed.2022.1023224. eCollection 2022.
PubMed 36325389
Study at a glance
From the registry record, last updated by the sponsor on 9 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Open-label, Multiple Dose Study of the Safety, Tolerability and Efficacy of Intravitreal KIO-104 in Patients With Macular Edema (KLARITY-1)
- ClinicalTrials.gov ID
- NCT06825702
- Acronym / sponsor ID
- KLARITY-1 / KIO-104-2100
- Conditions
- Macular Edema
- Keywords
- Macular Edema
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 28 participants (estimated)
- Lead sponsor
- Kiora Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 1 May 2025 (actual)
- Primary completion
- Nov 2026 (estimated)
- Study completion
- Dec 2026 (estimated)
- First posted
- 13 Feb 2025 (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.
This is a multi-center, open label study to assess the safety, tolerability, and efficacy of KIO-104 administered by IVT injection to the study eye of eligible participants with macular edema.
Detailed description
In Part A of the study, the safety and efficacy of up to three doses (administrations) of KIO-104, administered every 2 weeks, will be investigated in 2 possible dose cohorts.
Cohort 1: Low dose KIO-104 administered to the study eye Cohort 2: High dose KIO-104 administered to the study eye Part B of the study will investigate the safety and efficacy of up to three doses (administrations) of KIO-104 with 2 different dosing regimens (every 2 weeks or every 4 weeks), in participants with macular edema at a dose selected from Part A.
Cohort 3A: KIO-104 administered to the study eye every 2 weeks Cohort 3B: KIO-104 administered to the study eye every 4 weeks
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Low Dose KIO-104: 2-weekly | Experimental | Drug: KIO-1042-weekly dosing |
| High Dose KIO-104: 2-weekly | Experimental | Drug: KIO-1042-weekly dosing |
| Low or High Dose KIO-104: 2-weekly | Experimental | Drug: KIO-1042-weekly dosing |
| Low or High Dose KIO-104: 4-weekly | Experimental | Drug: KIO-1044-weekly dosing |
Who may qualify
- Ages
- 18 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (17 listed)
- Be aged 18 to 85 years inclusive 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 ME in the study eye secondary to non-infectious uveitis, retinal vein occlusion, diabetic retinopathy or cataract surgery.
- If currently receiving systemic corticosteroid therapy or immunosuppressive therapy (or any combination thereof), be on a stable dose of therapy for at least 3 months prior to Screening and during the study.
Key exclusion criteria (27 listed)
- Have media opacities (cornea, anterior or posterior synechia, cataract, vitreous haze and others) of either eye that preclude investigation and documentation of the posterior pole and intravenous fluorescein angiography, or optical coherence tomography evaluation in the study eye.
- Receive local or systemic biologicals (i.e. tumour necrosis factor [TNF]-blockers, B-cell blockers, cytokines, cytokine-blockers, receptor antagonists) 90 days prior to Day 1 or planned during the study.
- Receive treatment with cyclophosphamide or chlorambucil during the study.
- Receive intravitreal injections (including but not limited to anti-vascular endothelial growth factors) 90 days prior to Day 1 or planned during the study.
- Receive a posterior subtenon's or orbital floor injection of steroids 90 days prior to Day 1 or planned during the study.
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 |
|---|---|---|
| KIO-104KIO-104 is an ophthalmic formulation for IVT injection of the active pharmaceutical ingredient (API) KIO-100, a novel, non-steroidal, small molecule inhibitor of dihydroorotate dehydrogenase (DHODH). | Drug | High Dose KIO-104: 2-weekly, Low Dose KIO-104: 2-weekly, Low or High Dose KIO-104: 2-weekly, Low or High Dose KIO-104: 4-weekly |
Primary outcome
Safety and Tolerability
Time frame: 23months
The primary objective is safety and tolerability. The incidence of AEs will be presented by System Organ Class (SOC) and Preferred Term (PT) by dose cohorts.
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 9 Sep 2026.
- Background: PubMed 36325389Thurau S, Deuter CME, Heiligenhaus A, Pleyer U, Van Calster J, Barisani-Asenbauer T, Obermayr F, Sperl S, Seda-Zehetner R, Wildner G. A new small molecule DHODH-inhibitor [KIO-100 (PP-001)] targeting activated T cells for intraocular treatment of uveitis - A phase I clinical trial. Front Med (Lausanne). 2022 Oct 17;9:1023224. doi: 10.3389/fmed.2022.1023224. eCollection 2022.
- Individual participant dataSharing planned: No
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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.
6 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 KIO-104 in people with Macular Edema, run by Kiora Pharmaceuticals, Inc. at 6 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 17 inclusion and 27 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 9 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 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?
Safety and Tolerability (time frame: 23months). 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 NCT06825702, 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 9 Sep 2026.