Safety, Tolerability and Efficacy of Intravitreal KIO-104 in Patients With Macular Edema

NCT06825702 | Recruiting Phase 2

• 1 other identifier: KIO-104-2100
• Study Type: interventional
• Target: 28
• Locations: 1 country
• Sites: 4

Brief 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.

Conditions

Macular Edema

Keywords

Macular Edema

Outcome Measures

Primary Outcomes (1)

• 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.

  23months

Study Arms (4)

Low Dose KIO-104: 2-weekly

EXPERIMENTAL

2-weekly dosing

Drug: KIO-104

High Dose KIO-104: 2-weekly

EXPERIMENTAL

2-weekly dosing

Drug: KIO-104

Low or High Dose KIO-104: 2-weekly

EXPERIMENTAL

2-weekly dosing

Drug: KIO-104

Low or High Dose KIO-104: 4-weekly

EXPERIMENTAL

4-weekly dosing

Drug: KIO-104

Interventions

KIO-104 DRUG

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).

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

Eligibility Criteria

• Age: 18 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• Have a Central Subfield Thickness (CST) of ≥ 350 µm.
• Have a Best Corrected Visual Acuity (BCVA) in the study eye of:
• ≤ 20/32 (Feet); logMAR ≥ 0.2
• ≥ 20/800 (Feet); log MAR ≤ 1.6
• 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.

You may not qualify if:

• 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.
• 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, 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).

Sponsors & Collaborators

• Kiora Pharmaceuticals, Inc.: lead

Study Sites (4)

Sydney Eye Hospital

Sydney, New South Wales, 2001, Australia

RECRUITING

Royal Adelaide Hospital

Adelaide, South Australia, 5000, Australia

NOT YET RECRUITING

Centre for Eye Research Australia

East Melbourne, Victoria, 3002, Australia

RECRUITING

Lions Eye Institute Limited

Nedlands, Western Australia, 6009, Australia

RECRUITING

Related Publications (1)

• 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.

  PMID: 36325389 BACKGROUND

MeSH Terms

Conditions

Macular Edema

Condition Hierarchy (Ancestors)

Macular Degeneration; Retinal Degeneration; Retinal Diseases; Eye Diseases

Central Study Contacts

Claudia Gregorio-King

CONTACT

+61 421 992 076; clinical@kiorapharma.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 4, 2025
• First Posted: February 13, 2025
• Study Start: May 1, 2025
• Primary Completion (Estimated): November 1, 2026
• Study Completion (Estimated): December 1, 2026
• Last Updated: April 30, 2026

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

AU (4)