Testing KIO-301 for patients with Retinitis Pigmentosa and Choroideremia

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)

This study is testing a new eye treatment called KIO-301 to see if it can help improve vision for people with retinitis pigmentosa and choroideremia.

Quick facts

• Phase: Phase1; Phase2
• Study type: Interventional
• Enrollment: 12 (estimated)
• Ages: 18 Years to 80 Years
• Sex: All
• Sponsor: Kiora Pharmaceuticals, Inc. (Industry-sponsored)
• Locations: 2 sites (Adelaide, South Australia and 1 other locations)
• Trial ID: NCT05282953 on ClinicalTrials.gov

What this trial studies

This study evaluates the safety, tolerability, and efficacy of KIO-301, administered directly into the eye, for patients diagnosed with retinitis pigmentosa and choroideremia. It is a phase I/II dose-escalating trial, meaning that the dosage of KIO-301 will increase in a controlled manner to assess its effects. The study is open-label, allowing both researchers and participants to know the treatment being administered. The goal is to determine how well this treatment works in improving vision for affected patients.

Who should consider this trial

Good fit: Ideal candidates include individuals with a clinical diagnosis of retinitis pigmentosa or choroideremia and specific visual acuity criteria.

Not a fit: Patients with significant optic nerve disease, previous retinal detachments, or other serious ocular conditions may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could potentially improve vision and quality of life for patients suffering from these degenerative eye conditions.

How similar studies have performed: While this approach is novel, similar studies targeting retinal diseases have shown promise, indicating potential for success.

Eligibility criteria

Inclusion Criteria:

1. Have a clinical diagnosis of retinitis pigmentosa (Cohorts 1 - 3) or choroideremia (Cohort 3 only).
2. 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.
3. Have similar visual acuity in both eyes as defined as a LogMar difference between eyes of \< 0.05 using the BRVT.

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.

Where this trial is running

Adelaide, South Australia and 1 other locations

• Royal Adeliade Hospital — Adelaide, South Australia, Australia (Recruiting)
• Harley Eye Clinic — North Adelaide, South Australia, Australia (Recruiting)

Study contacts

• Principal investigator: Robert Casson — Royal Adelaide Hospital
• Study coordinator: Eric Daniels, M.D., MBA

Conditions Retinitis Pigmentosa; Choroideremia