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)

Phase 1

Recruiting

• Conditions: Retinitis Pigmentosa; Choroideremia
• Interventions: Drug: KIO-301

• Registration Number: NCT05282953
• Lead Sponsor: Kiora Pharmaceuticals, Inc.

Brief 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). Open label.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2022-03-08
• Study First Submitted QC: 2022-03-15
• Study First Posted: 2022-03-16
• Study Start: 2022-11-10
• Status Verified: 2024-03
• Last Update Submitted: 2025-03-04
• Last Update Posted: 2025-03-06
• Primary Completion: 2025-06
• Study Completion: 2025-09

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 12

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Patients with Retinitis Pigmentosa and Choroideremia	KIO-301	-

Primary Outcome Measures

Name	Time	Method
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.	84 days

Trial Locations

Locations (2)

Royal Adeliade Hospital; 🇦🇺 Adelaide, South Australia, Australia

Harley Eye Clinic; 🇦🇺 North Adelaide, South Australia, Australia