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)

NCT05282953 | Completed Phase 1

• 1 other identifier: KIO-301-1101
• Study Type: interventional
• Target: 32
• Locations: 1 country
• Sites: 3

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).

Conditions

Retinitis Pigmentosa; Choroideremia; Non-Syndromic Rod-Dominant Inherited Retinal Diseases

Outcome Measures

Primary Outcomes (1)

• Main Study Primary Outcome Measure

  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.

Other Outcomes (1)

• Sub-study Outcome Measure

  42 days.

Study Arms (1)

Patients with Retinitis Pigmentosa and Choroideremia

EXPERIMENTAL

Drug: KIO-301

Interventions

KIO-301 DRUG

KIO-301 intravitreal injection at ascending doses

Patients with Retinitis Pigmentosa and Choroideremia

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Be aged 18 to 80 years at Visit 1 of either sex and of any race.
• Be willing and able to provide informed consent either written, or if the Participant is not able to read, provide consent as stipulated by local laws and Human Research Ethics Committee (HREC) guidelines.
• Be willing and able to follow all instructions and attend all study visits.
• Have a clinical diagnosis of retinitis pigmentosa (Cohorts 1 - 3) or choroideremia (Cohort 3 only).
• Have a visual acuity of:
• no light perception for Cohort 3 or
• no light perception or bare light perception for Cohort 1 confirmed with a LogMar \> 2.9 using the Berkeley Rudimentary Vision Test (BRVT), or
• count fingers or hand motion for Cohort 2 as confirmed by a LogMar ≤ 2.9 and \> 1.6 using the BRVT.
• Have similar visual acuity in both eyes as defined as a LogMar difference between eyes of \< 0.05 using the BRVT.
• Be willing to avoid disallowed medications and treatments for the duration of the study.
• Contraception:
• If female, agree to submit to a pregnancy test at Visit 1, or not be of childbearing potential. All participants of childbearing potential must agree to use an acceptable method of contraception for the duration of the study. Acceptable methods of birth control include oral, transdermal, injectable, or implantable contraception, intrauterine device, abstinence, and surgical sterilisation of partner. Female Participants are not of childbearing potential if they have had a hysterectomy, bilateral oophorectomy, bilateral tubal ligation, or are post-menopausal by at least 12 months.
• Male participants will be surgically sterile for at least 3 months, or when sexually active with female partners of childbearing potential will be required to use a male condom from Screening until 3 months the last dose of study drug. Sexual intercourse with female partners who are pregnant, or breastfeeding should be avoided unless condoms (or double barrier method) are used from the time of the first dose until 3 months after the last dose of study drug. Male participants are required to refrain from donation of sperm from Screening until 3 months after the last dose of study drug.
• Participants who have practiced true abstinence for at least 1 year because of the participant's lifestyle choice (i.e., the participant should not become abstinent just for the purpose of study participation) are exempt from contraceptive requirements. Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception. If a participant who is abstinent at the time of signing the ICF becomes sexually active they must agree to use contraception as described above and as outlined in the ICF.
• For participants who are exclusively in same sex relationships, contraceptive requirements do not apply. If a participant who is in a same sex relationship at the time of signing the ICF becomes engaged in a heterosexual relationship, they must agree to use contraception as described above and as outlined in the ICF.

You may not qualify if:

• Have evidence of material/substantial optic nerve disease.
• Have a history of retinal detachments.
• 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.
• Have high intraocular pressure (IOP) \>22 mm Hg.
• Have had a previous intraocular surgery (excluding phakocataract surgery).
• Have aphakia.
• Have a psychiatric condition that 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, blood chemistry, haematology, urinalysis or a 12-lead electrocardiogram (ECG), as assessed by the investigator.
• 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 infection and/or a temperature greater than 38.0°C at the time of Screening. Study entry may be deferred at the discretion of the Principal Investigator (PI).
• Have implanted pacemakers.
• Have intracranial aneurysm clips.
• Have cochlear implants.
• Have implanted drug infusion and prosthetic devices.
• Have certain intrauterine contraceptive devices.

Sponsors & Collaborators

• Kiora Pharmaceuticals, Inc.: lead

Study Sites (3)

Save Sight Institute

Sydney, New South Wales, 2001, Australia

Location

Royal Adelaide Hospital

Adelaide, South Australia, 5000, Australia

Location

Harley Eye Clinic

North Adelaide, South Australia, 5006, Australia

Location

Related Publications (1)

• Casson RJ, Daniels E, Barras CD, Dwyer A, Strem BM, Wykoff CC, Gregorio-King C, Schuh C, Kramer RH, Van Gelder RN. Intravitreal photoswitch therapy in advanced retinitis pigmentosa: a phase 1 open-label trial. Nat Med. 2026 Apr 14. doi: 10.1038/s41591-026-04317-6. Online ahead of print.

  PMID: 41981307 DERIVED

MeSH Terms

Conditions

Retinitis Pigmentosa; Choroideremia

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Retinal Dystrophies; Retinal Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Choroid Diseases; Uveal Diseases; Genetic Diseases, X-Linked

Study Officials

• Robert Casson

  Royal Adelaide Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 8, 2022
• First Posted: March 16, 2022
• Study Start: November 10, 2022
• Primary Completion: August 10, 2023
• Study Completion: March 24, 2026
• Last Updated: May 4, 2026

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share

Locations

AU (3)