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A Phase II Study of Intravitreal KIO-301 in Patients With Late-stage Retinitis Pigmentosa | Clinical Trials | Clareo Health

RECRUITINGPHASE2

A Phase II Study of Intravitreal KIO-301 in Patients With Late-stage Retinitis Pigmentosa

A Phase II, Randomised, Controlled, Double Masked, Multiple Dose Study of the Safety, Tolerability and Efficacy of Intravitreal KIO-301 in Patients With Late-stage Retinitis Pigmentosa

NCT06628947•Kiora Pharmaceuticals, Inc.

View on ClinicalTrials.gov

Summary

The goal of the study is to investigate the safety, tolerability and efficacy of up to 3 doses of KIO-301 administered by intravitreal (IVT) injection bilaterally every 6 weeks in patients with late-stage retinitis pigmentosa (RP). Late-stage RP patients will include those patients with No Light Perception (NLP), or Low Vision (LV).

Detailed Description

All enrolled and randomised study participants will attend study visits every 3 weeks during treatment (12 weeks) and follow-up (12 weeks) for PK, safety, tolerability, and efficacy assessments. The Screening period may be up to 45 days. Total duration of the main study may be up to 30 weeks. For participants who received placebo in the main study and choose to participate in the open label (OL) extension, duration of participation will be a further 24 weeks with total participation dependent upon time between completion of the main study and initiation of the OL extension.

Eligibility

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•1. Be aged 18 years or older at the time of consent.
•2. Provide informed consent prior to any study procedures, as stipulated by local laws, Ethics Committee (EC) and Regulatory Authority (RA) guidelines.
•3. Be willing and able to follow all study instructions, attend all study visits, and complete all study assessments.
•4. Have a clinical diagnosis of non-syndromic RP, with the exception of Usher's Syndrome Type II (USH2) which is allowed.
•5. Have a visual acuity as per the Berkeley Rudimentary Vision Test (BRVT) at Screening of:
•* NLP OU confirmed by inability to see pen torch light at 25 cm OD, OS, and OU (assigned logMAR of 4.0).
•* LV OU limited to logMAR \> 1.6 and \< 4.0.
•6. Other than intravitreal corticosteroids, participants must not receive intravitreal concomitant medications from Screening until end of study.
•7. For Low Vision (LV) OU participants only: must pass at least one multi-luminance functional vision (MLFV) test at two successive light levels (between 1 and 500 lux), or at 1400 lux. Additionally, they must fail the same test at 0.125 and 0.35 lux.
•8. Must agree to follow appropriate contraception requirements from Screening until 3 months after the last dose of IMP.
+3 more criteria

Exclusion Criteria

•1. Pregnant or breast-feeding, or plan to become pregnant during the study.
•2. Have, in the investigator's opinion, evidence of material/substantial optic nerve disease.
•3. Have a history of one or more retinal detachments.
•4. Other than RP related macular pathologies, have in the investigator's opinion, clinically significant ocular disease (e.g., corneal oedema, uveitis, severe keratoconjunctivitis sicca), or clinically significant opacities of the media which might interfere with the study assessments, or the ability of the participant to complete the study.
•5. Have a history of high myopia (\> 6 diopters).
•6. Have uncontrolled severe glaucoma defined as intraocular pressure (IOP) of \> 26 mmHg when on 2 or more IOP lowering medications and cup disc ratio of ≥ 0.8, as diagnosed by an ophthalmologist.
•7. Have had a previous intraocular surgery (with the exception of phacoemulsification cataract surgery and YAG capsulotomy more than 12 months prior to first study drug administration, which is allowed).
•8. Have aphakia or a subluxed intraocular lens, or have evidence of zonular weakness that in the opinion of the investigator would result in light obfuscation.
•9. 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.
•10. Have any clinically significant abnormality at screening determined by medical history, vital signs, clinical biochemistry, haematology, 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.
+5 more criteria

Locations (5)

Save Sight Institute

Sydney, New South Wales, Australia

Queensland Eye Institute

Woolloongabba, Queensland, Australia

Royal Adelaide Hospital

Adelaide, South Australia, Australia

Cerulea Clinical Trials

East Melbourne, Victoria, Australia

Lions Eye Institute

Nedlands, Western Australia, Australia

Key Dates

Start Date

August 29, 2025

Primary Completion Date

August 1, 2027

Completion Date

August 1, 2027

Last Updated

November 17, 2025

Enrollment

ESTIMATED participants

Conditions

Retinitis Pigmentosa

Interventions

Placebo (Sterile Saline or Balanced Salt Solution)

OTHER

100 μg KIO-301

DRUG

50 μg KIO-301

DRUG

Contacts

Eric Daniels, MD

CONTACT

+61 448 787 315 edaniels@kiorapharma.com

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: November 17, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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A Phase II Study of Intravitreal KIO-301 in Patients With Late-stage Retinitis Pigmentosa

Inclusion Criteria

Exclusion Criteria

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