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

ConditionRetinitis Pigmentosa

Trial statusRecruiting

Trial phasePhase 2

Trial typeInterventional

Biological sexAll

Age18+

SponsorKiora Pharmaceuticals, Inc.

About this trial

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).

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Be aged 18 years or older 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 non-syndromic RP, with the exception of Usher's Syndrome Type II (USH2) which is allowed.

Disqualifiers

Pregnant or breast-feeding, or plan to become pregnant during the study.

Have, in the investigator's opinion, evidence of material/substantial optic nerve disease.

Have a history of one or more retinal detachments.

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.

Trial design

Design model

Parallel

Treatments tested in this trial

Placebo (Sterile Saline or Balanced Salt Solution)
Other intervention
A control 50 μl injection of clear sterile saline or balanced salt solution (BSS) liquid.
100 μg KIO-301
Drug
KIO-301 drug product is an ophthalmic formulation of the drug substance KIO-300-Cl in sulfobutylether-β-cyclodextrin, sucrose, phosphate buffer salts and water suitable for IVT injection. The drug substance KIO-300-Cl is a quaternary ammonium chloride salt of the active compound KIO-300.
50 μg KIO-301
Drug
KIO-301 drug product is an ophthalmic formulation of the drug substance KIO-300-Cl in sulfobutylether-β-cyclodextrin, sucrose, phosphate buffer salts and water suitable for IVT injection. The drug substance KIO-300-Cl is a quaternary ammonium chloride salt of the active compound KIO-300.

Treatment groups

36 Participants

are divided into 2 treatment groups

Group A: 50 μg KIO-301Experimental treatment 2 interventions

Group B: 100 μg KIO-301Experimental treatment 2 interventions

Trial outcomes

Primary outcomes

To assess the safety and tolerability of multiple doses of KIO-301 administered by IVT injection bilaterally of KIO-301 in patients with late-stage RP.

Change in ophthalmic and non-ophthalmic adverse events

Time frame

Baseline (Week 1/pre-dose) to Week 25 (12 weeks post 3rd IVT administration of KIO-301)

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Kiora Pharmaceuticals, Inc.

Lead sponsor

Source ClinicalTrials.gov

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