NCT06628947

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

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 36 (estimated)
• Sponsor: Kiora Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Retinitis Pigmentosa

Interventions

Type	Name	Description
OTHER	Placebo (Sterile Saline or Balanced Salt Solution)	A control 50 μl injection of clear sterile saline or balanced salt solution (BSS) liquid.
DRUG	100 μg KIO-301	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.
DRUG	50 μg KIO-301	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.

Timeline

• Start date: 2025-08-29
• Primary completion: 2027-08-01
• Completion: 2027-08-01
• First posted: 2024-10-08
• Last updated: 2025-11-17

Locations

5 sites across 1 country: Australia