A Study of the Efficacy and Safety of KUS121 in Participants With Acute Non-Arteritic Central Retinal Artery Occlusion (CRAO)

NCT06178055 | Completed Phase 2 DMC FDA Drug

• 1 other identifier: KDK-1101-02
• Study Type: interventional
• Target: 17
• Locations: 1 country
• Sites: 11

Brief Summary

Central retinal artery occlusion (CRAO) is an ophthalmologic emergency which leads to severe and permanent vision loss. There is no evidence-based therapy for CRAO. The objective of this GION study is to evaluate the efficacy and safety of KUS121 intravitreal (IVT) injection in participants with acute non-arteritic CRAO.

Conditions

Central Retinal Artery Occlusion

Outcome Measures

Primary Outcomes (1)

• Proportion of study eyes which achieve a gain of 15 ETDRS letters or more in BCVA at Week 12 compared to baseline

  BCVA: Best-Corrected Visual Acuity; ETDRS: Early Treatment Diabetic Retinopathy Study. A higher ETDRS letter score means a better outcome (better visual acuity).

  At baseline, Week 12

Secondary Outcomes (8)

• Proportion of study eyes which achieve a gain of 15 ETDRS letters or more in BCVA at Week 4 compared to baseline

  At baseline, Week 4

• Proportion of study eyes which achieve a gain of 0.3 Logarithm of Minimum Angle of Resolution (Log MAR) in BCVA at Week 12 compared to baseline

  At baseline, Week 12

• Proportion of study eyes which achieve a gain of 10 ETDRS letters or more in BCVA at Week 12 compared to baseline

  At baseline, Week 12

• Mean change in BCVA (readable letters and Log MAR) at Week 12 compared to baseline

  At baseline, Week 12

• Change in retinal thickness from baseline at Week 12 as measured by SD-OCT

  At baseline, Week 12

Study Arms (3)

KUS121 high dose group

EXPERIMENTAL

Drug: KUS121 high dose

KUS121 low dose group

EXPERIMENTAL

Drug: KUS121 low dose

Control group

SHAM COMPARATOR

Drug: Sham procedure

Interventions

KUS121 high dose DRUG

Participants will receive KUS121 high dose intravitreal (IVT) injection once on each of 3 consecutive days (Day 1 through Day 3).

KUS121 high dose group

KUS121 low dose DRUG

Participants will receive KUS121 low dose intravitreal (IVT) injection once on each of 3 consecutive days (Day 1 through Day 3).

KUS121 low dose group

Sham procedure DRUG

Participants will receive a sham procedure that mimics an intravitreal (IVT) injection once on each of 3 consecutive days (Day 1 through Day 3).

Control group

Eligibility Criteria

• Age: 20 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Participants who are willing and able to comply with clinic visits and study-related procedures and able to provide signed informed consent in person or from their legally authorized representative
• Males and females ≥ 20 years of age at that time of providing signed informed consent
• Diagnosed as non-arteritic Central Retinal Artery Occlusion from 3 hours until no more than 48 hours after the onset of significant visual acuity changes
• Best Corrected Visual Acuity (BCVA) Early Treatment Diabetic Retinopathy Study (ETDRS) letter score of 0 to 35 in the study eye at screening prior to enrollment (eyes with 'no light perception' or 'light perception' are to be excluded)
• Retinal thickening or hyper-reflectivity in retinal inner layers in spectral domain optical coherence tomography (SD-OCT), or white turbid edema in fundus examination

You may not qualify if:

• Presence of the following conditions in the study eye:
• Infection in or around the eye
• Uncontrolled intraocular pressure
• Abnormality in macula other than CRAO findings
• History of macular photocoagulation
• Opacity in visual axis preventing fundus examination or fundus imaging (e.g., corneal dystrophy)
• Neovascularization of iris and retina
• Any inflammatory disease involving the eye
• Optic atrophy
• Glaucomatous cupping greater than 0.9
• Prior vitrectomy
• Aphakia with the absence of posterior capsule
• Any IVT injection or sub-Tenon's injection within 1 month of screening
• Any intraocular surgery or ocular implant within 3 months of screening
• Any history of ocular trauma within 3 months of screening

Sponsors & Collaborators

• Kyoto Drug Discovery and Development Co., Ltd.: lead

Study Sites (11)

The Retina Partners

Encino, California, 91436, United States

Location

Salehi Retina Institute, Inc

Huntington Beach, California, 92647, United States

Location

Kaiser Permanente Oakland Med Ctr.

Oakland, California, 94611, United States

Location

Florida Retina Institute

Jacksonville, Florida, 32216, United States

Location

Cumberland Valley Retina Consultants

Hagerstown, Maryland, 21740, United States

Location

Kresge Eye Institute/Wayne State University

Detroit, Michigan, 48201, United States

Location

NYEE Infirmary of Mount Sinai

New York, New York, 10003, United States

Location

UH Eye Institute

Cleveland, Ohio, 44106, United States

Location

Austin Retina Associates

Austin, Texas, 78705, United States

Location

Retina Consultants of Texas

San Antonio, Texas, 78240, United States

Location

Retina Consultants of Texas

The Woodlands, Texas, 77384, United States

Location

Related Publications (1)

• Ikeda HO, Muraoka Y, Hata M, Sumi E, Ikeda T, Nakagawa T, Abe H, Tada H, Morita S, Kakizuka A, Yoshimura N, Tsujikawa A. Safety and effectiveness of a novel neuroprotectant, KUS121, in patients with non-arteritic central retinal artery occlusion: An open-label, non-randomized, first-in-humans, phase 1/2 trial. PLoS One. 2020 Feb 13;15(2):e0229068. doi: 10.1371/journal.pone.0229068. eCollection 2020.

  PMID: 32053676 BACKGROUND

MeSH Terms

Conditions

Retinal Artery Occlusion

Interventions

KUS121

Condition Hierarchy (Ancestors)

Retinal Diseases; Eye Diseases; Arterial Occlusive Diseases; Vascular Diseases; Cardiovascular Diseases

Study Officials

• Kunihiro Musashi, MD., Ph.D.

  Kyoto Drug Discovery ＆ Development Co., Ltd.

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 1, 2023
• First Posted: December 20, 2023
• Study Start: April 16, 2024
• Primary Completion: March 3, 2025
• Study Completion: November 30, 2025
• Last Updated: February 18, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (11)