Trial reportPloS one2020
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.
Trial report in PloS one, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT06178055 (A Phase 2, Double-masked, Randomized, Sham-controlled, Multiple-dose Study of the Efficacy and Safety of Intravitreal KUS121 in the Treatment of Non-Arteritic Central Retinal Artery Occlusion), which is not on this map. Cited by 20 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Phase 2, Double-masked, Randomized, Sham-controlled, Multiple-dose Study of the Efficacy and Safety of Intravitreal KUS121 in the Treatment of Non-Arteritic Central Retinal Artery Occlusion (CRAO)
Who cites it
20 citing papers in PubMed, 27 citations in OpenAlex.
- Post hoc analysis of a randomized placebo-controlled trial suggests potential visual benefits of branched-chain amino acids in retinitis pigmentosa.Scientific reports · 2025Trial
- Efficacy and Safety of Branched Chain Amino Acids on Retinitis Pigmentosa: A Randomized, Double-Blind, Placebo-Controlled Clinical Trial.Translational vision science & technology · 2024Trial
- Branch retinal artery occlusion time to presentation and diagnosis: a retrospective review.International journal of retina and vitreous · 2026Article
- Safety and Efficacy of Orally Administered SJP-0008 in Central Retinal Artery Occlusion: A Phase IIa Randomized Clinical Trial.Ophthalmology science · 2026Article
- Valosin-containing protein modulator KUS121 protects retinal neurons in a rat model of anterior ischemic optic neuropathy.Scientific reports · 2025Article
- Retinal Artery Occlusion: A Review of Current Management Practices.Journal of ophthalmic & vision research · 2024Review
- A telemedicine-enabled intravenous thrombolytic treatment pathway for patients with hyperacute non-arteritic central retinal artery occlusion.American journal of ophthalmology case reports · 2024Article
- Therapeutic Strategies for Retinal Artery Occlusion-A Literature Review.Journal of clinical medicine · 2024Review
- The effect of KUS121, a novel VCP modulator, against ischemic injury in random pattern flaps.PloS one · 2024Article
- Central Retinal Artery Occlusion: A Review of Pathophysiological Features and Management.Stroke (Hoboken, N.J.) · 2024Review
- Valosin containing protein (VCP): initiator, modifier, and potential drug target for neurodegenerative diseases.Molecular neurodegeneration · 2023Review
- Neuroprotection for Nonarteritic Central Retinal Artery Occlusion: Lessons from Acute Ischemic Stroke.Clinical ophthalmology (Auckland, N.Z.) · 2023Review
- Involvement of endothelins in neuroprotection of valosin-containing protein modulators against retinal ganglion cell damage.Scientific reports · 2022Article
- Branched Chain Amino Acids Promote ATP Production Via Translocation of Glucose Transporters.Investigative ophthalmology & visual science · 2022Article
- Mitochondrial quality control in the brain: The physiological and pathological roles.Frontiers in neuroscience · 2022Review
- Hypoxia Tolerant Species: The Wisdom of Nature Translated into Targets for Stroke Therapy.International journal of molecular sciences · 2021Review
- KUS121 attenuates the progression of monosodium iodoacetate-induced osteoarthritis in rats.Scientific reports · 2021Article
- Fenofibrate Protects against Retinal Dysfunction in a Murine Model of Common Carotid Artery Occlusion-Induced Ocular Ischemia.Pharmaceuticals (Basel, Switzerland) · 2021Article
- A VCP modulator, KUS121, as a promising therapeutic agent for post-traumatic osteoarthritis.Scientific reports · 2020Article
- Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
12 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Kyoto University Substance (KUS) 121, an ATPase inhibitor of valosin-containing protein, is a novel neuroprotectant. We tested the safety and effectiveness of KUS121 in patients with acute central retinal artery occlusion (CRAO). We conducted an investigator-initiated, first-in-humans, phase 1/2 clinical trial. Nine patients with non-arteritic CRAO symptoms lasting for 4-48 h were enrolled. These patients received daily intravitreal injections of KUS121 for 3 days: 25 μg (low-dose) in the first three patients and 50 μg (high-dose) in the next six patients. The primary endpoint was the safety of the drug. As a secondary endpoint, pharmacokinetics was evaluated. Other key secondary endpoints were changes in best-corrected visual acuity (BCVA), measured using the Early Treatment Diabetic Retinopathy Study chart, visual field scores, and retinal sensitivities between baseline and week 12; and decimal BCVA at week 12. Administration of KUS121 did not result in serious adverse events. All nine patients (100%) showed significant improvement of BCVA. Average readable letter counts, visual field scores, and retinal sensitivities also improved. Decimal BCVA at week 12 was better than 0.1 in four patients (44%) and equal to or better than 0.05 in seven patients (78%). This first-in-humans clinical trial provides support for the safety and efficacy of intravitreal KUS121 injection. To substantiate the safety and effectiveness for patients with acute CRAO, further larger scale clinical studies will be needed.
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What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.