OCS-05 in Patients With Optic Neuritis (ACUITY)

A Randomized, Double-blind, Placebo-controlled Study to Evaluate the Safety and Tolerability of OCS-05 in Patients With Acute Optic Neuritis

To evaluate the safety and tolerability of OCS-05 compared to placebo in patients with optic neuritis receiving the standard of care

Study Overview

• Status: Completed
• Conditions: Optic Neuritis
• Intervention / Treatment: Drug: OCS-05 +SoC (corticosteroid) IV administration; Other: Placebo + SoC (corticosteroid) IV administration

Detailed Description

ACUITY is a phase 2, multicentric, two-arm, randomized, double-blind, placebo-controlled study to evaluate the safety and tolerability of OCS-05 compared to placebo in patients with acute optic neuritis (AON) receiving the standard of care. Patients received OCS-05 2mg/kg/day, 3mg/kg/day or placebo for five days in addition corticosteroid standard of care (SoC).

• Study Type: Interventional
• Enrollment (Actual): 36
• Phase: Phase 2

Contacts and Locations

Study Locations

• France
  • Lyon, France, 69677
    • Hospices Civils de Lyon
  • Nice, France, 06000
    • CHU - Nice
  • Paris, France, 75013
    • CIC Neurosciences - La Pitié Salpêtrière
  • Paris, France, 75019
    • Foundation Rothschild

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 60 years (Adult)
• Accepts Healthy Volunteers: No

Description

Main Inclusion Criteria:

• Diagnosed with a unilateral optic neuritis
• Onset of visual loss symptoms in the last 12 days before randomization

Main Exclusion Criteria:

• Optic neuropathy of non-demyelinating origin
• Known Neuromyelitis optica with autoantibodies against aquaporin-4 (AQP4-Abs)
• Patients with widespread and symmetric white matter alterations in the screening MRI suggestive of other demyelinating disorders (e.g. metabolic disorders, mitochondrial disorders)
• Active, chronic disease (or stable but treated with immune therapy) of the immune system other than Multiple Sclerosis (MS) or Myelin Oligodendrocyte Glycoprotein Antibody associated Disorder (MOGAD)(e.g. Sjögren's disease, systemic lupus erythematosus) or with a known immunodeficiency syndrome (AIDS, hereditary immune deficiency, drug induced immune deficiency)
• An alternative cause of visual loss (e.g. compressive or infiltrative lesion of the optic nerve, infections, genetic forms of visual loss.
• Diagnosed with macular edema, severe myopia (>6 δ) or other disease of the retina at inclusion
• Known diabetic retinopathy
• Known glaucoma
• Female patients of child-bearing potential who are unwilling to use an effective contraception while enrolled on study and for the duration of the study.
• Male patients not willing to use contraception (abstinence, condom etc..) while enrolled in the study and receiving the experimental drug, and for at least 2 days after the last experimental drug administration.
• Breastfeeding or pregnant women

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: OCS-05 +SoC (corticosteroid IV); Once daily IV infusions of OCS-05 + SoC (corticosteroid) (n=18) for 5 consecutive days	Drug: OCS-05 +SoC (corticosteroid) IV administration; OCS-05 + SoC (corticosteroid) IV administration for 5 consecutive days
Placebo Comparator: Placebo +SoC (corticosteroid IV); Once daily IV infusions of Placebo + SoC (corticosteroid) (n=18) for 5 consecutive days	Other: Placebo + SoC (corticosteroid) IV administration; Placebo + SoC (corticosteroid) IV administration for 5 consecutive days

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of patients with shift from normal (baseline) to abnormal in ECG parameters.	To determine the shift from normal to abnormal ECG parameters.	From Day 1 (V3-t1 after investigational drug administration) to Day 15 (V4)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To summarize the Visual Evoked Potential latency and amplitude and the change from baseline of the affected eye to each time point (M3 and M6) by treatment group and OCS-05 pooled group	Change in electrophysiological parameters in the affected eye as compared to baseline	Up to 6 months
To summarize the EDSS (Expanded Disability Status Scale) scores and the change from baseline to each time point (M1, M3 and M6) by treatment group and OCS-05 pooled group	Change in neurological parameters in the affected eye as compared to baseline	Up to 6 months
To describe the Cmax of OCS-05 for patients having the full PK scheme and for patients having the single PK point at 1.5h post infusion on Day 1	Characterize the PK profile of OCS-05 3mg/kg	Day 1
To describe the Tmax of OCS-05 for patients having the full PK scheme and for patients having the single PK point at 1.5h post infusion on Day 1	Characterize the PK profile of OCS-05 3mg/kg	Day 1
To describe the AUC0-t of OCS-05 for patients having the full PK scheme and for patients having the single PK point at 1.5h post infusion on Day 1	Characterize the PK profile of OCS-05 3mg/kg	Day 1
Describe the Ganglion Cell and Inner Plexiform Layer (GCIPL) thickness, absolute and relative change from baseline (of the affected eye) to each time point (t5, M1, M3, M6)	To determine the change in retinal layers thickness from baseline in the affected eye	Up to 6 months
Describe the Retinal Nerve Fiber Layer (RNFL) thickness, absolute and relative change from baseline (of the affected eye) to each time point (t5, M1, M3, M6).	To determine the change in retinal layers thickness from baseline in the affected eye.	Up to 6 months
To describe the visual function on the 2.5% ETDRS Low Contrast Letter Acuity (LCVA) chart change from baseline (of the affected eye) to each time point (D15, M1, M3, M6)	To determine change in clinical vision parameters in the affected eye as compared to baseline.	Up to 6 months
To describe the visual function on the 2.5% ETDRS High Contrast Letter Acuity (HCVA) chart change from baseline (of the affected eye) to each time point (D15, M1, M3, M6)	Change in clinical vision parameters in the affected eye as compared to baseline	Up to 6 months
To describe the visual function on the Humphrey visual fields evaluations change from baseline (of the affected eye) to each time point (M1, M3, M6)	Change in clinical vision parameters in the affected eye as compared to baseline	Up to 6 months
To describe the rate of treatment switch at 6 months for subjects receiving Disease Modifying Therapy (DMT) for multiple sclerosis	Change in rate of treatment switch for subjects receiving Disease Modifying Therapy (DMT) for multiple sclerosis	6 months
To summarize the incidence of safety parameters including clinically notable laboratory abnormalities	Change in safety laboratory parameters as compared to baseline	Up to 6 months

Collaborators and Investigators

• Sponsor: Oculis
• Collaborators: Neurotrials
• Investigators: Study Director: Sharon Klier, MD, MPH, Oculis

Publications and helpful links

General Publications

• Villoslada P, Masso M, Paris S, Hutchings S, Koch A. A Phase 1 randomized study on the safety and pharmacokinetics of OCS-05, a neuroprotective disease modifying treatment for Acute Optic Neuritis and Multiple Sclerosis. Sci Rep. 2023 Mar 29;13(1):5099. doi: 10.1038/s41598-023-32278-0.

Study record dates

Study Major Dates

• Study Start (Actual): February 10, 2021
• Primary Completion (Actual): September 16, 2024
• Study Completion (Actual): September 16, 2024

Study Registration Dates

• First Submitted: February 17, 2021
• First Submitted That Met QC Criteria: February 17, 2021
• First Posted (Actual): February 21, 2021

Study Record Updates

• Last Update Posted (Actual): September 22, 2025
• Last Update Submitted That Met QC Criteria: September 16, 2025
• Last Verified: September 1, 2025

More Information

Terms related to this study

• Keywords: Optic Neuritis; Acute Optic Neuritis (AON)
• Additional Relevant MeSH Terms: Nervous System Diseases; Eye Diseases; Optic Nerve Diseases; Cranial Nerve Diseases; Optic Neuritis; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Health Services Administration; Adrenal Cortex Hormones; Organization and Administration
• Other Study ID Numbers: OCS-05_P2_01; 2020-003147-29 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No