OCS-05 in Patients With Acute 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 acute optic neuritis (AON) receiving the standard of care

Study Overview

• Status: Recruiting
• Conditions: Optic Neuritis; Optic; Neuritis, With Demyelination
• Intervention / Treatment: Other: Placebo IV administration; Drug: OCS-05 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. Fourty-two (42) eligible patients aged 18 to 60, with recent onset (visual loss symptoms) of unilateral AON (idiopathic or associated with multiple sclerosis) will be randomized to receive OCS-05 or placebo.

• Study Type: Interventional
• Enrollment (Anticipated): 42
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Acuity Study Team; Phone Number: +1 617 928 5886; Email: info@oculis.com

Study Locations

• France
  • Lyon, France, 69677
    • Recruiting
    • Hospices Civils de Lyon
  • Nice, France, 06000
    • Recruiting
    • CHU - Nice
  • Paris, France, 75013
    • Recruiting
    • CIC Neurosciences - La Pitié Salpêtrière
  • Paris, France, 75019
    • Recruiting
    • 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 acute optic neuritis with a demyelinating origin
• 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
Placebo Comparator: Placebo; Once daily IV infusions of Placebo (n=18) for 5 consecutive days	Other: Placebo IV administration; Placebo IV administration for 5 consecutive days
Experimental: OCS-05; Once daily IV infusions of OCS-05 (n=18) for 5 consecutive days	Drug: OCS-05 IV administration; Multiple Dose of OCS-05 IV administration for 5 consecutive days

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of patients with treatment-related adverse events (frequencies and percentages tabulated by treatment group)	To determine if OCS-05 treatment is associated within increase of adverse event	up to 6 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Describe the change in 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) by treatment group and OCS-05 pooled group	To determine the change in retinal layers thickness as compared to baseline in the affected eye	up to 6 months
To describe the change in visual function (high to low contrast visual acuity and Humphrey visual fields) from baseline to each time point (M1, M3, M6) by treatment group and OCS-05 pooled group	Change in clinical vision parameters in the affected eye as compared to baseline	up to 6 months
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 summarize the incidence of clinically notable laboratory abnormalities	Change in safety laboratory parameters 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

Collaborators and Investigators

• Sponsor: Oculis
• Collaborators: Neurotrials
• Investigators: Study Director: Joanne Chang, MD, Oculis

Study record dates

Study Major Dates

• Study Start (Actual): February 11, 2021
• Primary Completion (Anticipated): October 30, 2023
• Study Completion (Anticipated): March 31, 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 (Estimate): May 11, 2023
• Last Update Submitted That Met QC Criteria: May 9, 2023
• Last Verified: May 1, 2023

More Information

Terms related to this study

• Keywords: Optic Neuritis; Acute Optic Neuritis (AON); Demyelination
• Additional Relevant MeSH Terms: Nervous System Diseases; Eye Diseases; Neuromuscular Diseases; Peripheral Nervous System Diseases; Optic Nerve Diseases; Cranial Nerve Diseases; Neuritis; Optic Neuritis; Demyelinating Diseases
• 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