OCS-05 in Patients With Acute Optic Neuritis

Study Overview
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Brief Summary
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To evaluate the safety and tolerability of OCS-05 compared to placebo in patients with acute optic neuritis (AON) receiving the standard of care

Detailed Description
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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.

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

Conditions
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Optic Neuritis, Optic; Neuritis, With Demyelination

Intervention / Treatment
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* Drug: OCS-05 IV administration
* Other: Placebo IV administration


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 60 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: OCS-05<br>Once daily IV infusions of OCS-05 (n=18) for 5 consecutive days | Drug: OCS-05 IV administration<br>* Multiple Dose of OCS-05 IV administration for 5 consecutive days<br>|
| Placebo Comparator: Placebo<br>Once daily IV infusions of Placebo (n=18) for 5 consecutive days | Other: Placebo IV administration<br>* Placebo IV administration for 5 consecutive days<br>|

What is the study measuring?
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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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Acute Optic Neuritis (AON), Demyelination, Optic Neuritis