Clinical trial NCT04762017: OCS-05 in Patients with Acute Optic Neuritis
Official title: A Randomized, Double-blind, Placebo-controlled Study to Evaluate the Safety and Tolerability of OCS-05 in Patients with Acute Optic Neuritis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Optic Neuritis
Optic; Neuritis, with Demyelination
Keywords: Acute Optic Neuritis (AON)
Demyelination
Optic Neuritis
Lead sponsor: Oculis
Sponsor class: INDUSTRY
Collaborators: name: Neurotrials
First posted: 2021-02-21
Enrollment: 36
Sex: ALL
Minimum age (years): 18
Maximum age (years): 60
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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: 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. The study randomized 36 eligible patients aged between 18 to 60 with recent onset (visual loss symptoms) of unilateral acute optic neuritis with a demyelinating origin, of which 33 patients received OCS-05 2mg/kg/day, 3mg/kg/day or placebo for five days in addition corticosteroid standard of care (SoC).

Eligibility criteria: 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

Primary outcomes:
measure: Percentage of patients with shift from normal (baseline) to abnormal in ECG parameters.
timeFrame: From Day 1 (V3-t1 after investigational drug administration) to Day 15 (V4)
Secondary outcomes:
measure: 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)
timeFrame: Up to 6 months
measure: 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).
timeFrame: Up to 6 months
measure: 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)
timeFrame: Up to 6 months
measure: 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)
timeFrame: Up to 6 months
measure: 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)
timeFrame: Up to 6 months
measure: 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
timeFrame: Up to 6 months
measure: 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
timeFrame: Up to 6 months
measure: To describe the rate of treatment switch at 6 months for subjects receiving Disease Modifying Therapy (DMT) for multiple sclerosis
timeFrame: 6 months
measure: To summarize the incidence of safety parameters including clinically notable laboratory abnormalities
timeFrame: Up to 6 months
measure: 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
timeFrame: Day 1
measure: 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
timeFrame: Day 1
measure: 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
timeFrame: Day 1
Other outcomes:
Locations:
city: Lyon
country: France
facility: Hospices Civils de Lyon
city: Nice
country: France
facility: CHU - Nice
city: Paris
country: France
facility: CIC Neurosciences - La Pitié Salpêtrière
city: Paris
country: France
facility: Foundation Rothschild
Study officials:
affiliation: Oculis
name: Sharon Klier, MD, MPH
role: STUDY_DIRECTOR
References: