ACUITY: ACT-01 in Patients With Acute Optic Neuritis

Sponsor

Accure Therapeutics (Industry)

Overall Status

Recruiting

CT.gov ID

NCT04762017

Collaborator

Neurotrials (Other)

Enrollment

Location

Arms

37.6

Anticipated Duration (Months)

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

To evaluate the safety and tolerability of ACT-01 compared to placebo in patients with acute optic neuritis (AON) receiving the standard of care.

Condition or Disease	Intervention/Treatment	Phase
Optic Neuritis Optic; Neuritis, With Demyelination	Drug: ACT-01 IV administration Other: Placebo IV administration	Phase 2

Detailed Description

ACUITY is a phase 2a, monocentric, two-arm, randomised, double-blind, placebo-controlled study to evaluate the safety and tolerability of ACT-01 compared to placebo in patients with acute optic neuritis (AON) receiving the standard of care. Thirty-six eligible subjects aged 18 to 60, with recent onset (visual loss symptoms) of unilateral AON (idiopathic or associated with multiple sclerosis) will be randomized to receive ACT-01 or placebo

Study Design

Study Type:

Interventional

Anticipated Enrollment :

36 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Triple (Participant, Care Provider, Investigator)

Primary Purpose:

Treatment

Official Title:

A Two-arm, Randomized, Double-blind, Placebocontrolled, Monocentric Study to Evaluate the Safety and Tolerability of ACT-01 Compared to Placebo in Patients With Acute Optic Neuritis

Actual Study Start Date :

Feb 11, 2021

Anticipated Primary Completion Date :

Oct 30, 2023

Anticipated Study Completion Date :

Mar 31, 2024

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Multidose ACT-01 Once daily IV infusions of ACT-01 (n=18) for 5 consecutive days	Drug: ACT-01 IV administration Multiple Dose of ACT-01 IV administration for 5 consecutive days
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

Arm

Intervention/Treatment

Experimental: Multidose ACT-01

Once daily IV infusions of ACT-01 (n=18) for 5 consecutive days

Drug: ACT-01 IV administration

Multiple Dose of ACT-01 IV administration for 5 consecutive days

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

Outcome Measures

Primary Outcome Measures

Safety and tolerability: adverse events [up to 6 months]
To determine if ACT-01 treatment is associated within increase of adverse event

Secondary Outcome Measures

Neuroimaging [up to 12 months]
Change in ganglion cell and inner plexiform layer thickness in the affected eye measured with optical coherence tomography (OCT) compared to baseline in the affected eye
Neuroimaging [up to 12 months]
Change in Retinal Nerve Fibre Layer thickness in the affected eye measured with OCT compared to baseline in the affected eye
Visual Acuity [Up to 12 months]
Change on the 2.5% Early Treatment Diabetic retinopathy study (ETDRS) Low Contrast Letter Acuity chart and ETDRS high contrast visual acuity measured in the affected eye compared to baseline

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 60 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Main Inclusion Criteria:

Diagnosed with a unilateral acute optic neuritis with a demyelinating origin
Onset of visual loss symptoms in the last 10 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 MOG antibody associated disorder (MOGAD) or Multiple Sclerosis (MS) (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 oedema, 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