ACT-01 in Patients With Acute Optic Neuritis (ACUITY)

• ClinicalTrials.gov Identifier: NCT04762017
• Recruitment Status: Recruiting
• First Posted: February 21, 2021
• Last Update Posted: March 3, 2021
• Sponsor: Accure Therapeutics
• Collaborator: Neurotrials
• Information provided by (Responsible Party): Accure Therapeutics

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 (Clinical Trial)
• Estimated 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: February 11, 2021
• Estimated Primary Completion Date: August 25, 2021
• Estimated Study Completion Date: December 25, 2022

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

Outcome Measures

Primary Outcome Measures :

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

Secondary Outcome Measures :

1. Neuroimaging [ Time Frame: 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
2. Neuroimaging [ Time Frame: 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
3. Visual Acuity [ Time Frame: 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

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

Criteria

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

Contacts and Locations

Contacts

Contact: Jennifer Thibault, MSc; jennifer.thibault@icm-institute.org

Contact: STEPHANE PARIS, MSc; sparis@accure.health

Locations

France

CIC Neurosciences - La Pitié Salpêtrière; Recruiting

Paris, France, 75013

Sponsors and Collaborators

Accure Therapeutics

Neurotrials

Investigators

• Study Director: Rossella Medori, MD; Accure Therapeutics

More Information

• Responsible Party: Accure Therapeutics
• ClinicalTrials.gov Identifier: NCT04762017
• Other Study ID Numbers: ACT-01_P2_01; 2020-003147-29 ( EudraCT Number )
• First Posted: February 21, 2021
• Last Update Posted: March 3, 2021
• Last Verified: March 2021

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Accure Therapeutics:

Acute Optic Neuritis (AON); Demyelination; Optic Neuritis

Additional relevant MeSH terms:

Neuritis; Optic Neuritis; Demyelinating Diseases; Peripheral Nervous System Diseases; Neuromuscular Diseases; Nervous System Diseases; Optic Nerve Diseases; Cranial Nerve Diseases; Eye Diseases