OCS-05 in Patients With Acute Optic Neuritis (ACUITY)

• ClinicalTrials.gov Identifier: NCT04762017
• Recruitment Status: Recruiting
• First Posted: February 21, 2021
• Last Update Posted: May 11, 2023
• Sponsor: Oculis
• Collaborator: Neurotrials
• Information provided by (Responsible Party): Oculis

Tracking Information

• First Submitted Date: February 17, 2021
• First Posted Date: February 21, 2021
• Last Update Posted Date: May 11, 2023
• Actual Study Start Date: February 11, 2021
• Estimated Primary Completion Date: October 30, 2023 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: March 6, 2023)

Number of patients with treatment-related adverse events (frequencies and percentages tabulated by treatment group) [ Time Frame: up to 6 months ]

To determine if OCS-05 treatment is associated within increase of adverse event

Original Primary Outcome Measures (submitted: February 17, 2021)

Safety and tolerability: adverse events [ Time Frame: up to 6 months ]

To determine if ACT-01 treatment is associated within increase of adverse event

Current Secondary Outcome Measures (submitted: March 6, 2023)

• 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 [ Time Frame: up to 6 months ]
  To determine the change in retinal layers thickness as compared to baseline in the affected eye
• 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 [ Time Frame: up to 6 months ]
  Change in clinical vision parameters in the affected eye as compared to baseline
• 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 [ Time Frame: Up to 6 months ]
  Change in electrophysiological parameters in the affected eye as compared to baseline
• 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 [ Time Frame: Up to 6 months ]
  Change in neurological parameters in the affected eye as compared to baseline
• To summarize the incidence of clinically notable laboratory abnormalities [ Time Frame: Up to 6 months ]
  Change in safety laboratory parameters as compared to baseline
• 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 [ Time Frame: Day 1 ]
  Characterize the PK profile of OCS-05 3mg/kg
• 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 [ Time Frame: Day 1 ]
  Characterize the PK profile of OCS-05 3mg/kg
• 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 [ Time Frame: Day 1 ]
  Characterize the PK profile of OCS-05 3mg/kg

Original Secondary Outcome Measures (submitted: February 17, 2021)

• 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
• 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

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: 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
• 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. 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
• Study Phase: Phase 2
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Triple (Participant, Care Provider, Investigator); Primary Purpose: Treatment

Condition

• Optic Neuritis
• Optic; Neuritis, With Demyelination

Intervention

• Drug: OCS-05 IV administration
  Multiple Dose of OCS-05 IV administration for 5 consecutive days
• Other: Placebo IV administration
  Placebo IV administration for 5 consecutive days

Study Arms

• Experimental: OCS-05
  Once daily IV infusions of OCS-05 (n=18) for 5 consecutive days
  Intervention: Drug: OCS-05 IV administration
• Placebo Comparator: Placebo
  Once daily IV infusions of Placebo (n=18) for 5 consecutive days
  Intervention: Other: Placebo IV administration

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: March 6, 2023): 42
• Original Estimated Enrollment (submitted: February 17, 2021): 36
• Estimated Study Completion Date: March 31, 2024
• Estimated Primary Completion Date: October 30, 2023 (Final data collection date for primary outcome measure)

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years to 60 Years (Adult)
• Accepts Healthy Volunteers: No

Contacts

Contact: Acuity Study Team; +1 617 928 5886; info@oculis.com

• Listed Location Countries: France

Administrative Information

• NCT Number: NCT04762017
• Other Study ID Numbers: OCS-05_P2_01; 2020-003147-29 ( EudraCT Number )
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

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

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: Oculis
• Original Responsible Party: Accure Therapeutics
• Current Study Sponsor: Oculis
• Original Study Sponsor: Accure Therapeutics
• Collaborators: Neurotrials

Investigators

• Study Director: Joanne Chang, MD; Oculis

• PRS Account: Oculis
• Verification Date: May 2023