Brain Mind Institute
Neurology

Author Of 1 Presentation

Clinical Trials Poster Presentation

P0243 - VISIONARY-MS: A Phase 2 clinical trial of catalytic gold nanocrystals, CNM-Au8, for the treatment of chronic optic neuropathy (ID 431)

Abstract

Background

Therapies that promote neuroprotection and remyelination are a high priority for multiple sclerosis (MS). CNM-Au8 is an orally delivered suspension of clean-surfaced, faceted gold nanocrystals. CNM-Au8 acts as a nanocatalyst, enhancing cellular bioenergetics, with robust activity in multiple preclinical models of neuroprotection and remyelination. Phase 1 studies identified 15mg and 30 mg per day as well-tolerated doses, with human exposures exceeding those demonstrating preclinical efficacy. No safety issues emerged.

Objectives

To present the rationale for, and design of, the VISIONARY-MS study (NCT03536559), provide clinical trial baseline demographics and visual characteristics, and report interim, blinded efficacy data.

Methods

VISIONARY-MS is a Phase 2, multicenter, randomized, double-blind, placebo-controlled study evaluating the efficacy, safety, and pharmacokinetics of CNM-Au8 (15 mg or 30 mg daily) versus placebo for 24 weeks in stable relapsing multiple sclerosis (RMS) patients with chronic optic neuropathy, currently receiving disease-modifying therapy (DMT). Key inclusion criteria: age 18-55 years, diagnosis of RMS, disease duration £ 15 years, no history of optic neuritis (ON) for 6 months prior to entry (+/- ON prior to that), clinically stable on an approved DMT, Best Corrected Low Contrast Letter Acuity (BC-LCLA) ≤ 20/40 in the affected eye and ≤ 20/32 in the unaffected eye [BC-LCLA ≤ Best Corrected High Contrast Letter Acuity (BC-HCVA) in both eyes], and mean retinal nerve fibre layer thickness ³ 70 mm in both eyes.

Primary endpoint: mean change in BC-LCLA, as measured by 2.5% Sloan letter chart, from baseline to week 24. Key secondary endpoint: mean change in average mf-VEP latency in the mf-VEP affected eye (greatest mf-VEP latency delay at Baseline) from baseline to Week 24.

Other exploratory outcomes: changes in mf-VEP amplitude and latency measures, retinal morphology, nerve fibre layer thickness, magnetic resonance imaging metrics [T1 Black Hole lesion volume, diffusion tensor imaging, magnetization transfer imaging (MTR) and myelin water imaging], EDSS, 25-Foot Timed Walk, Symbol Digit Modality Test, 9-Hole Peg Test and quality of life from baseline to Week 24.

Results

The study commenced in December 2018. Enrolment is ongoing at selected sites in Australia and North America. Baseline demographics and visual characteristics will be presented. Available interim, blinded efficacy data will be presented.

Conclusions

VISIONARY-MS represents the first efficacy study for a completely novel therapeutic catagory. CNM-Au8 is a nanotherapeutic agent providing bioenergetic, catalyitc support to neurons and oligodendroglia precursor cells, resultng in neuroprotection and improved remyelination in an MS population.

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