Therapeutic Nanocatalysis to Slow Disease Progression of Amyotrophic Lateral Sclerosis (ALS)

Summary

The objective of this trial was to assess the efficacy, safety, and PK/PD effects of CNM-Au8 as a disease-modifying agent for the treatment of ALS by utilizing electrophysiological measures to detect preservation of motor neuron function. The primary endpoint was the mean change in the average difference between active treatment and placebo from Baseline through Week 36 evaluated by electromyography.

Timeline

Start

2019-12-19

Primary completion

2021-07-13

Completion

2021-07-13

Outcome

Missed primary endpoint

paper there was no significant difference in the summated MUNIX score percent change PMID 37396808 ↗

paper While the primary and secondary outcomes of this trial were not significant PMID 37396808 ↗

Publications

• Results Vucic S, Menon P, Huynh W, Mahoney C, Ho KS, Hartford A, Rynders A, Evan J, Evan J, Ligozio S, Glanzman R, Hotchkin MT, Kiernan MC. Efficacy and safety of CNM-Au8 in amyotrophic lateral sclerosis (RESCUE-ALS study): a phase 2, randomised, double-blind, placebo-controlled trial and open label extension. EClinicalMedicine. 2023 Jun 8;60:102036. doi: 10.1016/j.eclinm.2023.102036. eCollection 2023 Jun.
• Saha S, Tripathy S, Patra CR. Neuritogenic activity of metal nanoparticles. Nanomedicine (Lond). 2024 Feb;19(5):363-366. doi: 10.2217/nnm-2023-0297. Epub 2024 Jan 12. No abstract available.
• Vucic S, Kiernan MC, Menon P, Huynh W, Rynders A, Ho KS, Glanzman R, Hotchkin MT. Study protocol of RESCUE-ALS: A Phase 2, randomised, double-blind, placebo-controlled study in early symptomatic amyotrophic lateral sclerosis patients to assess bioenergetic catalysis with CNM-Au8 as a mechanism to slow disease progression. BMJ Open. 2021 Jan 11;11(1):e041479. doi: 10.1136/bmjopen-2020-041479.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	CNM-Au8	Other / unclassified	30 mg	Oral

Indications