NCT05299658

An Open-Label Extension for the Phase 2, Randomised, Double-Blind, Placebo-Controlled Study in Early Symptomatic Amyotrophic Lateral Sclerosis Patients on Stable Background Therapy to Assess Bioenergetic Catalysis With CNM-Au8 to Slow Disease Progression in ALS

Active, enrolled Phase 2 Last updated 17 December 2025

What this trial tests

Phase 2 trial testing CNMAu8 in Amyotrophic Lateral Sclerosis in 40 participants. Participants enrolled and being followed up; not accepting new ones.

Timeline

13 November 2021

Primary endpoint
1 June 2026

1 August 2026

Quick facts

Lead sponsor	Clene Nanomedicine
Phase	Phase 2
Status	Active, enrolled
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	40
Start date	13 November 2021
Primary completion	1 June 2026
Estimated completion	1 August 2026
Sites	2 locations across Australia

Drugs / interventions tested

• CNMAu8 — full drug profile →

Conditions studied

• Amyotrophic Lateral Sclerosis — all drugs for Amyotrophic Lateral Sclerosis →

Sponsor

Clene Nanomedicine — full company profile →

Who can join

Adults 30 to 80, any sex, with Amyotrophic Lateral Sclerosis. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Safety measures
  Time frame: Up to an estimated 96 weeks.
  Safety endpoints include incidence of treatment-emergent AEs, drug-related AEs, deaths, SAEs, and AEs leading to discontinuation from the study. Changes from baseline (Week 36 of the placebo controlled phase) in clinical laboratory results, physical examination findings, vital signs, ECGs, and C-SSRS will be summarized descriptively by group and timepoint.
• Electromyography measures
  Time frame: Up to an estimated 96 weeks.
  Mean change in the average difference between active treatment and placebo from Baseline through End of Study for the MUNIXscore(4), which is the sum of the respective MUNIX values for the Abductor Digiti Minimi (ADM), Abductor Pollicis Brevis (APB), Biceps Brachii (BB), and Tibialis Anterior (TA).

Sponsor's own description

This is an optional open-label extension to participants that have completed the clinical trial CNMAu8.205.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Clinical translation of gold nanoparticles.
   Zhang R, Kiessling F, Lammers T, Pallares RM. · · 2023 · cited 108× · PMID 36045273 · DOI 10.1007/s13346-022-01232-4
2. 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.
   Vucic S, Menon P, Huynh W, Mahoney C, et al · · 2023 · cited 40× · PMID 37396808 · DOI 10.1016/j.eclinm.2023.102036
3. Gold nanostructures in melanoma: Advances in treatment, diagnosis, and theranostic applications.
   Dastgheib ZS, Abolmaali SS, Farahavar G, Salmanpour M, et al · · 2024 · cited 14× · PMID 39170173 · DOI 10.1016/j.heliyon.2024.e35655
4. Unlocking the Potential of Gold as Nanomedicine in Cancer Immunotherapy.
   Kumar PPP, Lee M, Kim T. · · 2024 · cited 3× · PMID 40808797 · DOI 10.3390/jnt5020003
5. Amyotrophic Lateral Sclerosis: Pathophysiological Mechanisms and Treatment Strategies (Part 2).
   Tolochko C, Shiryaeva O, Alekseeva T, Dyachuk V. · · 2025 · cited 2× · PMID 40508048 · DOI 10.3390/ijms26115240
6. Latest progress and challenges in drug development for degenerative motor neuron diseases.
   Wen X, Lan T, Su W, Cao B, et al · · 2026 · cited 2× · PMID 40364643 · DOI 10.4103/nrr.nrr-d-24-01266
7. Advances in Gold Nanoparticles for the Diagnosis and Management of Alzheimer's Disease.
   Sivamaruthi BS, Kesika P, Sisubalan N, Chaiyasut C. · · 2025 · PMID 41012495 · DOI 10.3390/pharmaceutics17091158
8. Programmable self-replicating JEV nanotherapeutics redefine RNA delivery in ALS.
   Loo YS, Yusoh NA, Yap K, Ng CS. · · 2025 · PMID 40858858 · DOI 10.1038/s42003-025-08579-7