An Open-Label Extension for the Phase 2 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

NCT05299658 | Active Not Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: CNMAu8.205 OLE
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 2

Brief Summary

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

Conditions

Amyotrophic Lateral Sclerosis

Keywords

ALS; Open-label extension

Outcome Measures

Primary Outcomes (2)

• Safety measures

  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.

  Up to an estimated 96 weeks.

• Electromyography measures

  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).

  Up to an estimated 96 weeks.

Other Outcomes (4)

• MScanFit MUNE

  Up to an estimated 96 weeks.

• MUSIX

  Up to an estimated 96 weeks.

• Spilt Hand Index

  Up to an estimated 96 weeks.

Study Arms (1)

Open label extension

EXPERIMENTAL

This is an unblinded open-label extension, all participants will be on active drug.

Drug: CNMAu8

Interventions

CNMAu8 DRUG

CNM-Au8 is an aqueous suspension of clean surfaced faceted nanocrystals consisting of gold atoms self-organized into crystals of various geometrical shapes (hexagonal bi-pyramid, pentagonal bi-pyramid, tetrahedron, decahedron, planar spheroids). Those choosing to participate in the OLE period will orally receive 30 mg of CNM-Au8, once daily.

Open label extension

Eligibility Criteria

• Age: 30 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Participants must have completed the randomized placebo-controlled Treatment Period without compliance issues.
• Able to understand and give written informed consent to participate in the open-label extension.
• If referred from a third party (neurologist or a State based ALS organisation), participant agrees to maintain transfer of care to a neurologist participating in the study.

You may not qualify if:

• Lack of treatment compliance during the randomized placebo controlled Treatment Period.
• Positive pregnancy test at the Week 36 visit, or, females who plan to get pregnant during the course of this extension or within 6 months of the end of this extension.
• Based on the investigator's judgment, patients who may have difficulty complying with the protocol and/or any study procedures.
• Patient with clinically significant abnormalities in haematology, blood chemistry, ECG, or physical examination identified during the W36 visit which according to Investigator may interfere with continued participation.
• Patients with clinically significant hepatic or renal dysfunction or clinical laboratory findings that would limit the interpretability of change in liver or kidney function, or those with low platelet counts (\< 150 x 10\^9 per liter) or eosinophilia (absolute eosinophil count of ≥ 500 eosinophils per microliter) at the Week 36 visit.
• Patient is considered a suicide risk in the opinion of the Investigator, has previously made a suicide attempt, or is currently demonstrating active suicidal ideation. Subjects with intermittent passive suicidal ideation are not necessarily excluded based on the assessment of the Investigator.

Sponsors & Collaborators

• Clene Nanomedicine: lead

Study Sites (2)

Concord Hospital

Concord, New South Wales, 2139, Australia

Location

Neuroscience Research Australia

Randwick, New South Wales, 2031, Australia

Location

Related Publications (1)

• 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.

  PMID: 37396808 DERIVED

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR
• Expanded Access: Yes

Model Details: Open-label extension

Study Record Dates

• First Submitted: March 8, 2022
• First Posted: March 29, 2022
• Study Start: November 13, 2021
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): August 1, 2026
• Last Updated: December 19, 2025

Record last verified: 2025-12

Locations

AU (2)