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

NCT ID: NCT05299658

Last Updated: 2025-12-19

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 40 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-11-13
• Study Completion Date: 2026-08-31

Brief Summary

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

Detailed Description

A forty-eight (48) week optional open-label extension period (Open- Label Period), which may be extended by 12-week increments until discontinued by the Sponsor for participants that have completed protocol CNMAu8.205.

Conditions

Amyotrophic Lateral Sclerosis

Keywords

ALS; Open-label extension

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Open label extension

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

Group Type: EXPERIMENTAL

CNMAu8

Intervention Type: 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.

Interventions

CNMAu8

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.

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. Participants must have completed the randomized placebo-controlled Treatment Period without compliance issues.

2. Able to understand and give written informed consent to participate in the open-label extension.

3. 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.

Exclusion Criteria

1. Lack of treatment compliance during the randomized placebo controlled Treatment Period.

2. 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.

3. Based on the investigator's judgment, patients who may have difficulty complying with the protocol and/or any study procedures.

4. 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.

5. 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.

6. 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.

• Minimum Eligible Age: 30 Years
• Maximum Eligible Age: 80 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Clene Nanomedicine

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Concord Hospital

Concord, New South Wales, Australia

Neuroscience Research Australia

Randwick, New South Wales, Australia

Countries

Australia

References

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.

Reference Type: DERIVED

PMID: 37396808 (View on PubMed)

Other Identifiers

CNMAu8.205 OLE

Identifier Type: -

Identifier Source: org_study_id