Amyotrophic Lateral SclerosisPhase 2NCT05299658
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
A Phase 2 study testing CNMAu8 in people with Amyotrophic Lateral Sclerosis, run by Clene Nanomedicine at 2 sites in Australia.
Trial summary
This is an optional open-label extension to participants that have completed the clinical trial CNMAu8.205. Read more about this study
- Who is it for?
- Ages 30 to 80 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- CNMAu8
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 2 sitesAustralia
- Study dates
- 13 Nov 2021to Aug 2026
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 30 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
2 sites in 1 country.
Australia2 sites
Concord Hospital
Concord, New South Wales, 2139
Neuroscience Research Australia
Randwick, New South Wales, 2031
About this study
Official title: 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
This is an optional open-label extension to participants that have completed the clinical trial CNMAu8.205.
Read the 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
- Being tested
- CNMAu8
- Keywords
- ALS, Open-label extension
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CNMAu8Drug
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.
| Study group | Role | What they receive |
|---|---|---|
| Open label extension | Experimental | This is an unblinded open-label extension, all participants will be on active drug. Receives: Drug: CNMAu8 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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).
Measured: Up to an estimated 96 weeks.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Mar 2022
- Study start13 Nov 2021
- Primary completionJun 2026 (estimated)
- Study completionAug 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Open-label extension
- Masking
- None (open label)
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Clene Nanomedicine
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05299658
- Sponsor's protocol ID
- CNMAu8.205 OLE
- Status last verified
- Dec 2025
- First submitted
- 8 Mar 2022
- Last updated
- 19 Dec 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
- Expanded access
- Available
Publications
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.
PubMed 37396808
Study at a glance
From the registry record, last updated by the sponsor on 19 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT05299658
- Acronym / sponsor ID
- CNMAu8.205 OLE
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- ALS, Open-label extension
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Clene NanomedicineIndustry (pharma, biotech, devices)
- Study start
- 13 Nov 2021 (actual)
- Primary completion
- Jun 2026 (estimated)
- Study completion
- Aug 2026 (estimated)
- First posted
- 29 Mar 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Open-label extension
| Arm | Type | Receives |
|---|---|---|
| Open label extension | Experimental | Drug: CNMAu8This is an unblinded open-label extension, all participants will be on active drug. |
Who may qualify
- Ages
- 30 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CNMAu8CNM-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. | Drug | Open label extension |
Primary outcome
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).
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Dec 2025.
- Derived from this study: PubMed 37396808Vucic 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.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing CNMAu8 in people with Amyotrophic Lateral Sclerosis, run by Clene Nanomedicine at 2 sites in Australia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 19 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety measures (time frame: Up to an estimated 96 weeks.). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05299658, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Dec 2025.