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This is an optional open-label extension to participants that have completed the clinical trial CNMAu8.205.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Open label extension | Experimental | This is an unblinded open-label extension, all participants will be on active drug. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
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| Measure | Description | Time Frame |
|---|---|---|
| MScanFit MUNE | Mean change in the average difference between active treatment and placebo from Baseline through Week 36 (overall difference at all time points) as measured by MScanFit MUNE (Jacobsen et al., 2017) of the APB in the least clinically affected hand. The baseline average will be indexed to 100%, and changes at Week 12, 24 and Week 36 will be calculated as the percent change from the Baseline index of 100%. |
Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Concord Hospital | Concord | New South Wales | 2139 | Australia | ||
| Neuroscience Research Australia |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37396808 | Derived | 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. |
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Open-label extension
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|
| Up to an estimated 96 weeks. |
| MUSIX | Mean change in the average difference between active treatment and placebo from Baseline through Week 36 for the MUSIXscore(4) (Neuwirth et al., 2015), which is the mean of the respective MUSIX values for the ADM, APB, BB, and TA. The average baseline mean values will be indexed to 100%. Changes will be calculated as the percent change from the Baseline index of 100%. baseline average will be indexed to 100%, and changes at Week 12, 24 and Week 36 will be calculated as the percent change from the Baseline index of 100%. | Up to an estimated 96 weeks. |
| Spilt Hand Index | Mean change in the average difference between active treatment and placebo from Baseline through Week 36 for the Split Hand Index (SI) (Menon et al., 2013), defined as the first dorsal interosseous (FDI)Peak CMAP Amplitude * APBPeak CMAP Amplitude/ADMPeak CMAP Amplitude. | Up to an estimated 96 weeks. |
| Neurophysiological index | Mean change in the average difference between active treatment and placebo from Baseline through Week 36 for the Neurophysiological Index (NPI) of the ADM (Cheah et al., 2011), defined as the ulnar nerve (ADMCMAP Peak Amplitude / ADMDistal Motor Latency) * ADMF-Wave (%). | Up to an estimated 96 weeks. |
| Randwick |
| New South Wales |
| 2031 |
| Australia |
| ID | Term |
|---|---|
| D000690 | Amyotrophic Lateral Sclerosis |
| ID | Term |
|---|---|
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D016472 | Motor Neuron Disease |
| D019636 | Neurodegenerative Diseases |
| D057177 | TDP-43 Proteinopathies |
| D009468 | Neuromuscular Diseases |
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
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