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

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

Detailed Description
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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.

Official Title
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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

Conditions
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Amyotrophic Lateral Sclerosis

Intervention / Treatment
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* Drug: CNMAu8


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 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. Exclusion Criteria: 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.

Ages Eligible for Study
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Minimum Age: 30 Years
Maximum Age: 80 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Interventional Model Description: Open-label extension
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Open label extension<br>This is an unblinded open-label extension, all participants will be on active drug. | Drug: CNMAu8<br>* 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.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | 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. | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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ALS, Open-label extension