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

Amyotrophic Lateral Sclerosis Clinical Trial

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05299658
• Other study ID #: CNMAu8.205 OLE
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: November 13, 2021
• Est. completion date: December 2024

Study information

• Verified date: May 2024
• Source: Clene Nanomedicine
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 40
• Est. completion date: December 2024
• Est. primary completion date: November 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 30 Years to 80 Years

Eligibility

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.

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Disease Progression
• Motor Neuron Disease
• Sclerosis

Intervention

Drug:
• 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.

Locations

Country	Name	City	State
Australia	Concord Hospital	Concord	New South Wales
Australia	Neuroscience Research Australia	Randwick	New South Wales

Sponsors (1)

Lead Sponsor	Collaborator
Clene Nanomedicine

Country where clinical trial is conducted

Australia, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	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%.	Up to an estimated 96 weeks.
Other	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.
Other	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.
Other	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.
Primary	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.
Primary	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.