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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

Primary Purpose

Amyotrophic Lateral Sclerosis

Status

Active

Phase

Phase 2

Locations

Australia

Study Type

Interventional

Intervention

CNMAu8

About this trial

This is an interventional treatment trial for Amyotrophic Lateral Sclerosis focused on measuring ALS, Open-label extension

Eligibility Criteria

30 Years - 80 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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 109 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.

Sites / Locations

Brain Mind Centre
Concord Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Open label extension

Arm Description

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

Outcomes

Primary Outcome Measures

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.

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).

Secondary Outcome Measures

Full Information

NCT ID

NCT05299658

First Posted

March 8, 2022

Last Updated

March 30, 2023

Sponsor

Clene Nanomedicine

1. Study Identification

Unique Protocol Identification Number

NCT05299658

Brief Title

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

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

Study Type

Interventional

2. Study Status

Record Verification Date

March 2023

Overall Recruitment Status

Active, not recruiting

Study Start Date

November 13, 2021 (Actual)

Primary Completion Date

November 2023 (Anticipated)

Study Completion Date

December 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Clene Nanomedicine

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Amyotrophic Lateral Sclerosis

Keywords

ALS, Open-label extension

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Model Description

Open-label extension

Masking

None (Open Label)

Allocation

N/A

Enrollment

40 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Open label extension

Arm Type

Experimental

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

CNMAu8

Intervention Description

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.

Primary Outcome Measure Information:

Title

Safety measures

Description

Time Frame

Up to an estimated 96 weeks.

Title

Electromyography measures

Description

Time Frame

Up to an estimated 96 weeks.

Other Pre-specified Outcome Measures:

Title

MScanFit MUNE

Description

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%.

Time Frame

Up to an estimated 96 weeks.

Title

MUSIX

Description

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%.

Time Frame

Up to an estimated 96 weeks.

Title

Spilt Hand Index

Description

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.

Time Frame

Up to an estimated 96 weeks.

Title

Neurophysiological index

Description

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 (%).

Time Frame

Up to an estimated 96 weeks.

10. Eligibility

Sex

All

Minimum Age & Unit of Time

30 Years

Maximum Age & Unit of Time

80 Years

Accepts Healthy Volunteers

Eligibility Criteria

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.

Facility Information:

Facility Name

Brain Mind Centre

City

Camperdown

State/Province

New South Wales

ZIP/Postal Code

2050

Country

Australia

Facility Name

Concord Hospital

City

Concord

State/Province

New South Wales

ZIP/Postal Code

2139

Country

Australia

12. IPD Sharing Statement

Plan to Share IPD

Undecided

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