Therapeutic Nanocatalysis to Slow Disease Progression of Amyotrophic Lateral Sclerosis (ALS)

• Register: ClinicalTrials.gov
• Last refreshed on: 21 March 2022
• Main ID: NCT04098406
• Date of registration: 19/09/2019
• Prospective Registration: Yes
• Primary sponsor: Clene Nanomedicine
• Public title: Therapeutic Nanocatalysis to Slow Disease Progression of Amyotrophic Lateral Sclerosis (ALS) RESCUE-ALS
• Scientific title: A Phase 2, Randomized, 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.
• Date of first enrolment: December 19, 2019
• Target sample size: 45
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT04098406
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

Australia

Contacts

• Name: Parvarthi Menon, PhD, MD, MBBS
• Affiliation: Westmead Hospital

• Name: William Huynh, MD
• Affiliation: University of Sydney, Brain and Mind Centre

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Able to understand and give written informed consent.

2. Male or female patients aged 30 years or greater (inclusive) and less than 80 years of
age at the time of Screening.

3. Patients whose conditions are defined as possible or probable or definite ALS per the
diagnostic criteria by Awaji-Shima criteria as determined by a neurologist sub-
specialising in ALS (e.g., the Principal Investigator by study site).

4. For patients taking riluzole, stable dosing of riluzole over the prior 30-days from
Screening.

5. At the time of Screening either disease duration less than or equal to 24-months from
symptom onset, or disease duration less than or equal to 12-months from diagnosis.

6. Forced vital capacity (FVC) 60% of predicted value as adjusted for gender, height, and
age at the Screening Visit.

7. Patient who has established care with a neurologist at one of the specialized ALS
clinics involved in the study and will maintain this clinical care throughout the
study. If a patient is referred from a third party (neurologist or a State based ALS
organization) they must be willing to transfer care to the neurologist participating
in the study.

Following completion of the 36-week randomized placebo controlled treatment period,
interested participants must meet the following inclusion criteria to enroll in the
open-label extension:

1. Participants must have completed the randomized placebo controlled Treatment Period
without compliance issues

2. Able to understand and give written informed consent to participant in the open-label
extension.

3. If referred from a third party (neurologist or a State based ALS organization),
participant agrees to maintain transfer of care to a neurologist participating in the
study.

Exclusion Criteria:

1. Patients will be excluded from the study if they meet any of the following criteria:

2. At Screening patients who utilize, or in the Investigator's judgment will be
imminently dependent upon:

1. Non-invasive ventilation > 22 hours per day, or

2. Tracheostomy Note: If the patient requires non-invasive ventilation
postrandomisation, they will be allowed to continue in the study.

3. Patients with Familial ALS (e.g., 2 or more family members with ALS or motor neuron
disease)

4. Patients with a history of carpal tunnel syndrome, polyneuropathy, or in the
investigators judgement diseases that could induce polyneuropathy and interfere with
electromyography (EMG) recordings.

5. Patients with too severe atrophy of the Abductor Digiti Minimi (ADM), Abductor
Pollicis Brevis (APB), Biceps Brachii (BB), or Tibialis Anterior (TA) muscles in the
least clinically affected hand and leg, respectively, to allow for reliable EMG
recordings.

6. Patient with a history of significant other major medical conditions based on the
Investigator's judgment.

7. Based on the investigator's judgment, patients who may have difficulty complying with
the protocol and/or any study procedures.

8. Patient with clinically significant abnormalities in haematology, blood chemistry,
ECG, or physical examination not resolved by the Baseline visit which according to
Investigator can interfere with study participation.

9. 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 Screening.

10. Patient participating in any other investigational drug trial or using investigational
drug (within 12 weeks prior to screening and thereafter).

11. Females who are pregnant or nursing or who plan to get pregnant during the course of
this clinical trial or within 6 months of the end of this trial.

12. Females of child-bearing potential, or men, who are unwilling or unable to use
accepted methods of birth control.

13. Active inflammatory condition or autoimmune disorder.

14. Positive screen for drugs of abuse.

15. History of gold allergy.

16. 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.

Following completion of the 36-week randomized placebo controlled treatment period,
interested participants will be excluded from participating in the open-label extension
phase if they meet any of the following 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 hematology, 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 109 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.



Age minimum: 30 Years
Age maximum: 80 Years
Gender: All

Health Condition(s) or Problem(s) studied

Amyotrophic Lateral Sclerosis

Intervention(s)

Drug: CNM-Au8

Drug: Placebo

Primary Outcome(s)

Electromyography measures of disease progression. [Time Frame: 36 weeks]

Secondary Outcome(s)

Mean absolute change of the average difference between active treatment and placebo from Baseline through Week 36 for the MUNIXscore(4). [Time Frame: 36 weeks]

Mean change in the average difference between active treatment and placebo from Baseline for respiratory function as measured by forced vital capacity (FVC). [Time Frame: 36 weeks]

Secondary ID(s)

CNMAu8.205

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Clene Australia Pty Ltd