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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04098406




Registration number
NCT04098406
Ethics application status
Date submitted
19/09/2019
Date registered
23/09/2019

Titles & IDs
Public title
Therapeutic Nanocatalysis to Slow Disease Progression of Amyotrophic Lateral Sclerosis (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.
Secondary ID [1] 0 0
CNMAu8.205
Universal Trial Number (UTN)
Trial acronym
RESCUE-ALS
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Amyotrophic Lateral Sclerosis 0 0
Condition category
Condition code
Neurological 0 0 0 0
Neurodegenerative diseases
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional(has expanded access)
Description of intervention(s) / exposure
Treatment: Drugs - CNM-Au8
Treatment: Drugs - Placebo

Placebo comparator: Placebo - The matched placebo used in this study consisted of water, sodium bicarbonate, and food coloring to match volume and color of the experimental treatment

Experimental: 30 mg CNM-Au8 - 30mg suspension of clean-surfaced, faceted, gold nanocrystals in 60ml of sodium bicarbonate buffered water


Treatment: Drugs: CNM-Au8
CNM-Au8 is a dark red/purple-colored liquid formulation consisting of a stable suspension of faceted clean surfaced elemental gold nanocrystals in buffered deionized water with a nominal concentration of 0.5 mg/mL of gold. The formulation is buffered by sodium bicarbonate present at a concentration of 0.546 mg/mL. There are no other excipients. The drug product is formulated to be taken orally and will be provided in single dose 60 mL HDPE containers.

Treatment: Drugs: Placebo
Placebo was liquid with identical color and taste
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Electromyography Measures of Disease Progression.
Timepoint [1] 0 0
36 weeks
Secondary outcome [1] 0 0
Percentage Mean Change in the Average Difference Between Active Treatment and Placebo From Baseline for Respiratory Function as Measured by Forced Vital Capacity (FVC).
Timepoint [1] 0 0
36 weeks
Secondary outcome [2] 0 0
Mean Absolute Change of the Average Difference Between Active Treatment and Placebo From Baseline Through Week 36 for the MUNIXscore(4).
Timepoint [2] 0 0
36 weeks

Eligibility
Key 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.
Minimum age
30 Years
Maximum age
80 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
19/12/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
13/07/2021
Sample size
Target
Accrual to date
Final
45
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
University of Sydney Brain and Mind Centre - Sydney
Recruitment hospital [2] 0 0
Westmead Hospital - Sydney
Recruitment postcode(s) [1] 0 0
2050 - Sydney
Recruitment postcode(s) [2] 0 0
2145 - Sydney

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Clene Nanomedicine
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
Clene Australia Pty Ltd
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Parvarthi Menon, PhD, MD, MBBS
Address 0 0
Westmead Hospital
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?




Results publications and other study-related documents

TypeCitations or Other Details
Journal Vucic S, Menon P, Huynh W, Mahoney C, Ho KS, Hartf... [More Details]


Results are available at https://clinicaltrials.gov/study/NCT04098406