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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06177431




Registration number
NCT06177431
Ethics application status
Date submitted
11/12/2023
Date registered
20/12/2023

Titles & IDs
Public title
An Open Label Extension Study of Monepantel in Individuals With Motor Neurone Disease
Scientific title
An Open Label Extension Study to Investigate the Long Term Safety, Tolerability And Efficacy of Oral Monepantel in Individuals With Motor Neurone Disease Who Previously Completed Study MON-2021-001
Secondary ID [1] 0 0
MON-2023-001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Motor Neuron Disease 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
Description of intervention(s) / exposure
Treatment: Drugs - Monepantel

Experimental: Monepantel treatment arm - Daily dose of 10 mg/kg body weight (QD)


Treatment: Drugs: Monepantel
Monepantel is provided to individuals living with ALS/MND as a white oval tablet to be administered once a day following meals
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Long-term safety and tolerability
Timepoint [1] 0 0
From Baseline to End of Treatment, up to 12 months.
Secondary outcome [1] 0 0
Treatment-related changes in serum neurofilament light (NfL) chain levels
Timepoint [1] 0 0
From Baseline to End of Treatment, up to 12 months.
Secondary outcome [2] 0 0
Treatment-related changes in urinary p75 levels
Timepoint [2] 0 0
From Baseline to End of Treatment, up to 12 months.
Secondary outcome [3] 0 0
Treatment-related changes on the ALS Functional Rating Scale (ALSFRS)
Timepoint [3] 0 0
From Baseline to End of Treatment, up to 12 months.
Secondary outcome [4] 0 0
Treatment-related changes on the Edinburgh Cognitive and Behavioural Amyotrophic Lateral Sclerosis Screen (ECAS)
Timepoint [4] 0 0
From Baseline to End of Treatment, up to 12 months.
Secondary outcome [5] 0 0
Treatment-related changes in slow vital capacity (in participants with the capacity to undergo testing)
Timepoint [5] 0 0
From Baseline to End of Treatment, up to 12 months.
Secondary outcome [6] 0 0
Treatment-related changes in Quality of Life (QOL) Assessment: ALSSQOL-R
Timepoint [6] 0 0
From Baseline to End of Treatment, up to 12 months.
Secondary outcome [7] 0 0
Treatment-related changes in central spinal fluid (CSF) neurofilament light (NfL) chain levels
Timepoint [7] 0 0
From Baseline to End of Treatment, up to 12 months.

Eligibility
Key inclusion criteria
1. Signed informed consent obtained prior to initiation of any study specific procedures and treatment.
2. Individuals who are willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures.
3. Able to swallow study drug tablets.
4. Individuals must have completed Study MON-2021-001 and, in the opinion of the investigator, have been compliant with the study procedures and study treatment.
5. Diagnosis of ALS/MND defined as clinically possible, probable, or definite according to Awaji-shima Consensus Recommendations.
6. Not taking riluzole or on a stable dose of riluzole for at least 4 weeks prior to the screening visit; subjects are not allowed to start taking riluzole during the study.
7. Individual has a competent caregiver/support person who can and will be able to support the individual's participation in the study, including assisting with the administration of study drug.
8. Adequate bone marrow reserve, renal and liver function:

* absolute neutrophil count = 1500/µl.
* platelet count = 120,000/µl.
* hemoglobin = 11 g/dL.
* creatinine clearance = 60 mL/min (Cockroft & Gault formula).
* alanine aminotransferase and/or aspartate aminotransferase = 3 x upper limit of normal.
* total bilirubin = 2.0 x ULN.
* serum albumin = 2.8 g/dL.
9. Women and men with partners of childbearing potential must use effective contraception while on study treatment and women of childbearing potential must be non-lactating.
Minimum age
18 Years
Maximum age
75 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Inability to swallow oral medications or presence of a gastrointestinal disorder (e.g., malabsorption) deemed to jeopardize intestinal absorption of study drug.
2. Participated in another investigational drug research study within 4 weeks (28 days) of the Baseline Visit or five half-lives of the drug, whichever is longer.
3. Any other significant illness or condition that in the opinion of the study investigator would interfere with the study conduct.
4. Dementia that may affect either outcome measures or subject understanding and/or compliance with study requirements and procedures.
5. Women and men of childbearing potential not using effective contraception while on study treatment.
6. Women who are breast feeding.
7. Individuals at risk of or are known to carry a SOD1 mutation or VCP mutation.

Study design
Purpose of the study
Treatment
Allocation to intervention
NA
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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
13/02/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/04/2025
Actual
Sample size
Target
12
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Macquarie University - Sydney
Recruitment hospital [2] 0 0
Calvary Health Care Bethlehem - Melbourne
Recruitment postcode(s) [1] 0 0
2109 - Sydney
Recruitment postcode(s) [2] 0 0
3195 - Melbourne

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
PharmAust Ltd
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
Calvary Health Care Bethlehem
Address [1] 0 0
Country [1] 0 0
Other collaborator category [2] 0 0
Other
Name [2] 0 0
Macquarie University, Australia
Address [2] 0 0
Country [2] 0 0

Ethics approval
Ethics application status

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

Contacts
Principal investigator
Name 0 0
Susan Mathers, BSc, MB ChB, MRCP(UK), FRACP
Address 0 0
Calvary Health Care Bethlehem
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Michael Thurn
Address 0 0
Country 0 0
Phone 0 0
+61 8 9202 6814
Fax 0 0
Email 0 0
[email protected]
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?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT06177431