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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
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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
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Secondary ID [1]
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MON-2023-001
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Motor Neuron Disease
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Condition category
Condition code
Neurological
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Neurodegenerative diseases
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Intervention/exposure
Study type
Interventional
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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
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Long-term safety and tolerability
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Assessment method [1]
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Assess long-term safety and tolerability of 10 mg/kg body weight (BW) once daily (QD) of monepantel in individuals with Motor Neurone Disease (MND)
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Timepoint [1]
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From Baseline to End of Treatment, up to 12 months.
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Secondary outcome [1]
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Treatment-related changes in serum neurofilament light (NfL) chain levels
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Assessment method [1]
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Serum NfL chain levels correlate with disease progression, so stable NfL levels would correlate with stable disease (pg/ml)
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Timepoint [1]
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From Baseline to End of Treatment, up to 12 months.
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Secondary outcome [2]
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Treatment-related changes in urinary p75 levels
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Assessment method [2]
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Urinary p75 level reflect nerve damage and therefore increased levels would act as a proxy to disease progression (ng/mg creatinine)
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Timepoint [2]
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From Baseline to End of Treatment, up to 12 months.
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Secondary outcome [3]
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Treatment-related changes on the ALS Functional Rating Scale (ALSFRS)
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Assessment method [3]
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The ALS Functional Rating Scale (ALSFRS) is a validated rating instrument for monitoring the progression of disability in patients with amyotrophic lateral sclerosis (ALS). Measurements include: (1) speech (2) salivation (3) swallowing (4) handwriting (5) cutting food and handling utensils (with or without gastrostomy) (6) dressing and hygiene (7) turning in bed and adjusting bed clothes (8) walking (9) climbing stairs and (10) breathing. Possible scores range from 0 (normal function) to 4 (severe loss of function). Change = (Week 4 score - Baseline score)
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Timepoint [3]
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From Baseline to End of Treatment, up to 12 months.
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Secondary outcome [4]
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Treatment-related changes on the Edinburgh Cognitive and Behavioural Amyotrophic Lateral Sclerosis Screen (ECAS)
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Assessment method [4]
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The ECAS-cognitive screen is a validated screen comprises 16 items organized into two sub-scales. An ALS-specific sub-scale taps into the cognitive domains of language, verbal fluency, and executive and social functions. A non-ALS-specific sub-scale specifically assesses memory and visuospatial function. The sub-scales of the ECAS-cognitive screen range, respectively, from 0 to 100 and from 0 to 36. Low scores indicate a greater deficit. Change = (Week 4 score - Baseline score)
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Timepoint [4]
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From Baseline to End of Treatment, up to 12 months.
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Secondary outcome [5]
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Treatment-related changes in slow vital capacity (in participants with the capacity to undergo testing)
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Assessment method [5]
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A decline in SVC would indicate a decline in respiratory function and is an important indicator of any clinical progression (L/s)
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Timepoint [5]
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From Baseline to End of Treatment, up to 12 months.
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Secondary outcome [6]
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Treatment-related changes in Quality of Life (QOL) Assessment: ALSSQOL-R
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Assessment method [6]
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ALSSQOL-R is a 50-item disease-specific questionnaire. Each item is rated on a 10-point Likert scale, with 0 being the least desirable situation and 10 being the most desirable. The instrument produces a single-item QoL score and six domain scores (negative emotion, interaction with people and environment, intimacy, religiosity, physical symptoms, and bulbar function). The ALSSQOL-R is used as QoL outcomes measure to examine the effects of experimental treatments on individual's QoL.
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Timepoint [6]
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From Baseline to End of Treatment, up to 12 months.
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Secondary outcome [7]
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Treatment-related changes in central spinal fluid (CSF) neurofilament light (NfL) chain levels
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Assessment method [7]
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CSF NfL chain levels correlate with disease progression. Levels in individuals living with MND are 5 to 10-fold higher than those of healthy individuals (pg/ml)
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Timepoint [7]
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From Baseline to End of Treatment, up to 12 months.
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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.
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Minimum age
18
Years
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Maximum age
75
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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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.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
NA
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 1
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
13/02/2024
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
1/04/2025
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Actual
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Sample size
Target
12
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
NSW,VIC
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Recruitment hospital [1]
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Macquarie University - Sydney
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Recruitment hospital [2]
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Calvary Health Care Bethlehem - Melbourne
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Recruitment postcode(s) [1]
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2109 - Sydney
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Recruitment postcode(s) [2]
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3195 - Melbourne
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
PharmAust Ltd
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Address
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Country
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Other collaborator category [1]
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Other
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Name [1]
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Calvary Health Care Bethlehem
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Address [1]
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Country [1]
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Other collaborator category [2]
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Other
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Name [2]
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Macquarie University, Australia
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Address [2]
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Country [2]
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Ethics approval
Ethics application status
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Summary
Brief summary
This study is a multicenter, 12-month open label extension study, following Phase 1 Study MON-2021-001, with a single dose of monepantel (MPL) once daily (QD) for the treatment of individuals with MND.
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Trial website
https://clinicaltrials.gov/study/NCT06177431
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Susan Mathers, BSc, MB ChB, MRCP(UK), FRACP
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Address
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Calvary Health Care Bethlehem
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Michael Thurn
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Address
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Country
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Phone
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+61 8 9202 6814
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Fax
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Email
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[email protected]
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Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
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No/undecided IPD sharing reason/comment
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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