Status:
COMPLETED
An Open Label Extension Study of Monepantel in Individuals With Motor Neurone Disease
Lead Sponsor:
Neurizon Therapeutics Limited
Collaborating Sponsors:
Calvary Health Care Bethlehem
Macquarie University, Australia
Conditions:
Motor Neuron Disease
Eligibility:
All Genders
18-75 years
Phase:
PHASE1
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...
Detailed Description
ALS/MND is a progressive, fatal neurodegenerative disease; characterized by motor neuron loss resulting in muscle weakness and atrophy, disability, and eventually death from failure of the ventilatory...
Eligibility Criteria
Inclusion
- Signed informed consent obtained prior to initiation of any study specific procedures and treatment.
- Individuals who are willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures.
- Able to swallow study drug tablets.
- 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.
- Diagnosis of ALS/MND defined as clinically possible, probable, or definite according to Awaji-shima Consensus Recommendations.
- 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.
- 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.
- 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.
- 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.
Exclusion
- Inability to swallow oral medications or presence of a gastrointestinal disorder (e.g., malabsorption) deemed to jeopardize intestinal absorption of study drug.
- 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.
- Any other significant illness or condition that in the opinion of the study investigator would interfere with the study conduct.
- Dementia that may affect either outcome measures or subject understanding and/or compliance with study requirements and procedures.
- Women and men of childbearing potential not using effective contraception while on study treatment.
- Women who are breast feeding.
- Individuals at risk of or are known to carry a SOD1 mutation or VCP mutation.
Key Trial Info
Start Date :
February 13 2024
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 30 2025
Estimated Enrollment :
12 Patients enrolled
Trial Details
Trial ID
NCT06177431
Start Date
February 13 2024
End Date
May 30 2025
Last Update
June 4 2025
Active Locations (2)
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1
Macquarie University
Sydney, New South Wales, Australia, 2109
2
Calvary Health Care Bethlehem
Melbourne, Victoria, Australia, 3195