An Open Label Extension Study of Monepantel in Individuals With Motor Neurone Disease

NCT06177431 | Completed Phase 1 FDA Drug

• 1 other identifier: MON-2023-001
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 2

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.

Conditions

Motor Neuron Disease

Outcome Measures

Primary Outcomes (1)

• Long-term safety and tolerability

  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)

  From Baseline to End of Treatment, up to 12 months.

Secondary Outcomes (7)

• Treatment-related changes in serum neurofilament light (NfL) chain levels

  From Baseline to End of Treatment, up to 12 months.

• Treatment-related changes in urinary p75 levels

  From Baseline to End of Treatment, up to 12 months.

• Treatment-related changes on the ALS Functional Rating Scale (ALSFRS)

  From Baseline to End of Treatment, up to 12 months.

• Treatment-related changes on the Edinburgh Cognitive and Behavioural Amyotrophic Lateral Sclerosis Screen (ECAS)

  From Baseline to End of Treatment, up to 12 months.

• Treatment-related changes in slow vital capacity (in participants with the capacity to undergo testing)

  From Baseline to End of Treatment, up to 12 months.

Study Arms (1)

Monepantel treatment arm

EXPERIMENTAL

Daily dose of 10 mg/kg body weight (QD)

Drug: Monepantel

Interventions

Monepantel DRUG

Monepantel is provided to individuals living with ALS/MND as a white oval tablet to be administered once a day following meals

Monepantel treatment arm

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Neurizon Therapeutics Limited: lead
• Calvary Health Care Bethlehem: collaborator
• Macquarie University, Australia: collaborator

Study Sites (2)

Macquarie University

Sydney, New South Wales, 2109, Australia

Location

Calvary Health Care Bethlehem

Melbourne, Victoria, 3195, Australia

Location

MeSH Terms

Conditions

Motor Neuron Disease

Interventions

monepantel

Condition Hierarchy (Ancestors)

Neurodegenerative Diseases; Nervous System Diseases; Neuromuscular Diseases

Study Officials

• Susan Mathers, BSc, MB ChB, MRCP(UK), FRACP

  Calvary Health Care Bethlehem

  PRINCIPAL INVESTIGATOR

• Dominic Rowe, AM, BSc(Med), MBBS(Hons), PhD

  Macquarie University, Sydney

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Open Label Extension

Study Record Dates

• First Submitted: December 11, 2023
• First Posted: December 20, 2023
• Study Start: February 13, 2024
• Primary Completion: May 30, 2025
• Study Completion: May 30, 2025
• Last Updated: June 4, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

AU (2)