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

• Status: Recruiting
• Trial end date: 2025-04-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: PharmAust Ltd
• Collaborators: Calvary Health Care Bethlehem; Macquarie University, Australia

Criteria

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.

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.