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

Prior treatment

• 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.

Other illness

• Inability to swallow oral medications or presence of a gastrointestinal disorder (e.g., malabsorption) deemed to jeopardize intestinal absorption of study drug.
• 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.

Other

• 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.

• 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.