A Phase 2 Study to Evaluate AL001 in C9orf72-Associated ALS

Open to people ages 18 years and up

• Confirmation of C9orf72 mutation
• Diagnosis of ALS by revised El Escorial criteria
• Time since onset of muscle weakness due to ALS ≤36 months at the time of the Screening Visit
• Slow Vital Capacity (VC) ≥50% of predicted capacity at the time of the Screening Visit
• If taking riluzole, must be on a stable dose of riluzole for at least 30 days prior to the Screening Visit. Riluzole naive participants are allowed.
• If taking edaravone, must have completed at least one cycle of edaravone prior to the Screening Visit and plan to continue edaravone during the study. Edaravone naive participants are allowed.
• Females must not be pregnant, breastfeeding or planning to conceive within the study period. Males must agree to use acceptable contraception
• Capable of providing informed consent at the Screening visit and complying with study procedures throughout the study

• Clinically significant, unstable, medical condition (other than ALS)
• Clinically significant heart disease, liver disease or kidney disease
• Cognitive impairment or dementia
• Current uncontrolled hypertension
• History of unresolved cancer
• Any experimental gene therapy
• Any experimental vaccine (any vaccine against COVID-19 either approved or administered under an Emergency Use Authorization is allowed)