A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AL001 in C9orf72-Associated Amyotrophic Lateral Sclerosis

Minimum Age:

18 Years

Maximum Age:

N/A

Can participants use Riluzole?

Yes

Inclusion Criteria:

- 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

Exclusion Criteria:

- 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)