A phase 2 double-blind, placebo-controlled study of AL001 in participants with C9orf72-associated ALS.
This is a phase 2 double-blind, placebo-controlled trial to test the safety, tolerability, pharmacokinetics, and pharmacodynamics of AL001 in participants with C9orf72-associated Amyotrophic Lateral Sclerosis.
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)