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

• Status: Recruiting
• Trial end date: 2023-02-01
• Target enrollment: 0
• Participant gender: All

Summary

A phase 2 double-blind, placebo-controlled study of AL001 in participants with C9orf72-associated ALS.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Alector Inc.

Criteria

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)