for people ages 18 years and up (full criteria)
at San Francisco, California and other locations
study started
estimated completion



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

Official Title

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


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.


Amyotrophic Lateral Sclerosis Motor Neuron Disease Sclerosis


You can join if…

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

You CAN'T join if...

  • 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)


  • University of California, San Francisco not yet accepting patients
    San Francisco California 94117 United States
  • Barrow Neurological Instiute not yet accepting patients
    Phoenix Arizona 85013 United States


accepting new patients
Start Date
Completion Date
Alector Inc.
Phase 2
Study Type
Last Updated