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

Study Purpose:

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


Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Not enrolling


Phase II

Study Chair(s)/Principal Investigator(s):

Katharine Nicholson, MD Massachusetts General Hospital

Clinicaltrials.gov ID (11 digit #):


Neals Affiliated?


Coordinating Center Contact Information

Study Lead / .(JavaScript must be enabled to view this email address)
.(JavaScript must be enabled to view this email address) United States

Full Study Summary:

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.

Study Sponsor:

Alector Inc.

Participant Duration:

Estimated Enrollment:


Estimated Study Start Date:


Estimated Study Completion Date:


Posting Last Modified Date:


Date Study Added to alsconsortium.org:

  • Eligibility Criteria



    Minimum Age:


    Maximum Age:


    Min Vital Capacity (% predicted normal):


    Time since Symptom Onset:

    <36 months

    Time since Diagnosis:

    Can participants use Riluzole?


    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)

  • Site Contact Information

    Barrow Neurological Instiute
    Phoenix, Arizona 85013
    United States

    University of California, San Francisco
    San Francisco, California 94117
    United States

    University of Colorado
    Aurora, Colorado 80045
    United States

    University of South Florida
    Tampa, Florida 33612
    United States

    Mayo Clinic Florida
    acksonville, Florida 32224
    United States

    Indiana University
    Indianapolis, Indiana 46202
    United States

    Johns Hopkins University
    Baltimore, Maryland 21205
    United States

    Massachusetts General Hospital
    Boston, Massachusetts 02114
    United States

    University of Michigan
    Ann Arbor, Michigan 48109
    United States

    Washington University School of Medicine
    Saint Louis, Missouri 63110
    United States

    Jefferson University
    Philadelphia, Pennsylvania 19107
    United States

    University of Washington
    Seattle, Washington 98195
    United States