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.

Study Status:

Not recruiting


Amyotrophic Lateral Sclerosis

Study Type:


Type of Intervention:


Intervention Name:

AL001, Placebo




Phase 2

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

Sabrina Paganoni, MD, Massachusetts General Hospital

Clinicaltrials.gov ID:


Neals Affiliated?


Coordinating Center Contact Information

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.

Estimated Enrollment:


Estimated Study Start Date:

09 / 02 / 2021

Estimated Study Completion Date:

02 / 01 / 2023

Posting Last Modified Date:

04 / 15 / 2022

Date Study Added to neals.org:

09 / 22 / 2021

Minimum Age:

18 Years

Maximum Age:


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)

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

Mayo Clinic Florida

Jacksonville, Florida 32224
United States

University of South Florida

Tampa, Florida 33612
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