A Randomized, Double-Blind, Placebo-Controlled Study to Assess Safety, Tolerability, and Pharmacokinetics Following Multiple Doses of ABBV-CLS-7262 in Subjects With Amyotrophic Lateral Sclerosis Followed by an Active Treatment Extension

Study Purpose:

ABBV-CLS-7262 is an investigational drug being researched for the treatment of Amyotrophic Lateral Sclerosis. This is a 48-week, 2-part study. Part 1 will be a 4-week, randomized, double-blind, placebo-controlled study; Part 2 will be a 44-week active treatment extension (ATE) during which all subjects will receive ABBV-CLS-7262.

Subjects will attend regular visits during the course of the study and complete medical assessments, blood tests, checking for side effects, and completing questionnaires.


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

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:



Phase I

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

Clinicaltrials.gov ID (11 digit #):


Neals Affiliated?


Coordinating Center Contact Information

Calico Call Center / .(JavaScript must be enabled to view this email address) / 844-463-2423
.(JavaScript must be enabled to view this email address)

Full Study Summary:

Study Sponsor:

Calico Life Sciences LLC

Participant Duration:

48 weeks

Estimated Enrollment:


Estimated Study Start Date:


Estimated Study Completion Date:


Posting Last Modified Date:


Date Study Added to alsconsortium.org:

  • Eligibility Criteria


    Female, Male

    Minimum Age:


    Maximum Age:


    Min Vital Capacity (% predicted normal):


    Time since Symptom Onset:

    <36 months

    Time since Diagnosis:

    Can participants use Riluzole?


    Inclusion Criteria:

    • Must have an identified, reliable caregiver
    • Confirmed diagnosis of Familial ALS or Sporadic ALS
    • First ALS symptoms occurred ≤36 months before screening
    • Able to swallow solids
    • No known active COVID-19 infection at screening
    • Vital capacity ≥50% predicted value (for sex, age, ethnic origin, and height) at screening

    Exclusion Criteria:

    • History of dementia/severe cognitive problems at screening
    • Use of riluzole (Rilutek®) at a dose which has NOT been stable for >1 months prior to Baseline.
    • Use of edaravone (Radicava®) within 1 month prior to screening or intention of initiating edaravone within 3 months after receiving the first dose of ABBV-CLS-7262.
    • History of clinically significant medical conditions (other than ALS) or any other reason, including any physical, psychological, or psychiatric condition that, in the opinion of the Investigator, would compromise the safety or interfere with the subject's participation in the study, or would make the subject an unsuitable candidate to receive study drug, or would put the subject at risk by participating in the study.
    • History of abnormal screening laboratory or imaging results that, in the opinion of the Investigator, are indicative of any significant cardiac, endocrinologic, hematologic, hepatic, immunologic, infectious, metabolic, urologic, pulmonary, gastrointestinal, dermatologic, psychiatric, renal, neurologic, and/or other major disease that would preclude administration of ABBV-CLS-7262.
    • If female, is known to be pregnant, breastfeeding, considering becoming pregnant, or donating/banking eggs during the study or within 30 days or >5 half-lives (whichever is longer) after the last dose of study drug
    • If male, plans to donate sperm or father a child during the study or within 30 days after the last dose of study drug.
    • Known to have received any investigational product within 30 days or 5 half-lives of the drug (whichever is longer) prior to the first dose of study drug or is currently enrolled in another clinical study.
    • History of ABBV-CLS-7262 use prior to participation in this study
    • Recent (within 6 months prior to Screening) history of drug or alcohol abuse
    • Previous participation in a stem cell clinical study
    • Current or anticipated use of diaphragmatic pacing during the study period
    • Tracheostomy or use of non-invasive ventilatory support ≥22 hours a day

  • Site Contact Information

    UC Irvine Health ALS and Neuromuscular Center
    Irvine, California 92868
    United States

    Mayo Clinic
    Bjorn Oskarsson, MD / 904-953-3647
    Jacksonville, Florida 32224
    United States

    Massachusetts General Hospital
    Darshana Hari / .(JavaScript must be enabled to view this email address) / 617-643-3530
    Boston, Massachusetts 02114
    United States

    Mayo Clinic
    Nathan Staff, MD / 507-293-0545
    Rochester, Minnesota 55905
    United States

    University of Calgary
    Calgary, Alberta

    University of Alberta
    Edmonton, Alberta

    Horizon Health Network
    Fredericton, New Brunswick

    London Health Sciences Centre
    London, Ontario

    Sunnybrook Research Institute
    Toronto, Ontario

    Centre Hospitalier de l'Universite de Montreal (CHUM)
    Montreal, Quebec

    Montreal Neurological Institute and Hospital
    Montreal, Quebec