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

Full Study Summary:

• Eligibility Criteria

  Gender:

  Female, Male

  Minimum Age:

  18

  Maximum Age:

  80

  Min Vital Capacity (% predicted normal):

  50

  Time since Symptom Onset:

  <36 months

  Time since Diagnosis:

  Can participants use Riluzole?

  Yes

  
  

  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
  Canada

  University of Alberta
  Edmonton, Alberta
  Canada

  Horizon Health Network
  Fredericton, New Brunswick
  Canada

  London Health Sciences Centre
  London, Ontario
  Canada

  Sunnybrook Research Institute
  Toronto, Ontario
  Canada

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

  Montreal Neurological Institute and Hospital
  Montreal, Quebec
  Canada