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A Phase 1 Study to Investigate the Safety and Pharmacokinetics of ABBV-CLS-7262 in Patients With Amyotrophic Lateral Sclerosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT04948645
Recruitment Status : Recruiting
First Posted : July 2, 2021
Last Update Posted : June 22, 2022
Information provided by (Responsible Party):
Calico Life Sciences LLC

Brief Summary:

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.

Condition or disease Intervention/treatment Phase
ALS Amyotrophic Lateral Sclerosis Drug: ABBV-CLS-7262 Drug: Placebo Phase 1

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : September 22, 2021
Estimated Primary Completion Date : June 2022
Estimated Study Completion Date : May 2023

Arm Intervention/treatment
Active Comparator: ABBV-CLS-7262 LOW DOSE Drug: ABBV-CLS-7262

Active Comparator: ABBV-CLS-7262 MEDIUM DOSE Drug: ABBV-CLS-7262

Active Comparator: ABBV-CLS-7262 HIGH DOSE Drug: ABBV-CLS-7262

Placebo Comparator: PLACEBO Drug: Placebo

Primary Outcome Measures :
  1. Safety and Tolerability [ Time Frame: Baseline Up to Approximately Day 28 ]
    Number of patients with treatment-related adverse events as assessed by CTCAE v4.03

  2. Pharmacokinetics [ Time Frame: Baseline Up to Approximately Day 28 ]
    Maximum Plasma Concentration [Cmax]

  3. Pharmacokinetics [ Time Frame: Baseline Up to Approximately Day 28 ]
    Area Under the Curve [AUC]

Secondary Outcome Measures :
  1. CSF Pharmacokinetics [ Time Frame: Baseline Up to Approximately Day 28 ]
    Concentration at steady state in CSF

  2. Safety and Tolerability [ Time Frame: Baseline Up to Approximately Week 48 ]
    Number of patients with treatment-related adverse events as assessed by CTCAE v4.03

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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
  • Subjects must be a) naïve to, or b) off riluzole (Rilutek), or c) on a stable dose >30 days prior to Baseline visit to enter study
  • Subjects must be a) naïve to, or b) off edaravone (Radicava), or c) have completed 2 treatment cycles prior to the Baseline visit.

Exclusion Criteria:

  • History of dementia/severe cognitive problems at screening
  • Use of riluzole (Rilutek®) if dose has NOT been stable for > 30 days prior to Baseline visit
  • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04948645

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Contact: Calico Call Center 1-844-463-2423 CalicoALS@patientrm.com

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United States, California
UC Irvine Health ALS and Neuromuscular Center Recruiting
Irvine, California, United States, 92868
Principal Investigator: Namita Goyal         
Forbes Norris MDA/ALS Research and Treatment Center Recruiting
San Francisco, California, United States, 94109
Principal Investigator: Jonathan Katz         
United States, Florida
Mayo Clinic Recruiting
Jacksonville, Florida, United States, 32224
Principal Investigator: Bjorn Oskarsson         
United States, Massachusetts
Healey & AMG Center for ALS Research Recruiting
Boston, Massachusetts, United States, 02114
Contact: Darshana Hari    617-643-3530    DHARI@mgh.harvard.edu   
Contact: Max Higgins    617 643-2522    mphiggins@mgh.harvard.edu   
Principal Investigator: Doreen Ho         
United States, Minnesota
Mayo Clinic Recruiting
Rochester, Minnesota, United States, 55905
Principal Investigator: Nathan Staff         
Canada, Alberta
University of Calgary - Heritage Medical Research Clinic Recruiting
Calgary, Alberta, Canada, T2V1P9
Principal Investigator: Rodney Li Pi Shan         
University of Alberta Recruiting
Edmonton, Alberta, Canada, T6G 2G3
Principal Investigator: Wendy Johnston         
Canada, New Brunswick
Stan Cassidy Centre for Rehabilitation Recruiting
Fredericton, New Brunswick, Canada, E38 0C7
Principal Investigator: Colleen O'Connell         
Canada, Ontario
London Health Sciences Centre Recruiting
London, Ontario, Canada, N6A585
Principal Investigator: Christen Shoesmith         
Sunnybrook Research Institute Recruiting
Toronto, Ontario, Canada, M4N 3M5
Principal Investigator: Lorne Zinman         
Canada, Quebec
Centre Hospitalier de l'Universite de Montreal (CHUM) Recruiting
Montreal, Quebec, Canada, H2L 4M1
Principal Investigator: Genevieve Matte         
Montreal Neurological Institute and Hospital Recruiting
Montreal, Quebec, Canada, H3A 2B4
Principal Investigator: Angela Genge         
Sponsors and Collaborators
Calico Life Sciences LLC
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Responsible Party: Calico Life Sciences LLC
ClinicalTrials.gov Identifier: NCT04948645    
Other Study ID Numbers: M20-405
First Posted: July 2, 2021    Key Record Dates
Last Update Posted: June 22, 2022
Last Verified: June 2022

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
Additional relevant MeSH terms:
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Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases