A Phase 1 Study to Investigate the Safety and Pharmacokinetics of Fosigotifator in Patients With Amyotrophic Lateral Sclerosis

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

Fosigotifator is an investigational drug being researched for the treatment of Amyotrophic Lateral Sclerosis. This is an up to 156-week, 2-part study. Part 1 will be a 4-week, randomized, double-blind, placebo-controlled study; Part 2 will be up to a 152-week active treatment extension (ATE) during which all subjects will receive Fosigotifator.

Study Overview

• Status: Terminated
• Conditions: Amyotrophic Lateral Sclerosis; ALS
• Intervention / Treatment: Drug: Placebo; Drug: Fosigotifator

• Study Type: Interventional
• Enrollment (Actual): 31
• Phase: Phase 1

Contacts and Locations

Study Locations

• Canada
  • Alberta
    • Calgary, Alberta, Canada, T2V1P9
      • University of Calgary - Heritage Medical Research Clinic
    • Edmonton, Alberta, Canada, T6G 2G3
      • University of Alberta
  • New Brunswick
    • Fredericton, New Brunswick, Canada, E38 0C7
      • Stan Cassidy Centre for Rehabilitation
  • Ontario
    • London, Ontario, Canada, N6A585
      • London Health Sciences Centre
    • Toronto, Ontario, Canada, M4N 3M5
      • Sunnybrook Research Institute
  • Quebec
    • Montreal, Quebec, Canada, H3A 2B4
      • Montreal Neurological Institute and Hospital
    • Montreal, Quebec, Canada, H2L 4M1
      • Centre Hospitalier de l'Universite de Montreal (CHUM)
• United States
  • California
    • Irvine, California, United States, 92868
      • UC Irvine Health ALS and Neuromuscular Center
    • Los Angeles, California, United States, 90048
      • Cedars-Sinai Medical Center
    • San Francisco, California, United States, 94109
      • Forbes Norris MDA/ALS Research and Treatment Center
  • Florida
    • Jacksonville, Florida, United States, 32224
      • Mayo Clinic
  • Maryland
    • Baltimore, Maryland, United States, 21205
      • Johns Hopkins ALS Clinical Trials Unit
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Healey & AMG Center for ALS Research
  • Minnesota
    • Rochester, Minnesota, United States, 55905
      • Mayo Clinic

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 80 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Must have an identified, reliable caregiver.
• Confirmed diagnosis of Familial Amyotrophic Lateral Sclerosis (ALS) or Sporadic ALS.
• First ALS symptoms occurred <= 36 months before screening.
• Able to swallow solids.
• No known active COVID-19 infection at screening.
• Slow vital capacity (SVC) >= 50% predicted value (for sex, age, ethnic origin, and height) at screening.
• If taking concomitant standard-of-care medications approved for the treatment of ALS (or their components), subjects must be on a stable dose of the medication(s) for >30 days prior to Baseline in order to enter the study. For edaravone, a stable dose is defined by having completed 2 treatment cycles prior to Baseline.

Exclusion Criteria:

• History of dementia/severe cognitive problems at screening.
• 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 Fosigotifator.
• Documented active or suspected malignancy or history of any malignancy within the last 5 years except for successfully treated non-melanoma skin cancer or localized carcinoma in situ of the cervix.
• 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 Fosigotifator 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 for treatment of ALS.
• Current or anticipated use of diaphragmatic pacing during the study period.
• Tracheostomy or use of non-invasive ventilatory support >= 22 hours a day.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo	Drug: Placebo; Oral
Active Comparator: Fosigotifator Low Dose	Drug: Fosigotifator; Oral; Other Names: ABBV-CLS-7262
Active Comparator: Fosigotifator Medium Dose	Drug: Fosigotifator; Oral; Other Names: ABBV-CLS-7262
Active Comparator: Fosigotifator High Dose	Drug: Fosigotifator; Oral; Other Names: ABBV-CLS-7262

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety and Tolerability	Number of patients with treatment-related adverse events as assessed by CTCAE v4.03	Baseline Up to Approximately Day 28
Pharmacokinetics	Maximum Plasma Concentration [Cmax]	Baseline Up to Approximately Day 28
Pharmacokinetics	Area Under the Curve [AUC]	Baseline Up to Approximately Day 28

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
CSF Pharmacokinetics	Concentration at steady state in CSF	Baseline Up to Approximately Day 28
Safety and Tolerability	Number of patients with treatment-related adverse events as assessed by CTCAE v4.03	Baseline Up to Approximately Week 156

Collaborators and Investigators

• Sponsor: AbbVie
• Collaborators: Calico Life Sciences LLC

Study record dates

Study Major Dates

• Study Start (Actual): September 22, 2021
• Primary Completion (Actual): July 15, 2025
• Study Completion (Actual): July 15, 2025

Study Registration Dates

• First Submitted: June 23, 2021
• First Submitted That Met QC Criteria: June 30, 2021
• First Posted (Actual): July 2, 2021

Study Record Updates

• Last Update Posted (Actual): August 1, 2025
• Last Update Submitted That Met QC Criteria: July 30, 2025
• Last Verified: July 1, 2025

More Information

Terms related to this study

• Keywords: ABBV-CLS-7262; Fosigotifator
• Additional Relevant MeSH Terms: Central Nervous System Diseases; Nervous System Diseases; Pathologic Processes; Neuromuscular Diseases; Metabolic Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Sclerosis; Motor Neuron Disease; Amyotrophic Lateral Sclerosis
• Other Study ID Numbers: M20-405

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• 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