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

NCT04948645 | Terminated Phase 1 DMC FDA Drug

• 1 other identifier: M20-405
• Study Type: interventional
• Target: 31
• Locations: 2 countries
• Sites: 14

Brief Summary

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.

Conditions

ALS; Amyotrophic Lateral Sclerosis

Keywords

Fosigotifator; ABBV-CLS-7262

Outcome Measures

Primary Outcomes (3)

• 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 Outcomes (2)

• CSF Pharmacokinetics

  Baseline Up to Approximately Day 28

• Safety and Tolerability

  Baseline Up to Approximately Week 156

Study Arms (4)

Fosigotifator Low Dose

ACTIVE COMPARATOR

Drug: Fosigotifator

Fosigotifator Medium Dose

ACTIVE COMPARATOR

Drug: Fosigotifator

Fosigotifator High Dose

ACTIVE COMPARATOR

Drug: Fosigotifator

Placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

Fosigotifator DRUG

Oral

Also known as: ABBV-CLS-7262

Fosigotifator High Dose; Fosigotifator Low Dose; Fosigotifator Medium Dose

Placebo DRUG

Oral

Placebo

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• AbbVie: lead
• Calico Life Sciences LLC: collaborator

Study Sites (14)

UC Irvine Health ALS and Neuromuscular Center

Irvine, California, 92868, United States

Location

Cedars-Sinai Medical Center

Los Angeles, California, 90048, United States

Location

Forbes Norris MDA/ALS Research and Treatment Center

San Francisco, California, 94109, United States

Location

Mayo Clinic

Jacksonville, Florida, 32224, United States

Location

Johns Hopkins ALS Clinical Trials Unit

Baltimore, Maryland, 21205, United States

Location

Healey & AMG Center for ALS Research

Boston, Massachusetts, 02114, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

University of Calgary - Heritage Medical Research Clinic

Calgary, Alberta, T2V1P9, Canada

Location

University of Alberta

Edmonton, Alberta, T6G 2G3, Canada

Location

Stan Cassidy Centre for Rehabilitation

Fredericton, New Brunswick, E38 0C7, Canada

Location

London Health Sciences Centre

London, Ontario, N6A585, Canada

Location

Sunnybrook Research Institute

Toronto, Ontario, M4N 3M5, Canada

Location

Centre Hospitalier de l'Universite de Montreal (CHUM)

Montreal, Quebec, H2L 4M1, Canada

Location

Montreal Neurological Institute and Hospital

Montreal, Quebec, H3A 2B4, Canada

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 23, 2021
• First Posted: July 2, 2021
• Study Start: September 22, 2021
• Primary Completion: July 15, 2025
• Study Completion: July 15, 2025
• Last Updated: August 1, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

CA (7); US (7)