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

Phase 1

Active, not recruiting

• Conditions: ALS; Amyotrophic Lateral Sclerosis
• Interventions: Drug: Fosigotifator; Drug: Placebo

• Registration Number: NCT04948645
• Lead Sponsor: Calico Life Sciences LLC

Brief Summary

ABBV-CLS-7262 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 ABBV-CLS-7262.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2021-06-23
• Study First Submitted QC: 2021-06-30
• Study First Posted: 2021-07-02
• Study Start: 2021-09-22
• Primary Completion: 2023-01-12
• Status Verified: 2024-05
• Last Update Submitted: 2024-05-24
• Last Update Posted: 2024-05-28
• Study Completion: 2025-12

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 31

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
• 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.

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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 ABBV-CLS-7262.
• 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 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 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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Fosigotifator Low Dose	Fosigotifator	-
Fosigotifator Medium Dose	Fosigotifator	-
Fosigotifator High Dose	Fosigotifator	-
Placebo	Placebo	-

Primary Outcome Measures

Name	Time	Method
Safety and Tolerability	Baseline Up to Approximately Day 28	Number of patients with treatment-related adverse events as assessed by CTCAE v4.03
Pharmacokinetics	Baseline Up to Approximately Day 28	Area Under the Curve \[AUC\]

Secondary Outcome Measures

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

Trial Locations

Locations (13)

Cedars-Sinai Medical Center; 🇺🇸 Los Angeles, California, United States

Johns Hopkins ALS Clinical Trials Unit; 🇺🇸 Baltimore, Maryland, United States

Healey & AMG Center for ALS Research; 🇺🇸 Boston, Massachusetts, United States

Sunnybrook Research Institute; 🇨🇦 Toronto, Ontario, Canada

Stan Cassidy Centre for Rehabilitation; 🇨🇦 Fredericton, New Brunswick, Canada

UC Irvine Health ALS and Neuromuscular Center; 🇺🇸 Irvine, California, United States

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

University of Calgary - Heritage Medical Research Clinic; 🇨🇦 Calgary, Alberta, Canada

University of Alberta; 🇨🇦 Edmonton, Alberta, Canada

London Health Sciences Centre; 🇨🇦 London, Ontario, Canada

Montreal Neurological Institute and Hospital; 🇨🇦 Montreal, Quebec, Canada

Forbes Norris MDA/ALS Research and Treatment Center; 🇺🇸 San Francisco, California, United States

Mayo Clinic; 🇺🇸 Rochester, Minnesota, United States