Clinical trial NCT04948645: A Phase 1 Study to Investigate the Safety and Pharmacokinetics of Fosigotifator in Patients With Amyotrophic Lateral Sclerosis
Official title: 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
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE1
Conditions: ALS
Amyotrophic Lateral Sclerosis
Keywords: Fosigotifator
ABBV-CLS-7262
Lead sponsor: AbbVie
Sponsor class: INDUSTRY
Collaborators: name: Calico Life Sciences LLC
First posted: 2021-07-02
Enrollment: 31
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Safety and Tolerability
timeFrame: Baseline Up to Approximately Day 28
measure: Pharmacokinetics
timeFrame: Baseline Up to Approximately Day 28
measure: Pharmacokinetics
timeFrame: Baseline Up to Approximately Day 28
Secondary outcomes:
measure: CSF Pharmacokinetics
timeFrame: Baseline Up to Approximately Day 28
measure: Safety and Tolerability
timeFrame: Baseline Up to Approximately Week 156
Other outcomes:
Locations:
city: Irvine
country: United States
facility: UC Irvine Health ALS and Neuromuscular Center
state: California
city: Los Angeles
country: United States
facility: Cedars-Sinai Medical Center
state: California
city: San Francisco
country: United States
facility: Forbes Norris MDA/ALS Research and Treatment Center
state: California
city: Jacksonville
country: United States
facility: Mayo Clinic
state: Florida
city: Baltimore
country: United States
facility: Johns Hopkins ALS Clinical Trials Unit
state: Maryland
city: Boston
country: United States
facility: Healey & AMG Center for ALS Research
state: Massachusetts
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
city: Calgary
country: Canada
facility: University of Calgary - Heritage Medical Research Clinic
state: Alberta
city: Edmonton
country: Canada
facility: University of Alberta
state: Alberta
city: Fredericton
country: Canada
facility: Stan Cassidy Centre for Rehabilitation
state: New Brunswick
city: London
country: Canada
facility: London Health Sciences Centre
state: Ontario
city: Toronto
country: Canada
facility: Sunnybrook Research Institute
state: Ontario
city: Montreal
country: Canada
facility: Centre Hospitalier de l'Universite de Montreal (CHUM)
state: Quebec
city: Montreal
country: Canada
facility: Montreal Neurological Institute and Hospital
state: Quebec
Study officials:
References: