Clinical trial NCT06441682: A Safety and Efficacy Study of ARGX-119 in Adult Patients with Amyotrophic Lateral Sclerosis (ALS)
Official title: A Phase 2a, Double-Blinded, Randomized, Placebo-Controlled, and Active-Treatment Extension Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of ARGX-119 in Participants with Amyotrophic Lateral Sclerosis
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Amyotrophic Lateral Sclerosis
Lead sponsor: argenx
Sponsor class: INDUSTRY
First posted: 2024-06-04
Enrollment: 60
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: This study aims to evaluate the safety of ARGX-119 in adults with ALS. The study will also assess the impact of ARGX-119 on ALS disease outcomes, including muscle function. The study consists of 2 periods: a treatment period when participants will receive one of three ARGX-119 doses or placebo and an extension period when all participants will receive the same dose of ARGX-119. Participation in the study will last up to approximately 100 weeks.


Eligibility criteria: Inclusion Criteria:

* The participant is at least 18 and ≤80 years of age
* The participant is diagnosed with familial or sporadic ALS according to Gold Coast criteria
* The participant has a Treatment Research Initiative to Cure ALS (TRICALS) risk profile of ≥ -6.0 to \< -2.0
* Slow vital capacity (SVC) of ≥ 60% of the predicted value according to Global Lung Function Initiative 2012

Exclusion Criteria:

* Use of noninvasive ventilation more than 10 hours a day or use of a tracheostomy for ventilatory support
* Any history of or current exposure to any gene or cell therapies (off-label use or investigational) for ALS
* Pregnant or lactating state or intention to become pregnant during the study

Primary outcomes:
measure: Assessment of adverse events (AEs)
timeFrame: Up to week 96
Secondary outcomes:
measure: Rate of change from baseline in electrophysiological muscle scan (MScan)-derived motor unit number (MUN)
timeFrame: Up to week 24
measure: Maximum observed serum concentration (Cmax) of ARGX-119
timeFrame: Up to week 96
measure: Incidence of anti-drug antibodies (ADA) against ARGX-119 in serum over time
timeFrame: Up to week 96
measure: Prevalence of anti-drug antibodies (ADA) against ARGX-119 in serum over time
timeFrame: Up to week 96
Other outcomes:
Locations:
city: Leuven
country: Belgium
facility: UZ Leuven
city: Edmonton
country: Canada
facility: Kaye Edmonton Clinic
city: Montreal
country: Canada
facility: Montreal Neurological Institute and Hospital
city: Aarhus
country: Denmark
facility: Aarhus Universitets Hospital
city: Kopenhagen
country: Denmark
facility: Bispebjerg University Hospital
city: Paris
country: France
facility: Hôpital La Pitié Salpêtrière
city: Tours
country: France
facility: CHU Bretonneau
city: Utrecht
country: Netherlands
facility: UMC Utrecht
city: Stockholm
country: Sweden
facility: Akademiskt specialistcentrum Karolinska Institutet
Study officials:
References: