A Safety and Efficacy Study of ARGX-119 in Adult Patients with Amyotrophic Lateral Sclerosis (ALS)

Phase 2

Recruiting

• Conditions: Amyotrophic Lateral Sclerosis
• Interventions: Biological: ARGX-119; Other: Placebo

• Registration Number: NCT06441682
• Lead Sponsor: argenx

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-05-29
• Study First Submitted QC: 2024-05-29
• Study First Posted: 2024-06-04
• Study Start: 2024-10-23
• Status Verified: 2025-03
• Last Update Submitted: 2025-03-18
• Last Update Posted: 2025-03-19
• Primary Completion: 2027-07
• Study Completion: 2027-07

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 60

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
ARGX-119 - Dose 3	ARGX-119	Participants will receive third dosage level of ARGX-119 intravenously during the double blinded treatment period followed by ARGX-119 in active treatment extension period
Placebo	ARGX-119	Participants will receive placebo intravenously during the double-blinded treatment period followed by ARGX-119 in active treatment extension period
Placebo	Placebo	Participants will receive placebo intravenously during the double-blinded treatment period followed by ARGX-119 in active treatment extension period
ARGX-119 - Dose 1	ARGX-119	Participants will receive first dosage level of ARGX-119 intravenously during the double blinded treatment period followed by ARGX-119 in active treatment extension period
ARGX-119 - Dose 2	ARGX-119	Participants will receive second dosage level of ARGX-119 intravenously during the double blinded treatment period followed by ARGX-119 in active treatment extension period

Primary Outcome Measures

Name	Time	Method
Assessment of adverse events (AEs)	Up to week 96

Secondary Outcome Measures

Name	Time	Method
Incidence of anti-drug antibodies (ADA) against ARGX-119 in serum over time	Up to week 96
Rate of change from baseline in electrophysiological muscle scan (MScan)-derived motor unit number (MUN)	Up to week 24
Prevalence of anti-drug antibodies (ADA) against ARGX-119 in serum over time	Up to week 96
Maximum observed serum concentration (Cmax) of ARGX-119	Up to week 96

Trial Locations

Locations (9)

Hôpital La Pitié Salpêtrière; 🇫🇷 Paris, France

Akademiskt specialistcentrum Karolinska Institutet; 🇸🇪 Stockholm, Sweden

CHU Bretonneau; 🇫🇷 Tours, France

UZ Leuven; 🇧🇪 Leuven, Belgium

Kaye Edmonton Clinic; 🇨🇦 Edmonton, Canada

Montreal Neurological Institute and Hospital; 🇨🇦 Montreal, Canada

Bispebjerg University Hospital; 🇩🇰 Kopenhagen, Denmark

UMC Utrecht; 🇳🇱 Utrecht, Netherlands

Aarhus Universitets Hospital; 🇩🇰 Aarhus, Denmark