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

NCT06441682 | Active Not Recruiting Phase 2 DMC

• 2 other identifiers: ARGX-119-23032024-511318-19-00
• Study Type: interventional
• Target: 60
• Locations: 6 countries
• Sites: 9

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. The study was terminated early following review of interim data indicating that continuation was unlikely to demonstrate a clinically meaningful treatment effect. The decision was made to minimize unnecessary participant burden. This decision is not related to safety concerns. End-of-study and Safety-Follow-Up visits are ongoing for the participants of this trial

Conditions

Amyotrophic Lateral Sclerosis

Outcome Measures

Primary Outcomes (1)

• Assessment of adverse events (AEs)

  Up to week 96

Secondary Outcomes (4)

• Rate of change from baseline in electrophysiological muscle scan (MScan)-derived motor unit number (MUN)

  Up to week 24

• Maximum observed serum concentration (Cmax) of ARGX-119

  Up to week 96

• Incidence of anti-drug antibodies (ADA) against ARGX-119 in serum over time

  Up to week 96

• Prevalence of anti-drug antibodies (ADA) against ARGX-119 in serum over time

  Up to week 96

Study Arms (4)

ARGX-119 - Dose 1

EXPERIMENTAL

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

Biological: ARGX-119

ARGX-119 - Dose 2

EXPERIMENTAL

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

Biological: ARGX-119

ARGX-119 - Dose 3

EXPERIMENTAL

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

Biological: ARGX-119

Placebo

PLACEBO COMPARATOR

Participants will receive placebo intravenously during the double-blinded treatment period followed by ARGX-119 in active treatment extension period

Biological: ARGX-119; Other: Placebo

Interventions

ARGX-119 BIOLOGICAL

Intravenous infusion of ARGX-119

ARGX-119 - Dose 1; ARGX-119 - Dose 2; ARGX-119 - Dose 3; Placebo

Placebo OTHER

Intravenous infusion of placebo

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:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• argenx: lead

Study Sites (9)

UZ Leuven

Leuven, 3000, Belgium

Location

Kaye Edmonton Clinic

Edmonton, 11400, Canada

Location

Montreal Neurological Institute and Hospital

Montreal, H3A 2B4, Canada

Location

Aarhus Universitets Hospital

Aarhus, 8200, Denmark

Location

Bispebjerg University Hospital

Copenhagen, 2400, Denmark

Location

Hôpital La Pitié Salpêtrière

Paris, 75013, France

Location

CHU Bretonneau

Tours, 37000, France

Location

UMC Utrecht

Utrecht, 3584 CX, Netherlands

Location

Akademiskt specialistcentrum Karolinska Institutet

Stockholm, 113 61, Sweden

Location

Related Publications (1)

• Moss KR, Darvishi FB, Badawi Y, Fish LA, Funke JR, Pedersen TH, Robitaille R, Arnold WD, Burgess RW, Meriney SD, Nishimune H, Saxena S. The Neuromuscular Junction: A Shared Vulnerability in Aging and Disease. J Neurosci. 2025 Nov 12;45(46):e1353252025. doi: 10.1523/JNEUROSCI.1353-25.2025.

  PMID: 41224659 DERIVED

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 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 29, 2024
• First Posted: June 4, 2024
• Study Start: October 23, 2024
• Primary Completion (Estimated): August 1, 2026
• Study Completion (Estimated): August 1, 2026
• Last Updated: April 8, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

CA (2); BE (1); FR (2); NL (1); SE (1); DK (2)