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

April 3, 2026 updated by: argenx

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

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

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

60

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Belgium
      • Leuven, Belgium, 3000
        • UZ Leuven
  • Canada
      • Edmonton, Canada, 11400
        • Kaye Edmonton Clinic
      • Montreal, Canada, H3A 2B4
        • Montreal Neurological Institute and Hospital
  • Denmark
      • Aarhus, Denmark, 8200
        • Aarhus Universitets Hospital
      • Copenhagen, Denmark, 2400
        • Bispebjerg University Hospital
  • France
      • Paris, France, 75013
        • Hôpital La Pitié Salpêtrière
      • Tours, France, 37000
        • Chu Bretonneau
  • Netherlands
      • Utrecht, Netherlands, 3584 CX
        • UMC Utrecht
  • Sweden
      • Stockholm, Sweden, 113 61
        • Akademiskt specialistcentrum Karolinska Institutet

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: ARGX-119 - Dose 1
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
Intravenous infusion of ARGX-119
Experimental: ARGX-119 - Dose 2
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
Intravenous infusion of ARGX-119
Experimental: ARGX-119 - Dose 3
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
Intravenous infusion of ARGX-119
Placebo Comparator: Placebo
Participants will receive placebo intravenously during the double-blinded treatment period followed by ARGX-119 in active treatment extension period
Other: Placebo
Intravenous infusion of placebo
Biological: ARGX-119
Intravenous infusion of ARGX-119

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Assessment of adverse events (AEs)
Time Frame: Up to week 96
Up to week 96

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

argenx

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

October 23, 2024

Primary Completion (Estimated)

August 1, 2026

Study Completion (Estimated)

August 1, 2026

Study Registration Dates

First Submitted

May 29, 2024

First Submitted That Met QC Criteria

May 29, 2024

First Posted (Actual)

June 4, 2024

Study Record Updates

Last Update Posted (Actual)

April 8, 2026

Last Update Submitted That Met QC Criteria

April 3, 2026

Last Verified

April 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ARGX-119-2303
  • 2024-511318-19-00 (Ctis)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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