Evaluating ARGX-119 for treating ALS in adults
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 is testing a new treatment called ARGX-119 to see if it can help adults with ALS improve their muscle function over about two years.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 60 (estimated) |
| Ages | 18 Years to 80 Years |
| Sex | All |
| Sponsor | argenx Industry-sponsored |
| Locations | 9 sites (Leuven and 8 other locations) |
| Trial ID | NCT06441682 on ClinicalTrials.gov |
What this trial studies
This study evaluates the safety and efficacy of ARGX-119 in adult patients diagnosed with Amyotrophic Lateral Sclerosis (ALS). Participants will be assigned to receive one of three doses of ARGX-119 or a placebo during a treatment period, followed by an extension period where all will receive ARGX-119. The study aims to assess the impact of the treatment on ALS disease outcomes, particularly muscle function, over a duration of approximately 100 weeks.
Who should consider this trial
Good fit: Ideal candidates are adults aged 18 to 80 with a confirmed diagnosis of familial or sporadic ALS and specific respiratory function criteria.
Not a fit: Patients requiring extensive ventilatory support or those with prior gene or cell therapy exposure may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could improve muscle function and overall outcomes for patients with ALS.
How similar studies have performed: While there have been studies on ALS treatments, the specific approach with ARGX-119 is novel and has not been extensively tested.
Eligibility criteria
Show full inclusion / exclusion 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
Where this trial is running
Leuven and 8 other locations
- UZ Leuven — Leuven, Belgium (Recruiting)
- Kaye Edmonton Clinic — Edmonton, Canada (Recruiting)
- Montreal Neurological Institute and Hospital — Montreal, Canada (Recruiting)
- Aarhus Universitets Hospital — Aarhus, Denmark (Recruiting)
- Bispebjerg University Hospital — Kopenhagen, Denmark (Recruiting)
- Hôpital La Pitié Salpêtrière — Paris, France (Recruiting)
- CHU Bretonneau — Tours, France (Recruiting)
- UMC Utrecht — Utrecht, Netherlands (Recruiting)
- Akademiskt specialistcentrum Karolinska Institutet — Stockholm, Sweden (Recruiting)
Study contacts
- Study coordinator: Sabine Coppieters, MD
- Email: clinicaltrials@argenx.com
- Phone: 857-350-4834
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.