Topic / Pathology
- ACT4ALS-MND
- Phase 2a
- ALS
Objectives
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
Find out more: https://clinicaltrials.gov/study/NCT06441682
Sponsor
ARGENX
Investigator
French coordinator: Prof Philippe Corcia
Critères d'inclusion
- The participant is at least 18 years old and ≤80 years old.
- The participant is diagnosed with familial or sporadic ALS according to Gold Coast criteria.
- The participant has a TRICALS (Treatment Research Initiative to Cure ALS) risk profile of ≥ -6.0 to < -2.0.
- Slow vital capacity (LVC) ≥ 60% of predicted value according to the Global Lung Function Initiative 2012.
Critères de non-inclusion
- 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
Status
Completed
