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

ARGX-119-2303

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.

2024-09-02

2027-05-01

2027-05-01

Not yet recruiting

Early phase I

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

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'PARALLEL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}}, 'enrollmentInfo': {'count': 60, 'type': 'ESTIMATED'}}

2024-06-04