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

argenx

Status and phase: Enrolling; Phase 2

Conditions: Amyotrophic Lateral Sclerosis

Treatments: Other: Placebo; Biological: ARGX-119

Study type: Interventional

Funder types: Industry

Identifiers

NCT06441682

ARGX-119-2303

2024-511318-19-00 (EU Trial (CTIS) Number)

Details and patient eligibility

About

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.

Enrollment: 60 estimated patients

Sex: All

Ages: 18 to 80 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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60 participants in 4 patient groups, including a placebo group

ARGX-119 - Dose 1

Experimental group

Description:

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

Treatment:

Biological: ARGX-119

ARGX-119 - Dose 2

Experimental group

Description:

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

Treatment:

Biological: ARGX-119

ARGX-119 - Dose 3

Experimental group

Description:

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

Treatment:

Biological: ARGX-119

Placebo

Placebo Comparator group

Description:

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

Treatment:

Biological: ARGX-119

Other: Placebo