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

Amyotrophic Lateral Sclerosis Clinical Trial

— ReALiSe  

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06441682
• Other study ID #: ARGX-119-2303
• Secondary ID: 2024-511318-19-0
• Status: Not yet recruiting
• Phase: Phase 2
• Start date: September 2, 2024
• Est. completion date: May 2027

Study information

• Verified date: May 2024
• Source: argenx
• Contact: Sabine Coppieters, MD
• Phone: 857-350-4834
• Email: clinicaltrials[@]argenx.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 60
• Est. completion date: May 2027
• Est. primary completion date: May 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 80 Years

Eligibility

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

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease
• Sclerosis

Intervention

Biological:
• ARGX-119
Intravenous infusion of ARGX-119

Other:
• Placebo
Intravenous infusion of placebo

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
argenx

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Assessment of adverse events (AEs)		Up to week 96
Secondary	Rate of change from baseline in electrophysiological muscle scan (MScan)-derived motor unit number (MUN)		Up to week 24
Secondary	Maximum observed serum concentration (Cmax) of ARGX-119		Up to week 96
Secondary	Incidence of anti-drug antibodies (ADA) against ARGX-119 in serum over time		Up to week 96
Secondary	Prevalence of anti-drug antibodies (ADA) against ARGX-119 in serum over time		Up to week 96