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Clinical Trials in France / NCT06436742
Active, not recruiting Phase 1

A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS)

NCT06436742 · tracked via the Priya Life Science France tracker
Sponsor
argenx
Phase
Phase 1
Started
2024-09-24
Last updated
2026-07-24

Condition(s) studied

Congenital Myasthenic SyndromeCMS

Investigational drug(s) / intervention(s)

ARGX-119 →Placebo

ARGX-119: Intravenous infusion of ARGX-119

Placebo: Intravenous infusion of placebo

Study summary

The purpose of this study is to assess the safety and tolerability of ARGX-119 in adult participants with DOK7- Congenital Myasthenic Syndromes. The study will also assess how ARGX-119 is processed by the body (pharmacokinetics), how the immune system reacts to it (immunogenicity), and how it may improve the way patients feel and function.

After the screening period, eligible participants will be randomized in a 4:1 ratio to receive intravenous infusions of ARGX-119 or placebo during the double-blinded treatment period. Participants will then enter the follow-up period. After the follow-up period, participants may enrol in the active-treatment period, where they will receive open-label ARGX-119.

The full duration of the study is approximately 38 months.

Eligibility

Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria: * At least 18 years of age. * Has genetically confirmed congenital myasthenic syndromes due to mutation of downstream of kinase 7 (DOK7-CMS). * Participants taking oral beta agonists (eg, albuterol, salbutamol, ephedrine) must have been receiving the medication for more than 3 months and agree to remain on a same stable dosing regimen of the same medication until the end of the study. Exclusion Criteria: * Diagnosis of CMS due to mutation of any gene other than DOK7. * Known medical condition that would interfere with an accurate assessment of CMS, confound the results of the study, or put the patient at undue risk, as assessed by the investigator. * History of malignancy, cancer, unless considered cured by adequate treatment with no evidence of recurrence for more than 5 years. Adequately treated participants with the following cancers can be included at any time: Basal cell or squamous cell skin cancer, Carcinoma in situ of the cervix, Carcinoma in situ of the breast, Incidental histological findings of prostate cancer. * Pregnant or lactating state or intention to become pregnant during the study.

Primary outcome measure(s)

  • Assessment of adverse events (AEs) — Up to week 42
  • Change from active-treatment baseline over time for 6MWT distance — Up to 72 weeks
    The 6-minute walk test (6MWT) measures the distance a participant walks in 6 minutes. Before and after the 6MWT assessment, the participant's blood pressure, heart rate, and SPO2 will be recorded, and the participant's perception of fatigue and dyspnea will be measured.

Trial sites (9)

FacilityCityRegionStatus
UC Davis Medical Center Sacramento California
Ann and Robert H Lurie Childrens Hospital of Chicago Chicago Illinois
Ottawa Hospital Research Institute - Civic Campus Ottawa Canada
CHU - Hospital de la Timone Marseille France
Group Hospitalier Pitie-Salpetriere Paris France
Fondazione IRCCS Istituto Neurologico Carlo Besta Milan Italy
Universitat de Valencia - Hospital Universitari i Politecnic La Fe de Valencia (Hospital La Fe Bulevar Sur) Valencia Spain
Clinical Trials Centre - South Eastern Health and Social Care Trust - The Ulster Hospital Belfast United Kingdom
John Radcliffe Hospital - Oxford University Hospitals NHS Foundation Trust Oxford United Kingdom

More argenx trials in France

A Study to Assess the Efficacy and Safety of Empasiprubart Versus IVIg in Adults With Multifocal Motor Neuropathy Active, not recruiting
A Prospective Longitudinal Study in Adults With Multifocal Motor Neuropathy Active, not recruiting
A Study to Assess the Efficacy and Safety of Efgartigimod PH20 SC in Adults With Systemic Sclerosis Recruiting
A Study to Assess the Long-term Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod in Adults With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP, an Autoimmune Disorder That Affects the Peripheral Nerves) Active, not recruiting
Evaluating the Pharmacokinetics, Pharmacodynamics, and Safety of Efgartigimod Administered Intravenously in Children With Generalized Myasthenia Gravis Recruiting

Other trials for the same condition

A Natural History Study in Participants With Congenital Myasthenic Syndromes (CMS) Due to Mutations in DOK7, MUSK, AGRN, or LRP4 Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06436742 on ClinicalTrials.gov ↗ ← All trials in France