A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS) (NCT06436742)
A Phase 1b, Double-Blinded, Randomized, Placebo-Controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, Immunogenicity, and Efficacy of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: UC Davis Medical Center — Sacramento, California
Age range: 18 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (9 Sites)
- UC Davis Medical Center — Sacramento, California
- Ann and Robert H Lurie Childrens Hospital of Chicago — Chicago, Illinois
- Ottawa Hospital Research Institute - Civic Campus — Ottawa,
- CHU - Hospital de la Timone — Marseille,
- Group Hospitalier Pitie-Salpetriere — Paris,
- Fondazione IRCCS Istituto Neurologico Carlo Besta — Milan,
- Universitat de Valencia - Hospital Universitari i Politecnic La Fe de Valencia (Hospital La Fe Bulevar Sur) — Valencia,
- Clinical Trials Centre - South Eastern Health and Social Care Trust - The Ulster Hospital — Belfast,
- John Radcliffe Hospital - Oxford University Hospitals NHS Foundation Trust — Oxford,
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
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💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.