Actively Recruiting
A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS)
Led by argenx · Updated on 2026-04-23
16
Participants Needed
9
Research Sites
173 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- At least 18 years of age.
- Genetically confirmed congenital myasthenic syndromes due to DOK7 mutation.
- If taking oral beta agonists (e.g., albuterol, salbutamol, ephedrine), must have used them for more than 3 months and agree to keep the same stable dose until study end.
You will not qualify if you...
- Diagnosis of CMS caused by gene mutations other than DOK7.
- Any medical condition that could affect accurate CMS assessment, study results, or participant safety, as judged by the investigator.
- History of cancer unless cured with no recurrence for over 5 years, except for treated basal cell or squamous cell skin cancer, cervical carcinoma in situ, breast carcinoma in situ, or incidental prostate cancer findings.
- Pregnant, breastfeeding, or planning pregnancy during the study.
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 9 locations
1
UC Davis Medical Center
Sacramento, California, United States, 95817
Active, Not Recruiting
2
Ann and Robert H Lurie Childrens Hospital of Chicago
Chicago, Illinois, United States, 60611
Actively Recruiting
3
Ottawa Hospital Research Institute - Civic Campus
Ottawa, Canada, K1Y 4E9
Actively Recruiting
4
CHU - Hospital de la Timone
Marseille, France, 13385
Completed
5
Group Hospitalier Pitie-Salpetriere
Paris, France, 75013
Actively Recruiting
6
Fondazione IRCCS Istituto Neurologico Carlo Besta
Milan, Italy, 20133
Actively Recruiting
7
Universitat de Valencia - Hospital Universitari i Politecnic La Fe de Valencia (Hospital La Fe Bulevar Sur)
Valencia, Spain, 46026
Active, Not Recruiting
8
Clinical Trials Centre - South Eastern Health and Social Care Trust - The Ulster Hospital
Belfast, United Kingdom, BT16 1RH
Actively Recruiting
9
John Radcliffe Hospital - Oxford University Hospitals NHS Foundation Trust
Oxford, United Kingdom, OX3 9DU
Active, Not Recruiting
Research Team
S
Sabine Coppieters, MD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
3
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