Actively Recruiting
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
Led by argenx · Updated on 2026-04-23
16
Participants Needed
9
Research Sites
21 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and tolerability of ARGX-119 in adults with DOK7-Congenital Myasthenic Syndromes, a rare genetic condition affecting muscle function. The study also looks at how the body processes ARGX-119, the immune response to the treatment, and its potential impact on patients' symptoms and daily functioning. This Phase 1b trial is sponsored by argenx and aims to gather important early data about ARGX-119. Participants will be randomly assigned in a 4:1 ratio to receive either ARGX-119 or a placebo through intravenous infusions during a double-blinded treatment period. After this phase, they will enter a follow-up period. Those who complete follow-up may join an active-treatment period where they receive open-label ARGX-119. The entire study lasts about 38 months. Throughout the study, participants will undergo regular assessments including monitoring for adverse events, measuring changes in walking distance using the 6-minute walk test, and evaluations of muscle strength and daily living activities. Blood samples will be collected to study drug levels and immune reactions. Researchers will track various health scales and questionnaires to understand how the treatment affects fatigue, lung function, and quality of life over time.
CONDITIONS
Brief 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 mutation of downstream of kinase 7 (DOK7-CMS).
- If taking oral beta agonists (such as albuterol, salbutamol, ephedrine), must have been on the same stable dose for more than 3 months and agree to continue this regimen until the end of the study.
You will not qualify if you...
- Diagnosis of CMS due to mutation of any gene other than DOK7.
- Known medical condition that interferes with accurate CMS assessment, confounds study results, or poses undue risk as judged by the investigator.
- History of malignancy or cancer unless cured with no recurrence for over 5 years, except treated basal cell or squamous cell skin cancer, carcinoma in situ of cervix or breast, or incidental prostate cancer findings.
- Pregnant, lactating, or intending to become pregnant during the study.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Up to 42 weeks for double-blinded treatment period plus additional active-treatment period
Participants receive intravenous infusions of ARGX-119 or placebo during the double-blinded treatment period, followed by an active-treatment period with ARGX-119.
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
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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