Congenital neuromuscular disorders Nervous System Diseases
Conditions
Interventions
After the screening period, eligible participants will be randomized through IRT in a 4:1 ratio to receive intravenous infusions of ARGX-119 or placebo during the treatment period. Participants will t
Sponsors
Argenx (Belgium)
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. At least 18 years of age 2. Has genetically confirmed congenital myasthenic syndromes due to mutation downstream of kinase 7 (DOK7-CMS). 3. 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 the same stable dosing regimen of the same medication until the end of the study
Exclusion criteria
Exclusion criteria: 1. Diagnosis of CMS due to mutation of any gene other than DOK7. 2. 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. 3. History of malignancy, or 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, and Incidental histological findings of prostate cancer 4. Different study drugs received in another clinical study within 12 weeks or 5 half-lives before screening 5. Current participation in another interventional clinical study or prior participation in any gene therapy or cell therapy study 6. Pregnant or lactating state or intention to become pregnant during the study The complete list of exclusion criteria can be found in the protocol.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Assessment of adverse events, clinical laboratory tests, electrocardiograms, and vital signs up to week 42 | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Pharmacokinetic (PK) parameters of ARGX-119 measured up to week 42 2. Incidence and prevalence of anti-drug antibodies (ADA) against ARGX-119 measured up to week 42 3. Change from baseline up to week 42 in: 3.1. Disease severity measured using the Quantitative Myasthenia Gravis (QMG) scale 3.2. Impact of myasthenia gravis on daily functioning measured using the Myasthenia Gravis Activities of Daily Living (MG-ADL) 3.3. Changes in patient quality of life measured using the Patient-Reported Outcomes Measurement Information System Global Health (PROMIS-GH) scale | — |
Countries
Austria, Canada, England, France, Italy, Spain, United Kingdom, United States of America
Contacts
Public ContactPublic Contact
Outcome results
None listed